Adrenaline

近年来，国内改良型新药研发如火如荼，但一个残酷的现实是：90%的改良药从立项第一步就埋下隐患。企业投入数千万、耗时5-7年，最终却因市场表现惨淡而陷入困境。问题究竟出在哪里？真正的破局之道又是什么？01改良新药的“生死劫”：立项即定成败"选择远大于努力"——这句话在改良药领域尤为残酷。数据显示，90%的改良药失败根源在于立项阶段的三重误判：伪需求陷阱：仅关注药学改进（如缓释、依从性），却未精准匹配临床痛点；竞品盲区：低估替代疗法（如长效注射剂、创新器械）的竞争压力；医生认知断层：忽视处方药需撬动医生处方习惯的底层逻辑。案例直击：某缓控释制剂耗时6年改良，最终因临床价值模糊，被医生评价"可有可无"；全球首个阿立哌唑"芯片药物"因价格高昂、无法抗衡长效注射剂，遭遇市场滑铁卢。02破解困局的三大核心法则法则1：真需求=未被满足的临床缺口改良药的终极价值在于填补"临床空白"，而非单纯技术炫技。关键三问：患者的实际治疗困境是什么？（如急救场景、特殊人群需求）医生的诊疗习惯中是否存在未被优化的痛点？现有疗法是否存在安全性、便捷性或疗效的明显短板？成功范例：肾上腺素鼻喷剂的突围——瞄准过敏人群急救场景，以"无需注射、便携即用"直击传统注射剂的临床短板，上市即获医患双端认可。法则2：竞品替代战，赢在"降维打击"改良药的本质是"替代战争"，必须建立差异化竞争优势：技术降维：用更低成本实现更高临床价值（如国产微电子技术突破芯片药成本瓶颈）；场景切割：锁定细分人群（如儿童剂型、居家护理场景）；价值量化：用真实数据证明"多花的钱值得"（如减少住院次数、降低护理成本）。警惕误区："自我视角"立项——仅对比原研药，却忽视同类改良药、跨赛道疗法的竞争。法则3：医生的"认知战"必须提前打响处方药的商业成功=临床价值×医生认知度×准入效率。破局策略：临床KOL前置介入：立项阶段即邀请权威医生参与需求验证；证据链全程打造：从药学数据到真实世界研究，构建医生信服的科学依据；市场教育"抢跑"：在研发中后期同步启动医生学术推广，避免上市后"从零开始"。03未来趋势：从"技术改良"到"生态创新"改良药的下一站，是成为医疗场景的解决方案：药械结合（如可监测用药数据的智能制剂）；支付创新（如按疗效付费、商保定制产品）；患者全周期管理（从治疗延伸到预防、康复场景）。行业信号：国家药审中心（CDE）已明确释放信号——仅以"依从性提升"为卖点的改良药将面临更严苛的审评标准，真正的临床获益才是通行证。04结语改良新药的黄金时代，属于那些以临床为锚点、以生态为战场的创新者。当行业集体走出"为改而改"的误区，中国医药创新的第二增长曲线才能真正爆发。【内容参考视频号“慧药王立峰”】特此声明如下：本文数据与观点仅供参考，不构成投资、医疗等专业建议，据此决策风险自担。文章信息源于多方，仅供参考，读者应核实信息，决策后果自行负责。文中内容为一般性分析，不构成具体操作指引，使用需谨慎，责任自负。•中国改良型新药技术要求与相关政策解读•医改新政对改良新药市场和营销的影响•改良型新药的申报、审批状况分析及立项布局策略考量•改良型创新药物的行业分析• 改良型新药调释制剂临床药代动力学研究技术指导原则• 已确认改良新药大方向下，如何发掘未被满足的临床需求• 改良型新药研发的处方开发和临床设计• 缓释制剂的改良立项及临床开发策略• 改良型新药调释制剂临床药代动力学研究技术指导原则• 已确认改良新药大方向下，如何发掘未被满足的临床需求• 改良型新药研发的处方开发和临床设计• 缓释制剂的改良立项及临床开发策略• 儿童改良型新药研发关键技术及实例解析• 基于新辅料创新的改良型新药研发策略• 改良型新药开发立题的合理性探讨• 面对技术壁垒，仿制药和改良新药立项的策略是什么• 高壁垒仿制药的市场机会及赛道布局• 陈  震郑州大学教授，曾担任CDE化药一部副部长• 甘  勇上海药物所研究员，CDE评审专家、药典委委员• 韩  军聊城大学生物制药研究院院长• 杨  劲中国药科大学教授• 涂家生中国药科大学药剂学教授，国家药典委药用辅料和药包材专业委员会主任委员• 郑爱萍中国人民解放军军事医学科学院，毒物药物研究所药物制剂研究室主任• 余  立原北京市药品检验所所长助理，国家药典委生化专业组委员，中国药学会抗生素专业委员会委员• 赵秀丽首都医科大学附属北京同仁医院，国家药物临床试验机构副主任/办公室主任• 魏世峰北京罗诺强施医药技术研发中心有限公司董事长兼CEO• 郭新峰南京循证生物科技有限公司创始人• 王立峰慧药咨询创始人• 袁 弘浙江大学药学院教授展会参展，赞助，协办等商务合作洽谈，欢迎咨询：商务合作CMC药博会 400-1662-808 参会咨询胡女士 13065724378（微信同号）同期会议全览↓↓

Geometric mean baseline adjusted epinephrine concentration over time after administration of Anaphylm 12mg in pediatric subjects (Study AQ109302) compared to adult subjects (Study AQ109301B) Reports positive pharmacokinetic (PK) results from the recently completed pediatric study for Anaphylm™ Completes submission of its New Drug Application (NDA) for Anaphylm to the FDA; NDA acceptance expected in Q2 2025 WARREN, N.J., April 01, 2025 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (“Aquestive” or the “Company”), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today announced positive topline results from its pediatric study for Anaphylm™ (epinephrine) sublingual film in patients aged seven to seventeen and weighing greater that thirty kilograms with a personal history of allergic reactions. This marks the completion of the Anaphylm clinical program and supports the clinical data needed for the NDA submission. Aquestive has submitted the Anaphylm NDA to the U.S. Food and Drug Administration (FDA) and expects to receive potential acceptance of the NDA during the second quarter of 2025. "We are extremely pleased with the positive results from our pediatric study, which further validate Anaphylm's potential as the first-ever sublingual film and convenient treatment option for all patients with severe allergic reactions, including anaphylaxis," said Daniel Barber, President and Chief Executive Officer of Aquestive. "These results demonstrate that Anaphylm