EX-99.2 3 q32024supplementalearnin.htm EX-99.2 q32024supplementalearnin
Advancing medicines. Solving problems. Improving lives. September 2024 Advancing medicines. Solving problems. Improving lives. Advancing medicines. Solving problems. Improving lives. 1 Third Quarter 2024 Earnings Supplemental Materials November 4, 2024

 
© 2024 Aquestive Therapeutics, Inc. 2 ® Disclaimer Certain statements in this presentation include “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “believe,” “anticipate,” “plan,” “expect,” “estimate,” “intend,” “may,” “will,” or the negative of those terms, and similar expressions, are intended to identify forward-looking statements. These forward-looking statements include, but are not limited to, statements regarding the advancement and related timing of our product candidate Anaphylm (epinephrine) Sublingual Film through clinical development and approval by the U.S. Food and Drug Administration (FDA), including the timing of submission of supporting and pediatric clinical studies, holding a pre-New Drug Application (NDA) meeting with the FDA and filing the NDA for Anaphylm with the FDA; that the results of the Company’s clinical studies for Anaphylm are sufficient to support submission of the NDA for approval of Anaphylm by the FDA; that Anaphylm will be the first and only oral administration of epinephrine and accepted as an alternative to existing standards of care, if Anaphylm is approved by the FDA; the advancement and related timing of our Adrenaverse pipeline epinephrine prodrug product candidates, including AQSt-108, through clinical development and FDA regulatory approval process, including holding a pre-IND meeting with the FDA for AQSt-108 to support the targeted indication of alopecia areata; the potential benefits our products and product candidates could bring to patients; our cash and financial position, including with respect to our 2024 financial outlook; and business strategies, market opportunities, and other statements that are not historical facts. These forward-looking statements are based on our current expectations and beliefs and are subject to a number of risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Such risks and uncertainties include, but are not limited to, risks associated with our development work, including any delays or changes to the timing, cost and success of our product development activities and clinical trials and plans, including those relating to Anaphylm (including for pediatric patients), AQSt-108, and the Company's other product candidates; risks associated with the Company’s distribution work for Libervant, including any delays or changes to the timing, cost and success of Company's distribution activities and expansion of market access to patients aged two to five years for Libervant; risk of delays in advancement of the regulatory approval process through the FDA of our product candidates, including the filing of the respective NDAs, including for Anaphylm, AQSt-108, Libervant for patients aged between six and eleven and other product candidates, or failure to receive FDA approval at all of any of these product candidates; risk of the Company’s ability to generate sufficient clinical data for approval of our product candidates, including with respect to our pharmacokinetics and pharmacodynamics comparability submission for FDA approval of Anaphylm; risk of the Company’s ability to address the FDA’s comments on the Company’s future clinical trials and other concerns identified in the FDA Type C meeting minutes for Anaphylm, including the risk that the FDA may require additional clinical studies for approval of Anaphylm; risk that we may not overcome the seven year orphan drug market exclusivity granted by the FDA for the approved nasal spray product of another company in the U.S. in order for Libervant to be granted U.S. market access for patients aged six years and older until the expiration of the orphan drug market exclusivity period of the nasal spray product due to expire in January 2027, or for other reasons; risk of loss of U.S. market approval of Libervant for patients aged between two and five resulting from a legal challenge relating to U.S. orphan drug market exclusivity by the owner of the approved nasal spray product with respect to the FDA’s approval for U.S. market access of Libervant for this pediatric patient population, or for other reasons; risks and uncertainties inherent in commercializing a new product (including technology risks, financial risks, market risks and implementation risks and regulatory limitations); risk of development of a sales and marketing capability for commercialization of our product Libervant and other product candidates, including Anaphylm and AQSt-108; the potential impact on the value of the Company of the sale or outlicensing of our product and product candidates, including Libervant and Anaphylm and other product candidates; risk of sufficient capital and cash resources, including sufficient access to available debt and equity financing, including under our ATm facility, and revenues from operations, to satisfy all of our short-term and longer-term liquidity and cash requirements and other cash needs, at the times and in the amounts needed, including to fund commercialization activities relating to Libervant for patients between two and five years of age and to fund future clinical development and commercial activities for our product candidates, including Anaphylm, AQSt-108 and Libervant for patients aged between six and eleven, should these product candidates be approved by the FDA, and for Libervant patients of six years and older upon expiration of the orphan drug marketing exclusivity period of the nasal spray product; risk that our manufacturing capabilities will be sufficient to support demand for Libervant for patients between two and five years of age and for older patients, should Libervant receive U.S. market access for these older patients, and for demand for our licensed products in the U.S. and abroad; risk of eroding market share for Suboxone® and risk as a sunsetting product, which accounts for the substantial part of our current operating revenue; risk of default of our debt instruments; risks related to the outsourcing of certain sales, marketing and other operational and staff functions to third parties; risk of the rate and degree of market acceptance in the U.S. and abroad of Libervant for epilepsy patients between two and five years of age, and for older epilepsy patients if approved for U.S. market access and after the expiration of the orphan drug market exclusivity period in January 2027; risk of the rate and degree of market acceptance in the U.S. and abroad of Libervant and Anaphylm, AQSt-108 and our other product candidates, should these product candidates be approved by the FDA, and for our licensed products in the U.S. and abroad; risk of the success of any competing products including generics; risk of the size and growth of our product markets; risk of compliance with all FDA and other governmental and customer requirements for our manufacturing facilities; risks associated with intellectual property rights and infringement claims relating to our products; risk that our patent applications for our product candidates, including for Anaphylm and AQSt-108, will not be timely issued, or issued at all, by the U.S. Patent and Trademark Office; risk of unexpected patent developments; risk of legislation and regulatory actions and changes in laws or regulations affecting our business including relating to our products and products candidates and product pricing, reimbursement or access therefor; risk of loss of significant customers; risks related to claims and legal proceedings against Aquestive including patent infringement, securities, business torts, investigative, product safety or efficacy and antitrust litigation matters; risk of product recalls and withdrawals; risks related to any disruptions in our information technology networks and systems, including the impact of cybersecurity attacks; risk of increased cybersecurity attacks and data accessibility disruptions due to remote working arrangements; risk of adverse developments affecting the financial services industry; risks related to inflation and rising interest rates; risks related to the impact of the COVID-19 global pandemic and other pandemic diseases on our business, including with respect to our clinical trials and the site initiation, patient enrollment and timing and adequacy of those clinical trials, regulatory submissions and regulatory reviews and approvals of our product candidates, availability of pharmaceutical ingredients and other raw materials used in our products and product candidates, supply chain, manufacture and distribution of our products and product candidates; risks and uncertainties related to general economic, political (including the Ukraine and Israel wars and other acts of war and terrorism), business, industry, regulatory, financial and market conditions and other unusual items; and other uncertainties affecting us. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from those expressed or implied by these statements. These factors include the matters discussed and referenced in the risk factors of the Company’s 2023 Annual Report on Form 10-k and our other Quarterly Reports on Form 10-Q and in our Current Reports on Form 8-k and our other filings with the U.S. Securities and Exchange Commission. Given these uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as the date made. All subsequent forward-looking statements attributable to us or any person acting on our behalf are expressly qualified in their entirety by this cautionary statement. All subsequent forward-looking statements attributable to the Company or any person acting on its behalf are expressly qualified in their entirety by this cautionary statement. The Company assumes no obligation to update forward-looking statements or outlook or guidance after the date of this presentation whether as a result of new information, future events or otherwise, except as may be required by applicable law.

