2021.12.06 Press Release

A novel therapeutic drug, World's first, from Japan: First step to overcoming mitochondrial Disease MA-5, the Mitochondrial Disease Drug, Begins Clinical Trials in Healthy Adult Subjects

Mitochondrial disease Note 1. is a rare disorder that causes damage to mitochondria, the energy-producing factories in cells, resulting in decreased energy production (ATP ) in the neuromuscular, cardiovascular, metabolic, renal-urinary, hematological, visual, endocrine, and digestive systems. At present, however, there is no treatment other than taurine that has been proven to be effective in a rigorous clinical trial. The principal investigator, Professor Takaaki Abe, has developed Mitochonic acid-5 (MA-5), a novel compound for mitochondrial diseases with a completely new mechanism different from existing drugs. MA-5 inhibits cell death in cultured cells derived from patients with mitochondrial disease. Moreover, it improved cardiac and renal respiratory function and increased survival rates in mitochondrial disease mouse models. By accelerating ATPase dimerization through binding with mitofilin/Mic60. The principal investigator’s group with the support of AMED Moonshot Research and Development Project will conduct a Phase I clinical trial from January 2022 to confirm the safety of MA-5, which is expected to be the world's first mitochondrial disease treatment from Japan, in healthy adult subjects. This study will confirm whether MA-5 can be safely administered to humans (safety), how the human body absorbs MA-5, how it is transported in the blood, and metabolized (pharmacokinetics). Upon completing this study, we plan to conduct a phase II clinical trial in which MA-5 will be administered to actual patients. This clinical trial of MA-5 is expected to lead to the inhibition of the progression and treatment of mitochondrial diseases, which are rare and intractable diseases for which there is currently no effective treatment, and in the future, MA-5 is expected to be helpful in the prevention and treatment of diseases such as deafness, sarcopenia, ALS, and Parkinson's disease, which are also caused by reduced mitochondrial function.