Thomson Reuters—one of the world’s leading source of information—reports the approval to initiate a First-In-Man clinical study for CMT1A.

The study will be conducted by Addex Therapeutics, a company based in Geneva, Switzerland, famous for its research in the field of small molecule drugs. Past products of the same company are being used to treat Parkinson’s disease.

The company website confirms the news, and names this new experimental drug ADX71441. Although the company headquarter is based in Switzerland, the approval to start a First-In-Man clinical trial has been issued by the regulatory authorities in the Netherlands.

In fact, the company plans to initiate the testing at the Centre for Human Drug Research in Leiden, The Netherlands.

A drug designed to target the orphan disease CMT1A

The molecule drug is designed to be exquisitely selective and target (some of) the causes of Charcot-Marie-Tooth Type 1A and eventually other similar neuropathy. It expects to deliver Phase 1 top-line safety data by the end of 2013, and move to a more practical Phase 2a testing in 2014.

The doctor in charge of the project is the Italian-born Sonia Poli, Ph.D., Head of Non-Clinical Development at Addex. Dr. Poli has obtained a doctorate in Industrial Chemistry at the University of Milan, Italy, and completed a post-doctoral fellowship at the CNRS, in Paris, France.

Besides her work with Charcot-Marie-Tooth, she has developed an expertise in many central nervous system diseases (CNS), including Alzheimer’s disease, Parkinson’s disease, bipolar disorders and anxiety.

This is a very exciting news for all the staff at CMT journal. However, it’s important to stress that it’s the beginning of a trial, not the release of a cure.

Research takes years, and Charcot-Marie-Tooth is not an easy subject. Nevertheless, until a few years ago, the possibility of a First-In-Man clinical study in CMT has been strongly excluded, and the top research was conducted on mice.

Starting such a trial is a milestone, and we are optimistic. Until an effective drug is released on the market, we (or our beloved) should manage CMT in the old way: “smarter than harder”. Be active as much as you can, and always cheer your spirit. It’s not easy, but optimism is the best drug we have yet.

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[Image: our photo of San Thomas Hospital, London, UK]


  1. Good Night,

    I read a news that a laboratory frances, Pharnext, is developing a drug to CMT1A. They have already completed all phases of research, including the use in humans. Do you know anything about this?
    Best regards, Naira

    • This is not the first drug trial for CMT1A. Pharnext conducted a phase 2 trial and presented some promising data at the CMT meeting in Antwerp. There was a vitamin C trial also for CMT1A, but unfortunately that trial had no impact. Its important that anyone with CMT join the Global Registry for Inherited Neuropathies (GRIN) – to advance research as well as to be notified when Addex, Pharnext and other pharma companies are conducting clinical trials.

  2. Hello!!

    I am a woman how has CMT disease and I would like to know when will this treatment be in the markets.

    Thank you very much.



    • hi Hessu,

      I have CMT-A1 and would like to test the formulation of PXT3003 as you described. Could you please list the dosages and how you add the Sorbitol to the other 2 drugs?

      thank you so much,


  3. When will it be available for treatment in the United States I’m a lady of 43yrs old with CMT1A and my daughter has it also and she’s 15 in a half so at my age will we be treatable ??

    Thank you!


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