BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) reported second-quarter 2025 results, highlighting progress toward a planned Biologics License Application (BLA) for nomlabofusp, a potential first disease-modifying therapy for Friedreich’s ataxia (FA). The company said it remains on track to submit the application in the second quarter of 2026 under a potential accelerated approval pathway.
CEO Carole Ben-Maimon, MD, said Larimar has received “written communications in hand from the Food and Drug Administration (FDA) for key elements of our BLA submission,” including safety database recommendations and support for using skin frataxin (FXN) levels as a surrogate endpoint. “With our balance sheet strengthened through our recent capital raise, key clinical data approaching, and a clear regulatory path in place, we are well-positioned,” she added.
The company expects initial data in September from two key efforts: the 50 mg dose cohort in its open-label study and the adolescent pharmacokinetic run-in study. Enrollment is ongoing, with plans to include adolescent participants, FA patients new to nomlabofusp studies, and children aged 2 to 11. The FDA has recommended safety data from at least 30 participants with six months of continuous treatment, and a subset of at least 10 with one year of exposure, primarily at the 50 mg dose.
Global Phase 3 preparations are underway, with sites in the U.S., E.U., U.K., Canada, and Australia expected to begin recruiting later this year. Larimar has also transitioned its open-label study to the lyophilized commercial formulation of nomlabofusp.
Financially, the company ended June 30 with $138.5 million in cash, equivalents, and marketable securities. Including $65.1 million raised in July, Larimar projects its cash runway into the fourth quarter of 2026.
For the second quarter, Larimar posted a net loss of $26.2 million, or $0.41 per share, compared with $21.6 million, or $0.34 per share, a year earlier. Research and development expenses rose to $23.4 million from $19.7 million, driven by higher consulting, personnel, and clinical costs tied to advancing nomlabofusp. General and administrative costs declined slightly to $4.4 million.
The company said it anticipates multiple near-term catalysts, led by September’s clinical updates, which could be pivotal in advancing nomlabofusp toward regulatory submission.
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