Tag Archives: icpt

2 Hotly Debated Drugs Due For FDA Decisions This Week

The FDA is due to decide whether to approve two of the most divisive drugs in the biotech industry this week, both of them crucial for the companies that make them, Sarepta Therapeutics and Intercept Pharmaceuticals. Thursday is the deadline for eteplirsen, Sarepta Therapeutics ‘ ( SRPT ) treatment for Duchenne muscular dystrophy (DMD). Just getting to the filing took years of negotiation between Sarepta and the FDA, and May 26 is three months later than the decision was originally supposed to be made. And after all that, Wall Street is placing low odds on the drug’s approval. That’s based mainly on last month’s advisory committee meeting on eteplirsen, which voted 7 to 3 (with 3 abstentions) that the drug’s effectiveness wasn’t adequately proven by Sarepta’s tiny clinical trial. Sarepta stock hit a four-year low of 8, on April 26, as multiple analysts downgraded it on the assumption the drug will be rejected. Nonetheless, DMD is a devastating disease without a current treatment, and the families of the children affected have turned into a formidable lobby to get something on the market — especially after BioMarin Pharmaceutical ‘s ( BMRN ) rival drug Kyndrisa was rejected in January. Some observers say the families’ desperation might be enough to move the agency. Oppenheimer analyst Christopher Marai on May 2 upgraded Sarepta on the grounds that, in the past, the FDA has been more flexible in such cases of unmet need. He also pointed out that the stock was now cheap. Sarepta stock has remained volatile but has steadied somewhat the past couple of weeks. On Friday, shares jumped 8.8%, to 19.15. Intercept’s Liver-Disease Treatment Expected To Get OK A day after the Sarepta decision, Intercept Pharmaceuticals ‘ ( ICPT ) liver-disease treatment Ocaliva is due for a verdict. Ocaliva’s advisory committee vote last month went the opposite way of eteplirsen’s, with the panel unanimously supporting approval for the drug, which treats the rare disease primary biliary cholangitis (PBC). As a result, Wall Street generally expects approval. Bears, however, are still growling about Ocaliva’s safety, which they say could limit the drug’s label. The day after the panel vote, Morgan Stanley analyst Andrew Berens drove down Intercept’s stock with the theory that the FDA would discourage use in patients with advanced cirrhosis. This might affect not only the relatively small PBC market, but also the much, much bigger possibilities for Ocaliva in non-alcoholic steatohepatitis (NASH). Intercept is testing the drug in NASH, and approval wouldn’t be for a couple of years, but it’s a major part of the stock’s bull thesis, as analysts see annual sales in the multi-billions. Intercept stock has been on a downtrend this month, but it rose 2.4% this past week. On Friday, shares closed up 2.2%, at 134.28.

