美國
證券交易委員會
華盛頓特區20549
表格
截至本季度末
或
由_至_的過渡期
佣金文件編號
(註冊人的確切姓名載於其章程)
(註冊成立或組織的國家或其他司法管轄區) |
(國際稅務局僱主身分證號碼) |
（主要行政辦公室地址） |
(郵政編碼) |
註冊人的電話號碼，包括區號：(
用複選標記表示註冊人是否：(1)在過去12個月內(或註冊人被要求提交此類報告的較短時間內)提交了1934年《證券交易法》第13或15(D)節要求提交的所有報告，以及(2)在過去90天內一直遵守此類提交要求。
用複選標記表示註冊人是否在過去12個月內(或在註冊人被要求提交此類文件的較短時間內)以電子方式提交了根據S-T規則405規定必須提交的每一份交互數據文件。
通過複選標記來確定註冊人是大型加速申報人、加速申報人、非加速申報人、小型報告公司還是新興成長型公司。請參閱《交易法》第120億.2條規則中「大型加速申報人」、「加速申報人」、「小型報告公司」和「新興成長型公司」的定義。：
☒ |
加速文件管理器 |
☐ |
|
非加速文件服務器 |
☐ |
規模較小的報告公司 |
|
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新興成長型公司 |
如果是一家新興的成長型公司，用複選標記表示註冊人是否已選擇不使用延長的過渡期來遵守根據《交易所法》第13(A)節提供的任何新的或修訂的財務會計準則。☐
用複選標記表示註冊人是否是空殼公司(如《交易法》第12b-2條所定義)。是，☐不是
根據該法第12(B)條登記的證券：
每節課的題目： |
|
交易代碼 |
|
在其註冊的每個交易所的名稱： |
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有幾個
Axome Therapeutics，Inc.
Form 10-Q季度報告
截至2024年9月30日的季度
表格 的 C一家企業
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頁面 |
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3 |
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第1項 |
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4 |
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第2項 |
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35 |
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第3項 |
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49 |
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項目4 |
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49 |
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第1項 |
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50 |
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第1A項 |
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52 |
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第5項 |
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112 |
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項目6 |
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113 |
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簽名s |
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114 |
2
警示說明 關於 前瞻性陳述
本報告中討論的某些事項，包括「管理層對財務狀況和經營結果的討論和分析」中討論的事項，可能構成前瞻性陳述，以符合1933年修訂的「證券法」或修訂的「證券法」和1934年的「證券交易法」或「交易法」的規定，涉及已知和未知的風險、不確定因素和其他因素，可能會導致我們的實際結果、業績或成就與這些前瞻性陳述明示或暗示的未來結果、業績或成就大不相同。「預期」、「相信」、「估計」、「可能」、「預期」以及類似的表述通常用於識別前瞻性陳述。由於各種因素的影響，我們的實際結果可能與這些前瞻性聲明中的預期結果大不相同，包括但不限於本報告中「風險因素」、「管理層對財務狀況和經營結果的討論和分析」以及本報告其他部分中討論的那些因素，以及其他可能在我們提交給美國證券交易委員會或美國證券交易委員會的其他文件中，或在出現此類前瞻性聲明的文件中不時提到的其他因素。可歸因於我們的所有書面或口頭前瞻性聲明都明確地受到這些警告性聲明的限制。此類前瞻性陳述包括但不限於關於以下方面的陳述：
本報告中包含的前瞻性陳述僅反映我們截至本報告簽署之日的觀點和假設。除法律要求外，我們不承擔更新任何前瞻性陳述的責任。
我們通過這些警示性陳述來限制我們所有的前瞻性陳述。此外，對於我們的所有前瞻性陳述，我們主張1995年《私人證券訴訟改革法案》所載前瞻性陳述的安全港保護。
3
第一部分.金融公司IAL信息
項目1.金融公司ALI報表
Axsome Therapeutics，Inc
綜合資產負債表
(單位爲千，不包括每股和每股金額)
|
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9月30日， |
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12月31日， |
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（未經審計） |
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資產 |
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流動資產： |
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現金及現金等價物 |
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$ |
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$ |
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應收賬款淨額 |
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庫存，淨額 |
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預付資產和其他流動資產 |
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流動資產總額 |
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設備，網絡 |
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使用權資產-經營租賃 |
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商譽 |
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無形資產，淨額 |
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非流動庫存和其他資產 |
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總資產 |
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$ |
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$ |
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負債和股東權益 |
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流動負債： |
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應付賬款 |
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$ |
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$ |
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應計費用和其他流動負債 |
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經營租賃負債，本期部分 |
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當前或有考慮因素 |
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流動負債總額 |
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或有考慮，非流動 |
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應付貸款，長期 |
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長期經營租賃負債 |
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融資租賃負債，長期 |
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— |
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總負債 |
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股東權益： |
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優先股，$ |
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普通股，$ |
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額外實收資本 |
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累計赤字 |
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( |
) |
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( |
) |
股東權益總額 |
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總負債和股東權益 |
|
$ |
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$ |
|
||
附註是綜合財務報表的組成部分。
4
Axsome Therapeutics，Inc
合併業務報表(未經審計)
(單位爲千，不包括每股和每股金額)
|
|
截至9月30日的三個月裏， |
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截至9月30日的9個月， |
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2024 |
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2023 |
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2024 |
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2023 |
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收入： |
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產品銷售，淨額 |
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$ |
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$ |
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$ |
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$ |
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許可證收入 |
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— |
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— |
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— |
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使用費收入 |
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總收入 |
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運營費用： |
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收入成本（不包括攤銷和折舊） |
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研發 |
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銷售，一般和行政 |
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或有對價公允價值的損失（收益） |
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( |
) |
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無形資產攤銷 |
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總運營支出 |
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運營虧損 |
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( |
) |
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( |
) |
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( |
) |
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( |
) |
利息支出，淨額 |
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( |
) |
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( |
) |
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( |
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( |
) |
所得稅前虧損 |
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( |
) |
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( |
) |
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( |
) |
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( |
) |
所得稅優惠(費用) |
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— |
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— |
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( |
) |
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淨虧損 |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
每股普通股基本虧損和攤薄後淨虧損 |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
加權平均已發行普通股、基本普通股和稀釋後普通股 |
|
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||||
附註是綜合財務報表的組成部分。
5
Axsome Therapeutics，Inc
合併股東權益報表（未經審計）
(單位爲千，不包括份額)
|
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普通股 |
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額外實收 |
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積累 |
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總 |
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||||||||
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股份 |
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量 |
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資本 |
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赤字 |
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股權 |
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|||||
2022年12月31日餘額 |
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( |
) |
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股票補償 |
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— |
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— |
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— |
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行使期權後發行普通股 |
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— |
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— |
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受限制股份單位歸屬後發行普通股 |
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— |
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— |
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— |
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— |
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債務融資時發行認購證 |
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— |
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— |
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— |
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預扣稅投標股份 |
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— |
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— |
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( |
) |
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— |
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( |
) |
淨虧損 |
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— |
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— |
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— |
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|
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( |
) |
|
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( |
) |
2023年3月31日的餘額 |
|
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|
|
|
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|
|
( |
) |
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|
||||
股票補償 |
|
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— |
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— |
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— |
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||
行使期權後發行普通股 |
|
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— |
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||||
受限制股份單位歸屬後發行普通股 |
|
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— |
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— |
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— |
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— |
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融資後發行普通股 |
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— |
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債務融資時發行認購證 |
|
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— |
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— |
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— |
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預扣稅投標股份 |
|
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— |
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— |
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( |
) |
|
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— |
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( |
) |
淨虧損 |
|
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— |
|
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— |
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|
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— |
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( |
) |
|
|
( |
) |
2023年6月30日的餘額 |
|
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|
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( |
) |
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||||
股票補償 |
|
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— |
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— |
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— |
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行使期權後發行普通股 |
|
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— |
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— |
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受限制股份單位歸屬後發行普通股 |
|
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— |
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— |
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— |
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— |
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融資後發行普通股 |
|
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— |
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— |
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|||
預扣稅投標股份 |
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— |
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— |
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( |
) |
|
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— |
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( |
) |
淨虧損 |
|
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— |
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— |
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— |
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( |
) |
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( |
) |
2023年9月30日餘額 |
|
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( |
) |
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2023年12月31日餘額 |
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( |
) |
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股票補償 |
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— |
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— |
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— |
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行使期權後發行普通股 |
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— |
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— |
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受限制股份單位歸屬後發行普通股 |
|
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— |
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— |
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— |
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— |
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預扣稅投標股份 |
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— |
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— |
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( |
) |
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— |
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( |
) |
淨虧損 |
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— |
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— |
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— |
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( |
) |
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( |
) |
2024年3月31日的餘額 |
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( |
) |
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股票補償 |
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— |
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— |
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— |
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行使期權並根據員工股票購買計劃發行普通股 |
|
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— |
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— |
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受限制股份單位歸屬後發行普通股 |
|
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— |
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— |
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— |
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— |
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融資後發行普通股 |
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— |
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— |
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預扣稅投標股份 |
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— |
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— |
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( |
) |
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— |
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( |
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淨虧損 |
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— |
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— |
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— |
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( |
) |
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( |
) |
2024年6月30日的餘額 |
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( |
) |
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股票補償 |
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— |
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— |
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— |
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行使期權後發行普通股 |
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— |
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— |
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受限制股份單位歸屬後發行普通股 |
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— |
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— |
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— |
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— |
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融資後發行普通股 |
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— |
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— |
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預扣稅投標股份 |
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— |
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— |
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( |
) |
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— |
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( |
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淨虧損 |
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附註是綜合財務報表的組成部分。
6
Axsome Therapeutics，Inc
合併現金流量表(未經審計)
(In數千）
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基於股票的補償費用 |
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折舊 |
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融資租賃使用權資產攤銷 |
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經營租賃負債變化 |
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應收賬款淨額 |
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庫存，淨額 |
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預付費用和其他流動資產 |
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用於經營活動的現金淨額 |
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投資活動產生的現金流 |
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購買設備 |
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投資活動所用現金淨額 |
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融資活動現金流量 |
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債務提取收益 |
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支付債務發行成本 |
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融資租賃義務本金部分的付款 |
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行使期權並根據員工股票購買計劃發行普通股的收益 |
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支付或有對價 |
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股票獎勵的預扣稅支付 |
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融資活動提供的現金淨額 |
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現金淨(減)增 |
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支付的利息 |
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爲換取經營租賃負債而取得的經營租賃使用權資產 |
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融資租賃使用權資產換取融資租賃負債 |
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附註是綜合財務報表的組成部分。
7
Axsome Therapeutics，Inc
合併財務報表附註(未經審計)
(單位爲千，不包括每股和每股金額)
注1.業務性質和列報依據
Axome治療公司(「Axome」或「公司」)是一家引領中樞神經系統(「CNS」)疾病治療新紀元的生物製藥公司。該公司通過識別護理方面的關鍵差距來實現科學突破，並開發差異化產品，重點放在新的行動機制上，使患者結果能夠取得有意義的進步。Axome於2012年1月12日在特拉華州註冊成立。該公司的CNS投資組合包括
該公司於2022年5月從Jazz PharmPharmticals Plc(「Jazz」)手中收購了Sunosi在美國的權利，並於2022年11月從Jazz手中收購了除美國以外的全球權利(不包括某些亞洲市場)(統稱爲「收購」)。Sunosi是一種經美國食品和藥物管理局(FDA)批准並在美國銷售的產品，用於改善與發作性睡病或阻塞性睡眠呼吸暫停相關的日間過度嗜睡(EDS)成年患者的清醒程度。Sunosi於2020年1月在歐洲獲得歐盟委員會的批准。2023年2月，該公司宣佈與Atnahs Pharma UK Limited(「Pharmanovia」)達成許可交易，在歐洲和中東/北非的某些國家營銷Sunosi。
該公司於2022年8月宣佈FDA批准Auvelity，並於2022年10月宣佈Auvelity在美國商業化用於治療成年人的嚴重抑鬱障礙。
隨附的未經審核中期綜合財務報表乃由本公司根據美國公認會計原則(「美國公認會計原則」)及美國證券交易委員會(「美國證券交易委員會」)就Form 10-Q呈報的規則及規定而編制。因此，按照美國公認會計原則編制的財務報表中通常包含的某些信息和腳註披露已根據此類規則和規定予以濃縮或省略。這些未經審計的中期合併財務報表應與本公司於2024年2月23日提交給美國證券交易委員會的截至2023年12月31日的10-K表格年度報告中包含的已審計財務報表及相關附註結合閱讀。
管理層認爲，未經審計的中期綜合財務報表反映了公平列報中期財務信息所需的所有調整，這些調整是正常的經常性調整。截至2024年9月30日的三個月和九個月的經營業績不一定代表整個會計年度或未來任何時期的經營業績。
流動性與資本資源
該公司自成立以來一直出現經營虧損，預計將繼續出現經營虧損，可能永遠不會盈利。截至2024年9月30日，該公司的累計虧損爲$
該公司的主要現金來源是出售Auvelity和Sunosi的收益、在公開發行中發行和出售其普通股以及發行債務。該公司實現盈利的能力取決於許多因素，包括其獲得監管部門對其候選產品的批准、成功完成任何批准後的監管義務以及成功地將其候選產品單獨或與第三方合作商業化的能力。即使該公司繼續從其產品中獲得收入，它也可能繼續遭受巨大的運營虧損。
8
該公司相信，其現有現金將足以滿足本申請之日起至少十二個月內的預期運營現金需求。在此期間，公司預計其費用將主要由於Auvelity和Sunosi的商業化而增加，同時繼續進一步開發公司的管道資產。如果市場條件有利或出於其他戰略考慮，公司可能會使用公開和私募股權發行、債務融資、其他第三方融資、戰略聯盟、許可安排或營銷和分銷安排的組合來爲其未來的現金需求提供資金。
該公司的普通股在納斯達克全球市場上市，交易代碼爲「AXSmm」。
附註2.主要會計政策摘要
重大風險和不確定性
該公司的運營受到許多因素的影響，這些因素可能會影響其經營業績和財務狀況。這些因素包括但不限於：公司候選產品的臨床測試和試驗活動的結果；公司獲得監管機構批准銷售其產品的能力；來自其他公司製造和銷售或正在開發的產品的競爭；公司產品的價格和需求；公司就其產品談判有利的許可或其他製造和營銷協議的能力；以及公司籌集額外資本的能力。如果該公司的產品商業化在財務上不成功，它將無法產生足夠的經常性產品收入來實現和保持盈利。
該公司目前有兩種經商業批准的產品，Auvelity和Sunosi，不能保證公司的研究和開發努力將導致除Auvelity和Sunosi之外的產品成功商業化。開發和商業化一種產品需要大量的時間和資金，並受到監管審查和批准以及來自其他生物技術和製藥公司的競爭。該公司在快速變化的環境中運營，依賴於其員工和顧問的持續服務以及獲得和保護知識產權。
預算的使用
管理層在制定編制這些財務報表時使用的估計和假設時考慮了許多因素。管理層必須在這一過程中做出重大判斷。此外，其他因素可能會影響估計，包括預期的業務和運營變化、與編制估計時使用的假設相關的敏感性和波動性，以及歷史趨勢是否有望代表未來趨勢。估算過程通常可能會對最終的未來結果產生一系列潛在的合理估算，管理層必須選擇一個在該合理估算範圍內的數額。這一過程可能會導致實際結果與編制財務報表時使用的估計金額大不相同，如果這些結果與歷史經驗不同，或者其他假設被證明不是實質上準確的，即使這些假設在做出時是合理的。在編制這些財務報表時，管理層在以下方面使用了重大估計：基於股票的薪酬支出；認股權證公允價值的確定；研究和開發成本的會計；收購的會計；商譽和無形資產的減值；或有對價公允價值的確定；扣款、現金折扣、銷售回扣、回報和其他調整；以及公司遞延稅項淨資產和相關估值撥備的可回收性。
9
收入確認
根據會計準則編纂(「ASC」)主題606，與客戶的合同收入(「ASC 606」)，當客戶獲得承諾的貨物或服務的控制權時，公司確認收入，其數額反映了公司預期從貨物或服務交換中獲得的對價。控制權的轉讓以合同履行義務爲基礎，該義務發生在所有權轉移以及公司貨物實際轉讓給客戶時，因爲客戶已經獲得了對所有重大經濟利益的控制權，公司獲得了支付權。
爲了確定本公司認定屬於ASC606範圍內的安排的收入確認，本公司執行以下五個步驟：(I)確定與客戶的合同(S)，(Ii)確定合同中的履約義務，(Iii)確定交易價格，(Iv)將交易價格分配到合同中的履約義務，以及(V)在實體履行履約義務時確認收入。本公司僅將五步模式應用於符合ASC 606規定的合同定義的安排，包括本公司很可能會收取其有權收取的對價，以換取其轉讓給客戶的商品或服務。在合同開始時，一旦合同被確定在ASC 606的範圍內，公司就評估每份合同中承諾的商品或服務，確定哪些是履行義務，並評估每一項承諾的商品或服務是否不同。然後，公司確認在履行履約義務時分配給各自履約義務的交易價格的金額爲收入。有關產品銷售會計的完整討論，請參閱產品銷售淨額(見下文)和附註13.收入.
許可協議
該公司從與製藥公司就某些產品的開發和商業化達成的許可證或類似協議中獲得收入。這類協議可以包括以許可證形式轉讓知識產權。客戶支付的款項可能包括不可退還的預付費用、基於所定義里程碑的實現情況的付款以及產品銷售的版稅。
如果本公司的知識產權許可被確定有別於安排中確定的其他履行義務，本公司在轉讓許可控制權時將分配給該許可的交易價格確認爲收入。對協議中所有其他承諾的商品或服務進行評估，以確定它們是否不同。如果它們不是不同的，它們就與其他承諾的商品或服務相結合，形成一個不同的承諾商品或服務捆綁包。向公司支付的任何額外對價反映其獨立銷售價格的可選未來服務不向客戶提供實質性權利，因此不被視爲履行義務。如果可選的未來服務的定價方式爲客戶提供顯著或遞增的折扣，則這些服務屬於實質性權利，並作爲單獨的績效義務入賬。
合同開始時的或有里程碑被估計到很可能不會發生重大收入逆轉的程度，並使用最可能金額法計入交易價格。不在公司控制範圍內的里程碑付款，如監管批准，在收到這些批准之前不被認爲是可能實現的，因此，可變對價受到限制。然後，交易價格按相對獨立的銷售價格分配給每項履約義務，公司在履行合同項下的履約義務時確認收入。在每個報告期結束時，本公司重新評估實現開發或基於銷售的里程碑付款的可能性，即不會發生重大收入逆轉，並在必要時調整整體交易價格的估計。任何此類調整均按累積追趕原則入賬，這將影響許可證和其他收入以及調整期間的收益。
對於包括基於銷售的特許權使用費的安排，包括基於銷售的里程碑付款，以及被視爲與特許權使用費相關的主要項目的知識產權許可，收入在(I)發生相關銷售時，或(Ii)部分或全部特許權使用費分配給的履約義務已經履行(或部分履行)時確認。
10
產品銷售，淨額
產品銷售收入在扣除準備金後入賬，以備變動對價。這些準備金反映了公司根據合同條款對公司有權獲得的對價金額的最佳估計。本公司向與本公司簽訂正式協議的批發商(統稱爲「分銷商」)銷售美國的Auvelity和Sunosi。這些分銷商隨後將該公司的產品轉售給零售藥店。該公司還向加拿大的經銷商銷售Sunosi，並在產品供應的基礎上向Pharmanovia銷售。Sunosi隨後被Pharmanovia在某些前美國市場出售。本公司不以寄售安排銷售產品，產品銷售收益的收取不取決於客戶將商品出售給第三方。見注13.截至2024年9月30日和2023年9月30日的三個月和九個月的產品銷售收入進一步細分，淨額。
可變對價準備金
該公司對可變對價的估計和對是否將估計金額計入交易價格的決定主要基於對其預期業績的評估和所有合理可用的信息(歷史、當前和預測)。這些準備金反映了公司根據合同條款對公司有權獲得的對價金額的最佳估計，並被歸類爲應收賬款的減少額(如果應付給客戶)或應計費用和其他流動負債(如果應付給第三方)。交易價格中包含的可變對價金額可能受到限制，只有在被認爲未來期間確認的累計收入金額可能不會發生重大逆轉的情況下，才會包含在淨銷售價格中。最終收到的實際對價金額可能與估計不同。如果未來的實際結果與我們的估計不同，公司將調整這些估計，這將影響產品淨收入和在這些差異已知期間的收益。
回扣、折扣和其他激勵措施的撥備基於預期的患者使用量以及分銷渠道中的庫存水平，以確定對福利提供者的合同義務。此外，在某些情況下，銷售通常具有有限的返回權。收入是在銷售時確定的回扣、折扣和其他獎勵和回報撥備後計入的淨額。該公司使用客戶群利用組合數據、產品價格變化、政府定價計算和以前的付款歷史來估計可變對價。當趨勢表明調整是適當的並反映實際經驗時，將調整回扣、折扣和其他激勵措施的應計金額。
貿易折扣和津貼-該公司通常提供折扣，其中包括在公司合同中明確規定的獎勵費用，並在確認相關產品收入的期間記錄爲收入減少。此外，該公司還(通過貿易折扣和補貼)補償其分銷商的分銷服務和數據。這些付款被記錄爲產品銷售額的減少以及綜合資產負債表上淨額的應收賬款的減少。
產品退貨-根據產品的到期日，本公司一般對從本公司購買的產品提供有限的退貨權利。該公司估計可能退回的產品銷售金額，並將這一估計記錄爲相關產品銷售確認期間的收入減少，以及應計費用和其他流動負債的組成部分。該公司目前使用現有的行業數據、歷史產品銷售信息和實際退貨經驗來估計產品退貨負債。
退款和折扣-向供應商收取的費用和折扣是指合同承諾以低於向分銷商收取的標價銷售產品所產生的估計債務。經銷商按他們爲產品支付的價格與最終售價之間的差額向公司收取費用。這些準備金是在確認相關產品銷售的同一期間建立的，導致產品銷售和應收賬款淨額減少。
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回扣-返點適用於：醫療補助、管理式醫療保健和所有適用州的補充返點，這些返點由醫療補助藥品返點計劃下的法定政府定價計算要求定義。Tricare對TRICARE第三方管理員的回扣是根據與國防衛生局達成的協議中定義的法定計算得出的。D部分和商業管理醫療返點是根據與藥房福利經理(「PBM」)和管理醫療組織簽訂的合同支付的。在收到簽約實體的發票後，根據簽約實體成員對產品的使用情況，向這些實體支付回扣。回扣津貼還包括根據2022年《通貨膨脹降低法案》爲聯邦醫療保險D部分應支付的金額。公司估計這些回扣，並在相關產品銷售確認的同一時期記錄這些估計，導致產品銷售減少以及應計費用和其他流動負債的一部分。
覆蓋範圍差距-聯邦醫療保險D部分承保缺口是指當患者是由聯邦醫療保險和醫療補助服務中心管理的聯邦醫療保險D部分處方藥計劃的成員時，消費者爲處方藥費用支付的一段時間，該期間位於初始承保限額和災難性承保閾值之間。本公司估計出售給患者的商品在保險缺口中的百分比，並在銷售時調整此類折扣的交易價格，從而減少產品銷售以及應計費用和其他流動負債的一部分。
其他激勵措施-該公司提供的其他激勵措施包括自願患者援助計劃，如共同支付援助計劃，旨在向符合條件的商業保險患者提供財政援助，這些患者擁有付款人要求的處方藥共同付款。自付援助應計費用的計算依據是索賠估計數和公司預計收到的與已確認爲收入的產品相關的每項索賠的成本。準備金在確認相關收入的同一期間入賬，導致產品銷售減少以及應計費用和其他流動負債的一部分。
該公司作出的重大估計和判斷對其產品淨收入的確認有重大影響。第三方付款人對回扣、退款和折扣的索賠經常在相關銷售之後向公司提交，這可能會導致新信息知曉的時間段的調整。該公司將根據新的信息調整其估計，這些信息包括有關其產品的實際回扣、退款和折扣的信息。
收入成本
該公司的收入成本包括產品銷售成本和與收到的預付許可收入相關的費用分攤費用。產品銷售成本主要包括直接成本(包括材料、運輸、搬運和製造成本)、間接費用和產品使用費。產品銷售成本不包括折舊和攤銷。在2023年第一季度，該公司錄得
該公司承擔了Jazz對Sk生物製藥有限公司(「SK」)和AIR Biophma，LLC(「AIR」)的特許權使用費和基於銷售的里程碑承諾。SK是Sunosi的發起人，並保留在
外幣折算
以外幣計價的收入和支出在發生之日按匯率換算成美元。境外業務的資產和負債按期末匯率折算。匯率波動對將外幣兌換成美元的影響包括在經營報表中，對公司的綜合財務報表並不重要。
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細分市場信息
運營部門被定義爲企業的組成部分，其獨立的離散信息可供首席運營決策者或決策小組在決定如何分配資源和評估業績時進行評估。該公司將其運營和管理業務視爲
現金和現金等價物
本公司將收購時到期日在三個月或以下的所有高流動性投資視爲現金等價物。該公司的現金和現金等價物包括支票帳戶和隔夜清償帳戶中的持有量。該公司的現金等價物，即在一個清掃帳戶中持有的貨幣市場基金，按公允價值經常性計量。截至2024年9月30日，現金和現金等價物餘額爲#美元。
風險集中
信用風險集中-可能使公司面臨集中信用風險的金融工具包括現金和現金等價物。本公司維持其在金融機構的現金存款，這些現金存款超過保險限額。2024年9月30日，公司的大部分現金由
風險集中，其他- 該公司產品的合同製造商數量有限。有時，該公司的產品可能只有一個製造商或供應商。
業務合併
本公司採用收購會計方法將收購作爲一項業務合併入賬，該方法要求收購的所有可識別資產和承擔的負債均按其估計公允價值入賬。購買對價的公允價值超過可確認資產和負債的公允價值的部分計入商譽。在確定收購資產和承擔負債的公允價值時，管理層作出重大估計和假設。評估無形資產的關鍵估計包括但不限於收購專利技術帶來的未來預期現金流。管理層對公允價值的估計是基於被認爲是合理的假設，但本質上是不確定和不可預測的，因此，實際結果可能與估計不同。
作爲收購的結果，該公司記錄了商譽和無形資產。
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商譽
商譽被認爲是無限期的，因此不攤銷。本公司每年測試商譽賬面值是否可收回，或在事件或環境變化顯示資產可能減值時更頻繁地測試商譽賬面值是否可收回。在審核減值商譽時，本公司首先評估定性因素，以確定報告單位的公允價值是否更有可能低於其賬面價值。如果定性因素確定有必要完成商譽減值測試，則相關報告單位的公允價值將被確定並與其賬面價值進行比較。如果公允價值大於賬面價值，則賬面價值被視爲可收回，無需採取進一步行動。如果公允價值估計低於賬面價值，商譽將按賬面金額超過報告單位公允價值的金額被視爲減值，並在本公司的綜合經營報表中計入商譽減值。截至12月31日，公司完成了年度商譽評估。截至2024年9月30日，該公司已確定它已
無形資產
該公司的無形資產在其估計的十年受益期內採用直線法攤銷。本公司通過考慮可能需要修訂使用壽命估計或表明資產可能減值的事件或情況變化，定期評估無形資產的可回收性。於截至二零二四年九月三十日止九個月內，本公司並無發現任何顯示該無形資產存在潛在減值的事件或情況變化.
或有對價
在企業合併中支付的對價可能包括潛在的未來付款，這取決於被收購的企業在未來實現某些里程碑(「或有對價」)。Jazz支付的特許權使用費是公司目前在美國的Sunosi淨銷售額的高個位數特許權使用費，以及公司在美國未來適應症的Sunosi淨銷售額的中位數特許權使用費。或有對價負債按購入之日的估計公允價值計量，並於確認期間於綜合經營報表中記錄公允價值的後續變動。本公司採用概率加權收益法估計收購日期及其後報告期的或有對價的公允價值，並作出重大假設，包括當前和未來跡象下Sunosi的估計未來銷售額、監管和商業里程碑成就的時間、技術和監管成功率的概率和貼現率。預期於結算日後12個月內結清的或有對價負債以流動負債列報，非流動部分計入綜合資產負債表的總負債內。
公允價值計量
公允價值被定義爲在計量日市場參與者之間的有序交易中，爲資產或負債在本金或最有利的市場上轉移負債而收取或支付的交換價格。按公允價值計量的資產和負債採用三級公允價值層次結構進行報告，該層次結構對用於計量公允價值的投入進行了優先排序。這種層次結構最大限度地利用了可觀察到的輸入，最大限度地減少了不可觀察到的輸入。用於計量公允價值的三種投入水平如下：
第1級-在活躍市場上對公司在計量日期有能力獲得的相同資產或負債的報價。
第2級--資產或負債可直接或間接觀察到的活躍市場報價以外的投入。
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第3級--沒有或很少有市場數據的不可觀察到的輸入，這需要報告實體制定自己的假設。
在某種程度上，估值是基於市場上較難觀察到或無法觀察到的模型或投入，公允價值的確定需要更多的判斷。因此，本公司在厘定公允價值時所作出的判斷，對分類爲第3級的工具的判斷程度最高。公允價值層次內的資產或負債水平是以對公允價值計量有重大意義的任何投入中的最低水平爲基礎。
公司的金融工具包括現金和現金等價物、應收賬款、應付賬款、應計費用和其他負債、或有權證負債、流動和長期債務以及流動和非流動或有對價。該公司的一級金融工具包括現金和現金等價物、應收賬款、應付賬款、應計費用和其他負債。由於它們的短期到期日，在隨附的綜合財務報表中報告的賬面價值接近其各自的公允價值，因此它們被視爲1級。本公司資產負債表上債務的賬面價值估計爲接近其公允價值。本公司的3級金融工具包括或有認股權證負債、當期及非當期或有對價，這是由於在確定其各自的公允價值時所需的重大不可觀察的投入。
該公司將或有對價負債的公允價值歸類爲公允價值等級中的第三級，因爲該估計是基於需要管理層判斷的重大不可觀察輸入。或有對價負債的公允價值採用概率加權收益法進行估計，並使用重大假設，包括Sunosi在當前和未來跡象中的估計未來銷售額、監管和商業里程碑成就的時間、技術和監管成功的可能性以及貼現率。或有對價負債須於每個預期資產負債表日重新計量，公允價值的任何變動均記錄在綜合經營報表中。詳情見附註6.金融工具公允價值。
該公司根據關鍵假設和投入，採用布萊克-斯科爾斯模型估計認股權證負債的公允價值。本公司利用概率評估評估剩餘貸款協議(定義見下文)認股權證歸屬的可能性，並將發生概率百分比分配至所計算的公允價值，因此被視爲公允價值層次中的第三級。本公司將根據貸款協議預期將於未來發行的認股權證入賬爲負債，並採用Black-Scholes估值模型按公允價值計量。認股權證須於每個預期資產負債表日重新計量，公允價值的任何變動均記錄在綜合經營報表內。
應收賬款淨額
公司的應收賬款淨額來自產品銷售，代表其客戶的應收賬款。它們通常以銷售總額減去貿易折扣、津貼和按存儲容量使用計費所產生的準備金來表示。應收賬款通常有一個標準的p的付款期限
公司監控客戶的財務表現和信譽，以便對客戶信用狀況的變化作出適當的評估和回應。2023年第一季度，該公司開始通過批發客戶分銷產品。本公司通過評估損失風險和可收回的相關信息，包括歷史信用損失、現有合同付款條款、客戶的實際付款模式、個別客戶的情況以及對預計在應收賬款整個合同期限內存在的經濟狀況的合理和可支持的預測，來估計其應收賬款的預期信用損失。從歷史上看，該公司沒有經歷過重大的信貸損失。根據其評估，截至2024年9月30日，該公司擁有
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發債成本
債務發行成本包括獲得長期融資所產生的成本。該等成本在綜合資產負債表中被分類爲直接從相關債務負債的賬面金額中扣除，然後按實際利率法在綜合經營報表中攤銷爲利息支出。
本公司根據ASC 470-50對其債務工具的修訂進行評估，債務修改和清償(「ASC 470」)，以確定該修正案應被視爲修改還是終止。當新債務與原始票據的條款並無「實質不同」(如ASC 470中的債務修改指引所界定)時，修訂可被視爲已修改。被視爲修訂的修訂將根據經修訂條款計入預期收益調整，而貸款人費用及與第三方直接產生的成本(如屬重大事項)則按實際利率法記作債務貼現及攤銷利息開支。
庫存
本公司以成本或估計可變現淨值中較低者對其存貨進行估值。由於採購會計原因，與收購相關的剩餘存貨按公允價值列報。本公司於每個報告期內對資本化存貨的可回收性進行評估，並將任何過剩及陳舊存貨減記至首次確認減值期間的估計可變現淨值。該等減值費用如發生，則記入收入成本內。
當根據管理層的判斷，認爲未來的商業化是可能的，並且未來的經濟效益有望實現時，公司在監管部門批准後對與公司產品相關的庫存成本進行資本化。在收到監管機構批准的候選產品之前獲得和製造的庫存在發生時計入研究和開發費用。可用於臨床或商業產品生產的庫存，如果選擇用於臨床生產活動，則作爲研發費用支出。
庫存水平是根據一年內銷售的數量進行評估的。如果庫存水平超過未來12個月後的估計銷售金額，本公司將此類庫存的估計歸類爲非流動庫存。
設備，網絡
設備主要由計算機設備組成，按成本入賬。設備在其估計使用年限內以直線方式折舊，該公司估計使用年限爲
研發成本
研究和開發成本在發生時計入費用。研發支出主要包括與員工相關的支出，包括工資、福利、差旅和股票薪酬支出、合同服務、第三方服務提供商進行研究、臨床前和臨床研究的成本、實驗室用品、產品許可費、諮詢和其他相關支出。研究、臨床前和臨床研究費用是根據與代表公司進行和管理研究、臨床前和臨床活動的第三方研究和開發組織簽訂的合同，包括與內部管理人員和外部服務提供商討論服務的進度或完成階段以及爲該等服務支付的合同費用而估計的。如果服務業績的實際時間安排或努力程度與最初估計數不同，應計項目也會相應調整。爲獲得開發、使用、製造和商業化尚未達到技術可行性且未來沒有替代用途的產品的許可協議而支付的相關費用計入已發生的費用。
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廣告費
廣告成本計入銷售、一般和行政費用，並在發生時計入費用。本公司將廣告費用視爲與推廣本公司商業產品有關的費用。截至2024年9月30日的三個月和九個月，廣告費是$
所得稅
所得稅按資產負債法覈算。根據這一方法，遞延稅項資產和負債被確認爲可歸因於現有資產和負債的賬面金額與其各自稅基之間的差額、營業虧損和稅項抵免結轉之間的差額的未來稅務後果。遞延稅項資產及負債以制定稅率計量，預期適用於預計收回或結算該等暫時性差額的年度的應稅收入。稅率變動對遞延稅項資產和負債的影響在包括頒佈日期在內的期間的收入中確認。如果根據現有證據的份量，部分或全部遞延稅項資產很可能無法變現，則會提供估值減值準備。
該公司估計每年的有效稅率爲
本公司只有在根據不確定稅務狀況的技術價值以及現有事實和情況進行審查後更有可能維持的情況下，才會確認來自該不確定稅務狀況的稅務利益。當存在不確定的稅收頭寸時，本公司確認稅收頭寸的稅收優惠，以使其更有可能實現。截至2024年9月30日，本公司不認爲存在任何重大不確定的稅務頭寸。如果公司認爲未來有必要計提利息或罰款，則該金額將作爲所得稅支出的組成部分列報。
基於股票的薪酬
對於已發行的股票期權，公司使用Black-Scholes期權定價模型估計每個期權的授予日期公允價值。布萊克-斯科爾斯模型考慮了公司普通股的預期波動率、無風險利率、期權的估計壽命、公司普通股的收盤價和行使價。布萊克-斯科爾斯計算中使用的估計涉及固有的不確定性和管理層判斷的應用。此外，當股權獎勵被沒收時，公司會對其進行覈算。
對於限制性股票單位，公司以公司普通股的形式發行。這些獎勵的公平市場價值以授予日每股市場收盤價爲基礎。
本公司確認授予日期股票期權和RSU在必要的服務期內的公允價值，這通常是歸屬期限。對於僅受基於服務的歸屬條件約束的獎勵，本公司選擇以直線基礎確認基於股票的薪酬支出。對於受業績歸屬條件約束的獎勵，當業績條件有可能實現時，本公司採用加速歸因法確認基於股票的薪酬支出。與基於股票的補償有關的費用與受贈人的現金補償記錄在同一財務報表行項目內。
本公司在行使購股權及RSU時的政策是，股份將根據股東於2015年11月採納的本公司2015年綜合激勵薪酬計劃股份池作爲新股發行。
17
普通股基本淨虧損和攤薄淨虧損
普通股每股基本淨虧損的計算方法是用淨虧損除以當期已發行普通股的加權平均股數。普通股每股攤薄淨虧損包括根據2023年員工購股計劃(「ESPP」)可能行使或轉換證券的影響(如有)，例如認股權證、股票期權、RSU及/或普通股，這將導致發行普通股的增量股份。由於這些項目的影響在淨虧損期間是反攤薄的，截至2024年9月30日和2023年9月30日的三個月和九個月的普通股每股基本淨虧損和稀釋後每股淨虧損沒有差異.
