Larimar Therapeutics (LRMR) investor relations material

Executive summary

• Nomlabofusp, a therapy for Friedreich's ataxia, demonstrated a favorable long-term safety profile, sustained increases in skin frataxin (FXN) levels, and improvements in key clinical outcomes compared to natural history data, with high patient and investigator enthusiasm.

• Rolling BLA submission for accelerated approval was initiated in June 2026, with the first module submitted and completion expected in the second half of 2026; FDA and EMA engagement ongoing, and FDA agreed to consider FXN as a surrogate endpoint.

• Four clinical studies completed and an ongoing open-label study with over 10,000 doses administered; ten participants experienced anaphylaxis and discontinued, all recovered with standard therapy.

• Dosing of the first patient in the global confirmatory Phase 3 study is expected in Q3 2026, with sites in the US, EU, UK, Canada, and Australia.

Financial highlights

• Cash, cash equivalents, and marketable securities totaled $156.3 million as of June 30, 2026, with a projected cash runway into Q3 2027.

• Net loss was $32.8 million for Q2 2026 and $62.4 million for the six months ended June 30, 2026, compared to $26.2 million and $55.5 million for the same periods in 2025.

• Research and development expenses for Q2 2026 were $28.0 million, up from $23.4 million in Q2 2025, mainly due to manufacturing, consulting, and BLA preparation costs.

• General and administrative expenses rose to $6.4 million in Q2 2026 from $4.4 million in Q2 2025, driven by commercial readiness activities.

• Stockholders' equity increased to $127.4 million as of June 30, 2026, from $78.1 million at December 31, 2025.

Outlook and guidance

• Cash resources expected to fund operations into Q3 2027, but not for a full year from the financial statement issuance date; additional capital will be needed.

• Preparing for a potential mid-2027 commercial launch of nomlabofusp, pending regulatory approval.

• Rolling BLA submission completion expected in H2 2026; first patient dosing in Phase 3 study anticipated in Q3 2026.