Larimar Therapeutics

Larimar Therapeutics reported Q3 2024 net loss of $15.5 million ($0.24 per share) with $203.7 million in cash, projecting runway into 2026. Their lead program, nomlabofusp for Friedreich's ataxia, advances with milestones: program update in mid-December 2024, PK run-in study in adolescents by year-end, global confirmatory study mid-2025, and BLA submission targeted for 2H 2025. The company received ILAP designation from MHRA for accelerated UK market access.

Larimar presented positive early clinical results for nomlabofusp (CTI-1601), a drug designed to correct frataxin deficiency in FRDA patients. The phase 1 study involved 55 patients, with primary endpoints of safety and tolerability. A phase 2 trial showed increased frataxin levels with doses of 25 mg and 50 mg. An ongoing open-label extension study will include adolescents and children, with preliminary data expected in Q4 2024. Larimar aims for accelerated FDA approval with a BLA submission planned for mid-next year.