Reneo Pharmaceuticals has expanded its clinical development program for REN001, into glycogen storage disease type V (GSD V), also known as McArdle disease. The company is currently recruiting participants into a Phase 1b clinical trial in the United Kingdom and Spain.
McArdle disease is an inherited disorder caused by the inability of the body to break down glycogen in muscle cells. Many patients experience significant exercise intolerance, muscle pain and cramping, fatigue, the breakdown of muscle tissue and potentially kidney failure. There are no approved drugs to treat the disease.
"We believe that activating PPAR delta could provide an alternate strategy for accessing the energy needed to perform daily tasks, which is sorely needed for patients living every day with McArdle disease," said Niall O'Donnell, Ph.D., president and CEO of Reneo. "With this expansion of our REN001 clinical program, we are presently conducting clinical trials in three separate clinical indications which, if successful, could bring about meaningful improvements for patients suffering from these debilitating diseases."
In addition to GSD V, Reneo is developing REN001 to treat other genetic myopathies including long-chain fatty acid oxidation defects (FAOD) and primary mitochondrial myopathies (PMM).
