Assess the therapeutic effects of orally bioavailable, small molecule modulators of the RyR
The focus of the research project is to assess the therapeutic effects of orally bioavailable, small molecule modulators of the RyR (known as Rycals) which repair pathophysiologic calcium leak in skeletal muscle disease to reverse chronic muscle weakness. Given recent evidence that Kennedy's Disease may affect muscles directly, causing them to become weak independent of the nervous system, such modulators have the potential to improve motor function in patients affected by KD (SBMA). Rycals restore normal RyR function, improve skeletal muscle biochemical markers and improve function of pathologic muscle in various preclinical models
of skeletal muscle disease. Recent evidence from a mouse model of KD suggests that toxic androgen receptor may cause muscle weakness by impairing the function of RyR, resulting in an elevated concentration of cytosolic calcium and the loss of muscle strength in KD affected men.
"We are very pleased to have been selected by the KDA to receive funding which will support the advancement of our scientific platform against Kennedy’s Disease, complementing our lead Rycal programs for heart failure and Duchenne Muscular Dystrophy" said Dr. Sapan Shah, President and CEO of ARMGO Pharma. "We are
excited at the possibility to expand our Rycal research and development program into skeletal muscle diseases, such as Kennedy's Disease, where new treatment options for patients are desperately needed” remarked Dr. Michael Blanar, Chief Scientific Officer of ARMGO Pharma and Principal Investigator of the KDA-funded
research project.
