Larimar Therapeutics Announces FDA Clearance to Initiate the 25 mg Cohort of a Phase 2 Dose Exploration Trial of CTI-1601 in Friedreich’s Ataxia Patients
- Initiation of the Phase 2 trial is expected in Q4 2022, with top-line data expected in 2H 2023
- Company management hosting webcast and conference call today at 8:30 a.m. ET
Excerpt from the Press Release:
BALA CYNWYD, Pa., Sept. 14, 2022 (GLOBE NEWSWIRE) — Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has cleared the initiation of the 25 mg cohort of a Phase 2, four-week, placebo-controlled, dose exploration trial of CTI-1601 in Friedreich’s ataxia (FA) patients. In a written communication to Larimar, the FDA indicated it was lifting its full clinical hold on the CTI-1601 program and imposing a partial hold. The design of the upcoming Phase 2 trial is identical to the design proposed by Larimar, with the exception of a requirement for the FDA to review data from the 25 mg cohort prior to escalating the dose in the second cohort. Larimar expects to begin the Phase 2 trial in Q4 2022, with top-line data expected in 2H 2023.
“We thank the FDA for their engagement and are pleased with their decision to clear CTI-1601’s return to the clinic,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “Given the strength of our Phase 1 data and the urgent need for a disease-modifying FA therapy, we believe today’s news is an important event for not only Larimar, but for the entire FA community. We are now working expeditiously to initiate our Phase 2 dose exploration trial next quarter. We anticipate that the results of this trial will provide crucial safety, pharmacokinetic, and pharmacodynamic data that will inform the design of future studies.”
The CTI-1601 program was placed on a clinical hold by the FDA following the Company’s notification to the agency of 3 mortalities out of a total of 34 animals in a 26-week non-human primate (NHP) toxicology study designed to support extended dosing of patients with CTI-1601.
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