Boehringer Ingelheim has put up $201 million (€173 million) to work with Dicerna Pharmaceuticals on RNAi treatments for NASH and other chronic liver diseases. The goal is to use Dicerna’s GalXC technology to silence previously inaccessible drug targets, thereby restoring liver function.
Germany’s Boehringer is paying an upfront fee of undisclosed size and committing to a package of milestones and R&D funding that could theoretically top $200 million to work with Dicerna. In return, Boehringer gets to add Dicerna’s RNAi technology platform to the toolkit it is using to treat cardiometabolic diseases.
Dicerna designed GalXC to enable the subcutaneous administration of RNAi therapeutics, without using formulation components such as lipid nanoparticles to aid delivery. Once in the bloodstream, the GalXC molecules travel to the liver, where they enter hepatocyte cells and act on their RNAi machinery.
Neither Boehringer nor Dicerna have fleshed out exactly how they will apply this approach to the treatment of NASH and other chronic diseases. Most importantly, the companies have yet to say which target they are going after.
Other companies have also tried to treat NASH and its symptoms with RNA-based approaches. AstraZeneca and Regulus Therapeutics took their microRNA AZD4076 as far as phase 2, only to can the program earlier this year.
Bristol-Myers Squibb bought its way into an overlapping area one year ago when it struck a deal for Nitto Denko’s phase 1b targeted siRNA lipid nanoparticle ND-L02-s0201. That drug is designed to target heat shock protein 47.
Boehringer’s decision to explore the use of RNAi to treat NASH comes two years after it moved into the indication through a deal with Pharmaxis. That deal gave Boehringer the rights to a drug designed to block leukocyte adhesion and tissue infiltration during inflammation in return for $31 million upfront and up to $222 million in milestones.
The Dicerna program, while less advanced than the now-phase 2a BI 1467335, gives Boehringer a different, potentially more effective way of treating the growing population of NASH patients. Those patients are positioned to gain access to a barrage of new treatment options over the coming years if front-runners Genfit and Intercept and a chasing pack that includes Allergan and Gilead can get their drugs over the finish line.