Larimar Therapeutics (LRMR) investor relations material

Executive summary

• Breakthrough Therapy Designation granted for nomlabofusp in Friedreich's ataxia (FA) based on open label study data.

• FDA alignment on BLA submission using skin FXN as a surrogate endpoint; BLA submission planned for June 2026.

• Topline open label study data expected Q2 2026; global Phase 3 confirmatory study to begin screening in Q2 2026.

• $115 million public offering in February 2026 strengthens balance sheet and extends cash runway into Q2 2027.

Financial highlights

• Cash, cash equivalents, and marketable securities totaled $136.9 million as of December 31, 2025; pro forma $244.5 million including February 2026 offering.

• Net loss for Q4 2025 was $62.5 million ($0.73/share) vs. $28.8 million ($0.45/share) in Q4 2024.

• Full year 2025 net loss was $165.7 million ($2.27/share) vs. $80.6 million ($1.32/share) in 2024.

• Research and development expenses rose to $59.4 million in Q4 2025 and $154.2 million for 2025, mainly due to manufacturing scale-up and clinical activities.

• General and administrative expenses were $4.6 million in Q4 2025 and $18.3 million for 2025.

Outlook and guidance

• BLA submission for nomlabofusp seeking accelerated approval on track for June 2026; potential U.S. launch in first half of 2027 if approved.

• Topline data from open label study expected Q2 2026; Phase 3 confirmatory study to initiate screening Q2 2026, first patient dosing mid-2026.

• Projected cash runway extends into Q2 2027.