BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) has announced the publication of two new peer-reviewed articles detailing nonclinical data supporting the therapeutic potential of its investigational frataxin replacement therapy, nomlabofusp, for the treatment of Friedreich’s ataxia (FA).
The publications, now available online, focus on nomlabofusp’s pharmacology, mechanism of action, and its impact on disease-relevant tissues. Titled “Pharmacokinetics and Pharmacodynamics of Nomlabofusp in Non‑clinical Studies of Friedreich’s Ataxia” and “Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria,” the articles highlight evidence that nomlabofusp effectively increases frataxin (FXN) levels in critical tissues such as the dorsal root ganglia, heart, and skeletal muscle.
Larimar emphasized that these nonclinical findings played a key role in discussions with the U.S. Food and Drug Administration (FDA), contributing to the agency’s openness to consider skin FXN concentrations as a reasonably likely surrogate endpoint to support accelerated approval.
“We are pleased to share these two recent publications featuring nonclinical findings that provide evidence of nomlabofusp’s mechanism of action, its ability to increase FXN levels in disease-relevant tissues after administration of doses equivalent to those in our ongoing open-label study,” said Carole Ben-Maimon, MD, President and CEO of Larimar Therapeutics. “Importantly, 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.”
Larimar plans to submit a Biologics License Application (BLA) for nomlabofusp in the second quarter of 2026, positioning it as a potential first disease-modifying therapy for FA, a debilitating rare neuromuscular disorder.
As the company continues to execute on its development milestones, these new data publications mark a significant step in advancing nomlabofusp toward potential regulatory approval and, ultimately, providing a much-needed treatment option for patients living with Friedreich’s ataxia.
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