Key Takeaways Replimune shares fell 54.2% in three months after the FDA issued a CRL against its RP1 BLA in July.The FDA cited shortcomings in the IGNYTE study data but noted no safety concerns with RP1.Replimune met with the FDA to discuss next steps, with a clear regulatory path still uncertain.Replimune Group’s (REPL) share price tumbled 54.2% over the past three months. The massive setback was observed after the FDA issued a complete response letter (CRL) in July, against the biologics license applicati ...

Key Points A crucial meeting with a regulator didn't seem to go well. It was about a leading pipeline drug being developed by the company. 10 stocks we like better than Replimune Group › On Thursday, Replimune (NASDAQ: REPL) had a crucial meeting with federal regulators regarding one of its leading drug candidates. While the outcome of the event wasn't necessarily negative, it left the future of the drug up in the air, and investors didn't like that uncertainty. As a result, they assertively traded ...

公司研发进展 - 公司宣布其新药bitopertin用于治疗红细胞生成性原卟啉症（EPP）的预新药申请（NDA）会议获得美国食品药品监督管理局（FDA）的积极反馈 [1] - 公司与FDA就NDA提交的预期时间和内容达成一致，计划于2025年10月提交NDA [2] - NDA将基于公司现有的数据包，通过FDA的加速批准途径进行提交 [2] 核心产品信息 - Bitopertin是一种研究性、临床阶段的口服甘氨酸转运体1（GlyT1）抑制剂，旨在调节血红素生物合成 [3] - 公司计划开发bitopertin作为一系列血液疾病的潜在治疗方法，在EPP中可能成为首个疾病修饰疗法 [3] - 公司于2021年5月通过一项许可协议从罗氏获得bitopertin的全球权利 [4] 目标疾病领域 - 红细胞生成性原卟啉症（EPP）是一种罕见、使人衰弱且可能危及生命的疾病，由影响血红素生物合成的突变引起 [5] - 该病导致患者暴露在阳光下时产生严重反应，包括剧痛、水肿、灼烧感，并可能导致起泡和毁容 [5] - 20-30%的患者会出现并发症，包括胆结石、胆汁淤积和肝损伤，极端情况下会导致肝功能衰竭 [5] - 目前EPP无法治愈，仅有一种FDA批准的疗法Scenesse® [5] 公司战略定位 - 公司是一家临床阶段的生物制药公司，致力于发现、开发和商业化治疗严重血液疾病的新型疗法 [6] - 公司正在构建一系列创新的、可能成为同类首选的候选疗法，旨在通过靶向红细胞生物学的基本生物学途径（特别是血红素生物合成和铁稳态）来应对广泛的血液疾病 [6]