Larimar Therapeutics (LRMR) investor relations material

Disease overview and unmet need

• Friedreich's ataxia (FA) is a rare, progressive, and debilitating disease affecting about 5,000 in the U.S. and 20,000 globally, with most patients presenting before age 14.

• FA is caused by a genetic defect leading to frataxin (FXN) deficiency, resulting in severe neurological and cardiac symptoms and early mortality.

• Current approved treatments do not address the underlying FXN deficiency, highlighting a significant unmet medical need.

Nomlabofusp mechanism and preclinical data

• Nomlabofusp is designed to deliver mature human FXN to mitochondria, targeting the root cause of FA.

• Preclinical studies showed nomlabofusp increases mitochondrial FXN, restores mitochondrial function, and improves survival and cardiac function in FXN-deficient mouse models.

Clinical development and regulatory status

• Nomlabofusp has received multiple expedited regulatory designations, including FDA Breakthrough Therapy, Fast Track, Orphan Drug, and Rare Pediatric Disease designations, as well as global designations in the EU and UK.

• The program is part of the FDA START Pilot Program, providing enhanced regulatory support and guidance.

• Rolling BLA submission is planned for June 2026, with final modules expected in 2H 2026 and a potential U.S. launch in 1H 2027.