Nomlabofusp (CTI-1601)

March 19, 2026: Larimar Therapeutics, Inc. (NASDAQ: LRMR) reported Q4/full-year 2025 results and provided regulatory and clinical updates for nomlabofusp in Friedreich’s ataxia (FA). In February 2026, FDA granted Breakthrough Therapy Designation for nomlabofusp for adults and children with FA based on available clinical data from an ongoing open-label study. Following an FDA START pilot program meeting and review of preliminary data, the company said it remains aligned with FDA on key elements for a planned accelerated-approval BLA, including potential use of skin frataxin (FXN) as a novel surrogate endpoint, use of matched FACOMS natural history controls and advance statistical plan review, and that safety adequacy will be determined at BLA review. FDA also agreed the confirmatory global Phase 3 trial should be underway at BLA submission, with change from baseline in Upright Stability Score (USS; mFARS subscale) as the proposed primary endpoint.

Upcoming catalysts: topline open-label data in Q2 2026; Phase 3 screening to begin Q2 2026 with first patient dosing expected mid-2026; BLA submission planned for June 2026; targeted U.S. launch in 1H 2027 if approved. The company also noted a February 2026 public offering with $107.6M net proceeds and pro forma cash of $244.5M as of Dec. 31, 2025, extending runway into Q2 2027.