Reneo Pharmaceuticals has initiated a phase 1b clinical trial of REN001 in glycogen storage disease type V (GSD V), also called McArdle disease.
The California-based clinical-stage pharma company is currently enrolling participants into the early-stage clinical trial in the UK and Spain.
Considered as an inherited disorder, McArdle disease is caused by the body’s inability to break down glycogen in muscle cells. Several patients face considerable exercise intolerance, muscle pain and cramping, fatigue, the breakdown of muscle tissue and possibly kidney failure as well.
Currently, there are no approved drugs for the treatment of McArdle disease.
Niall O’Donnell – president and CEO of Reneo Pharmaceuticals said: “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.
“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.”
REN001 is also being developed for the treatment of long-chain fatty acid oxidation defects (FAOD), primary mitochondrial myopathies (PMM), and other genetic myopathies.