Larimar Therapeutics publishes nonclinical data on nomlabofusp

Larimar Therapeutics, Inc. (NASDAQ: LRMR) published two peer-reviewed articles detailing nonclinical data on nomlabofusp, its experimental treatment for Friedreich's ataxia. The company announced Monday that the research supports the drug's mechanism of action and its potential use of skin frataxin concentrations as a surrogate endpoint for regulatory approval.

The articles, published online, examine the pharmacokinetics and pharmacodynamics of nomlabofusp and its ability to deliver functional frataxin protein into mitochondria. According to the company, the data demonstrate that nomlabofusp can increase frataxin levels in disease-relevant tissues including dorsal root ganglia, heart, and skeletal muscle at doses equivalent to those used in ongoing clinical studies.

Larimar included this nonclinical data in its briefing package to the U.S. Food and Drug Administration to support using skin frataxin concentrations as a reasonably likely surrogate endpoint for accelerated approval. The company plans to submit its Biologics License Application in the second quarter of 2026.

"These encouraging data contributed to FDA's openness to consider the use of skin FXN concentrations as a reasonably likely surrogate endpoint in support of an accelerated approval," said Carole Ben-Maimon, the company's president and chief executive officer.

Friedreich's ataxia is a rare genetic disorder that affects the nervous system and causes movement problems. Nomlabofusp is designed as a frataxin protein replacement therapy to address the underlying protein deficiency that causes the disease.

The Bala Cynwyd, Pennsylvania-based biotechnology company focuses on developing treatments for complex rare diseases. Information is based on a company press release.

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