Allos Therapeutics, Inc.

正文共：1716字  2图预计阅读时间：7分钟作者 | 子系良文章来源 | 药时代公众号FDA收紧加速批准要求已是近年来大势所趋。为什么加速批准的要求越来越严格，还不是以前有太多药物钻了加速批准的空子，非但没有达成”患者获益“的目标，还可能因为其模糊的疗效，导致患者的生命健康受到损害。FDA也不得不为曾经不够严格的加速批准还债……将在明日（2023年11月16日）召开的FDA肿瘤药物咨询委员会（ODAC）正是例证：本次ODAC会议将直接决定两款加速批准后迟迟未完成确证性试验的”复发或难治性外周t细胞淋巴瘤“药物的前路命运。Folotyn (pralatrexate)和Beleodaq (belinostat)是两款治疗复发或难治性外周t细胞淋巴瘤的产品，曾经分属于不同药企，但都在2019年由Acrotech Biopharma以3亿美元的打包价格和其他五款药物打包买下。这两种药物已经超过了最初计划的完成验证研究的日期，在报告发布之时，Beleodaq已逾期15个月，而Folotyn已逾期72个月（整整6年！）……而且两款药物均通过客观缓解率（ORR）取得了加速批准，至今仍没有总生存期（OS）甚至是中位生存期（PFS）的数据。虽然Beleodaq也有不少问题，但“头铁”程度显然没有Folotyn这么严重，感觉更像是“反正药企代表来都来了，不如趁这个机会给你旗下两款药全掰扯明白”这种感觉。我们还是把重点放在这款确证性试验逾期72个月的药物上。被导过多手的天价抗癌药Folotyn（普拉曲沙）由Allos Therapeutics公司开发，是首个用于治疗外周 T 细胞淋巴瘤 (PTCL）的二氢叶酸还原酶（DHFR）抑制剂，最早于2009年9月获得FDA批准上市。Folotyn也是全球首款DHFR抑制剂，在当时确实有不可替代性。2012年4月，Spectrum以2.06亿美元收购Allos获得这款药物。2019年，该公司又以3亿美元的价格将这款药物出售给印度仿制药巨头阿拉宾度的子公司Acrotech Biopharma。但无论是哪家公司，似乎对于推进Folotyn的确证性试验兴趣不高。至今FOLOTYN的药物官网仍然标注“通过ORR获得批准，仍未取得PFS或者OS数据”。尽管迟迟没有完成确证性试验，Folotyn的销售价格却居高不下，甚至在近几年还进行了涨价。由Fierce Pharma发布的2023年美国最昂贵药物排名TOP10中，以每年84.26万美元的花费位居第十名。要知道Folotyn是一款已经上市14年的药物，在基因治疗药物还没有如此兴盛之前，Folotyn的排名还要更高。2020年国内上市仍靠ORR在美国之外的市场，Folotyn一直都有动静，不光地区权益多次易手，还在中国做了一个新的3期试验。这款药物于2020年8月26日以优先审评方式获得NMPA批准，在中国大陆地区上市，本次批准基于FOT12-CN-301研究，主要终点仍是ORR。丁香园用药助手提供的Folotyn中文说明书显示，FOT12-CN-301研究是一项在中国复发或难治性 PTCL 患者中评价普拉曲沙有效性和安全性的单臂、多中心的3期临床试验。主要分析基于所有受试者完成了 5 个周期治疗的时间点(数据截止日期)。主要终点是基于国际工作组标准(IWC)的客观缓解率 (ORR)。次要疗效终点为至缓解时间 (TTR)、缓解持续时间 (DOR) 、无进展生存期(PFS) 、总生存期(OS)。按照独立中心审核 WC 法评估，安全性人群 (n= 71)的客观缓解率为 52.1% (37171 例受试者，95%CI: 39.9-64.1%)。目前该试验还未给出完整的PFS、OS数据。连中国大陆都已上市3年，FDA终于忍不住要过问一句，FOLOTYN的确证性试验，做得咋样了啊？小结本次ODAC会议将会要求Acrotech递交两款药物的临床试验情况说明，确实不由得让人期待药企代表将会怎么解释上市14年没做完确证性试验的情况。当然对于FDA而言更重要的是——如何杜绝类似情况再次发生，让加速批准成为真正有助于解决临床需求的好规定，而不是“获得批准后直接当撒手掌柜，勒令撤市时，商业生命早就吃干抹净了 ”参考资料：1.FDA官网2.Jun Zhu, Ee Min Yeoh, Yoshinobu Maeda & Kensei Tobinai (2020) Efficacy and safety of single-agent pralatrexate for treatment of angioimmunoblastic T-cell lymphoma after failure of first line therapy: a pooled analysis, Leukemia & Lymphoma, 61:9, 2145-2152, DOI: 10.1080/10428194.2020.17652323.丁香园用药助手4.其他公开资料血脂，礼来的下一个狙击点……我国第一个天然药物1.1类新药批准上市！糖足溃疡年需求高达数百亿元，多家药企积极争取合作商机司美格鲁肽心血管获益试验详细结果公布，主要不良心血管事件风险降低20%，且不仅与减重有关！点击在看 共济新药研发浪潮

HAMILTON, ON and BOSTON, Oct. 16, 2023 /PRNewswire/ -- Fusion Pharmaceuticals Inc. (Nasdaq: FUSN), a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines, announced the appointment of Jeremy Bender, Ph.D., M.B.A., Teresa Bitetti, M.B.A., and David Meek to its Board of Directors, effective today. "We are excited to welcome Jeremy, Teresa and David to Fusion's Board of Directors, each bringing an exceptional breadth of experience and track record of success in oncology drug development, business development and commercialization. Their experiences introducing innovative therapies to cancer patients as leaders of top-tier oncology companies will be invaluable as we advance our pipeline, particularly our lead program FPI-2265, which is on track to be the first actinium-based PSMA targeted alpha therapy (TAT) to market," said Fusion Chief Executive Officer John Valliant, Ph.D. "Fusion is undergoing an exciting evolution to a later stage radiopharmaceutical company and with these appointments we are transforming our Board to maximize the value generation of our strategy." Dr. Bender, Ms. Bitetti and Mr. Meek succeed Johan Christenson, Ph.D., Chau Khuong and Heather Preston, M.D., who will step down from Fusion's Board effective today. "I want to thank Johan, Chau and Heather for their significant contributions and unwavering support that enabled Fusion to become a vertically integrated radiopharmaceutical company with a deep clinical-stage pipeline and a proprietary radiopharmaceutical manufacturing facility," Dr. Valliant said. Dr. Jeremy Bender is currently Chief Executive Officer of Day One Biopharmaceuticals, a clinical-stage biopharmaceutical company focused on pediatric cancer. Previously, Dr. Bender served as Vice President of Corporate Development at Gilead Sciences, overseeing acquisitions, partnerships and investments. He also served as Chief Operating Officer at Tizona Therapeutics, Chief Business Officer at Sutro Biopharma, and Vice President of