Larimar Therapeutics(LRMR) Newsfilter·2025-03-24 11:00Core Viewpoint - Larimar Therapeutics is making significant progress in its nomlabofusp program, with plans for a Biologics License Application (BLA) submission by the end of 2025, aiming for accelerated approval based on promising clinical data and FDA feedback regarding skin frataxin (FXN) concentrations as a potential surrogate endpoint [2][4][11]. Clinical and Regulatory Progress - The company is enthusiastic about recent interactions with the FDA, which are aligned with its approach to using skin FXN concentrations as a novel surrogate endpoint for Friedreich's ataxia (FA) [2][4]. - Larimar is actively enrolling participants in its open label extension (OLE) study, with some participants receiving treatment for up to one year, and plans to report data from this study in September 2025 [2][11]. - The company is on track to initiate a global Phase 3 trial in mid-2025, having received feedback from both the FDA and European Medicines Agency (EMA) on the trial protocol [4][11]. Financial Results - As of December 31, 2024, Larimar reported cash, cash equivalents, and marketable securities totaling 28.8 million for Q4 2024, compared to a net loss of 80.6 million for 2024, up from 26.7 million, significantly higher than $10.6 million in Q4 2023, primarily due to increased manufacturing costs for nomlabofusp [8][12]. Upcoming Developments - Larimar plans to provide an update on the OLE study data in September 2025, focusing on participants receiving the 50 mg dose of nomlabofusp [11]. - The company has amended the OLE protocol to include premedication to mitigate the risk of allergic reactions, including anaphylaxis [11]. - The FDA has accepted the data supporting the transition to a lyophilized form of nomlabofusp, which is expected to be introduced into the clinical development program in mid-2025 [11].