STAT+：施维雅15.5亿美元肌营养不良药物未能通过首次重大考验
摘要
法国制药公司施维雅周二表示，其于6月以15.5亿美元现金外加约10亿美元潜在里程碑付款收购的一款实验性药物，在一项长期安慰剂对照试验中未能延缓一种罕见类型的肌营养不良症。这对施维雅和贝克尔肌营养不良症患者而言都是一个出人意料的挫折，后者目前尚无获批的治疗方案。该药物名为sevasemten，最初由Edgewise Therapeutics开发，此前在一项规模较小的随机研究中显示出有希望的结果。施维雅在一份声明中表示：“关键的GRAND CANYON研究结果与既往临床研究中观察到的某些发现不一致。”该公司用这一名称指代其3期试验。“施维雅正在对GRAND CANYO进行全面分析”
English
The French pharma Servier said Tuesday that an experimental drug it purchased in June for $1.55 billion in cash, plus about $1 billion more in potential milestones, failed to slow a rare form of muscular dystrophy in a long placebo-controlled trial. It’s a surprising setback for both Servier and the patients with Becker muscular dystrophy, who currently have no approved treatments. The drug, called sevasemten and originally developed by Edgewise Therapeutics, had previously shown promising results in a smaller, randomized study. “The results of the pivotal GRAND CANYON study are not aligned with certain findings observed in prior clinical studies,” Servier said in a statement, using the company’s name for the Phase 3 trial. “Servier is conducting a comprehensive analysis of the GRAND CANYO