Adrabetadex

突破性疗法向FDA递交上市申请，有望获优先审评资格 Beren Therapeutics宣布，已通过其子公司Mandos为其在研药物adrabetadex向美国FDA递交新药申请（NDA），用于治疗婴儿期发病的C型尼曼匹克病（NPC）。公司同时要求FDA授予该申请优先审评资格。公司表示，此次申报所提交的研究结果显示，接受adrabetadex治疗的婴儿期发病NPC患者生存期显著改善。除临床生存获益证据外，申报资料还纳入了生物标志物及非临床数据等支持性证据，提示该疗法可对疾病的基础病理机制产生作用。此外，adrabetadex整体耐受性良好。 Adrabetadex（VTS-270）是一种由2-hydroxypropyl-β-cyclodextrin异构体构成的专有混合物，目前正被用于NPC治疗的临床研究。通过重建细胞内胆固醇转运，adrabetadex可直接针对NPC的根本病理机制进行干预。Adrabetadex之前曾获FDA授予孤儿药资格、罕见儿科疾病认定与突破性疗法认定（BTD）。 新锐获约1亿美元开发抗体降解剂偶联物 Orum Therapeutics日前宣布完成1450亿韩元（约合1亿美元）的可转换优先股融资。本轮融资由KB Investment领投，现有投资方IMM Investment、Woori Venture Partners及Stassets Investment继续参与；同时引入多家新投资者，包括全球投资机构Weiss Asset Management，以及Korea Investment Partners。除此之外，DSC Investment、Company K Partners、AON Investment和DAYLI Partners也作为新投资者加入本轮融资。 公司表示，本轮资金将用于推进ORM-1153及管线中的其他项目，并持续完善其抗体降解剂偶联物（DACs）平台能力，包括在GSPT1之外开发更多新型药物载荷类别。资金亦将支持公司科研与运营基础设施的扩张，以推动更多靶向DAC项目进入后续开发阶段；相关项目将围绕选择性、效力以及基于理性设计的药物载荷策略展开。上述投入构成Orum核心战略的重要支撑，旨在打造面向肿瘤及其他严重疾病、具备差异化的治疗性DAC产品组合。 获1.65亿美元A轮融资！新锐聚焦心理健康疾病 Syremis Therapeutics宣布正式成立，并完成1.65亿美元融资。本轮A轮融资由Dexcel Pharma与Third Rock Ventures共同领投，Bain Capital Life Sciences、Google Ventures（GV）、QVT以及Pictet参与投资。Syremis表示这笔资金将用于推动其管线项目完成临床概念验证，以加速关键候选项目的临床开发进程。 Syremis聚焦精神健康领域的重大未满足需求，强调其药理学策略建立在新兴且已获得临床验证的神经精神生物学基础之上。Syremis目前披露了两项核心项目：其中，主打项目ST-905是一款高效的M1/M4毒蕈碱受体激动剂，现处于1期临床开发阶段，用于治疗精神分裂症；第二个项目ST-901为新型NMDA受体拮抗剂，当前处于IND支持性研究阶段，预计将于明年进入1期临床开发，用于治疗抑郁症。 参考资料： [1] Orum Therapeutics Secures Approximately US$100 Million to Accelerate Development of Leading Degrader Antibody Conjugate Programs to Treat Serious Diseases. Retrieved December 19, 2025 from https://www.businesswire.com/news/home/20251216175984/en/Orum-Therapeutics-Secures-Approximately-US%24100-Million-to-Accelerate-Development-of-Leading-Degrader-Antibody-Conjugate-Programs-to-Treat-Serious-Diseases [2] Syremis Therapeutics Launches with $165M to Develop Best-in-Class Medicines for Mental Health Conditions. Retrieved December 19, 2025 from https://www.businesswire.com/news/home/20251218743253/en/Syremis-Therapeutics-Launches-with-%24165M-to-Develop-Best-in-Class-Medicines-for-Mental-Health-Conditions [3] Beren, Through its Subsidiary Mandos, Submits New Drug Application to U.S. FDA for Adrabetadex in Infantile-Onset NPC. Retrieved December 19, 2025 from https://www.businesswire.com/news/home/20251219752330/en/Beren-Through-its-Subsidiary-Mandos-Submits-New-Drug-Application-to-U.S.-FDA-for-Adrabetadex-in-Infantile-Onset-NPC 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

THOUSAND OAKS, Calif.--(BUSINESS WIRE)--Beren Therapeutics P.B.C.® (“Beren”) today announced that its subsidiary Mandos LLC® (“Mandos”) has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for adrabetadex, an investigational therapy for the treatment of infantile-onset Niemann-Pick disease type C (NPC). The application was based on data supporting a finding of improved survival in an externally controlled survival analysis, biomarker and nonclinical confirmatory evidence, and patient experience narratives. Adrabetadex is an investigational, proprietary cyclodextrin therapy designed to increase intracellular cholesterol trafficking to target the underlying pathology of NPC. “The submission of the NDA marks an important milestone for adrabetadex,” said Dr. Alexander Gold, Chief Medical Officer, Beren Therapeutics P.B.C. “The totality of the evidence – including a rigorous survival analysis compared to external controls – supports the potential of adrabetadex to provide meaningful clinical benefit for patients living with NPC. We are grateful to the patients with NPC and their caregivers, clinicians, and advocates who have worked with the Mandos development team to make this possible.” At the American Neurological Association (ANA) and Child Neurology Society (CNS) annual meetings in 2025, Beren/Mandos presented analyses that were featured in the NDA submission, including: Survival analyses that support improved outcomes in adrabetadex-treated patients with infantile-onset NPC compared to matched external controls Biomarker analyses demonstrating reductions in markers of neuronal injury, including