Larimar Therapeutics (LRMR) investor relations material

Executive summary

• Nomlabofusp clinical development advanced with positive initial and long-term open label study data, showing all participants achieved skin FXN levels similar to asymptomatic carriers after 6 months and consistent improvement in four key clinical outcomes after 1 year.

• Seven cases of anaphylaxis occurred in the first 6 weeks of dosing, all resolved with standard treatment; a modified dosing regimen is being implemented and accepted by the FDA to mitigate this risk.

• Regulatory engagement ongoing in the US and Europe, with plans to update on discussions in Q1 2026 and target BLA submission for accelerated approval in Q2 2026, aiming for a U.S. launch in early 2027.

• Focused on developing nomlabofusp as a potential first disease-modifying therapy for Friedreich's ataxia, with expanded open label studies including adolescents and children.

Financial highlights

• Net loss of $47.7 million for Q3 2025 ($0.61 per share), compared to $15.5 million ($0.24 per share) in Q3 2024; net loss of $103.2 million ($1.50 per share) for the nine months ended September 30, 2025, versus $51.8 million ($0.86 per share) in the prior year period.

• Operating expenses rose to $49.5 million in Q3 2025, driven by higher R&D and manufacturing costs; R&D expenses were $44.9 million and G&A expenses $4.6 million in Q3 2025.

• Cash, cash equivalents, and marketable securities totaled $175.4 million as of September 30, 2025.

• Completed a $65 million public offering in July 2025.

Outlook and guidance

• Cash resources expected to fund operations into Q4 2026, supported by recent equity financing.

• Modified starting dose regimen is being incorporated into the Phase 3 protocol, with global site qualification and study initiation preparations underway.

• Update on regulatory discussions and open label study status planned for Q1 2026; process performance qualification for commercial-scale drug substance planned for Q4 2025.