BALA CYNWYD, Pa. — Larimar Therapeutics, Inc. (Larimar) (Nasdaq: LRMR), a clinical-stage biotechnology company focused on developing treatments for complex rare diseases, today announced the publication of two peer-reviewed articles highlighting nonclinical data on the therapeutic potential, pharmacology, and mechanism of action of nomlabofusp as a novel frataxin (FXN) protein replacement therapy designed to address the underlying cause of Friedreich’s ataxia (FA). These data were included in the briefing package reviewed by the U.S. Food and Drug Administration (FDA) in support of potentially using skin FXN concentrations as a reasonably likely surrogate endpoint (RLSE) for Larimar’s registrational program seeking accelerated approval for nomlabofusp.
The articles, now available online, are titled:
- “Pharmacokinetics and Pharmacodynamics of Nomlabofusp in Non‑clinical Studies of Friedreich’s Ataxia” (link)
- “Nomlabofusp, a Fusion Protein of Human Frataxin and a Cell Penetrant Peptide, Delivers Mature and Functional Frataxin into Mitochondria” (link)
“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 like dorsal root ganglia, heart, and skeletal muscle after administration of doses equivalent to the dose administered in our ongoing open label study,” said Carole Ben-Maimon, MD, President and Chief Executive Officer of Larimar. “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. Our Biologics License Application (BLA) submission is planned for the second quarter of 2026. We continue to focus on executing across our near-term catalysts to further advance nomlabofusp as the first potential disease modifying therapy for patients with FA.”
About Larimar Therapeutics
Larimar Therapeutics, Inc. (Nasdaq: LRMR), is a clinical-stage biotechnology company focused on developing treatments for complex rare diseases. Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich’s ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion proteins to target additional rare diseases characterized by deficiencies in intracellular bioactive compounds. For more information, please visit: https://larimartx.com.
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