Pharnext, a biopharmaceutical company, has announced new positive results from the ongoing open-label extension study of their drug PXT3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A), called the PLEO-CMT-FU trial. The trial has been conducted for a total of 6 years, and the data is consistent with a long-term treatment benefit of PXT3003 in patients with mild-to-moderate CMT1A. The results indicate that PXT3003 has a good safety profile and shows a long-term treatment effect on the Overall Neuropathy Limitation Scale (ONLS), which measures functional motor disability. A group of 117 patients with mild-to-moderate CMT1A are still receiving treatment with the high dose of PXT3003 in the trial.
Pharnext Opens First U.S. Study Site For Its PLEO-CMT International Pivotal Phase 3 Trial In Charcot-Marie-Tooth Disease Type 1A
French pharmaceutical company Pharnext announced the opening of the first U.S. trial site for its PLEO-CMT pivotal Phase 3 clinical trial of its lead pleodrug PXT-3003 in Charcot-Marie-Tooth Disease Type 1A (CMT1A).