Dicerna Announces That Kyowa Hakko Kirin Has Selected a Second DsiRNA Therapeutic Candidate for Development, Triggering $5

  Dicerna Announces That Kyowa Hakko Kirin Has Selected a Second DsiRNA
  Therapeutic Candidate for Development, Triggering $5 Million Milestone

Business Wire

WATERTOWN, Mass. -- January 3, 2013

Dicerna Pharmaceuticals, Inc. (Dicerna), a RNA interference (RNAi) company
focused on developing breakthrough oncology therapeutics, today announced that
Kyowa Hakko Kirin Co., Ltd. (KHK) (TSE: 4151) has elected to advance the
second therapeutic oncology candidate from the research to development stage.
The achievement of this milestone triggers a $5 million payment from KHK to

Dicerna has a research collaboration and license agreement with KHK worth up
to $1.4 billion for the research, development and commercialization of Dicer
substrate siRNA (DsiRNA) pharmaceuticals and drug delivery systems for gene
targets with therapeutic potential in oncology. In 2011, Dicerna announced
that KHK had moved its first oncology candidate under the collaboration into
development and also nominated a second oncology target. The milestone
announced today is in regard to this second oncology target. DsiRNA delivered
by KHK’s drug delivery system to the target has yielded compelling activity in
animal models of difficult-to-treat tumors.

“We are delighted that our collaboration with KHK has delivered a second
product candidate that will advance to the development stage,” said Douglas M.
Fambrough, Ph.D., chief executive officer of Dicerna. “Along with Dicerna’s
own hepatocellular carcinoma program, we now have three announced oncology
candidates, all utilizing Dicerna’s Dicer Substrate Technology™, in
development. Each of these is moving toward the clinic with the goal of
addressing unmet cancer therapy needs.”

“When we first entered this collaboration with Dicerna to access the Dicer
Substrate Technology™, we saw the potential for RNAi technology to create
novel therapeutics targeting key genes at the root of cancer,” said Etsuo
Ohshima, Ph.D., managing officer and vice president, head, research division
at Kyowa Hakko Kirin. “This strategy is now providing KHK with multiple
promising development candidates and helping to complement our oncology
pipeline. The speed with which this second oncology program has moved from
concept to development candidate is impressive. Highly active DsiRNAs combined
with KHK’s proprietary lipid nanoparticle delivery technology can be validated
in vivo in less than a year.”

“We have now reached a point where the platforms are sufficiently developed
that there is a ‘plug and play’ reality to bringing therapeutic opportunities
forward,” said Bob Brown, Ph.D., chief scientific officer of Dicerna. “We will
fully leverage this to create near-term product opportunities while pushing
the technology to the next level.”

About Dicer Substrate RNAi (DsiRNA)

Dicerna’s DsiRNA molecules are uniquely structured and different from first
generation siRNAs. Specifically, the company’s DsiRNA molecules are 25 or more
base pairs in length and are processed by the Dicer enzyme. The ability to
engage the Dicer enzyme, which operates early in the gene silencing cascade,
provides functional benefits over conventional, shorter siRNA molecules.

About Dicerna

Dicerna is a private, venture-backed biopharmaceutical company harnessing RNA
interference (RNAi) to develop breakthrough, targeted cancer therapeutics.
Utilizing its Dicer Substrate Technology™ platform and EnCore™ lipid
nanoparticle delivery technology, Dicerna is developing a pipeline of programs
to reach previously undruggable intracellular disease targets. The initial
program Dicerna will bring to the clinic targets hepatocellular carcinoma, a
disease of significant worldwide unmet need, through a DsiRNA delivered by an
EnCore particle, directed to the MYC gene. The company has collaborations and
major alliances with global pharmaceutical companies and will continue to
build on this momentum to advance its research and development efforts.
Founded in 2007, Dicerna is based in Watertown, Massachusetts. For more
information, please visit www.dicerna.com.

About Kyowa Hakko Kirin

Kyowa Hakko Kirin is a leading biopharmaceutical company in Japan focusing on
its core business area of oncology, nephrology and immunology/allergy.

Kyowa Hakko Kirin leverages antibody-related leading-edge technologies to
discover and develop innovative new drugs aiming to become a global specialty
pharmaceutical company which contributes to the health and well-being of
people around the world. For more information, visit


Dicerna Pharmaceuticals
Jim Weissman, 617-612-6214
Chief Business Officer
Pure Communications
Michele Rozen, 617-730-8284
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