Pharma company Boehringer Ingelheim and leading developer of RNAi therapeutics Dicerna Pharmaceuticals has 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.

Douglas M. Fambrough, President and Chief Executive Officer of Dicerna said, “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. 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.” 

Clive R. Wood, Ph.D., Corporate Senior Vice President Discovery Research at Boehringer Ingelheim said, “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. This partnership complements our existing research efforts and expertise and offers distinct advantages in developing exciting new therapy options.”

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.