regulatory
confidence high
sentiment positive
materiality 0.70
FDA clears Larimar's Phase 2 CTI-1601 trial to proceed to 50 mg cohort and OLE trial
Larimar Therapeutics, Inc.
- FDA cleared Phase 2 dose exploration trial to proceed to 50 mg cohort; dosed daily for 14 days then every other day to day 28.
- FDA also cleared initiation of open label extension (OLE) trial with 25 mg daily CTI-1601; enrollment expected Q1 2024, interim data Q4 2024.
- Top-line safety, PK, and frataxin data from the 50 mg cohort expected in first half of 2024.
- Clearance follows FDA review of unblinded data from completed 25 mg cohort (n=13) showing frataxin increases vs placebo.
- Further dose escalation and additional U.S. trials remain under partial clinical hold pending FDA review of 50 mg cohort results.