For the development of SNT-317 in congenital muscular dystrophy
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The French patient advocacy organisation, Association Française contre les Myopathies (AFM) has awarded a grant of CHF1m for the development of Santhera Pharmaceuticals’ (Santhera) SNT-317 (INN: omigapil) in congenital muscular dystrophy (CMD).
The funding of two non-clinical studies by AFM is expected to enable the clinical development of SNT-317/ omigapil in pediatric CMD patients. AFM will be eligible to a success-based repayment of the grant after product launch.
In preclinical studies, the compound inhibited GAPDH-Siah1 mediated apoptosis in muscle tissue and reduced body weight loss and skeletal deformation while increasing locomotive activity and protecting from early mortality. Santhera has been granted orphan drug designations by the FDA and European Medicines Agency for CMD subtypes caused by collagen-VI and laminin-alpha-2 deficiency.
Thomas Meier, chief scientific officer of Santhera, said: “Receiving this financial support from AFM reinforces our commitment to develop SNT-317/omigapil for CMD. Professional patient advocacy organisation such as AFM are tremendously important in advancing translational research into the clinic, particularly for diseases such as CMD for which there is no pharmacological therapy available or in advanced clinical development.”
Serge Braun, scientific director of AFM, said: “AFM’s main objective is to pave the way for new treatments that ultimately bring help and hope for patients suffering from rare neuromuscular diseases. SNT-317/omigapil is a first therapy for Congenital Muscular Dystrophy. Through this grant, we support Santhera in driving the development of this important compound”
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