Larimar Therapeutics Announces the Dosing of the First Patient in Long-term Open Label Extension Study for Nomlabofusp in Patients with Friedreich’s Ataxia

• Study will inform on long-term safety profile and tissue frataxin levels
• OLE initiated with 25 mg daily subcutaneous injections of nomlabofusp
• Frataxin data and safety data from the OLE study are intended to help support a potential Biologics License Application (“BLA”) submission for accelerated approval targeted for H2 2025
• Initial data expected in Q4 2024

BALA CYNWYD, Pa., March 11, 2024 (GLOBE NEWSWIRE) -- Larimar Therapeutics, Inc. (“Larimar”) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced dosing of the first patient in an open label extension (OLE) study evaluating 25 mg daily subcutaneous injections of nomlabofusp in participants with Friedreich’s ataxia (FA). Nomlabofusp (CTI-1601) is a novel protein replacement therapy designed to address the root cause of Friedreich's ataxia (FA) by delivering frataxin to mitochondria.

“We are pleased to dose the first patient in our OLE study, further advancing the nomlabofusp clinical program and building on the successful completion of our Phase 2 dose escalation study. Importantly, the OLE study will inform on the long-term safety profile and tissue frataxin levels and provide a first look at real-life experience with self-administration by patients or administration by a caregiver. Participants who completed treatment in the recent Phase 2 dose exploration trial, or who previously completed a prior Phase 1 clinical trial of nomlabofusp are potentially eligible to screen for the OLE. Based on our Phase 1 and Phase 2 findings, we expect to continue daily dosing throughout the study,” said Carole Ben-Maimon, MD, President, and Chief Executive Officer of Larimar. “In February we announced that we intend to pursue frataxin as a potential novel surrogate endpoint to support accelerated approval. The frataxin data, supportive pharmacodynamics and clinical outcomes information and safety data from the OLE study, along with additional nonclinical pharmacology information will be used to help support a potential BLA submission for accelerated approval targeted for the second half of 2025. We look forward to reporting initial data from the OLE study in the fourth quarter of 2024.”