Larimar Therapeutics (LRMR) Corporate presentation summary

Program highlights and clinical progress

• Nomlabofusp is a first-in-class mitochondrial protein replacement therapy targeting frataxin deficiency in Friedreich's Ataxia (FA), a rare neurodegenerative disease.

• Four clinical studies completed, with ongoing open-label study showing sustained increases in tissue FXN levels and clinical improvements up to 18 months.

• Rolling BLA submission initiated in June 2026, with remaining modules expected in 2H 2026 and a targeted US launch in mid-2027 if approved.

• $156.3 million in cash and investments as of June 30, 2026, with projected runway into Q3 2027.

Disease background and unmet need

• FA affects approximately 5,000 patients in the US and 20,000 globally, with most presenting before age 14.

• Caused by genetic frataxin deficiency, leading to progressive loss of coordination, speech, and early mortality, often from heart disease.

• Current approved treatments do not address the underlying frataxin deficiency.

Mechanism of action and preclinical data

• Nomlabofusp delivers mature human frataxin to mitochondria by leveraging a mitochondrial targeting sequence and cell-penetrating peptide.

• Preclinical studies show mitochondrial localization, restoration of mitochondrial function, and improved survival and cardiac function in FXN knockout mouse models.