Levacetylleucine

A case study presented at Child Neurology Society Annual Meeting demonstrates preservation of neurological function in sisters with infantile-onset Niemann-Pick disease, type C Additional data presentations reinforce benefit of early and sustained adrabetadex treatment in preserving function and improving long-term outcomes THOUSAND OAKS, Calif.--(BUSINESS WIRE)-- Beren Therapeutics P.B.C. , a leader in cholesterol-trafficking biology and novel cyclodextrin-based therapeutics, today announced new long-term data demonstrating neurological function was preserved for more than 10 years following initiation of treatment with adrabetadex, an investigational cyclodextrin-based medicine under review by the U.S. Food and Drug Administration (FDA) for infantile-onset Niemann-Pick disease, type C (I-NPC). A case study containing these data will be presented at the 55th Annual Meeting of the Child Neurology Society (CNS 2026), taking place October 14–17, 2026, in Montréal, Québec, Canada. Additional poster presentations highlight data showing that adrabetadex was associated with improved survival and slower disease progression and that it was generally well tolerated alone and in combination with other NPC treatments. “Infantile-onset NPC is associated with the most rapid neurodegeneration, accelerated disease progression, and markedly increased risk of premature death,” said Kristina Julich, M.D., Chief of the Pediatric Neurogenetics Center at the Comprehensive Pediatric Epilepsy Center within UT Health Austin Pediatric Neurosciences at Dell Children’s and treating physician for the case study. “Given this natural history of the disease, it is especially impressive to see that neurological function was preserved for more than 10 years in sisters with infantile-onset NPC who began treatment with adrabetadex at very different stages of disease.” Case report shows benefits of early treatment for I-NPC Poster 284: Case Report of Siblings With Infantile-Onset Niemann-Pick Type C (NPC) Initiating Adrabetadex at Different Ages and Intervals From Disease Onset (presented October 15, 2026) This case study follows two sisters who have I-NPC through 10 years of adrabetadex treatment. The older sister began adrabetadex more than four years after the onset of symptoms, which began before she was six years old and included ataxia, gross motor decline, and tremors/dysmetria, when she was classified as having moderate disease. Despite this delayed treatment initiation, she still experienced stabilization or improvement of her symptoms and she maintains moderate disease symptoms after approximately 10 years on adrabetadex. The younger sister began treatment with adrabetadex approximately 10 months after her first neurological symptoms appeared, which has resulted in maintenance of minimal disease activity and symptoms over 10 years. Importantly, treatment with adrabetadex has preserved neurological