Pharnext Supports the Fight Against Rare Diseases through Development of Pleotherapy-based Treatments

PARIS, Feb. 16, 2016 /PRNewswire/ -- Pharnext confirms its support and commitment to the fight against rare diseases on the occasion of the Worldwide Rare Disease Day taking place this year on February 29(th). Pharnext is deeply involved in the development of new pleotherapy-based treatments using its network pharmacology approach for patients with rare neurological diseases as well as other neurological conditions with high unmet medical needs.

The 6,000 existing rare diseases affect approximately 580 million people worldwide including 60 million in Europe and North America. Many of these individuals suffer from the lack of safe and efficacious therapies for their serious conditions and from a lack of up-to-date information on their diseases.

Pharnext's Pleodrugs are synergistic low-dose combinations of repositioned drugs with established safety profiles. These innovative therapies under development tackle simultaneously several disease targets potentially offering an excellent efficacy and safety profile. These are essential features for patients suffering from debilitating rare diseases.

Pharnext has two lead therapeutic candidates in development for severe orphan neurological diseases:

-- PXT-3003 for the treatment of Charcot-Marie-Tooth type 1A disease (CMT
1A) in an international pivotal phase 3 trial - To date, no curative or
symptom alleviating medications have been approved for CMT 1A patients
and treatments for this rare neuromuscular disease consist only of
supportive care

-- PXT-864 for the treatment of Amyotrophic Lateral Sclerosis (ALS) in a
completed phase 1 trial - ALS is a very severe, life-threatening
neurodegenerative disease; Currently approved treatments have very
limited impact on life expectancy and do not improve quality of life for

Daniel Cohen, M.D., Ph.D., chairman, chief executive officer and co-founder of Pharnext, said, "We are absolutely committed to developing efficacious and safe new therapies for patients suffering from various rare diseases. From Pharnext's perspective, every patient counts and deserves the best therapeutic solution."

About Pharnext
Pharnext is an advanced clinical stage biopharmaceutical company developing novel therapeutics that simultaneously target multiple key disease pathways for severe orphan and common neurological disorders. The proprietary research and development platform of Pharnext, based on network pharmacology, is applicable to a broad spectrum of diseases and allows the rapid development of "pleodrugs", synergistic combinations of repositioned drugs with established safety profiles. The company's two lead pleodrugs are PXT-3003 for the treatment of orphan disease Charcot Marie Tooth type 1A (Phase 2 clinical trial completed) and PXT-864 for Alzheimer's disease (Phase 2 clinical trial ongoing) and other neurologic indications (including Parkinson's disease and amyotrophic lateral sclerosis).

For further information, visit []


Pharnext contact
Daniel Cohen, M.D., Ph.D.
Chairman and Chief Executive Officer []

Media Contacts
Tony Russo, Ph.D.
Matt Middleman, M.D.
Russo Partners
T: 212-845-4251
T: 212-845-4272 [] []

Florence Portejoie
Caroline Carmagnol
T : +33 1 44 54 36 64 []


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