附錄99.1
protara therapeutics宣布2024年第三季度財務業績並提供業務更新。
| · | 計劃在2024年第4季度報告ADVANCED-2試驗中TARA-002在NMIBC的六個月可評估患者的中期數據。 |
| · | 預計於2025年中報告NMIBC中TARA-002在ADVANCED-2試驗的12個月中期數據。 |
| · | 在小兒LM患者中，TARA-002在第2期STARBORN-1試驗的第一隊中展示出令人鼓舞的結果並且一般上耐受良好；預計在2025年上半年從下一隊中獲得初始結果。 |
| · | 獲得美國FDA對IV氯化膽鹼的快速通道指定；預計在2025年第1季度對THRIVE-3登記試驗的第一位患者進行劑量。 |
NEW YORk，2024年11月12日 - protara therapeutics（納斯達克代碼：TARA）公佈，這家臨床階段公司為治療癌症和罕見疾病開發轉變性療法，今天提供了業務更新和截至2024年9月30日的第三季度財務業績。
“我們持續取得重大進展，推進所有進行中的項目，努力實現將改變生命的療法帶給受癌症和罕見疾病影響的患者的目標，” protara therapeutics首席執行官Jesse Shefferman說。“值得一提的是，我們仍在計劃中，計劃在2024年第4季度從我們對非肌肉浸潤性膀胱癌（NMIBC）患者的TARA-002的ADVANCED-2研究中報告中期數據。考慮到其易於使用、良好的安全性概況以及令人鼓舞的早期臨床數據，我們相信TARA-002可能成為NMIBC治療格局中有影響力的補充物。”
Shefferman先生補充說，“除了我們的NMIBC計劃外，我們仍然按計劃將於2025年第一季度開始最關鍵的THRIVE-3研究，該研究探討靜脈注射(IV)氯化膽鹼，我們最近從美國食品和藥物管理局(FDA)獲得了快速通道指定。最後，我們預計將於2025年上半年從進行中的TARA-002對淋巴管畸形(LMs)進行的第2期STARBORN-1試驗中獲得中期數據，這是一種罕見的疾病，主要影響兒童，目前尚無FDA批准的治療方案。
最新進展和亮點
NMIBC中的TARA-002
| · | 公司仍然按計劃預計在本季度晚些時候報告正在進行中的第2期開放標籤ADVANCED-2試驗中六個月可評估患者的預先計劃中期分析結果。ADVANCED-2試驗正在評估TARA-002對NMIBC患者的膀胱內注射，這些患者患有原位癌或CIS(± Ta/T1)，這些患者對卡西华卡尔莫特‧格林巴氏菌(BCG)不敏感(n≈100)且BCG未曾接觸(n=27)。對於BCG不敏感的群體設計符合FDA的2024年BCG不敏感非肌層浸潤性膀胱癌：為治療研製藥物和生物制品制定的指南。 |
| · | 公司預計將於2025年中期從正在進行的第2期開放標籤ADVANCED-2試驗中12個月可評估患者報告初步結果。 |
| · | 公司持續探索靜脈注射之前的系統性預先劑量給藥，以及與CIS的NMIBC患者TARA-002進行聯合治療。鑒於TARA-002的作用機制和安全性概況，公司認為它在聯合治療中具有強大潛力，並正在努力確定臨床計劃的各種機會。 |
用於接受輸液支持(PS)的靜脈注射氯化膽鹼
| · | 在2024年10月，protara宣布FDA已為該公司的調查性IV磷脂基底替代療法IV氯化膽鹼授予快速通道指定，該療法供應給無法進行口服或腸鐵營養，或者營養不足或禁忌的患者。protara目前正在開發IV氯化膽鹼作為成人和青少年PS患者的膽鹼來源。 |
| · | In September 2024, the Company announced results from THRIVE-1, a prospective, observational study evaluating the prevalence of choline deficiency and liver injury in patients dependent on PS. The study found that 78% of patients who are dependent on PS were choline deficient, with 63% of these patients demonstrating liver dysfunction, including steatosis, cholestasis, and hepatobiliary injury. |
| · | In September 2024, the U.S. Patent and Trademark Office issued the Company a second patent in this program, claiming a method for treating choline deficiency with a choline composition with a term until 2041. |
| · | The Company expects to begin dosing patients in the THRIVE-3 registrational trial in the first quarter of 2025. |
TARA-002 in LMs
