Larimar Therapeutics announced positive 25 mg and 50 mg data from its ongoing long-term open label (OL) study of nomlabofusp for Friedreich's ataxia. The data showed consistent directional improvements across four key clinical outcomes, including modified Friedreich Ataxia Rating Scale (mFARS), FARS-Activities of Daily Living (ADL), 9 Hole Peg Test (9-HPT), and Modified Fatigue Impact Scale (MFIS), when compared to a Friedreich’s Ataxia Clinical Outcomes Measure Study (FACOMS) reference population.
The study also observed increased skin frataxin (FXN) levels, with participants achieving levels equivalent to more than 50% of those found in healthy volunteers, which are comparable to levels in asymptomatic carriers. Long-term treatment with daily nomlabofusp was generally well-tolerated, with 8 participants treated for over one year. As of August 27, 2025, 39 participants in the OL study had received at least one dose of nomlabofusp, and 25 participants were receiving daily dosing for up to 527 days.
However, seven OL study participants experienced anaphylaxis and were withdrawn from the study, with most events occurring on the initial day of administration and all within the first six weeks. In response, Larimar modified its starting dose regimen to include a 5 mg test dose, followed by a 25 mg dose one hour later under observation, then 25 mg daily for 30 days, before increasing to 50 mg daily. The Biologics License Application (BLA) submission remains on track for the second quarter of 2026.
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