租賃
本公司在合同開始時確定一項安排是否爲租賃。使用權資產代表公司在租賃期內使用標的資產的權利，租賃負債代表公司支付租賃所產生的租賃款項的義務。在評估合同是否包含租賃時，本公司考慮(1)合同是否明確或隱含地確定了合同定義的資產，以及(2)公司從使用標的資產中獲得了幾乎所有的經濟利益，並指示在合同期限內如何使用該資產以及用於什麼目的。
該公司的租賃協議包含租賃和非租賃部分。非租賃部分主要包括維修費和水電費。本公司已採取實際權宜之計，將非租賃部分的固定付款與租賃付款合併，並將其作爲單一租賃部分進行會計處理，從而增加了租賃資產和相應的負債額。本公司租賃安排項下的付款主要爲固定付款，但變動付款於已發生時列支，並不包括在經營租賃資產及負債內。
租賃資產及負債於租賃開始日按租賃期內租賃付款的現值確認。在確定租賃期限時，公司包括在合理確定公司將行使該選擇權時延長或終止租約的選擇權。本公司使用合同中隱含的利率，當該利率容易確定時，使用公司的遞增借款利率，當合同中隱含的利率不能根據開始日期可獲得的信息確定租賃付款的現值時，使用公司的遞增借款利率。
公司的經營租賃反映在公司綜合資產負債表中的使用權經營資產、經營租賃負債的當前部分和經營租賃負債的長期部分。經營租賃費用在租賃期間以直線法確認，並計入銷售、一般和行政費用。融資租賃計入非流動存貨和其他資產；應計費用和其他流動負債；以及融資租賃負債，長期計入公司的綜合資產負債表。融資租賃項下的資產在租賃期內按直線攤銷，並計入銷售、一般及行政費用。短期租賃被定義爲在開始日期的租期爲12個月或以下的租賃，不包括延長期限或購買本公司合理確定將行使的標的資產的選擇權，不包括這種處理，並按直線法在租賃期內確認。
近期會計公告
2023年11月，財務會計準則委員會(FASB)發佈了會計準則更新(ASU)2023-07分部報告，要求在年度和中期基礎上披露增量分部信息。該標準在2023年12月15日之後的年份和2024年12月15日之後的過渡期內有效，並允許及早採用。該公司目前正在評估在其合併財務報表上採用這一指導意見的效果。
18
2023年12月，FASB發佈了ASU 2023-09，《所得稅披露的改進》，其中要求披露司法管轄區支付的分類所得稅，加強有效稅率調節中的披露，並修改其他與所得稅相關的披露。這些修正案在2024年12月15日之後的年度期間生效。該公司目前正在評估在其合併財務報表上採用這一指導意見的效果。
2024年11月，FASB發佈了ASU 2024-03，損益表-報告全面收入-費用分解披露(子主題220-40)：損益表費用分解。這個ASU要求在財務報表附註中對損益表中所列某些類別的費用進行額外的分類披露。該指南適用於2026年12月15日之後開始的財政年度，以及2027年12月15日之後開始的財政年度內的過渡期，並允許提前採用。該公司目前正在評估在其合併財務報表上採用這一指導意見的效果。
說明3.應收賬款，淨額
應收賬款淨額包括：
|
|
9月30日， |
|
|
12月31日， |
|
||
貿易應收款項 |
|
$ |
|
|
$ |
|
||
減：可變考慮的準備金 |
|
|
( |
) |
|
|
( |
) |
應收賬款淨額 |
|
$ |
|
|
$ |
|
||
該公司應收賬款餘額最大的客戶是Cardinal Health，Inc.，森科拉公司，和McKesson Corporation，該公司約佔
說明4.庫存
庫存包括以下內容：
|
|
9月30日， |
|
|
12月31日， |
|
||
原料 |
|
$ |
|
|
$ |
|
||
Oracle Work in Process |
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|
|
|
|
|
||
成品 |
|
|
|
|
|
|
||
總 |
|
$ |
|
|
$ |
|
||
有幾個
下表總結了所示每個時期公司庫存的資產負債表分類：
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9月30日， |
|
|
12月31日， |
|
||
資產負債表分類 |
|
|
|
|
|
|
||
庫存，淨額 |
|
$ |
|
|
$ |
|
||
非流動庫存和其他資產 |
|
|
|
|
|
|
||
總 |
|
$ |
|
|
$ |
|
||
19
說明5.無形資產
下表提供了公司在所示各個期間無形資產的公允價值。
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|
總賬面金額 |
|
|
累計攤銷 |
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|
賬面淨額 |
|
|
剩餘加權平均使用壽命 |
|||
2023年12月31日餘額 |
|
|
|
|
|
|
|
|
|
|
|
|||
壽命較長的無形資產 |
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$ |
|
|
$ |
|
|
$ |
|
|
||||
2024年9月30日餘額 |
|
|
|
|
|
|
|
|
|
|
|
|||
壽命較長的無形資產 |
|
$ |
|
|
$ |
|
|
$ |
|
|
||||
基於截至年記錄的有限壽命無形資產 2024年9月30日，假設基礎資產不會發生減損且公司不會改變資產的預期壽命，未來五年及此後期間的未來攤銷費用估計如下：
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|
預計攤銷費用 |
|
|
2024 |
|
$ |
|
|
2025 |
|
|
|
|
2026 |
|
|
|
|
2027 |
|
|
|
|
2028 |
|
|
|
|
此後 |
|
|
|
|
總 |
|
$ |
|
|
說明6.金融工具公允價值
就收購而言，公司向Jazz支付Sunosi在美國淨銷售額的特許權使用費。用於估值該或有對價的貼現現金流量法包括不容易觀察的市場數據的輸入，即第三級輸入。或有對價的公允價值反映爲當前應計或有對價美元
按經常性基準計量的金融工具的公允價值如下：
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2024年9月30日 |
|
|||||||||||||
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1級 |
|
|
2級 |
|
|
3級 |
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總 |
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||||
資產： |
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|
|
|
|
|
|
|
|
|
|
|
||||
現金及現金等值物-貨幣市場基金 |
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
||
負債： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
或然代價 |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
|
$ |
|
||
|
|
2023年12月31日 |
|
|||||||||||||
|
|
1級 |
|
|
2級 |
|
|
3級 |
|
|
總 |
|
||||
資產： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
現金及現金等值物-貨幣市場基金 |
|
$ |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
||
負債： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
或然代價 |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
|
|
$ |
|
||
20
或然代價負債
或有對價負債的公允價值在每個報告期按市價計價，並重新計量 2024年9月30日。截至2024年9月30日，或有對價負債的公允價值變化如下：
|
|
或然代價 |
|
|
2023年12月31日餘額 |
|
$ |
|
|
公允價值調整 |
|
|
|
|
付款 |
|
|
( |
) |
2024年9月30日餘額（3級） |
|
$ |
|
|
記錄負債的或有代價的經常性第三級公允價值計量包括以下重大不可觀察輸入數據：
|
|
|
|
|
|
截至2024年9月30日 |
|
截至2023年12月31日 |
|
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估值方法 |
|
重大不可觀察輸入數據 |
|
加權平均值（範圍，如果適用） |
|
加權平均值（範圍，如果適用） |
或然代價 |
|
概率加權收益法 |
|
貼現率 |
|
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||
|
|
|
|
收入貼現率 |
|
|
該公司對或有對價負債的公允價值計量已被歸類爲第3級，因爲其估值需要對需要使用不可觀察輸入的因素進行實質性判斷和估計。或有對價負債的公允價值是通過使用概率加權收益法進行估計的，其中使用了重要假設，包括Sunosi在當前和未來跡象中的估計未來銷售額、監管和商業里程碑成就的時間、技術和監管成功率的可能性以及貼現率。如果其中一項或多項假設發生重大變化，或有對價負債的估計公允價值可能會導致公允價值計量顯着提高或降低。
說明7.應計費用和其他流動負債
應計費用和其他流動負債包括：
|
|
9月30日， |
|
|
12月31日， |
|
||
應計研究和開發 |
|
$ |
|
|
$ |
|
||
應計補償 |
|
|
|
|
|
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||
應計銷售，一般和行政 |
|
|
|
|
|
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應計銷售折扣、回扣和津貼 |
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|
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||
應計版稅 |
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|
|
|
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||
應計利息 |
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|
|
|
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||
應計稅 |
|
|
|
|
|
— |
|
|
融資租賃負債，流動 |
|
|
|
|
|
— |
|
|
總 |
|
$ |
|
|
$ |
|
||
21
附註8.貸款和擔保協議
大力神資本公司
就本附註8而言，本附註8所使用但未另作定義的大寫術語應具有貸款協議(定義如下)所賦予它們的涵義。
《貸款協議》第五修正案
於2024年9月30日，本公司訂立日期爲2020年9月25日的貸款及擔保協議第五修正案(「第五修正案」)(經日期爲2021年10月14日的貸款及擔保協議第一修正案修訂，經日期爲2022年3月27日的貸款及擔保協議第二修正案進一步修訂，並經日期爲2023年1月9日的貸款及擔保協議第三修正案進一步修訂，並經日期爲5月8日的貸款及擔保協議豁免及第四修正案進一步修訂)，2023年)(「貸款協議」)與作爲行政代理和抵押品代理的馬里蘭州公司Hercules Capital，Inc.以及作爲貸款人的其他金融機構或實體(統稱爲「貸款人」)就該協議項下的定期貸款(「2020定期貸款」)達成協議。
《第五修正案》修訂了貸款協議的條款，除其他事項外：(1)將第3檔的承諾額從#美元增加到
《貸款協議》第四修正案
於2023年5月8日，本公司以行政代理及抵押品代理身分及貸款人身分與Hercules訂立貸款協議豁免及第四修正案(「第四修正案」)。
第四修正案增加了Axome馬耳他在美國境外可以持有的現金金額，從#美元
《貸款協議》第三修正案
於2023年1月9日，本公司以行政代理及抵押品代理身分及貸款人身分與Hercules訂立貸款協議第三修正案(「第三修正案」)。
第三修正案修改了貸款協議的條款，除其他事項外：
22
截至2024年9月30日，該公司允許第二批，總額爲#美元。
2021年10月14日，該公司與Hercules簽訂了貸款和擔保協議第一修正案。於2022年3月27日，就收購事項(如上所述)，本公司與Hercules訂立貸款及抵押協議第二修正案(「第二修正案」)。第二修正案於2022年5月9日結束，與收購結束同時。
作爲債務的抵押品，公司已向Hercules授予公司對公司所有財產(包括知識產權)的所有權利、所有權和權益的優先擔保權益，其中包括公司與安盛首席執行官兼董事會主席Herriot Tabuteau萬.D.擁有的實體Antecip的一項現有許可協議(「許可協議」)，但有限的例外情況除外。Antecip同意(其中包括)根據與本公司、Antecip和Hercules的直接協議(「直接協議」)轉讓許可協議的抵押品。
貸款協議包含慣例陳述、擔保和契諾，包括本公司限制額外債務的契諾、留置權(包括知識產權和其他資產的負質押)、擔保、合併和合並、大規模資產出售、投資和貸款、某些公司變更、與關聯公司的交易和重大變更。於最初成交時，貸款協議並無載有適用的財務契諾。《第五修正案》於2024年9月結束後生效，適用以下有限的金融契約：
23
本公司於貸款協議項下的責任會在發生特定違約事件時加速履行，包括拖欠款項、無力償債及借款人的業務、營運或財務或其他狀況出現重大不利變化。
此外，本公司須支付某些期末費用，包括(A)初始期末費用#美元。
如果滿足某些期限延長條件，公司必須支付相當於
本公司可選擇預付全部或部分定期貸款，但須繳交相等於(I)的預付款罰金。
本公司評估了2023年1月生效的第三修正案是否代表根據ASC 470-50進行的債務修改或清償，債務修改和清償。因爲第三修正案規定的現金流的現值小於
本公司還評估了2024年9月生效的第五修正案是否代表根據ASC 470進行的債務修改或清償。由於第五修正案的條款與第四修正案的條款沒有實質性差異，本公司將該修正案視爲債務修改。
貸款利息支出和攤銷
長期債務和未攤銷債務貼現餘額如下：
|
|
9月30日， |
|
|
12月31日， |
|
||
未償債務總額 |
|
$ |
|
|
$ |
|
||
加：附加的最終付款費 |
|
|
|
|
|
|
||
減：未攤銷債務貼現，長期 |
|
|
( |
) |
|
|
( |
) |
減去：長期債務的當前部分 |
|
|
— |
|
|
|
— |
|
應付貸款，長期 |
|
$ |
|
|
$ |
|
||
鑑於其可變利率，債務的公允價值接近其公允價值。
24
利息費用、期末付款費用攤銷、與發行成本相關的債務折扣攤銷以及公司債務的認購權如下：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
利息開支 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
期末付款費攤銷 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
債務貼現相關發行成本和認購證攤銷 |
|
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|
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|
|
|
|
|
|
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|
||||
截至2011年，未償債務的計劃本金支付 2024年9月30日，具體如下：
2024 |
|
$ |
— |
|
2025 |
|
|
— |
|
2026 |
|
|
— |
|
2027 |
|
|
— |
|
2028 |
|
|
|
|
此後 |
|
|
— |
|
未償本金付款總額 |
|
$ |
|
附註9.承付款和或有事項
租賃
租賃在ASC主題842下說明。該公司作出會計政策選擇，不將確認要求應用於短期租賃。本公司在綜合經營報表中以直線方式確認短期租賃的租賃付款，並在產生該等付款義務的期間內確認可變租賃付款。因此，本公司不會確認任何租賃的租賃負債或使用權資產，而在開始日期，租賃期限爲
在
該公司從2024年第一季度開始實施機隊租賃計劃。租賃協議包括一份初步的
25
確認的租賃費用如下：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
經營租賃費用 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
融資租賃費用： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
使用權資產攤銷 |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
租賃負債利息 |
|
|
|
|
|
— |
|
|
|
|
|
|
— |
|
||
公司租賃的未來最低租賃付款額 2024年9月30日如下：
|
|
經營租賃 |
|
|
融資租賃 |
|
||
2024 |
|
$ |
|
|
$ |
|
||
2025 |
|
|
|
|
|
|
||
2026 |
|
|
|
|
|
|
||
2027 |
|
|
|
|
|
|
||
2028 |
|
|
|
|
|
|
||
此後 |
|
|
— |
|
|
|
— |
|
租賃付款總額 |
|
|
|
|
|
|
||
扣除計入的利息 |
|
|
( |
) |
|
|
( |
) |
租賃負債現值 |
|
$ |
|
|
$ |
|
||
說明10.股東權益
公開招股
市場上的產品
於2019年12月，本公司與SVB Securities LLC(現稱Leerink Partners LLC)(「Leerink」)訂立銷售協議(「2019年12月銷售協議」)，根據該協議，本公司可出售最多$
於2022年3月，本公司與Leerink訂立銷售協議(「2022年3月銷售協議」)，並提交招股說明書補充文件，根據該協議，本公司可出售最多$
2022年8月，公司提交了《2019年貨架登記說明書》的招股說明書補充文件，用於發行和銷售(如果有的話)高達$
26
於2022年12月，就2022年貨架登記聲明(定義見下文)，本公司提交了新的銷售協議招股說明書，以取代先前於2022年8月提交的與到期的2019年貨架登記聲明相關的招股說明書補充資料。新的銷售協議招股說明書涵蓋了公司發行和出售的相同金額的
根據2022年3月的銷售協議，截至2024年9月30日止三個月，本公司收到約
《公司》做到了
在2022年3月第二修正案結束後，大力神還購買了
普通股持有者有權
2023年6月公開發行
2023年6月，本公司完成了普通股的包銷公開發行(以下簡稱《2023年6月公開發行》)。該公司出售了
貨架登記表
2022年12月2日，本公司向美國證券交易委員會備案了發行普通股、優先股、權證、權利、債務證券和單位的自動擱置登記書(《2022年擱置登記書》)。於美國證券交易委員會備案後生效，是本公司目前唯一活躍的貨架登記。
根據美國證券交易委員會的規則，2022年貨架登記聲明允許公司未來不時以一次或多次不確定數量的公司普通股、優先股、債務證券和單位的公開發行形式要約和出售。價格不確定。當2022年貨架登記聲明涵蓋的任何證券發售時，將編寫招股說明書補充文件並向SEC提交，其中包含有關任何此類發行條款的具體信息。
27
股權激勵計劃
2015年11月，公司股東通過了2015年綜合激勵薪酬計劃（「2015年計劃」）。截至2024年9月30日，已有
股票期權
下表列出了截至 2024年9月30日：
|
|
Number |
|
|
加權 |
|
|
加權 |
|
|
骨料 |
|
||||
截至2023年12月31日的未償還債務 |
|
|
|
|
$ |
|
|
|
|
|
|
|
||||
授予 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
已鍛鍊 |
|
|
( |
) |
|
|
|
|
|
|
|
|
|
|||
被沒收/取消 |
|
|
( |
) |
|
|
|
|
|
|
|
|
|
|||
截至2024年9月30日未完成 |
|
|
|
|
$ |
|
|
|
|
|
$ |
|
||||
已歸屬，預計將於2024年9月30日歸屬 |
|
|
|
|
$ |
|
|
|
|
|
$ |
|
||||
可於2024年9月30日取消 |
|
|
|
|
$ |
|
|
|
|
|
$ |
|
||||
每個股票期權授予的公允價值在授予之日使用Black-Scholes期權定價模型進行估計。公司股票期權的預期期限是採用美國證券交易委員會關於股票薪酬的第107號工作人員會計公告中所述的「簡化」方法確定的。之所以選擇簡化方法，是因爲該公司由於經營歷史較短，其歷史期權行使經驗有限。無風險利率以授予時有效的美國國債收益率爲基礎，期限大致等於授予的預期期限。預期股息收益率是基於公司從未支付過現金股利，並且確實支付了現金股息
已授出期權的加權平均授出日期公允價值爲$
限售股單位
RSU的公允價值是在授予之日根據其普通股在該日的市場價格確定的。RSU的公允價值在歸屬期間按比例確認爲費用
28
下表列出了RSU的活動 截至2024年9月30日的九個月：
|
|
數 |
|
|
加權 |
|
||
截至2023年12月31日的未償還債務 |
|
|
|
|
$ |
|
||
授予 |
|
|
|
|
|
|
||
既得 |
|
|
( |
) |
|
|
|
|
沒收 |
|
|
( |
) |
|
|
|
|
截至2024年9月30日未完成 |
|
|
|
|
$ |
|
||
員工購股計劃
ESPP允許符合條件的員工購買公司普通股股份。收購價格等於
截至2024年9月30日,
基於股票的薪酬費用
確認的股票補償費用如下：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
研發 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
銷售，一般和行政 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
總 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
資本化爲庫存的股票補償費用總計美元
注11。權證
下表總結了 截至2024年9月30日的九個月：
|
|
權證 |
|
|
加權平均 |
|
||
截至2023年12月31日的未償還債務 |
|
|
|
|
$ |
|
||
發佈 |
|
|
|
|
|
|
||
已鍛鍊 |
|
|
|
|
|
|
||
截至2024年9月30日未完成 |
|
|
|
|
$ |
|
||
29
未清償認股權證
關於加入第三修正案，Hercules收到了購買總計
2023年權證、2022年權證和2020年權證的定價採用本公司普通股的成交量加權平均價格
說明12.每股普通股淨虧損
下表列出了普通股基本淨虧損和攤薄淨虧損的計算方法：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
每股普通股基本和稀釋淨虧損： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
淨虧損 |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
加權平均已發行普通股-基本普通股和稀釋普通股 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
每股普通股淨虧損-基本和稀釋 |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
|
$ |
( |
) |
以下潛在稀釋證券已被排除在稀釋加權平均股的計算之外，因爲它們具有反稀釋作用：
|
|
9月30日， |
|
|||||
|
|
2024 |
|
|
2023 |
|
||
股票期權 |
|
|
|
|
|
|
||
限制性股票單位 |
|
|
|
|
|
|
||
權證 |
|
|
|
|
|
|
||
ESPP |
|
|
|
|
|
— |
|
|
總 |
|
|
|
|
|
|
||
30
注13。收入
該公司通過分銷商在美國銷售Auvelity和Sunosi。該公司還將Sunosi出售給加拿大的分銷商，並通過向Pharmacia供應產品。Sunosi隨後由Pharacco在某些美國前市場出售。
許可證收入包括確認公司於2023年2月從Pharamerovia收到的預付款，特許權使用費收入與Pharamerovia在某些美國以外市場銷售Sunosi有關。
下表按產品列出了總收入摘要：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
產品銷售，淨額 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
奧維利蒂 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
蘇諾西 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
產品總銷售額(淨額) |
|
|
|
|
|
|
|
|
|
|
|
|
||||
Sunosi許可收入 |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
|
|
Sunosi特許權使用費收入 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
總收入 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
下表按地理位置列出了總收入摘要：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
產品銷售，淨額 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
美國 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
美國以外 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
產品總銷售額(淨額) |
|
|
|
|
|
|
|
|
|
|
|
|
||||
許可證收入 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
美國以外 |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
|
|
使用費收入 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
美國以外 |
|
|
|
|
|
|
|
|
|
|
|
|
||||
總收入 |
|
$ |
|
|
$ |
|
|
$ |
|
|
$ |
|
||||
截至2024年9月30日的九個月產品銷售額（淨額）包括因估計數變化而對2023年產品銷售撥備的調整
31
注14.許可協議
與Pharmanovia達成許可協議
於2023年2月，馬耳他有限公司及本公司全資附屬公司Axome馬耳他與Pharmanovia訂立獨家許可協議(「Pharmanovia許可協議」)，以在歐洲及中東及北非若干國家(「地區」)商業化及進一步開發Sunosi。根據Pharmanovia許可協議的條款，本公司保留其在Sunosi知識產權中的現有權益，並將領土上的這些權利許可給Pharmanovia。Pharmanovia公司獨自負責Sunosi在該領土的臨床開發和商業化。該公司將在無限期內繼續生產Sunosi並向Pharmanovia公司提供產品供應，當產品供應給Pharmanovia公司時，公司將確認收入爲產品銷售淨額的組成部分。
作爲簽署Pharmanovia許可協議的代價，該公司收到了一筆不可退還的歐元預付款
該公司根據ASC 606評估了Pharmanovia許可協議，並得出結論，Pharmanovia在交易中代表客戶。初始交易價格包括不可退還的預付款，這筆款項在2023年第一季度向Pharmanovia轉讓許可證時確認爲許可證收入，因爲符合ASC 606規定的收入確認要求。其餘的對價形式是可變的，因爲它們取決於基於銷售的目標或其他里程碑的實現情況。本公司評估了可變對價的限制，並得出結論，里程碑付款取決於監管機構的批准和第三方的行動，因此非常容易受到公司影響之外的因素的影響。因此，在合同開始時，里程碑不包括在交易價格中，因爲收入不可能不發生重大逆轉。以銷售爲基礎的里程碑將在達到相關銷售門檻的期間確認爲收入。所有其他開發或管理里程碑將在基本里程碑實現期間立即確認爲收入。與基於銷售的版稅相關的任何對價將在相關銷售發生時確認。該公司確認的特許權使用費收入爲$
與輝瑞達成獨家許可協議
2020年1月，該公司與輝瑞公司(「輝瑞」)就輝瑞的臨床和非臨床數據以及瑞波西汀的知識產權簽訂了獨家許可協議。瑞波西汀是該公司正在開發的用於治療發作性睡病的AXS-12中的活性藥物成分。該協議還規定該公司有獨家權利在美國開發和商業化埃斯波西汀，這是一種新的晚期候選產品，稱爲AXS-14，用於治療纖維肌痛。
根據協議條款，輝瑞獲得了
輝瑞還可以獲得最高美元的賠償。
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與Antecip達成獨家許可協議
於二零一二年，本公司訂立
關於貸款協議，本公司與Antecip及Hercules訂立直接協議，據此Antecip同意根據貸款協議進行許可協議的抵押品轉讓(其中包括)。
注15.版稅協議
2022年3月25日，本公司與Jazz簽訂了資產購買協議(「購買協議」)，根據該協議，本公司將從Jazz手中收購Sunosi在某些美國和前美國市場的商業和開發權。此次收購是在兩次獨立的交易中完成的。於滿足或豁免購買協議項下的成交條件後，買賣協議擬於二零二二年五月九日(「初步成交」)進行的指定初始資產的買賣。購買協議預期的特定美國以外資產的出售和購買發生在購買協議下的成交條件(「最終成交」)得到滿足或豁免之後的2022年11月14日。本公司採用收購會計方法將初始結賬作爲業務合併進行會計處理，將最終結賬作爲資產收購進行會計處理。
根據購買協議，該公司同意向Jazz支付的不可退還、不可入賬的特許權使用費相當於(A)任何當前指示的高個位數特許權使用費，或(B)在適用特許權使用費期限內對美國領土淨銷售額的任何未來指示的中位數個位數特許權使用費。Jazz在美國境外的淨銷售額不需要向Jazz支付版稅。
在最初的交易中，該公司承擔了Jazz對Sk和AIR的所有承諾。SK是Sunosi的發起人，並保留在
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附註16.所得稅
下表列出了該公司所有時期的所得稅前虧損和有效稅率：
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截至9月30日的9個月， |
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所得稅前虧損 |
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$ |
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$ |
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所得稅優惠(費用) |
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實際稅率 |
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該公司在美國和其業務所在的外國司法管轄區繳納所得稅。這些外國司法管轄區的法定稅率與美國不同。因此，公司的有效稅率將根據外國收入與美國收入的相對比例、淨營業虧損和稅收抵免結轉的利用情況、收入和支出的地理組合的變化以及管理層對遞延稅項資產變現能力和稅法變化等事項的評估變化而變化。本公司定期評估由美國國稅局(「IRS」)及其他稅務機關審核本公司的報稅表，以確定其所得稅儲備及開支是否足夠而產生不利結果的可能性。如果實際事件或結果與公司目前的預期不同，可能需要對其所得稅支出進行費用或抵免。
《公司》做到了
《公司》做到了
該公司目前正在接受國稅局的審查，以獲取公司2021年美國所得稅申報表。該公司目前尚未接受州一級審查。該公司的美國聯邦和州淨運營虧損自2012年成立以來就已發生，因此，接受潛在稅務審查的納稅年度可能從該日期起適用，因爲利用前幾年的淨運營虧損將開啓相關年度的審計。國稅局和/或州稅務當局。
說明17.關聯交易
自公司成立之日起，Herriot Tabuteau SEARCH. D一直是該公司的創始人、首席執行官、公司董事會主席，以及超過
該公司是一方
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項目2.管理層的討論與分析F財務狀況和業務成果
以下討論和分析包含有關我們的計劃和對未來可能發生的事情的預期的前瞻性陳述。前瞻性陳述基於一系列假設和估計，這些假設和估計固有地受到重大風險和不確定性的影響，由於許多已知或未知因素，包括但不限於「風險因素」中討論的那些因素，我們的結果可能與前瞻性陳述預期的結果存在重大差異。另請參閱本報告開頭列出的「關於前瞻性陳述的警告」。
您應結合本報告其他地方出現的未經審計的中期綜合財務報表及其相關腳註，以及管理層的討論和分析以及我們截至12月31日年度報告中包含的截至10-k表格年度報告中的已審計綜合財務報表，閱讀以下討論和分析，2023年，於2024年2月23日向美國證券交易委員會（SEC）提交.