function for more than 10 years in both sisters, regardless of their stage of disease when they began adrabetadex. “These findings are meaningful in showing that neurological function was preserved for these patients for a decade,” said Irene von Hennigs, PharmD, SVP of Medical Affairs of Beren Therapeutics P.B.C. “In a disease where children can progressively lose the ability to walk, communicate, and develop, preserving function over many years represents an important clinical outcome. These sisters also illustrate why early diagnosis and treatment matter.” Additional Poster Presentations The following posters will also be presented during the conference poster sessions on Thursday, October 15, 2026. Poster 283: Survival outcomes by baseline disease burden in participants with infantile-onset Niemann-Pick disease type C treated with adrabetadex ​ Among 76 individuals treated with adrabetadex for up to 11 years, those with the lowest baseline disease severity (as assessed by NPC Clinical Severity Scale, NPCCSS) demonstrated the longest survival observed (n=29; 100% survival at 5 years), supporting the importance of initiating adrabetadex treatment before advanced disease progression. The findings suggest that earlier restoration of cholesterol trafficking, before substantial neurological disease burden accumulates, may improve long-term outcomes. Poster 281: Outcomes in participants with infantile-onset Niemann-Pick disease type C receiving prompt and sustained treatment with adrabetadex ​ Among 25 patients who received adrabetadex for at least five years, both prompt and delayed treatment initiation were associated with slower disease progression relative to published natural history data. Participants who initiated adrabetadex within one year of neurological symptom onset demonstrated less progression from baseline in NPCCSS scores compared with later treatment initiation, suggesting that earlier initiation of treatment with adrabetadex may result in greater long-term clinical benefit in I-NPC. Poster 275: Early evidence of an emerging NPC treatment paradigm: adrabetadex use alongside arimoclomol and/or N-acetyl-L-leucine ​ The safety pro adrabetadex was generally similar with and without the use of additional therapies, supporting its use as a foundational therapy that can be safely combined with other approved NPC treatments. Most participants initiated adrabetadex prior to complementary therapies, with arimoclomol and/or levacetylleucine frequently added after adrabetadex (when they became commercially available) rather than used instead of adrabetadex. Together, these findings add to the growing body of long-term clinical evidence supporting the potential for adrabetadex to slow progression of I-NPC by addressing impaired cholesterol trafficking, the underlying cause of the disease. About Niemann-Pick Disease, Type C Niemann-Pick disease, type C (NPC) is a rare, autosomal-recessive, severe, heterogeneous, neurodegenerative disorder caused by pathogenic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired intracellular cholesterol trafficking resulting in progressive neurological decline and premature mortality. Infantile-onset NPC (I-NPC) refers to NPC in infants and children who first experience neurological symptoms <6 years of age. