Larimar Therapeutics, Inc., a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, last week presented data from the Company’s Phase 1 studies and the Phase 2 dose exploration study of nomlabofusp at the International Congress for Ataxia Research (ICAR) in London, U.K.
Data from a total of 61 adults with FA who participated in these studies evaluating short-term (up to 28 days) subcutaneous administration of 25, 50, 75, and 100 mg nomlabofusp were further evaluated and presented in three posters during the conference.
- Treatment with nomlabofusp modified gene expression and lipid profiles in addition to increasing frataxin (FXN) levels in study participants with Friedreich’s ataxia (FA)
- Modeling and simulation predict that, in most patients with FA, 50 mg of nomlabofusp administered daily is likely to achieve FXN levels that are ≥50% 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
- Nomlabofusp program update expected mid-December 2024