The Pipeline

ARMGO’s lead Rycal candidate (ARM210) is in development for Duchenne Muscular Dystrophy (DMD). Multiple studies in mdx mouse model of DMD demonstrate improvements in voluntary wheel activity, grip strength, EDL muscle and diaphragm specific force, and muscle histopathology. Robust GLP safety/toxicity package completed, with Phase 1 clinical trials planned for July 2015. Other skeletal muscle disease opportunities for ARM210 include Becker and Limb-Girdle Muscular Dystrophies, Sarcopenia, Cachexia, Congenital Myopathies, and Spinal-Bulbar Muscular Atrophy (Kennedy’s Disease), among others.

ARMGO’s cardiovascular program targets heart failure, arrhythmias and CPVT. ARM036 (aladorian) was the first in-house discovered, novel, small molecule NCE (Rycal®) from ARMGO’s library that was advanced into clinical studies. ARM036 completed multiple Phase IIa clinical studies in chronic heart failure patients, demonstrating positive activity and safety in patients, and validating ARMGO’s approach for drug discovery and candidate selection on the RyR target. Second generation candidates with improved profile are in preclinical development.

A third effort targets CNS disorders including PTSD, Alzheimer’s Disease (AD), and Huntington’s Disease (HD). There is growing evidence that neuronal RyRs are intimately involved in the pathology of learning & memory disorders and of neurodegenerative diseases, and that restoration of normal intracellular calcium homeostasis is important to neuronal cell survival and normal function.