Corporate Development at Allos Therapeutics, and began his career at Boston Consulting Group in the life sciences practice. He currently sits on the Board of Mereo BioPharma as an Independent Board Member. Dr. Bender holds a B.S. in Biological Sciences from Stanford University, a Ph.D. in Microbiology and Immunology from the University of Colorado and an M.B.A. from the MIT Sloan School of Management. Ms. Teresa Bitetti currently serves as President of the Global Oncology Business Unit of Takeda. Previously, Ms. Bitetti held various roles of increasing responsibility at Bristol-Myers Squibb Company (BMS), including as Senior Vice President, Head of Worldwide Oncology Commercialization. At BMS, she also served as Senior Vice President and Head of the U.S. Oncology business, President and General Manager of BMS Canada, and Worldwide Head of the BMS Virology business. Before BMS, Ms. Bitetti also held various roles of increasing responsibility at Mobil Oil Corporation. Ms. Bitetti currently serves on the board of Osmol Therapeutics as an independent director. Ms. Bitetti holds a B.A. from Wellesley College and an M.B.A. from the Darden School of Business at the University of Virginia. Mr. David Meek most recently served as Chief Executive Officer and Member of the Board of Directors of Mirati Therapeutics. Prior to this role, he served as the President, CEO and Board Member of FerGene and CEO and Board Member of Ipsen. Mr. Meek was previously an Executive Vice President and President of Oncology at Baxalta Incorporated in the leadup to the acquisition by Shire and Chief Commercial Officer of Endocyte, and he held executive leadership roles at Novartis Pharmaceuticals Corporation and Novartis Oncology. Mr. Meek has served on the boards of Pharmaceutical Research & Manufacturers of America and European Federation of Pharmaceutical Industries & Associations. He currently serves on the Board of Directors of uniQure N.V. and previously served on the Board of Directors of Entasis Therapeutics (acquired by Innoviva). Mr. Meek holds a B.A. from the University of Cincinnati. About Fusion Fusion Pharmaceuticals is a clinical-stage oncology company focused on developing next-generation radiopharmaceuticals as precision medicines. Fusion connects alpha particle emitting isotopes to various targeting molecules to selectively deliver the alpha emitting payloads to tumors. Fusion's clinical portfolio includes: FPI-2265 targeting prostate specific membrane antigen (PSMA) for metastatic castration resistant prostate cancer currently in a Phase 2 trial; FPI-1434 targeting insulin-like growth factor 1 receptor currently in a Phase 1 trial; and FPI-2059, a small molecule targeting neurotensin receptor 1 (NTSR1), currently in a Phase 1 trial. In addition to a robust proprietary pipeline, Fusion has a collaboration with AstraZeneca to jointly develop novel targeted alpha therapies (TATs) and combination programs between Fusion's TATs and AstraZeneca's DNA Damage Response Inhibitors (DDRis) and immuno-oncology agents. The Company recently received IND clearance for FPI-2068, the first novel TAT under the collaboration, which targets EGFR-cMET. Fusion has also entered into a collaboration with Merck to evaluate FPI-1434 in combination with Merck's KEYTRUDA® (pembrolizumab) in patients with solid tumors expressing IGF-1R. To support Fusion's growing pipeline of TATs, the Company has signed strategic actinium supply agreements with TRIUMF, Niowave, Inc. and BWXT Medical. Contact: Amanda Cray Senior Director of Investor Relations & Corporate Communications (617) 967-0207 [email protected] SOURCE Fusion Pharmaceuticals

