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’) - Seite 2

Adrian Hepner, MD, PhD, Chief Medical Officer of Pharnext, said: “Although these new data were generated from an open-label study, the findings are consistent with the safety and efficacy results of PXT3003 observed in prior clinical studies in CMT1A. In addition, the fact we have just initiated the PREMIER trial in a similar patient population, using the same High Dose of PXT3003 and measuring the same efficacy endpoint ONLS, reinforces our confidence in the potential positive outcome of our ongoing pivotal Phase III study”.

Florian P. Thomas, MD, PhD, Founding Chair & Professor, Department of Neurology, Hackensack University Medical Center & Hackensack Meridian School of Medicine (NJ, USA) and U.S. lead investigator of the PLEO-CMT trial, said: “These new results from the interim analysis of the ongoing open-label Phase III extension study show very promising safety and efficacy data of PXT3003 in CMT1A after more than four years of treatment. It reinforces our hope that PXT3003 could be the first treatment approved for patients suffering from this debilitating disease”.

Results of First Double-Blind Phase III (PLEO-CMT) & Open-Label Extension (PLEO-CMT-FU)
Studies of PXT3003 with an ONLS Data Readout at 54 Months of Total Trial Time

^aCohort of CMT1A patients treated with PXT3003 High Dose during PLEO-CMT and ongoing PLEO-CMT-FU trials
^bCohort of CMT1A patients treated with PXT3003 Low Dose during PLEO-CMT + PLEO-CMT-FU Period 1, and then switched to PXT3003 High Dose during PLEO-CMT-FU period 2
^cCohort of CMT1A patients treated with placebo during PLEO-CMT, PXT3003 Low Dose or High Dose during PLEO-CMT-FU Period 1 and PXT3003 High Dose during PLEO-CMT-FU Period 2
Please refer to a graphic illustration of first double-blind Phase III (PLEO-CMT) and open-label extension (PLEO-CMT-FU) studies design in the “About the PLEO-CMT-FU Trial” section below.

Conference Call Details

Pharnext will host a live conference call and webcast at 2:00 p.m. CET / 8:00 a.m. ET tomorrow Thursday April 29^th, 2021 to discuss the data. The conference call may be accessed by dialing [phone] (France), [phone] (USA), or [phone] (International) and using conference ID 6197704. The live webcast and accompanying slides can be accessed via the Pharnext’s website at https://pharnext.com/en in the ‘Events’ section or by clicking here. An archived webcast will then be available on the Pharnext’s website approximately 4 hours after the conference call.