| · | In September 2024, Protara announced completion of the first safety cohort of the ongoing Phase 2 STARBORN-1 clinical trial of TARA-002 in pediatric patients with macrocystic and mixed cystic LMs. Of the three patients treated in the first cohort, which enrolled individuals six years to less than 18 years of age, two patients treated with TARA-002, including one with a macrocystic Lm and one with a ranula (a type of maxillofacial cyst) achieved a complete response after receiving one dose of TARA-002. TARA-002 was generally well-tolerated. Enrollment is underway in additional cohorts, with initial results expected in the first half of 2025. |
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2024年第三季度財務結果
| · | As of September 30, 2024, cash, cash equivalents, and investments in marketable debt securities totaled approximately $8150萬. The Company expects its cash, cash equivalents, and investments in marketable debt securities will be sufficient to fund its planned operations and data milestones into 2026. |
| · | Research and development expenses for the third quarter of 2024 increased to $810萬 from $620萬 for the prior year period. This increase was primarily due to an increase in expenses related to TARA-002 of $150萬 as well as an increase in expenses related to IV Choline Chloride of $40萬. |
| · | 2024年第三季度的總務及管理費用從去年同期的450萬美元下降至430萬美元。這個降低主要是由於人員相關費用減少了60萬美元，部分抵消了市場開發、業務拓展和投資者關係活動增加30萬美元。 |
| · | 2024年第三季度，protara therapeutics的淨虧損為1120萬美元，每股0.50美元，較2023年同期的淨虧損990萬美元，每股0.87美元。2024年第三季度的淨虧損中包括約90萬美元的股份報酬支出。 |
關於TARA-002
TARA-002是一種研究性細胞治療，用於治療NMIBC和LMS，並已獲得美國食品和藥物管理局授予的罕見小兒疾病認定。TARA-002是從日本中外製藥市場上市的廣泛免疫增強劑Picibanil®所使用的一種基因不同的A群鏈球菌主細胞庫開發而來。protara therapeutics已成功展示了TARA-002與Ok-432之間的製造可比性。
當TARA-002被施用時，假設囊腫或腫瘤內的固有和適應性免疫細胞被激活，並釋放細胞激素如腫瘤壞死因子（TNF）-alpha、干擾素（IFN）-γ IL-6、IL-10、IL-12等進行炎症反應。TARA-002也直接殺死腫瘤細胞，並通過誘導免疫原性細胞死亡觸發宿主免疫反應，從而進一步增強抗腫瘤的免疫反應。
關於非肌層侵襲性膀胱癌（NMIBC）
膀胱癌在美國是第六大常見的癌症，非肌侵性膀胱癌約佔膀胱癌診斷的約80％。每年在美國約有6.5萬名患者被診斷為非肌侵性膀胱癌。非肌侵性膀胱癌是指發現在膀胱內表面內層組織中的癌症，並且未擴散到膀胱肌層。
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關於淋巴管畸形（LMs）
LMs是淋巴管的罕見先天畸形，導致這些結構無法連接或排出到靜脈系統。大多數LMs存在於頭部和頸部區域，並在早期兒童中進行診斷，期間淋巴生長活躍，超過50％在出生時檢測到，90％在三歲之前診斷。該病最常見的弊端和嚴重表現包括壓迫上呼吸消化道，包括需要插管和可能需要氣管造口依賴；局部出血；對重要結構的侵襲，包括神經，血管，淋巴管；反復感染，以及外觀和其他功能殘疾。
關於IV磷脂醯膽鹼（Choline Chloride）
IV磷脂醯膽鹼是一種正在開發中的探索性藥物，用於接受輸液支持的病人的靜脈磷脂質替代療法。膽鹼是磷脂質的已知重要基質，對於健康的肝臟功能至關重要，同時在調節基因表達，細胞膜信號，大腦發育和神經傳導，肌肉功能和骨骼健康中起著重要作用。接受輸液支持的病人無法從腸道營養來源合成膽鹼，目前沒有含有膽鹼的輸液支持制劑。依賴輸液支持的患者約80％缺乏膽鹼，並且有一定程度的肝損傷，可能導致肝功能衰竭。每年在美國約有9萬人需要在家接受輸液支持，其中約有3萬人長期需要輸液支持。IV磷脂醯膽鹼有潛力成為首個獲得美國食品和藥物管理局（FDA）批准的用於輸液支持患者的IV膽鹼配方，並獲得FDA授予孤兒藥品稱號，用於預防輸液支持患者的膽鹼缺乏。美國專利商標局已向該公司授予了請求膽鹼組成的美國專利和一種用膽鹼組成治療膽鹼缺乏的方法的美國專利，每個專利期均至2041年到期。
About Protara Therapeutics, Inc.
