Pharnext SA announced new results from the ongoing open-label follow-up extension study of PXT3003 in Charcot-Marie-Tooth Disease Type 1A (‘CMT1A'), the PLEO-CMT-FU trial, which followed the first double-blind, placebo controlled Phase III study, the PLEO-CMT trial. In January 2020 and April 2021, Pharnext reported results, based on previous data extraction from the PLEO-CMT and PLEO-CMT-FU trials, suggesting sustained safety and efficacy of PXT3003 in patients with mild-to-moderate CMT1A. The new results announced are derived from a data extraction performed on April 25th, 2022, which shows the continuous treatment effect for CMT1A patients treated with PXT3003 High Dose (‘HD') in the PLEO-CMT-FU Period 2 trial with a data readout at 60 months of total trial time (15 months of PLEO-CMT trial + 9 months of PLEO-CMT-FU trial period 1 + 36 months of PLEO-CMT-FU trial period 2).

Please refer to an illustration of the first PXT3003 Phase III program design in the “About the PLEO-CMT-FU Trial” section below for more details.