PMM is a complex mitochondrial disease in which genetic mutations primarily impair the function of mitochondria, resulting in reduced muscle function, reduced endurance to exercise (i.e., exercise intolerance), increased fatigue, and muscle atrophy. In addition, PMM may present serious and life-threatening health conditions due to multiple organ involvement. For example, decreased myocardial function may lead to cardiomyopathy and heart failure. Decreased function of respiratory muscles may cause respiratory failure and pneumonia. In patients with mitochondrial disease due to pathogenic mutations in either the mtDNA or nDNA genome, the minimum point prevalence is estimated at 12.5 in 100,000 (approximately 1 in 8,000) for adults with clinical manifestations, and 23 in 100,000 (approximately 1 in 4,300) for adults with or without clinical manifestations.1 There is no FDA-approved treatment for PMM, a rare disease with a high unmet medical need.
Preclinical data and results from Astellas' Phase I healthy volunteer study collectively suggest that ASP0367 an orally administered PPAR modulator could improve exercise intolerance and fatigue in PMM patients by increasing the number and enhancing the function of mitochondria in patient's cells. ASP0367 will be entering a Phase II / III study (MOUNTAINSIDE) to validate the efficacy and safety in PMM patients.
Astellas explores mitochondrial biology as a Primary Focus of its R&D strategy and is committed to providing treatments using new modalities. Targeting mitochondrial function is an innovative approach to address diseases with significant unmet medical needs in novel and effective ways. With the acquisition of
The
'Primary mitochondrial myopathies are a serious, complex disease with significant unmet need and no approved therapies,' said
'At Astellas, mitochondrial biology is one of our Primary Focus Areas, driving our commitment to bring new biological concepts into the clinic for rapid proof of concept,' said
About ASP0367
ASP0367, a selective modulator of PPAR, has the potential to be a first-in-class treatment for PMM. In a preclinical study, ASP0367 increased the expression of PPAR target genes and enhanced mitochondrial function in fibroblasts collected from patients with PMMs. A phase I study in healthy adults demonstrated dose-dependent increased expression of PPAR target genes and ASP0367 doses were found to be safe and well tolerated in this study. ASP0367 is also being developed as a potential treatment for Duchenne muscular dystrophy. Other Phase Ib studies are also under preparation.
About Mitobridge, an
About Astellas
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In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management's current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas' intellectual property rights by third parties.
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