Fatty Liver Disease: The Next Pharma Gold Mine?

Drug giants eye potential $35 billion market

With cash in their pockets, big pharma companies are on the prowl for promising medications being developed at smaller firms to treat patients with nonalcoholic steatohepatitis (NASH), a progressive fatty liver disease that is on its way to becoming the number one cause of liver transplants by 2020, according to a Reuters report. The NASH market is expected to reach between $20 billion and $35 billion as populations pay the price for fatty diets. There are no approved treatments for the disease.

Pfizer currently has three early-stage drugs in the clinic aiming to block or reverse fat accumulation in the liver, according to the report. Bristol-Myers Squibb is also looking for additional compounds to enhance its internally developed NASH drugs.

Up to 15 million people have NASH in the United States, making the disease and its consequences––advanced fibrosis and liver-destroying cirrhosis––prime targets for new therapies.

A few years ago, Gilead Sciences was the lone large drug maker talking about NASH, the report notes. The company was undeterred after its most advanced antifibrosis candidate failed, making deals with two small companies to acquire additional NASH programs.

Allergan became a top NASH contender with its acquisition of Tobira Therapeutics and a deal with Akarna Therapeutics last year. Other big drug makers with licensing deals or options on future deals in the NASH arena include Novartis, Merck, Bristol-Myers Squibb, and Johnson & Johnson, Reuters says.

Many small companies developing NASH drugs do not have partners. They include Intercept Pharmaceuticals, Galectin Therapeutics, Genfit, Galmed Pharmaceuticals, Enanta Pharmaceuticals, Durect Corp, and Tiziana Life Sciences––all with a chance to be among the first to reach the marketplace with a NASH medication.

Drug makers are taking a wide range of approaches to treating the complex disease, given the multiple health issues that contribute to liver damage, such as heart disease and diabetes. Some investigational drugs target inflammation to prevent or reduce fibrotic scarring, and some address lipid regulation to reduce liver fat, while others attempt to directly halt or reverse fibrosis. Some companies are also testing diabetes treatments to assess their ability to improve NASH.

Reuters estimates that many of the NASH drugs in development are two to five years away from reaching the market, if they get that far.

In related news, NASH front-runner Genfit has announced that it will delay a phase 3 trial of its NASH candidate elafibranor by four to six months in patients with liver scarring. The double-blind, placebo-controlled study, which initiated enrollment approximately one year ago, will include about 2,000 patients at 250 treatment centers worldwide. The patients will receive once-daily elafibranor 120 mg or placebo for 72 weeks. A few years ago, elafibranor failed to beat placebo in a midstage study.

Sources: Reuters; April 24, 2017; and Fierce Biotech; April 24, 2017.