Larimar Therapeutics Announces FDA Lifted Partial Hold on Nomlabofusp Program for Friedreich’s Ataxia

Larimar Therapeutics Inc., a clinical-stage biotech firm, has announced that the U.S. Food and Drug Administration (FDA) has lifted a partial clinical hold on its nomlabofusp (CTI-1601) program. Nomlabofusp is currently being developed as a treatment for Friedreich’s Ataxia (FA), a rare genetic disorder. This novel protein replacement therapy aims to address the primary cause of FA by delivering frataxin, a crucial protein, to mitochondria. The FDA's decision follows a review of data from a completed four-week, placebo-controlled Phase 2 dose exploration study that featured 25 mg and 50 mg doses administered to patients.

Carole Ben-Maimon, MD, the President and CEO of Larimar, expressed excitement over the FDA's approval to escalate to a 50 mg dose in their ongoing open label extension (OLE) study. The study, which initially focused on evaluating a 25 mg dose, aims to assess long-term safety and frataxin levels in patients. Interim data from this study is expected by the fourth quarter of 2024, and a Biologics License Application (BLA) submission is planned for the second half of 2025.

The Phase 2 dose exploration study showed that nomlabofusp was generally well-tolerated over the four-week period. The study demonstrated predictable pharmacokinetics and dose-dependent increases in frataxin levels in both skin and buccal cells. Notably, in the 50 mg cohort, all patients with quantifiable baseline levels achieved significant increases in frataxin levels in skin cells, some reaching over 50% of the average level found in healthy individuals by Day 14.

The ongoing OLE study is set to evaluate the long-term impacts of daily subcutaneous injections of 25 mg of nomlabofusp, either self-administered or administered by a caregiver. Larimar plans to escalate the dose to 50 mg after further characterizing the pharmacodynamics of frataxin at the 25 mg dose. Any escalation beyond 50 mg will require additional FDA review and data submission.

Nomlabofusp has received multiple designations from the FDA, including Rare Pediatric Disease, Fast Track, and Orphan Drug designations. It has also been granted Orphan Drug designation by the European Commission and a PRIME designation by the European Medicines Agency. These designations highlight the significance and potential of nomlabofusp in treating Friedreich's Ataxia.

Larimar Therapeutics is focused on developing treatments for complex rare diseases. The company is leveraging its intracellular delivery platform to design other fusion proteins targeting rare diseases characterized by deficiencies in intracellular bioactive compounds. Larimar's lead compound, nomlabofusp, exemplifies their commitment to addressing unmet medical needs in rare disease communities.