Protara is a clinical-stage biotechnology company committed to advancing transformative therapies for people with cancer and rare diseases. Protara’s portfolio includes its lead candidate, TARA-002, an investigational cell-based therapy in development for the treatment of non-muscle invasive bladder cancer (NMIBC) and lymphatic malformations (LMs). The Company is evaluating TARA-002 in an ongoing Phase 2 trial in NMIBC patients with carcinoma in situ (CIS) who are unresponsive or naïve to treatment with Bacillus Calmette-Guérin (BCG), as well as a Phase 2 trial in pediatric patients with LMs. Additionally, Protara is developing IV Choline Chloride, an investigational phospholipid substrate replacement for patients on parenteral nutrition who are otherwise unable to meet their choline needs via oral or enteral routes. For more information, visit www.protaratx.com.
參考文獻
1. Klinische Einheit, or KE, is a German term indicating a specified weight of dried cells in a vial.
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前瞻性陳述
Statements contained in this press release regarding matters that are not historical facts are “forward looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Protara may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “designed,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words or expressions referencing future events, conditions or circumstances that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such forward-looking statements include but are not limited to, statements regarding Protara’s intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: Protara’s business strategy, including its development plans for its product candidates and plans regarding the timing or outcome of existing or future clinical trials; statements related to expectations regarding interactions with the FDA; Protara’s financial position; statements regarding the anticipated safety or efficacy of Protara’s product candidates; and Protara’s outlook for the remainder of the year. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Factors that contribute to the uncertain nature of the forward-looking statements include: risks that Protara’s financial guidance may not be as expected, as well as risks and uncertainties associated with: Protara’s development programs, including the initiation and completion of non-clinical studies and clinical trials and the timing of required filings with the FDA and other regulatory agencies; general market conditions; changes in the competitive landscape; changes in Protara’s strategic and commercial plans; Protara’s ability to obtain sufficient financing to fund its strategic plans and commercialization efforts; having to use cash in ways or on timing other than expected; the impact of market volatility on cash reserves; failure to attract and retain management and key personnel; the impact of general U.S. and foreign, economic, industry, market, regulatory, political or public health conditions; and the risks and uncertainties associated with Protara’s business and financial condition in general, including the risks and uncertainties described more fully under the caption “Risk Factors” and elsewhere in Protara’s filings and reports with the United States Securities and Exchange Commission. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management’s assumptions and estimates as of such date. Protara undertakes no obligation to update any forward-looking statements, whether as a result of the receipt of new information, the occurrence of future events or otherwise, except as required by law.
