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.


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  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?


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