Data from First Phase III Clinical Study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A, the PLEO-CMT Trial, Published in the Orphanet Journal of Rare Diseases
18 October 2021
PHARNEXT SA reported on a first phase clinical study of PXT3003 in Charcot-Marie-Tooth disease Type 1A. PXT3003 is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day. Based on their conclusion that the high-dose PXT3003 group demonstrated a statistically significant improvement in the primary endpoint, the Overall Neuropathy Limitations Scale ('ONLS'), compared to placebo, and a good safety profile, the authors state high-dose PXT3003 is considered a promising treatment option for patients with Charcot-Marie-Tooth disease Type 1A.