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protara therapeutics, inc.及其子（或附屬）公司
未經審核的簡化合併資產負債表
(以千為單位，每股數據除外)
| 截至目前為止 | ||||||||
| 9月30日， 2024 | 12月31日 2023 | |||||||
| 資產 | ||||||||
| 流動資產: | ||||||||
| 現金及約當現金 | $ | 51,757 | $ | 39,586 | ||||
| 可銷負債證券 | 29,742 | 25,994 | ||||||
| 預付費用及其他流動資產 | 3,583 | 3,125 | ||||||
| 全部流動資產 | 85,082 | 68,705 | ||||||
| 限制性現金，非流動 | 745 | 745 | ||||||
| 物業及設備，扣除折舊後淨值 | 1,109 | 1,296 | ||||||
| 經營租賃權使用資產 | 4,514 | 5,264 | ||||||
| 其他資產 | 2,640 | 2,944 | ||||||
| 資產總額 | $ | 94,090 | $ | 78,954 | ||||
| 550,714 | ||||||||
| 流動負債： | ||||||||
| 應付賬款 | $ | 2,295 | $ | 2,434 | ||||
| 應計費用及其他流動負債 | 5,268 | 2,732 | ||||||
| 營業租賃負債 | 1,079 | 983 | ||||||
| 流動負債合計 | 8,642 | 6,149 | ||||||
| 非流動營業租賃負債 | 3,657 | 4,484 | ||||||
| 總負債 | 12,299 | 10,633 | ||||||
| 合約和可能負債 | ||||||||
| 股東權益： | ||||||||
| 優先股票，0.001美元面值，授權股份為10,000,000股：序列1可換股優先股，2024年9月30日和2023年12月31日授權8,028股，2024年9月30日和2023年12月31日發行和流通股為7,991股。 | - | - | ||||||
| 普通股票，0.001美元面值，授權股份為100,000,000股：普通股，2024年9月30日和2023年12月31日分別發行和流通股為20,629,772股和11,364,903股。 | 21 | 11 | ||||||
| 資本公積額額外增資 | 313,952 | 268,725 | ||||||
| 累積虧損 | (232,211 | ) | (200,384 | ) | ||||
| 其他綜合損益（損失）累積額 | 29 | (31 | ) | |||||
| 股東權益總額 | 81,791 | 68,321 | ||||||
| 負債和股東權益總額 | $ | 94,090 | $ | 78,954 | ||||
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protara therapeutics, inc.及其附屬公司
未經審計之綜合損益及綜合虧損表
(以千為單位，每股數據除外)
| 截至三個月結束之日 9月30日， | 截至九個月結束 9月30日， | |||||||||||||||
| 2024 | 2023 | 2024 | 2023 | |||||||||||||
| 營運開支： | ||||||||||||||||
| 研究與開發 | $ | 8,070 | $ | 6,218 | $ | 22,205 | $ | 18,608 | ||||||||
| 一般行政管理 | 4,260 | 4,482 | 12,637 | 13,964 | ||||||||||||
| 營業費用總計 | 12,330 | 10,700 | 34,842 | 32,572 | ||||||||||||
| 營運虧損 | (12,330 | ) | (10,700 | ) | (34,842 | ) | (32,572 | ) | ||||||||
| 其他收益（支出），淨額： | ||||||||||||||||
| 利息和投資收入 | 1,111 | 840 | 3,015 | 2,373 | ||||||||||||
| 其他收入（費用），淨額 | 1,111 | 840 | 3,015 | 2,373 | ||||||||||||
| 淨收入（虧損） | $ | (11,219 | ) | $ | (9,860 | ) | $ | (31,827 | ) | $ | (30,199 | ) | ||||
| 每股凈利潤（損失）歸於普通股股東，基本及稀釋 | $ | (0.50 | ) | $ | (0.87 | ) | $ | (1.74 | ) | $ | (2.67 | ) | ||||
| 基本和稀釋平均股數 | 22,329,772 | 11,347,887 | 18,342,566 | 11,320,027 | ||||||||||||
| 其他綜合損益： | ||||||||||||||||
| 可轉讓債券投資淨未實現收益（損失） | 29 | 171 | 60 | 523 | ||||||||||||
| 其他全面收益（損失） | 29 | 171 | 60 | 523 | ||||||||||||
| 綜合收益（損失） | $ | (11,190 | ) | $ | (9,689 | ) | $ | (31,767 | ) | $ | (29,676 | ) | ||||
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公司聯絡人:
Justine O’Malley
protara therapeutics
Justine.OMalley@protaratx.com
646-817-2836
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