Larimar Therapeutics (LRMR) Corporate presentation summary

Regulatory designations and FDA engagement

• Nomlabofusp received FDA Breakthrough Therapy, Orphan Drug, Fast Track, and Rare Pediatric Disease designations, with a priority review voucher program extended to 2029.

• Global designations include Orphan Drug and PRIME in the EU and ILAP in the UK.

• FDA aligned on using skin FXN as a surrogate endpoint and confirmed the exposure-response analysis could support a future BLA submission.

• BLA submission is targeted for June 2026, with global Phase 3 study underway at submission.

Clinical development and efficacy data

• Open label study showed 100% of participants at 6 months achieved FXN levels similar to asymptomatic carriers.

• Consistent improvements were observed across mFARS, ADL, 9-HPT, and MFIS after 1 year of treatment.

• Dose-dependent increases in FXN levels were observed in skin and buccal cells across Phase 1 and 2 studies.

• Modeling predicts 50 mg daily dosing can achieve ≥50% of healthy control FXN levels in most patients.

Safety and tolerability

• Over 8,000 doses administered; most common adverse events were mild/moderate injection site reactions.

• Seven cases of anaphylaxis occurred, all resolved with standard treatment and no long-term sequelae.

• Long-term daily dosing was generally well tolerated, with several participants on treatment for over a year.