Larimar Therapeutics Reports Positive Initial Data from Nomlabofusp OLE Study for Friedreich’s Ataxia

Dec. 16, 2024. Larimar Therapeutics announced positive results from its ongoing open label extension study evaluating the daily subcutaneous administration of 25 mg nomlabofusp in participants with Friedreich’s ataxia. In a cohort of 14 patients, the treatment was generally well tolerated for up to 260 days, with tissue frataxin levels significantly increasing in both buccal and skin cells by Day 90. 

Plans for a global confirmatory study are set for mid-2025, with a Biologics License Application targeted for submission in the second half of 2025. Early trends towards improvement in clinical outcomes observed at Day 90, supporting the potential for nomlabofusp to benefit a broad spectrum of patients with Friedreich’s ataxia.

Serious adverse events occurred in two study participants during the OLE study, which, despite resolving quickly, may raise concerns about the safety profile of nomlabofusp.

Larimar Therapeutics Reports Positive Initial Data from Nomlabofusp OLE Study for Friedreich’s Ataxia