Alex Dorenbaum, MD, Chief Medical Officer of Reneo Pharmaceuticals, gives an overview of the STRIDE study of mavodelpar (REN001) in primary mitochondrial myopathies (PMM).
PMM are a group of rare metabolic disorders caused by mutations or deletions in mitochondrial or nuclear DNA. These alterations impair the ability of mitochondria to generate energy, resulting in energy deficits that are most pronounced in tissues with high energy demand such as muscle, brain, and heart. The symptoms of PMM include muscle weakness, exercise intolerance, movement disorder, deafness, blindness, and droopy eyelids. The prognosis for these disorders ranges in severity from progressive weakness to death. Currently, there is no approved treatment for PMM.
Recently, Reneo Pharmaceuticals announced that their most recent trial investigating mavodelpar, STRIDE, has achieved its target enrollment. As Dr. Dorembaum explains, mavodelpar is a selective peroxisome proliferator-activated receptor delta agonist currently in clinical development for PMM.
STRIDE is a randomized, double-blind, placebo-controlled pivotal phase 2b trial of mavodelpar in adult patients with PMM due to mitochondrial DNA defects. The study is designed to assess the efficacy and safety of 100 mg mavodelpar administered orally, once daily for 24 weeks. The primary efficacy endpoint is the change from baseline in the distance walked during the 12-minute walk test at week 24.
There is also an open-label extension study, STRIDE AHEAD, which is a 24-month, long-term safety trial for patients with PMM outside of the United States. The STRIDE AHEAD study was recently amended to allow enrollment of patients with PMM due to both mitochondrial and nuclear DNA defects.
Reneo intends to submit the data from STRIDE and STRIDE AHEAD to the U.S. Food and Drug Administration and the European Medicines Agency in 2024.
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