- Boehringer Ingelheim GmBH and Dicerna Pharmaceuticals Inc. have entered into a research and licensing pact focused on RNA interference (RNAi) technology and its ability to treat an increasingly prevalent liver disease.
- In a deal announced Thursday, the companies will use Dicerna's GalXC platform to develop a candidate that works on an undisclosed non-alcoholic steatohepatitis (NASH) target. For its part, Boehringer has agreed to hand its new collaborator upfront, potential milestone and royalties payments worth more than $200 million combined.
- NASH is a subset of nonalcoholic fatty liver disease (NAFLD), which affects about 30% to 40% of adults in the U.S., according to the National Institute of Diabetes and Digestive and Kidney Diseases.
NASH has become one of the hottest therapeutic areas in the pharmaceutical industry, due to the high unmet medical need and the lack of actual treatments. As people around the world become more obese, the liver disease is becoming a much more frequent diagnosis. And pharma companies have been willing to pay out big bucks in recent years to enter the space.
Just in the past couple of years, Allergan plc shelled out $1.7 billion to acquire Tobira Therapeutics and its NASH drugs cenicriviroc and evogliptin; Bristol-Myers Squibb Co. dropped $100 million for an exclusive license to Nitto Denko's ND-L02-s0201 and some other small interfering RNA treatments; and Novartis AG paid Conatus Pharmaceuticals Inc. $50 million for an exclusive option to emricasan.
The NASH drug market is primed to grow from $618 million in 2016 to $25.3 billion in 2026 across the seven major pharmaceutical markets — France, Germany, Italy, Japan, Spain, the UK and the U.S.— according to a May report from research firm GlobalData.
Boehringer Ingelheim has worked to secure its seat at the NASH table as well. Following a deal with Pharmaxis, for example, the German drugmaker took hold of what's now called BI1467335, an oral inhibitor of amine oxidase, copper containing 3 (AOC3) that works to combat the inflammation that promotes NASH. In August, Boehringer Ingelheim initiated a Phase 2a study to establish the drug's proof of clinical principle as well as assess dosing and safety.
And tapping Dicerna and its GalXC technology to bring another NASH-focused drug under its belt signals another step toward Boehringer Ingelheim's goal.
"Dicerna’s GalXC technology platform uses RNAi to inhibit the expression of disease-causing genes by destroying the messenger RNAs of those genes," Boehringer Ingelheim said in a Nov. 2 statement. "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."
Dicerna, meanwhile, gains a big-name partner and the potential to bring in some big bucks. Shares in the Massachusetts biotech opened at $6 apiece on Thursday, up 11.3% from the prior day's close.