Santhera awarded CHF 1M to develop muscular dystrophy drug

Grant

Santhera Pharmaceuticals, of Liestal, Switzerland, a specialist in treatments for rare neuromuscular diseases, has announced that the French patient advocacy organisation Association Française contre les Myopathies (AFM) has awarded a grant of CHF 1 million (€660,000) for the development of SNT-317 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 pre-clinical studies needed before the compound can enter clinical development in pediatric patients.

Originally developed by Novartis, SNT-317 was in-licenced by Santhera in 2007. In preclinical studies, the compound reduced 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 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 for CMD. Professional patient advocacy organisations such as AFM are tremendously important in advancing translational research into the clinic,” 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 is a promising first therapy for Congenital Muscular Dystrophy. Through this grant, we support Santhera in driving the development of this important compound,” says Serge Braun, scientific director of AFM.