Larimar receives FDA clearance to proceed to 50mg in Phase 2 trial of CTI-1601

Larimar Therapeutics announced that the U.S. Food and Drug Administration, FDA, has cleared the Company’s four-week, placebo-controlled, Phase 2 dose exploration trial of CTI-1601 in patients with Friedreich’s ataxia to proceed to a 50 mg cohort in which participants will be dosed daily for the first 14 days, and then every other day until day 28. In addition, Larimar’s open label extension, OLE, trial was also cleared for initiation by the FDA. Participants in the OLE will receive 25 mg of CTI-1601 daily. Larimar received clearance to advance its Phase 2 trial to a 50 mg cohort and initiate its OLE trial following a review by the FDA of Larimar’s complete response to its partial clinical hold that included unblinded safety, pharmacokinetic, and pharmacodynamic data from the Phase 2 trial’s completed 25 mg cohort. Data from the completed 25 mg cohort indicated that CTI-1601 was generally well tolerated and showed increases in frataxin levels from baseline compared to placebo in all evaluated tissues at day 14.