The companies will use Dicerna’s RNA interference (RNAi)-based GalXC™ platform.

Boehringer Ingelheim (BI) is making a concerted effort to develop treatments against nonalcoholic steatohepatitis (NASH). In August 2017, the company launched a Phase IIa study in the EU and US to assess its candidate BI 1467335 (formerly PXS-4728A), which it acquired from Pharmaxis in May 2015. The oral inhibitor of amine oxidase, also known as copper-containing 3 (AOC3), vascular adhesion protein-1 (VAP-1) or semicarbazide-sensitive amine oxidase (SSAO), works by blocking leukocyte adhesion and tissue infiltration in inflammatory processes underlying NASH, has received Fast Track designation from the US Food and Drug Administration.

More recently, BI announced that it is partnering with Dicerna Pharmaceuticals to develop treatments for chronic liver diseases, initially focusing on NASH. Dicerna has the potential to earn up to $201 million from the collaboration and license agreement. Dicerna will receive $10 million upfront and up to $191 million in development and commercial milestone payments, as well as royalty payments on potential global net sales and additional money if BI opts to add a second target.

The companies will use Dicerna’s RNA interference (RNAi)-based GalXC™ platform, in which RNAi destroys the messenger RNAs of specific disease-causing genes, thus preventing their expression. The technology is suitable for application across many different diseases, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases

BI is looking to the partnership with Dicerna to develop candidates for use in combination therapies and breakthrough therapies for the treatment of cardiometabolic diseases, including type 2 diabetes and various contributing factors and consequences of the disease.

Initially, the companies will be developing product candidates for a “specific disease-linked gene in the hepatocytes” for the treatment of chronic liver disease using the GalXC platform. BI has the option to add the development of compounds against a second target.

“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,” said Dicerna President and CEO Douglas M. Fambrough, Ph.D., said in a statement. “We believe that Dicerna’s GalXC technology platform is ideally suited for the development of novel RNAi therapies for NASH and other chronic liver diseases.”