Larimar Therapeutics(US:LRMR) Globenewswireยท2025-06-23 11:00Core Viewpoint - Larimar Therapeutics, Inc. is progressing towards the submission of a Biologics License Application (BLA) for nomlabofusp, a potential treatment for Friedreich's Ataxia, with a planned submission in the second quarter of 2026 following FDA recommendations for safety data inclusion [1][4][9] FDA Recommendations - The FDA has provided clear recommendations for the safety database, requiring at least 30 participants with continuous exposure for 6 months, including a subset of at least 10 participants with 1-year exposure, primarily from the 50 mg dose group [4][5] - The FDA is open to the use of skin frataxin (FXN) concentrations as a reasonably likely surrogate endpoint for the BLA submission, acknowledging the relationship between increased skin FXN and relevant tissues [5][9] Clinical Development Progress - Enrollment in the open label extension (OLE) study is ongoing, with plans to include adolescents and patients who have not previously participated in clinical studies [2][4] - Data from the OLE study, expected in September 2025, will include results from 30-40 participants who received at least one dose of nomlabofusp, focusing on the 50 mg dose [4][9] - Adolescent pharmacokinetic (PK) run-in data is also anticipated in September 2025 from 14 participants, some of whom received a placebo [4][9] Global Phase 3 Study - Activities for the global Phase 3 study are ongoing, with site identification and qualification in the U.S., Europe, U.K., Canada, and Australia [4][9] - The Phase 3 study is intended to serve as a confirmatory study to verify clinical benefit as required by the FDA's accelerated approval pathway [9] Company Overview - Larimar Therapeutics is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp as its lead compound targeting Friedreich's Ataxia [7]