概述
我們是一家生物製藥公司，引領着中樞神經系統疾病治療的新紀元。我們通過識別護理中的關鍵差距來實現科學突破，並開發差異化產品，重點放在新的行動機制上，使患者結果能夠取得有意義的進步。我們的CNS產品組合包括兩個已批准的產品--Auvelity®和Sunosi®，這兩個產品也正在爲進一步的適應症而開發，還有三個尚未批准的候選產品，AXS-07、AXS-12和AXS-14，正在爲多個適應症而開發。2022年5月，Axome Treateutics，Inc.或Axome或本公司完成了從Jazz PharmPharmticals或Jazz手中收購Sunosi的美國交易，2022年11月，該公司從Jazz手中收購了Sunosi的前美國資產，用於某些國際市場，或集體收購。Sunosi是一種獲得美國食品和藥物管理局(FDA)批准並在美國上市的產品，用於改善與發作性睡病或阻塞性睡眠呼吸暫停相關的白天過度嗜睡的成年患者的覺醒能力。2020年1月，歐盟委員會也在歐洲批准了Sunosi。2022年8月，該公司宣佈獲得FDA的批准，並於2022年10月，Auvelity在美國商用。Auvelity被用於治療成人的主要抑鬱障礙。
商業產品
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發展計劃
當用於進一步開發的計劃時，我們將Auvelity中包含的專利右美沙芬-安非他酮製劑稱爲「AXS-05」。AXS-05是一種新型的、口服的、研究中的NMDA受體拮抗劑，具有多模式活性，正在開發中，用於治療阿爾茨海默病激動症或AD激動症和戒菸。AXS-05利用專利配方和劑量的右美沙芬和安非他酮，以及Axome的代謝抑制技術，來調節組件的傳遞。我們已經完成了AXS-05在AD激盪中的2/3階段試驗，我們稱之爲Advance-1試驗。AXS-05在Advance-1試驗中達到了主要終點。我們還完成了ACCORD-1試驗，這是一項第三階段的雙盲、安慰劑對照、隨機停藥試驗，用於AD激動症患者，我們正在進行一項開放標籤的AD激動症長期安全性研究。我們目前正在進行ADVANCE-2試驗，另一個階段3，雙盲、安慰劑對照試驗，用於AD激動症患者。我們還在進行ACCORD-2研究，這是一項對AD激動症患者進行的3期雙盲、安慰劑對照、隨機停藥試驗。在與杜克大學的研究合作下，AXS-05用於戒菸的積極第二階段試驗已經完成。
AXS-07是一種新型的口服、快速吸收、多機制的研究藥物，正在開發中，用於偏頭痛的急性治療。AXS-07由分子溶解性增強型包合物™、美洛昔康和利扎曲普坦組成。我們已經完成了AXS-07用於急性治療偏頭痛的兩個3期試驗，我們稱之爲動量試驗和攔截試驗。AXS-07在動量試驗和攔截試驗中都達到了共同的主要終點。我們還完成了AXS-07在偏頭痛患者中的一項開放標籤、長期、安全的研究，稱爲MOVE試驗。在MOVE試驗中，服用AXS-07可以迅速、實質性地緩解偏頭痛及其相關症狀，而且長期服用耐受性良好。我們目前正在進行Emerge研究，這是一項多中心、3期、單組試驗，評估AXS-07對口服降鈣素基因相關肽抑制劑反應不足的成人偏頭痛的急性治療的有效性和安全性。我們在2021年提交了針對AXS-07的新藥申請(NDA)，該申請被接受，並於2022年4月收到了FDA的完整回覆信(CRL)。CRL中給出的主要原因與化學、製造和控制或CMC考慮有關。我們向FDA重新提交了AXS-07用於偏頭痛急性治療的NDA，FDA承認並將其歸類爲2類重新提交，並將處方藥使用費法案(PDUFA)的行動目標日期定爲2025年1月31日。
AxS-12，瑞波西汀，是一種正在開發的新型口服研究藥物，用於治療發作性睡病。AxS-12是一種高度選擇性且強效的去甲腎上腺素再吸收抑制劑。AxS-12已獲得FDA孤兒藥稱號，用於治療發作性睡病。我們已經完成了AxS-12的II期試驗，我們將其稱爲CONCERT研究。我們最近完成了AxS-12治療發作性睡病的隨機、安慰劑對照3期試驗，我們將其稱爲SYMPHONY研究，其中AxS-12在減少發作的試驗中達到了主要終點。我們目前正在對AxS-12進行一項開放標籤長期安全性擴展研究。
AxS-14，esreboxextra，是一種正在開發的新型口服研究藥物，用於治療纖維肌痛。AxS-14是一種高度選擇性且強效的去甲腎上腺素再吸收抑制劑。艾瑞波西汀是瑞波西汀的SS-對映體，比外鏈瑞波西汀更有效和選擇性。我們擁有輝瑞公司的許可數據，或輝瑞公司，其中包括一項已完成的纖維肌痛II期試驗和III期試驗，兩項試驗均呈陽性。
索連菲醇是蘇諾西的活性成分。它是一種口服雙作用多巴胺和去甲腎上腺素再吸收抑制劑和TAAR 1激動劑。我們目前正在進行四項3期、雙盲、安慰劑對照、隨機多中心索林菲醇試驗：第一項是在DDD中，我們稱之爲Paradigm研究，第二項是在暴飲暴食症（BED）中，我們稱之爲ENGAGE研究，第三項是在患有注意力缺陷/多動症的成年人中，我們稱之爲FOCUS研究，第四項是在輪班工作障礙（SWD）中，我們將其稱爲SUSTAIN研究。
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此外，我們目前正在評估其他候選產品，我們打算開發這些產品來治療中樞神經系統疾病。我們的目標是成爲一家完全整合的生物製藥公司，開發和商業化差異化療法，以增加可用的治療選擇並改善患有中樞神經系統疾病的患者的生活。
我們的候選產品通過專利、商業祕密和專有技術的組合得到保護。如果獲得批准，它們還可能有資格獲得監管排他期。我們的知識產權組合包括已頒發的美國和外國專利，AXS-05的權利要求延伸到2034、2040、2041和2043，AXS-07的權利要求延伸到2038，AXS-12的權利要求延伸到2040，以及AXS-05、AXS-07、AXS-12和AXS-14的美國和外國專利申請。與Sunosi一樣，Orange Book在美國列出的專利延長至2042年。我們在其他多個國家也擁有與Sunosi相關的專利。2024年6月，我們與聯昌實驗室有限公司達成和解協議，解決了與Sunosi相關的專利訴訟，允許聯通在2042年6月30日或更早的情況下開始銷售其仿製版本的Sunosi。2024年8月，我們與Sandoz Inc.或Sandoz達成協議，駁回與Sunosi相關的專利訴訟，因此，訴訟已被無罪駁回。
自2012年1月成立以來，我們迄今爲止的業務包括組織和人員配備我們的公司、業務規劃、籌集資金、開發我們的化合物、從事其他發現和臨床前活動以及Auvelity和Sunosi的商業發佈。首次公開募股後，我們主要通過向股權投資者出售普通股和債務借款的收益爲我們的運營提供資金。如需進一步討論，請參閱下文題爲「流動性和資本資源」的部分。
我們的盈利能力取決於我們創造收入的能力。我們已開始商業銷售Auvelity和Sunosi，但我們在將這些或任何產品商業化方面的經驗有限。
自成立以來，我們發生了重大的運營和淨虧損。在截至2024年和2023年9月30日的9個月中，我們的淨虧損分別爲21230美元萬和14060美元萬。截至2024年9月30日，我們的累計赤字爲104790美元萬，我們預計將產生巨額費用和持續的運營虧損。我們預計，隨着我們繼續將目前上市的產品商業化，以及開發和臨床試驗，並尋求監管部門對我們目前的候選產品和我們開發或許可並進入臨床開發的任何其他候選產品的批准，與我們正在進行的活動相關的費用將會增加。此外，作爲一家上市公司，我們已經並將繼續承擔與運營相關的額外成本。因此，我們可能需要額外的資金來支持我們的持續運營。我們可能尋求通過公共或私人股本、債務融資或其他來源爲我們的運營提供資金。我們可能無法以可接受的條款獲得足夠的額外融資，或者根本不能。我們未能在需要時籌集資金，將對我們的財務狀況和我們實施業務戰略的能力產生負面影響。我們將需要創造可觀的收入來實現盈利，而且我們可能永遠不會做到這一點。
今年迄今爲止和最近的發展
AxS-05
2024年5月，我們宣佈啓動ACCORD-2研究，這是一項3期、雙盲、安慰劑對照、隨機戒斷試驗，旨在評估AxS-05治療AD激越的有效性和安全性。
AxS-07
2024年9月，我們宣佈FDA確認我們重新提交了用於急性治療偏頭痛的AX-07的NDA。FDA將重新提交指定爲2類重新提交，並將PDUFA行動目標日期設定爲2025年1月31日。
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AxS-12
2024年3月，我們報告了AxS-12治療發作性睡病的3期試驗（我們稱之爲SYMPHONY研究）的陽性頂線數據。
索連菲醇
2024年3月，我們宣佈啓動PARADIGm研究，這是一項關於索林菲醇治療DDD的3期、隨機、雙盲、安慰劑對照、多中心試驗。
2024年4月，我們宣佈啓動ENGAGE研究，這是一項關於索林菲醇治療BED的3期、隨機、雙盲、安慰劑對照、多中心試驗。
2024年8月，我們宣佈啓動SUSTAIN研究，這是一項關於索林菲醇治療SWD的3期、隨機、雙盲、安慰劑對照、多中心試驗。
財務概述
收入
截至2024年9月30日和2023年9月30日的三個月，我們的產品銷售淨收入分別爲10370萬美元和5710萬美元，截至2024年和2023年9月30日的九個月，我們的產品銷售淨收入分別爲26440萬美元和13170萬美元。
我們預計Auvelity和Sunosi的收入可能會根據季度需求而波動。除非我們成功開發當前或未來的候選產品之一、獲得監管機構批准並商業化，否則我們不會從其他產品中產生收入。自成立以來，我們已經遭受了巨額運營損失。如果我們未能及時完成候選產品的開發或獲得監管機構的批准，我們從此類候選產品中產生未來收入的能力以及我們的運營業績和財務狀況將受到重大不利影響。如果我們達成許可或合作安排，此類協議未來可能會或不會產生收入。
此外，2023年第一季度，我們通過許可協議（定義如下）記錄了與Pharacco相關的許可收入6570萬美元。更多詳細信息請參閱下文。
與Pharmanovia達成許可協議
2023年2月，我們通過與Pharacco簽訂了Pharacco許可協議，以在該地區商業化和進一步開發Sunosi®。Pharacevia是一家總部位於英國的全球生命週期管理醫療保健公司，專注於四個核心治療領域--腫瘤學、內分泌學、神經病學和心血管學。
2023年第一季度，我們收到了6200萬歐元（6570萬美元）的預付款，並有資格收到總額高達9450萬歐元的基於銷售的付款和其他里程碑付款。我們將在20世紀20年代中期就該地區的許可產品（如《Pharican》通過許可協議的定義）的淨銷售額獲得特許權使用費百分比。我們在截至2024年9月30日和2023年9月30日的三個月內分別確認了100萬美元和70萬美元的特許權使用費收入，在截至2024年和2023年9月30日的九個月內確認了260萬美元和160萬美元的特許權使用費收入，這些收入與Pharacco via出售Sunosi有關。
38
收入成本
收入成本包括配製、生產和包裝藥品的直接成本、間接成本（包括勞動力、海關、庫存補償、運輸、外部庫存管理、特許權使用費和其他雜項運營成本）。
研發費用
研發費用主要包括臨床前研究、臨床試驗、製造成本、與員工相關的費用（包括工資、福利、差旅和股票補償費用）、合同服務，包括根據與第三方（例如合同研究組織或CROs）的安排產生的外部研發費用、設施成本、管理費用、折舊和其他相關成本。
研發活動是我們商業模式的核心。爲了爲我們的任何候選產品提交新藥申請或NDA，我們已經並將產生超出我們目前和計劃中的臨床試驗的巨額成本。很難確定我們目前或未來的臨床試驗和臨床前研究的成本和持續時間，或者如果我們獲得監管部門的批准，我們將從Auvelity和Sunosi或我們的候選產品的商業化和銷售中獲得多大程度的收入。我們候選產品的臨床試驗和開發的持續時間、成本和時間將取決於各種因素，包括未來臨床試驗和臨床前研究的不確定性、臨床試驗註冊率的不確定性以及重大和不斷變化的政府監管。此外，每種候選產品的成功概率將取決於許多因素，包括競爭、製造能力和商業可行性。我們將根據每個候選產品在科學和臨床上的成功，以及對每個候選產品的商業潛力的評估，確定要追求哪些項目，以及爲每個項目提供多少資金。
管理層在制定編制這些財務報表時使用的估計和假設時考慮了許多因素。管理層必須在此過程中做出重大判斷。此外，其他因素可能會影響估計，包括預期的業務和運營變化、與制定估計時使用的假設相關的敏感性和波動性，以及歷史趨勢是否預計代表未來趨勢。估計過程通常可能會對最終未來結果產生一系列潛在合理的估計，管理層必須選擇在該合理估計範圍內的金額。如果實際結果與歷史經驗不同，或者其他假設實際結果並非基本準確，即使這些假設在做出時是合理的，則此過程可能會導致實際結果與編制財務報表時使用的估計金額存在重大差異。
銷售、一般和管理費用
銷售、一般和行政費用主要包括執行、商業、財務和運營職能人員的工資和相關成本，包括基於股票的薪酬和差旅費。銷售、一般和管理費用還包括營銷成本、其他商業成本、商業化前成本、設施相關成本、保險費用、法律和會計服務的專業費用以及專利申請和起訴費用。銷售、一般和行政費用在發生時列爲費用。
利息支出，淨額
淨利息費用主要包括與我們的定期貸款相關的現金利息和非現金成本（進一步討論，請參閱下文「流動性和資本資源」）。我們在債務協議期限內將這些成本攤銷爲綜合運營報表中的利息費用。利息支出，淨額還包括現金賺取的利息收入。
39
關鍵會計政策與重大判斷和估計
對我們財務狀況和經營業績的討論和分析基於我們的綜合財務報表，這些報表是根據美利堅合衆國公認會計原則或美國公認會計原則編制的。編制該等綜合財務報表要求我們做出影響資產和負債的報告金額、綜合財務報表日期或有資產和負債的披露以及報告期內的報告費用金額的估計和假設。根據GAAP，我們的估計基於歷史經驗和我們認爲在當時情況下合理的各種其他假設。在不同的假設或條件下，實際結果可能與這些估計不同。
除與許可協議相關的收入確認外，我們2023年10-k表格年度報告中披露的關鍵會計政策沒有重大變化。我們的關鍵會計政策在本季度報告10-Q表格其他地方出現的合併財務報表註釋中進行了描述。
經營成果
下表彙總了我們在所示期間的業務成果(以千計)：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
收入： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
產品銷售，淨額 |
|
$ |
103,736 |
|
|
$ |
57,127 |
|
|
$ |
264,352 |
|
|
$ |
131,713 |
|
許可證收入 |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
65,735 |
|
使用費收入 |
|
|
1,026 |
|
|
|
667 |
|
|
|
2,575 |
|
|
|
1,622 |
|
總收入 |
|
|
104,762 |
|
|
|
57,794 |
|
|
|
266,927 |
|
|
|
199,070 |
|
運營費用： |
|
|
|
|
|
|
|
|
|
|
|
|
||||
收入成本（不包括攤銷和折舊） |
|
|
8,437 |
|
|
|
6,532 |
|
|
|
22,789 |
|
|
|
18,687 |
|
研發 |
|
|
45,388 |
|
|
|
28,767 |
|
|
|
132,071 |
|
|
|
67,141 |
|
銷售，一般和行政 |
|
|
95,564 |
|
|
|
83,188 |
|
|
|
298,088 |
|
|
|
236,314 |
|
或有對價公允價值的損失（收益） |
|
|
16,391 |
|
|
|
(180 |
) |
|
|
17,139 |
|
|
|
5,711 |
|
無形資產攤銷 |
|
|
1,606 |
|
|
|
1,607 |
|
|
|
4,785 |
|
|
|
4,768 |
|
總運營支出 |
|
|
167,386 |
|
|
|
119,914 |
|
|
|
474,872 |
|
|
|
332,621 |
|
運營虧損 |
|
|
(62,624 |
) |
|
|
(62,120 |
) |
|
|
(207,945 |
) |
|
|
(133,551 |
) |
利息支出，淨額 |
|
|
(1,978 |
) |
|
|
(757 |
) |
|
|
(4,359 |
) |
|
|
(5,751 |
) |
所得稅前虧損 |
|
|
(64,602 |
) |
|
|
(62,877 |
) |
|
|
(212,304 |
) |
|
|
(139,302 |
) |
所得稅優惠(費用) |
|
|
— |
|
|
|
678 |
|
|
|
— |
|
|
|
(1,285 |
) |
淨虧損 |
|
$ |
(64,602 |
) |
|
$ |
(62,199 |
) |
|
$ |
(212,304 |
) |
|
$ |
(140,587 |
) |
每股普通股基本虧損和攤薄後淨虧損 |
|
$ |
(1.34 |
) |
|
$ |
(1.32 |
) |
|
$ |
(4.45 |
) |
|
$ |
(3.14 |
) |
加權平均已發行普通股、基本普通股和稀釋後普通股 |
|
|
48,140,519 |
|
|
|
47,117,196 |
|
|
|
47,703,508 |
|
|
|
44,783,380 |
|
產品銷售，淨。 截至2024年9月30日的三個月和九個月，Auvelity美國淨銷售額分別爲8040萬美元和19880萬美元，而2023年同期爲3770萬美元和8100萬美元。截至2024年9月30日的三個月和九個月，Sunosi的淨銷售額分別爲2340萬美元和6560萬美元，而2023年同期的淨銷售額分別爲1940萬美元和5070萬美元。兩個可比期間的增長主要是由於Auvelity和Sunosi的單位銷量增加。
40
下表總結了截至2024年9月30日的九個月我們的銷售津貼和儲備金活動（以千計）：
|
|
商業折扣和回扣、退貨和其他 |
|
|
現金折扣和退款 |
|
|
醫療補助和醫療保險回扣 |
|
|
總 |
|
||||
2023年12月31日餘額 |
|
$ |
37,492 |
|
|
$ |
12,501 |
|
|
$ |
9,222 |
|
|
$ |
59,215 |
|
規定 |
|
|
172,040 |
|
|
|
69,227 |
|
|
|
26,737 |
|
|
|
268,004 |
|
付款/貸方 |
|
|
(160,825 |
) |
|
|
(67,429 |
) |
|
|
(15,902 |
) |
|
|
(244,156 |
) |
2024年9月30日餘額 |
|
$ |
48,707 |
|
|
$ |
14,299 |
|
|
$ |
20,057 |
|
|
$ |
83,063 |
|
許可證收入。 2023年2月，我們通過許可協議簽訂了Pharacco，將Sunosi在某些前美國市場商業化。2023年第一季度，我們將Pharacco via的6570萬美元預付款確認爲許可收入。截至2024年9月30日的九個月內，我們沒有許可收入。
版稅收入。 關於2023年2月通過許可協議在某些美國以外市場商業化Sunosi的Pharacco，我們在截至2024年9月30日的三個月和九個月內分別確認了100萬美元和260萬美元的特許權使用費收入，相比之下，2023年同期，Pharacco通過在許可外市場銷售Sunosi的收入分別爲70萬美元和160萬美元。
收入成本。 截至2024年9月30日的三個月和九個月的收入成本分別爲840萬美元和2280萬美元，而2023年同期的收入成本爲650萬美元和1870萬美元。兩個可比期間的增長與Auvelity和Sunosi銷售額的增長一致。截至2023年9月30日止九個月的收入成本包括通過許可協議與Pharacco相關的500萬美元許可內收入分成費用。
研究和開發。 下表總結了截至2024年和2023年9月30日的三個月和九個月我們主要產品的研發費用（單位：千）：
|
|
截至9月30日的三個月裏， |
|
|
截至9月30日的9個月， |
|
||||||||||
|
|
2024 |
|
|
2023 |
|
|
2024 |
|
|
2023 |
|
||||
索連菲醇 |
|
$ |
13,711 |
|
|
$ |
7,317 |
|
|
$ |
37,908 |
|
|
$ |
11,903 |
|
AxS-05 |
|
|
14,802 |
|
|
|
8,964 |
|
|
|
44,090 |
|
|
|
23,967 |
|
AxS-07 |
|
|
2,512 |
|
|
|
1,764 |
|
|
|
9,457 |
|
|
|
4,869 |
|
AxS-12 |
|
|
1,911 |
|
|
|
2,503 |
|
|
|
7,035 |
|
|
|
7,364 |
|
AxS-14 |
|
|
3,516 |
|
|
|
2,364 |
|
|
|
9,886 |
|
|
|
5,062 |
|
其他研究與開發（*） |
|
|
3,067 |
|
|
|
1,826 |
|
|
|
8,186 |
|
|
|
4,295 |
|
股票補償 |
|
|
5,869 |
|
|
|
4,029 |
|
|
|
15,509 |
|
|
|
9,681 |
|
研發費用總額 |
|
$ |
45,388 |
|
|
$ |
28,767 |
|
|
$ |
132,071 |
|
|
$ |
67,141 |
|
（*）其他研發費用主要包括與其他候選產品相關的成本、設施和管理費用
與2023年同期相比，截至2024年9月30日的三個月和九個月的研發費用分別增加了1660萬美元和6500萬美元。兩個可比時期的增加主要與針對其他適應症的III期試驗有關，包括針對索利芬醇的ADHD、DDD、BED和SWD研究、正在進行的AX-05和AX-12 3期試驗的進展、AX-07和AX-14的製造成本上升以及組織增長導致的人員成本上升。我們預計，隨着現有試驗即將完成，而新試驗即將啓動，研發成本將穩定在當前水平。
41
銷售、一般和行政。 截至2024年9月30日的三個月和九個月，銷售、一般和行政費用分別爲9560萬美元和29810萬美元，而2023年同期爲8320萬美元和23630萬美元。兩個可比時期的增長主要與Auvelity和Sunosi的商業活動增加以及與組織增長相關的人員成本增加有關，包括非現金股票薪酬。隨着我們擴大Auvelity、Sunosi以及潛在未來產品發佈的營銷、促銷和廣告成本，我們預計銷售、一般和行政費用將會增加。
或有對價的公允價值損失。 截至2024年9月30日的三個月和九個月的虧損頭寸分別爲1640萬美元和1710萬美元，而2023年同期的收益頭寸分別爲20萬美元和570萬美元的虧損頭寸與重大不可觀察輸入（包括貼現率）和重大假設（包括未來銷售估計）的變化有關。
無形資產攤銷.我們在10年的使用壽命內攤銷無形資產（我們將其確認爲收購的一部分）。截至2024年9月30日和2023年9月30日的三個月無形資產攤銷均爲160萬美元，截至2024年9月30日和2023年9月30日的九個月無形資產攤銷均爲480萬美元。
利息費用，淨額。 截至2024年9月30日的三個月和九個月，淨利息費用分別爲200萬美元和440萬美元，而2023年同期爲80萬美元和580萬美元。截至可比期間三個月的增長主要是由於現金餘額減少的利息收入減少。截至同期的九個月減少主要是由於現金餘額增加帶來的利息收入增加，部分抵消了與貸款協議相關的利息費用、發行成本和費用。
所得稅支出。 我們沒有記錄截至2024年9月30日的三個月和九個月的所得稅費用，原因是本季度所有司法管轄區均出現了虧損，而由於我們的遞延所得稅資產的全額估值備抵狀況，我們沒有從中受益。截至2023年9月30日的三個月，我們記錄了70萬美元的稅收優惠，截至2023年9月30日的九個月，我們記錄了130萬美元的稅收費用，這是由於在馬耳他賺取的收入與通過許可協議從Pharaman確認的許可收入有關。
淨虧損。截至2024年9月30日的三個月和九個月的淨虧損分別爲6，460萬美元和21，230萬美元，而2023年同期的淨虧損分別爲6，220萬美元和14，060萬美元。兩個可比時期的增長主要是由於臨床前和正在進行的臨床試驗費用中的研發支出增加、與Auvelity和Sunosi相關的商業活動的銷售、一般和管理費用增加，包括銷售人員和營銷支出，以及由於組織增長而導致的人員成本增加，包括非現金股票補償費用。此外，截至2024年9月30日的九個月淨虧損與2023年同期相比有所增加，這受到2023年第一季度從Pharacco via收到的預付款6570萬美元的影響。
流動性與資本資源
自成立以來至2024年9月30日，我們主要通過股權發行、債務借款和產品銷售收益爲運營提供資金。請參閱下面的討論。
2022年12月2日，我們向美國證券交易委員會提交了一份自動貨架登記聲明，用於發行普通股、優先股、配股、債務證券和不超過限額的單位，我們稱之爲2022年貨架登記聲明。美國證券交易委員會在提交文件後宣佈生效。未來，我們可能會利用2022年貨架登記聲明對其中一種或多種證券進行額外發行，其金額、價格和條款將在證券發行時公佈。當我們2022年貨架登記聲明涵蓋的任何證券發售時，將準備招股說明書補充文件並向SEC提交，其中包含有關任何此類發行條款的具體信息。
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2019年12月，我們與SVb Securities LLC簽訂了銷售協議或2019年12月銷售協議（現稱爲Leerink Partners LLC）或Leerink，根據該規定，我們可以通過Leerink（作爲我們的銷售代理）不時出售高達8000萬美元的普通股，利用我們於2019年12月5日向SEC提交的自動貨架登記聲明，在一項或多項市場發行中發行普通股、優先股、配股、權利，債務證券和金額不限的單位，我們稱其爲2019年貨架登記聲明。Leerink有權就根據2019年12月銷售協議出售的任何股份收取總收益3.0%的佣金。
2022年3月，我們與Leerink簽訂了銷售協議或2022年3月銷售協議，並提交了招股說明書補充，根據該補充說明書，我們可以通過Leerink（作爲我們的銷售代理）不時出售高達20000萬美元的普通股，利用2019年貨架登記聲明在一項或多項市場上出售。Leerink有權就根據2022年3月銷售協議出售的任何股份收取高達總收益3.0%的佣金。2022年3月的銷售協議取代了我們與Leerink之間的2019年12月的銷售協議。根據之前的市場發售計劃，我們耗盡了普通股的銷售。
2022年8月，我們提交了對2019年貨架登記聲明的招股說明書補充，以額外發行和出售高達25000萬美元的普通股股票（如果有的話）。Leerink有權就根據2022年3月銷售協議出售的任何股份收取高達總收益3.0%的佣金。
2022年12月，針對2022年貨架登記聲明，我們提交了新的銷售協議招股說明書，以取代之前於2022年8月提交的與過期的2019年貨架登記聲明相關的招股說明書補充。新的銷售協議招股說明書涵蓋了我們根據2022年3月的銷售協議發行和銷售的價值高達25000萬美元的普通股，這些普通股可能通過Leerink作爲銷售代理不時發行和銷售。
根據2022年3月的銷售協議，在截至2024年9月30日的九個月內，我們通過出售297，135股股份獲得了約2440萬美元的總收益，其中淨收益約爲2390萬美元。截至2024年3月31日的三個月內，我們沒有利用與Leerink的2022年3月銷售協議。截至2023年12月31日止年度，我們也沒有利用與Leerink的2022年3月銷售協議。
2023年1月，我們與大力神簽訂了貸款協議第三修正案，即第三修正案。第三修正案將定期貸款墊款的金額(定義見貸款協議)提高至35000美元萬，降低利率，並延長貸款協議的到期日和僅限利息的期限。2024年9月，我們與大力神簽訂了貸款協議第五修正案，或稱第五修正案。第五修正案修訂了貸款協議的條款，其中包括：(I)將2020年定期貸款(定義如下)第3批的本金總額從75.0億美元增加到8000美元萬；(Ii)延長2020年定期貸款某些部分的可用期；(Iii)更改貸款協議中包含的業績契約的條款，並增加新的業績契約；(Iv)有條件地免除安盛市值超過15美元億期間的最低現金要求；以及(V)允許安盛馬耳他有限公司或馬耳他子公司向貸款人申請一定數額的預付款(定義見貸款協議)，前提是安盛可要求預付款，並增加馬耳他子公司在美國境外持有的現金金額，這在第五修正案中有更詳細的規定。本公司動用了2020年定期貸款的1C部分，截至2024年9月30日，本公司在2020年定期貸款下尚有約18000美元的未償還萬和15000美元的萬。有關更多信息，請參閱下面的「合同義務和承諾--2023年1月貸款和擔保協議第三修正案--大力神」和「合同義務和承諾--2024年9月貸款和擔保協議第五修正案--大力神」部分。
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2023年6月，我們完成了普通股的承銷公開發行，並以每股75.00美元的公開發行價格出售了3億股普通股。淨收益爲21130萬美元，扣除承保折扣和佣金1350萬美元以及其他發行成本20萬美元。此外，與此次公開發行有關，承銷商於2023年7月完全行使了選擇權，以每股75.00美元的公開發行價格購買了我們額外的450，000股普通股。扣除承保折扣和2億美元佣金以及其他最低發行成本，淨收益爲3170萬美元。
未來，我們可能會對2022年貨架登記聲明涵蓋的一種或多種證券進行額外發行，其金額、價格和條款將在證券發行時公佈。當我們2022年貨架登記聲明涵蓋的任何證券發售時，將準備招股說明書補充文件並向SEC提交，其中包含有關任何此類發行條款的具體信息。
我們相信，根據當前的運營計劃，我們當前的現金足以爲預期的運營提供資金，使其成爲現金流正向。由於臨床試驗中產品商業化和評估候選產品的過程成本高昂，而且這些試驗的進展時間也不確定，因此我們做出這一估計的假設可能被證明是錯誤的，我們可以比目前預期更早地使用我們的資本資源。
現金流
下表總結了所示期間我們的主要來源和現金用途（以千計）：
|
|
截至9月30日的9個月， |
|
|||||
|
|
2024 |
|
|
2023 |
|
||
提供的現金淨額(用於)： |
|
|
|
|
|
|
||
經營活動 |
|
$ |
(102,208 |
) |
|
$ |
(114,718 |
) |
投資活動 |
|
|
(240 |
) |
|
|
(575 |
) |
融資活動 |
|
|
43,596 |
|
|
|
331,015 |
|
現金淨增(減) |
|
$ |
(58,852 |
) |
|
$ |
215,722 |
|
經營活動。 截至2024年9月30日止九個月的經營活動使用的現金爲10220萬美元，而截至2023年9月30日止九個月的現金爲11470萬美元。減少1250萬美元的影響是2023年第一季度從Pharacco收到的預付款6570萬美元以及2024年Auvelity和Sunosi淨產品收入增加，但這被2024年商業和臨床活動中使用的現金增加所抵消。
投資活動。 截至2024年9月30日止九個月投資活動使用的現金爲24萬美元，而截至2023年9月30日止九個月爲57.5萬美元。我們在兩個可比期間的投資活動主要涉及購買與商業和臨床活動相關的設備。
融資活動。在截至2024年9月30日的9個月中，融資活動提供的現金爲4,360萬，其中包括爲融資目的發行普通股的淨收益2,390美元萬，以及根據員工股票期權和特別提款權發行普通股的2,950美元萬收益，這些收益被或有對價支付和股票獎勵預扣稅部分抵消，總計920美元萬。截至2023年9月30日的9個月，融資活動提供的現金爲33100萬，其中包括與2023年6月公開發售相關的21130美元萬的淨收益和由於承銷商充分行使其購買額外股份的選擇權而額外的3 170萬淨收益，與提取與大力神的貸款協議部分有關的8 270萬淨收益，以及因行使員工股票期權而發行普通股的11 20萬收益，被或有對價的支付和股票獎勵的預扣稅抵消，總計600萬。
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資金需求
自成立以來，我們尚未實現盈利，隨着我們繼續開發候選產品並尋求監管機構批准，並開始商業化任何額外批准的產品，同時進一步投資Auvelity和Sunosi，我們預計將繼續虧損。我們面臨與新候選產品開發相關的所有風險，我們可能會遇到不可預見的費用、困難、併發症、延誤和其他可能損害我們業務的未知因素。
我們未來可能需要籌集額外融資來資助我們的運營。如果我們需要額外的融資，我們可能會產生額外的債務、許可某些知識產權，並尋求出售可能導致股東稀釋的額外股權或可轉換證券。如果我們通過發行股票或可轉換證券籌集額外資金，這些證券可能擁有優先於我們普通股的權利或優先權，並且可能包含限制我們運營的契約。無法保證我們能夠以我們可以接受的條款獲得額外的股權或債務融資（如果有的話）。我們未來的資本要求將取決於許多因素，包括：
請參閱「風險因素」，了解與我們巨額資本要求相關的額外風險。
合同義務和承諾
與輝瑞的許可協議
2020年1月，我們與輝瑞簽訂了許可協議。根據我們與輝瑞的獨家許可協議的條款，輝瑞收到了82，019股我們普通股，掛牌價值爲800萬美元，基於我們普通股前十個交易日的平均收盤價97.54美元，作爲許可和權利的對價。輝瑞還收到了300萬美元的預付現金付款。根據該日我們普通股的收盤價，我們確定2020年1月9日收盤日授予輝瑞的每股普通股的公允價值爲87.24美元。因此，發行股票的公允價值爲720萬美元。
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在實現某些監管和銷售里程碑後，輝瑞還可以獲得高達32300萬美元的資金，並對任何此類已批准的含有化合物的臨床產品的未來銷售獲得從個位數到低兩位數的分層特許權使用費。輝瑞還將有權就涉及AX-12和AX-14的任何潛在戰略交易進行首次談判。
與Antecip Bioventures的許可協議
根據與Antecip Bioventures II LLC（或Antecip）簽訂的三項獨家許可協議，Antecip是我們首席執行官兼董事會主席Herriot Tabuteau.D.擁有的實體，我們有義務對包含AxS-02、AxS-05和AxS-04許可技術的產品的淨銷售額支付1.5%至4.5%的指定特許使用費，但根據向第三方支付的費用最多可減少50%。
就貸款協議（見下文）而言，Antecip同意根據與我們和Hercules的直接協議轉讓其中一項許可協議的抵押品。
與Hercules Capital，Inc.的貸款和擔保協議
本文中使用但未另行定義的大寫術語應具有貸款協議中賦予的含義。
2024年9月貸款和擔保協議第五修正案
2024年9月30日，我們簽署了第五修正案。第五修正案修改了貸款協議的條款，除其他事項外：(1)將第3檔的承諾額從75美元增加到8,000美元萬；(2)如第五修正案中更詳細地規定，將第1 D檔的可用期延長至2025年6月15日，將第1 E檔的可用期延長至2025年12月15日；(3)修改履約公約A、履約公約b和履約公約C的條款，並增加履約公約D，如第五修正案中更詳細地規定的那樣；(Iv)在本公司市值超過$15億期間，有條件地豁免要求本公司維持大於或等於$3000萬加合格現金應付金額之和的合格現金；及(V)允許馬耳他附屬公司向貸款人申請墊款，最多可達至本公司可要求墊付金額的某一數額，並增加子公司馬耳他可持有的美國境外現金金額，詳情載於第五修正案。
2023年5月貸款和擔保協議第四修正案
2023年5月8日，我們與Hercules（作爲行政代理人和抵押代理人）以及貸方簽訂了貸款協議的豁免和第四修正案（即第四修正案）。第四修正案將第四修正案結束後45天內馬耳他子公司在美國境外可持有的現金金額從300萬美元增加到1500萬美元，此後增加到1000萬美元。第四修正案還免除了馬耳他子公司在第四修正案日期之前持有的現金金額的任何所謂違約。2023年8月，Hercules授予Axsome對第四修正案的豁免，允許馬耳他子公司在美國境外持有不超過1250萬美元的現金，直至2023年12月31日。
2023年1月貸款和擔保協議第三修正案
2023年1月9日，我們加入了第三修正案。
第三修正案修改了貸款協議的條款，除其他事項外：
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版稅協議
根據日期爲2022年3月25日的資產購買協議或購買協議，我們同意向Jazz支付不可退還、不可抵免的特許權使用費，相當於（A）任何當前指示的高個位數特許權使用費或（B）任何未來指示的中個位數特許權使用費，適用特許權使用費期限內在美國領土內淨銷售額（在每種情況下，這些條款在購買協議中定義）。Jazz在美國領土以外的淨銷售額無需支付特許權使用費。
在最初的收盤時，我們承擔了Jazz對Sk和Aerial Bizerma，LLC或Aerial的所有承諾。Sk是Sunosi的創始人，在包括中國、韓國和日本在內的12個亞洲市場保留版權。2014年，Jazz從Aerial手中收購了Sunosi的全球版權，不包括之前提到的亞洲市場。對Sk和Aerial的假設承諾包括基於Sunosi銷售額的個位數分層特許權使用費，我們承諾根據收入里程碑支付高達16500萬美元，根據開發里程碑支付高達100萬美元。
世貿中心租賃
2023年2月21日，我們與Advance Magazine Publishers d/b/a Conde Nast簽訂了紐約州紐約One Word Trade Center整個二十二層的分包合同或分包合同。該公司將該空間用作其公司和行政辦公室。該分包合同於2023年4月7日開始，爲期十（10）年。公司在向轉房東支付費用後，可以一次性選擇在第五週年時終止該分包。公司負責分包合同下的基本租金和某些額外的習慣可變成本，例如建築稅和運營費用的可分配部分。就分包而言，公司從分房東處獲得了一定的租金和工作優惠。
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員工與人力資本管理
截至2024年11月5日，我們擁有607名全職員工。我們的員工中沒有一個是由集體談判協議代表的，我們從未經歷過任何停工。我們相信，我們與員工保持着良好的關係。我們的員工技能很高，許多人擁有高級學位。我們的許多員工都有藥物商業化或開發的經驗。我們未來的業績在很大程度上取決於我們關鍵的科學、技術和高級管理人員的持續服務，以及我們吸引和留住高技能員工的持續能力。我們爲我們的員工提供有競爭力的工資和獎金，擁有股權的機會，支持持續學習和增長的發展計劃，以及促進他們生活方方面面福祉的強勁就業方案。除了工資外，這些計劃還包括潛在的年度可自由支配獎金、股票獎勵、醫療和保險福利、健康儲蓄和靈活支出帳戶、帶薪休假、探親假和靈活工作時間安排，以及其他福利。我們可能會根據所有適當的政府法規，採取我們認爲最符合員工利益的進一步行動。
表外安排
根據適用的SEC法規的定義，我們在所列期間沒有、目前也沒有任何表外安排。
近期會計公告
請參閱注2。我們合併財務報表的重要會計政策摘要包含在本季度報告10-Q表格第一部分財務信息第1項財務報表中，以討論最近發佈的會計公告。
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項目3.量化和假設關於市場風險的有效披露
利率風險
我們在正常業務過程中面臨市場風險。這些市場風險主要限於利率波動。截至2024年9月30日和2023年12月31日，我們的現金分別爲32730萬美元和38620萬美元。我們投資活動的主要目標是保護本金和流動性，同時在不顯着增加風險的情況下最大化收入。我們不會出於交易或投機目的進行投資。由於我們的投資組合和債務協議的短期性質（分別使用短期利率和最優惠利率），我們認爲利率立即上升100個點子不會對我們投資組合的公平市場價值產生重大影響，因此，我們預計我們的經營業績或現金流不會受到市場利率突然變化的重大影響。
外幣兌換風險
我們與位於歐洲的供應商和第三方製造商簽訂合同，某些發票以外幣計價。Pharacco via的版稅收入來自其在美國以外市場銷售Sunosi，這些銷售以歐元計價。因此，我們會受到與這些協議相關的歐元、瑞士法郎和英鎊外幣匯率波動的影響，並在我們的運營報表中確認外匯損益。我們歷史上沒有對沖外幣匯率風險。迄今爲止，我們尚未因外幣變化對這些交易產生任何重大影響。
我們認爲2024年9月30日這些貨幣發生10%的變化不會對我們的經營業績或財務狀況產生重大影響。
通貨膨脹風險
通貨膨脹通常通過增加勞動力成本和合同定價來影響我們。我們認爲，截至2024年9月30日的九個月內，通貨膨脹對我們的業務、財務狀況或經營業績沒有產生重大影響。
第四項。 控制 和程序
披露控制和程序。在首席執行官和首席財務官的參與下，我們的管理層在10-Q表格季度報告所涵蓋的期間結束時評估了我們的披露控制和程序的有效性。基於這一評估，我們的首席執行官和首席財務官得出結論，截至目前，我們的披露控制和程序有效地確保了我們根據1934年證券交易法(經修訂)或交易法提交的報告中要求披露的信息在美國證券交易委員會規則和表格中指定的時間段內得到記錄、處理、彙總和報告。披露控制和程序包括但不限於控制和程序，旨在確保我們根據交易所法案提交或提交的報告中要求披露的信息得到積累，並酌情傳達給我們的管理層，包括我們的首席執行官和首席財務官，以便及時做出有關要求披露的決定。
財務報告內部控制的變化。 截至2024年9月30日的季度，財務報告內部控制沒有發生對我們的財務報告內部控制產生重大影響或合理可能產生重大影響的變化。
