BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) began a rolling U.S. application for accelerated approval of its Friedreich’s ataxia drug candidate as development and launch preparations pushed its second-quarter net loss to $32.8 million.
The clinical-stage biotechnology company submitted the first module of its Biologics License Application for nomlabofusp in June and expects to complete the filing in the second half of 2026. Larimar is seeking accelerated approval based on data from its ongoing open-label study.
The company expects to dose the first patient in a global confirmatory Phase 3 study during the third quarter. It is targeting a mid-2027 commercial launch if the drug receives approval.
The regulatory push follows a Type B pre-BLA meeting with the Food and Drug Administration. According to Larimar, the FDA indicated that the existing data package appears capable of supporting submission and review of an accelerated-approval application, though any approval remains subject to the agency’s review.
The FDA also agreed to a rolling submission and reaffirmed its willingness to consider frataxin, or FXN, as a novel surrogate endpoint, according to the company. Larimar reported that the agency indicated an exposure-response analysis connecting nomlabofusp exposure with clinical outcomes may support the application.
Nomlabofusp is being developed for Friedreich’s ataxia, a rare inherited disease. Larimar is evaluating whether increasing frataxin levels can address the underlying biology of the condition.
As of June, 43 adolescents and adults had received at least one dose in Larimar’s open-label study, with 22 participants remaining in the trial. Maximum treatment duration exceeded 800 days, and more than 10,000 doses had been administered.
The study has also produced significant treatment-related safety events. Ten participants experienced anaphylaxis and discontinued treatment, including nine who had previously been exposed to nomlabofusp.
All 10 responded to standard therapy and returned to their usual state of health without further complications, according to Larimar. Among 11 participants without previous exposure to the drug, one experienced anaphylaxis.
Three additional participants discontinued after experiencing generalized urticaria. Larimar reported no new occurrences following the introduction of antihistamine therapy.
Another three participants discontinued because of other adverse events, while five left for reasons unrelated to treatment, primarily logistical factors. In total, 21 participants have discontinued since the study began in January 2024.
Larimar reported sustained increases in skin frataxin levels among participants remaining in the study. Nine of 11 participants reached levels above those observed in asymptomatic carriers by six months, while all nine participants evaluated at one year and all three evaluated at 18 months reached that threshold.
The company also reported directional improvements in clinical measures compared with the Friedreich’s Ataxia Clinical Outcome Measures Study natural-history reference population. Among 13 participants evaluated after one year, nomlabofusp was associated with a 2.6-point benefit on the Modified Friedreich Ataxia Rating Scale relative to that reference population.
The comparisons were not described in the release as results from a randomized controlled trial.
Larimar President and Chief Executive Officer Carole Ben-Maimon characterized the period as a critical stage in the drug’s development, pointing to the rolling BLA, open-label data and preparations for the confirmatory trial.
The company had $156.3 million in cash, cash equivalents and marketable securities at June 30 and expects that capital to fund operations into the third quarter of 2027.
Larimar’s second-quarter net loss widened to $32.8 million, or 30 cents per share, from $26.2 million, or 41 cents per share, a year earlier.
Research and development expenses increased to $28 million from $23.4 million. Larimar attributed the increase primarily to manufacturing activities and higher professional and consulting costs associated with clinical trials, data analysis, FDA inspection readiness and preparation of the BLA.
General and administrative expenses rose to $6.4 million from $4.4 million as Larimar increased spending ahead of a potential commercial launch. Those costs included additional personnel, market development, commercial-readiness activities and legal expenses.
For the first six months of 2026, Larimar recorded a net loss of $62.4 million, or 61 cents per share, compared with $55.5 million, or 87 cents per share, a year earlier.
Six-month research and development expenses increased to $53 million from $49.9 million, while general and administrative expenses rose to $12.4 million from $9.1 million.
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