Santhera Repositions Omigapil in Congenital Muscular Dystrophy
News Jul 17, 2014
Santhera Pharmaceuticals has 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.
"The initiation of the CALLISTO trial constitutes the fulfillment of one major goal for the EndoStem project, namely to get new drugs into the clinic that target diseased muscle. Funded by the European Commission and now in its 5th year, EndoStem is a collaborative network of leaders in stem cell biology and private entities that came together to drive research from the laboratory to the clinic with a particular focus on muscular dystrophies. A central strategy is to target the diseased tissue in order to promote regeneration and prevent degeneration, for which omigapil is a perfect example", explained Professor David Sassoon, coordinator of the EndoStem Programme.
"With currently no effective treatment available for patients, the CALLISTO trial offers hope to patients with Congenital Muscular Dystrophies. As the leading organization representing the interests of patients with this disease, Cure CMD welcomes CALLISTO as a major milestone for our foundation", added Patrick May, President of Cure CMD.
"Our foundation is dedicated to support clinical research for the development of therapies for rare neuromuscular diseases both in Switzerland and internationally. We are glad to support the consortium of experts in order to conduct the CALLISTO trial", added Jacques Rognon, Chairman of Swiss Foundation for Research on Muscle Diseases (FSRMM).
"Combined international efforts of private and public institutions are needed to advance medical research into orphan diseases. This externally funded collaboration is a perfect strategic fit for Santhera to which it brings orphan drug and muscular dystrophy clinical development expertise whilst retaining global commercial rights for omigapil in CMD", concluded Thomas Meier, Chief Executive Officer of Santhera.
PhoreMost and Plexxikon Collaborate to Identify Novel Drug TargetsNews
PhoreMost, the UK-based biopharmaceutical company dedicated to drugging ‘undruggable’ disease targets, today announced it has signed a collaborative agreement with Plexxikon Inc., the small molecule structure-guided R&D centre of Daiichi Sankyo.READ MORE
Anti-Malaria Drugs Delivered With CaffeineNews
Many drug delivery systems, such as capsules and tablets, can be difficult to swallow, especially for children. Making delivery systems out of polymer gels is an attractive option, but usually requires hazardous levels of heavy metals to be used. Now, scientists have found a way to build these gels from caffeine molecules.READ MORE
Lonza Opens World’s Largest Dedicated Cell-and-Gene-Therapy Manufacturing Facility in TexasNews
First-of-its-kind, state-of-the-art manufacturing facility with capacity to produce treatment for thousands of patients suffering from rare genetic disorders or life-threatening diseases.READ MORE
Comments | 0 ADD COMMENT
World Congress on Advanced Biomaterials and Tissue Engineering
Jun 20 - Jun 21, 2018