• Dicerna to receive up to $201 million in upfront and success-based development and commercialization milestones, excluding royalties, for an undisclosed target
• The collaboration will investigate a new therapeutic approach that enables addressing previously inaccessible drug targets to protect and restore liver functionality in NASH

INGELHEIM, Germany & CAMBRIDGE, Mass. -- (BUSINESS WIRE) --

Boehringer Ingelheim and Dicerna Pharmaceuticals (NASDQ: DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, today announced a research collaboration and license agreement to discover and develop novel GalXC™ RNAi therapeutics for the treatment of chronic liver diseases. The partnership will initially focus on nonalcoholic steatohepatitis (NASH), a devastating, chronic liver disease for which there is no approved treatment option.

NASH is caused by the buildup of fat in the liver, potentially leading to liver fibrosis and cirrhosis. It has an especially high prevalence among obese and diabetic patients, and is an area of high unmet medical need. NASH is expected to soon become the most common cause of advanced liver disorders, and it often necessitates liver transplantation.

Dicerna’s GalXC technology platform uses RNAi to inhibit the expression of disease-causing genes by destroying the messenger RNAs (mRNAs) of those genes. This new approach has the potential to treat diseases by silencing previously inaccessible drug targets. It adds a further breakthrough therapy opportunity to Boehringer Ingelheim’s cardiometabolic pipeline and provides additional combination options.

“We believe that Dicerna’s GalXC technology platform is ideally suited for the development of novel RNAi therapies for nonalcoholic steatohepatitis and other chronic liver diseases,” said Douglas M. Fambrough, President and Chief Executive Officer of Dicerna. “With strong capabilities in drug discovery, deep expertise in the cardiometabolic space, and proven commercial experience, Boehringer Ingelheim is a natural partner to speed the development of the first GalXC RNAi program targeting chronic liver disease. The collaboration combines the strong capabilities of both companies to pursue the full potential of Dicerna’s GalXC technology to bring valuable and differentiated RNAi therapies to patients with liver diseases and their healthcare teams, and reflects both the promise of the GalXC technology and the strength of its underlying intellectual property.”

“At Boehringer Ingelheim, our research team is diligently working to discover effective new therapies for NASH and other chronic liver diseases, which is a priority area for us,” said Clive R. Wood, Ph.D., Corporate Senior Vice President Discovery Research at Boehringer Ingelheim. “This partnership complements our existing research efforts and expertise and offers distinct advantages in developing exciting new therapy options,” added Wood.

Boehringer Ingelheim has a long history of excellence in the discovery and development of medicines for cardiometabolic disease patients. It has established a broad portfolio of marketed products for thromboembolic diseases, type 2 diabetes, acute myocardial infarction, hypertension, and cardio-renal risk reduction. The cardiometabolic diseases pipeline extends beyond type 2 diabetes and anticoagulation with a focus on innovative drugs for the treatment of the devastating consequences of diabetes as well as contributing factors like obesity.

Dicerna is building a portfolio of research and development programs to advance the treatment of diseases involving the liver, including chronic liver diseases, viral infectious diseases, cardiovascular diseases and rare diseases. The company aims to leverage its proprietary GalXC technology to develop innovative RNAi therapeutics to positively transform the lives of individuals living with these diseases.

Under the terms of the agreement, Dicerna may receive more than $200 million from Boehringer Ingelheim, including an upfront payment, development and commercial milestone payments, and research and development reimbursement for a GalXC candidate product addressing an undisclosed NASH target. Dicerna is also eligible to receive royalties staggered up to double-digits on worldwide net sales.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Examples of forward-looking statements include, among others, statements we make regarding: (i) the therapeutic and commercial potential of GalXC™; (ii) research and development plans related to GalXC; and (iii) the potential of our technology and drug candidates in our research and development pipeline. The process by which an early stage platform such as GalXC could potentially lead to an approved product is long and subject to highly significant risks, particularly with respect to a pre-clinical research collaboration. Applicable risks and uncertainties include those relating to our preclinical research and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q filing and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Please click on the link below for ‘Notes to Editors’:

http://www.boehringer-ingelheim.com/press-release/collaboration-dicerna-develop-nash-treatments

View source version on businesswire.com: http://www.businesswire.com/news/home/20171102005614/en/

CONTACT:

Boehringer Ingelheim Corporate Center GmbH
Dr. Reinhard Malin
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Media + PR
reinhard.malin@boehringeringelheim.com
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or
Linda Ruckel
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Boehringer Ingelheim Pharmaceuticals, Inc.
Ridgefield, Connecticut
linda.ruckel@boehringeringelheim.com
P: 203-791-6672 :: C: 203-957-5114
www.boehringeringelheim.com
or
For Dicerna Pharmaceuticals
Paula Schwartz
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Rx Communications Group
pschwartz@rxir.com
P: 917-322-2216
www.rxir.com
or
Alex Van Rees
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SmithSolve Communications
alex.vanrees@smithsolve.com
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www.smithsolve.com