Larimar Therapeutics (LRMR) Study update summary

Program and Regulatory Milestones

• Rolling BLA submission for nomlabofusp initiated, with the first module submitted and remaining modules expected in the second half of 2026; FDA confirmed the data package is sufficient and reaffirmed willingness to consider FXN as a surrogate endpoint.

• Global confirmatory Phase 3 trial to begin dosing in Q3 2026, enrolling ambulatory patients aged 2–40 years, with sites in the U.S., EU, U.K., Canada, and Australia.

• Multiple regulatory designations awarded in the U.S., EU, and U.K. to expedite development, including Breakthrough Therapy and Orphan Drug Designations.

• Priority review voucher expected at approval, with a potential U.S. launch in mid-2027.

• Financial runway extends into Q2 2027, with $200 million in cash as of March 31.

Study Design and Patient Population

• Open-label study evaluates long-term safety, tolerability, pharmacokinetics, and clinical outcomes of daily 50 mg nomlabofusp, expanded to include adults, adolescents, and children aged 2–11 without prior exposure.

• 76 participants enrolled across the program; 66 received at least one dose; over 10,000 doses administered.

• As of June 2026, 43 participants had received at least one dose in the open-label study; 22 remain in the study, with up to 800 days of treatment.

• Patient population is more severe than typical FA trials, with a mean baseline mFARS score of 55 and about half non-ambulatory; 51% ambulatory and 49% previously exposed to omaveloxolone.

• Baseline characteristics of the open-label group matched to a FACOMS reference group for comparative analysis.

Efficacy and Clinical Outcomes

• Nomlabofusp increased skin FXN levels to those seen in asymptomatic carriers, with 82% reaching target at 6 months and 100% at 1 year and 18 months.

• At 1 year, a 2.6-point mFARS advantage was observed compared to the FACOMS reference group; at 18 months, the advantage increased to 4.6 points.

• Mean mFARS improvement of 1.0 point at 1 year and 2.3 points at 18 months in treated participants, compared to worsening in the reference group.

• Improvements observed in Nine Hole Peg Test and FARS-ADL, with one non-ambulatory participant regaining ambulation and no ambulatory participants losing mobility at one year.

• Correlations observed between FXN increases and clinical improvements, supported by gene expression and lipid biomarker data.