Liver Transplants For Non-Alcoholics Spurs Drug Search.

http://www.bloomberg.com/news/2014-11-07/liver-transplants-for-non-alcoholics-spurs-drug-search.html

Next to Alice Gorman’s bed is a small suitcase packed with clothes and a fully charged mobile phone she carries wherever she goes. The news she’s waiting for: a donated liver that matches her blood type.

Gorman isn’t an alcoholic, an assumption nurses and emergency room doctors have made over the years when the 51-year-old Scottish woman tells them she has fatty liver disease. Her condition is non-alcoholic steatohepatitis, or NASH, which is primarily caused by obesity and diabetes, and can go undetected for decades.

Awareness of fatty liver disease that isn’t caused by drinking is scant, even as it affects a fifth of the global population and up to 70 percent of people with type 2 diabetes. NASH is an advanced form that can lead to cirrhosis, where the liver hardens and stops working properly. For Gorman, that meant a near-death episode of vomiting blood, trips to emergency rooms and a decision by doctors in September that she needs a new liver.

“I was quite shell-shocked,” Gorman, a manager at a telecommunications company, said in an interview. “But I’ve come to terms with it. My bags are packed, and I’m just waiting for the phone call.”

NASH is on track to become the most frequent cause of liver transplantation by 2025, as rates of obesity and diabetes rise globally, according to Bloomberg Intelligence. Biotechnology companies such as Intercept Pharmaceuticals Inc. (ICPT), Genfit and Gilead Sciences Inc. (GILD) are racing to develop the first treatments for NASH, an untapped market that Deutsche Bank AG says may reach $40 billion globally.

Intercept Study

Intercept’s publication today of a mid-stage study of its experimental NASH drug is the “most anticipated presentation in biotech for the year,” according to Andrew Berens, an analyst at FBR Capital Markets & Co. in New York.

Patients who took Intercept’s obeticholic acid showed an improvement in fibrosis, or scarring of the liver, which suggests the drug may help prevent progression to cirrhosis, according to a paper published in the Lancet journal.

Even so, the treatment elevated levels of bad cholesterol and its “long-term safety requires further clarification,” the researchers said. The benefit to the liver from Intercept’s therapy was similar to that from treatment with vitamin E and pioglitazone, a diabetes drug.

Companies including Genfit and Galectin Therapeutics Inc. (GALT) will give presentations on NASH therapies at the American Association for the Study of Liver Diseases annual meeting, which starts today in Boston.

Investor Interest

Investor expectations are soaring for NASH treatments. New York-based Intercept’s shares more than tripled on Jan. 9 on promising trial results for its drug. The shares slipped as much as 13 percent in late trading yesterday after data from the latest study were released.

Genfit (GNFT)’s stock has more than quadrupled this year in Paris trading, while Galectin has dropped about 38 percent in New York on investor doubts about its trial results.

As no drugs are available for NASH, the standard therapies are better diet, exercise and avoiding alcohol.

Awareness of the risks of the disease, until recently dismissed even by diabetes specialists, is gradually rising among primary doctors, Forrest said. As treatments for diabetes and associated ailments have improved, patients are living longer, allowing more time for liver conditions to appear, he said.

“It’s about people who don’t eat healthily and eat too much fat. Lifestyle has a lot to do with it.”

Surgeons perform a liver transplant. Awareness of fatty liver disease that isn’t caused by drinking is scant, even as it affects a fifth of the global population and up to 70 percent of people with type 2 diabetes.