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean ages of death 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). About Adrabetadex Adrabetadex is an investigational cyclodextrin-based therapy designed to address the underlying cause of Niemann-Pick disease, type C (NPC) by restoring impaired intracellular cholesterol trafficking. By targeting the biological defect responsible for cholesterol accumulation and downstream neurological injury, adrabetadex is being developed as a potential disease-modifying treatment for infantile-onset NPC. Clinical studies and expanded access experience have generated long-term data on survival, neurological function, and safety, including treatment extending beyond a decade in some individuals. 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. The U.S. Food and Drug Administration (FDA) granted adrabetadex Breakthrough Therapy Designation. In February 2026, the FDA accepted Beren Therapeutics’ New Drug Application (NDA) for adrabetadex in infantile-onset NPC for Priority Review. The agency assigned the NDA a Prescription Drug User Fee Act (PDUFA) target action date of November 17, 2026. Adrabetadex has not been approved by the FDA or any other health authority at this time. About Beren Therapeutics 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 LLC are committed to the development of adrabetadex for individuals living with Niemann-Pick disease, type C (NPC) and have supported the NPC community by providing access to adrabetadex through an Expanded Access Program (EAP). Beren’s public benefit purpose is to discover, develop, and deliver novel therapies that provide optimal benefit for patients, and to do so by integrating the needs of patients, caregivers, clinicians, and health systems from the beginning of the development process and maintaining a long-term focus on delivering meaningful therapies and access. Beren is headquartered in Thousand Oaks, Calif. To learn more about Beren, the adrabetadex program, and Beren’s cholesterol-trafficking focused therapeutic strategy, visit the company’s website , LinkedIn channel or Facebook page . Forward-Looking Statement This press release contains forward-looking statements regarding Beren’s clinical development program, regulatory plans, and the potential of adrabetadex, which is investigational and not yet approved by any regulatory authority. These statements are based on current data — including data from small, uncontrolled case reports and observational analyses — and are subject to risks and uncertainties inherent in drug development. Actual results may differ materially. Contacts Media Contact Beren Therapeutics Media Relations media@berentx.com

