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.