Larimar Therapeutics provides updates on nomlabofusp program for FA

Informative Adolescent PK Run-In Data: Adolescents 12 to 17 years of age received a weight-based equivalent of 50 mg for 7 days. Exposure and PK in adolescents spanning 12 to 17 years of agewere similar to adults on 50 mg of nomlabofusp. Six adolescents are currently enrolled in the OL study Planned Enrollment in OL Study: The OL study protocol was amended to include adolescent and adult patients who have not participated in a prior nomlabofusp study. Recently, Larimar modified the starting dose regimen and is implementing this change. The new dosing regimen will include a 5 mg test dose followed by a 25 mg dose one hour later under observation. Nomlabofusp 25 mg will then be administered once daily through Day 30 and then the dose will be increased to 50 mg once daily. Larimar plans to enroll patients 2-11 years of age directly into the OL study in the future. Global Phase 3 Study: Sites are identified and being qualified in the U.S., Europe, U.K., Canada, and Australia. Developments in Chemistry Manufacturing and Control: Received agreement from FDA on analytical testing requirements including potency testing of nomlabofusp. Process performance qualification on the commercial scale drug substance is planned in Q4 2025, in preparation of data for BLA submission. Drug substance manufactured during PPQ activities are expected to be used as the initial commercial launch supply. Key Upcoming Milestones: Implement the new dosing regimen in the OL study in Q4 2025; PPQ on commercial scale drug substance planned in Q4 2025; BLA submission seeking accelerated approval targeted in Q2 2026