Forbion已通过两只基金筹集了23亿欧元（约26亿美元），旨在支持约30家公司，这是过去五年全球规模最大的生命科学风险投资募资之一。这家荷兰公司周二在公告中表示，此次募资规模创下其历史纪录，Forbion目前管理的资产规模约为75亿欧元（84.2亿美元）。这些资金将通过两只新基金部署，分别名为Forbion Growth Opportunities Fund IV和Forbion Ventures Fund VIII，两者都将优先投资于“高潜力”制药商。Forbion不设治疗领域限制，专注于在未满足医疗需求的关键领域开发新型疗法的高质量生命科学公司，这些公司有潜力对医学的未来产生重大影响。Forbion Ventures Fund VIII将“建立、投资并支持创新型生物技术公司”，尤其是临床前和早期临床阶段的制药商；而Growth Opportunities Fund IV则旨在通过“提供私募增长资本，以及精选的交叉投资和公开市场投资”来帮助企业扩张。礼来参与了Forbion的最新一轮募资，其他机构投资方包括KfW Capital、PGM和Kauffman Foundation。 拜耳投资22亿美元，在俄亥俄州建设一座生产原料药和药品制剂的设施，初期聚焦肿瘤、心血管疾病和肾脏护理领域。拜耳预计一个原料药模块将于2031年投入运营，第二个将于2034年上线。拜耳计划在该基地安装数字化和自动化技术。俄亥俄州官员预计，拜耳将围绕新基地创造约600个高价值岗位，以及约1500个建筑岗位。定于明年夏天开放的俄亥俄生命科学培训中心将通过培训生物制造操作员和技术人员，支持拜耳的招聘计划。JobsOhio正向该中心投资最高3000万美元，并将俄亥俄州的劳动力视为拜耳决定在该州建厂的一个因素。 盐野义已着手以20亿美元首付款收购处于商业化阶段的IntraBio。就在数周前，这家总部位于德克萨斯州的生物技术公司刚获得FDA批准，用于一种尚无其他疗法的罕见病。该交易的核心是IntraBio的口服修饰氨基酸Aqneursa。FDA于2024年9月批准其用于尼曼-匹克病C型（NPC），这是一种超罕见、进行性遗传病，影响溶酶体贮积，并可能导致危及生命的并发症，美国约有1000名潜在NPC患者。该公司去年净销售额为6800万美元，全部来自Aqneursa治疗NPC。就在几周前，IntraBio的Aqneursa又获得另一项罕见病批准，将其用途扩展至共济失调性毛细血管扩张症（A-T）患者。A-T是一种过早致命的疾病，涉及小脑进行性退化。该药是全球首个也是唯一一个获批用于A-T的治疗药物。IntraBio继续在其他罕见病中研究Aqneursa，包括GM2神经节苷脂贮积症、遗传性小脑共济失调和CACNA1A相关疾病。这家成立于2015年的生物技术公司还在开发偏头痛和痴呆等常见疾病的治疗方法。盐野义的罕见病产品组合目前以ALS药物Radicava为支柱。盐野义于2025年12月从田边制药收购Radicava，支付25亿美元外加某些基于销售额的特许权使用费。除Radicava外，盐野义还拥有针对脆性X综合征和Jordan综合征的罕见病管线项目。 在等待FDA对其新型非阿片类疼痛治疗药物做出决定之际，Viatris以16.5亿美元收购Pacira BioSciences，以加速其向非阿片类疼痛治疗领域的扩张。Viatris将以每股36.50美元现金收购Pacira。该股在交易宣布前一天收于25.20美元。Viatris预计将在年底前完成收购。Pacira销售Exparel和Zilretta，这两款疼痛治疗药物在2025年下半年和今年上半年分别实现了5.87亿美元和1.21亿美元的销售额。Exparel于2011年获批，是一种注射用布比卡因制剂，用于术后疼痛；而Zilretta于2017年上市，是一种注射用皮质类固醇，适用于膝关节骨关节炎疼痛的管理。Viatris表示，除了收购这些药物外，还将收购Pacira为商业化这些药物而建立的340人团队。这些产品和商业基础设施可能加速Viatris建立非阿片类疼痛缓解产品线的努力。Viatris已提交FDA批准申请，寻求批准一款用于中重度急性疼痛的非阿片类治疗药物。FDA计划在12月27日前对该申请做出裁决。Viatris认为其产品一种速效美洛昔康（FAM）制剂将服务于居家市场，而Exparel和Zilretta则分别面向住院和门诊患者。Viatris表示，尽管存在差异，但同一批术后及高量急性疼痛专科医生将开具这三种药物。公司旨在为FAM创造收入协同效应。Viatris表示，建立美国商业基础设施将使其能够对未来的业务发展采取“机会主义方法”。Pacira目前100%的销售额来自美国。Viatris计划扩张至“精选”国际市场。Pacira已启动全球扩张努力，包括与LG化学合作，将Exparel引入亚太市场。Pacira去年与三家公司达成诉讼和解，同意从2030年初开始允许部分美国仿制Exparel生产，同时正与另外两家希望推出该药仿制版本的公司进行诉讼。 临床前阶段的Matchpoint Therapeutics完成1.5亿美元的B轮融资。这笔资金将帮助Matchpoint将其主要资产MPT-062推进至人体试验。MPT-062设计为口服给药，是一种T细胞调节剂。Matchpoint预计明年启动MPT-062的临床开发。Matchpoint的研发工作依托其先进共价探索（ACE）平台，该平台利用机器学习模型设计小分子药物，这些药物能够与历史上难以靶向的致病蛋白结合。本轮B轮融资由瑞士生物技术投资方Nextech Invest和私募股权公司Norwest共同领投。赛诺菲风险投资曾于2022年10月领投该初创公司1亿美元的A轮融资，此次也参与了最新一轮融资。T1D基金以及Matchpoint的创始投资方Atlas Venture和Access Biotechnology等也加入了B轮融资。 礼来再投入约8.7亿美元，继续就一个新的小分子药物类别展开合作。礼来最初于2025年7月与Gate Bioscience建立合作，利用该生物技术公司的“分子门”引擎来治疗高度未满足需求的疾病，本次扩展协议将在现有协议基础上增加一个未披露的靶点，并有权选择第二个靶点。两项交易均利用Gate的“分子门”发现引擎，在高度未满足需求的疾病中消除特定的难以成药的细胞外蛋白。这家生物技术公司将由此产生的候选药物称为“分子门”。根据这项新的多年协议条款，Gate将获得一笔未披露的首付款，并在选定第二个靶点后再获得一笔付款。根据公告，这家生物技术公司还可能在完成某些临床前、临床和商业里程碑后获得付款，并就任何获批产品的全球净销售额获得分层特许权使用费。交易总价值可能超过8.7亿美元。