Larimar Therapeutics presents Phase 1 and 2 dose studies of nomlabofusp

Larimar Therapeutics (LRMR) presented data from the company’s Phase 1 studies and the Phase 2 dose exploration study of nomlabofusp in Friedreich’s ataxia, or FA, at the International Congress for Ataxia Research in London, U.K. FA is an autosomal recessive neurodegenerative disorder caused by GAA repeats in the FXN gene that result in FXN deficiency. Nomlabofusp study participants were representative of the broad population of adults with FA. Treatment with nomlabofusp modified gene expression and lipid profiles in addition to increasing frataxin – FXN – levels in study participants with Friedreich’s ataxia. Modeling and simulation predict that, in most patients with FA, 50 mg of nomlabofusp administered daily is likely to achieve FXN levels that are greater than or equal to50% of levels observed in healthy controls and similar to mean FXN levels reported in asymptomatic heterozygous carriers. Disease characteristics of adult participants in the nomlabofusp studies were representative of the broad population of adults with FA. Relationships between tissue FXN levels and onset of disease and GAA repeat length observed at baseline in nomlabofusp clinical study participants were consistent with prior published studies. A nomlabofusp program update is expected mid-December 2024.