 
2024年第3四半期の収益の主なメッセージ ・アナフィルム（エピネフリン）舌下フィルム ・陽性のトップライン結果 - 口内アレルギー症候群の挑戦研究（OASISと称される） ・NDA事前会議は2024年第4四半期に予定 ・NDA事前会議の直後に単回投与小児研究の開始を予定 ・2025年第1四半期にNDA提出を完了する予定 ・リバーバント®（ジアゼパム）歯へのフィルム 2歳から5歳の患者対象 ・既に12人の国内セールスチームを配置しています ・2024年10月より小売流通で入手可能 ・メディケイド患者には2歳から5歳の患者用のリバーバントが全州で払い戻し可能 ・健康計画の審査とPBm契約に基づいて、2歳から5歳の患者に対するリバーバントの商業患者アクセスは引き続き改善 ・AQSt-108（エピネフリン）局所ゲル ・目標適応は円形脱毛症 ・2024年第4四半期にFDAとの事前IND会議を予定 ・FDAとの調整を得た後、2025年第2四半期に第2a相研究を開始する計画 ・2026年までの現金ランウェイを延長する資産負債表を強化 ・2024年第3四半期を約7800万ドルの現金残高で終了 完

 
2024年を強力に終えるための位置づけ ● アナフィルム自己投与研究からの陽性トップラインデータを発表 ● 8月にOASIS研究の登録を開始 ● 7月に2歳から5歳のリバーバントの市場アクセスを拡大し続ける ● アナフィルムのNDA事前会議のリクエストを提出 ● OASIS研究の登録完了 ● インベスターデイを開催し、AQSt-108（エピネフリン）局所ゲルの円形脱毛症の目標適応を発表 9月 完

 
マイルストーン AQSt-108 リバーバント®（2歳から5歳） 2024年第3四半期 2024年第4四半期 2025年第1四半期 2025年第2四半期 AQSt-108 事前IND会議 AQSt-108 第2a相研究 開始 インベスターデイ - 9月27日開催 アナファラムTm FDAとのNDA事前会議 OAS研究 トップライン - 完了 販売範囲拡大 NDA提出 小児研究 拡大市場アクセス Open IND 完

 
アナフィルムプログラムの最新情報 更新6

 
7 アナフィルプログラムの更新および次のステップ • OASISスタディ完了 • フルトップライン臨床データがリリースされ、Aquestiveウェブサイト（https://investors.aquestive.com/events-and-presentations）で利用可能 • 浮腫または口腔浮腫の被験者に関する追加の分析を提供 • すべての予定された成人スタディが現在完了 • FDAとの事前NDAミーティング後、プロトコルについてFDAの合意を得ることを前提に小児スタディを開始する予定で、その直後にすぐにFDAとの事前NDAミーティングを予定している（2024年第4四半期）

