Larimar reports Q2 2026 update on nomlabofusp BLA, Phase 3 timing and cash

Larimar Therapeutics reported second quarter 2026 results on August 4, 2026, along with pipeline updates for nomlabofusp, its lead candidate for Friedreich's ataxia. The company said it initiated a rolling BLA submission in June, submitting the first module seeking accelerated approval.¹ Completion of that submission is expected in the second half of 2026, and Larimar is targeting a mid-2027 launch if the drug is approved.

Following a Type B multidisciplinary pre-BLA meeting, FDA said the existing data package looks capable of supporting a BLA submission and review for accelerated approval based on the open label study, with approval to be determined through the review process.¹ The agency also said it remains open to treating skin frataxin (FXN) as a novel surrogate endpoint, and indicated Larimar's analysis linking drug exposure to clinical outcomes could support the filing.¹ FDA further noted that gene expression and lipid biomarker data collected during the trial may help characterize nomlabofusp's biological activity beyond FXN tissue levels alone.¹

On the open label study, as of June 2026, 43 adolescent and adult participants had received at least one dose of nomlabofusp, with 22 remaining in the study and a maximum treatment duration exceeding 800 days; more than 10,000 doses have been given in total.¹ Skin FXN levels rose after dosing, with 82% of participants (9 of 11) exceeding asymptomatic-carrier levels by six months and 100% reaching that threshold at one year.¹ The company reported a 2.6-point benefit in mFARS at one year versus the FACOMS natural history comparison.¹

Larimar said it is on track to begin dosing in its global confirmatory Phase 3 study during the current quarter.¹

As of June 30, 2026, the company held $156.3 million in cash, cash equivalents and marketable securities, which it projects will fund operations into the third quarter of 2027.¹