Idebenone – First Treatment for Friedreich's Ataxia

  • Santhera and Takeda Establish Collaboration to Develop
    and Market Idebenone (SNT-MC17) in Neuromuscular

    Idebenone (SNT-MC17) Will Be the First Treatment for
    Friedreich's Ataxia

    LIESTAL, Switzerland and OSAKA, Japan, August 03, 2005
    /PRNewswire/ -- Santhera Pharmaceuticals AG
    ("Santhera") of Switzerland and Takeda Pharmaceutical
    Company Limited ("Takeda") of Japan announced today
    that they will collaborate to develop and
    commercialize Idebenone (SNT-MC17), a small molecule
    drug for the treatment of Friedreich's Ataxia
    ("FRDA"). The clinical development for FRDA is about
    to enter a Phase III clinical study in Europe and will
    enter a Phase III study in the US shortly thereafter.
    The product has orphan drug designation in the US and

    Under the terms of the collaboration, Santhera will
    conduct all clinical development for regulatory
    approval in Europe and in the US. Takeda will support
    the development work and will obtain an exclusive
    license to market Idebenone (SNT-MC17) in the European
    Union and Switzerland. Santhera will in return receive
    an upfront payment of Euro 5 million, development
    milestones and royalties from Takeda. Full financial
    terms were not disclosed. Santhera will supply drug
    product to Takeda for Europe. In the US, Santhera
    plans to market Idebenone (SNT-MC17) on its own.

    "We are pleased to be working with Santhera, a leader
    in neuromuscular drug research and development," said
    Yasuchika Hasegawa, President and Chief Operating
    Officer of Takeda. "Together with Santhera, we look
    forward to the successful development and
    commercialization of a medicine that will, for the
    first time, allow for the treatment of Friedreich's
    Ataxia, a life-threatening neuromuscular disease for
    which currently no pharmaceutical therapies exist."

    "I am pleased to announce Santhera's first development
    and marketing partnership in this area with a company
    of the stature of Takeda," said Klaus Schollmeier,
    Ph.D., Chief Executive Officer of Santhera. "Santhera
    has already built significant R&D expertise in
    neuromuscular diseases. With the help of Takeda's
    European organization, we are now leveraging that
    strength to bring our lead product, a decisive therapy
    breakthrough, to patients. We are very excited to work
    with Takeda, who is the ideal partner for us to bring
    the product to the European market."

    FRDA is a hereditary and disabling neuromuscular
    disorder that severely impacts quality of life and
    reduces patients' life expectancies substantially.
    Although FRDA is primarily a neurological disease,
    cardiac symptoms are well recognized to be a
    life-threatening complication of the disease. The most
    common cardiac manifestation is hypertrophic
    cardiomyopathy which ultimately causes death in the
    majority of FRDA patients.

    It has been shown that Idebenone (SNT-MC17), a small
    molecule oral therapy, can improve mitochondrial
    function and/or reduce oxidative stress in muscle
    cells, heart cells and neurons. By protecting heart
    muscle cells form the oxidative stress that is mainly
    responsible for the deterioration of the heart muscle
    in FRDA, Idebenone (SNT-MC17) could offer the first
    therapy option which can prolong the lives of

    About Takeda

    Takeda, located in Osaka, Japan, is a research-based
    global company with its main focus on pharmaceuticals.
    As the largest pharmaceutical company in Japan and one

    the global leaders of the industry, Takeda is
    committed to striving toward better health for
    individuals and progress in medicine by developing
    superior pharmaceutical products.

    Takeda's four core therapeutic areas in R&D are:
    - Franchise I: Life style-related diseases;
    - Franchise II: Oncology & Urologic diseases;
    - Franchise III: Central nervous system diseases;
    - Franchise IV: Gastroenterology diseases LCM
    For further information, please visit
    About Santhera

    Santhera Pharmaceuticals AG is a Swiss
    biopharmaceutical company founded in 2004 through a
    merger between Germany-based Graffinity
    Pharmaceuticals AG and Swiss-based MyoContract AG.
    Santhera is focused on the discovery, development and
    marketing of small molecule pharmaceutical products
    for the treatment of neuromuscular diseases and has a
    fully integrated platform for the discovery and
    development of drug candidates. The company,
    headquartered in Switzerland, has operations in
    Liestal, Switzerland (near Basel) and in Heidelberg,
    Germany, as well as a business development office in
    Boston, MA, USA. Santhera's investors comprise Merlin
    Biosciences Limited, Oxford Bioscience Partners, NGN
    Capital, 3i Group plc, Carnegie Asset Management, The
    Novartis Venture Fund, Varuma AG, GIMV, Clariden Bank,
    The Dow Chemical Company, Heidelberg Innovation,
    TechnoStart, tbg, the Swiss Foundation for Research on
    Muscle Diseases, and private investors.

    For further information, please visit

    CONTACT: For further information, please contact:
    SantheraPharmaceuticals: Klaus Schollmeier, Ph.D.,
    +41-(0)-61-906-89-52,; Citigate Dewe Rogerson: Mark
    Swallow,+44-(0)-207-638-9571; David Dible,
    +44-(0)7967566919,; Takeda: Seizo Masuda,
    +81-6-6204-2060,; Toshiyuki Ikeuchi, +81-6-6204-2050,

    Quelle: [internaf-news] Idebenone News