原始来源记录
重大申请已提交
Larimar Therapeutics 计划于 2026 年第二季度提交 nomlabofusp 的 BLA 申请，用于治疗 Friedreich’s Ataxia
该公司更新了其企业演示文稿，新增开放标签数据显示 FXN 水平持续升高且临床改善方向明确，并重申计划于 2026 年第二季度提交 BLA 申请，基于皮肤 FXN 水平寻求加速批准。FDA 已同意调整后的给药方案，此前曾出现过敏反应病例。
关键事实
- 申报时间
- 2025年10月14日 11:00
- 事件
- 申请已提交
- 方向
- 积极
- 来源
- SEC 8-K
- 可信度
- 有来源
- 现金
- 1.757亿美元
- 现金储备可维持至
- 2026年Q4
- FXN达成
- 第180天100%的参与者皮肤FXN水平超过健康志愿者50%
来源摘录
EX-99.1 2 lrmr-ex99_1.htm EX-99.1 pro October 2025 Larimar Therapeutics Corporate Deck Forward-Looking Statements This presentation contains forward-looking statements that are based on the beliefs and assumptions of Larimar Therapeutics, Inc. ( “Company”) and on information currently available to management. All statements contained in this presentation other than statements of historical fact are forward-looking statements, including but not limited to Larimar’s ability to develop and commercialize nomlabofusp (CTI-1601) and any other planned product candidates, Larimar’s planned research and development efforts, including the timing of its nomlabofusp clinical trials and non-clinical investigations and overall development plan expectations with respect to the FDA START pilot program,