Charcot-Marie-Tooth Association Enters Collaboration with Affectis to Advance Therapies for CMT1A

Top Quote The Charcot-Marie-Tooth Association (CMTA) announced today that it has entered into a collaboration with Affectis Pharmaceuticals AG to evaluate the efficacy of advanced Affectis compounds in neurological and behavioral models of CMT1A. End Quote
  • (1888PressRelease) April 22, 2015 - Glenolden, Pennsylvania - Affectis is a therapy development company and since 2013 a fully owned subsidiary of the Lead Discovery Center GmbH (LDC), a spin-off of Max Planck Innovation GmbH.

    The collaboration's aim is to demonstrate the potential of P2X7 antagonists that have high potency for the human form of P2X7 and are orally bioavailability in treating CMTA1. Use of such antagonists may impede the development of motor and sensory control defects associated with progression of the disease.

    Pre-clinical studies previously demonstrated a likely role for P2X7 over-activity in the pathogenesis of CMT1A (Nobbio et al. (2009) J. Biol.Chem. 284, 23146). An altered calcium homeostasis was observed in Schwann cells from rats that exhibit a CMT1A pathology due to the expression of extra copies of the pmp22 gene; this is hypothesized to lead to the disruption of myelination associated with the disease. The authors further showed that the changes in intracellular calcium coincided with overexpression of the P2X7 ligand-gated ion channel, and that its inhibition leads to myelin repair.

    Patrick Livney, CEO of the CMTA notes: "The association has assembled the scientific and clinical key opinion leaders in CMT disorders and the research tools necessary to validate therapeutic opportunities for their clinical potential. We have set out to engage drug makers to work together with the CMTA to advance new therapeutic approaches to our patients, and our STAR network combines this world class research expertise with an operational capability has been highly enabling to collaborative alliances formed for this purpose. Currently, there are no therapies for the different CMT disorders to halt either the onset or progression of the disease. This Affectis collaboration represents an exciting new opportunity for the CMTA to both de-risk and accelerate development of a novel drug class for the treatment of CMT1A, the most prevalent of the genetic neuropathies."

    Affectis CEO Michael Hamacher said of the collaboration: "Our P2X7 lead compounds have excellent pharmacological properties and repeatedly showed efficacy in various animal models. With initiation of the CMT1A studies we see the unique chance to evaluate both the role of P2X7 as well as the potency of the Affectis' compounds in the Charcot-Marie-Tooth 1A disorder, a demyelinating neuropathy with unmet medical need. We are very much looking forward to the collaboration with the excellent team of experts from the CMTA to jointly progress the P2X7 leads into an effective therapy for Charcot-Marie-Tooth 1A".

    About the Charcot-Marie-Tooth Association
    The Charcot-Marie-Tooth Association (CMTA) is a registered 501c3 dedicated to serving an international patient community that suffers from rare and disabling neuropathies of genetic origin. The CMTA directly engages its STAR scientific and clinical research network in the identification, validation and clinical development of therapies for the different Charcot-Marie-Tooth disorders.

    Patrick Livney, CEO
    Charcot-Marie-Tooth Association
    T. +1-312.750.9800
    Email: pal ( @ ) cmtausa dot org
    Further information at:

    About Affectis and the LDC
    Affectis Pharmaceuticals AG is a pharmaceuticals company that develops novel drugs for the treatment of neurodegenerative and neuroinflammatory diseases. Since January 2014 Affectis has relocated to Dortmund, Germany, and is now fully owned by and closely collaborates with the Lead Discovery Center GmbH (LDC) to further advance its core asset AFC-5128.

    Dr. Michael Hamacher, CEO
    Affectis Pharmaceuticals AG
    T. +49.231.97 42 70 00
    Email: hamacher ( @ ) affectis dot de
    Further information at:

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