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Clinical Trials


AI Companies (Drug Discovery)

Pharnext is an advanced clinical-stage biopharmaceutical company founded in April 2007 by renowned scientists and entrepreneurs including Professor Daniel Cohen and collaborators, pioneers in modern genomics. The company develops new therapeutics for neurodegenerative diseases - orphan and common – where there are currently no cures and existing therapies available.

Pharnext is the pioneer of a new paradigm for the discovery of medicinal drugs: PLEOTHERAPY™. The R&D approach protected by Pharnext systemizes new uses of approved drugs. It is based on network pharmacology utilizing complex and extensive genomic data to identify the thousands of molecules possibly involved in a disease. From this disease molecular network, Pharnext deduces synergistic combinations of drugs already approved but for unrelated indications. These novel therapeutics called PLEODRUG™ are then developed at new optimal lower doses and under new formulations. They offer several key advantages: efficacy, safety, and intellectual property including several composition of matter patents already granted.

The proof-of concept of the PLEOTHERAPY approach was obtained with PLEODRUG PXT3003 in Charcot-Marie-Tooth disease type 1A (CMT1A) through positive Phase 2 results. A Phase 3 clinical trial is currently ongoing in 323 patients with CMT1A. Top line results are expected in the second half of 2018.

PXT864, the second PLEODRUG, generated positive Phase 2 data in Alzheimer’s disease. We also plan to develop the same agent in other orphan and common neurodegenerative diseases such as Parkinson’s disease and amyotrophic lateral sclerosis (ALS).

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