3 Biotech Stocks Buoyed By Potential Blockbuster Drugs

Sentiment on biotech stocks overall ebbs and flows, but biotech companies can carve very individual paths. Drug company investors look to product pipelines to spot potential growth, especially for scientifically novel assets that can stand out from the pack. These three biotech stocks have outperformed the group in the past few months thanks to potential blockbuster drugs. • Juno Therapeutics ( JUNO ) is up 68% since its early February all-time low — which was also the IBD Medical-Biomed/Biotech group’s nearly two-year low point — as investors have regained confidence in its cancer-fighting pipeline. Juno is working in the cutting-edge field of cell therapy, in which patients’ own cells are re-engineered to help their immune systems kill cancer cells, in this case the chimeric antigen receptor (CAR) T cells. Juno’s leading candidate, JCAR015, is in midstage testing for acute lymphoblastic leukemia, for which it showed stunning success rates in earlier trials. Last month, its partner Celgene ( CELG ) said it would exercise its option to commercialize JCAR015 and two similar candidates outside of North America and China, under a 10-year, $1 billion partnership that the two companies inked last summer. Also last month, Juno showed off some of its earlier-stage CAR-T candidates at the annual meeting of the American Association for Cancer Research, targeting different receptors in other types of blood cancer. When the abstracts for the meeting were released in March, Leerink analyst Michael Schmidt wrote that Juno’s stock should be supported by “clinical data readouts supporting Juno’s business strategy to develop differentiated, potentially best-in-class CAR-T products that could generate translational insights in hematologic and solid tumor indications.” • BioMarin Pharmaceutical ( BMRN ) is up 29% since its early February 20-month low as it recovered from the FDA’s rejection of its muscular-dystrophy drug Kyndrisa in January. At its R&D day last month, BioMarin rolled out strong early data for two pipeline candidates, one for hemophilia and one for achondroplasia, a form of dwarfism. The hemophilia data brought particular attention, as it was the first-ever evidence on gene therapy for the disease in humans. After the therapy, BMN 270, was delivered once to eight patients, all of their conditions went from severe to mild-to-moderate. This was far better than expected. In a video recorded for clients a couple of weeks later, Evercore ISI analyst John Scotti described the mood at BioMarin’s event: “When they started presenting (the data), jaws just went to the floor. There was an audible gasp in the room.” The market for hemophilia A, the most common type, runs at $4 billion a year. Analysts say a successful gene therapy could massively disrupt the market of clotting factors that have to be infused weekly at least. • Intercept Pharmaceuticals ( ICPT ) is up 52% from its 28-month low in February, after its lead drug Ocaliva passed a major hurdle to market last month. An FDA advisory committee voted to grant it accelerated approval for a liver condition called primary biliary cholangitis (PBC), which the agency has until May 29 to decide on. While the PBC market is expected to bring in a healthy revenue stream for Intercept, the potential blockbuster indication is in nonalcoholic steatohepatitis (NASH), a liver-scarring disease that affects millions of Americans but has no treatment. Intercept is conducting a large phase-three trial of Ocaliva in the disease, with a launch not expected for several years. But the FDA panel vote did imply that the experts see the benefits as outweighing the drug’s known safety issues. Ocaliva’s potential has driven buyout speculation around Intercept stock, which has also helped to push up shares. A particular burst of this came on Feb. 12 when anonymous sources suggested that the company was exploring a sale, though nothing’s yet come of it.

Intercept Pharma Drops On Downgrade, Despite FDA Panel’s Yes Vote

Shares of Intercept Pharmaceuticals ( ICPT ) were falling Friday after Morgan Stanley downgraded the stock, saying that a positive FDA panel vote nonetheless pointed toward a restrictive label for its lead drug. Trading in Intercept stock was halted all day Thursday as the advisory committee discussed obeticholic acid, now branded Ocaliva, eventually resulting in a 17-to-0 vote in favor of approving the drug to treat a rare liver disease called primary biliary cholangitis (PBC). The FDA will make a final decision on the drug, and what recommendations will be on its label, by May 29. Morgan Stanley analyst Andrew Berens wrote in a research note that issues raised during the panel discussion could limit the potential market. “The panel unanimously recommended to approve the drug based on the surrogate endpoint used in the phase 3 trial, but failed to endorse usage in advanced PBC patients given the lack of data supporting efficacy, as well as safety concerns,” Berens wrote as he downgraded Intercept stock to underweight from equal weight and lowered his price target to 80 from 100. “According to external consultants, the primary unmet medical need in this patient population is in patients with advanced or aggressive disease, so this lack of an endorsement by the panel has important commercial implications in our opinion, especially if the label is similarly restrictive,” Berens wrote. Berens added that the panel also expressed reservations about using the drug in patients with advanced cirrhosis, or scarring of the liver, due to signs of liver toxicity at high doses. He wrote that this could have implications for Ocaliva’s much-anticipated future launch in non-alcoholic steatohepatitis (NASH), a market that could potentially bring in billions. “We had previously assumed usage of Ocaliva in up to 20% of cirrhotic patients,” Berens wrote. “Given the concerns voiced by the clinicians and the FDA, we have decreased our penetration into NASH patients with cirrhosis to 5% at peak.” Intercept stock, which rose more than 8% Wednesday to a nearly four-month high, fell by as much as 10% in the stock market today . Shares were down 5.5% midday Friday, near 155. Other analysts generally stuck by their ratings on the stock. RBC Capital Markets analyst Michael Yee wrote that the possible PBC restrictions were in line with his below-consensus sales estimates for that indication, but he added that the company could still be an interesting buyout target for a big pharma name like AstraZeneca ( AZN ) or Sanofi ( SNY ). Credit Suisse analyst Alethia Young still says the PBC launch could beat expectations and wrote that the severe cases may not be that much of the market. “We think perhaps at worst the severe population is 15% of patients,” she wrote in her research note affirming an outperform rating. “Based on the super-group study, Intercept thinks 12% may be a high watermark.”