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

Oct 18, 2021

PARIS, FRANCE / ACCESSWIRE / October 18, 2021 / Pharnext SA (FR0011191287 - ALPHA) (the 'Company'), an advanced late-stage clinical biopharmaceutical company pioneering new approaches to developing innovative drug combinations based on big genomics data and artificial intelligence using its PLEOTHERAPY(TM) platform, today announces the publication of the data from the Company's first phase III placebo-controlled clinical study of PXT3003 in Charcot-Marie-Tooth disease Type 1A ('CMT1A'), the PLEO-CMT trial, in the Orphanet Journal of Rare Diseases ('OJRD'). 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 CMT1A.

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