fatty acid-binding protein 3 (FABP3) and calbindin D, and increases in a marker of neuronal cholesterol trafficking (24S-OHC), supporting a mechanistic rationale for the observed clinical outcomes The survival analysis received Abstract of Distinction recognition at ANA. Beren/Mandos will present additional data at the WORLDSymposiumTM in San Diego, CA, February 2-6, 2026. “Submitting this NDA reflects our commitment to the NPC community and to advancing therapies grounded in rigorous science,” said Jason Camm, Chief Executive Officer, Beren Therapeutics P.B.C. “We believe the data included in this submission demonstrate adrabetadex has the potential to alter the course of disease. We are proud to take this step toward making a potential new treatment option available to families affected by NPC.” Data from the clinical trials and expanded access programs suggest that adrabetadex is well-tolerated. The most common adverse events associated with treatment include hearing impairment (manageable with hearing aids if needed) and transient post-dose fatigue and/or ataxia. The FDA granted Breakthrough Therapy Designation (BTD) in 2025 for adrabetadex in infantile-onset NPC, recognizing the potential to address a serious condition with substantial improvement over existing options. The FDA has a 60-day filing review period to determine whether the NDA is complete and accepted for review. Mandos intends to continue providing adrabetadex to eligible patients through an Expanded Access Program (EAP) during the FDA review process. About Niemann-Pick Disease Type C Niemann-Pick disease type C (NPC) is a rare, autosomal-recessive, severe, neurodegenerative disorder caused by pathologic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired cholesterol trafficking resulting in progressive neurological decline and premature death. Infantile-onset NPC refers to NPC in individuals who first experience neurological symptoms between 0 and 6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean survival of ~5.6 years for early-infantile-onset (age of neurological onset <2 years) and ~13.4 years for late-infantile-onset (2 to <6 years). Individuals with early- and late-infantile-onset NPC typically present with manifestations affecting multiple organs, with the most severe and debilitating effects occurring in the brain. About Adrabetadex (VTS-270) Adrabetadex (VTS-270) is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation as a treatment for Niemann-Pick disease type C (NPC). The data suggests that by re-establishing intracellular cholesterol trafficking, adrabetadex directly addresses the underlying pathology of NPC. Adrabetadex is generally well tolerated. The main adverse events associated with adrabetadex include hearing impairment that can be managed with hearing aids when necessary, and post-dose fatigue and/or ataxia. Adrabetadex has not been approved by the FDA or any other health authority at this time. About Beren Therapeutics P.B.C. and Mandos Beren Therapeutics P.B.C. is a founder-led, clinical-stage biotechnology company pioneering the discovery, development, and commercialization of cyclodextrin-based therapeutics for conditions characterized by defective cholesterol trafficking. Beren and its subsidiary, Mandos, are committed to the development of adrabetadex for individuals living with Niemann-Pick disease type C (NPC), a condition characterized by defects in intracellular cholesterol trafficking. Beren and Mandos have supported the NPC community by providing access to adrabetadex* through an Expanded Access Program (EAP). Beren will continue working closely with patients, families, researchers, regulators, and others on a path to bring forth this potentially transformative, investigational therapy for NPC. Beren is headquartered in Thousand Oaks, CA, and will launch its website in Q1 2026. For more information, please visit our public-facing subsidiary Mandos. * Adrabetadex is an investigational drug that has not been approved by the U.S. Food and Drug Administration and has not been found safe and effective to treat NPC or any other condition.