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Larimar Therapeutics Publishes Nonclinical Data Supporting the Therapeutic Potential of Nomlabofusp in Patients with Friedreich’s Ataxia
GlobeNewswire· 2025-07-08 11:00
Core Insights - Larimar Therapeutics is advancing its lead compound, nomlabofusp, as a potential treatment for Friedreich's ataxia, with a Biologics License Application (BLA) submission planned for Q2 2026 seeking accelerated approval [1][2] Company Overview - Larimar Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, with nomlabofusp being its lead compound targeting Friedreich's ataxia [3] Research Findings - Two peer-reviewed articles have been published that provide evidence of nomlabofusp's mechanism of action, demonstrating its ability to increase frataxin (FXN) levels in relevant tissues after administration [2][6] - The data from these studies support the potential use of skin FXN concentrations as a reasonably likely surrogate endpoint for the FDA's consideration in the accelerated approval process [1][2] Future Plans - The company is focused on executing near-term catalysts to advance nomlabofusp as the first potential disease-modifying therapy for patients with Friedreich's ataxia [2]