Pharnext: Successful intermediate analyses for PLEO-CMT Phase III clinical trial in Charcot-Marie-Tooth disease Type 1A.
Pharnext SA, a biopharmaceutical company working on a new approach for the development of drugs based on the combination and repositioning of known therapies, announced successful intermediate analyses for its Phase III clinical trial, PLEO-CMT, in Charcot-Marie-Tooth Disease Type 1A (CMT1A).
The ongoing study is part of a new company paradigm called PLEOTHERAPY, which centers on a blending of drugs previously established and targeted for the treatment of certain disorders that, when combined in optimal lower doses, can be repositioned and formulated into new, effective therapies for fighting other maladies, such as Alzheimer’s disease.
PLEO-CMT is a pivotal, multi-center, randomized, 15 months, double blind, placebo-controlled Phase III study that was initiated in December 2015 and has enrolled 323 patients with mild-to-moderate CMT1A in 30 sites across Europe, the U.S., and Canada.
With completion of the study expected by the end of next year, two intermediate analyses — a blind variability analysis followed by a futility analysis — were carried out as planned.
See related article on PLEOTHERAPY: “Two Drugs Can Be Better Than One,” an in-depth report featuring Rodolphe Hajj, Ph.D., chief pharmacology officer at Pharnext.
According to Professor Philippe Lehert, member of the independent Data Safety Monitoring Board, the variability of tests between patients is within predefined limits. In addition, a futility analysis concluded that PLEO-CMT is sufficiently powered to detect an effect of Pharnext’s lead PLEODRUG —PXT3003 — on the primary efficacy endpoint.
The Data Safety Monitoring Board is an independent body of experts drawn from the fields of clinical medicine, biostatistics, and study methodology, chartered to provide recommendations to Pharnext upon regular pre-specified review of the accumulated data during the conduct of the clinical trial.
The analyses follow two favorable intermediate safety analyses delivered by the monitoring board in November 2016 and September 2017 and indicate that PLEO-CMT can continue according to the original plan without having to increase the trial size, according to the company.
Final results of the study are expected in the second half of 2018.
(Source: Pharnext SA; Pictured: Pharnext headquarters in the southwestern suburban area of Paris, France, courtesy Pharnext)
Filed Under: Drug Discovery