ARMGO Pharma received a $1 million award from the Muscular Dystrophy Association (MDA) for preclinical work in support of an investigational new drug application with the FDA for ARM210, a small molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy (DMD). The funding to ARMGO Pharma comes from MDA Venture Philanthropy (MVP), part of the company’s translational research program.

The project being advanced by ARMGO is focused on a class of small molecule drugs known as Rycals, which target the ryanodine receptor (RyR), an intracellular calcium channel that becomes leaky in disease states including muscular dystrophy, contributing to loss of muscle strength and function. According to the company, Rycals have been shown in animal models of muscle disease to repair RyR-mediated calcium leak and thereby improve specific muscle force and exercise capacity.

“This is a very interesting potential therapeutic,” said Jane Larkindale, MDA’s vice president for research. “It works in a different way than the other potential therapeutics in development, which suggests that in addition to being effective on its own, it also might be able to be added on to other treatments, when other treatments become available.”

In August 2012, ARMGO Pharma, had a study published in the scientific journal Cell, which revealed the underlying role of calcium leak through the RyR as an important contributor to stress-induced cognitive dysfunction.

DMD is one of nine types of muscular dystrophy, a group of genetic, degenerative diseases primarily affecting voluntary muscles. Caused by mutations in the gene that makes dystrophin, DMD eventually weakens all voluntary muscles, the heart, and breathing muscles.

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