CMT 1A: Pharnext Pleodrug PXT3003 Phase 2 Trials BEGIN

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PHARNEXT Receives Clinical Trial Authorization for a Phase II Study with the

First Drug Generated by Its PleotherapyTM Technology

http://www.benzinga.com/press-releases/10/12/b665794/pharnext-receives-clinical-\

trial-authorization-for-a-phase-ii-study

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.

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 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.

About Pharnext

Pharnext is a biopharmaceutical company focused on discovering, developing and

licensing new pharmaceutical treatments based on intelligent Pleocompounds™ that

form the basis of Pleodrugs™. Pharnext was founded in Paris, France in April,

2007, by Professor Cohen, MD, PhD, his research group (composed of

pioneers in genome science and technology) and Philippe Pouletty, MD (General

Partner at Truffle Capital). Thanks to Truffle Capital's founding investment,

strong support from OSEO, a grant from the French Muscular Dystrophy Association

(AFM) and the benefits of France's research tax credit scheme, Pharnext has

developed a novel, proprietary platform (PleotherapyTM) based on network

pharmacology and which may provide a solution for the current decline in

pharmaceutical pipeline productivity. Pharnext is advised by La Compagnie

Financière Edmond de Rothschild.