PHARNEXT Has Completed Patient Recruitment for Its Phase II Clinical Trial of PXT3003 (the Company's First Pleodrug™) in Charcot-Marie-Tooth Disease

Published: Nov 22, 2011

Paris, 22 November 2011 - Pharnext SAS, a biopharmaceutical company specializing in the development of innovative treatments based on Pleotherapy™ for severe, unmet medical needs, today announced the on-schedule completion of the recruitment of 80 patients as part of its Phase II study of PXT3003 (the company's first Pleodrug™) in type 1A Charcot-Marie-Tooth disease (CMT1A). Pharnext's combination therapy was developed in just only three years, versus the eight years generally required for novel compounds; the company has been able to move directly from preclinical development to Phase II clinical trials of the Pleodrug™'s efficacy.

"PXT3003 provides a real prospect of improving the status of patients suffering from CMT1A. Recruitment went according to schedule and none of the enrolled patients have dropped out of the study so far, which is very encouraging", stated Dr Shahram Attarian (the CLN-PXT3003-01 study's coordinating investigator at La Timone University Medical Center (Marseilles, France)).

"We are delighted to have reached this key milestone with the first Pleodrug™ generated by our proprietary discovery platform. We are getting ever closer to our objective: to provide patients suffering from Charcot-Marie-Tooth disease (which is currently untreatable) with a safe, effective therapy. We are really looking forward to sharing some interesting results (expected in early 2013) with them", emphasized Professor Daniel Cohen, Pharnext's founder and CEO.

Patient recruitment started in late December 2010 in six investigating centers across France (Lille, Limoges, Lyons, Marseilles, Nantes and Paris) and completed on schedule. The Phase II clinical trial is a double-blind, placebo-controlled, randomized multicenter study. The patients were randomized between the study's 4 arms: a placebo arm and 3 arms with the PXT3003 study treatment administered as 3 oral doses twice a day.

PXT3003 is the first drug candidate to be generated by Pharnext's PleotherapyTM platform. PXT3003 is composed of a combination of off-patent, approved three drugs - each of which is marketed separately for the treatment of another disease and is administered here at much lower doses than in the usual indication. The study's objective is to evaluate the pharmacodynamics, pharmacokinetics, safety and efficacy of PXT3003 in CMT1A patients aged 18 to 65 and with genetic confirmation of a duplication on chromosome 17. The results are likely to be published in the first quarter of 2013. An article in the journal Nature Medicine ("Networking for new drugs", October 2011, vol. 17(10); 1166-1168) described the trial's novelty in its introduction and commented that whereas many of today's most celebrated drugs are designed to hit only one biological target with great precision, medicine's proverbial "magic bullet" might soon give way to a more sophisticated arsenal when using "network pharmacology" (like Pharnext's approach) to efficiently tackle severe medical needs.

Caroline Carmagnol


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