Larimar Therapeutics, Inc.

Champalimaud Centre for the Unknown is a clinical-stage biotechnology company focused on developing treatments for patients suffering from complex rare diseases using its proprietary cell penetrating peptide technology platform. The company's lead product candidate, nomlabofusp, is a subcutaneously administered recombinant fusion protein designed to deliver frataxin, an essential protein, to the mitochondria of patients with Friedreich's ataxia, a rare, progressive, and fatal genetic disease characterized by insufficient frataxin production. Friedreich's ataxia affects approximately 20,000 individuals globally, with roughly 5,000 in the United States, and currently lacks treatment options addressing the core deficit of low frataxin levels.

Nomlabofusp represents the first potential therapy designed to systemically increase frataxin levels in patients with this condition. The company's cell penetrating peptide platform enables therapeutic molecules to cross cell membranes and reach intracellular targets, with potential applications across multiple rare and orphan diseases characterized by intracellular deficiencies or alterations. The company has received significant regulatory recognition, including Orphan Drug Designation, Fast Track Designation, Pediatric Rare Disease Designation, and Breakthrough Therapy Designation from the FDA, as well as Orphan Drug Designation and access to the European Medicines Agency's Priority Medicines Program in the European Union.

The company has completed four clinical studies, including two Phase 1 trials in adults, a Phase 2 dose exploration study in adults, and a Phase 1 pharmacokinetic run-in study in adolescents. Approximately 8,000 doses of nomlabofusp have been administered throughout the clinical development program. An ongoing open-label study is currently enrolling adults and adolescents with Friedreich's ataxia, with participants receiving 50 milligrams of nomlabofusp daily. The company is targeting a Biologics License Application submission in June 2026 seeking accelerated approval and plans to initiate a global Phase 3 confirmatory study in the second quarter of 2026. As of December 31, 2025, the company had cash, cash equivalents, and marketable securities of $136.9 million, supplemented by $107.6 million in net proceeds from a February 2026 public offering.