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Pharnext logo

France based
$58.830 M

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).

Molecule combination Genomics Network pharmacology


AI Companies (Drug Discovery)  

R&D Platform

proprietary technology platform, systemizes the identification and development of new drug combinations for any disease that currently lack effective treatments. These new treatment candidates – called PLEODRUG™ – are expected to feature high levels of efficacy and safety as they target several disease pathways simultaneously and are formulated with optimal doses of their components.


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Clinical  |  Visit website

PXT3003 is a novel fixed-dose synergistic combination of baclofen, naltrexone and sorbitol formulated as an oral solution given twice a day. The three individual components of PXT3003 were selected to downregulate the overexpression of PMP22 protein, leading to improvement of neuronal signaling in dysfunctional peripheral nerves that are an essential part of the pathophysiology of this disease. PXT3003 could also have a positive effect on other cellular types of the motor unit such as the axon (direct protection), neuromuscular junctions or muscle cells.

Clinical  |  Visit website

PXT864 is a novel fixed-dose synergistic combination of baclofen and acamprosate formulated as a pill given twice a day. PXT864’s assumed main mechanism of action in neurodegenerative diseases is the restoration of balance between excitatory (glutamate activity) and inhibitory (GABA activity) pathways, disrupted by toxic factors such as Aβ oligomeric peptides in Alzheimer’s disease.
PXT864 showed positive safety and tolerability results in a Phase 1 clinical trial that enrolled 24 healthy volunteers.




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