作为投资回报，礼来获得独家全球许可，可研究、开发和商业化该联盟产生的最终产品。Gate将主导发现工作，而这家大型药企将负责从后期临床前开发到商业化的相关工作。与之前的交易一样，合作双方未披露任何感兴趣的适应症。然而，Gate在其新闻稿中指出，分子门的约4000个靶点中，有许多免疫学、神经科学和心血管代谢健康等类别中的“高价值”靶点，目前尚无获批疗法，或需要注射剂才能治疗。Gate成立于2021年，于2025年11月完成了一轮超额认购的B轮融资，筹集6500万美元，使公司当时的总融资额达到1.35亿美元。礼来作为新投资者加入了这轮由Forbion领投的融资。作为其最初与礼来合作的一部分，Gate有资格获得来自礼来ExploR&D的临床前研发支持，这是该公司早期生物技术生态系统项目Lilly Catalyze360的一部分，该项目提供获取公司专业知识、资源和网络的机会。 基因泰克达成一项价值最高达11.7亿美元潜在交易总额的协议，以获得Alector旨在穿透血脑屏障并治疗帕金森病和阿尔茨海默病等神经退行性疾病的临床前资产。基因泰克将支付1亿美元首付款，获得Alector的AL050在所有适应症上的独家开发和商业化权利。AL050是一种在研工程化葡萄糖脑苷脂酶（GCase）酶替代疗法（ERT），与Alector的血脑屏障递送平台配对使用。这11.7亿美元将以额外里程碑付款以及潜在销售的分层特许权使用费形式支付。作为交换，基因泰克将负责AL050的开发、监管、生产和商业化，而Alector将保留其平台的所有权。AL050旨在解决GCase缺乏症，该症被认为是帕金森病的一个重要风险因素。据Alector称，该酶替代疗法被递送至大脑后，最终应能减少细胞功能障碍并减缓疾病进展。该交易将这家临床前公司的现金跑道延长至2029年。此前，Alector表示其拥有1.728亿美元资金，这笔资金将至少维持到2027年。这笔新资金将用于推进Alector的全人源抗Aβ抗体AL137（该抗体同样源自其平台，设计为皮下注射）在阿尔茨海默病中的开发，并推动siRNA项目通过临床前开发。与Alector的交易将帮助基因泰克母公司罗氏加倍押注脑穿透技术。该药企已拥有自己的血脑屏障技术Brainshuttle，目前正在后期研究中评估trontinemab。这款基于Brainshuttle科学的在研双特异性抗体旨在减少阿尔茨海默病患者的淀粉样蛋白。对Alector而言，与基因泰克的合作是今年早些时候GSK退出之后一个受欢迎的变化。GSK最初于2021年与Alector合作，推进两款神经退行性疾病抗体，但在两款资产均未能显示出显著临床获益后，于7月终止了协议。2025年10月，合作中的一款候选药物latozinemab（Alector最先进的资产），在一项额颞叶痴呆患者的3期研究中未能减缓疾病进展，促使合作双方终止了该项目。在此之前，Alector与艾伯维合作抗体AL002在一项中期研究中未能减缓阿尔茨海默病进展，引发该生物技术公司17%的裁员潮，并最终结束了该联盟。Alector由联合创始人Rosenthal于2013年创立，最初围绕免疫神经学理论构建。但在经历一系列临床失败后，这家加州生物技术公司已围绕其血脑屏障递送项目进行了重建。 CSL将共同开发和共同推广瑞士生物技术公司Alentis Therapeutics的在研抗claudin-1疗法lixudebart，用于治疗肾脏和肝脏疾病，包括原发性硬化性胆管炎和一种以小血管炎症为特征的全身性自身免疫性疾病。CSL预付3.55亿美元，并承诺最高12亿美元。该协议的核心是靶向claudin-1的在研抗体lixudebart，目前正处于中期开发阶段，用于治疗抗中性粒细胞胞浆抗体相关性血管炎伴快速进展性肾小球肾炎（AAV-RPGN）。AAV-RPGN是一种罕见的自身免疫性疾病，患者免疫系统攻击肾脏血管。Lixudebart正在2期RENAL试验中接受评估。两家公司还计划利用lixudebart潜在抗炎和抗纤维化获益，用于治疗肾脏疾病局灶节段性肾小球硬化（FSGS）和肝脏疾病原发性硬化性胆管炎（PSC）。FDA于2024年5月授予lixudebart用于特发性肺纤维化（IPF）的孤儿药资格认定。根据协议，CSL将全额资助RENAL试验直至完成，以及计划中的AAV-RPGN 3期研究。CSL还将承担lixudebart在FSGS和PSC中的中期开发费用。任何获批产品的全球利润将按比例分配，CSL占55%，Alentis获得45%。 Varda Space Industries融资2.51亿美元，用于在太空为地球患者制造药物。Lux Capital和Natural Capital共同领投了Varda的D轮融资，公司估值达16亿美元。Varda由前SpaceX工程师Will Bruey和Founders Fund合伙人Delian Asparouhov于2021年创立，自成立以来已累计融资5.98亿美元。这家总部位于加利福尼亚的初创公司于2023年发射了首次任务，并已完成六次成功再入返回。Varda已在2027年至2029年间向SpaceX及其他提供商预订了28次发射。Lux Capital表示，这些支持反映出业界相信Varda是“首家也是唯一一家实现真正发射与再入节奏的公司”。太空较低的重力水平使物体看起来失重，会以可能影响药物特性（如稳定性和生物利用度）的方式影响晶体形成。微重力对药物分子影响的证据推动了数十年的研究。NASA于1978年资助了一份关于该主题的报告，默沙东的空间站研究为其皮下注射Keytruda制剂提供了信息。从1960年到2025年，将一公斤送入轨道的成本下降了96%，并预计到2040年进一步下降，这使得太空药物商业化生产的一个障碍正在降低。Varda正是寻求利用这一转变的公司之一，将其胶囊搭载商业火箭（如SpaceX的猎鹰9号）送入轨道。胶囊内装有在轨处理药品的设备。完成处理后，胶囊与轨道卫星分离，返回地球并被回收以供分析。其他团队已表明，少量太空生长晶体可支持多个地面生产批次。今年5月，United Therapeutics与Varda合作，开发用于罕见肺部适应症的改良疗法。United Therapeutics首席执行官表示，该协议支持探索“太空制造如何能为罕见肺部疾病治疗带来显著改善”。在轨道上发现和制造药物的竞赛中，Varda和United Therapeutics面临竞争。太空与国防技术公司Redwire旗下的Space Microgravity Development（SpaceMD）已聘请前默沙东研究员Paul Reichert担任战略顾问，以推进轨道研究与制造。SpaceMD已签署一份使用Starfall执行任务的合同，Starfall是SpaceX近期推出的太空制造项目。Voyager Technologies在国际空间站上运营研究设施。该公司的能力支持与Space LiinTech达成的微重力药物发现协议，并将在明年通过一套自动化药物筛选系统得到增强。