Date: 2014-12-04
Type of information: Filing of an IND
phase: 1
Announcement: filing of an IND
Company: Armgo Pharma (USA - NY) Servier (France)
Product: ARM210/S48168
Action
mechanism: Rycals target the Ryanodine Receptor (RyR), an intracellular calcium release channel that becomes leaky in disease states including Duchenne Muscular Dystrophy, contributing to muscle damage and loss of function. Rycals have been shown in animal models of muscle disease to repair RyR-mediated intracellular calcium leak and thereby improve muscle specific force and exercise capacity. Further, Rycals have been shown to exert similar beneficial effects on cardiac muscle in animal models of heart disease. The proprietary drug candidate ARM210/S48168, discovered by Armgo Pharma, was selected by the Armgo-Servier collaboration from a library of Rycal candidates for preclinical advancement as a potential treatment for DMD and other muscle disorders. With its mechanism of action and oral delivery formulation, ARM210/S48168 has the potential to provide benefit across skeletal muscle, diaphragm and heart muscle in DMD patients regardless of genetic background, both as a monotherapy as well as in conjunction with other treatments.
Disease: Duchenne muscular dystrophy
Therapeutic area: Genetic diseases - Neuromuscular diseases - Rare diseases
Country:
Trial details:
Latest
news: * On December 4, 2014, Armgo Pharma and Servier announced the successful completion of preclinical efficacy and IND/CTA enabling studies with ARM210/S48168, along with a formal decision to advance the program into early clinical development initially targeting treatment for patients with Duchenne Muscular Dystrophy (DMD). Armgo Pharma and Servier have collaborated since 2006 on the advancement of a novel class of small molecule drugs known as Rycals®, which were initially discovered through the pioneering work of Dr. Andrew R. Marks, the Clyde and Helen Wu Professor of Molecular Cardiology (in Medicine), Chairman of the Department of Physiology and Cellular Biophysics, and Founding Director of the Clyde and Helen Wu Center for Molecular Cardiology at Columbia University Medical Center. In preclinical efficacy studies using the mdx mouse model of Duchenne Muscular Dystrophy, oral treatment with ARM210/S48168 showed significant and robust improvements in exercise capacity, specific muscle force, grip strength and muscle histology compared to vehicle-treated controls. These improvements were validated in both short term (4 week) and longer term (3 month) efficacy studies in mdx mice conducted by multiple, internationally recognized independent laboratories. Results from one of these studies were recently presented at the 19th World Muscle Society Congress, November 7-11, 2014 (Abstract G.P. 90, Capogrosso et al., Neuromuscular Disorders 24 (2014) 791-924). In addition to the preclinical efficacy models, an IND/CTA enabling package of GLP safety/toxicology studies with ARM210/S48168 has been completed by Armgo and Servier, with no issues precluding further development. Based on the collective strength of the completed preclinical efficacy and safety studies, ARM210/S48168 has been formally selected for advancement for clinical development by the Armgo-Servier collaboration, resulting in a pre-agreed R&D payment by Servier to Armgo, along with a commitment to support clinical development costs. Clinical studies will begin in 2015, following finalization of a global clinical development plan, completion of manufacturing and formulation work, and receipt of input from regulatory agencies. The initial therapeutic indication for the ARM210/S48168 clinical program is the treatment of Duchenne Muscular Dystrophy (DMD). Given the results of preclinical efficacy studies, ARM210/S48168 treatment has not only the potential to result in improvements in muscle function but also due to its unique mode of action, which repairs intracellular calcium leak in myopathy regardless of the causative mutation to have an impact on the progression of the disease. Once efficacy is demonstrated in DMD patients, other forms of myopathy will be evaluated for advancement and may also benefit from this new treatment.
