Specialty pharmaceutical company Edison Pharmaceuticals has entered into a R&D and commercialization agreement with Dainippon Sumitomo Pharma, a Japanese pharmaceutical company, for the development of EPI-743 and EPI-589 in Japan.

DSP will gain development and commercialization rights in Japan in exchange for Edison receiving $35 million in upfront and $15 million in R&D support. In addition, Edison will be eligible to receive $10-$35 million in development milestones per indication and up to $460 million in commercial milestone payments as well as royalties on commercial sales.

The initial scope of the transaction includes both pediatric orphan inherited mitochondrial and adult central nervous system diseases. DSP will undertake activities required for development, approval and commercialization of EPI-743 in Japan. The work will initially focus on orphan pediatric mitochondrial disease. Edison will retain 100% ownership and direct all research, clinical, and commercial development of EPI-743 and EPI-589 outside of Japan.

In addition, the parties will collaborate on the R&D of EPI-589 with a focus on adult central nervous system disease. This collaboration is based upon the emerging body of data implicating redox and mitochondrial dysfunction as a root cause of neurologic disorders.

Edison's translational platform is based on redox biochemistry. It bridges key learnings derived from genetically confirmed pediatric rare mitochondrial disease to adult central nervous system disorders where redox and mitochondrial dysfunction are likely to also play a critical role. The Edison redox platform consists of proprietary laboratory and clinical tools that enable the discovery, optimization and clinical validation of redox-directed drugs. Specifically, through employing both laboratory and clinically derived redox signatures of disease and drug action, Edison is able to develop drugs at a fraction of the time and cost of current drug development processes, thereby reducing risk and cost and increasing the likelihood of success.

Edison will use proceeds derived from the partnership to advance the late stage development and commercialization of EPI-743 for Leigh syndrome and Friedreich's ataxia, as well as to advance EPI-743 in other exploratory phase II trials for rare pediatric diseases with shared mitochondrial mechanisms.

"Our partnership with Dainippon Sumitomo Pharma will allow Edison to accelerate the development and approval of the first drug for inherited respiratory chain diseases of the mitochondria," said Guy Miller, MD, PhD, chairman and CEO of Edison. "Our shared vision of the role of redox control and the mitochondria in human disease will help us extend our learnings derived from rare pediatric diseases to poorly treated acute and chronic adult CNS diseases."