Larimar Therapeutics Reports Positive Top-line Data from Phase 2 Dose Exploration Study from 25 mg and 50 mg Cohorts of Nomlabofusp in Patients with Friedreich’s Ataxia

  • Nomlabofusp was generally well tolerated following repeated subcutaneous injections in patients in the 25 and 50 mg cohorts with no serious adverse events
  • Dose dependent increases in frataxin levels were observed in skin and buccal cells
  • Open Label Extension (OLE) study initiated in January 2024 to dose 25 mg daily of nomlabofusp with dosing anticipated to be starting later this quarter; Initial data expected in Q4 2024
  • Initiated discussions with the FDA on use of tissue frataxin levels as a novel surrogate endpoint to support a potential Biologics License Application (“BLA”) submission for accelerated approval targeted for 2H 2025