Reneo Pharmaceuticals has been granted the orphan drug designation (ODD) for its lead drug candidate REN001 from the US Food and Drug Administration (FDA) for the treatment of primary mitochondrial myopathies (PMM).
According to the California-based clinical-stage company, REN001 is a selective PPAR delta agonist, which is being developed for the treatment of genetic myopathies, including primary mitochondrial myopathies.
Reneo Pharmaceuticals said that a recently concluded 12-week clinical study of the PPAR delta agonist in patients with primary mitochondrial myopathies yielded positive preliminary results.
Primary mitochondrial myopathies are a group of life-threatening diseases that result from genetic mutations impairing mitochondrial function. Patients with primary mitochondrial myopathies usually have reduced muscle function, which adversely affects daily functions and are faced with decreased life expectancy.
Currently, there are no FDA approved drugs for the treatment of primary mitochondrial myopathies.
Niall O’Donnell – CEO of Reneo Pharmaceuticals said: “Patients with primary mitochondrial myopathies have many aspects of their lives impacted.
“The preliminary safety data and exploratory endpoint results from our first PMM clinical study has driven us to move forward, into a large, global clinical trial in PMM. We hope this will result in a great stride forward for patients with PMM.”
REN001 also holds FDA orphan drug designation for the treatment of fatty acid oxidation disorders, which was granted to it in 2019. The PPAR delta agonist is also in clinical development for glycogen storage disease type V, which is also called McArdle disease.