ARMGO Pharma Servier Launch Clinical Trials For Duchenne Muscular Dystrophy Drug

Margarida Azevedo, MSc avatar

by Margarida Azevedo, MSc |

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shutterstock_81404410A new preclinical trial revealed the efficacy and IND/CTA enabling studies with ARM210/S48168. ARMGO Pharma and Servier are set to initiate clinical trials for the treatment of Duchenne Muscular Dystrophy (DMD).

The companies have been working on a drug called Rycals®. Rycals targets the Ryanodine Receptor (RyR), an intracellular calcium channel that becomes leaky in conditions such as Duchenne Muscular Dystrophy, and that is known to contribute to the muscle atrophy. Studies with animal models found that Rycals repairs RyR-mediated intracellular calcium leak, thus improving muscle capacity. Similar effects have been found in the on cardiac muscle in animal models of heart disease.

One of the Rycal drugs discovered by ARMGO Pharma, called ARM210/S48168, is going to the tested in the trials as a mono-therapy and in combination with other drugs as treatment for DMD and other disorders affecting the muscles.

Studies with this compound in mdx mouse models of DMD revealed improvements (both short- term, 4 weeks and longer- term, 3 months) in the capacity to perform exercise, specific muscle and grip force and in muscle histology in comparison with vehicle-treated controls.

The results of preclinical efficacy and safety studies, showed that ARM210/S48168 might improve muscle function and also repair intracellular calcium leak in myopathy irrespective of the mutation causing the pathology. After the efficacy studies in DMD patients, the companies are going to test the compound efficacy in other forms of myopathy.

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The trials for ARM210/S48168 treatment are going to start in 2015, and in a recent press release Sapan Shah, President and CEO of ARMGO Pharma commented, “We are very pleased to have formally reached this critical clinical advancement decision for ARM210/S48168 with our partner Servier,”  “We are excited at the prospect of bringing a novel therapy to patients suffering from skeletal muscle diseases such as DMD, where new treatments are desperately needed.”, Shash added.

In the press release Dr. Emmanuel Canet, MD PhD, Vice-President Research and Development at Servier said, “This agreement demonstrates our long-term commitment in research to discover new therapy that will benefit young patients in a disease that has a major impact on the quality of life and life expectancy in the boys affected.”

Additionally, Dr. Patricia Belissa-Mathiot, Director of Innovative Center for Rheumatology at Servier said in the press release, “Given the unique mode of action of ARM210/S48168, which corrects intracellular calcium leak regardless of the underlying genetic mutation, its therapeutic value could be assessed in other myopathies once efficacy is established in DMD.”