Larimar Therapeutics(US:LRMR) Globenewswireยท2025-09-29 11:00Core Insights - Larimar Therapeutics announced positive results from an ongoing long-term open label study of nomlabofusp for treating Friedreich's ataxia, showing improvements in clinical outcomes and skin frataxin levels [1][2][3] Clinical Study Results - The open label study involved 65 participants, with 39 receiving nomlabofusp, and demonstrated consistent improvements across four key clinical outcomes compared to a reference population [3][12] - After one year of treatment, participants showed a median improvement in the modified Friedreich Ataxia Rating Scale (mFARS) score of -2.25, while the reference population experienced a worsening of 1.00 [12][14] - All participants who received nomlabofusp for at least six months achieved skin frataxin levels similar to asymptomatic carriers, with 10 out of 10 participants exceeding 50% of median levels found in healthy volunteers [3][8] Safety and Tolerability - Nomlabofusp was generally well-tolerated, with most adverse events being mild to moderate local injection site reactions [9][10] - Anaphylaxis was reported in 7 participants, all occurring within the first six weeks of treatment, leading to a modification of the starting dose regimen [3][9] Development Program Updates - Larimar plans to submit a Biologics License Application (BLA) seeking accelerated approval in Q2 2026 [3][20] - The dosing regimen has been modified to include a 5 mg test dose followed by a 25 mg dose, with plans to enroll younger patients in the future [15][20] Company Overview - Larimar Therapeutics is focused on developing treatments for complex rare diseases, with nomlabofusp as its lead candidate for Friedreich's ataxia [17]