maintains its consistent PK profile in pediatric patients between the ages of seven and seventeen and weighing greater than thirty kilograms. These data are an important component of the FDA review process and could enable Anaphylm to have a label that includes this pediatric patient population. We continue to prepare for commercial readiness and plan to launch Anaphylm in the first quarter of 2026, if approved by the FDA.” The pediatric study was a multi-site, single treatment study designed to evaluate the PK, pharmacodynamics (PD), safety, and tolerability of Anaphylm. A total of thirty-two patients completed the study. The PK results were consistent with previous adult studies. Importantly, Anaphylm was shown to be safe and well-tolerated with no serious adverse events (SAEs) reported. Figure 1 below compares the pediatric study results to the previously announced pivotal adult study results. Figure 1: Geometric mean baseline adjusted epinephrine concentration over time after administration of Anaphylm 12mg in pediatric subjects (Study AQ109302) compared to adult subjects (Study AQ109301B) About Anaphylm™ (epinephrine) Sublingual Film Anaphylm™ (epinephrine) Sublingual Film is a polymer matrix-based epinephrine prodrug product candidate in development for the treatment of severe allergic reactions, including anaphylaxis. Anaphylm is similar in size to a postage stamp, weighs less than an ounce, and begins to dissolve on contact. No water or swallowing is required for administration. The packaging for Anaphylm is thinner and smaller than an average credit card, can be carried in a pocket, and is designed to withstand weather excursions such as exposure to rain and/or sunlight. The Anaphylm trade name for AQST-109 has been conditionally approved by the FDA. Final approval of the Anaphylm proprietary name is conditioned on FDA approval of the product candidate. About Aquestive Therapeutics Aquestive is a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies. We are developing orally administered products to deliver complex molecules, providing novel alternatives to invasive and inconvenient standard of care therapies. Aquestive has five commercialized products marketed by the Company and its licensees in the U.S. and around the world, and is the exclusive manufacturer of these licensed products. The Company also collaborates with pharmaceutical companies to bring new molecules to market using proprietary, best-in-class technologies, like PharmFilm®, and has proven drug development and commercialization capabilities. Aquestive is advancing a late-stage proprietary product candidate for the treatment of severe allergic reactions, including anaphylaxis, and an earlier stage epinephrine prodrug topical gel for possible various dermatology conditions, including alopecia areata. For more information, visit Aquestive.com and follow us on LinkedIn. Forward-Looking Statement Certain statements in this press release 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 FDA, including the timing and acceptance of the NDA for Anaphylm with the FDA and potential approved label indications, and the following launch of Anaphylm, if approved by the FDA; the results of the Company’s clinical studies for Anaphylm and the ability of such results to support submission of the NDA for approval of Anaphylm to the FDA; Anaphylm's potential as a non-device, non-invasive based treatment option for severe allergic reactions, including anaphylaxis, if Anaphylm