-=Intercept Pharma (ICPT) : 'positive' top-line results from Phase 3 REGENERATE study of obeticholic acid in patients with liver fibrosis due to NASH

Intercept Pharmaceuticals (ICPT) stock popped Tuesday after its liver disease treatment succeeded in a Phase 3 study — a week after Gilead Sciences (GILD) failed in the same area.

Both companies are targeting nonalcoholic steatohepatitis, a liver disease known as NASH.

Early Tuesday, Intercept said its drug, Ocaliva, improved liver damage called fibrosis without worsening the symptoms of NASH after 18 months of treatment.

"We are thrilled to report the first positive registrational Phase 3 study results in patients with NASH, a devastating disease that is on track to become a leading cause of liver transplant in coming years," Intercept Chief Executive Mark Pruzanski said in a written statement.

Based on the results, Intercept plans to file for approval of Ocaliva in NASH treatment in the U.S. and Europe in the second half of 2019.

NASH Treatment Succeeds In Stage 2-3 Patients
Intercept's test included 931 patients with stage 2 or state 3 liver fibrosis due to NASH. The worst level of fibrosis is stage 4.

Researchers tested two doses of Ocaliva. At the low dose, 17.6% of patients showed fibrosis improvement without worsening NASH. The higher dose netted 23.1% of patients with the same result. Just 11.9% of the placebo group had the same result.

The biotech also tested Ocaliva as a NASH treatment. After 18 months, 11.2% and 11.7% of the Ocaliva patients showed no symptoms of NASH and hadn't experienced worsening in fibrosis. Just 8% of placebo patients had the same result.

The NASH treatment results weren't statistically significant, Intercept said. But the Food and Drug Administration only required the study to meet one of its goals to be successful.

Gilead is also testing a NASH treatment in patients with stage 3 fibrosis. Last week, the drug failed in a late-stage study in patients with stage 4 fibrosis.

=Long trade : ICPT +10% (4/18)

• Apr. 3:  vol. 1.02M;  $64

This week's biggest % winners & losers : Sept 18 - 22, 17 (wk 38)

The following are this week's top 20 percentage gainers and top 20 percentage losers, categorized by sectors (over $300 mln market cap and 100K average daily volume).

This week's top 20 % gainers

• Healthcare: ALNY (113.8 +49.32%), QDEL (43.1 +20.36%), LJPC (37.21 +18.2%), AXON (24.99 +17.1%), MCRB (16.75 +14.18%)
• Materials: CCC (21.4 +60.9%)
• Industrials: OA (132.64 +20.54%), AJRD (33.89 +19.63%), MTOR (26.4 +16.92%), CUB (51.6 +16.35%), HTZ (23.55 +14.49%)
• Information Technology: SSNI (16.18 +24.46%)
• Energy: SDRL (0.39 +64.84%), PGH (0.94 +50.62%), FMSA (4.19 +21.45%), CRC (10.18 +19.62%), EMES (9.17 +15.64%), TGS (20 +14.88%), CVE (10.28 +13.72%)
• Consumer Staples: REV (19.9 +23.99%)

This week's top 20 % losers

• Healthcare: ICPT (61.59 -35.09%), SUPN (39 -20.49%), KND (6.25 -13.79%), MNKD (1.98 -13.54%), IONS (51.32 -13.35%), NLNK (10.99 -13.26%), AERI (50.95 -12.61%)
• Materials: MUX (2.06 -20.16%), SBGL (4.69 -15.04%), CMP (60.1 -13.71%), HMY (1.86 -13.08%), KGC (4.25 -11.83%)
• Industrials: RUN (5.59 -16.82%), HDSN (7.94 -13.32%)
• Consumer Discretionary: BBBY (22.51 -21.49%), PIR (4.15 -20.19%), MMYT (28.45 -16.2%), RCII (10.8 -13.88%)
• Information Technology: SNCR (9.2 -42.89%), GOGO (12.45 -11.76%)

Intercept Pharma (ICPT): FDA warns doctors after 19 deaths on Intercept liver drug

• Some patients died after taking excessive doses, agency says
• Company previously reported 10 deaths connected to Ocaliva

  

• 2nd day

Nineteen patients died after taking a liver-disease drug from Intercept Pharmaceuticals Inc., the U.S. Food and Drug Administration said, warning doctors about risks from a product that the company is seeking to make into a blockbuster.