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第二部分：其他R信息
項目1.法律 法律程序。
除本文所述外，我們及其子公司目前不是任何重大未決法律訴訟的當事人，我們的財產目前也不是任何重大未決法律訴訟的主題;然而，我們也可能捲入日常業務過程中產生的各種索賠和法律訴訟。
證券集體訴訟
2022年5月13日，Evy Gru提交了一份可能的集體訴訟，標題爲Gru訴Axome治療公司等人。在美國紐約南區地區法院，或SDNY地區法院，針對公司及其某些現任和前任高級管理人員和一家董事，我們稱之爲證券集體訴訟。起訴書根據《交易法》第10(B)和20(A)條及其頒佈的第100條AXS.5提出索賠，並指控被告就公司的化學制造和控制做法以及就其候選產品億-07向美國食品和藥物管理局提交的保密協議做出了虛假陳述和遺漏。被點名的原告要求未指明的損害賠償、費用、利息和費用。2022年8月11日，SDNY地區法院任命了證券集體訴訟的聯合牽頭原告，其中一人後來退出。2022年10月7日，證券集體訴訟原告提交了一份修改後的起訴書，其中包含的指控與最初的起訴書基本相似。2023年9月25日，SDNY地區法院批准了被告駁回修改後的起訴書的動議。
2023年10月13日，原告律師提交了一封信，尋求允許提出修改後的投訴並替換新原告，但被告反對。紐約州地方法院重新啓動了主要原告的任命程序。托馬斯·吉布林、保羅·伯傑和保羅·薩瑟蘭共同任命爲替代原告。2024年1月22日，紐約州紐約州地方法院批准了該動議，並下令將案件名稱更改爲In re Axsome Therapeutics，Inc.證券訴訟。2024年1月26日，替代原告再次請求允許提交擬議的第二次修訂投訴，並於2024年2月6日，紐約州紐約地方法院批准了該請求。原告於2024年2月7日提交了第二次修訂投訴。2024年3月11日，被告提出駁回第二次修訂投訴。
股東派生訴訟
2022年7月21日，Daniel·恩格爾提起股東派生訴訟，標題爲恩格爾訴赫里奧特·塔布託等人案。在SDNY地區法院起訴本公司的現任董事、本公司的若干現任和前任高級管理人員以及本公司(名義上的被告)。2023年1月27日，凱爾·古特巴提交了一份名爲古特巴訴塔布託等人的股東衍生品起訴書。在SDNY地區法院起訴本公司的現任董事、本公司的若干現任和前任高級管理人員以及本公司(名義上的被告)。衍生品投訴源於與證券集體訴訟中的指控類似的指控。原告對所有被告提出違反受託責任的索賠，並就違反《交易法》第10(B)和21D條的責任提出索賠。原告尋求未指明的損害賠償、費用、利息和成本，以及公司治理變化。Engel和Guterba事件於2023年2月28日合併，目前仍在等待證券集體訴訟的進一步程序。
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第四條訴訟
2023年3月24日，我們開始對Teva PharmPharmticals，Inc.或Teva提起專利侵權訴訟，涉及Teva的Auvelity縮寫新藥申請(ANDA)。此操作的標題爲Axome治療公司和Antecip BioVentures II LLC訴Teva製藥公司。編號2：23-CV-01695在美國新澤西州地區法院或新澤西州地區法院。2023年12月15日，我們開始了對Teva的第二次專利侵權訴訟，涉及Teva的ANDA。此操作的標題爲Axome治療公司和Antecip BioVentures II LLC訴Teva製藥公司。新澤西州地區法院第2號：23-cv-23142。2024年2月26日，新澤西州地區法院合併了這些訴訟。事實發現目前計劃在綜合行動中於2025年2月7日結束。2024年5月28日，我們開始了對Teva的第三次專利侵權訴訟，涉及Teva的ANDA。此操作的標題爲Axome治療公司和Antecip BioVentures II LLC訴Teva製藥公司。第2號：24-cv-06489，新澤西州地區法院。2024年9月30日，我們開始了對Teva的第四次專利侵權訴訟，涉及Teva的ANDA。此操作的標題爲Axome治療公司和Antecip BioVentures II LLC訴Teva製藥公司。新澤西州地區法院第2-24-cv-09535號。所有操作目前都處於待定狀態。
Sunosi第四條訴訟
2023年9月13日，我們開始對Hikma和其他五家制藥公司提起專利侵權訴訟，涉及每個被告對Sunosi的ANDA。此操作的標題爲Axome馬耳他有限公司和Axome治療公司訴Alkem實驗室有限公司等人案。編號2：23-CV-20354，新澤西州地區法院。我們於2023年12月20日、2024年1月11日、2024年1月18日、2024年2月14日、2024年3月19日(提起2起訴訟)、2024年4月5日、2024年7月2日、2024年8月8日、2024年8月21日和2024年9月16日對被告提起相關專利侵權訴訟。這些操作的標題爲Axome馬耳他有限公司和Axome治療公司訴Unicem實驗室有限公司。2號：23-cv-23255；Axome馬耳他有限公司和Axome治療公司訴Hetero USA公司等人案。編號2：24-cv-00196；Axome馬耳他有限公司和Axome治療公司訴Aurobindo Pharma USA公司等人案。2號：24-cv-00309；Axome馬耳他有限公司和Axome治療公司訴Sandoz公司。2號：24-cv-00860；Axome馬耳他有限公司和Axome治療公司訴Hetero USA公司等人案。編號2：24-cv-03999；Axome馬耳他有限公司和Axome治療公司訴Aurobindo Pharma USA公司等人案。2號：24-cv-04002；Axome馬耳他有限公司和Axome治療公司訴Alkem實驗室有限公司等人案。2號：24-CV-04608；Axome馬耳他有限公司和Axome治療公司訴Aurobindo Pharma USA公司等人案。2-24-cv-07511；Axome馬耳他有限公司和Axome治療公司訴Alkem實驗室有限公司。2-24-cv-08365；Axome馬耳他有限公司和Axome治療公司訴Aurobindo Pharma USA，Inc.等人2-24-cv-08624；Axome馬耳他有限公司和Axome治療公司訴Alkem實驗室有限公司等人案。編號2-24-cv-09209，均在新澤西州地區法院。2024年6月4日，安盛和馬耳他子公司與聯昌達成和解協議，根據協議，聯昌同意在2042年6月30日之前或在某些情況下更早推出其仿製藥solriamfetol產品。2024年8月21日，Axome與馬耳他子公司達成協議，駁回對Sandoz的懸而未決的訴訟。所有其他行動目前都懸而未決。2024年9月25日，Hikma PharmPharmticals USA，Inc.向美國專利商標局專利審判和上訴委員會提交了對美國11560354號專利的跨方審查請願書。這份請願書的標題是Hikma製藥美國公司f/k/a West-Ward製藥公司訴Axome馬耳他有限公司。IPR2024-01418。
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項目1A. RISK因素。
該公司面臨許多風險，如果實現，可能會對其業務、經營業績、現金流、財務狀況或前景產生重大不利影響。以下是公司面臨的主要風險因素摘要。以下列表並不詳盡，公司面臨額外的挑戰和風險。我們已根據日期爲2024年2月23日提交給SEC的10-k表格年度報告中包含的風險因素，酌情重述和修改了本季度重大更新的風險因素。在決定投資公司的任何證券之前，投資者應仔細考慮本季度報告10-Q表格中列出的所有信息，包括以下風險因素。
風險因素摘要
我們的業務受到許多風險和不確定性的影響，包括下面詳細討論的那些風險。除其他外，這些風險包括：
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與我們的財務狀況和資本需求相關的風險
自成立以來，我們已經遭受了重大損失，預計我們將繼續遭受損失，並且可能永遠無法實現或維持盈利能力。
我們是一家經營歷史有限的生物製藥公司。自成立以來，我們已經出現了重大的運營損失。截至2024年9月30日和2023年9月30日的九個月，我們的淨虧損分別爲21230萬美元和14060萬美元。截至2024年9月30日，我們累計赤字爲104790萬美元。2022年，我們開始在美國和部分全球市場對Auvelity進行商業銷售，並開始在美國和部分全球市場對Sunosi進行商業銷售。除了Auvelity和Sunosi之外，我們沒有其他產品獲得監管機構批准。
隨着我們繼續開發當前和未來的候選產品，我們預計將繼續產生巨額費用和運營損失。此外，我們預計將產生與Auvelity、Sunosi以及任何其他可能獲得批准或我們可能獲得許可的候選產品的商業化相關的巨額銷售、營銷和製造費用。我們預計我們的費用將大幅增加，因爲我們：
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爲了成爲並保持盈利，我們必須成功地開發(或授權內)產生大量收入的產品並將其商業化。這將要求我們在一系列具有挑戰性的活動中取得成功，其中可能包括完成候選產品的臨床前測試和臨床試驗，發現更多候選產品，可能達成合作和許可協議，獲得候選產品的監管批准以及我們可能獲得監管批准的任何產品的製造、營銷和銷售，實現市場對我們產品的接受，滿足任何上市後要求，保持適當的分銷，設定價格，以及從私人保險或政府付款人那裏獲得我們產品的補償。就某些產品和候選產品而言，我們只是處於其中一些活動的初步階段。我們可能永遠不會在這些活動中取得成功，即使我們成功了，也可能永遠不會實現盈利。
由於與藥品開發相關的衆多風險和不確定性，我們無法準確預測我們可能發生的增加費用的時間或金額，或者何時或是否能夠實現盈利。如果FDA或類似的外國監管機構要求我們在當前預期的研究之外進行研究，或者如果完成我們的臨床試驗或任何候選產品的開發出現任何延誤，我們的費用可能會增加。
即使我們確實實現了盈利能力，我們也可能無法維持或提高季度或年度的盈利能力。我們無法實現並保持盈利將壓低我們公司的價值，並可能損害我們籌集資本、擴大業務、維持研發工作、實現產品多元化、繼續產品商業化甚至繼續運營的能力。我們公司價值的下降也可能導致您失去全部或部分投資。
我們可能需要額外的資金來進行未來的臨床試驗並完成候選產品的開發和商業化。如果我們無法在需要時籌集資金，我們將被迫推遲、減少或取消我們的產品開發計劃或商業化工作。
進行臨床試驗、尋求監管批准、建立外包製造關係以及成功製造和商業化我們的候選產品是一個耗時、昂貴且不確定的過程，需要數年時間才能完成。我們可能需要籌集額外資本以：
我們相信，根據當前的運營計劃，我們當前的現金足以爲預期的運營提供資金，使其成爲現金流正向。我們的假設可能被證明是錯誤的，我們可以比目前預期更快地使用可用的財政資源。此外，我們可能沒有足夠的財政資源來實現我們的所有目標。我們未來的資金需求將取決於許多因素，包括但不限於：
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未來的資本要求還取決於我們收購或投資額外業務、產品和技術的程度。在我們能夠產生足夠數量的產品收入（如果有的話）之前，我們可以通過公開或私募股權發行、債務融資、特許權使用費、企業合作和許可安排以及通過現金和投資餘額賺取的利息收入來滿足未來的現金需求。我們無法確定是否會以可接受的條件或根本無法確定額外資金。如果沒有足夠的資金，我們可能會被要求推遲、縮小範圍或取消我們的一個或多個開發計劃或商業化工作。
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由於與Hercules的貸款和擔保協議項下未償債務相關的契約，我們的運營活動可能會受到限制，並且如果發生違約，我們可能會被要求償還未償債務，這可能會對我們的業務產生重大不利影響。
2020年9月，我們與Hercules Capital，Inc.，或Hercules Capital，Inc.，作爲行政代理和抵押品代理以及貸款人的身份，以及不時成爲貸款協議當事方的其他金融機構，統稱爲貸款人，簽訂了一項定期貸款，我們將其稱爲2020年定期貸款，並以我們幾乎所有資產(包括知識產權)的留置權爲抵押。2021年10月，我們簽署了貸款協議第一修正案，其中包括增加2020年定期貸款的規模。於二零二二年三月，吾等訂立貸款協議第二修正案，其中包括於收購事項(定義見貸款協議)完成時更改定期貸款墊款的條款(定義見貸款協議)。於2023年1月，吾等訂立第三修正案，修訂貸款協議的條款，其中包括將2020年定期貸款的本金總額由30000美元萬增至35000美元萬，降低利率，並延長貸款協議的到期日及只限息期限。2023年5月，我們簽署了第四修正案，增加了馬耳他子公司在美國境外可以持有的現金金額，並免除了馬耳他子公司在第四修正案日期之前持有的現金金額的任何據稱違約。2023年8月，Hercules授予Axome對第四修正案的豁免權，增加了馬耳他子公司在美國以外可以持有的現金金額，直到2023年12月31日。我們於2024年9月30日訂立了《第五修正案》，修訂了貸款協議的條款，其中包括：(I)將2020年定期貸款第3批的本金總額從75.0美元提高至8,000萬；(Ii)延長2020年定期貸款某些部分的可獲得期；(Iii)更改貸款協議中包含的業績契約的條款，並增加新的業績契約；(Iv)有條件地在安盛市值超過15美元億的期間免除最低現金要求；以及(V)允許馬耳他子公司向貸款人申請不超過一定數額的預付款，只要Axome可以要求預付款，並增加馬耳他子公司在美國境外持有的現金金額，這在第五修正案中有更詳細的規定。
貸款協議包含各種契約，限制我們從事指定類型交易的能力。這些契約限制了我們出售、轉讓、租賃或處置某些資產的能力;承擔債務;對某些資產設定抵押權或允許對某些資產設定優先權;進行某些投資;進行某些限制性付款，包括支付股息、回購或分配我們的普通股;以及與關聯公司進行某些交易。我們的業務可能會受到這些對我們運營業務能力的限制的不利影響。
貸款協議下的契約還要求在貸方擁有第一優先擔保權益的一個或多個帳戶中保持最低金額的現金。
違反貸款協議項下的任何契諾可能導致2020年定期貸款違約。2020年定期貸款發生違約事件後，貸款人可以選擇宣佈所有未償金額（如有）立即到期和支付，並終止所有提供進一步信貸的承諾。如果我們無法償還任何未償款項，貸方可以利用授予其的抵押品來擔保此類債務。
我們的產品商業化運營歷史有限，這可能導致評估我們的業務和前景變得困難。
我們是一家商業階段的公司。在2022年將Auvelity和Sunosi商業化之前，我們尚未獲得任何候選產品的營銷批准，也沒有以商業規模生產產品或安排第三方代表我們這樣做，也沒有進行成功商業化所需的銷售和營銷活動。因此，如果我們有更長的成功開發和商業化產品的歷史，那麼對我們未來成功或生存能力的預測可能不會那麼準確。
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我們預計，由於多種因素，其中許多因素超出了我們的控制範圍，我們的財務狀況和經營業績將繼續逐季度和逐年波動。我們已經從一家僅以研發爲重點的公司轉變爲一家也有能力開展商業活動的公司。我們可能會遇到不可預見的費用、困難、併發症和延誤，並且在這樣的過渡中可能不會成功。
我們目前正處於經濟不確定和資本市場混亂的時期，這受到地緣政治不穩定的顯着影響。地緣政治緊張局勢對全球經濟和資本市場產生任何負面影響，我們的業務、財務狀況和運營業績可能會受到重大不利影響。
隨着地緣政治緊張局勢升級以及俄羅斯和烏克蘭之間軍事衝突的開始，美國和全球市場正在經歷波動和混亂。2022年2月24日，據報道俄羅斯軍隊對烏克蘭進行了全面軍事入侵。儘管持續軍事衝突的持續時間和影響高度不可預測，但烏克蘭衝突可能導致市場混亂，包括大宗商品價格、信貸和資本市場的大幅波動，以及供應鏈中斷。我們正在繼續監控烏克蘭和全球的局勢，並評估其對我們業務的潛在影響。
此外，烏克蘭軍事衝突導致美國、歐盟和其他國家對俄羅斯實施制裁和其他處罰。還提出和/或威脅實施額外的潛在制裁和處罰。俄羅斯的軍事行動以及由此產生的制裁可能會對全球經濟和金融市場產生不利影響，導致資本市場不穩定和流動性缺乏，可能使我們更難獲得額外資金。
此外，2023年10月7日，哈馬斯武裝分子和其他恐怖組織成員從加沙地帶滲透到以色列南部邊境，對平民和軍事目標實施了一系列恐怖襲擊。襲擊發生後不久，以色列安全內閣向哈馬斯宣戰，以色列對加沙地帶內的多個目標發動了空中轟炸，隨後還開始在加沙地帶的地面行動，該行動仍在進行中。包括黎巴嫩真主黨在內的其他恐怖分子和/或區域組織也加入了敵對行動，包括伊朗在內的其他中東國家可能會進一步捲入與以色列的敵對行動，導致衝突進一步擴大。以色列當前戰爭的強度和持續時間很難預測，此類戰爭對全球經濟的影響也很難預測。
此外，由於當前的地緣政治緊張局勢，拜登政府已經簽署了多項針對中國的行政命令。2022年9月12日簽署的一項名爲《推進生物技術和生物製造創新，打造可持續、安全和可靠的美國生物經濟》的行政命令可能會影響製藥業，鼓勵美國國內製造藥品。此外，美國國會還提出了一些立法建議，比如一項名爲《生物安全法》的法案，旨在阻止與中國公司就醫藥產品的開發或製造簽訂合同。《生物安全法》於2024年9月9日在美國衆議院獲得通過。美國衆議院通過的《生物安全法》版本包括一項不受限制的條款，該條款允許在該法案生效日期之前與其中提到的中國公司簽訂合同，直至2032年1月1日。生物安全法案還必須在美國參議院獲得通過，然後才能提交總裁·拜登否決或簽署，目前還不確定該法案是否會在2025年1月3日本屆立法會期結束前提交美國參議院進行表決。任何有關中國的額外行政命令或立法行動或潛在的制裁都可能對我們目前的製造合作伙伴產生實質性影響。
儘管迄今爲止，我們的業務尚未受到這些地緣政治問題或美國國內政治氣候的重大影響，但無法預測我們或供應商和製造商的業務將在短期和長期內受到影響的程度，或者衝突可能會影響我們的業務。軍事行動、制裁以及由此產生的市場混亂的程度和持續時間無法預測，但可能是巨大的。任何此類中斷也可能放大本文描述的其他風險的影響。
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政治不確定性可能會對我們的經營業績和經營業績產生不利影響。
總體政治不確定性可能會對我們的經營業績和經營業績產生不利影響。特別是，美國繼續經歷重大政治事件，給全球金融和經濟市場帶來不確定性，特別是在最近的總統選舉之後。目前尚不清楚美國新政府將採取哪些行動，以及如果實施，這些行動可能會如何影響美國的生物製藥行業。美國新政府採取的任何行動都可能對美國經濟以及我們的業務、財務狀況和運營業績產生負面影響。
Climate change or legal, regulatory or market measures to address climate change may negatively affect our business, results of operations, cash flows and prospects.
We believe that climate change has the potential to negatively affect our business and results of operations, cash flows and prospects. We are exposed to physical risks (such as extreme weather conditions or rising sea levels), risks in transitioning to a low-carbon economy (such as additional legal or regulatory requirements, changes in technology, market risk and reputational risk) and social and human effects (such as population dislocations and harm to health and well-being) associated with climate change. These risks can be either acute (short-term) or chronic (long-term).
The adverse impacts of climate change include increased frequency and severity of natural disasters and extreme weather events such as hurricanes, tornados, wildfires (exacerbated by drought), flooding, and extreme heat. Extreme weather and sea-level rise pose physical risks to our facilities as well as those of our suppliers. Such risks include losses incurred as a result of physical damage to facilities, loss or spoilage of inventory, and business interruption caused by such natural disasters and extreme weather events. Other potential physical impacts due to climate change include reduced access to high-quality water in certain regions and the loss of biodiversity, which could impact future product development. These risks could disrupt our operations and its supply chain, which may result in increased costs.
New legal or regulatory requirements may be enacted to prevent, mitigate, or adapt to the implications of a changing climate and its effects on the environment. These regulations, which may differ across jurisdictions, could result in us being subject to new or expanded carbon pricing or taxes, increased compliance costs, restrictions on greenhouse gas emissions, investment in new technologies, increased carbon disclosure and transparency, upgrade of facilities to meet new building codes, and the redesign of utility systems, which could increase our operating costs, including the cost of electricity and energy used by us. Our supply chain would likely be subject to these same transitional risks and would likely pass along any increased costs to us.
RISKS RELATED TO OUR BUSINESS AND THE DEVELOPMENT OF OUR PRODUCT CANDIDATES
We are substantially dependent on the success of our products and cannot guarantee that any of our product candidates will successfully complete any planned or ongoing clinical trials, receive regulatory approval, or be successfully commercialized.
We currently have two products approved for commercial distribution. We have invested a significant portion of our efforts and financial resources in the development of our product candidates. Our business, including our ability to generate revenue, depends entirely on the successful commercialization of Auvelity and Sunosi and the successful development and commercialization of our product candidates and/or future in-licensing activities, which may never occur. Furthermore, given the nature of our business, the biopharmaceutical industry in general and the uncertainty and costs associated with developing and commercializing our products within a complicated and costly regulatory regime, our goals, plans and assumptions with respect to our products may evolve or change. For example, we may not continue to emphasize, focus our research and development efforts on or direct resources to certain of our product candidates, and we may shift our focus and resources to our other current or future products. Any such change in our business strategy could harm our business, cause uncertainty or confusion in the marketplace or harm the clinical prospects of our products.
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Our product candidates will require additional clinical and non-clinical development, regulatory approval, commercial manufacturing arrangements, significant marketing efforts, and further investment before we generate any revenues from the sale of such product candidates. Multiple clinical trials are ongoing. As a result of one or more risks discussed in this section, we cannot assure you that we will meet projected timelines related to these trials.
We are not permitted to market or promote any of our product candidates before we receive regulatory approval from the FDA or comparable foreign regulatory authorities, and we may never receive such regulatory approval for any of our product candidates. Even if our product candidates are approved, they may be subject to limitations on the indicated uses for which they may be marketed, distribution restrictions, or to other conditions of approval, may contain significant safety warnings, including boxed warnings, contraindications, and precautions, may not be approved with label statements necessary or desirable for successful commercialization, or may contain requirements for costly post-market testing and surveillance, or other requirements, including the submission of a risk evaluation and mitigation strategy, or REMS, to monitor the safety or efficacy of the products. If we do not receive regulatory approval for, and successfully commercialize, our product candidates, we will not be able to generate revenue from these product candidates in the foreseeable future, or at all. Any significant delays in obtaining approval for and commercializing our product candidates will have a material adverse impact on our business and financial condition.
Although we submitted NDAs to the FDA for Auvelity (which was approved) and for AXS-07 for the acute treatment of migraines (which received a CRL and has now been resubmitted), we have not otherwise submitted an NDA to the FDA, or similar drug approval filings to comparable foreign authorities, for any product candidate, and we cannot be certain that our current or future product candidates will be successful in clinical trials or receive regulatory approval.
Our product candidates are susceptible to the risks of failure inherent at any stage of product development, including the appearance of unexpected adverse events or failure to achieve its primary endpoints in subsequent clinical trials, including our initiated and planned Phase 3 clinical trials. We conducted one interim analysis for the Phase 2/3 trial of AXS-05 in TRD and one interim analysis for the Phase 2/3 trial of AXS-05 for the treatment of AD agitation. We may elect to conduct interim analyses for our other clinical trials. Interim results of a clinical trial do not necessarily predict final results, and interim results may result in early stoppage of our clinical trials for futility or modifications to our clinical trials, including the addition of additional subjects. Further, our product candidates may not receive regulatory approval even if they are successful in clinical trials.
If approved for marketing by applicable regulatory authorities, our ability to generate revenues from our product candidates depend on our ability to:
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Potential conflicts of interest exist with respect to the intellectual property rights that we license from an entity owned by our Chief Executive Officer and Chairman of the Board, and it is possible that our interests and their interests may diverge.
In 2012, we entered into three exclusive license agreements with Antecip Bioventures II LLC, or Antecip, an entity owned by our Chief Executive Officer and Chairman of the Board, Herriot Tabuteau, M.D., in which we were granted exclusive licenses to develop, manufacture, and commercialize Antecip’s patents and applications related to the development of certain of the Company’s then current product candidates. The patents licensed from Antecip include certain intellectual property pertaining to the Company’s Auvelity product / AXS-05 portfolio product. Although Dr. Tabuteau dedicates all of his working time to us, because Antecip is an inactive intellectual property holding company, he may face potential conflicts of interest regarding these licensing transactions as a result of his ownership of Antecip. The license agreements provide that, subject to the reasonable consent of Antecip, we have the right to control the prosecution or defense, as the case may require, of a patent infringement claim involving the licensed intellectual property. Our interests with respect to pleadings and settlements in such cases may be at odds with those of Antecip. If there is a dispute between us and Antecip, Dr. Tabuteau will have a conflict of interest because he may, at the time of a prospective dispute, simultaneously have a financial interest in and owe a fiduciary duty to Antecip and simultaneously have a financial interest in and owe a fiduciary duty to us. For example, if a contractual dispute arises between us and Antecip under any of the license agreements we have with Antecip, Dr. Tabuteau may be in a position where he would benefit if Antecip prevails, to the detriment of our business or our investors, even though he is an officer and director of our company, because he is the sole owner of Antecip. Similarly, if we have a claim of any kind against Antecip, Dr. Tabuteau may be, even as our Chief Executive Officer and Chairman of the Board, reluctant to assert a claim by us against Antecip because of his financial interest in Antecip. We cannot assure you that any conflicts will be resolved in our favor, and as a result, our business could be impeded or materially harmed.
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We may expend our limited resources to pursue a particular product candidate or indication and fail to capitalize on product candidates or indications that may be more profitable or for which there is a greater likelihood of success.
