BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) plans to begin a rolling FDA biologics license application submission next month for its experimental Friedreich’s ataxia treatment nomlabofusp as the company advances toward potential accelerated approval.
Larimar said it intends to submit the nonclinical and clinical portions of the application in June, pending additional FDA feedback, with final chemistry, manufacturing, and controls modules expected later in 2026.
The company is seeking accelerated approval for nomlabofusp, which is being developed as a potential treatment for adults and children with Friedreich’s ataxia, a rare inherited neuromuscular disease.
Chief Executive Officer Carole Ben-Maimon said the company remains aligned with the FDA on key elements of its regulatory strategy and expects topline data from an ongoing open-label study later this quarter.
“We are planning to seek accelerated approval and initiate a rolling BLA submission in June with the nonclinical and clinical modules,” Ben-Maimon stated.
Larimar said it plans to begin dosing patients in a global confirmatory Phase 3 study in mid-2026.
The company also disclosed that the FDA granted Breakthrough Therapy Designation to nomlabofusp in February based on clinical data from the ongoing open-label study.
In April, Larimar published nonclinical findings supporting the use of skin frataxin levels as a potential surrogate endpoint for regulatory review. According to the company, the data demonstrated correlations between frataxin levels in skin tissue and other target tissues including heart, brain, and skeletal muscle.
The FDA has indicated willingness to consider frataxin as a novel surrogate endpoint for potential accelerated approval, the company stated.
Larimar reported cash, cash equivalents, and marketable securities totaling $200.4 million as of March 31 and projected its current cash runway into the second quarter of 2027.
The company completed a $115 million public stock offering in February, generating net proceeds of approximately $107.6 million.
Larimar reported a first-quarter net loss of $29.6 million, or $0.31 per share, compared with a net loss of $29.3 million, or $0.46 per share, a year earlier.
Research and development expenses declined to $25.0 million from $26.6 million, while general and administrative expenses increased to $6.1 million from $4.6 million.
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