Debiopharm International SA and Solid Biosciences, LLC, announce a collaboration to test the use of Alisporivir (Debio 025) in muscular dystrophy.
Lausanne, Switzerland and Cambridge, USA – September 8, 2015 – Debiopharm International SA (Debiopharm), part of Debiopharm Group™ and Solid Biosciences, LLC, are proud to announce the start of preclinical studies of Alisporivir, in the rare disease Duchenne Muscular Dystrophy (DMD). The goal of the collaboration is to reinforce existing preclinical trial data in additional DMD animal models. This preclinical work, fully funded by the Lausanne-based company, will therefore lead to Debiopharm starting clinical development of this promising new DMD therapy 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 the death of mitochondrial-dependent muscle cells and has shown promising activity in preclinical models of DMD but also in Limb-Sheath and Ullrich-Bethlem myopia. The aim of this collaboration is to confirm the efficacy of the compound in a new model of DMD that more closely resembles human pathology and to enable the initiation of clinical development in this indication. “We are delighted to begin the development of muscular dystrophy,” said Thierry Mauvernay, Delegate of the Board of Debiopharm Group. He added: “We are very pleased to be working with Solid Biosciences on this programme, combining our deep knowledge of Alisporivir with their great experience and network in DMD and their focus on finding solutions for these young patients suffering from this dreadful disease. . “We at Solid Biosciences are proud to partner with Debiopharm,” said Ilan Ganot, founder and CEO of Solid Biosciences. “Alisporivir has exciting potential for efficacy in DMD and we look forward to leveraging our experience in this condition to help advance Alisporivir in DMD patients. I am especially grateful to Bob McDonald and Ed Mascioli whose personal and professional commitment to DMD patients is reflected in this report. About Duchenne Muscular Dystrophy (DMD): Duchenne muscular dystrophy (DMD) is a severe form of X-linked muscular dystrophy that affects approximately 1 in 3500 males. DMD is caused by the absence of dystrophin, a structural protein critical to protecting skeletal and cardiac muscle from contraction-induced injury. Characterized by progressive and pervasive muscle wasting, patients with DMD lose the ability to walk in early to mid-adolescence and progress to complete loss of upper body function. Patients with DMD experience progressive cardiopulmonary complications, which are the primary cause of mortality, typically in their early to mid-20s. To find out more about DMD visit: About Debiopharm International SA: Debiopharm Group™ is a Switzerland-based global biopharmaceutical group of four companies active in drug development, proprietary drug manufacturing, diagnostics and investment management . Debiopharm International SA is focused on the development of prescription medicines that target unmet medical needs. The company licenses and develops promising drug candidates. The products are marketed by off-license pharmaceutical partners to enable access to the largest number of patients worldwide. For more information about the Debiopharm Group™, please visit: We are on Twitter. Follow us @DebiopharmNews at About Solid Biosciences, LLC: Solid Biosciences is a Cambridge, MA-based biotech company developing therapeutics for Duchenne muscular dystrophy. Solid is currently developing small molecules, gene therapies and medical devices, all targeting the many facets of DMD. For more information about Solid Biosciences, please visit: