Pharnext updates on PXT3003 for patients with CMT1A

French neurodegenerative diseases specialist Pharnext (EPA: ALPHA) has announced 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).

Market reaction was negative, as Pharnext’s shares closed down 3.3% at 3.24 euros following the announcement.