The award supports laboratory work that will enable the company to submit an investigational new drug (IND) application to the U.S. Food and Drug Administration. (An IND application is a step a company must take before it can test a new drug in humans.) Receiving an IND approval will allow ARMGO Pharma to prepare for clinical trials with ARM210 in healthy volunteers and for subsequent clinical studies in people with DMD.
The experimental class of compounds — which ARMGO calls Rycal drugs — is designed to normalize the way skeletal muscle fibers and heart muscle cells handle calcium.
The lead compound, ARM210, is aimed at treating DMD or BMD, which are caused by mutations in the dystrophin gene. However, the strategy is not dependent on the specific disease-causing genetic mutation and potentially could have application to other muscle disorders.
To learn more, read DMD/BMD: MDA Funds Development of Drug to Fix Calcium Leaks.