Edison Pharmaceuticals, a specialty pharmaceutical company dedicated to developing treatments for children and adults with orphan mitochondrial diseases, has entered into a strategic alliance valued at up to $4.295 billion with Dainippon Sumitomo Pharma (DSP) for the development of drugs targeting cellular energy metabolism.

DSP will gain select development and commercialization rights in Japan and North America to jointly discovered drugs in exchange for $10 million upfront and a $40 million payment in R&D support. In addition, DSP will fully fund the development of 10 new jointly discovered drugs through IND filing and broaden its rights to EPI-589, currently in phase Ib, to include North America. In exchange, Edison will be eligible to receive a total of $30 million to $105 million per indication associated with successful development of EPI-589 in North America; between $10 million and $30 million per indication in development milestones associated with successful development of jointly discovered compounds in Japan and North America; up to $3.86 billion in commercial milestone payments for jointly discovered compounds and EPI-589 in total; and double-digit royalties on commercial sales. DSP also will invest $50 million in Edison through a preferred stock purchase agreement. At the discretion of Edison, DSP shall invest an additional $50 million in the period between the first and fifth anniversaries of the initial equity closing.

The R&D program is directed at the characterization of the redox control energy system critical to the generation and regulation of cellular energy metabolism. The parties believe the cellular redox “network” is an untapped reservoir of new drug targets, especially for high energy-consuming organs such as the brain. DSP and Edison will work together under a novel collaborative framework to discover, characterize and translate drugs into clinical development.

“The broadening of our partnership with Edison reflects the success we have had to date in our current collaborative development programs. Compelling data suggest that the mitochondria and redox regulation play a central role in a variety of disease mechanisms,” said Hiroshi Noguchi, Ph.D., chief scientific officer and member, board of directors, of DSP.

The term redox refers to a set of chemical reactions involving the tandem accepting and donating of electrons. Redox biochemical and enzyme-catalyzed reactions underlie the vast majority of the chemistry responsible for the generation of energy within the cellular powerhouse, the mitochondrion. In addition, redox reactions play a critical role in the regulation of energy metabolism.

Edison’s initial clinical focus is on a set of pediatric diseases where there are unambiguous genetic alterations in mitochondrial proteinsresponsible for energy generation and regulation. These rare (orphan) diseases are collectively referred to as inherited respiratory chain diseases of the mitochondria. They form the basis of Edison’s initial drug development efforts; the breadth of Edison’s proprietary technology platform extends well beyond these conditions. Inherited pediatric mitochondrial disease and a variety of adult neurodegenerative diseases may arise through analogous genetic errors in mitochondrial proteins, giving rise to common biochemical consequences. For example, in the case of Parkinson’s disease, a variety of inherited forms of the disease are caused by defects in proteins associated with mitochondrial function. Additionally, both pediatric mitochondrial diseases and adult neurodegenerative diseases display a common biochemical signature of excess electrons. This is commonly referred to as oxidative stress.

Edison is leveraging its core experience in redox biochemistry and its proprietary technology platform to develop novel drugs targeting redox pathways common to pediatric mitochondrial disease and adult neurodegenerative diseases, as well as fundamental aging mechanisms.

“Our partnership with DSP codifies a common vision for developing drugs targeting unmet medical needs. We will be able to grow and explore a frontier of science through a collaborative and iterative learning process,” said Guy Miller, M.D., Ph.D., chairman and CEO, Edison Pharmaceuticals. “Resources committed by DSP to the partnership will accelerate our first-mover advantage in redox drug development. This will allow us together to expand Edison’s pipeline, bringing 10 new compounds into clinical development over the next five years. In addition, the DSP investment will allow Edison to build its commercial enterprise in pediatric medicines. This will start with EPI-743, Edison’s drug candidate currently in phase II development for a variety of inherited rare pediatric mitochondrial diseases.”