Because we have limited financial and managerial resources, we focus on developing product candidates for specific indications that we identify as most likely to succeed, in terms of both its regulatory approval and commercialization. As such, we are currently primarily focused on the development of solriamfetol for additional indications, AXS-05 for the treatment of agitation associated with AD, and smoking cessation, AXS-07 for the acute treatment of migraines, AXS-12 for the treatment of narcolepsy and AXS-14 for the treatment of fibromyalgia. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that may prove to have greater commercial potential. Additionally, as more fully described in “Business—Material License Agreements,” we are required to pay to an entity owned by our Chief Executive Officer and Chairman of the Board certain royalty payments related to the sales of the Company’s Auvelity product / AXS-05 portfolio product, as well as two product candidates that are not currently in active development. This may influence management’s decision concerning which product candidates or indications to pursue and/or the manner in which our products are commercialized. Our resource allocation decisions may cause us to fail to capitalize on viable commercial products or profitable market opportunities. Our spending on current and future research and development programs and product candidates for specific indications may not yield any commercially viable products. If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate through collaboration, licensing, or other royalty arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate.
Our future growth may depend on our ability to identify and develop product candidates, and if we do not successfully identify and develop product candidates or integrate them into our operations, we may have limited growth opportunities.
A component of our business strategy is to continue to develop a pipeline of product candidates by developing products that we believe are a strategic fit with our focus on central nervous system, or CNS, therapeutics. However, these business activities may entail numerous operational and financial risks, including:
For instance, our prior efforts have resulted in our decision not to further develop certain product candidates that, at one time, appeared to be promising. Likewise, we received a CRL from the FDA relating to the Company’s AXS-07 portfolio product in 2022 (we have since resubmitted the NDA for AXS-07). We have limited resources to identify and execute the development of products. Moreover, we may devote resources to potential development that are never completed, or we may fail to realize the anticipated benefits of such efforts. If we do not successfully develop and commercialize product candidates, we may not be able to obtain revenues from such product candidates in future periods.
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If safety and efficacy data for our product candidates, a reference drug, or published literature does not satisfactorily demonstrate safety and efficacy to the FDA, or if the FDA and other regulators do not permit us to rely on the data of a reference drug or published literature, we may incur additional costs or experience delays in completing, or ultimately be unable to complete, the development and commercialization of our product candidates.
We are not permitted to commercialize, market, promote, or sell any product candidate in the United States without obtaining marketing approval from the FDA. Comparable foreign regulatory authorities, such as the European Medicines Agency, or EMA, impose similar restrictions.
In the United States, we currently plan to, at least initially, seek approval of some of our product candidates using the 505(b)(2) pathway. These 505(b)(2) product candidates include AXS-05 and AXS-07. The FDA interprets Section 505(b)(2) of the Federal Food, Drug, and Cosmetic Act, or FDCA, for purposes of approving an NDA, to permit the applicant to rely, in part, upon published literature or the FDA’s previous findings of safety and efficacy for an approved product. The FDA, though, requires companies to perform additional clinical trials or preclinical studies to support any deviation from the previously approved product and to support reliance on the FDA’s prior findings of safety and efficacy or published literature.
Under the 505(b)(2) pathway, the FDA may approve our product candidates for all or some of the label indications for which the referenced product has been approved, as well as for any new indication sought pursuant to the Section 505(b)(2) process. The label, however, may require all or some of the limitations, contraindications, warnings, or precautions included in the reference product’s label, including a box warning (commonly referred to as a “black box warning”), or may require additional limitations, contraindications, warnings, or precautions, including class‑wide warnings. For instance, antidepressants, including Auvelity, include a class‑wide black box warning regarding the increased risk of suicidal thoughts and behavior.
In addition, because we plan to file certain product candidates under an NDA submitted pursuant to 505(b)(2), we will rely, at least in part, upon a reference drug and published literature. For example, we have and/or intend to rely on third-party studies in the published literature as well as FDA findings of safety and efficacy for approved drug products containing the same active molecules in AXS-05 and AXS-07. If the FDA disagrees with our conclusions regarding the appropriateness of our reliance on a reference drug or published literature, we could be required to conduct additional clinical trials or other studies to support our NDA, which could lead to unanticipated costs and delays or to the termination of our development program. If we are unable to obtain approval for our pharmaceutical formulations through the 505(b)(2) NDA process, we may be required to pursue the more expensive and time consuming 505(b)(1) approval process, which consists of full reports of investigations of safety and effectiveness conducted by or for the applicant. In addition, because we have submitted NDAs for AXS-05 and AXS-07 pursuant to the 505(b)(2) process, we have not conducted certain additional clinical trials for these product candidates and, as such, we will have less experience with actual testing of these product candidates.
There may also be circumstances under which the FDA would not allow us to pursue a 505(b)(2) application. For instance, should the FDA approve a pharmaceutically equivalent product to our product candidates before we obtain approval, we would no longer be able to use the 505(b)(2) pathway. In that case, it is the FDA’s policy that the appropriate submission would be an ANDA, for a generic version of the approved product. We may, however, not be able to immediately submit an ANDA or have an ANDA approval made effective, as we could be blocked by others’ periods of patent and regulatory exclusivity protection.
Notwithstanding the approval of a number of products by the FDA under 505(b)(2) over the last few years, pharmaceutical companies and others have objected to the FDA’s interpretation of Section 505(b)(2). If the FDA’s interpretation of Section 505(b)(2) is successfully challenged, the FDA may change its policies and practices with respect to Section 505(b)(2) regulatory approvals, which could delay or even prevent the FDA from approving any NDA that we submit pursuant to the 505(b)(2) process. Moreover, our inability to pursue a 505(b)(2) application could result in new competitive products reaching the market more quickly than our product candidates, which could hurt our competitive position and our business prospects.
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An NDA submitted under Section 505(b)(2) subjects us to the risk that we may be subject to a patent infringement lawsuit or regulatory actions that would delay or prevent the review or approval of our product candidates.
Under the Hatch-Waxman Act, the holder of patents listed in the Orange Book for NDAs that a 505(b)(2) application references may file a patent infringement lawsuit after receiving notice of the paragraph IV certification. Filing of a patent infringement lawsuit against the filer of the 505(b)(2) applicant within 45 days of the patent or NDA owner’s receipt of notice triggers a one time, automatic, 30 months stay of the FDA’s ability to make the 505(b)(2) NDA approval effective. In such a case, the FDA may not make the 505(b)(2) NDA approval effective until the earlier of 30 months from the receipt of the notice of the paragraph IV certification, the expiration of the patent, when the infringement case concerning each such patent was favorably decided in the applicant’s favor or settled, or such shorter or longer period as may be ordered by a court. Accordingly, we may invest a significant amount of time and expense in the development of one or more product candidates only to be subject to significant delay and patent litigation before such product candidates may be commercialized, if at all. In addition, a 505(b)(2) application approval will not be made effective until any existing non-patent exclusivity, such as exclusivity for obtaining approval of a new chemical entity, or NCE, or exclusivities for changes to NCEs listed in the Orange Book for the referenced product have expired or, if possible, are carved out from the label.
In practice, companies that produce branded reference listed drugs often bring patent litigation against applicants that seek regulatory approval to market generic or reformulated versions of their products. Litigation to enforce or defend intellectual property rights is often complex and often involves significant expense and can delay or prevent introduction or sale of our product candidates. If a court finds patents valid and infringed by our product candidates, we may be required to cease selling, relinquish or destroy existing stock, or pay monetary damages unless we can obtain a license from the patent holder. There may also be situations where we use our business judgment and decide to market and sell our approved products, notwithstanding the fact that allegations of patent infringement have not been finally resolved by the courts, an approach known as an “at risk launch.” The risk involved in doing so can be substantial because the remedies available to the owner of a patent for infringement may include, among other things, damages measured by the profits lost by the patent owner which may be greater than the profits earned by the infringer. In the case of willful infringement, such damages may be increased up to three times. An adverse decision in patent litigation could have a material adverse effect on our business, financial position, and results of operations and could cause the market value of our common stock to decline.
The regulatory approval timelines and processes of the FDA and comparable foreign authorities are lengthy, time consuming, and inherently unpredictable. If we are not able to obtain, or if there are delays in obtaining, required regulatory approvals, we will not be able to commercialize our product candidates as expected, and our ability to generate revenue will be materially impaired.
The timeline for review and time required to obtain approval by the FDA and comparable foreign authorities is unpredictable but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including the substantial discretion of the regulatory authorities and the availability and prioritization of regulatory agency resources. The timeline for regulatory approval can be affected by a variety of factors, including budget and funding levels, agency staffing, and statutory, regulatory, and policy changes. In addition, approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the course of a product candidate’s clinical development, vary among jurisdictions, and/or require us to amend our clinical trial protocols or conduct additional studies that require regulatory or institutional review board, or IRB, approval, or otherwise cause delays in the approval or rejection of an application. To date, we have submitted two NDAs to the FDA and have obtained regulatory approval for one of our product candidates, Auvelity. It is possible that none of our other existing product candidates, or any product candidates we may seek to develop in the future, will ever obtain regulatory approval. Any delay in obtaining or failure to obtain required approvals or uncertainty in the timing of regulatory action could materially adversely impact our development efforts and affect our ability or that of any of our collaborators to generate revenue from the particular product candidate, which likely would result in significant harm to our financial position and adversely impact our stock price.
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Our products and the activities associated with their development and commercialization, including their design, testing, manufacture, safety, efficacy, recordkeeping, labeling, storage, approval, advertising, promotion, sale, and distribution, are subject to comprehensive regulation by the FDA and other regulatory agencies in the United States, and by the EMA, in Europe, and similar regulatory authorities outside the United States and Europe. Failure to obtain marketing approval for a product candidate will prevent us from commercializing the product candidate. We have limited experience in filing and supporting the applications necessary to gain marketing approvals and rely on third‑party contract research organizations, or CROs, and consultants to assist us in this process. Securing marketing approval requires the submission of extensive preclinical and clinical data and supporting information to regulatory authorities for each therapeutic indication to establish the product candidate’s safety and efficacy for that indication and the submission of information about the product manufacturing process to, and inspection of manufacturing facilities and clinical trial sites by, the regulatory authorities.
Clinical testing is expensive and can take many years to complete, and its outcome is inherently uncertain. Failure can occur at any time during the clinical trial process. The results of preclinical studies; our product candidates’ mechanism of action; studies conducted by third parties in different patient populations, using different products, or using different study designs; and early clinical trials of our product candidates may not be predictive of the results of later‑stage clinical trials. Product candidates in later stages of clinical trials may fail to show the desired safety and efficacy traits despite having progressed through preclinical studies and initial clinical trials. Preclinical studies may also reveal unfavorable product candidate characteristics, including safety concerns. A number of companies in the pharmaceutical industry have suffered significant setbacks in advanced clinical trials due to lack of efficacy or adverse safety profiles, notwithstanding promising results in earlier trials. Our future clinical trial results may not be successful. Moreover, should there be a flaw in a clinical trial, it may not become apparent until the clinical trial is well advanced.
We may also experience numerous unforeseen events during, or as a result of, clinical trials and in the course of our preparation, submission, and review of NDA filings that could delay or prevent our ability to receive marketing approval or commercialize our product candidates, including:
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Moreover, if we are required to conduct additional clinical trials or other testing of our product candidates beyond that which we currently contemplate, if we are unable to successfully complete clinical trials or other testing of our product candidates, if the results of these trials or tests are not positive, or are only modestly positive or if there are safety concerns, we may:
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Our product candidate development costs will also increase if we experience delays in testing or approvals and we may not have sufficient funding to complete the testing and approval process for any of our product candidates. We may be required to obtain additional funds to complete clinical trials and prepare for possible commercialization of our product candidates. We do not know whether any additional preclinical tests or clinical trials will be required, will begin as planned, will need to be restructured, or will be completed on schedule, or at all. Significant delays relating to any preclinical studies or clinical trials also could shorten any periods during which we may have the exclusive right to commercialize our product candidates or allow our competitors, or the competitors of our collaborators, to bring products to market before we do and impair our ability to successfully commercialize our product candidates and may harm our business and results of operations. In addition, many of the factors that cause, or lead to, such delays may ultimately lead to the denial of marketing approval of any of our product candidates. If any of this occurs, our business, financial condition, results of operations, and prospects may be materially harmed.
Regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data are insufficient for approval and require additional preclinical studies, clinical trials, or other studies. In addition, varying interpretations of the data obtained from preclinical and clinical testing could delay, limit, or prevent marketing approval of a product candidate. During the course of review, the FDA may also request or require additional CMC, or other data and information, and the development and provision of these data and information may be time consuming and expensive. For example, in the CRL with respect to our NDA for AXS-07, the FDA noted the need for additional CMC data. Furthermore, there is the possibility that the FDA or comparable foreign regulatory authorities have not previously reviewed product candidates for the indications we are pursuing, such as AD agitation or smoking cessation. As a result, we may experience delays in regulatory approval due to uncertainties in the approval process.
If we experience delays in obtaining approval, if we fail to obtain approval of a product candidate or if the label for a product candidate does not include the labeling claims necessary or desirable for the successful commercialization of that product candidate, the commercial prospects for such product candidate may be harmed and our ability to generate revenues from that product candidate will be materially impaired. Furthermore, there is the possibility that the FDA or comparable foreign regulatory authorities have not previously reviewed product candidates for the indications we are pursuing, such as AD agitation or smoking cessation. As a result, we may experience delays in regulatory approval due to uncertainties in the approval process.
If we cannot demonstrate an acceptable safety and toxicity profile for our product candidates, we will not be able to continue our clinical trials of or obtain approval for those product candidates.
In order to obtain approval of a product candidate we must demonstrate safety in various nonclinical tests (including, for example, carcinogenicity studies, drug-drug interaction studies, and toxicity studies), in addition to human clinical trials. At the time of initiating human clinical trials, we may not have conducted or may not conduct all the types of nonclinical testing ultimately required by regulatory authorities, or future nonclinical tests may indicate safety concerns regarding our product candidates. Nonclinical testing and clinical testing are both expensive and time-consuming and have uncertain outcomes. Even if initial tests appear favorable, later testing may have unfavorable results. We may experience numerous unforeseen events during, or as a result of, the testing process, which could delay or prevent our ability to develop or commercialize our product candidates, including:
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Any such events would increase our costs and could delay or prevent our ability to commercialize our product candidates, which could adversely impact our business, financial condition and results of operation.
The FDA may determine that any of our current or future product candidates have undesirable side effects that could delay or prevent their regulatory approval or commercialization.
Undesirable side effects caused by our product candidates could cause us, IRBs, and other reviewing entities or regulatory authorities to interrupt, delay, or halt clinical trials and could result in a more restrictive label or the delay or denial of regulatory approval by the FDA or other comparable foreign authorities. For example, if concerns are raised regarding the safety of a new drug as a result of undesirable side effects identified during clinical or preclinical testing, the FDA may order us to cease further development, decline to approve the drug, or issue a letter requesting additional data or information prior to making a final decision regarding whether or not to approve the drug.
The number of requests for additional data or information issued by the FDA in recent years has increased and resulted in substantial delays in the approval of several new drugs. Undesirable side effects caused by any of our current or future product candidates could also result in denial of regulatory approval by the FDA or other comparable foreign authorities for any or all targeted indications or the inclusion of unfavorable information in our product labeling, such as limitations on the indicated uses for which the products may be marketed or distributed, a label with significant safety warnings, including boxed warnings, contraindications, and precautions, a label without statements necessary or desirable for successful commercialization, or may result in requirements for costly post‑marketing testing and surveillance, or other requirements, including REMS, to monitor the safety or efficacy of the products, and in turn prevent us from commercializing and generating revenues from the sale of any of our current or future product candidates.
Based on the side effects disclosed in FDA product labeling for marketed drugs that contain the same active molecules as our product candidate, AXS-07 may result in fatigue, confusion, dry mouth, diarrhea, nausea, insomnia, anemia, increased appetite, anxiety, sweating, dizziness, palpitations, arrythmia, tachycardia, abnormal vision, syncope, seizure, tremor, tinnitus, dizziness, somnolence, paresthesia, dysgeusia, dyspepsia, constipation, weight increase or decrease, gastritis, hematuria, flatulence, esophagitis, gastric ulcers, gastroesophageal reflux, gastrointestinal hemorrhages, colitis, rash, pain or tightness in the chest, neck, throat or jaw, upper respiratory tract infections, influenza-like symptoms, or other adverse events or potential adverse events reported or discussed in the product labels for meloxicam‑containing or rizatriptan-containing products including Anjeso, Vivlodex, Mobic, and Maxalt.
Based on the side effects disclosed in the EMA required product label for marketed drugs that contain the same active molecule as our product candidate, AXS-12 and AXS-14 may result in decreased appetite, insomnia, agitation, anxiety, dizziness, headache, paresthesia, akathisia, dysgeusia, accommodation disorder, mydriasis, glaucoma, vertigo, tachycardia, palpitations, vasodilation, hypotension, hypertension, dry mouth, vomiting, hyperhidrosis, rash, sensation of incomplete bladder emptying, urinary tract infection, dysuria, urinary retention, erectile dysfunction, ejaculatory pain, ejaculatory delay, chills, or other adverse events or potential adverse events reported or discussed in the product labels for reboxetine containing products including Edronax.
Known side effects for Auvelity and Sunosi are described on the approved labels for those products. In relation to further development efforts with respect to these compounds, different patient populations may react to these compounds differently. For example, AD agitation patients in the case of AXS-05 or ADHD patients in the case of solriamfetol may experience different side effects than patients taking these products for their currently approved indications. This is particularly true where different dosing, formulations or methods of administration are implicated.
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If any of our other product candidates are associated with serious adverse events or undesirable side effects or have properties that are unexpected, we may need to abandon development or limit development of that product candidate to certain uses or subpopulations in which the undesirable side effects or other characteristics are less prevalent, less severe, or more acceptable from a risk‑benefit perspective. The drug‑related side effects could affect patient recruitment or the ability of enrolled patients to complete the trial or result in potential product liability claims. Any of these occurrences may significantly harm our business, financial condition, results of operations, and prospects.
If we experience delays or difficulties in the enrollment of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.
We may not be able to initiate or continue conducting clinical trials for our product candidates if we are unable to locate and enroll a sufficient number of eligible patients to participate in these trials as required by the FDA or similar regulatory authorities outside the United States. Some of our competitors have ongoing clinical trials for product candidates that treat the same indications as our product candidates, and patients who would otherwise be eligible for our clinical trials may instead enroll in clinical trials of our competitors’ product candidates. Patient enrollment is affected by other factors including:
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Our inability to enroll a sufficient number of patients for our clinical trials would result in significant delays which would cause us to miss our projected timelines and could require us to abandon one or more clinical trials altogether. For instance, because we are seeking regulatory approval for certain indications that may have a narrow or small patient population, it may be difficult to find patients eligible to participate in our clinical studies at a sufficient rate or in a sufficient quantity. We may be required by the FDA to modify the entry criteria for our planned Phase 3 clinical trials and these changes may make it more difficult to enroll patients in our clinical trials. Moreover, patients in our clinical trials, especially patients in our control groups, may be at risk for dropping out of our studies if they are not experiencing relief of their symptoms. A significant number of withdrawn patients would compromise the quality of our data.
Enrollment delays or slower periods of enrollment in our clinical trials may result in increased development costs for our product candidates, or the inability to complete development of our product candidates, which would cause the value of our company to decline, limit our ability to obtain additional financing, and materially impair our ability to generate revenues.
Development of combination product candidates may present more or different challenges than development of single agent product candidates.
Certain product candidates of ours, including AXS-05 and AXS-07, are combination therapies. A combination therapy is a single drug product that consists of two or more active ingredients, with each component making a contribution to the claimed effect of the drug. The development of combination drugs may be more complex than the development of single agent products and generally requires that sponsors demonstrate the contribution of each component to the claimed effect and the safety and efficacy of the product as a whole. This requirement may make the design and conduct of clinical trials more complex, requiring more clinical trial subjects. We also may not be able to meet the FDA’s approval standards required for combination products. The FDA’s requirements concerning combination products may change in the future. Moreover, the applicable requirements for approval may differ from country to country.
Changes in product candidate manufacturing or formulation may result in additional costs or delay.
As product candidates are developed through preclinical studies to late-stage clinical trials towards approval and commercialization, it is common that various aspects of the development program, such as manufacturing methods and formulation, are altered along the way in an effort to optimize processes and results. For instance, as we begin scale-up efforts for commercial-size manufacturing batches, formulation changes may be necessary to improve tablet robustness. Such changes carry the risk that they will not achieve these intended objectives. Any of these changes could cause our product candidates to perform differently and affect the results of planned clinical trials or other future clinical trials conducted with the altered materials. Such changes may also require additional testing, FDA notification, or FDA approval. This could delay completion of clinical trials; require the conduct of bridging clinical trials or studies, or the repetition of one or more clinical trials; increase clinical trial costs; delay approval of our product candidates; and jeopardize our ability to commence product sales and generate revenue.
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Failure to obtain marketing approval in international jurisdictions would prevent our products from being marketed abroad.
In order to market and sell our products in the European Union, or EU, and many other jurisdictions, we or our third‑party collaborators must obtain separate marketing approvals and comply with numerous and varying regulatory requirements. The approval procedure varies among countries and can involve additional testing. The time required to obtain approval may differ substantially from that required to obtain FDA approval. The regulatory approval process outside the United States generally includes all of the risks associated with obtaining FDA approval. In addition, in many countries outside the United States, it is required that the product be approved for reimbursement before the product can be approved for sale in that country. We or these third parties may not obtain approvals from regulatory authorities outside the United States on a timely basis, if at all. Approval by the FDA does not ensure approval by regulatory authorities in other countries or jurisdictions, and approval by one regulatory authority outside the United States does not ensure approval by regulatory authorities in other countries or jurisdictions or by the FDA. However, the failure to obtain approval in one jurisdiction may compromise our ability to obtain approval elsewhere. We may not be able to file for marketing approvals and may not receive necessary approvals to commercialize our products in any market.
Although Breakthrough Therapy, Fast Track, and other designations are designed to expedite the development and review of drugs, they may not ultimately lead to a faster approval process or faster development of regulatory review, and they will not increase the likelihood that our product candidates will receive marketing approval, for example, Breakthrough Therapy designation by the FDA for AXS-05 for the treatment of AD agitation.
We have received a Fast Track product designation for AXS-05 for both the treatment of TRD as well as for the treatment of AD agitation, and we may seek Fast Track designation for other of our current or future product candidates. The FDA may designate a product for Fast Track review if it is intended, whether alone or in combination with one or more other products, for the treatment of a serious or life-threatening disease or condition, and it demonstrates the potential to address unmet medical needs for such a disease or condition. For Fast Track products, sponsors may have greater interactions with the FDA, and the FDA may initiate review of sections of a Fast Track product’s application before the application is complete. This rolling review may be available if the FDA determines, after preliminary evaluation of clinical data submitted by the sponsor, that a Fast Track product may be effective. The sponsor must also provide, and the FDA must approve, a schedule for the submission of the remaining information, and the sponsor must pay applicable user fees.
We also received Breakthrough Therapy designation for AXS-05 for both the treatment of MDD and the treatment of AD agitation, and we may seek Breakthrough Therapy designation for other current or future product candidates. A Breakthrough Therapy is defined as a product candidate that is intended, alone or in combination with one or more other drugs, to treat a serious or life-threatening disease or condition, and preliminary clinical evidence indicates that the product candidate may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development. For product candidates that have been designated as breakthrough therapies, interaction and communication between the FDA and the sponsor of the trial can help to identify the most efficient path for clinical development while minimizing the number of patients placed in ineffective control regimens. Breakthrough Therapy designation also allows the sponsor to request a Priority Review or file sections of the NDA on an ongoing basis for rolling review where the FDA may consider beginning review portions of a marketing application before the full submission is complete. Product candidates designated as Breakthrough Therapies by the FDA are also eligible for Priority Review if supported by clinical data at the time of the submission of the NDA.
Breakthrough Therapy or Fast Track designation is within the discretion of the FDA. The receipt of a Breakthrough Therapy or Fast Track designation for a product candidate may not ultimately result in a faster development process or review, and it does not in any way assure approval of product candidates by the FDA. In addition, the FDA may later decide to rescind the Breakthrough Therapy or Fast Track designation for one or more of our applicable product candidates if such product candidates no longer meet the conditions for qualification of this program. For example, we were initially granted Breakthrough Therapy designation for AXS-12 for the treatment of cataplexy in patients with narcolepsy in August 2020. In July 2021, the FDA rescinded our Breakthrough Therapy designation due to the FDA approving an additional drug product for the treatment of cataplexy in narcolepsy.
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Regulatory approval is limited by the FDA or comparable foreign regulatory authorities to those specific indications and conditions for which clinical safety and efficacy have been demonstrated, and we may be subject to fines, penalties, injunctions, or other enforcement actions if we are determined to be promoting the use of our products for unapproved or “off‑label” uses, resulting in damage to our reputation and business.
We, and any of our collaborators, must comply with requirements concerning advertising and promotion for any of our products for which we or they obtain marketing approval. Promotional communications with respect to prescription drugs are subject to a variety of legal and regulatory restrictions and continuing review by the FDA, Department of Justice, HHS’s Office of Inspector General, state attorneys general, members of Congress, and the public. When the FDA or comparable foreign regulatory authorities issue regulatory approval for a product candidate, the regulatory approval is limited to those specific uses and indications for which a product is approved. If we are not able to obtain FDA approval for any desired uses or indications for our products and product candidates, we may not market or promote our products for those indications and uses, referred to as off‑label uses, and our business may be adversely affected. We further must be able to sufficiently substantiate any claims that we make for our products including claims comparing our products to other companies’ products.
While physicians may choose to prescribe drugs for uses that are not described in the product’s labeling and for uses that differ from those tested in clinical studies and approved by the regulatory authorities, we are prohibited from marketing and promoting the products for indications and uses that are not specifically approved by the FDA or comparable foreign regulatory authorities. These off‑label uses are common across medical specialties and may constitute an appropriate treatment for some patients in varied circumstances. Regulatory authorities in the United States and in many other major markets do not generally restrict or regulate the behavior of physicians in their choice of treatment within the practice of medicine. Regulatory authorities do, however, restrict communications by pharmaceutical companies concerning off‑label use.
If we are found to have impermissibly promoted any of our products, we may become subject to significant liability and government fines. The FDA and other agencies actively enforce the laws and regulations regarding product promotion, particularly those prohibiting the promotion of off‑label uses, and a company that is found to have improperly promoted a product may be subject to significant sanctions. The federal government has levied large civil and criminal fines against companies for alleged improper promotion and has enjoined several companies from engaging in off‑label promotion. The FDA has also requested that companies enter into consent decrees of permanent injunctions under which specified promotional conduct is changed or curtailed. Thus, we and any of our collaborators will not be able to promote any products we develop for indications or uses for which they are not approved.
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In the United States, engaging in the impermissible promotion of our products, following approval, for off-label uses can also subject us to false claims and other litigation under federal and state statutes, including fraud and abuse and consumer protection laws, which can lead to civil and criminal penalties and fines, agreements with governmental authorities that materially restrict the manner in which we promote or distribute drug products and do business through, for example, corporate integrity agreements, suspension or exclusion from participation in federal and state healthcare programs, and debarment from government contracts and refusal of future orders under existing contracts. Recent court decisions have impacted the FDA’s enforcement activity regarding off-label promotion in light of First Amendment considerations; however, there are still significant risks in this area in part due to the potential False Claims Act exposure. The False Claims Act allows any individual to bring a lawsuit against a pharmaceutical company on behalf of the federal government alleging submission of false or fraudulent claims or causing others to present such false or fraudulent claims, for payment by a federal program such as Medicare or Medicaid. If the government decides to intervene and prevails in the qui tam lawsuit, the individual will share in the proceeds from any fines or settlement funds. If the government declines to intervene, the individual may pursue the case alone. Under the False Claims Act, a penalty may be imposed for each false claim, for example, a claim for payment for each prescription for the product, and, when aggregated, these penalties often total millions of dollars and incentivize qui tam lawsuits. These False Claims Act lawsuits against pharmaceutical companies have increased significantly in volume and breadth, leading to several substantial civil and criminal settlements, up to $3.0 billion, pertaining to certain sales practices and promoting off-label drug uses. This growth in litigation has increased the risk that a pharmaceutical company will have to defend a false claim action; pay settlement fines or restitution, as well as criminal and civil penalties; agree to comply with burdensome reporting and compliance obligations; and be excluded from Medicare, Medicaid, or other federal and state healthcare programs. If we or our collaborators do not lawfully promote our approved products, if any, we may become subject to such litigation and, if we do not successfully defend against such actions, those actions may have a material adverse effect on our business, financial condition, results of operations, and prospects.
In the United States, the distribution of product samples to physicians must further comply with the requirements of the U.S. Prescription Drug Marketing Act. If the FDA determines that our promotional materials or activities violate its regulations and policies pertaining to product promotion, it could request that we modify our promotional materials or activities or subject us to regulatory or other enforcement actions, including issuance of warning letters or untitled letters, suspension or withdrawal of an approved product from the market, requests for recalls, payment of civil fines, disgorgement of money, imposition of operating restrictions, injunctions, or criminal prosecution. These regulatory and enforcement actions could significantly harm our business, financial condition, results of operations, and prospects.
We are, and will continue to be subject to, ongoing obligations and continued regulatory review, which may result in significant additional expense. Additionally, any of our products, could be subject to labeling and other restrictions and market withdrawal and we may be subject to penalties if we fail to comply with regulatory requirements or experience unanticipated problems with our products.
Our product(s) are subject to extensive and ongoing requirements of and review by the FDA and other regulatory authorities, including requirements related to the manufacturing processes, post approval clinical data, labeling, packaging, distribution, adverse event reporting, storage, recordkeeping, export, import, advertising, marketing, and promotional activities for such product. These requirements further include submissions of safety and other post-marketing information, including manufacturing deviations and reports; registration and listing requirements; the payment of annual program fees for our products; continued compliance with cGMP requirements relating to manufacturing, quality control, quality assurance, and corresponding maintenance of records and documents; requirements regarding the distribution of samples to physicians and recordkeeping; and GCP, for any clinical trials that we conduct post approval.
We and any of our collaborators, including our contract manufacturers, could be subject to periodic unannounced inspections by the FDA to monitor and ensure compliance with cGMP and GCP. Application holders must further notify the FDA, and depending on the nature of the change, obtain FDA pre-approval for product and manufacturing changes. Application fees may apply to certain changes.
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In addition, later discovery of previously unknown adverse events or that the drug is less effective than previously thought or other problems with our products, manufacturers, or manufacturing processes, or failure to comply with regulatory requirements both before and after approval, may yield various results, including:
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Any of these events could prevent us from achieving or maintaining market acceptance of the particular products, or could substantially increase the costs and expenses of commercializing such product, which in turn could delay or prevent us from generating significant revenues from its sale. Any of these events could further have other material and adverse effects on our operations and business and could adversely impact our stock price and could significantly harm our business, financial condition, results of operations, and prospects.
The FDA’s policies may change and additional government regulations may be enacted that could prevent, limit, or delay regulatory approval of our product candidates or that could impose additional regulatory obligations on our products. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained and be subject to regulatory enforcement action.
Should any of the above actions take place, they could adversely affect our ability to achieve or sustain profitability. Further, the cost of compliance with post‑approval regulations may have a negative effect on our operating results and financial condition.
A variety of risks associated with international operations could materially adversely affect our business.
We are, and may become party to further agreements, pursuant to which we out-license our products outside of the United States. The Company also currently markets Sunosi in Canada. We expect that we will be subject to additional risks related to entering into international business relationships, including:
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These and other risks associated with our international operations may materially adversely affect our ability to attain or maintain profitable operations.
We are exposed to market risk from fluctuations in currency exchange rates and interest rates.
We operate in multiple jurisdictions, and virtually all sales are denominated in currencies of the local jurisdiction. Additionally, we have entered and may enter into business development transactions, borrowings, or other financial transactions that may give rise to currency and interest rate exposure.
Since we cannot, with certainty, foresee and mitigate against such adverse changes, fluctuations in currency exchange rates, interest rates, and inflation could negatively affect our business, cash flow, results of operations, financial condition, and prospects.
In order to mitigate against the adverse impact of these market fluctuations, we may from time to time enter into hedging agreements. While hedging agreements, such as currency options and forwards and interest rate swaps, may limit some of the exposure to exchange rate and interest rate fluctuations, such attempts to mitigate these risks may be costly and not always successful.
We will need to obtain FDA approval (and that of comparable foreign regulatory authorities) of any proposed product names, and any failure or delay associated with such approval may adversely affect our business.
Any name we intend to use for our product candidates will require approval from the FDA regardless of whether we have secured a formal trademark registration from the U.S. Patent and Trademark Office, or USPTO. The FDA typically conducts a review of proposed product names, including an evaluation of the potential for confusion with other product names. The FDA may also object to a product name if it believes the name inappropriately implies medical claims or contributes to an overstatement of efficacy. If the FDA objects to any of our proposed product names, we may be required to adopt alternative names for our product candidates. If we adopt alternative names, we would lose the benefit of any existing trademark applications for such product candidate and may be required to expend significant additional resources in an effort to identify a suitable product name that would qualify under applicable trademark laws, not infringe the existing rights of third parties, and be acceptable to the FDA. We may be unable to build a successful brand identity for a new trademark in a timely manner or at all, which would limit our ability to commercialize our product candidates.
