Dainippon Sumitomo inks $545M-plus orphan drug R&D pact with Edison

Up until now, Edison Pharmaceuticals in Mountain View, CA, has been flying under the radar. But today it shed stealth mode and went public with a $545 million-plus collaboration deal with Japan's Dainippon Sumitomo that centers on its lead late-stage drug for rare diseases. And with the backing of a well-known billionaire investor, the biotech is already at work laying the foundation for commercialization efforts.

Dainippon is putting $50 million on the table in an upfront fee and R&D support for Edison, along with up to $35 million in development milestones per indication and $460 million in commercialization milestones and royalties.

The Japanese company, which has done a number of research deals with U.S. biotech companies, gains the Japanese rights to EPI-743 and will now take the reins on development and approval in their home country, initially focusing on orphan pediatric mitochondrial disease. The deal also covers EPI-589, now the subject of a collaboration between the two companies on adult central nervous system diseases, which points to the potential to branch out into much larger markets ahead.

Mitochondrial disease, which afflicts one in every 4,000 children, interferes with the production of energy in cells, triggering a cascade of cell damage and death that typically starts in childhood. There are no effective treatments.

CEO Guy Miller tells FierceBiotech that the company got started about 6 years ago and was largely funded by billionaire real estate investor, venture capitalist and sometime biotech investor Carl Berg. Mitsui Global Venture stepped in recently to lead a $20 million round and now the company is focusing on wrapping its first pivotal Phase IIb/III studies by the end of this year or the beginning of next.

"Our mission is to get these kids a drug," says Miller.

The 35 staffers on the team have initially set their sights on Leigh syndrome and Friedreich's ataxia. There are also possibilities for more work ahead on big CNS targets, including Alzheimer's and Parkinson's, which share some of the same mitochondria triggers. And Miller says he's confident that a positive round of results from ongoing studies will set the stage for regulatory approvals in Europe and the U.S. The company has already begun to set up a commercialization unit for their drugs.

- here's the press release

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