Larimar Therapeutics Reports Preliminary Top-line Data from Phase 2 Trial’s 25 mg Cohort Showing Increases in Frataxin Levels in Patients with Friedreich’s Ataxia and First Quarter 2023 Financial Results

  • Safety data indicate that repeated subcutaneous injections of 25 mg CTI-1601 were generally well tolerated when administered daily for 14 days and then every-other-day thereafter until day 28
  • Daily subcutaneous injections of 25 mg CTI-1601 for 14 days led to increases in frataxin levels from baseline compared to placebo in all evaluated tissues (skin and buccal cells)
  • Median placebo-adjusted increases from baseline of 3.5 pg/µg and 0.9 pg/µg in frataxin levels observed in skin and buccal cells, respectively, with 14 days of daily dosing of CTI-1601
  • Larimar has submitted data to FDA and will meet with the Agency to discuss next steps; update expected in Q3 2023
  • Company management to host webcast and conference call today at 8:00 a.m. ET