Biotech Sarepta Therapeutics ( SRPT ) soared in early trading Wednesday after the Food and Drug Administration delayed making a decision on whether to approve its muscular dystrophy drug, giving new hope for a product that Wall Street had generally given low odds. The FDA’s self-imposed deadline had been Thursday, which itself was pushed back three months from the original decision date. Sarepta’s statement didn’t mention a new deadline, just saying that the reviewers “will not be able to complete their work” by the current deadline. Sarepta’s drug eteplirsen, for a subgroup of patients with Duchenne muscular dystrophy (DMD), had always had a tough case to make based on a clinical trial with only 12 subjects and no internal control group. The odds of success looked even lower when the majority of an FDA advisory committee last month voted not to recommend approval. However, families of patients with DMD, who generally don’t live past the age of 30, have been lobbying hard for some treatment to come on the market, since currently none are available. Analyst Simos Simeonidis of RBC Capital Markets suspects that this is the reason for the delay, but he still gives the drug only a 30% chance of approval. “Despite the fact that today’s news slightly increases the chances of an approval, we have a hard time imagining the agency setting the precedent of approving a drug with such a limited dataset,” Simeonidis wrote in a research note. “On the other hand, we cannot overlook the tremendous pressure being put on the FDA, and it is very difficult to gauge what may happen behind the scenes at the agency at the last minute.” Sarepta stock shot up 20% soon after the opening bell on the stock market today , near 22.
Big Pharma AbbVie ( ABBV ) said Tuesday that a recently licensed drug candidate succeeded in a phase-two trial of Crohn’s disease, as the company seeks to refresh its blockbuster immunology franchise. In the late morning at the Digestive Disease Week conference in San Diego, AbbVie’s researchers presented results from an ongoing phase-two study of risankizumab, a drug AbbVie licensed from Boehringer Ingelheim during the first quarter, in Crohn’s disease. They said that after 12 weeks of treatment, 24% to 37% of patients were in remission — depending on the dose — compared with just 15% in the placebo group. The treated group also had fewer adverse events than the placebo group. Risankizumab is one of a newer group of drugs targeting interleukins, small proteins in cells associated with inflammation, among the proteins is TNF, or tumor necrosis factor. “These results are particularly encouraging because of the difficult-to-treat population within the study,” researcher Brian Feagan said in a statement. “Our patients had endoscopically confirmed moderate or more severe disease activity at study entry and the majority had previously failed treatment with one or more TNF antagonists.” The most popular TNF blocker happens to be Humira, AbbVie’s flagship drug treating Crohn’s disease as well as other immunological conditions like rheumatoid arthritis and psoriasis. Humira’s patents are under fire , however, and last week AbbVie’s stock tumbled when the patent board agreed to review a challenge to a methods patent brought by Coherus BioSciences ( CHRS ). Other drugs targeting interleukins include Novartis ‘ ( NVS ) Cosentyx, launched early last year for psoriasis, as well as Eli Lilly ‘s ( LLY ) Taltz, approved for psoriasis in March. Those drugs target interleukin (IL) 17, while risankizumab targets IL-23 through a novel mechanism of action. “The efficacy of this drug looks to be as good (as), or perhaps even better than IL-17,” Evercore ISI analyst John Scotti said in a video for clients recorded May 3. “And remember, Cosentyx peak sales in consensus right now are about $4 billion.” AbbVie stock was up 2.5% in afternoon trading on the stock market today , near 61, and shares in the past week have found support at the 50-day moving average line.
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.