ARMGO Pharma and the Muscular Dystrophy Association (MDA) today announced that $1 Million has been awarded for preclinical work in support of an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for ARM210, a novel, orally available, small-molecule Rycal drug that has potential as a treatment for Duchenne muscular dystrophy (DMD). The funding award to ARMGO Pharma comes from MDA Venture Philanthropy (MVP), part of MDA's translational research program.
The project being advanced by ARMGO Pharma is focused on a novel 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. 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. ARMGO Pharma's experimental proprietary drug ARM210 has been selected by the company from its library of Rycal candidates for preclinical advancement as a potential treatment for DMD. With its unique mechanism of action and oral delivery formulation, ARM210 has the potential to provide benefit across skeletal muscle, diaphragm and heart muscle in DMD patients regardless of genetic background, both as a mono-therapy as well as in conjunction with other treatments.
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, a protein that normally protects muscle cells and keeps them intact, DMD eventually weakens all voluntary muscles, and the heart and breathing muscles. DMD affects approximately 1 in 3,500 boys with an estimated patient population exceeding 50,000 worldwide.
"We are very pleased to have been selected by the MDA to receive funding which will support the advancement of ARM210," said Dr. Sapan Shah , President and CEO of ARMGO Pharma. "We are excited at the possibility to expand our Rycal program into skeletal muscle diseases such as DMD, where new treatment options for patients are desperately needed."
"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."
ARMGO Pharma; Muscular Dystrophy Association