 
8 OASISスタディ - 症状的な口腔浮腫はAnaphylmの薬物動態（PK）に影響しない¹ アレルゲン挑戦を伴う/伴わない状態でのAnaphylmの投与: • アドレナリンマニュアル筋内注射（Manual IM）よりも45分以上高い • 類似したPKプロファイルを有する • 一貫した中央値最大薬物濃度（Tmax）を示す 1. アクエスティブセラピューティクスのファイル内データ。口腔浮腫が報告された被験者の単回投与（n=7）と同じ被験者がアレルゲン挑戦を受けた場合を比較

 
9 予定された小児スタディ1 単回投与、単一治療、小児患者を対象としたマルチセンター、平行デザインのスタディ 7-17歳（体重≥30kg）アナフィラキシスのリスクが高い患者18-24人 薬物動態学（PK）、薬力学（PD）、および治療前有害事象（TEAEs） アドレナリン（IM）非アレルゲン曝露 アナフィルムの単回投薬、医療提供者による 1. プロトコルに関するFDAの合意を前提にしたスタディデザイン。スタディデザインエンドポイント

 
AQSt-108プログラムの更新10

 
11 決定症alopecia areata¹のためのAQSt-108計画された第2a相臨床スタディ アライメント後のスタディ開始を予定 フェーズ2aスタディデザイン • 24-48被験者、4回投与 • 12-24週² • 進行段階の重症度（SALT）スコアの基準値からの変化が12週目において≥10% • トリコスコピー評価と基準値時の検査 第2aスタディの目標: • 進行段階の重症度（SALT）スコアが基準値から12週目において≥10%の変化を指摘することにより、alopecia areata患者においてAQSt-108の安全性と有効性を評価する 1. アライメント後にスタディを開始する予定。 2. 途中データは12週後に利用可能で、主要エンドポイントデータは24週で利用可能と見込まれている。

 
Libervant® ローンチ更新 12

 
2歳から5歳の患者のLibervant処方が増加し、患者のアクセスとセールスフォースが拡大するにつれ、継続的に伸びています。 ・ Libervantのローンチは拡大し続け、目標通りに進行中 ・ 処方箋あたりの平均カートン数は1.8箱¹ ・ セールスフォースは活発で、医療提供者（HCPs）2,300人をカバーする規模となっています ・ 2024年10月1日から全国の小売り流通が完了しました。 Aquestive Therapeuticsのファイルにデータがあります。

 
14 支払い者カバレッジと市場アクセスの拡大続く ● すべての州でマイペイマーケット向けに年齢などの臨床的に適切な編集（例：年齢）が含まれるメディケイドがリンバーセーブル ● 商業薬局給付管理会社（PBMs）との交渉が続行中（リババン患者のトップ3のうち2社との契約が完了） ● リババン患者2-5対象の商業保険計画がカバレッジ追加開始 ● リバヴァント患者2-5のための完全な小売り流通がすでに開始しています ● リババン患者2-5のために、全米70か所の地域的卸売流通センターに在庫があり、小売り薬局へのアクセスが可能です。市場アクセス - 支払者カバレッジ市場アクセス - 流通

 
財務結果 15

 
16 キャッシュポジションは、2024年第1四半期の株式調達により大幅に改善しました 23.9 95.2 89.9 77.9 0 20 40 60 80 100 120 Q4 23 Q1 24 Q2 24 Q3 24 米ドル（百万ドル） 四半期末の現金残高

 
17 研究開発費を除くEBITDA¹ （4.6） （5.1） （0.5） 0.1 1.7 （0.1） （1.4） 5.6 -1.6 （6.0） （5.0） （4.0） （3.0） （2.0） （1.0） 0.0 1.0 2.0 3.0 4.0 5.0 6.0 7.0 第3四半期22第4四半期22 第1四半期23 第2四半期23 第3四半期23 第4四半期23 第1四半期24 第2四半期24 第3四半期24米ドル（百万ドル） 非GAAP調整後EBITDA（調整後R＆D費用を除く）四半期ごとの指標 1.AnaphylmおよびAQSt-108のR＆Dパイプライン投資を除く；AnaphylmおよびLibervantへの商業投資を含む

 
18 42,182 47,041 41,097 36,863 33,458 47,953 46,241 45,328 42,516 34,418 43,818 0 10,000 20,000 30,000 40,000 50,000 60,000 Q1 22 Q2 22 Q3 22 Q4 22 Q1 23 Q2 23 Q3 23 Q4 23 Q1 24 Q2 24 Q3 24出荷された量（千単位） 四半期単位の出荷量 製造業務は引き続きキャッシュフローを生み出しています

 
19現在の通期ガイダンス2024年の展望 ・ 約5700万〜6000万ドルの総収入 ・ 約2000万〜2300万ドルの調整後非GAAP EBITDA損失

 
ありがとうございました20