PMM is a complex mitochondrial disease in which genetic mutations mainly impair the function of mitochondria that may result in reduced muscle function, reduced endurance to exercise, increased fatigue and muscle atrophy.
Preclinical data and results from the company’s phase I healthy volunteer study collectively demonstrate that ASP0367 can improve exercise intolerance and fatigue in PMM patients by increasing the number and enhancing the function of mitochondria in patient’s cells.
ASP0367, a selective modulator of PPARδ, has increased the expression of PPARδ target genes and improved mitochondrial function in fibroblasts collected from patients with PMMs in a preclinical study.
A phase I study in healthy adults showed dose-dependent enhanced expression of PPARδ target genes, and it found to be safe and well-tolerated in the study.
The company is also focused on the development of ASP0367 as a potential treatment for Duchenne muscular dystrophy.
In 2018, Astellas Pharma acquired Mitobridge to gain access to its expertise in mitochondrial biology and a pipeline of programmes such as ASP0367.
Astellas medical specialities and therapeutic area head and senior vice president Dr Salim Mujais said: “Primary mitochondrial myopathies are a serious, complex disease with significant unmet need and no approved therapies.
“With the development of ASP0367, an oral, once-daily modulator of mitochondrial energy production, we are hoping to alleviate the serious burden of this disease on patients, their families and caregivers.”
In February this year, Pfizer and Astellas Pharma announced that Xtandi (enzalutamide) has shown significant improvement in overall survival (OS) in phase 3 PROSPER trial.