ARMGO Pharma has received a research grant from the Kennedy's Disease Association (KDA) to support preclinical work on new, orally available compounds that have potential as a novel treatment for Kennedy's Disease (KD) also known as spinal bulbar muscular atrophy (SBMA).
With the grant, ARMGO Pharma will focus on new class of small molecule drugs called Rycals which target the Ryanodine Receptor (RyR), an intracellular calcium channel that becomes leaky in disease states, including KD and contributes to loss of muscle strength and function.
KD is an incurable and non-treatable recessive X-linked, genetic progressive neuromuscular disease, under which spinal and bulbar motor neurons are primarily affected causing muscle weakness and wasting throughout the body including the extremities (legs/arms) as well as in the face and throat.
Symptoms of KD usually appear in adults aged between 30 and 50, and it is estimated that one in 40,000 male individuals across the world have the disease, however many go undiagnosed or misdiagnosed for years.
The company said that the most frequent misdiagnosis is the fatal Lou Gehrig's Disease (ALS).
The project is mainly focused at evaluating 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.
Rycals restore normal RyR function, enhances skeletal muscle biochemical markers and function of pathologic muscle in several preclinical models of skeletal muscle disease.
ARMGO Pharma president and CEO Sapan Shah said, "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."