Pharnext, a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, has enroled the first two patients to the international Phase 3 Extension Study PLEO-CMT-FU of PXT3003 at La Timone University Hospital (Marseille, France).
PXT3003 is Pharnext’s lead Pleodrug 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 company said.
The PXT3003 Phase 3 clinical program consists of two international trials: PLEO-CMT, a pivotal, multi-center, randomized, double blind, fifteen-month, placebo-controlled, adaptive design Phase 3 study that was initiated in December 2015 and PLEO-CMT-FU, a multi-center, double blind, nine-month, Phase 3 follow-up extension study designed to assess the long-term safety and tolerability of PXT3003.
Top-line results from the PLEO-CMT trial are expected in the second quarter of 2018 and in the second quarter of 2019 for PLEO-CMT-FU
Also, Pharnext plans to apply for marketing authorization for PXT3003 in Europe and the United States in the first quarter of 2019. Long term safety data from PLEO-CMT-FU would then be submitted to regulatory authorities during their review of the marketing authorization application. The company said that this should lead to PXT3003 market approval during the second half of 2019.
“The initiation of this second international Phase 3 trial marks an important milestone for the whole PXT3003 clinical development program as it aims at confirming the long-term safety and tolerability profile of PXT3003.” said René Goedkoop, M.D., Chief Medical Officer of Pharnext. “This advancement underscores once more the ability of our clinical team to deliver on time, while bringing us one step closer to providing PXT3003 as a potential treatment option to patients suffering from CMT1A where only supportive care is available today.”