By Anne Steele
The next frontier for multibillion-dollar drug therapies is a
silent disease many people don't know they have.
Dozens of pharmaceutical companies including Gilead Sciences
Inc., Allergan PLC and Intercept Pharmaceuticals Inc. have joined
the fray to bring a treatment for nonalcoholic steatohepatitis -- a
common but often undetected fatty-liver disease -- to market.
Interest in the disease, known as NASH, has spurred at least six
deals over two years valued at $3.52 billion or more.
Overall, more than 40 drugs in mid- and late-stage trials are
targeting various aspects of the complex metabolic disorder.
NASH is the progressive form of nonalcoholic fatty-liver
disease. Fat buildup causes inflammation, cell damage and
eventually fibrosis.
The companies are lured by the promise of a large and growing
pool of patients for whom there is currently no treatment. As much
as 12% of the U.S. population is believed to have the disease,
which is linked with other metabolic conditions on the rise
including obesity and Type 2 diabetes, according to the National
Institute of Diabetes and Digestive and Kidney Diseases. NASH
generally has no symptoms early on but can lead to cirrhosis and
liver cancer.
SunTrust Robinson Humphrey's analyst Edward Nash, who has called
NASH "one of the last untapped multibillion-dollar therapeutic
areas," forecasts the space to grow to more than $5 billion in the
U.S. alone by 2028. Research and consulting firm GlobalData
estimates the NASH space across seven major markets -- the U.S.,
France, Germany, Italy, Spain, the U.K. and Japan -- is set to rise
to $25.3 billion by 2026.
As markets for diseases such as diabetes and hepatitis C become
crowded, companies are looking to NASH for their next big
break.
With so many players chasing the market, some analysts are
concerned there aren't enough patients to support multiple
treatments. A definitive diagnosis requires an invasive biopsy, and
awareness of the disease is low. At least two companies have
disclosed issues filling their drug trials.
Pricing could also come under pressure from payers that have
experience playing hepatitis C drugs against each other to win
rebate concession. A drug would need to demonstrate clear clinical
benefit over rivals to command premium pricing.
But companies say it is likely different treatments will be used
in combination to treat patients in different stages of the
disease. Some drugs attempt to reduce fat in the liver while others
aim at symptoms such as inflammation and fibrosis, or scarring of
the liver. Some drugs address combinations of these.
"This is a race," says Mark Pruzanski, chief executive of
Intercept, which has one of the drugs furthest along in
development. "But it's not a zero-sum game."
Drug-company interest got a boost in January 2014, when the NIH
halted a clinical trial of Intercept's drug obeticholic acid in
NASH patients because the drug significantly improved measures of
liver health versus a placebo. That disclosure nearly quadrupled
Intercept's market value, though it ebbed amid reports of the
drug's effects on cholesterol levels.
Results from a study released later that year eased concerns
over cholesterol and the company's stock surged again.
"Those positive results suddenly lit a fire under the field and
woke people up to the possibility that this is an untapped market,"
said Scott Friedman, liver-disease chief at the Icahn School of
Medicine at Mount Sinai Hospital in New York.
Intercept had an early advantage because its obeticholic acid,
whose brand name is Ocaliva, is already approved for another liver
disease. Mr. Pruzanski said Intercept intends to position the drug
as the first-line treatment to which others can be added.
Other companies say they have their own particular edge.
In September, Allergan snapped up Tobira Therapeutics Inc. and
Akarna Therapeutics Ltd. for $1.7 billion and $50 million,
respectively, adding a trio of NASH therapies to its pipeline. It
also is collaborating to combine one of its drugs with one of
Novartis AG's liver treatments, which is in phase 2 trials.
A Novartis spokesman said the company believes combination
therapy that treats both the metabolic and fibrotic components of
NASH will be critical.
"NASH is not a single disease for everybody, it manifests
differently," said Allergan's chief medical officer, Gavin
Corcoran. "We'd like to have different series of therapies that
have been tested together."
Gilead last year bought Nimbus Therapeutics LLC's liver-drug
program and now also has three NASH candidates in its pipeline.
Mani Subramanian, senior vice president of liver-disease
therapeutics at Gilead, says the company's program is aimed at
patients in the later stages of sickness, where the liver is
scarred and losing function.
"It is by far the biggest clinical need because it's what leads
to mortality," he said.
Mike Burgess, Bristol-Myers Squibb Co.'s head of cardiovascular,
fibrosis and immunoscience development, insists the company's phase
2 candidate has the trifecta: It reduces fat in the liver and is
anti-inflammatory and antifibrotic. He says it also addresses
issues outside of NASH, such as increasing insulin sensitivity in
patients who are diabetic.
Much will depend on identifying patients with the disease. Right
now, diagnosis is often by chance.
Diabetes and heart disease run in Robin A. Rothman's family, so
she has been careful to keep a close watch on her health. She was
diagnosed with NASH three years ago, after an episode with hives,
and her doctors initially suspected hepatitis C because her partner
had the virus.
The 45-year-old Brooklyn, N.Y. resident, who has since taken off
12 pounds, has joined Intercept's latest clinical trial. The
day-to-day is simple: one pill. But Ms. Rothman also volunteers to
do regular blood draws and provide stool samples.
"I want them to have as much information as possible to do as
much good as possible," she says.
Write to Anne Steele at Anne.Steele@wsj.com
(END) Dow Jones Newswires
June 23, 2017 07:14 ET (11:14 GMT)
Copyright (c) 2017 Dow Jones & Company, Inc.
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