Edison Pharmaceuticals, Inc. enters into strategic alliance with Dainippon Sumitomo Pharma Co., Ltd.

Edison Pharmaceuticals, Inc. enters into strategic alliance with Dainippon Sumitomo
Pharma Co., Ltd.

Novel partnership in drugs targeting biological energy

Mountain View, CA. January 31, 2014. Edison Pharmaceuticals today announced that it has entered into
a strategic alliance valued up to $4.295 billion with Dainippon Sumitomo Pharma Co., Ltd. (DSP) for
the development of drugs targeting cellular energy metabolism. Under the terms of the agreement, DSP
will gain select development and commercialization rights in Japan and North America to jointly
discovered drugs in exchange for $10M upfront and $40M 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 1B, to include North America. In exchange, Edison will be eligible to receive in total between $30M and $105M per indication associated with successful development of EPI-589 in North America; between $10M and $30M 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 will also invest $50M in Edison through a preferred stock purchase agreement. At the discretion of Edison, DSP shall invest an additional $50M in the period between the first and fifth anniversaries of the initial equity closing.

Building on the March 2013 Edison/DSP collaboration agreement, the primary clinical focus of the
partnership is the adult central nervous system. Under the terms of the expanded alliance agreement,
DSP will gain access to three jointly discovered compounds and fully fund their development in Japan
and North America. Edison will retain rights to these compounds outside of Japan and North America.
Edison will have worldwide rights to up to seven jointly discovered compounds, which DSP will fund
through IND filing. In addition, DSP will broaden its rights to EPI-589 to include North America. Edison
will retain 100% ownership of its drug EPI-743, and it will continue to direct all research, clinical
development, and commercial development outside of Japan.
The research and development 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 that
the cellular redox “network” is an untapped reservoir of new drug targets, especially for high energyconsuming
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,” stated Hiroshi Noguchi, PhD, Chief
Scientific Officer and Member, Board of Directors of DSP. He further stated, “As Edison is a leader in
redox drug development, we see the highly integrated alliance structure as a way to bring our companies
closer together in order to leverage our mutual strengths, make new and important discoveries, and bring
valuable new drugs to the marketplace for critical diseases with unmet medical need.”
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
proteins responsible 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
Edison Press Release January 31, 2014
Page 2
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 expertise
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,” stated Guy Miller, MD, PhD, Chairman, CEO, Edison Pharmaceuticals, Inc.
“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 2 development for a variety of inherited rare (orphan) pediatric mitochondrial
Edison Pharmaceuticals received financial advisory services on the transaction from J.P. Morgan
Securities LLC and Barclays, and legal counsel from Morrison & Foerster, LLP.
Edison Pharmaceuticals
Edison Pharmaceuticals is a specialty pharmaceutical company dedicated to developing treatments for
children and adults with orphan mitochondrial diseases. EPI-743 is in phase 2 clinical development for
the treatment of inherited respiratory chain disorders. Double-blind placebo-controlled trials are ongoing
for the following indications: Leigh syndrome, Friedreich’s ataxia, Cobalamin C defect, and
Undiagnosed Disorders of Oxidation-Reduction.
Dainippon Sumitomo Pharma Co., Ltd.
Dainippon Sumitomo Pharma Co., Ltd. (DSP) is a multi-billion dollar, top-ten listed pharmaceutical
company in Japan with a diverse portfolio of pharmaceutical products. DSP aims to produce innovative
pharmaceutical products in the psychiatry and neurology, which is designated as a key therapeutic area,
as well as oncology. DSP has about 7,000 employees worldwide. Additional information about DSP is
available through its corporate website at www.ds-pharma.com.
Edison Information
Edison Pharmaceuticals, Inc.
350 North Bernardo Avenue
Mountain View, CA 94043

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