Pharnext was founded in 2007 by Prof. Daniel Cohen, a pioneer in genomics, co-founder of Millennium Pharmaceuticals, and his main collaborators. The company develops new therapeutics for severe orphan and common neurological diseases.
The R&D approach pioneered and 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 are then developed at new optimal lower doses and under new formulations.
These novel therapeutics – called Pleodrugs – present significant potential medical advantages: strong efficacy and safety profile well suited for disease prevention. Intellectual property is also strong and robust.
The proof-of concept of the Pleotherapy approach was obtained with the investigational Pleodrug, PXT3003, in Charcot-Marie-Tooth disease type 1A (CMT1A) through positive Phase 2 results. A pivotal, multicentric, international Phase 3 clinical trial started in CMT1A in December 2015.
PXT864, the second investigational Pleodrug, is currently in Phase 2a clinical trial for Alzheimer’s disease with encouraging intermediate preliminary data. It is also planned to develop the same agent in other orphan and common neurodegenerative diseases such as Parkinson’s disease and amyotrophic lateral sclerosis (ALS).
Pharnext is a privately-held company which has raised a total of €65M since inception.