Date: 2015-09-08
Type of information: Collaboration agreement
Compound: alisporivir (DEB025)
Company: Debiopharm (Switzerland) Solid Biosciences (USA - MA)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Type agreement: collaboration
Action mechanism: cyclophilin inhibitor. Alisporivir is a non-immunosuppressive cyclophilin inhibitor, with multiple potential indications and has already been tested in more than 1500 patients during clinical development for hepatitis C, demonstrating an acceptable safety profile. Alisporivir is able to prevent mitochondrial-dependent muscular cell death and has shown promising activity in preclinical models of DMD but also Limb-Girdle and Ullrich-Bethlem myopathy.
Disease: Duchenne muscular dystrophy (DMD)
Details: * On September 8, 2015, Debiopharm and Solid Biosciences announced the start of preclinical studies of alisporivir, in the rare disease Duchenne Muscular Dystrophy (DMD). The objective of the collaboration is to reinforce existing preclinical proof of concept data in additional DMD animal models. This preclinical work, fully funded by the Lausanne-based company, will then lead to Debiopharm initiating the clinical development of this new and promising therapeutic for DMD at its own expense in the near future. Alisporivir is a non-immunosuppressive cyclophilin inhibitor, with multiple potential indications and has already been tested in more than 1500 patients during clinical development for hepatitis C, demonstrating an acceptable safety profile. Alisporivir is able to prevent mitochondrial-dependent muscular cell death and has shown promising activity in preclinical models of DMD but also Limb-Girdle and Ullrich-Bethlem myopathy. The aim of this collaboration is to confirm the efficacy of the compound in a new model of DMD that more closely resembles the human pathology and to enable the start of clinical development in this indication.
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