Larimar Therapeutics, a biotechnology company, saw a significant increase in its shares, rising by 10% to $3.44. This surge follows the approval of the company’s Phase 2 trial for CTI-1601 in patients with Friedreich’s ataxia by the U.S. Food and Drug Administration (FDA).
Advancing to the Next Phase
With the FDA’s clearance, Larimar Therapeutics will now proceed to a 50-mg cohort in the dose-exploration trial. Participants in this phase will receive daily doses for the first 14 days and then alternate every other day until day 28.
Extension Trial Receives Green Light
In addition to the phase advancement, Larimar Therapeutics also received FDA approval for its open-label extension trial. Participants in this trial will be administered a daily dosage of 25 mg of CTI-1601.
A Promising Therapy for Friedreich’s Ataxia
CTI-1601, a novel protein replacement therapy developed by Larimar Therapeutics, aims to deliver frataxin to the mitochondria of patients with Friedreich’s ataxia who have low levels of frataxin. Previous data from the completed 25 mg cohort of the Phase 2 trial showed promising results, with CTI-1601 being well tolerated and demonstrating increases in frataxin levels from baseline.
Special Designations Highlighting Potential
CTI-1601 has been granted several notable designations for its potential in treating Friedreich’s ataxia. These include Orphan Drug, Rare Pediatric Disease, Fast Track, and PRIME designations.
