Larimar Therapeutics Announces FDA has Removed Partial Clinical Hold for Nomlabofusp Program in Friedreich’s Ataxia

• Food and Drug Administration (FDA) removed partial clinical hold following review of Phase 2 dose exploration study data
• Ongoing open label extension (OLE) study initially evaluating 25 mg; Larimar plans to dose escalate to 50 mg following further characterization of frataxin pharmacodynamics (PD) at the 25 mg dose
• Interim data from OLE study remains on track for Q4 2024
• Biologics License Application (BLA) submission targeted for 2H 2025

BALA CYNWYD, Pa., May 20, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the U.S. FDA has removed the partial clinical hold previously placed on the company's nomlabofusp (CTI-1601) clinical program. Nomlabofusp is currently in development for the treatment of patients with Friedreich’s Ataxia (FA). Nomlabofusp is a novel protein replacement therapy designed to address the root cause of FA by delivering frataxin to mitochondria. The FDA removed the partial clinical hold after a review of data from the Company’s recently completed four-week, placebo-controlled Phase 2 dose exploration study. The review included data from both the 25 mg and 50 mg cohorts in patients who received daily dosing of nomlabofusp for 14 days followed by every other day dosing until day 28.

“We are very excited the FDA has removed the partial clinical hold on our nomlabofusp program following review of our Phase 2 data. Helping patients with FA is our top priority and we appreciate the attention and thorough review by the FDA of all submitted data,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “Importantly, we are now cleared to dose escalate to the 50 mg dose in our ongoing OLE study which we plan to do following further characterization of frataxin PD at the 25 mg dose. The OLE study is evaluating the long-term safety as well as frataxin levels following daily administration of nomlabofusp and we look forward to interim data in the fourth quarter of the year.”

In the Phase 2 dose exploration study, nomlabofusp was generally well-tolerated throughout the four-week treatment period. Nomlabofusp had a predictable pharmacokinetic profile and demonstrated dose-dependent increases in frataxin levels in skin and buccal cells. All patients with quantifiable levels at baseline and Day 14 in the 50 mg cohort achieved frataxin levels in skin cells over 33% of the average level observed in healthy volunteers at Day 14, and 3 patients achieved levels greater than 50% of the average healthy volunteer level.