While the cause of death wasn’t available in most cases, seven of the patients who died were taking the drug, called Ocaliva, more frequently than is recommended, the agency said in a safety announcement on Thursday.

The drug “is being incorrectly dosed in some patients with moderate to severe decreases in liver function, resulting in an increased risk of serious liver injury and death,” the FDA said in its warning. But it also said that the medication may be associated with liver damage in some patients with mild disease receiving the correct dose.

Intercept declined to immediately comment as it reviewed the FDA’s statement. Shares of the drugmaker sank 9.2 percent to $89.10 at 12:21 p.m. in New York.

The deaths could lead to restrictions on how Ocaliva is prescribed and additional warnings on the drug’s label, which doesn’t currently list death as a risk. The company would face a potential major safety issue as it tries to expand the drug’s use to millions of patients with other conditions.

Joseph Schwartz, an analyst at Leerink Partners, said that the new information “could tilt the FDA more toward a black box warning” calling attention to life-threatening risks.

Earlier this month, Intercept said it had reviewed 10 deaths of patients who had taken the drug. The company warned doctors that Ocaliva can cause injuries, organ failure, or death if it’s not used exactly as intended in patients with primary biliary cholangitis, a relatively rare liver condition for which the drug was approved last year.

About 15,000 prescriptions for Ocaliva have been written since it was introduced, according to data from Symphony Health Solutions compiled by Bloomberg Intelligence. The drug is Intercept’s only commercial product and was approved in the U.S. in May last year. It brought in $30.4 million in sales last quarter.

Rival treatments in testing at other drugmakers could threaten Ocaliva’s market potential if they don’t have the same risks. Intercept is in the final stages of testing Ocaliva for nonalcoholic steatohepatitis, or NASH, which is associated with being overweight.

Three percent to 12 percent of Americans have NASH, according to the U.S. National Institutes of Health -- a huge potential market. Analysts have projected that the drug will become a blockbuster, with more than $1 billion in sales by 2021, though the disclosure of deaths may change that.

Intercept Pharmaceuticals (ICPT) : issues warning regarding Ocaliva

** charts before warning**

  

** charts after warning**

  

Intercept Pharmaceuticals (ICPT) dove Tuesday after the firm issued a letter warning physicians against overdosing some patients with Ocaliva, which has been tied to liver injuries and death.

In early trading on the stock market today, Intercept toppled 18%, near 93.10. Shares hit their lowest point in 19 months. More broadly biotech stocks dipped 1.4%.

The letter warned physicians about post-marketing cases of liver injury, liver decompensation, liver failure and deaths in patients with liver impairment and a rare disease known as primary biliary cholangitis who receive Ocaliva more frequently than recommended.

"In addition, serious liver adverse events have been reported in patients initiating therapy without cirrhosis or with mild liver impairment," Intercept said in its letter. "Liver-related adverse events have occurred both early in treatment and after months of treatment."

Current Ocaliva dosing recommendations are for 5 milligrams once a week in primary biliary cholangitis patients with liver impairment. Primary biliary cholangitis is a chronic liver disease resulting from the progressive destruction of bile ducts in the liver.

The warning could weigh on Ocaliva sales and help eventual sales of seladelpar, a treatment from CymaBay Therapeutics (CBAY) that is being investigated in primary biliary cholangitis, says Leerink analyst Joseph Schwartz. Seladelpar has a different mechanism and better safety profile.

"Further down the road, if others such as etrasimod (from Arena Pharmaceuticals (ARNA)) can show competitive (effectiveness) and safety profile, Ocaliva could face competition on multiple fronts, thereby increasing the important of Ocaliva's prospects in nonalcoholic steatohepatitis," he said.

Schwartz reiterated outperform ratings on CymaBay and Arena stocks. He rates Intercept at market perform.

He notes the warnings regarding liver injury and death were included on Ocaliva's label for some patients with liver impairment. These issues were also raised at the advisory committee meeting. Still, the safety notice is a headwind for Ocaliva's base business, he said in a note to clients.

"Intercept has enjoyed a robust launch trajectory to date," he said. "In the second quarter, however, management's more tempered expectation for the remainder of 2017 increased investor caution and the stock has accordingly been weak."

He added: "Today's letter/disclosure, in our view, will likely add another layer of caution as physicians may find Ocaliva dosing more cumbersome and patients more reluctant to initiate therapy."