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RISKS RELATED TO THE COMMERCIALIZATION OF OUR PRODUCTS
We face significant competition from other pharmaceutical and biotechnology companies, academic institutions, government agencies, and other research organizations. Our operating results will suffer if we fail to compete effectively.
The development and commercialization of new drug products is highly competitive. We face competition with respect to our current products and product candidates and will face competition with respect to any product candidates that we may seek to develop or commercialize in the future, from major pharmaceutical companies, specialty pharmaceutical companies, and biotechnology companies worldwide. There are a number of large pharmaceutical and biotechnology companies that currently market and sell products or are pursuing the development of products for the treatment of CNS disorders. Potential competitors also include academic institutions, government agencies, and other public and private research organizations that conduct research, seek patent protection, and establish collaborative arrangements for research, development, manufacturing, and commercialization.
Specifically, there are a large number of companies developing or marketing therapies for CNS disorders, including many major pharmaceutical and biotechnology companies. Among the companies that currently market or are developing therapies that, if approved, our product candidates would potentially compete with include: AbbVie Inc.; Amgen Inc.; Avadel Pharmaceuticals plc; Biogen Inc.; Eli Lilly and Company; H. Lundbeck A/S; Harmony Biosciences; Intra-Cellular Therapies, Inc.; Janssen; Jazz; Otsuka Pharmaceutical Co. Ltd.; Pfizer Inc.; Relmada Therapeutics Inc.; Sage Therapeutics, Inc.; and Takeda Pharmaceutical Company Limited.
Our commercial opportunities could be reduced or eliminated if our competitors develop and commercialize products that are safer, are more effective, have fewer or less severe side effects, are more convenient, or are less expensive than any products that we may develop. Our competitors also may obtain FDA or other regulatory approval for their products more rapidly than we may obtain approval for ours, which could result in our competitors establishing a strong market position before we are able to enter the market. In addition, our ability to compete may be affected in many cases by insurers or other third‑party payors seeking to encourage the use of generic products or therapeutically similar lower cost brands. If our product candidates achieve marketing approval, we expect that they will be priced at a significant premium over competitive generic products, which would further impact our commercialization efforts.
Generic forms of the active ingredients of our product candidates, including dextromethorphan, bupropion, meloxicam, rizatriptan, and reboxetine, are available in the United States and abroad and could be used off-label. Any such off-label use could adversely affect our profitability and have a negative effect on our operating results and financial condition. For example, even though meloxicam is not currently approved for the treatment of acute migraine, we would not be able to prevent a physician from prescribing it for such treatment. Nor could we prevent a payor from offering favorable coverage for such product and disadvantaging our product candidates, even if the generics would be used off-label.
Many of the companies against which we are competing or against which we may compete in the future have significantly greater financial resources and expertise in research and development, manufacturing, preclinical testing, conducting clinical trials, obtaining regulatory approvals, and marketing approved products than we do. Mergers and acquisitions in the pharmaceutical and biotechnology industries may result in even more resources being concentrated among a smaller number of our competitors. Early-stage companies may also prove to be significant competitors, particularly through collaborative arrangements with, or acquisition by large and established companies. These third parties compete with us in recruiting and retaining qualified scientific and management personnel and establishing clinical trial sites and patient registration for clinical trials, as well as in acquiring technologies complementary to, or necessary for, our programs.
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If the FDA or comparable foreign regulatory authorities approve generic or similar versions of any of our products that receive marketing approval, or such authorities do not grant our products appropriate periods of exclusivity before approving generic or similar versions of our products, the sales of our products could be adversely affected.
Once an NDA is approved, the covered product becomes a “reference listed drug” in the FDA’s Orange Book. Manufacturers may seek approval of generic versions of reference listed drugs through submission of ANDAs in the United States. In support of an ANDA, a generic manufacturer need not conduct full clinical studies. Rather, the applicant generally must show that its product has the same active ingredient(s), dosage form, strength, route of administration, and conditions of use or labeling, among other commonalities, as the reference listed drug and that the generic version is bioequivalent to the reference listed drug, meaning it is absorbed in the body at the same rate and to the same extent. For example, in February 2023, we received a paragraph IV certification notice letter from Teva providing notification to the Company that Teva has submitted an ANDA to the FDA seeking approval to manufacture, use, or sell a generic version of Auvelity. Additionally, beginning in August 2023, we received paragraph IV certification notice letters from six other pharmaceutical companies providing notification to the Company that each such filer has submitted an ANDA to the FDA seeking approval to manufacture, use, or sell a generic version of Sunosi.
Recently, the FDA and Congress have also taken steps to encourage increased generic drug competition in the market. Generic products may be significantly less costly to bring to market than the reference listed drug and companies that produce generic products are generally able to offer them at lower prices and are generally preferred by third‑party payors. Thus, following the introduction of a generic drug, a significant percentage of the sales of any branded product or reference listed drug is typically lost to the generic product.
Moreover, in addition to generic competition, we could face competition from other companies seeking approval of drug products that are similar to ours using the 505(b)(2) pathway. Such applicants may be able to rely on our products, or other approved drug products or published literature to develop drug products that are similar to ours. The introduction of a drug product similar to our product candidates could expose us to increased competition.
Further, if we do not file a patent infringement lawsuit against a generic manufacturer within 45 days of receiving notice of its paragraph IV certification, the ANDA or 505(b)(2) applicant may not be subject to a 30‑month stay. Litigation or other proceedings to enforce or defend intellectual property rights are often very complex in nature, may be expensive and time consuming, may divert our management’s attention from our core business, and may result in unfavorable results that could adversely impact our ability to prevent third parties from competing with our products. Accordingly, we may be subject to generic competition or competition from similar products, or may need to commence patent infringement proceedings, which would divert our resources.
Competition that our products may face from generic or similar versions of our products could materially and adversely impact our future revenue, profitability, and cash flows and substantially limit our ability to obtain a return on the investments we have made in our product candidates.
AXS-12 received Orphan Drug Designation from the FDA. However, there is no guarantee that we will receive this designation for any of our other product candidates or receive or maintain any corresponding benefits for any of our other product candidates that may receive Orphan Drug Designation in the future, including periods of exclusivity.
AXS-12 received Orphan Drug Designation from FDA for the treatment of narcolepsy. We may also seek Orphan Drug Designation for our other products, as appropriate.
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Orphan Drug Designation, however, may be lost if the indications for which we develop any of our future product candidates do not meet the orphan drug criteria. Moreover, following product approval, orphan drug exclusivity may be lost if the FDA determines, among other reasons, that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the product to meet the needs of patients with the rare disease or condition. Even if we obtain orphan drug exclusivity for any of our current or future product candidates, that exclusivity may not effectively protect the product from competition because different products can be approved for the same condition. Even after an orphan product is approved, the FDA can subsequently approve a product containing the same principal molecular features for the same condition if the FDA concludes that the later product is clinically superior in that it is shown to be safer or more effective or makes a major contribution to patient care.
The FDA or the EMA may grant orphan exclusivity to two different sponsors for the same compound or active molecule and for the same indication. For example, if another sponsor receives FDA approval for a reboxetine containing product for the treatment of narcolepsy before we obtain FDA approval for AXS-12 for the treatment of narcolepsy, we would be prevented from launching our product in the United States for this indication for a period of at least 7 years. If another sponsor receives EMA approval for a reboxetine containing product for the treatment of narcolepsy before we obtain EMA approval for AXS-12 for the treatment of narcolepsy, we would be prevented from launching our product in the EU for this indication for a period of at least 10 to 12 years.
The FDA may undertake a reevaluation of aspects of its orphan drug regulations and policies at any time and may possibly do so in response to a recent court decision regarding the plain meaning of the exclusivity provision of the Orphan Drug Act. We do not know if, when, or how the FDA may change the orphan drug regulations and policies, and it is uncertain how any changes might affect our business. Depending on what changes the FDA may make to its orphan drug regulations and policies, our business, financial condition, results of operations, and prospects could be harmed.
If we are unable to establish effective marketing, sales and distribution capabilities or enter into agreements with third parties to market, sell and distribute our products, we may be unable to generate substantial product revenues.
We recently expanded our commercial infrastructure for the marketing, sale, and distribution of pharmaceutical products, which included the creation of a sales force to launch our commercial stage products throughout the United States. This requires additional compliance with a range of federal and state laws. Additionally, we currently commercialize Sunosi outside the United States. Each global market we commercialize Sunosi in has its own set of applicable laws.
We have limited experience in the marketing, sale, and distribution of pharmaceutical products, and there are significant risks involved in the building and managing of a commercial infrastructure. We have to compete with other pharmaceutical and biotechnology companies to recruit, hire, train, manage, and retain marketing and sales personnel. In the event we are unable to maintain our marketing and sales infrastructure, we may not be able to successfully commercialize any of our existing commercial stage products or future product candidates, which would limit our ability to generate revenue. Factors that may inhibit our efforts to commercialize any of our products on our own include:
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If additional product candidates are approved, we may incur expenses prior to product launch in expanding our sales force and compliant marketing and sales infrastructure. If a commercial launch is delayed as a result of FDA requirements or other reasons, we may incur these expenses prior to being able to realize any revenue from sales of such product candidate(s). Furthermore, our sales force and marketing teams may not be successful in commercializing any of our current or future product candidates.
If any of our products do not achieve broad market acceptance, the revenues that we generate from their sales will be limited.
Our products, and, if approved, our product candidates, may not gain acceptance among physicians, patients, third-party payors, and others in the medical community. If any of our products or product candidates, for which we obtain regulatory approval, do not gain an adequate level of market acceptance, we may not generate significant product revenues or become profitable. Market acceptance of any of our products by the medical community, patients, and third-party payors will depend on a number of factors, some of which are beyond our control. For example, physicians are often reluctant to switch their patients from existing therapies even when new and potentially more effective or convenient treatments enter the market. Further, patients often acclimate to the therapy that they are currently taking and do not want to switch unless their physicians recommend switching products or they are required to switch therapies due to lack of reimbursement for existing therapies. Even if physicians prescribe our products, third-party payors may not consider them cost effective without a significant price concession, which could negatively impact our revenue. Third-party payors may also implement onerous access controls, which could further impede our efforts to effectively transition eligible patients to our therapies.
Efforts to educate the medical community and third-party payors on the benefits of our products may require significant resources and may not be successful. If any of our product candidates is approved but does not achieve an adequate level of market acceptance, we may not generate significant revenues and we may not become profitable. The degree of market acceptance of any of our products will depend on a number of factors, including:
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Our efforts to educate the medical community and third‑party payors on the benefits of our products may require significant resources and may never be successful. Even if the medical community accepts that one of our product candidates is safe and effective for its approved indications, physicians and patients may not immediately be receptive to such product candidate and may be slow to adopt it as an accepted treatment of the approved indication. It is unlikely that any labeling approved by the FDA will contain claims that one of our product candidates is safer or more effective than competitive products or will permit us to promote such product candidate as being superior to competing products.
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The potential market opportunities for our products and/or product candidates are difficult to precisely estimate. Our estimates of the potential market opportunities are predicated on many assumptions including industry knowledge and publications, third‑party research reports, and other surveys. While we believe that our internal assumptions are reasonable, these assumptions involve the exercise of significant judgment on the part of our management and are inherently uncertain, and the reasonableness of these assumptions has not been assessed by an independent source. If any of the assumptions proves to be inaccurate, the actual markets for our product candidates could be smaller than our estimates of the potential market opportunities.
We face potential product liability exposure, and if successful claims are brought against us, we may incur substantial liability and may have to limit our products’ commercialization.
The use of any of our current or future product candidates in clinical trials, and the sale of any of our products exposes us to the risk of product liability claims. We face inherent risk of product liability related to the testing of our product candidates in human clinical trials and face an even greater risk for our commercialized products. For example, we may be sued if any products we develop allegedly causes injury or is found to be otherwise unsuitable during clinical testing, manufacturing, marketing, or sale. Any such product liability claims may include allegations of defects in manufacturing, defects in design, a failure to warn of dangers inherent in the product, negligence, strict liability or a breach of warranties. Claims could also be asserted under state consumer protection acts. Product liability claims might be brought against us by consumers, healthcare providers, or others using, administering, or selling our products. If we cannot successfully defend ourselves against these claims, we will incur substantial liabilities or be required to limit commercialization of our products. Even successful defense would require significant financial and management resources. Regardless of merit or eventual outcome, liability claims may result in loss of revenue from including from:
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We have obtained limited product liability insurance coverage for our products and our clinical trials. We have also obtained local policies in those foreign jurisdictions where it was appropriate. However, our insurance coverage may not reimburse us or may not be sufficient to reimburse us for any expenses or losses we may suffer. Moreover, insurance coverage is becoming increasingly expensive, and, in the future, we may not be able to maintain insurance coverage at a reasonable cost or in sufficient amounts to protect us against losses due to liability. On occasion, large judgments have been awarded in class action lawsuits based on drugs that had unanticipated side effects. A successful product liability claim or series of claims brought against us could cause our stock price to fall and, if judgments exceed our insurance coverage, could decrease our cash and adversely affect our business and our prospects.
Sunosi is a controlled substance and may be subject to U.S. federal and state controlled substance laws and regulations, and our failure to comply with these laws and regulations, or the cost of compliance with these laws and regulations, could materially and adversely affect our business, results of operations, financial condition and growth prospects.
Sunosi contains controlled substances as defined in the Federal Controlled Substances Act, or CSA. Controlled substances are subject to a number of requirements and restrictions under the CSA and implementing regulations, including certain registration, security, recordkeeping, reporting, import, export and other requirements administered by the U.S. Drug Enforcement Administration, or DEA. The DEA classifies controlled substances into five schedules: Schedule I, II, III, IV or V substances. Schedule I substances by definition have a high potential for abuse, no currently “accepted medical use” in the U.S., lack accepted safety for use under medical supervision, and may not be prescribed, marketed or sold in the U.S. Pharmaceutical products approved for use in the U.S. which contain a controlled substance are listed as Schedule II, III, IV or V, with Schedule II substances considered to present the highest potential for abuse or dependence and Schedule V substances the lowest relative risk of abuse among such substances. Schedule I and II drugs are subject to the strictest controls under the CSA, including manufacturing and procurement quotas, heightened security requirements and additional criteria for importation. In addition, dispensing of Schedule II drugs is further restricted. For example, they may not be refilled without a new prescription. Sunosi is a Schedule IV controlled substance.
Individual states have also established controlled substance laws and regulations. Though state-controlled substances laws often mirror federal law, they may separately schedule our products or our product candidates as well. We or our partners may also be required to obtain separate state registrations, permits or licenses in order to be able to manufacture, distribute, administer or prescribe controlled substances for clinical trials or commercial sale, and failure to meet applicable regulatory requirements could lead to enforcement and sanctions by the states in addition to those from the DEA or otherwise arising under federal law.
U.S facilities conducting research, manufacturing, distributing, importing or exporting, or dispensing controlled substances must be registered (licensed) to perform these activities and must comply with the security, control, recordkeeping and reporting obligations under the CSA, DEA regulations and corresponding state requirements. DEA and state regulatory bodies conduct periodic inspections of certain registered establishments that handle controlled substances. Obtaining and maintaining the necessary registrations and complying with the regulatory obligations may result in delay of the importation, manufacturing, distribution or clinical research of our products and products candidates. Furthermore, failure to maintain compliance with the CSA and DEA and state regulations by us or any of our contractors, distributors or pharmacies can result in regulatory action that could have a material adverse effect on our business, financial condition and results of operations. DEA and state regulatory bodies may seek civil penalties, refuse to renew necessary registrations, or initiate proceedings to restrict, suspend or revoke those registrations. In certain circumstances, violations could lead to criminal penalties. Any penalties imposed by the DEA to us or our third-party manufacturers which could have a material adverse effect on our business, results of operations, financial condition and growth prospects.
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RISKS RELATED TO OUR DEPENDENCE ON THIRD PARTIES
We rely, and expect to continue to rely, on third parties to perform many essential services for our products and product candidates, including services related to our preclinical studies and clinical trials, warehousing and inventory control, distribution, government price reporting, customer service, and adverse event reporting. If these third parties fail to perform satisfactorily, including by failing to meet deadlines for the completion of our preclinical studies and clinical trials, or fail to comply with legal and regulatory requirements, our ability to commercialize any of our products will be significantly impacted and we may be subject to regulatory sanctions.
We rely on third‑parties to conduct, supervise, and monitor our preclinical studies and certain clinical trials for our product candidates and do not currently plan to independently conduct preclinical studies or clinical trials of any other potential product candidates. We expect to continue to rely on third parties, such as CROs, clinical data management organizations, medical institutions, and clinical investigators, to conduct our preclinical studies and clinical trials. While we have agreements governing their activities, we have limited influence over their actual performance and control only certain aspects of their activities. The failure of these third parties to successfully carry out their contractual duties or meet expected deadlines could substantially harm our business because we may not obtain marketing approval for or commercialize our product candidates in a timely manner or at all. Moreover, these agreements might terminate for a variety of reasons, including a failure to perform by the third parties. If we need to enter into alternative arrangements, that could delay our product development activities and adversely affect our business.
Our reliance on these third parties for development activities will reduce our control over these activities. Nevertheless, we are responsible for ensuring that each of our studies is conducted in accordance with the applicable protocol, legal, regulatory, and scientific standards and our reliance on third parties does not relieve us of our regulatory responsibilities. For example, we will remain responsible for ensuring that each of our clinical trials is conducted in accordance with the general investigational plan and protocols for the trial and for ensuring that our preclinical trials are conducted in accordance with good laboratory practice, or GLP, as appropriate. Moreover, the FDA and comparable foreign regulatory authorities require us to comply with standards, such as GCP for conducting, monitoring, recording, and reporting the results of clinical trials to assure that data and reported results are credible and accurate and that the rights, integrity, and confidentiality of trial participants are protected. As a clinical trial sponsor, we also have regulatory requirements that directly apply to us. Regulatory authorities enforce these requirements through periodic inspections of trial sponsors, clinical investigators, and trial sites. If we or any of the third parties we engage fail to comply with applicable GCP, we or those third parties may be subject to enforcement or other legal actions, the clinical data generated in our clinical trials may be deemed unreliable and the FDA or comparable foreign regulatory authorities may require us to perform additional clinical trials.
In addition, when we submit an NDA for review, we are required to report certain financial interests of our third‑party investigators if these relationships exceed certain financial thresholds or meet other criteria. The FDA and comparable foreign regulatory authorities may question the integrity of the data from those clinical trials conducted by investigators who previously served or currently serve as scientific advisors or consultants to us from time to time and receive cash compensation in connection with such services or otherwise receive compensation from us that could be deemed to impact study outcome, proprietary interests in a product candidate, certain company equity interests, or significant payments of other sorts.
We cannot assure you that upon inspection by a given regulatory authority, such regulatory authority will determine that any of our clinical trials complies with GCP regulations. In addition, our clinical trials must be conducted with product candidates that were produced under cGMP regulations. Our failure to comply with these regulations may require us to repeat clinical trials, which would delay the regulatory approval process. We also are required to register certain clinical trials and post the results of certain completed clinical trials on a government sponsored database, ClinicalTrials.gov, within specified timeframes. Failure to do so or to meet the related submission requirements can result in enforcement actions, including civil monetary penalties and adverse publicity.
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Third parties we engage to conduct research may also have relationships with other entities, some of which may be our competitors, for whom they may also be conducting clinical trials or other drug development activities that could harm our competitive position. In addition, these third parties are not our employees, and except for remedies available to us under our agreements with such third parties, we cannot control whether or not they devote sufficient time and resources to our ongoing clinical, non-clinical, and preclinical programs. If these third parties do not successfully carry out their contractual duties, meet expected deadlines or conduct our preclinical studies or clinical trials in accordance with regulatory requirements or our stated protocols, if they need to be replaced or if the quality or accuracy of the clinical data they obtain is compromised due to the failure to adhere to our clinical protocols or regulatory requirements or for other reasons, our clinical trials may be extended, delayed, or terminated and we may not be able to obtain, or may be delayed in obtaining, marketing approvals for our product candidates and will not be able to, or may be delayed in our efforts to, successfully commercialize our product candidates, or we or they may be subject to regulatory enforcement actions. As a result, our results of operations and the commercial prospects for our product candidates would be harmed, our costs could increase and our ability to generate revenues could be delayed. To the extent we are unable to successfully identify and manage the performance of third-party service providers in the future, our business may be materially and adversely affected.
If any of our relationships with these third parties terminates, we may not be able to enter into arrangements with alternative resources or to do so on commercially reasonable terms. Switching or adding additional third parties involves additional cost and requires management time and focus. In addition, there is a natural transition period when a new third party commences work. As a result, delays could occur, which could compromise our ability to meet our desired development timelines. Though we carefully manage our relationships with these third-party vendors, there can be no assurance that we will not encounter similar challenges or delays in the future or that these delays or challenges will not have a material adverse impact on our business, financial condition and prospects, and results of operations.
If the manufacturers upon whom we rely fail to produce our products in the volumes that we require on a timely basis, or to comply with stringent regulations applicable to pharmaceutical drug manufacturers, we may face delays in the development and commercialization of, or be unable to meet demand for, our products and may lose potential revenues.
We do not manufacture any of our products, and we do not currently plan to develop any capacity to do so. We currently outsource all manufacturing of our products to third parties typically without any guarantee that there will be sufficient supplies to fulfill our requirements or that we may obtain such supplies on acceptable terms. Any delays in obtaining adequate supplies with respect to our products may delay or disrupt the development or commercialization of our products. Moreover, we do not yet in all cases have agreements established regarding commercial supply of our product candidates, and we may not be able to establish or maintain commercial manufacturing arrangements on commercially reasonable terms for any of our current or future product candidates for which we obtain approval in the future.
We may not succeed in our efforts to establish manufacturing relationships or other alternative arrangements for any of our existing or future products and programs. Our products may compete with other products and product candidates for access to manufacturing facilities. There are a limited number of manufacturers that operate under cGMP regulations and that are both capable of manufacturing for us and willing to do so. If our existing third‑party manufacturers, or the third parties that we engage in the future to manufacture a product for commercial sale or for our clinical trials, should cease to continue to do so for any reason, we likely would experience delays in obtaining sufficient quantities of our product for us to meet commercial demand or to advance our clinical trials while we identify and qualify replacement suppliers. If for any reason we are unable to obtain adequate supplies of our products or the drug substances used to manufacture them, it will be more difficult for us to develop our products and compete effectively. Further, even if we do establish such collaborations or arrangements, our third‑party manufacturers may breach, terminate, or not renew these agreements.
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Any problems or delays we experience in preparing for commercial‑scale manufacturing of a product candidate may result in a delay in FDA approval of the product candidate or may impair our ability to manufacture commercial quantities or such quantities at an acceptable cost, which could result in the delay, prevention, or impairment of clinical development and commercialization of our product candidates and could adversely affect our business. For example, our manufacturers will need to produce specific batches of our product candidates to demonstrate acceptable stability under various conditions and for commercially viable lengths of time. We and our contract manufacturers will need to demonstrate to the FDA and other regulatory authorities that this is acceptable stability data for our product candidates, as well as validate methods and manufacturing processes, in order to receive regulatory approval to commercialize any of our current or future product candidates. Furthermore, if our commercial manufacturers fail to deliver the required commercial quantities of bulk drug substance or finished product on a timely basis and at commercially reasonable prices, we would likely be unable to meet demand for our products and we would lose potential revenues.
We have a limited number of contract manufacturers for our products. At times we may have only one manufacturer for a product. In addition, we do not have any long‑term commitments from our suppliers of clinical trial material or guaranteed prices for our product candidates. The manufacture of pharmaceutical products requires significant expertise and capital investment, including the development of advanced manufacturing techniques and process controls. Manufacturers of pharmaceutical products often encounter difficulties in production, particularly in scaling up initial production. These problems include difficulties with production costs and yields; quality control, including stability of the product candidate and quality assurance testing; shortages of qualified personnel; and compliance with strictly enforced federal, state, and foreign regulations. Our manufacturers may not perform as agreed. If our manufacturers were to encounter any of these difficulties, our ability to provide product candidates to patients in our clinical trials and for commercial use, if approved, would be jeopardized.
In addition, all manufacturers of our products must comply with cGMP requirements enforced by the FDA and comparable foreign regulatory authorities that are applicable to both finished drug products and active pharmaceutical ingredients used both for clinical and commercial supply, through its facilities inspection program. The FDA must verify our contract manufacturers’ compliance with cGMP requirements and comparable foreign regulatory authorities will similarly inspect our contract manufacturers’ facilities after we submit our marketing applications to the agency and comparable foreign regulatory authorities. The cGMP requirements include quality control, quality assurance, and the maintenance of records and documentation. Manufacturers of our products may be unable to comply with our specifications, these cGMP requirements and with other FDA, state, and foreign regulatory requirements. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA or other regulatory authorities, they will not be able to secure or maintain regulatory approval for their manufacturing facilities. While we are ultimately responsible for the manufacture of our products, other than through our contractual arrangements, we have little control over our manufacturers’ compliance with these regulations and standards. If the FDA or a comparable foreign regulatory authority does not approve these facilities for the manufacture of our products or if it withdraws any such approval in the future, we may need to find alternative manufacturing facilities, which would significantly impact our ability to develop or market our products, or obtain regulatory approval for, our product candidates. A failure to comply with these requirements may result in regulatory enforcement actions against our manufacturers or us, including fines and civil and criminal penalties, including imprisonment; suspension or restrictions of production; suspension, delay, or denial of product approval or supplements to approved products; clinical holds or termination of clinical studies; warning or untitled letters; regulatory authority communications warning the public about safety issues with the drug; refusal to permit the import or export of the products; product seizure, detention, or recall; suits under the civil False Claims Act; corporate integrity agreements; consent decrees; or withdrawal of product approval. If the safety of any quantities supplied is compromised due to our manufacturers’ failure to adhere to applicable laws or for other reasons, we may not be able to obtain regulatory approval for our product candidates or successfully commercialize our products.
Any failure or refusal to supply our products or components for our current or future product candidates that we may develop could delay, prevent, or impair our clinical development or commercialization efforts. Any change in our manufacturers could be costly because the commercial terms of any new arrangement could be less favorable and because the expenses relating to the transfer of necessary technology and processes could be significant.
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As an NDA applicant and commercial “virtual manufacturer,” we may rely in many cases on third parties to perform many essential services for our products, including services related to warehousing and inventory control, distribution, government price reporting, customer service, and adverse event reporting. If these third parties fail to perform as expected or to comply with legal and regulatory requirements, our ability to commercialize any of our products will be significantly impacted and we may be subject to regulatory sanctions.
We have retained third-party service providers to perform a variety of functions related to the sale and distribution of our products, key aspects of which are out of our direct control. These service providers provide key services related to warehousing and inventory control, distribution, government price reporting, and customer service, and, as a result, much of our inventory is stored at a single warehouse maintained by one such service provider. We substantially rely on this service provider as well as other third-party providers that perform services for us, including entrusting our inventories of products to their care and handling. If these third-party service providers fail to comply with applicable laws and regulations, fail to meet expected deadlines, or otherwise do not carry out their contractual duties to us, or encounter physical or natural damage at their facilities, our ability to deliver product to meet commercial demand would be significantly impaired and we may be subject to regulatory enforcement action.
In addition, we may engage third parties to perform various other services for us relating to adverse event reporting, safety database management, fulfillment of requests for medical information regarding our products and related services. If the quality or accuracy of the data maintained by these service providers is insufficient, or these third parties otherwise fail to comply with regulatory requirements related to adverse event reporting, we could be subject to regulatory sanctions.
Additionally, if a third party errs in calculating government pricing information from transactional data in our financial records, it could impact our discount and rebate liability and potentially cause government programs to overpay providers for our products, which could expose us to significant False Claims Act liability and other civil monetary penalties.
Any collaboration arrangements that we are a party to or may enter into in the future may not be successful, which could adversely affect our ability to develop and commercialize our product candidates.
Our business model is to commercialize our product candidates in the United States, and we may either commercialize products outside the United States ourselves or collaborate with pharmaceutical or biotechnology companies, or academic institutions, for the development or commercialization of our product candidates in the rest of the world. For example, we currently commercialize Sunosi in Canada. In February 2023, we announced a licensing transaction with Pharmanovia to market Sunosi in Europe and certain countries in the Middle East / North Africa. Our current and future collaboration arrangements may not be successful, and the success of them will depend heavily on the efforts and activities of our collaborators. Collaborators generally have significant discretion in determining the efforts and resources that they will apply to these collaboration arrangements. For clinical trials of our product candidates being conducted by our collaborators, for example, the Phase 2 clinical trial of AXS-05 for smoking cessation in collaboration with Duke University, we relied on timeline estimates provided by our collaborators for these trials. Such timeline estimates may differ materially from actual trial completion dates. Disagreements between parties to a collaboration arrangement regarding clinical development and commercialization matters can lead to delays in the development process or commercializing the applicable product candidate and, in some cases, termination of the collaboration arrangement. These disagreements can be difficult to resolve if neither of the parties has final decision-making authority.
We may license the right to market and sell our products under our collaborators’ labeler codes. Alternatively, we may enter into agreements with collaborators to market and sell our products under our own labeler code, in which case errors and omissions by collaborators in capturing and transmitting transactional data may impact the accuracy of our government price reporting.
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Collaborations with pharmaceutical companies and other third parties often are terminated or allowed to expire by the other party. Any such termination or expiration would adversely affect us financially and could harm our business reputation. Any future collaborations we might enter into may pose a number of risks, including:
If any collaborations we might enter into in the future do not result in the successful development and commercialization of products or if one of our collaborators subsequently terminates its agreement with us, we may not receive any future research funding or milestone or royalty payments under the collaboration. If we do not receive the funding we expect under the agreements, our development of our product candidates could be delayed and we may need additional resources to develop our product candidates and our product platform.
Additionally, if any future collaborator of ours is involved in a business combination, the collaborator might deemphasize or terminate development or commercialization of any product candidate licensed to it by us. If one of our collaborators terminates its agreement with us, we may find it more difficult to attract new collaborators and our reputation in the business and financial communities could be adversely affected.
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RISKS RELATED TO INTELLECTUAL PROPERTY
It is difficult and costly to protect our proprietary rights, and, as a result, we may not be able to ensure their protection. In addition, patents have a limited lifespan and will eventually expire.
Market exclusivity awarded by the FDA upon the approval of an NDA is limited in scope and duration. For example, our New Chemical Entity exclusivity for Sunosi expired on June 17, 2024 with an Orphan Drug Exclusivity relating to the product’s narcolepsy indication expiring on June 17, 2026. For Auvelity, the New Product Exclusivity expires on August 18, 2025. Neither of these expiry dates take into account the effect of the statutory 30-month stay should we timely commence litigation against any generic filer. A generic filer may be permitted to launch a generic version of either of our products following expiry of these exclusivities if our patents do not preclude a generic launch. Patent litigation is inherently uncertain, and we cannot guarantee the outcome of any such proceedings or that we would succeed in stopping the “at risk” launch of a generic version of either of our currently commercialized products during the pendency of litigation following expiry of the 30-month stay. Such a generic launch could materially impact our commercial success.
We seek to protect intellectual property relating to our products and portfolio products by prosecuting patents in the United States and elsewhere. The patent prosecution process is expensive and time consuming, and we may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. It is also possible that we will fail to identify patentable aspects of our research and development output before it is too late to obtain patent protection. Moreover, should we enter into additional collaborations we may be required to consult with or cede control to collaborators regarding the prosecution, maintenance, and enforcement of our patent applications and patents. Therefore, these patents and patent applications may not be prosecuted and enforced in a manner consistent with the best interests of our business. The patent positions of pharmaceutical and biotechnology companies can be highly uncertain and involve complex legal and factual questions for which important legal principles remain unresolved. No consistent policy regarding the breadth of claims allowed in pharmaceutical or biotechnology patents has emerged to date in the United States. The patent situation outside the United States is even more uncertain. Changes in either the patent laws or in interpretations of patent laws in the United States and other countries may diminish the value of our intellectual property. Accordingly, we cannot predict the breadth of claims that may be allowed or enforced in our patents and patent applications or in third‑party patents and patent applications. The degree of future protection for our proprietary rights is uncertain because legal means afford only limited protection and may not adequately protect our rights or permit us to gain or keep our competitive advantage. Moreover, the patent application process is also subject to numerous risks and uncertainties, and there can be no assurance that we or any of our future development partners will be successful in protecting any of our current or future product candidates that we may develop, license, or acquire by obtaining and defending patents. For example:
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Patents have a limited lifespan. In most countries, including the United States, the expiration of a patent is typically 20 years from the date that the application for the patent is filed or 20 years from the earliest non-provisional filing date to which priority is claimed if the patent is granted from a continuing application (e.g., continuation, divisional, or continuation-in-part). Various extensions of patent term may be available in particular countries; however, in all circumstances the life of a patent, and the protection it affords, has a limited term. If we encounter delays in obtaining regulatory approvals, the period of time during which we could market a product under patent protection could be reduced. We expect to seek extensions of patent terms where these are available in any countries where we are prosecuting patents. Such possible extensions include those permitted under the Drug Price Competition and Patent Term Restoration Act of 1984 in the United States, which permits a patent term extension of up to five years to cover an FDA-approved product. The actual length of the extension will depend on the amount of patent term lost while the product was in clinical trials. However, the applicable authorities, including the U.S. Patent and Trademark Office, or USPTO and the FDA in the United States, and any equivalent regulatory authority in other countries, may not agree with our assessment of whether such extensions are available, and may refuse to grant extensions to our patents, or may grant more limited extensions than we request. If this occurs, our competitors may be able to take advantage of our investment in development and clinical trials by referencing our clinical and preclinical data, and then may be able to launch their product earlier than might otherwise be the case.
