Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)

Interim Analysis from the Ongoing Open-Label Phase III Extension Study Shows Sustained Benefits of PXT3003 for Patients with Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’)

New results suggest good safety profile and sustained efficacy of PXT3003 as measured with the Overall Neuropathy Limitation Scale (‘ONLS’), after 4.5 years of total trial time.

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PARIS, France, April 28, 2021, 6:00 p.m. CET – 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 platform, today announces new results from an interim analysis of an ongoing open-label follow-up extension study (‘PLEO-CMT-FU trial’) following the first double-blind, placebo-controlled Phase III study (‘PLEO-CMT trial’) of PXT3003 for the treatment of Charcot-Marie-Tooth Disease Type 1A (‘CMT1A’). There is currently no approved drug treatment for CMT1A.

In January 2020, the company reported interim results suggesting sustained safety and efficacy of PXT3003 in patients with mild-to-moderate CMT1A after 24 months of total trial time (PLEO-CMT and PLEO-CMT-FU Period 1 trials). The new results announced today continue to show sustained treatment benefits for CMT1A patients treated with PXT3003 at High Dose (‘HD’) in the PLEO-CMT-FU Period 2 trial with a data readout at 54 months of total trial time (double-blind + open-label). Highlights from an analysis of available data include:

• PXT3003 was safe and well tolerated. Data are consistent with observed safety profile in prior clinical trials.
• Data on the Overall Neuropathy Limitations Scale (‘ONLS’), which measures patients’ functional motor disability, are as follows (please refer to the diagram below for graphical illustration):
  • During PLEO-CMT (double-blind Phase III study), patients treated with placebo on average declined on ONLS while patients treated with PXT3003 on average improved. The best efficacy signal was observed in the cohort of patients treated with PXT3003 HD.
  • During PLEO-CMT-FU (open-label Phase III Extension study), on average patients improved on ONLS across all patient cohorts.
    • 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.
    • Patients treated with PXT3003 during the double-blind phase continue to improve when pursuing their treatment with PXT3003 in the ongoing open-label phase.
• New results with available ONLS data after 54 months of total trial time suggest a better efficacy signal with PXT3003 HD in this patient population.