BMN-331

上个月，BioMarin已经宣布将其研发管线削减一半。近日，该公司已经确认，作为研发重组的一部分，170名员工也将离开公司。在上个月底公布第一季度财报的同时，BioMarin表示将停止4个项目的开发而优先开发另外3个更具潜力资产。被放弃的管线包括两个已进入临床的项目BMN 331和BMN 255和两个临床前项目。BMN 331是一种用于遗传性血管性水肿患者的基因疗法，正在进行I/II期试验，BMN 255正在进行代谢功能障碍相关脂肪性肝炎（MASH）的I期试验。临床前项目BMN 355和BMN 365均为治疗心脏病候选药物，前者是一款单克隆抗体，旨在治疗2型和3型长QT间期综合征（LQTS）；后者旨在用于治疗plakophilin-2突变和致心律失常性右心室发育不良/心肌病。BioMarin发言人昨天在一份电子邮件声明中表示，此次裁员约占公司员工总数的5%，与“投资组合评估”有关。BioMarin在提交给美国证券交易委员会的文件中解释说，公司管理层上周同意裁员，由于遣散费和其他员工解雇福利，第二季度可能会产生约1500万至2000万美元的税前费用。虽然BioMarin在公布第一季度财报时并未明确提及裁员，但该公司当时确实表示，管线重组将在今年节省多达6000万美元的研发成本，其中2000万美元将用于加速优先项目。Copyright © 2024 PHARMCUBE. All Rights Reserved.欢迎转发分享及合理引用，引用时请在显要位置标明文章来源；如需转载，请给微信公众号后台留言或发送消息，并注明公众号名称及ID。免责申明：本微信文章中的信息仅供一般参考之用，不可直接作为决策内容，医药魔方不对任何主体因使用本文内容而导致的任何损失承担责任。精彩预告线下活动↑长按扫码，填表报名↑

The majority of affected employees were notified yesterday, BioMarin said. BioMarin had already announced plans to cut its pipeline in half, but now the company has confirmed that 170 employees are also leaving the building as part of the R&D restructure. The Voxzogo and Roctavian maker revealed last month that it was halting four programs including BMN 331, a gene therapy being assessed in a phase 1/2 trial for patients with hereditary angioedema, and BMN 255, which was being evaluated in a phase 1 trial for metabolic dysfunction-associated steatohepatitis (MASH). Two preclinical programs were also terminated: a monoclonal antibody called BMN 355 designed to treat long-QT syndrome types 2 and 3; and BMN 365, which was targeted at plakophilin-2 mutations and arrhythmogenic right ventricular dysplasia/cardiomyopathy. Instead, the company is focusing on three programs that “met the highest bar for advancement.” They include BMN 351, a next-gen oligonucleotide in a phase 1/2 trial for patients with Duchenne muscular dystrophy, as well as BMN 349, a phase 1-stage oral small molecule for alpha-1 antitrypsin deficiency-associated liver disease. The final program to have been spared the ax is BMN 333, a preclinical C-type natriuretic peptide being developed for multiple growth disorders. The job cuts represent around 5% of the company’s workforce and are related to this “portfolio assessment,” a BioMarin spokesperson told Fierce Biotech in an emailed statement yesterday. The layoffs, which the company’s management agreed on last week, are likely to incur pre-tax charges of around $15 to $20 million during the second quarter as a result of severance and other employee termination benefits, BioMarin explained in a SEC filing (PDF). The majority of affected employees were notified yesterday, with the layoffs likely to be completed by the end of July, according to the filing. “We are grateful to the impacted employees for their dedication to our mission of developing transformative medicines for patients,” the spokesperson told Fierce Biotech. “BioMarin is committed to providing support through this transition for those employees impacted.” While BioMarin didn’t explicitly refer to any layoffs when the strategic rethink was outlined during first-quarter earnings in April, the company did say at the time that the pipeline restructure would save up to $60 million in R&D costs this year, offset by up to $20 million that will be spent on accelerating the prioritized programs.