Santhera Repositions Omigapil in Congenital Muscular Dystrophy

18-Jul-2014 - Switzerland

Santhera Pharmaceuticals announced the initiation of a clinical program with omigapil, a drug candidate in-licensed from Novartis and repositioned for therapeutic use in Congenital Muscular Dystrophy (CMD). The clinical development program will be initiated with a Phase I study in pediatric CMD patients, to be conducted at the National Institute of Neurological Disorders and Stroke (NINDS), a component of the US National Institutes of Health (NIH). The program is supported financially in the amount of CHF 1.3 million by EndoStem, an EU 7th Framework Programme, and two patient organizations, the US-based Cure CMD and the Swiss Foundation for Research on Muscle Diseases. Patient enrolment is expected to start in late 2014.

Following Santhera's successful repositioning of idebenone for the treatment of Leber's Hereditary Optic Neuropathy (LHON) and for Duchenne Muscular Dystrophy (DMD), omigapil, as an anti-apoptotic which previously reached late-stage clinical development for other neurological indications, represents an ideal candidate for the treatment of CMD in which muscle cell death by apoptosis is a major contributing factor.

The Phase I study (CALLISTO) will evaluate the pharmacokinetic profile, safety and tolerability of oral omigapil in pediatric and adolescent CMD patients and establish the feasibility of conducting disease-relevant clinical assessments for the design of future efficacy trials. A new liquid formulation of omigapil has been developed by Santhera specifically for this patient population. CALLISTO will be conducted at the NIH's National Institute of Neurological Disorders and Stroke (NINDS) in Bethesda, Maryland, and will include 20 ambulatory and non-ambulatory patients aged between 5 and 16 years suffering either from the Ullrich or from MDC1A subtypes of CMD who will be treated for 12 weeks. An independent drug safety monitoring board (DSMB) will monitor patient safety and study progress.

"Congenital Muscular Dystrophies are inherited neuromuscular conditions characterized by progressive loss of muscle tissue. Frequently, these children are affected with devastating muscle loss and no treatment is currently available to slow down or stop progression of the disease. The NIH is pleased to participate in a study that will explore the feasibility of omigapil in pediatric and adolescent patients with CMD", commented Carsten Bönnemann, MD, PhD, Senior investigator and Chief of the NINDS Neuromuscular and Neurogenetic Disorders of Childhood Section at NINDS who will serve as the Principal Investigator of this study.

https://www.bionity.com/en/news/149180/santhera-repositions-omigapil-in-congenital-muscular-dystrophy.html