Pharnext’s lead PLEODRUG © for treatment of CMT1

by | Nov 9, 2017 | 5 comments

The Hereditary Neuropathy Foundation (HNF) is a staunch champion for accurate and early Charcot-Marie-Tooth (CMT) disease diagnosis, recognizing its critical importance for patient care, research and treatments. For those with CMT and suffering with its progressive muscle atrophy and weakness in their legs, feet, arms and hands, there are currently no cures nor approved curative or symptomatic medications.

A change is on the horizon, though!

In December 2016, Pharnext, a French biopharmaceutical company, completed patient enrollment for the international Phase 3 clinical trial of PXT3003, Pharnext’s lead PLEODRUG ©, for treatment of CMT1A (the most common form of CMT). The trial’s primary endpoint is the change in the Overall Neuropathy Limitation Score (ONLS) to determine improvement of patient’s disability after 12 and 15 months of treatment.The trial results are expected in the second half of 2018.

HNF has been partnering with Pharnext to encourage this clinical trial’s development, and we want to thank all who are involved with the study, from the researchers and developers at Pharnext, the clinicians in the field and at our HNF-designated Centers of Excellence, to the patient-participants; all contribute and play a critical role.

HNF has been pleased to contribute to this groundbreaking research by providing U.S. clinical site identification and patient recruitment support for this pivotal Phase 3 trial of PXT3003. HNF resources such as the Global Registry for Inherited Neuropathies (GRIN), our online Inspire Patient Community and our CMT-Connect local patient empowerment programs have been instrumental. We are enthusiastic that our joint effort with Pharnext could result in the first ever therapy for CMT, and we look forward to continuing our collaboration to tackle the more rare forms of CMT in the future.

We can’t rest on our laurels, though!

It will take many, many clinical trials to build the pipeline of potential therapeutics necessary to serve the diverse needs of the entire CMT community. That is why we have created the Clinical Trial Support fund, specifically to raise the money for the clinical trials desperately needed by our patients. Your tax deductible donation will ensure that HNF can continue to support clinical trials for years to come.


Learn more on this topic

Related Blog Posts

Pharnext Announces PXT3003 for the Treatment of Charcot-Marie-Tooth Disease Type 1A has Been Granted Promising Innovative Medicine (PIM) Designation by UK Medicines and Healthcare Products Regulatory Agency

United Kingdom’s Medicine and Healthcare products Regulatory Agency (MHRA) has granted Promising Innovative Medicine (PIM) designation to its lead drug candidate, PXT3003, for the treatment of Charcot-Marie-Tooth Disease Type 1A (CMT1A) in patients 16 years and older.

Pharnext raises € 7.7 million in a private placement

Pharnext SA (FR0011191287 – ALPHA) (the “Company”), a biopharmaceutical company pioneering a new approach to developing innovative drug combinations based on its PLEOTHERAPY artificial intelligence platform harnessing big genomics data and network pharmacology, today announced a capital raise of circa € 7.7 million by way of issuance of 1,799,061 new ordinary shares (the “New Shares”) with one warrant attached each (together with the New Shares, the “ABSA”).

Hot Off the Press – Potential Treatment for CMT1A

Two recent publications from Pharnext describe a novel synergistic combination of 3 drugs (baclofen, naltrexone and sorbitol) and its effect on CMT1A both in the lab and in a phase II clinical trial. These 3 drugs already approved but for unrelated conditions, are combined at new optimal lower doses and under a new formulation. This novel potential therapeutic is called PXT-3003.

Breaking News: First Therapeutic Gene Therapy to Treat an Inherited Neuropathy is Approved for Clinical Trial!

The first disease community to receive a therapeutic gene to the spinal cord for an ultra rare inherited neuropathy is Giant Axonal Neuropathy (GAN). Congratulations to Hannah’s Hope Fund (HHF), a 501(c)3 public charity, which has driven this collaborative research in less than six years. Six million dollars has been raised to date to fund pre-clinical and clinical research on this rare disease.

Join the conversation

Leave a Comment


  1. Elsadig Ahmed

    This disease is found in the three sons — Have you discovered a cure for it

    • Estela Lugo

      Hello Elsadig,

      We are working tirelessly towards research and clinical trials towards effective treatments. At this time there is no cure for CMT.

  2. Sherry Weatherly

    It i s wonderful to hear there may be a treatment that works! I have CMT1A, my sister has it, my mother had it, all 3 of my children show signs of it already. I’d be thrilled if there was something we could to to stop it from getting worse, or help us to improve! Do keep us informed!

  3. Ronald Riskevich

    I have Charcot-Marie-Tooth, When will Clinical Trials be concluded? What is the time frame for FDA approval?


Submit a Comment

Your email address will not be published. Required fields are marked *


Join for notifications on events, campaigns, & news