Patent reform legislation could increase the uncertainties and costs surrounding the prosecution of our patent applications and the enforcement or defense of our issued patents.
2011年9月16日，《萊希-史密斯美國發明法》或《萊希-史密斯法案》簽署成爲法律。《萊希-史密斯法案》對美國專利法進行了多項重大修改。這些條款包括影響專利申請起訴方式的條款，也可能影響專利訴訟。特別是，根據《萊希-史密斯法案》，美國於2013年3月過渡到「先提交專利」制度，在這種制度下，第一個提交專利申請的發明人將有權獲得專利。第三方被允許在美國專利商標局頒發專利之前提交現有技術，並可能參與授權後程序，包括重新審查、授權後審查、各方之間的審查，或質疑我們的專利權或其他人的專利權的派生或干擾程序。任何此類提交、訴訟或訴訟中的不利裁決可能會縮小我們專利權的範圍或可執行性，或使其無效，從而可能對我們的競爭地位產生不利影響。美國和/或美國以外司法管轄區未來的專利改革立法可能會進一步增加圍繞我們專利申請的起訴以及我們已頒發專利的執行或保護的不確定性和成本。
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獲得和維持我們的專利保護取決於是否遵守政府專利機構施加的各種程序、文件、費用支付和其他要求，如果不遵守這些要求，我們的專利保護可能會減少或取消。
美國專利商標局和各種外國政府專利機構要求在專利起訴過程中遵守一些程序、文件、費用支付和其他類似條款。定期維護費、續期費、年金費以及與專利或專利申請有關的各種其他政府費用將在專利和申請的有效期內分幾個階段支付給美國專利商標局和美國以外的各種專利機構。我們有提醒我們支付這些費用的系統，我們僱用並依賴信譽良好的律師事務所和其他專業人士向USPTO和非美國專利代理機構支付我們擁有的專利和專利申請以及我們授權的專利和專利申請的這些費用。我們還聘請信譽良好的律師事務所和其他專業人員來幫助我們遵守關於我們擁有的專利和專利申請以及我們授權的專利和專利申請的各種文件和其他程序要求。在某些情況下，疏忽可以通過支付滯納金或根據適用規則的其他方式予以補救。然而，在某些情況下，不遵守規定可能會導致專利或專利申請的放棄或失效，從而導致相關管轄區的專利權部分或全部喪失。在這種情況下，我們的競爭對手可能會進入市場，這種情況將對我們的業務產生實質性的不利影響。
如果我們或任何未來的合作伙伴因侵犯第三方知識產權而被起訴，成本高昂且耗時，任何訴訟的不利結果都將損害我們的業務。
我們開發、製造、營銷和銷售我們的任何產品的能力取決於我們避免侵犯第三方專有權利的能力，而我們的商業成功取決於我們和我們的合作者開發、製造、營銷和銷售我們的產品和使用我們的專有技術而不侵犯第三方專有權利的能力。在生物技術和製藥行業，有相當多的知識產權訴訟。在治療和管理中樞神經系統疾病的一般領域中，存在着大量由第三方擁有的美國和外國頒發的專利和正在申請的專利申請，涵蓋了我們目標市場中大量化合物和配方的使用。第三方可能會根據現有專利或未來可能授予的專利向我們提出侵權索賠。由於任何涉及專有權的專利或其他訴訟中固有的不確定性，我們和我們的許可人可能無法成功地捍衛第三方的知識產權索賠，這可能會對我們的業務、財務狀況、運營結果和前景產生實質性的不利影響。無論任何訴訟的結果如何，針對訴訟進行辯護可能是昂貴、耗時的，並且會分散管理層的注意力。此外，由於專利申請可能需要多年的時間才能發佈，因此可能會有我們不知道的當前正在處理的申請，這可能會導致我們當前或未來的任何產品可能會侵犯已頒發的專利。也可能存在我們不知道的現有專利，我們當前或未來的任何產品可能會無意中侵犯這些專利。
如果第三方聲稱我們侵犯了他們的知識產權，我們可能會面臨許多問題，包括：
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如果我們被發現侵犯了第三方的知識產權，我們可能被要求從該第三方獲得許可證，以繼續開發和營銷我們的產品和技術。然而，我們可能無法以商業上合理的條款獲得任何所需的許可證，或者根本無法獲得。即使我們能夠獲得許可，它也可能是非排他性的，從而使我們的競爭對手能夠訪問向我們許可的相同技術。我們可能會被迫停止將侵權技術或產品商業化，包括通過法院命令。此外，如果我們被發現故意侵犯專利，我們可能被判承擔金錢損害賠償責任，包括三倍損害賠償和律師費。侵權行爲的發現可能會阻止我們將產品商業化，或迫使我們停止一些業務運營，這可能會對我們的業務造成實質性損害。聲稱我們盜用了第三方的機密信息或商業祕密，可能會對我們的業務、財務狀況、運營結果和前景產生類似的負面影響。
我們可能會捲入訴訟以保護或執行我們的專利或我們許可者的專利，這可能會昂貴、耗時且不成功。
競爭對手可能會侵犯我們已頒發的專利、我們的許可內專利或我們擁有或許可內的其他知識產權。根據我們與Antecip的許可協議條款，如果我們認爲第三方侵犯了受許可約束的專利，我們有義務自費對這些第三方提起訴訟。爲了對抗侵權或未經授權的使用，我們可能會被要求提出侵權索賠，這可能是昂貴和耗時的。我們對被認定的侵權者提出的任何索賠都可能導致這些當事人對我們提出反訴，指控我們侵犯了他們的專利和/或向法院或美國專利商標局挑戰所主張的專利(S)的有效性(例如，在授權後程序中，如在美國專利商標局的專利審判和上訴委員會進行當事人間審查)。此外，在專利侵權或有效性訴訟中，決策者(例如法院或PTAB)可以決定我們的專利全部或部分無效或不可執行；狹義地解釋該專利的權利要求；或以我們的專利不涵蓋所涉技術爲由拒絕阻止另一方使用所涉技術。在USPTO的任何訴訟程序或相關程序中的不利結果可能會使我們的一項或多項專利面臨被無效或狹義解釋的風險。此外，由於知識產權訴訟需要大量的披露，在這類訴訟期間，我們的一些機密信息可能會因披露而被泄露。
我們的許多競爭對手比我們更大，並且擁有比我們更多的資源。因此，他們可能能夠比我們更長時間地承受複雜專利訴訟的成本。此外，與訴訟相關的不確定性可能會對我們籌集繼續臨床試驗所需資金、繼續我們的內部研究計劃、許可所需技術或建立開發合作伙伴關係的能力產生重大不利影響，以幫助我們將我們的候選產品推向市場。
我們已經許可並且未來可能需要許可第三方的某些知識產權。此類許可證可能無法提供或可能無法以商業上合理的條款提供。如果許可證因任何原因無法獲得或終止，我們的業務可能會受到重大損害。
我們是某些許可協議的一方，根據這些協議，我們被授予知識產權權利，包括對我們的業務非常重要的專利權。我們預計將來可能需要簽訂額外的許可協議才能將我們的產品商業化，在這種情況下，我們將被要求從更多的第三方獲得許可。此類許可證可能無法以商業上合理的條款獲得，或者根本無法獲得，這可能會對我們的業務、財務狀況、運營結果和前景造成嚴重損害。我們依賴這些許可證來使用知識產權，這些知識產權可能對我們的業務至關重要，對我們產品的開發或商業化也很重要或必要。我們現有的許可協議強加給我們，我們預計未來的許可協議將強加給我們各種排他性義務。如果我們未能履行我們在這些協議下的義務，適用的許可方可能有權終止我們的許可，在這種情況下，我們可能無法開發或商業化該許可涵蓋的產品。
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2020年1月，我們與輝瑞或輝瑞達成了一項協議，獲得輝瑞臨床和非臨床數據在美國的獨家許可，並獲得瑞波西汀的知識產權。瑞波西汀是Axome正在開發的用於治療發作性睡病的AXS-12的活性藥物成分。該協議還提供了Axome在美國開發和商業化Ereboxetine的獨家權利。Ereboxetine是一種新的晚期候選產品，現在被稱爲AXS-14，用於治療纖維肌痛。根據協議條款，我們從輝瑞獲得了輝瑞在美國的獨家許可，獲得了瑞波西汀和埃斯波西汀的輝瑞數據，包括全面的非臨床研究，以及涉及5000多名患者的短期和長期臨床試驗。獲得許可的數據包括埃斯波西汀治療纖維肌痛的陽性3期試驗和陽性2期試驗的結果。我們將擁有在美國開發AXS-14(埃斯波西汀)的獨家權利和獨家責任，用於治療纖維肌痛和其他適應症。輝瑞收到了82,019股我們的普通股，價值800美元萬，基於我們普通股在前10個交易日的平均收盤價97.538美元，作爲許可證和權利的代價。輝瑞還收到了300萬美元萬的預付現金，並將在監管和銷售里程碑中獲得高達32300美元的萬，並在未來的銷售中分級收取中位數至低兩位數的版稅。輝瑞還將擁有涉及AXS-12和AXS-14的任何潛在未來戰略交易的優先談判權。根據該協議，我們有義務使用商業上合理的努力在美國開發、製造和商業化這些化合物和產品，並尋求和保持對這些化合物和產品的監管批准。該協議將在此類產品的最後一個到期版稅期限到期時逐個產品到期。在到期(但不是更早終止)時，我們將根據許可的專利權和相關數據獲得永久、非獨家、全額支付、免版稅和不可撤銷的許可，以開發、製造、使用、商業化和以其他方式開發化合物。任何一方在一段治癒期後，對於另一方的實質性違約，均可終止協議。輝瑞可能會在發生與我們有關的某些破產事件時立即終止協議。我們可以在協議一週年後90天內隨時向輝瑞發出書面通知，以任何理由終止協議。如果與輝瑞的許可協議因任何原因終止，我們的業務、財務狀況、運營結果和前景都將受到實質性損害。
2012年，我們與Antecip BioVentures II LLC或Antecip簽訂了三項獨家許可協議，Antecip是由我們的首席執行官兼董事會主席Herriot Tabuteau萬.D.擁有的實體，根據協議，我們獲得了獨家許可，可以開發、製造和商業化Antecip的專利和與開發AXS-05相關的應用，以及世界上任何地方用於人類治療、獸醫和診斷用途的兩種目前未在開發中的候選產品。這些協議在2015年8月進行了修訂，以更新受許可協議約束的專利和申請的時間表。根據協議，我們需要使用商業上合理的努力來開發AXS-05，並獲得監管部門的批准，並將其商業化。根據協議條款，我們需要向Antecip支付相當於AXS-05的3.0%的特許權使用費，該使用費是我們、我們的關聯公司或被許可的分許可人銷售的包含許可技術的產品的淨銷售額的3.0%。這些特許權使用費最高可減少向第三方支付的任何所需費用的50.0%。除非一方當事人或我方出於方便而提前終止，否則協議將按產品和國家/地區保持有效，直至(1)適用產品在該國不再被有效索賠覆蓋或(2)適用產品在該國首次商業銷售後10年內發生。在與某個國家/地區的產品相關的協議到期後，我們在該國家/地區對該產品授予的許可將成爲全額支付、免版稅的永久非獨家許可。如果Antecip因故終止任何協議，或者如果我們出於方便行使我們的權利終止任何協議，則根據該終止協議授予我們的權利將恢復到Antecip。我們依賴與Antecip的許可協議，如果與Antecip的任何許可協議因任何原因終止，我們的業務、財務狀況、運營結果和前景將受到實質性損害。
在收購Sunosi方面，除了前期購買價格外，我們還承擔了與收購相關的某些責任，並同意就美國淨銷售額向Jazz支付不可退還、不可抵免的特許權使用費。Jazz在美國境外的淨銷售無需支付特許權使用費。此外，我們承擔了Jazz對Sk和Aerial的所有承諾。對Sk和Aerial的假設承諾包括個位數的分層特許權使用費以及某些銷售和開發里程碑。我們依賴這些協議，如果我們違反這些協議，我們的業務、財務狀況、運營業績和前景將受到重大損害。
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We may be subject to claims that our employees, independent contractors, or consultants have wrongfully used or disclosed alleged trade secrets of their former employers or other third parties.
As is common in the biotechnology and pharmaceutical industry, we employ individuals who were previously employed at other biotechnology or pharmaceutical companies, including our competitors or potential competitors. Although no claims against us are currently pending, we may be subject to claims that these individuals or we have inadvertently or otherwise used or disclosed trade secrets or other proprietary information of their former employers. Litigation may be necessary to defend against these claims. Even if we are successful in defending against these claims, litigation could result in substantial costs and be a distraction to management.
We may be unable to adequately prevent disclosure of trade secrets and other proprietary information.
我們依靠商業祕密來保護我們的專有技術進步和專有技術，特別是在我們認爲專利保護不合適或不可能獲得的情況下。然而，商業祕密很難保護。我們部分地依賴與我們的員工、顧問、承包商、外部科學合作者、贊助研究人員和其他顧問的保密協議，包括我們生產產品所依賴的第三方，以保護我們的商業祕密和其他專有信息。然而，與我們簽署此類協議的任何一方都可能違反該協議，泄露我們的專有信息，包括我們的商業祕密。因此，這些協議可能無法有效防止機密信息的披露，也可能無法在未經授權披露機密信息的情況下提供適當的補救措施。執行和確定我們專有權的範圍可能需要昂貴和耗時的訴訟。此外，其他人可能會獨立發現我們的商業祕密和專有信息。此外，作爲其透明度倡議的一部分，FDA目前正在考慮是否定期公開更多信息，包括我們可能認爲是商業祕密或其他專有信息的信息，目前尚不清楚FDA的披露政策未來可能會如何變化。如果不能獲得或維護商業祕密保護，競爭對手可能會利用我們的專有信息來開發與我們的產品競爭的產品，或者對我們的競爭業務地位和財務業績造成額外的重大不利影響。
我們或我們的許可人可能無法在世界各地保護我們的知識產權。
在世界所有國家提交、起訴和保護專利申請和產品專利的費用將高得令人望而卻步，而且我們在美國以外的一些國家的知識產權可能沒有美國那麼廣泛。此外，一些外國的法律對知識產權的保護程度不如美國的聯邦和州法律。因此，我們可能無法阻止第三方在美國以外的所有國家/地區實施我們的發明，或在美國或其他司法管轄區銷售或進口使用我們的發明製造的產品。競爭對手可以在我們沒有獲得專利保護的司法管轄區使用我們的技術來開發自己的產品，此外，還可以將其他侵權產品出口到我們擁有專利保護但執法權不如美國的地區。這些產品可能會與我們的產品競爭，而我們的專利或其他知識產權可能不能有效或不足以阻止他們競爭。
許多公司在外國司法管轄區保護和捍衛知識產權方面遇到了重大問題。某些國家的法律制度不支持專利的強制執行和其他知識產權保護，這可能會使我們難以從總體上阻止對我們專利的侵犯。在外國司法管轄區強制執行我們的專利權的訴訟程序可能會導致巨額成本，並將我們的努力和注意力從我們業務的其他方面轉移出去，可能會使我們的專利面臨被無效或狹義解釋的風險，可能會使我們的專利申請面臨無法發佈的風險，並可能引發第三方對我們提出索賠。我們可能不會在我們發起的任何訴訟中獲勝，所判給的損害賠償或其他補救措施(如果有的話)可能沒有商業意義。因此，我們在世界各地執行我們或我們許可人的知識產權的努力可能不足以從我們開發或許可的知識產權中獲得顯著的商業優勢。
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與法律和合規事項相關的風險
如果我們未能遵守聯邦、州和外國醫療保健法，包括欺詐和濫用、透明度和健康和其他數據保護、信息隱私和安全法，我們可能會面臨巨額處罰，我們的業務、財務狀況、運營業績和前景可能會受到不利影響。
作爲一家制藥公司，我們必須遵守許多聯邦和州醫療法律，包括在我們於2024年2月23日提交給美國美國證券交易委員會的10-k表格年度報告中「企業-政府監管和產品批准」部分描述的那些法律，例如聯邦反回扣法令、聯邦民事和刑事虛假索賠法、民事罰款法令、醫療補助藥品退稅法令和其他價格報告要求、1992年退伍軍人醫療法案、陽光法案、1977年外國腐敗行爲法、2010年患者保護和平價醫療法以及類似的州和外國法律。即使我們不會也不會控制醫療服務的轉介或直接向Medicare、Medicaid或其他第三方付款人收費，某些與欺詐和濫用以及患者權利有關的聯邦和州醫療法律法規現在和將來都適用於我們的業務。我們受到聯邦政府和我們開展業務的州的醫療欺詐和濫用法律的約束。
在聯邦和州一級還有許多其他法律以及立法和監管舉措來解決隱私和安全問題，一些州的隱私和安全法律的適用範圍比《健康保險可攜性和責任法》(HIPAA)及其實施條例更廣泛。例如，加利福尼亞州頒佈了立法-加州消費者隱私法，或CCPA-於2020年1月生效。CCPA除其他外，爲覆蓋的公司創造了數據隱私義務，併爲加州居民提供了新的隱私權，包括選擇不披露其信息的權利。CCPA還爲某些數據泄露創建了具有法定損害賠償的私人訴權，從而潛在地增加了與數據泄露相關的風險。此外，美國境內的許多數據隱私和安全法律具有並行管轄權，這可能會使我們受到多個機構根據多個法規對同一行爲的執行(例如，根據第5條執行FTC，根據HIPAA執行HHS-民權執法辦公室，以及州總檢察長因違反適用的州法律而提起的訴訟)。
此外，歐盟成員國和包括瑞士在內的其他外國司法管轄區通過了數據保護法律和法規，規定了重大的合規義務。此外，歐盟個人健康數據的收集和使用，以前由歐盟數據保護指令的條款管理，於2018年5月被歐盟一般數據保護條例或GDPR取代。GDPR的範圍很廣，對與個人數據有關的個人的同意、提供給個人的信息、個人數據的安全和保密、數據泄露通知以及在處理個人數據時使用第三方處理器等方面提出了幾項要求。GDPR還對將個人數據從歐盟轉移到美國實施了嚴格的規則，提供了執法機構，並對不遵守規定的公司施加了巨額處罰，包括可能被處以最高2000歐元的萬或不符合規定公司全球年收入的4%的罰款，以金額較大者爲準。GDPR要求不僅適用於第三方交易，也適用於我們與子公司之間的信息傳輸，包括員工信息。最近實施的GDPR增加了我們在處理個人數據方面的責任和責任，包括在臨床試驗中，我們未來可能需要建立額外的機制來確保遵守GDPR，這可能會轉移管理層的注意力並增加我們的業務成本。此外，有關數據隱私和安全的新法規或立法行動(連同適用的行業標準)可能會增加我們的業務成本。在這方面，我們預計美國、歐盟和其他司法管轄區將繼續有與隱私和數據保護有關的新的擬議法律、法規和行業標準，我們無法確定這些未來的法律、法規和標準可能對我們的業務產生什麼影響。
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如果我們或我們的業務被發現違反了任何聯邦或州醫療保健、數據或信息隱私法或適用於我們的任何其他政府法規，我們可能會受到懲罰，包括民事、刑事和行政處罰、損害賠償、罰款、返還、剝奪政府合同資格、拒絕現有合同下的訂單、禁止參與美國聯邦或州醫療保健計劃、公司誠信協議以及削減或重組我們的業務，其中任何一項都可能對我們的業務運營能力和財務業績產生實質性的不利影響。如果我們預期與之開展業務的任何醫生或其他醫療保健提供者或實體(包括我們的合作者)被發現不遵守適用法律，可能會受到刑事、民事或行政制裁，包括但不限於被排除在參與政府醫療保健計劃之外，這也可能對我們的業務產生重大影響。
儘管有效的合規計劃可以降低對違反這些法律的調查和起訴的風險，但這些風險無法完全消除。此外，實現和維持遵守適用的聯邦和州欺詐法可能會付出高昂的代價。任何因違反這些法律而針對我們採取的行動，即使我們成功進行了辯護，也可能導致我們承擔巨額法律費用並轉移我們管理層對業務運營的注意力。
如果政府或第三方付款人未能爲我們的任何產品提供足夠的保險範圍和付款率，或者如果健康維護組織（HMO）或長期護理機構選擇使用較便宜的療法，我們的收入和盈利前景將受到限制。
在國內外市場，我們產品的銷售在一定程度上取決於承保範圍和第三方付款人的報銷。此類第三方付款人包括政府健康計劃，如Medicare和Medicaid，管理型醫療保健提供者，私人健康保險公司和其他組織。覆蓋範圍的決定可能取決於臨床和經濟標準，當更成熟或更低成本的治療替代品已經可用或隨後可用時，這些標準不利於新藥產品。許多私人付款人對新推出的藥物和其他產品採用「新上市區塊」，直到付款人有機會根據他們對這些產品的內部審查做出承保決定。當一種藥物或其他產品不在保險範圍內時，患者有責任支付全額費用，這可能會顯著限制使用。如果無法獲得報銷或僅限於有限級別，我們的候選產品可能在競爭中處於劣勢，而我們或我們的合作者可能無法成功地將我們的候選產品商業化。即使提供了保險，批准的報銷金額也可能不足以讓我們或我們的合作者建立或保持足夠的市場份額，以實現我們或他們的投資的足夠回報。或者，爲了獲得有利的補償條款，我們可能需要在定價上做出妥協，並阻止我們實現相對於成本的足夠利潤率。
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與第三方付款人覆蓋範圍和新批准藥品的報銷有關的不確定性很大。新藥產品的上市審批、定價和報銷因國家而異。當前和未來的立法和/或行政行動可能會極大地改變審批要求，這可能涉及額外的費用，並導致獲得審批的延誤。例如，2024年9月20日，醫療保險和醫療補助服務中心發佈了一項最終規則，題爲「醫療補助計劃；藥品錯誤分類，醫療補助藥品返點計劃下的計劃完整性更新」，這可能會影響我們的報銷和返點戰略。一些國家要求藥品的銷售價格獲得批准後才能上市。在許多國家，定價審查期從批准營銷或產品許可後開始。在一些外國市場，處方藥定價即使在獲得初步批准後，仍然受到政府的持續控制。因此，我們可能會獲得產品在特定國家/地區的營銷批准，但隨後會受到價格法規的約束，這些法規會推遲產品的商業發佈，可能會推遲很長時間，這可能會對我們在該國家/地區銷售該產品所產生的收入產生負面影響。此外，藥品定價在美國是一個關鍵的州和聯邦問題，最近的立法和其他提案旨在提高藥品定價的透明度，降低聯邦醫療保險和醫療補助下的處方藥成本，審查定價與製造商患者計劃之間的關係，並改革政府計劃的藥品報銷方法。我們預計，未來對藥品定價的關注和壓力將繼續存在。不利的定價限制可能會阻礙我們或我們的合作者收回我們或他們在一個或多個產品或候選產品上的投資的能力。我們以及我們的合作伙伴將我們的候選產品商業化的能力將在一定程度上取決於這些產品和相關治療將在多大程度上從政府衛生行政當局、私人健康保險公司和其他組織獲得保險和補償。監管機構和第三方付款人，如私人健康保險公司和健康維護組織，決定他們將覆蓋哪些藥物並建立報銷水平。醫療保健行業非常關注成本控制，無論是在美國還是在其他地方。幾家第三方付款人要求製藥公司向他們提供標價的預定折扣，使用首選藥品清單在競爭類中利用更大的折扣，無視類內的治療差異化因素，並挑戰藥品的定價。沒有仿製藥等價物的品牌藥物通常與具有仿製藥的其他品牌一起列入治療類別，並可能同樣因類別中可用低成本替代品而處於不利地位，特別是如果同一藥物的仿製版本以另一種形式提供的話。
第三方支付者，無論是外國還是國內，還是政府還是商業，正在開發越來越複雜的控制醫療保健成本的方法。此外，在美國，第三方支付者之間沒有統一的藥品承保和報銷政策。因此，藥品的覆蓋範圍和報銷因付款人而異。此外，我們認爲未來的保險範圍和報銷可能會受到美國和國際市場更多的限制。我們獲得監管機構批准的產品或候選產品的第三方承保和報銷在美國或國際市場可能無法提供或不充分，這可能會對我們的業務、財務狀況、運營業績和前景產生負面影響。
假設保險獲得批准，由此產生的報銷付款率可能不夠高。如果付款人對我們的產品施加最高付款金額或施加限制，使其難以獲得報銷，提供商可能會選擇使用與我們的候選產品相比更便宜或訪問限制較少的療法。此外，如果付款人要求高額共同賠付，受益人可能會拒絕處方並尋求替代療法。我們可能需要進行上市後研究，以證明我們的任何產品的成本效益，使醫院和其他目標客戶及其第三方付款人滿意。這樣的研究可能需要我們投入大量的管理時間以及財政和其他資源。我們的產品最終可能不會被認爲具有成本效益。可能無法獲得足夠的第三方保險和報銷，使我們能夠維持足夠的價格水平，以實現產品開發投資的適當回報。
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此外，聯邦計劃對在保密協議下銷售的藥品(包括505(B)(2)藥品)的製造商進行處罰，如果商業價格的增長速度超過城市消費者價格指數，則將以強制額外回扣和/或折扣的形式進行處罰，這些回扣和/或折扣可能會影響我們提高商業價格的能力。監管機構和第三方付款人試圖通過限制特定藥物的覆蓋範圍和報銷金額來控制成本，這可能會影響我們或我們的合作者銷售我們的候選產品的盈利能力。這些付款人可能不會認爲我們的產品(如果有)具有成本效益，並且我們的客戶或我們的合作者可能無法獲得保險和報銷，或者可能不足以使我們的產品(如果有的話)在競爭的基礎上進行營銷。成本控制計劃可能會導致我們或我們的合作伙伴降低、折扣或回扣我們或他們可能爲產品制定的價格的一部分，這可能會導致產品收入低於預期。如果我們產品的實際價格(如果有的話)下降，或者如果政府和其他第三方付款人沒有提供足夠的保險或補償，我們的收入和盈利前景將受到影響。
新批准藥物的承保和報銷也可能會出現延遲，而且承保範圍可能比FDA或類似外國監管機構批准的藥物適應症更有限。此外，報銷資格並不意味着任何藥物都將在所有情況下或以涵蓋我們成本（包括研究、開發、製造、銷售和分銷）的費率付款。新藥的臨時報銷水平（如果適用）可能也不足以支付我們的成本，並且可能只是暫時的。例如，報銷率可能會根據藥物的用途和使用其的臨床環境而有所不同。報銷率也可以基於已經爲較低成本藥物設定的報銷水平，或者可以納入其他服務的現有付款中。
藥品的價格也因貿易類別而異。向政府客戶收取的價格受到價格管制，包括上限，私人機構通過團體採購組織獲得折扣。政府醫療保健計劃要求和私人支付者要求的強制折扣或回扣可能會進一步降低藥品淨價格。根據NDA批准的藥物，包括505（b）（2）藥物，與根據ANDA批准的藥物相比，根據聯邦計劃享受更大的折扣和報告義務，並且適用於這些產品的通貨膨脹罰款可以等於售價。市場條件保證向同一單位的不同客戶提供多次折扣的情況也並不罕見，例如向機構護理提供者提供購買折扣和向他們支付費用的健康計劃提供回扣，這會減少原始銷售的淨實現。
此外，第三方支付者越來越多地要求提供更高水平的新技術益處和臨床結果證據，並對收取的價格提出質疑。我們和我們的合作者無法確定我們或他們商業化的任何產品都能獲得保險，並且如果有的話，報銷率是否足夠。此外，如果目前限制從價格低於美國的國家進口藥品的法律發生變化，藥品的淨報銷可能會進一步減少。對於我們獲得營銷批准的任何候選產品，如果無法迅速從政府資助和私人支付者那裏獲得保險和足夠的付款率，可能會對我們的經營業績、我們籌集產品商業化所需資金的能力以及我們的整體財務狀況產生重大不利影響。
我們受到新立法、監管提案和醫療保健付款人計劃的約束，這可能會增加我們的合規成本，並對我們營銷產品、獲得合作者和籌集資本的能力產生不利影響。
在美國和一些外國司法管轄區，醫療保健系統發生了許多立法和監管變化以及擬議的變化，這些變化可能會阻止或推遲我們候選產品的營銷批准，限制或監管批准後活動，並影響我們或我們合作者的能力，以盈利方式銷售我們獲得營銷批准的任何產品。目前尚不清楚這些各種努力對我們的業務運營和由此產生的財務狀況產生了什麼影響。我們預計，現行法律以及未來可能採取的其他醫療改革措施可能會導致更嚴格的覆蓋標準，並對我們或我們的合作者可能收到的任何批准產品的價格帶來額外的下調壓力。
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目前尚不清楚不斷變化的法規和次級監管政策可能如何影響對ACA的解釋和進一步實施，以及它對我們業務的實際影響。我們無法預測美國聯邦或州醫療保健立法的未來走向，這些立法旨在擴大醫療保健的可獲得性，並控制或降低醫療保健的成本，包括藥品和生物製品。法律或監管框架的任何進一步變化，如果減少我們的收入或增加我們的成本，也可能對我們的業務、財務狀況和運營結果產生實質性的不利影響。此外，美國最高法院最近的裁決存在很大程度的不確定性，包括洛珀·光明企業訴雷蒙多案，603美國_(2024)和Corner Post，Inc.訴聯邦儲備系統理事會，603 U.S._(2024)，將影響FDA的執法和決策權。洛珀·布萊特明確地推翻了雪佛龍尊重，它以前給予行政部門機構的行政行動以司法上的尊重。此外，最高法院在角柱可能會導致新的訴訟當事人在很長一段時間內對FDA的決定提出挑戰。
While the full effect that the ACA may have on our business continues to evolve, we expect that the ACA, as well as other federal and state healthcare reform measures that may be adopted in the future, may result in more rigorous coverage criteria, increased regulatory burdens and operating costs, decreased net revenue from our pharmaceutical products, decreased potential returns from our development efforts, and additional downward pressure on the price that we receive for any approved drug. There is also an increasing focus on the price of drugs, both at the state and federal levels, and it is likely that additional pricing controls will be enacted and could harm our business, financial condition and results of operations. For instance, states such as California have begun enacting transparency laws aimed at curbing drug price increases. We continue to monitor the potential impact of proposals and recently enacted legislation to lower prescription drug costs at the federal and state level. For example, the Inflation Reduction Act of 2022, or the IRA, was signed into law by President Biden in August 2022. The IRA makes significant changes to how drugs are covered and paid for under the Medicare program, including the creation of financial penalties for drugs whose prices rise faster than the rate of inflation, redesign of the Medicare Part D program to require manufacturers to bear more of the liability for certain drug benefits, and government price-setting for certain Medicare Part D drugs, starting in 2026, and Medicare Part B drugs starting in 2028. We are evaluating what effect, if any, the IRA may have on our business. Any reduction in reimbursement from Medicare or other government healthcare programs may result in a similar reduction in payments from private payors. The implementation of cost containment measures or other healthcare reforms may prevent us from being able to generate revenue, attain profitability or commercialize our drugs.
Legislative and regulatory proposals may also be made to expand post approval requirements and restrict sales and promotional activities for drugs. We cannot be sure whether additional legislative changes will be enacted, or whether the FDA regulations, guidance, or interpretations will be changed, or what the impact of such changes on the marketing approvals of our product candidates, if any, may be. In addition, increased scrutiny by the U.S. Congress of the FDA’s approval process may significantly delay or prevent marketing approval, as well as subject us to more stringent product labeling and post-marketing testing and other requirements.
