The record launch of Gilead‘s hepatitis C drug Sovaldi turned up the spotlight on an already hot area of drug development.
On Friday, the annual meeting of the American Association for the Study of Liver Diseases, or AASLD, kicks off in Boston, where researchers, companies and Wall Street are converging to gain a sense of where the competition stands in bringing potentially multibillion dollar drugs to market.
While drugs for hepatitis C are the headliners, those for two other liver diseases, hepatitis B and NASH—nonalcoholic steatohepatitis—have been gaining investor attention. Here’s what to watch at the conference.
“I think we will see a lot of big-cap volatility, particularly if you look at Gilead, Merck and AbbVie,” said Brian Skorney, an analyst with Robert W. Baird. “Those three companies have a lot of information, both that they will be presenting for their internal programs, but I think they also very much affect each other.”
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Of chief interest is a Merck study called C-SWIFT that evaluates its two-drug combination with Gilead’s Sovaldi, aiming to cut treatment times down to as little as four weeks.
“Merck’s recent tone suggests the four-week regimen may not produce competitive SVRs [sustained viral responses], which if confirmed we believe would help reduce a potential overhang on Gilead,” Brian Abrahams, an analyst with Wells Fargo, wrote in a note to investors in October.
SVR is a measure of how well a drug works to cure a disease, determined by undetectable levels of the virus after a certain period of time.
In addition to the big names, analysts are also watching Achillion, a perennial takeover target with hepatitis C drugs in development. It’s unlikely the company will report key data on the drug being most watched, one in a class called nucleotides, said Deutsche Bank analyst Alethia Young. But Achillion will report data on another drug in combination with Gilead’s Sovaldi.
The story in hepatitis C has shifted from a revolution in treatment to how drugs such as Sovaldi and Harvoni do in the real world, and whether treatment can be shortened from 12 or eight weeks, Young said.
“It’s not like the old AASLDs, where everybody was seeing something completely revolutionary in hepatitis C,” she said.
Those revolutions haven’t come yet for hepatitis B and NASH, Young noted.
The commercial opportunity in both of those diseases is still uncertain, though the patient populations are as large as, or larger than, hepatitis C, which affects as many as 150 million people worldwide, according to the World Health Organization.
The overall market for hepatitis C drugs is likely to reach $15 billion to $20 billion, said Michael Yee, an analyst with RBC Capital Markets.
Companies to watch in hepatitis B include Arrowhead Research, whose shares took a massive tumble last month as data on the drug’s efficacy disappointed investors.
“I would argue it’s really too early to make a judgment call there, because we’re clearly going to need to see some fairly long duration treatment regimens at this point to see what’s truly going to happen to the hepatitis B virus,” Baird’s Skorney said.
Arrowhead is likely to present some more detailed data, though key questions from investors center around timelines for testing higher doses of the drug, Deutsche Bank’s Young said.
Investors are also likely to be curious about private company Replicor, which has “shown some hepatitis B data that I can only characterize as really fantastic,” Skorney said. “Most investors are scratching their heads if it can even be real,” he said, noting the company has only a handful of employees and very little capital, and ran its study in a developing country.
Though development of new drugs across hepatitis B is early, the opportunity is large—it’s the most common cause of liver disease. 5,000 patients die each year in the U.S. from liver failure, according to Cowen, and cure rates are very low. Skorney said cure rates are 10 percent or less, compared with more than 90 percent with new regimens in hepatitis C.
The number of patients with this chronic liver disease, which can lead to permanent liver scarring, eclipses that of hepatitis C in the U.S., RBC’s Yee said. While a significant number aren’t diagnosed, “we believe, based on survey work, that there are hundreds of thousands of patients with diagnosed NASH,” and that the global market for NASH drugs may reach $5 billion to $10 billion, he said. The disease’s prevalence may be growing along with obesity, according to the National Institutes of Health.
Companies working in NASH include Gilead, Intercept and Genfit. Intercept shares tumbled Friday after a study of its drug obeticholic acid, or OCA, was published in The Lancet. While analysts were largely unsurprised by the data, which showed the drug met study goals, they noted concern over a side effect of itchiness and the drug’s effect on LDL, or bad, cholesterol.
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The “overall publication is fine describing how it has positive effects on histology of NASH patients, but perhaps not surprising, in our view, given a need to be balanced for peer physicians, has some cautious/conservative comments and suggests a general need for more long-term efficacy and safety data, which is known,” Yee wrote in a research note.
But the AASLD meeting may perhaps be most helpful for gaining a sense of regulatory requirements—what the Food and Drug Administration will look for in order to approve a drug, Skorney said.
“Liver diseases on the whole, beyond hepatitis C, are a very significant commercial opportunity,” Skorney said. “While hepatitis C, I would argue, remains the focus because it’s so commercially near-term and we’re seeing massive sales figures, people are looking to the future to say, ‘Hey, is there anything else beyond hepatitis C?'”