EMERYVILLE, Calif.--(BUSINESS WIRE)-- OmniAb, Inc. (NASDAQ: OABI) announces that Director Sunil Patel passed away suddenly last week. The Board of Directors and leadership of OmniAb extend their deepest condolences to those who loved and cared for Sunil and express their gratitude for Sunil’s many contributions to OmniAb. Sunil Patel was appointed to OmniAb’s Board in November 2022 when OmniAb was spun-out of Ligand Pharmaceuticals as an independent, publicly-traded company, and previously served on the Ligand Pharmaceuticals Board of Directors since 2010. “It is with profound sadness that we announce Sunil’s passing,” said John Higgins, OmniAb Board Chair. “Sunil was a highly-valued and engaged member of the OmniAb Board of Directors and played an important role driving our investments and strategy. Above that, he was a friend and colleague for the past two decades. He will be remembered by all who had the pleasure of working with him as a very thoughtful business operator, strongly devoted to cutting-edge science supporting medical research. He was kind and gentle, and an independent thinker who brought levity and joy to any meeting or encounter. Sunil’s support for antibody discovery research and the formation of OmniAb are among his most important business contributions. He saw the future of where the field was going and at every turn helped to drive our strategy. He will be remembered by those who had the pleasure of knowing him as a warm and incredibly bright individual of high integrity. For all who knew Sunil, we will honor his legacy as we advance our work.” With more than 25 years of senior management and R&D experience in the biotechnology industry, Mr. Patel most recently worked as an independent consultant to biotechnology companies advising on strategy and corporate development initiatives. Previously, he served in executive roles at OncoMed Pharmaceuticals, Inc., including Executive Vice President and Chief Financial Officer. He held senior management positions in corporate development, marketing and strategy with BiPar Sciences, Inc., Allos Therapeutics, Inc., Connetics, Abgenix, Inc. and Gilead Sciences Inc. Early in his career Mr. Patel worked at McKinsey & Company serving biotechnology and pharmaceutical clients, and held scientific research positions at ZymoGenetics, Inc. and ProCyte Corporation. He received an undergraduate degree in chemistry from the University of California, Berkeley and a master’s degree in molecular bioengineering/biotechnology from the University of Washington. About OmniAb® OmniAb’s discovery platform provides pharmaceutical industry partners access to diverse antibody repertoires and high-throughput screening technologies to enable discovery of next-generation therapeutics. At the heart of the OmniAb platform is the Biological Intelligence™ (BI) of our proprietary transgenic animals, including OmniRat®, OmniChicken® and OmniMouse® that have been genetically modified to generate antibodies with human sequences to facilitate development of human therapeutic candidates. OmniFlic® (transgenic rat) and OmniClic® (transgenic chicken) address industry needs for bispecific antibody applications though a common light chain approach, and OmniTaur™ features unique structural attributes of cow antibodies for complex targets. We believe the OmniAb animals comprise the most diverse host systems available in the industry and they are optimally leveraged through computational antigen design and immunization methods, paired with high-throughput single B cell phenotypic screening and mining of next-generation sequencing datasets with custom algorithms to identify fully human antibodies with superior performance and developability characteristics. These proprietary technologies are joined with and leverage OmniDeep, which is a suite of in silico tools for therapeutic discovery and optimization that are woven throughout OmniAb’s various technologies and capabilities. Additionally, an established core competency focused on ion channels and transporters further differentiates OmniAb’s technology and creates opportunities in many emerging target classes. OmniAb antibodies have been leveraged across modalities, including bispecific antibodies, antibody-drug conjugates and others. The OmniAb suite of technologies span from BI-powered repertoire generation to cutting-edge antibody discovery and optimization offering a highly efficient and customizable end-to-end solution for the growing discovery needs of the global pharmaceutical industry. For more information, please visit . View source version on businesswire.com: Contacts OmniAb, Inc. Neha Singh, Ph.D. investors@OmniAb.com Twitter (X) @OmniAbTech (510) 768-7760 Source: OmniAb, Inc. View this news release online at: