Larimar Therapeutics (NASDAQ:LRMR) experienced a 17% drop in its share price on Monday following publication of open-label study results and the submission of the initial module of a rolling Biologics License Application (BLA) to the Food and Drug Administration for nomlabofusp, its investigational therapy for Friedreich's ataxia.
The company said it delivered the first BLA module to the FDA, with the remaining modules scheduled for submission in the second half of 2026. The filing follows an FDA Type B multidisciplinary pre-BLA meeting in which the agency and Larimar aligned on aspects of the submission process.
Larimar reported that daily administration of nomlabofusp in the ongoing open-label study produced increases in skin frataxin - the protein deficient in Friedreich's ataxia - that were sustained at both the one-year and 18-month assessments. All nine participants with one-year data reached and maintained skin frataxin levels above 50% of the mean observed in healthy volunteers, a level the company described as comparable to asymptomatic heterozygous carriers.
The open-label cohort included 43 adolescent and adult participants who had received at least one dose of nomlabofusp. As of June 2026, 22 participants remained actively enrolled in the study. Approximately 49% of enrollees were non-ambulatory at baseline.
On functional measures, participants demonstrated directional improvement across multiple clinical endpoints at one year when compared with a natural history reference population. Larimar highlighted that one of six participants who were non-ambulatory at baseline had regained ambulatory function after one year of dosing. Conversely, none of the seven participants who were ambulatory at baseline had progressed to non-ambulatory status during that period.
The company disclosed safety outcomes, reporting that anaphylaxis occurred in 10 of the 43 patients in the open-label study. Nine of those ten had prior exposure to nomlabofusp in an earlier study; among 11 participants with no prior exposure, one experienced anaphylaxis. Larimar characterized long-term dosing as generally well tolerated and noted that more than 10,000 doses of nomlabofusp have been administered within the open-label program.
Looking ahead, Larimar expects to dose the first patient in a global confirmatory Phase 3 trial in the third quarter of 2026. The company has publicly stated a target of launching the product in mid-2027, contingent on regulatory approval.
Context and next steps
The rolling BLA process allows the company to submit data modules to the FDA incrementally; Larimar has signaled that further modules will be provided in the latter half of 2026. The Phase 3 start and potential mid-2027 commercialization timeline remain dependent on successful progression through the confirmatory trial and regulatory review.