The big pharma companies are looking into a treatment for NASH, a progressive fatty liver disease poised to become the leading cause of liver transplants by 2020, since there are currently no approved treatments for it.
The potential market for the NASH, known as Non-alcoholic Steatohepatitis, is forecast to be $20 billion to $35 billion with a forecast from 5 to 20 percent of the population potentially affected in the United States alone.
Probably due to these data, Raghuram Selvaraju, managing director and senior healthcare analyst at Rodman and Renshaw has called NASH “one of the hottest spaces in the healthcare sector”.
Gilead has been an pioneer, being the only large drugmaker looking into this disease and after unfortunately its most advanced anti-fibrosis candidate failed, it stroke deals with two small companies to acquire additional NASH programs.
Last year Phase II data from a Gilead-developed drug demonstrated fibrosis regression after just six months.
Allergan tried to catch up acquiring on the same day last year Tobira Therapeutics and a Akarna Therapeutics, making the drugmaker a top NASH contender.
Big drugmakers with licensing deals or options on future deals in the space include Pfizer ( with three early-stage drugs in the clinic), Novartis, Merck & Co, Johnson & Johnson and Bristol-Myers that has presented promising data for its lead NASH candidate at the big European liver meeting in Amsterdam.
The large drugmaker companies are looking to small companies (and many of them do not have yet a partnership) developing promising medicines, among these: Intercept Pharmaceuticals, Galectin Therapeutics (expecting key data in December), Genfit and Galmed Pharmaceuticals, all with a chance to be among the first to market, as well as Enanta Pharmaceuticals , Durect Corp and little-known U.K.-traded Tiziana Life Sciences with assets much earlier in development.
Enanta Chief Executive Jay Luly said companies in earlier stages of development may have an advantage over the first wave of experimental treatments as regulators’ thinking on clinical trial goals and what makes an approvable product in such a new market evolves. “When we get there, the development pathway could be not only more clearly defined, but more simplified,” Luly said. Enanta among the other companies has also the advantage of cash flow from its hepatitis C partnership with AbbVie to fund its NASH program.
Different are also the approaches taken to treat the complex disease: there are drugs targeting inflammation to prevent or reduce fibrotic scarring, others address lipid regulation to reduce liver fat or attempt to directly halt or reverse fibrosis.
There are also companies testing diabetes treatments to assess their ability to improve NASH.
Some experts said drugs that are likely to gain earliest acceptance from insurers.
Conatus Pharmaceuticals research for example focuses to target late-stage fibrosis and cirrhosis, where the risk of cancer and liver failure is highest, with the goal of preventing transplants.
Shares of this small biotechnology company based in San Diego, more than doubled when it signed a $50 million collaboration deal with Novartis last December.
Still the millions of potential patients and expense of treating advanced disease, should be an incentive for insurers to embrace medicines that target earlier stage NASH as well.