BALA CYNWYD, PA — Larimar Therapeutics, Inc. (Nasdaq: LRMR) recently presented encouraging data from its studies on nomlabofusp at the International Congress for Ataxia Research in London. The data, derived from Phase 1 and Phase 2 studies involving 61 adults with Friedreich’s ataxia (FA), highlight the treatment’s potential benefits.
The studies showed that nomlabofusp altered gene expression, improved lipid profiles, and increased frataxin (FXN) levels in participants. Notably, a daily dose of 50 mg is predicted to achieve FXN levels similar to those in healthy carriers, which could be significant for managing FA, a genetic disorder characterized by FXN deficiency.
Carole Ben-Maimon, MD, President and CEO of Larimar, emphasized the therapy’s promise. “Nomlabofusp has shown dose-dependent increases in FXN levels and beneficial changes in gene expression and lipid profiles,” she said. The company is preparing for further studies, including a pediatric trial, and plans to submit a Biologics License Application in 2025.
Dr. Rusty Clayton, Chief Medical Officer, also noted the therapy’s potential, stating, “Our data supports the use of nomlabofusp across different patient demographics, and we are optimistic about its future development.”
As Larimar continues its research, the focus remains on understanding nomlabofusp’s long-term effects and its role in potentially improving life quality for those with FA. Further updates on the program are expected in December 2024.
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