Pharnext Initiates Phase 3 Extension Study PLEO-CMT-FU of PXT3003

Pharnext, a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, has announced that the first two patients have entered the international phase 3 Extension Study PLEO-CMT-FU of PXT3003 at La Timone University Hospital (Marseille, France). PXT3003 is Pharnext's lead PLEODRUG(TM) for the treatment of patients with mild-to-moderate Charcot-Marie-Tooth Disease Type 1A (CMT1A), a rare and debilitating inherited peripheral neuropathy for which there are no satisfactory approved treatments available.

The PXT3003 Phase 3 clinical program consists of two international trials PLEO-CMT and PLEO-CMT-FU. PLEO-CMT is a pivotal, multi-center, randomized, double blind, fifteen-month, placebocontrolled, adaptive design phase 3 study that was initiated in December 2015. In November 2016, the independent Data Safety Monitoring Board (DSMB) recommended continuing the PLEO-CMT study as planned after its review of safety data from 100 patients who completed at least three months of study treatment. Patient enrollment was completed in December 2016 with 323 patients in 30 sites across Europe, the United States and Canada. Top-line results from the PLEO-CMT phase 3 trial are expected in the second quarter of 2018. These data, if positive, will form the basis of the submission package for market approval to regulatory authorities in Europe and the United States.