New Data from the Open-Label PLEO-CMT-FU Trial Shows Sustained Benefit with PXT3003 in Patients with Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time

New Data from the Open-Label PLEO-CMT-FU Trial Shows Sustained Benefit with PXT3003 in Patients with Charcot-Marie-Tooth Disease Type 1A After 5 Years of Total Trial Time

• New data from the ongoing Open-Label Phase III Extension Study of PXT3003, the PLEO-CMT-FU trial, suggest good safety profile and continuous treatment effect of PXT3003 measured on the Overall Neuropathy Limitation Scale after 5 years of total trial time
  
  
• 126 patients with mild-to-moderate Charcot-Marie-Tooth Disease Type 1A are still on treatment with PXT3003 High Dose in the PLEO-CMT-FU trial
  
  

PARIS, France, May 16^th, 2022, 8:30 am CET – Pharnext SA (FR0011191287 – ALPHA) (the “Company”), an advanced late-clinical stage biopharmaceutical company developing novel therapeutics for neurodegenerative diseases with high unmet medical need, today announces 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 today are derived from a data extraction performed on April 25^th, 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.

Key features of the data analysis are as follows:

• PXT3003 continues to show a good tolerability and safety profile over the course of the first Phase III program (double-blind + ongoing open-label).
• PXT3003 continues to show encouraging efficacy results as measured on the Overall Neuropathy Limitations Scale (‘ONLS’) which evaluates the patient’s functional motor disability.
  • The best efficacy signal was observed in the cohort of patients treated with PXT3003 HD during 5 years of total trial time (double blind + ongoing open-label).
  • Patients treated with placebo declined on ONLS during the double-blind phase but then improved when switched to PXT3003 in the ongoing open-label phase.

Please refer to the illustration of the ONLS data below for more details.