Larimar Therapeutics (LRMR) Status Update summary

Regulatory and Clinical Development Updates

• FDA provided written recommendations for the BLA safety database, requiring at least 30 participants with six months' exposure and 10 with one year, primarily at the 50 mg dose.

• FDA is open to using skin frataxin (FXN) concentrations as a surrogate endpoint for accelerated approval, with final acceptability to be determined during BLA review.

• BLA submission for accelerated approval is targeted for Q2 2026, with a planned U.S. launch in early 2027.

• Participation in the FDA's START pilot program has expedited development and clarified regulatory expectations.

• The global Phase III study will serve as the confirmatory trial, enrolling 100–150 ambulatory patients aged 2–40 years, focusing on upright stability and mFARS as primary outcomes.

Clinical Data and Safety Profile

• Nomlabofusp has shown dose-dependent increases in tissue frataxin levels and early trends toward clinical improvement in multiple studies.

• The therapy is generally well tolerated, with the most common adverse events being mild, self-limited injection site reactions.

• Anaphylaxis has been observed, particularly in patients with prior exposure, and antihistamine premedication is now used.

• No withdrawals due to injection site reactions have occurred, and safety data continues to be monitored.

• OLE study data from 30–40 participants on the 50 mg dose, including adolescents and new patients, expected September 2025.

Study Design and Enrollment

• All patients in the open-label study are now on the 50 mg dose, with ongoing enrollment of adults, adolescents, and potentially children aged 2–11.

• The adolescent PK run-in study is complete, with data from 14 participants expected September 2025; eligible participants are transitioning into the open-label study.

• The open-label study is expanding to include adolescents, children, and patients new to Nomlabofusp trials.

• Lyophilized drug product formulation, stable at room temperature, has been introduced for commercialization.

• Global Phase 3 study site identification and qualification ongoing in the US, Europe, UK, Canada, and Australia.