Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments for severe neurological diseases and other major unmet medical needs, today announced that the French Agency for Healthcare Product Safety (AFSSAPS) has authorized a Phase II clinical trial with the first drug (a Pleodrug™) generated by the company's Pleotherapy™ technology platform in patients suffering from Charcot-Marie Tooth disease type 1A (CMT1A) - a chronic, severe, invalidating and currently incurable neuromuscular disease which affects 3 million people worldwide.
“This first clinical trial authorization constitutes key proof of the efficiency of our Pleotherapy™ technology. We are continuing to screen for Pleodrugs™ for six other serious diseases (including Alzheimer's disease); our model means that we can hope to make Pleodrugs™ available to patient more rapidly”
Pharnext's first Pleodrug™ is a composed of three generic drugs in a new formulation (a syrup) and which have already received marketing approval for therapeutic indications other than CMT1A. Given that the three compounds are present in the Pleodrug™ at 10- to 100-fold lower doses than in their usual indication, the clinical development programme in humans has not necessitated the performance of a Phase I safety trial and has enabled Pharnext to obtain a Phase II clinical trial authorization directly after just 3 years (rather than the 8 years required on average for most drugs).
The forthcoming Phase II randomized, comparative, placebo-controlled clinical trial will evaluate the safety, efficacy, pharmacodynamics and pharmacokinetics of PXT3003 in patients suffering from CMT1A. The trial will be performed in 5 investigating centres (Timone University Hospital in Marseilles, Salpêtrière Hospital in Paris, Limoges University Hospital, Lyons Sud University Hospital and Pierre Swynghedauw Hospital in Lille) and will be initiated first at Timone University Hospital (coordinating investigator: Shahram Attarian MD) in December.
"This first clinical trial authorization constitutes key proof of the efficiency of our Pleotherapy™ technology. We are continuing to screen for Pleodrugs™ for six other serious diseases (including Alzheimer's disease); our model means that we can hope to make Pleodrugs™ available to patient more rapidly", emphasized Pharnext founder and CEO Professor Daniel Cohen. "We are delighted to be able to start the first clinical trial of our first Pleodrug™ for the treatment of Charcot-Marie-Tooth disease", he concluded.
Pharnext has designed a novel, proprietary platform (Pleotherapy™) based on network pharmacology. The company's discovery engine identifies rational drug combinations (Pleodrugs™) made up of individual Pleocompounds™. This innovative approach could provide a solution to the ongoing and worrying decline in pharmaceutical pipeline productivity.