此外，還有其他一些旨在改變製藥業的立法和監管建議。例如，頒佈的《藥品供應鏈安全法》(DSCSA)規定了用於商業分銷的處方藥產品製造商的義務，規範了產品在聯邦一級的分銷，併爲供應鏈中的實體(製造商和分包商、批發分銷商、第三方物流提供商和分銷商)的聯邦或州註冊和合規設定了某些標準。DSCSA先發制人，先發制人某些先前頒佈的州譜系法律和《處方藥營銷法》(PDMA)的譜系要求。藥品供應鏈中的貿易伙伴現在必須確保滿足某些產品跟蹤要求，即他們正在與其他授權的貿易伙伴做生意；他們必須交換交易信息、交易歷史和交易對賬單。現在還需要產品標識信息(產品跟蹤方案的一個方面)。美國食品藥品監督管理局的要求、標準的制定和產品追溯系統已經並將繼續在一段時間內分階段實施，美國食品和藥物管理局表示，由於新冠肺炎大流行，它將允許某些豁免和排除，以及在某些方面的執法自由裁量權，儘管這種情況可能會繼續發展。產品樣品的分配繼續受到PDMA的監管，一些州還對藥品樣品的分配實施了規定。
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遵守聯邦追蹤要求可能會增加我們的運營費用並帶來重大行政負擔。由於這些和其他新提案，我們可能會決定改變我們當前的運營方式、提供額外福利或改變我們的合同安排，其中任何一項都可能對我們的業務、財務狀況和運營結果產生重大不利影響。
我們遵守各種美國和國際法律和法規。
我們目前受到一些政府法律法規的約束，未來可能會受到新的政府法律法規的約束。遵守此類法律和法規的成本或不遵守的負面結果可能對我們的業務、現金流、運營結果、財務狀況和前景產生不利影響；這些法律和法規包括：(I)美國或其他國家/地區的其他醫療改革舉措，包括額外的強制性折扣或費用；(Ii)1977年《反海外腐敗法》或其他反賄賂和腐敗法律；(Iii)新的法律、法規和影響定價、藥品報銷以及獲得或跨司法管轄區營銷的司法或其他政府決定；(Iv)知識產權法的變化；(V)會計準則的變化；(Vi)新的和不斷增加的數據隱私法規和執法，特別是在歐盟、美國和中國；(Vii)對本地製藥產品製造的立法授權或偏好；(Viii)關於報告向醫療保健專業人員支付款項和其他價值轉移的新興和新的全球法規要求；(Ix)環境法規，例如歐盟的企業可持續發展報告指令；以及(X)進口限制、禁運、貿易制裁以及立法和/或其他法規變化的潛在影響。
美國以外的政府傾向於實施嚴格的價格控制，這可能會對我們的收入產生不利影響，如果有的話。
在國際市場上，報銷和醫療保健支付制度因國家不同而有很大差異，許多國家對特定產品和療法設定了價格上限。在一些國家，特別是歐盟國家，處方藥的定價受到政府的控制。在這些國家，在收到產品上市許可後，與政府當局進行定價談判可能需要相當長的時間。爲了在一些國家獲得保險和報銷或定價批准，我們可能需要進行一項臨床試驗，將我們候選產品的成本效益與其他可用的療法進行比較。不能保證我們的產品會被第三方付款人認爲具有成本效益，不能保證有足夠的報銷水平，也不能保證第三方付款人的報銷政策不會對我們有利可圖地銷售產品的能力產生不利影響。如果我們的產品無法獲得報銷或在範圍或金額上受到限制，或者如果定價設定在不令人滿意的水平，我們的業務可能會受到損害，可能是實質性的。
我們的員工、獨立承包商、顧問、商業合作伙伴、主要調查員或CROs可能從事不當行爲或其他不當活動，包括不遵守監管標準和要求，這可能會對我們的業務產生重大不利影響。
我們面臨着員工欺詐或其他不當行爲的風險。員工、獨立承包商、顧問、商業合作伙伴、主要調查人員或CRO的不當行爲可能包括故意、魯莽、疏忽或無意未能遵守FDA法規、遵守適用的欺詐和濫用法律、向FDA提供準確信息、準確報告財務信息或數據，或向我們披露未經授權的活動。這種不當行爲還可能涉及對臨床試驗過程中獲得的信息的不當使用或虛假陳述，這可能導致監管制裁和對我們的聲譽造成嚴重損害。並非總是能夠識別和阻止此類不當行爲，我們爲發現和防止此類活動而採取的預防措施可能無法有效控制未知或未管理的風險或損失，或保護我們免受因不遵守此類法律或法規而引起的政府調查或其他行動或訴訟。如果對我們提起任何此類行動，而我們未能成功地爲自己辯護或維護自己的權利，這些行動可能會對我們的業務、財務狀況和運營結果產生重大影響，包括施加巨額罰款或其他制裁。此外，即使我們成功地進行了防禦，我們也可能在準備和維護我們的防禦時產生大量成本，任何此類行動都將是時間和資源密集型的，並可能轉移管理層對業務的注意力，這可能會對我們運營業務的能力和我們的運營結果產生不利影響。
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我們的第三方製造商可能會在生產我們的產品時使用危險材料，如果是這樣，他們必須遵守環境法律和法規，這些法律和法規可能成本高昂，並且限制了我們或他們的業務方式。
生產我們產品的製造活動涉及對危險材料的受控儲存、使用和處置，包括我們產品的組件和其他危險化合物。我們的第三方製造商和我們在使用、製造、儲存、搬運、釋放、處置和接觸這些危險材料時，必須遵守聯邦、州和地方的法律和法規。違反這些法律法規可能會導致巨額罰款和處罰。儘管我們相信我們和第三方製造商處理和處置這些材料的安全程序符合這些法律法規規定的標準，但我們不能消除這些材料意外污染或傷害的風險。一旦發生事故，州或聯邦當局可能會限制我們對這些材料的使用，並中斷我們的業務運營。此外，我們可能會承擔與調查和清理任何污染有關的潛在重大責任，無論是目前未知的還是由未來釋放造成的。
儘管我們購買了工人賠償保險，以支付因使用危險材料導致員工受傷而可能產生的成本和開支，但該保險可能無法爲潛在責任提供足夠的保障。我們不爲與我們儲存或處置生物、危險或放射性材料有關的環境責任或有毒侵權索賠購買保險。
此外，爲了遵守當前或未來的環境、健康和安全法律法規，我們可能會承擔巨額成本。這些當前或未來的法律和法規可能會損害我們的研究、開發或生產工作。我們未能遵守這些法律和法規也可能導致巨額罰款、處罰或其他制裁。
與我們的業務運營相關的風險
我們已經並可能繼續大幅擴大組織的規模，並且我們在管理增長方面可能會遇到困難。如果我們無法實施適當的控制和程序來管理我們的增長，我們將無法成功實施我們的業務計劃。
截至2024年11月5日，我們擁有607名全職員工。我們目前的管理、人員、系統和設施可能不足以支持未來的增長。此外，由於製藥企業之間的人員競爭，我們未來可能無法招聘和留住合格的人員，特別是銷售和營銷職位，如果不這樣做，可能會對我們未來的產品收入和業務業績產生重大負面影響。此外，隨着時間的推移，授予的股票期權或限制性股票單位對員工的價值受到我們股價變動的顯着影響，這些變動超出了我們的控制範圍，並且在任何時候都可能不足以抵消其他公司更有利可圖的報價。我們需要有效管理我們的運營、增長和各種項目，這要求我們：
We may be unable to successfully implement these tasks on a larger scale and, accordingly, may not achieve our development and commercialization goals.
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Our continued growth could strain our personnel resources and infrastructure, and if we are unable to implement appropriate controls and procedures to manage our growth, we will not be able to implement our business plan successfully.
As we continue to complete our clinical trials and commercialize our product candidates, and as our company continues to grow, we may experience significant strains on our resources, including to our administrative, operational and financial infrastructure, which will result in additional burdens on management. Our success will depend in part upon the ability of our senior management to manage this growth effectively. To do so, we must continue to hire, train and manage new employees as needed. If our new hires perform poorly, or if we are unsuccessful in hiring, training, managing and integrating these new employees, or if we are not successful in retaining our existing employees, our business would be harmed. To manage the expected growth of our operations and personnel, we will need to continue to improve our operational, financial and management controls and our reporting systems and procedures.
We may acquire businesses or products, or form strategic alliances in the future, and we may not realize the benefits of such acquisitions or alliances.
We may acquire additional businesses or products, form strategic alliances or create joint ventures with third parties that we believe will complement or augment our existing business. If we acquire businesses with promising markets or technologies, we may not be able to realize the benefit of acquiring such businesses if we are unable to successfully integrate them with our existing operations and company culture. We may encounter numerous difficulties in developing, manufacturing, and marketing any new products resulting from a strategic alliance or acquisition that delay or prevent us from realizing their expected benefits or enhancing our business. We cannot assure you that, following any such acquisition, we will achieve the expected synergies to justify the transaction.
We may not be able to manage our business effectively if we are unable to attract and retain key personnel.
近年來，我們行業的管理人員流失率很高。我們高度依賴我們管理團隊的技能和領導力，包括我們的首席執行官兼董事會主席Herriot Tabuteau博士。我們與我們的任何管理團隊都沒有正式的僱傭協議。然而，我們通常會與高管和關鍵人員簽訂聘書。我們的高級管理層可以隨時終止他們在我們公司的僱傭關係。如果我們失去了一名或多名高級管理團隊成員，我們成功實施業務戰略的能力可能會受到嚴重損害。替換這些員工可能很困難，而且可能需要很長一段時間，因爲我們行業中擁有成功開發、獲得監管部門批准並將產品商業化所需的技能和經驗的個人數量有限。從這個有限的人才庫中招聘的競爭非常激烈，我們可能無法招聘、培訓、留住或激勵更多的關鍵人員。我們不爲我們的任何高管或其他員工提供「關鍵人物」保險。
如果我們未能對財務報告保持有效的內部控制系統，我們可能無法準確報告我們的財務狀況、運營結果或現金流量，這可能會對投資者對我們的信心產生不利影響，從而對我們普通股的價值產生不利影響。
作爲一家上市公司，我們繼續承擔作爲私營公司沒有承擔的大量法律、會計和其他費用。此外，《薩班斯-奧克斯利法案》以及美國證券交易委員會和納斯達克股票市場有限責任公司（納斯達克）隨後實施的規則對上市公司提出了各種要求，包括要求建立和維護有效的披露控制和對財務報告的內部控制以及公司治理實踐的變化。我們的管理層和其他人員投入了大量時間用於這些合規舉措。此外，這些規則和法規增加了我們的法律和財務合規成本，並使一些活動更加耗時和成本。
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《薩班斯-奧克斯利法案》要求萬億對財務報告和披露控制及程序保持有效的內部控制。根據薩班斯-奧克斯利法案第404(A)條，我們必須提交一份由管理層提交的報告，其中包括我們對財務報告的內部控制的有效性。這份報告必須披露管理層在對財務報告的內部控制進行定期評估時發現的任何重大弱點。重大缺陷是指財務報告內部控制的缺陷或缺陷的組合，導致年度或中期財務報表的重大錯報不能得到及時防止或發現的可能性超過了合理的可能性。薩班斯-奧克斯利法案第404(B)條還要求我們的獨立核數師證明並報告這一管理評估。確保我們有足夠的內部控制措施，以便我們能夠及時編制準確的財務報表，這是一項既昂貴又耗時的努力。如果我們不能遵守第404條的要求，或者如果我們或我們的獨立註冊會計師事務所無法證明我們對財務報告的內部控制的有效性，投資者可能會對我們的財務報告的準確性和完整性失去信心，我們的股票的市場價格可能會下跌，我們可能會受到納斯達克、美國證券交易委員會或其他監管機構的制裁或調查，這將需要額外的財務和管理資源。
在評估和測試過程中，如果我們發現我們對財務報告的內部控制存在一個或多個重大弱點，我們將被要求實施旨在減輕控制弱點的補救程序。在這種補救程序成功緩解控制弱點之前，我們將不能斷言我們對財務報告的內部控制是有效的。我們不能向您保證，我們對財務報告的內部控制在未來不會出現重大缺陷。任何未能對財務報告進行內部控制的行爲都可能嚴重抑制我們及時準確地報告財務狀況、經營成果或現金流的能力。遵守第404條的成本要求我們在實施額外的公司治理實踐和遵守報告要求時，在與合規相關的問題上產生大量會計費用並花費大量管理時間。雖然我們目前使用第三方會計師事務所的服務來協助我們進行內部控制，但我們目前沒有內部審計小組，我們可能需要聘請更多具有適當上市公司經驗和技術會計知識的會計和財務人員。
此外，如果我們無法及時遵守這些要求，或者如果我們或我們的獨立註冊會計師事務所發現我們對財務報告的內部控制存在被視爲重大弱點的缺陷，我們股票的市場價格可能會下跌，我們可能會失去投資者對我們財務報告的準確性和完整性的信心，我們可能會受到納斯達克、美國證券交易委員會或其他監管機構的制裁或調查，這將需要額外的財務和管理資源。未能糾正我們對財務報告的內部控制中的任何重大缺陷，或實施或維護上市公司所需的其他有效控制系統，也可能會限制我們未來進入資本市場的機會。
我們的披露控制和程序可能無法阻止或檢測所有錯誤或欺詐行爲。
我們須遵守《交易法》的定期報告要求。我們的披露控制和程序旨在合理確保我們根據《交易法》提交或提交的報告中要求披露的信息在SEC規則和表格規定的時間內積累並傳達給管理層、記錄、處理、總結和報告。我們相信，任何披露控制和程序或內部控制和程序，無論構思和操作如何良好，只能提供合理而非絕對的保證，以實現控制系統的目標。
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此外，如上所述，薩班斯-奧克斯利法案要求我們保持有效的披露控制和程序，以及對財務報告的內部控制。特別是，《薩班斯-奧克斯利法案》第404條要求我們對我們的財務報告內部控制進行系統和過程評估和測試，以允許管理層報告我們的財務報告內部控制的有效性，並允許我們的獨立註冊會計師事務所證明我們財務報告內部控制的有效性。根據第404條，吾等須就本公司財務報告內部控制的有效性提供年度管理報告，並須在該年度報告中包括由本公司獨立註冊會計師事務所出具的財務報告內部控制證明報告。在未來，我們的獨立註冊會計師事務所可能會出具一份不利的報告，如果我們沒有在所有重要方面對財務報告保持有效的內部控制。任何未能對財務報告保持有效的披露控制和內部控制都可能對我們的業務、運營結果和財務狀況產生實質性的不利影響，並可能導致我們普通股的交易價格下降。
這些固有的侷限性包括這樣的現實，即決策過程中的判斷可能是錯誤的，故障可能因爲簡單的錯誤或錯誤而發生。此外，某些人的個人行爲、兩個或兩個以上人的串通或未經授權超越控制，都可以規避控制。因此，由於我們的控制系統的固有限制，由於錯誤或欺詐而導致的錯誤陳述或披露不足的情況可能會發生，而不會被發現。
如果系統發生故障，我們的業務和運營將受到影響。
儘管我們實施了安全措施，但我們的內部計算機系統以及我們的CRO和其他承包商和顧問的計算機系統很容易受到計算機病毒、未經授權的訪問、自然災害、恐怖主義、戰爭、電信和電氣故障、互聯網上的網絡攻擊或網絡入侵、電子郵件附件、組織內部人員或能夠訪問組織內部系統的人員的破壞。隨着來自世界各地的未遂攻擊和入侵的數量、強度和複雜性增加，安全漏洞或破壞的風險普遍增加，特別是通過網絡攻擊或網絡入侵，包括計算機黑客、外國政府和網絡恐怖分子。如果發生這樣的事件並導致我們的運營中斷，可能會導致我們的候選產品開發計劃發生實質性中斷。例如，已完成、正在進行或計劃中的臨床試驗的臨床試驗數據丟失可能會導致我們的監管審批工作延遲，並顯著增加我們恢復或複製數據的成本。如果任何中斷或安全漏洞導致我們的數據或應用程序丟失或損壞，或不適當地披露個人、機密或專有信息，我們可能會招致責任，我們的任何候選產品的進一步開發可能會被推遲。
環境、社會和治理問題可能會影響我們的業務和聲譽。
政府當局、非政府組織、客戶、投資者、外部利益攸關方和僱員對環境、社會和治理或ESG問題日益敏感，如多樣性和包容性、氣候變化、用水、包裝的可回收性或可回收性以及塑料垃圾。這種對ESG問題的關注可能會導致新的要求，這可能會導致與開發、製造和分銷我們的產品相關的成本增加。我們的競爭能力也可能受到客戶偏好和要求變化的影響，例如對更環保的產品、包裝或供應商做法的需求不斷增長，或者無法滿足客戶的期望或需求。在我們努力改善ESG表現的同時，如果我們不負責任地採取行動，或者如果我們被認爲在關鍵ESG領域沒有負責任地採取行動，包括公平獲得藥品和疫苗、產品質量和安全、多樣性和包容性、環境管理、對當地社區的支持、公司治理和透明度，以及解決我們運營中的人力資本因素，我們可能會面臨股東的負面反應，包括代理諮詢服務，以及其品牌和聲譽受到損害。如果我們沒有達到ESG投資者、客戶和其他利益相關者的期望，我們可能會經歷對其產品的需求減少、客戶流失，以及對我們的業務和運營結果的其他負面影響。
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與我們普通股所有權相關的風險
我們普通股的活躍交易市場可能無法持續。
2015年11月，我們完成了首次公開募股。在我們首次公開募股之前，我們的普通股沒有公開市場。儘管我們已完成首次公開募股，並且我們的普通股在納斯達克全球市場上市和交易，但我們股票的活躍交易市場可能無法維持。如果我們普通股的活躍市場不再持續，我們的股東可能很難在不壓低股票市場價格的情況下出售他們的股票，或者以或高於他們收購股票的價格出售他們的股票，或者在他們想出售的時候出售他們的股票。我們普通股的任何不活躍交易市場也可能損害我們通過出售股票籌集資金以繼續爲我們的運營提供資金的能力。
我們普通股的市場價格可能波動很大。
我們普通股的交易價格可能會高度波動。例如，2019年，我們的股價經歷了非同尋常的上漲。未來不太可能持續這樣的增長水平。從那時起，我們的股價出現了大幅升值和貶值。由於這種波動性，投資者可能無法以或高於股票價格出售其普通股。我們普通股的市場價格可能受到許多因素的影響，包括：
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In addition, the stock market in general, and the market for mid-cap pharmaceutical and biotechnology companies in particular, have experienced extreme price and volume fluctuations that have often been unrelated or disproportionate to the operating performance of these companies. Broad market and industry factors may negatively affect the market price of our common stock, regardless of our actual operating performance.
If equity research analysts do not publish research or reports, or publish unfavorable research or reports, about us, our business, or our market, our stock price and trading volume could decline.
The trading market for our common stock is influenced by the research and reports that equity research analysts publish about us and our business. We do not have any control over the equity research analysts that provide research coverage of our common stock or the content and opinions included in their reports. The price of our stock could decline if one or more equity research analysts downgrades our stock or issue other unfavorable commentary or research. If one or more equity research analysts ceases coverage of our company or fails to publish reports on us regularly, demand for our stock could decrease, which in turn could cause our stock price or trading volume to decline.
Our quarterly operating results may fluctuate significantly.
We expect our operating results to be subject to quarterly fluctuations. Our net loss and other operating results will be affected by numerous factors, including:
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If our quarterly or annual operating results fall below the expectations of investors or securities analysts, the price of our common stock could decline substantially. Furthermore, any quarterly or annual fluctuations in our operating results may, in turn, cause the price of our stock to fluctuate substantially. We believe that quarterly comparisons of our financial results are not necessarily meaningful and should not be relied upon as an indication of our future performance.
Raising additional funds by issuing securities may cause dilution to existing stockholders and raising funds through lending and licensing arrangements may restrict our operations or require us to relinquish proprietary rights.
We may finance our cash needs through a combination of equity offerings, debt financings, grants, and license and development agreements in connection with any collaborations until such time, if ever, as our product sales are sufficient to meet our cash needs. To the extent that we raise additional capital by issuing equity securities, our existing stockholders’ ownership will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a common stockholder. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends.
If we raise additional funds through collaborations, strategic alliances, or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs, or product candidates or grant licenses on terms that may not be favorable to us. Any debt financing we enter into may involve covenants that restrict our operations. These restrictive covenants may include limitations on additional borrowing and specific restrictions on the use of our assets as well as prohibitions on our ability to create liens, pay dividends, redeem our stock, or make investments. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce, or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves.
Our principal stockholders and management own a significant percentage of our stock and may be able to exert significant control over matters subject to stockholder approval.
As of November 5, 2024, our executive officers, directors, and 5% stockholders and their affiliates beneficially owned an aggregate of approximately 43% of our outstanding common stock. As a result, these stockholders have significant influence and may be able to determine all matters requiring stockholder approval. For example, these stockholders may be able to control elections of directors, amendments of our organizational documents, or approval of any merger, sale of assets, or other major corporate transaction. This concentration of ownership could delay or prevent any acquisition of our company on terms that other stockholders may desire and may adversely affect the market price of our common stock.
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Some of these persons or entities may have interests different than our other stockholders. For example, these stockholders, if they acted together, could significantly influence all matters requiring approval by our stockholders, including the election of directors and the approval of mergers or other business combination transactions. These stockholders may be able to determine all matters requiring stockholder approval. The interests of these stockholders may not always coincide with our interests or the interests of other stockholders. This may also prevent or discourage unsolicited acquisition proposals or offers for our common stock that other stockholders may feel are in their best interest and our large stockholders may act in a manner that advances their best interests and not necessarily those of other stockholders, including seeking a premium value for their common stock, and might affect the prevailing market price for our common stock.
Sales of a substantial number of shares of our common stock in the public market by our existing stockholders could cause our stock price to fall.
Sales of a substantial number of shares of our common stock in the public market or the perception that these sales might occur, could depress the market price of our common stock and could impair our ability to raise adequate capital through the sale of additional equity securities. We are unable to predict the effect that sales may have on the prevailing market price of our common stock.
As of November 5, 2024, we have outstanding 48,462,376 shares of common stock and 9,566,082 shares of common stock equivalents that would increase the number of common stock outstanding if these instruments were exercised or converted, including stock options to purchase common stock based on vesting requirements and warrants to purchase common stock, as well as outstanding restricted stock units. Of our currently outstanding shares of common stock, 40,870,017 are freely tradable. The remainder of the outstanding shares of common stock are held by our affiliates and may be considered “control securities” for purposes of Rule 144 under the Securities Act.
In addition, we have filed, or will soon file, one or more registration statements on Form S‑8 registering the issuance of an aggregate of 15,600,010 shares of common stock subject to options or other equity awards issued or reserved for issuance under our 2015 Omnibus Incentive Compensation Plan and 1,100,000 shares of common stock reserved for issuance under our 2023 Employee Stock Purchase Plan. Shares registered under registration statements on Form S-8 will be available for sale in the public market subject to vesting arrangements and exercise of options, the lock up agreements described above and the restrictions of Rule 144 in the case of our affiliates.
Our management will have broad discretion in the use of the net proceeds from our capital raises, including the proceeds from sales pursuant to our Sales Agreement, and may not use them effectively.
Our management will have broad discretion in the application of the net proceeds from our capital raises, which we refer to as our Capital Raises, including the proceeds from sales pursuant to the March 2022 Sales Agreement with Leerink, which provides for the sale of up to $250.0 million of our common stock from time to time, and our stockholders will not have the opportunity as part of their investment decision to assess whether the net proceeds from our Capital Raises are being used appropriately. Our stockholders may not agree with our decisions, and our use of the proceeds may not yield any return on investment for our stockholders. Because of the number and variability of factors that will determine our use of the net proceeds from our Capital Raises their ultimate use may vary substantially from their currently intended use. Our failure to apply the net proceeds of our Capital Raises effectively could compromise our ability to pursue our growth strategy and we might not be able to yield a significant return, if any, on our investment of those net proceeds. Our stockholders will not have the opportunity to influence our decisions on how to use our net proceeds from our Capital Raises. Pending their use, we may invest the net proceeds from our Capital Raises in short-term, investment-grade, interest-bearing instruments and U.S. government securities. These temporary investments are not likely to yield a significant return.
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The use of our net operating loss carryforwards and research tax credits may be limited.
Our net operating loss carryforwards and any future research and development tax credits may expire and not be used. As of December 31, 2023, we had U.S. federal net operating loss, NOL, carryforwards of approximately $549.9 million and foreign NOL carryforwards of $0.7 million. U.S. federal net operating loss carry forwards amounting to $59.8 million generated before the 2018 tax year will start expiring beginning 2032, if we have not used them prior to that time, and the U.S. federal net operating losses of approximately $490.1 million generated in 2018 and later have an indefinite carryforward period. Net operating loss carry forwards arising in taxable years ending after December 31, 2017, are no longer subject to expiration under the Internal Revenue Code of 1986, as amended, or the Code. Additionally, our ability to use any net operating loss and credit carryforwards to offset taxable income or tax, respectively, in the future will be limited under Sections 382 and 383 of the Code, respectively, if we have a cumulative change in ownership of more than 50% within a three-year period. The completion of our initial public offering, together with our other public and private Capital Raises, and other transactions that have occurred, may trigger, or may have already triggered, such an ownership change. In addition, since we may need to raise additional funding to finance our operations, we may undergo further ownership changes in the future. In the event a change of ownership occurs, we will be limited regarding the amount of net operating loss carryforwards and research tax credits that could be utilized annually in the future to offset taxable income or tax, respectively. Any such annual limitation may significantly reduce the utilization of the net operating loss carryforwards and research tax credits before they expire. In addition, certain states have suspended use of net operating loss carryforwards for certain taxable years, and other states are considering similar measures. As a result, we may incur higher state income tax expense in the future. Depending on our future tax position, continued suspension of our ability to use net operating loss carryforwards in states in which we are subject to income tax could have an adverse impact on our results of operations and financial condition.
Because we do not intend to pay dividends on our common stock, returns for our stockholders will be limited to any increase in the value of our stock.
We have never declared or paid any cash dividends on our capital stock. In addition, the terms of our existing credit facility with Hercules preclude us from paying cash dividends without Hercules’ consent. We currently intend to retain all available funds and any future earnings to support our operations and finance the growth and development of our business and do not anticipate declaring or paying any cash dividends on our common stock for the foreseeable future. Any return to stockholders will therefore be limited to the appreciation of their stock, if any. Investors seeking cash dividends should not purchase our common stock.
Provisions in our corporate charter documents and under Delaware law may prevent or frustrate attempts by our stockholders to change our management and hinder efforts to acquire a controlling interest in us, and the market price of our common stock may be lower as a result.
There are provisions in our amended and restated certificate of incorporation and amended and restated bylaws that may make it difficult for a third party to acquire, or attempt to acquire, control of our company, even if a change in control was considered favorable by you and other stockholders. For example, our Board will have the authority to issue up to 10,000,000 shares of preferred stock and to fix the price, rights, preferences, privileges, and restrictions of the preferred stock without any further vote or action by our stockholders. We do not currently have any preferred stock outstanding. The issuance of shares of preferred stock may delay or prevent a change in control transaction. As a result, the market price of our common stock and the voting and other rights of our stockholders may be adversely affected. An issuance of shares of preferred stock may result in the loss of voting control to other stockholders.
In addition, we are subject to the anti‑takeover provisions of Section 203 of the Delaware General Corporation Law, or DGCL, which regulates corporate acquisitions by prohibiting Delaware corporations from engaging in specified business combinations with particular stockholders of those companies. These provisions could discourage potential acquisition proposals and could delay or prevent a change in control transaction. They could also have the effect of discouraging others from making tender offers for our common stock, including transactions that may be in your best interests. These provisions may also prevent changes in our management or limit the price that investors are willing to pay for our stock.
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Our amended and restated certificate of incorporation designates the Court of Chancery of the State of Delaware as the sole and exclusive forum for certain types of actions and proceedings that may be initiated by our stockholders, which could limit our stockholders’ ability to obtain a favorable judicial forum for disputes with us or our directors, officers or employees.
Our amended and restated certificate of incorporation provides that, unless we consent in writing to the selection of an alternate form, the Court of Chancery of the State of Delaware will be the sole and exclusive forum for (1) any derivative action or proceeding brought on our behalf, (2) any action asserting a claim of breach of a fiduciary duty owed by any of our directors, officers, employees or agents to us or our stockholders, (3) any action asserting a claim arising pursuant to the DGCL, or (4) any other action asserting a claim against us that is governed by the internal affairs doctrine, in each such case subject to such Court of Chancery having personal jurisdiction over the indispensable parties named as defendants therein.
Any person or entity purchasing or otherwise acquiring any interest in shares of our capital stock shall be deemed to have notice of and to have consented to the provisions of our amended and restated certificate of incorporation described above. This choice of forum provision may limit a stockholder’s ability to bring a claim in a judicial forum that it finds favorable for disputes with us or our directors, officers, or other employees or agents, which may discourage such lawsuits against us and our directors, officers, employees, and agents. Further, this choice of forum provision does not preclude or contract the scope of exclusive federal or concurrent jurisdiction for any actions brought under the Securities Act or the Exchange Act. Section 27 of the Exchange Act creates exclusive federal jurisdiction over all suits brought to enforce any duty or liability created by the Exchange Act or the rules and regulations thereunder. As a result, the exclusive forum provision will not apply to suits brought to enforce any duty or liability created by the Exchange Act or any other claim for which the federal courts have exclusive jurisdiction. In addition, Section 22 of the Securities Act creates concurrent jurisdiction for federal and state courts over all suits brought to enforce any duty or liability created by the Securities Act or the rules and regulations thereunder. As a result, the exclusive forum provision will not apply to suits brought to enforce any duty or liability created by the Securities Act or any other claim for which the federal and state courts have concurrent jurisdiction. Accordingly, our exclusive forum provision will not relieve us of our duties to comply with the federal securities laws and the rules and regulations thereunder, and our stockholders will not be deemed to have waived our compliance with these laws, rules and regulations.
If a court were to find these provisions of our amended and restated certificate of incorporation inapplicable to, or unenforceable in respect of, one or more of the specified types of actions or proceedings, we may incur additional costs associated with resolving such matters in other jurisdictions, which could adversely affect our business, financial condition, and results of operations. Even if we are successful in defending against these claims, litigation could result in substantial costs and be a distraction to management and other employees.
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ITEM 5. OTHER INFORMATION.
During the third quarter of 2024, the following Rule 10b5-1 trading arrangements (as defined in Item 408(a)(1)(i) of Regulation S-K) and non-Rule 10b5-1 trading arrangements (as defined in Item 408(c) of Regulation S-K) intended to satisfy the affirmative defense of Rule 10b5-1(c) of the Exchange Act were adopted or terminated by our directors and/or executive officers:
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Title |
Date of |
Scheduled Expiration Date of Rule 10b5-1 Trading Arrangement |
Aggregate Number of Securities to Be Sold |
Up to |
(1) Date of adoption of Rule 10b5-1 trading arrangements is in accordance with both the Company’s insider trading policy and applicable SEC rules and regulations.
(2) The first trade pursuant to the Rule 10b5-1 trading arrangement will be, in accordance with both the Company’s insider trading policy and applicable SEC rules and regulations, on a date after the date of adoption of the Rule 10b5-1 trading arrangement.
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ITEM 6. EXHIBITS.
The exhibits filed as part of this Quarterly Report on Form 10-Q are set forth on the Exhibit Index, which is incorporated herein by reference.
INDEX OF EXHIBITS
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描述 |
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10.1**## |
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大力神貸款和安全協議第五修正案，日期爲2024年9月30日，由Axome治療公司和Hercules Capital，Inc.以其自身和貸款人的行政代理和抵押品代理的身份簽署。 |
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10.2** |
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10.3** |
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Axome Treeutics，Inc.根據修訂和重新發布的2015年綜合激勵薪酬計劃，以限制性股票單位協議(非執行人員)的形式。 |
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10.4** |
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Axome Treeutics，Inc.根據修訂和重新修訂的2015年綜合激勵薪酬計劃，以限制性股票單位協議的形式(高管和非僱員董事)。 |
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31.1** |
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根據2002年《薩班斯-奧克斯利法案》第302節通過的第13a-14(A)/15d-14(A)條頒發的特等執行幹事證書。 |
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31.2** |
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根據2002年《薩班斯-奧克斯利法案》第302條通過的第13a-14(A)/15d-14(A)條規定的首席財務幹事證書。 |
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32.1** |
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根據2002年《薩班斯-奧克斯利法案》第906節通過的《美國法典》第18篇第1350節規定的首席執行官證書(隨函提供)。 |
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32.2** |
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根據2002年《薩班斯-奧克斯利法案》第906節通過的《美國法典》第18篇第1350節規定的首席財務官證書(隨函提供)。 |
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101.INS |
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內聯XBRL實例文檔(該實例文檔不會出現在交互數據文件中，因爲其XBRL標記嵌入在內聯XBRL文檔中) |
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101.SCH |
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具有嵌入Linkbase文檔的內聯MBE分類擴展模式 |
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104 |
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封面交互數據文件(格式爲內聯XBRL，包含附件101中包含的適用分類擴展信息)。 |
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* * 隨附。
##根據S-k法規第601（b）（10）（iv）項，本展覽的附件和時間表以及某些條款已被省略，因爲它們包含的信息既不重要，又屬於註冊人視爲私人或機密的類型。註冊人同意應SEC的要求向SEC提供本附件的未經編輯的副本，包括此處的任何附件或時間表;但是，註冊人可以要求對此類未經編輯的副本進行保密處理。
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標牌縫隙
根據1934年《證券交易法》的要求，註冊人已正式促使本報告由正式授權的簽署人代表其簽署。
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Axome Therapeutics，Inc. |
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日期：2024年11月12日 |
通過 |
/s/ Herriot Tabuteau. D. |
|
|
Herriot Tabuteau萬. D. |
|
|
總裁與首席執行官 |
|
|
(首席行政主任) |
|
|
|
|
|
|
日期：2024年11月12日 |
通過 |
/s/尼克·皮齊 |
|
|
尼克·皮齊 |
|
|
首席財務官 |
|
|
(首席財務會計官) |
114