Larimar Therapeutics (LRMR) said Monday that initial data from its ongoing long-term study of its experimental therapy nomlabofusp in patients with Friedreich's ataxia leaned toward improved clinical outcomes at day 90.
The company said patients treated with daily injections of nomlabofusp saw tissue frataxin levels increase, supporting the drug's potential in treating Friedreich's ataxia.
Larimar said dose escalation has started in six participants, and screening for adolescents with Friedreich's ataxia is ongoing, with dosing expected in early 2025.
The company said it plans to begin a global confirmatory study in mid-2025 and submit a biologics license application in H2 2025 for potential accelerated approval.
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