November 7, 2014
By Riley McDermid, BioSpace.com Breaking News Staff
Intercept Pharmaceuticals Inc. ’s closely watched experimental liver drug has been able tor reduce liver damage but needs more safety studies because it significantly raised bad cholesterol levels in patients treated in a mid-stage trial, said the company Friday.
Intercept’s hotly anticipated drug, obeticholic acid, for nonalcoholic steatohepatitis (NASH), also caused itching so severe that it interfered with patient’s basic movements, said researchers.
News of the results pushed Intercept’s share price down more than 25 percent in early morning trading Friday. The company’s market value had been bulked up to $5.28 billion as Wall Street held out high hopes for the drug, but its side effects have chilled some of that anticipation.
Chief Executive Officer Mark Pruzanski said on a conference call Friday morning that as a result, the company will attempt smaller doses of the drug in upcoming trials to see if it reduced the itching side effect.
The results were published in British medical journal The Lancet and outlined a study treating 283 patients, almost half of whom saw an improvement. The data found that 45 percent improved, as opposed to only 21 percent on a placebo, while liver scarring (fibrosis) improved in 35 percent compared to 19 percent on a placebo.
Intercept said the effect of the drug was similar to therapies received from the treatment of increased vitamin E and diabetes drug pioglitazone. Researchers warned new studies “will need to address the consequences of these changes on cardiovascular outcomes” while the therapy’s “long-term safety requires further clarification.”
More concerning was a metric that saw 33 of 141 patients beset by the itching, with 21 saying it was “intense or widespread” and three who found it so distracting they could not conduct daily activities.
“If it’s very severe, it’s debilitating -- you can’t sleep and you can’t function,” Vijay Shah, chairman of gastroenterology and hepatology at the Mayo Clinic in Rochester, Minnesota, told Bloomberg. Shah said the elevated cholesterol and itching were “some reasons to pause and weigh the pros and cons.”Patients with NASH suffer from an accumulation of fat and inflammation around the liver that can damage the organ over time. The National Institutes of Health estimate 6 million people in the U.S. suffer from the disease, according to Bloomberg, and drugs for its treatment are rare.
Wall Street will be closely watching upcoming studies of the drug because today’s announcement could affect the flagship drug, dubbed OCA, which has held a lot of promise for investors because it would treat patients who are overweight or diabetic, a hugely lucrative market in the domestic U.S.
