Liestal, Switzerland, November 3, 2009 – Santhera Pharmaceuticals (SIX: SANN), a Swiss specialty pharmaceutical company focused on orphan neuromuscular diseases, announced today that the French patient advocacy organization Association Française contre les Myopathies (AFM) awarded a grant of CHF 1 million for the development of Santhera’s SNT-317 (INN: omigapil) in Congenital Muscular Dystrophy (CMD). This severe, genetically determined neuromuscular condition frequently affects infants or young children with life-threatening progressive muscle weakness. AFM’s grant will finance the remaining non-clini-cal studies needed before the compound can enter clinical development in pediatric patients. A recent publication in Journal of Pharmacology and Experimental Therapeutics highlights the beneficial effect of SNT-317/omigpail in a disease-relevant model .
Originally developed by Novartis, SNT-317/omigapil was inlicenced by Santhera in 2007 for devel-opment in CMD. 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 US Food and Drug Administration and European Medicines Agency for CMD subtypes caused by collagen-VI and laminin-alpha-2 deficiency.
The funding of two non-clinical studies by AFM will 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.
“Receiving this financial support from AFM reinforces our commitment to develop SNT-317/omigapil for CMD. Professional patient advocacy organization 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”, said Thomas Meier, Chief Scientific Officer of Santhera.
“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 promising first therapy for Congenital Muscular Dystrophy. Through this grant, we support Santhera in driving the devel-opment of this important compound”, says Serge Braun, scientific director of AFM.