Pharnext enrolls patients for Charcot-Marie-Tooth disease study in Europe, US, and Canada.


A French biopharmaceutical company Pharnext set to produce treatments to tackle neuropathy  diseases, has completed patient enrollment for its international pivotal Phase 3 PLEO-CMT trial of PXT3003, its lead Pleodrug, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A).

PLEO-CMT is a Phase 3 study started in December 2015 and has enrolled 323 patients with mild to moderate CMT1A in 30 sites with the primary endpoint to determine improvement of patients’ disability after 12 and 15 months of treatment with PXT3003.

Results of the should be available in the second half of 2018.

M.D., Chief Medical Officer of Pharnext, René Goedkoop, said:“Completing enrollment of our pivotal Phase 3 trial of PXT3003 is a significant milestone and highlights the strength of our clinical operations and management teams, as well as the support of our numerous clinical trial sites, our dedicated clinical investigators and the patient community.”

Daniel Cohen, M.D., Ph.D., Co-Founder and Chief Executive Officer of Pharnext added: “This clinical trial is highly significant for patients suffering from CMT1A where only supportive care is available today. Our Pleodrug represents a great hope for patients suffering from this debilitating disease.”

Michael W. Sereda, M.D., from the Max Planck Institute, Göttingen said that PXT3003 has shown much promise in previous preclinical and clinical studies, and the data Pharnext has published to date positions PXT3003 as a prominent and safe therapeutic candidate for patients with CMT1A.

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