Larimar Therapeutics (NASDAQ:LRMR) shares declined 17% on Monday after the company released new data from its ongoing open-label study and confirmed the submission of the first section of its rolling Biologics License Application (BLA) seeking accelerated approval for nomlabofusp as a treatment for Friedreich’s ataxia.
The biotechnology company said the initial BLA module has been submitted to the U.S. Food and Drug Administration, with the remaining sections scheduled for submission during the second half of 2026. The filing follows discussions with the FDA during a Type B multidisciplinary pre-BLA meeting.
Long-Term Study Shows Sustained Frataxin Levels
Updated results from the ongoing open-label trial demonstrated that once-daily treatment with nomlabofusp increased and maintained skin frataxin levels after one year and 18 months of therapy.
All nine participants with one-year follow-up data achieved and sustained frataxin levels above 50% of the average observed in healthy volunteers, reaching levels comparable with asymptomatic heterozygous carriers.
The study has enrolled 43 adolescent and adult patients who received at least one dose of nomlabofusp, with 22 participants still actively receiving treatment as of June 2026. Nearly half of the study population (49%) was non-ambulatory when treatment began.
Phase 3 Study Planned for Later This Year
Patients receiving nomlabofusp also demonstrated directional improvements across several clinical outcome measures after one year compared with a natural history reference population.
Among six participants who were non-ambulatory at baseline, one regained the ability to walk after one year of treatment, while none of the seven ambulatory participants experienced progression to non-ambulatory status during the same period.
The company reported anaphylaxis in 10 of the 43 treated patients, with nine of those individuals having previously received nomlabofusp in an earlier clinical study. Among the 11 participants without prior exposure, one experienced anaphylaxis. Larimar added that long-term treatment has generally been well tolerated, with more than 10,000 doses administered during the open-label study.
The company expects to enrol the first patient in its global confirmatory Phase 3 trial during the third quarter of 2026 and continues to target a potential commercial launch in mid-2027, subject to regulatory approval.