is approved by the FDA; the advancement, growth and related timing of our Adrenaverse™ pipeline of epinephrine prodrug product candidates, including AQST-108 (epinephrine) Topical Gel through clinical development and FDA regulatory approval process, including design and timing of clinical studies including those necessary to support the targeted indication of alopecia areata for AQST-108; the potential benefits our products and product candidates could bring to patients; 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 our other product candidates; risk of delays in advancement of the regulatory approval process through the FDA of our product candidates, including the filing of the respective NDAs, for Anaphylm, AQST-108and 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 PK/PD comparability submission for FDA approval of Anaphylm; risks associated with our ability to address the FDA’s comments on our 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; risks associated with the success of any competing products, including generics; 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 candidates, including Anaphylm and AQST-108; risks associated with the potential impact on the value of the Company of the sale or outlicensing of our product and product candidates, including Anaphylm and other product candidates; risk of insufficient capital and cash resources, including insufficient 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 future clinical development and commercial activities for our product candidates, including Anaphylm and AQST-108, should these product candidates be approved by the FDA; risk that our manufacturing capabilities will be insufficient to support demand for our licensed products in the U.S. and abroad; risk of eroding market share for Suboxone® as a sunsetting product, which accounts for a 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 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 associated with the size and growth of our product markets; risk associated with our 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 product candidates and product pricing, reimbursement or access therefor; risk of loss of significant customers; risks related to claims and legal proceedings against us 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 changing interest rates; risks related to the impact of other pandemic diseases, such as COVID-19, on our business; 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; risks related to uncertainty about presidential administration initiatives and their impact on our business; and other uncertainties affecting us including those described in the "Risk Factors" section and in other sections included in the Company’s 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K filed with the U.S. Securities and Exchange Commission. Given those uncertainties, you should not place undue reliance on these forward-looking statements, which speak only as of the date made. 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 press release whether as a result of new information, future events or otherwise, except as may be required by applicable law. PharmFilm® and the Aquestive logo are registered trademarks of Aquestive Therapeutics, Inc. Investor Inquiries Astr Partners Brian Korb brian.korb@asterpartners.com A photo accompanying this announcement is available at https://www.globenewswire.com/NewsRoom/AttachmentNg/f8452a4a-c453-4def-8656-7e571a206bf7