Tag Archives: biotech

BioMarin Gene Therapy For Hemophilia Scores In Trial; Stock Jumps

Rare-disease specialist BioMarin Pharmaceutical ( BMRN ) was trading up sharply Wednesday after it reported strong early-stage trial results for its gene therapy for hemophilia. BioMarin delivered a single dose of its candidate BMN 270 to eight patients with severe hemophilia A — the most common variety of the disease, which makes sufferers’ blood unable to clot. Six of those were on the highest dose, and all of those “improved from severe to either moderate, mild or normal range in terms of factor levels based on World Federation of Hemophilia criteria,” according to BioMarin’s press release. BioMarin is already preparing a large phase-three trial of BMN 270 in hopes of gaining FDA approval as quickly as possible. If successful, it could be a paradigm shift in hemophilia treatment. Currently, the only treatment is regular infusions of natural or recombinant blood-derived products to replace the missing clotting factors. A gene therapy, by attacking the disease’s origin in a defective X chromosome, could provide improvement for much longer, although since BioMarin’s trial only followed up after 16 weeks, it’s unclear just how long it is effective. Currently, hemophilia treatment is dominated by Baxalta ( BXLT ) — which is in the process of being acquired by Shire ( SHPG ) — and Biogen ( BIIB ). Both companies are working on their own gene therapies. Pfizer ( PFE ) holds the dominant position in the smaller hemophilia B market. RBC Capital Markets estimates that the hemophilia A market alone is about $4 billion a year. BioMarin stock was up 5% in early trading on the stock market today , near 90.50, and touched a three-month intraday high. Since last summer, the stock has been hit not only by the larger biotech sell-off, but also by the FDA’s rejection in January of its muscular dystrophy drug Kyndrisa, as well as mixed late-stage trial results last month on a treatment for another rare disease, called phenylketonuria. Currently its IBD Relative Strength Rating is a dismal 15, meaning the stock has performed among the lowest 15% of all stocks over the past 12 months. “While the data presented today were small numbers and of relatively limited follow-up … the data were encouraging (though looks like significant variability from patient to patient) and (1) suggest a path forward for BMN 270 in hemophilia A (and potentially for hemo A gene therapy in general) and (2) set a reasonably high bar for competition that will follow,” wrote Evercore ISI analyst Mark Schoenebaum in an email to clients. Image provided by Shutterstock .

Medivation Rises On Cancer Drug Data, AstraZeneca Buyout Rumor

Shares of drugmaker Medivation ( MDVN ) hit an eight-month high Monday after it reported successful early-stage cancer data as buyout rumors continued to circle the company. On Sunday at the annual meeting of the American Association for Cancer Research, Medivation said that its drug candidate talazoparib, combined with low-dose chemotherapy, produced a clinical benefit in 23 of 40 patients with heavily pretreated cancers. The company noted that four of seven patients with ovarian cancer showed objective responses, or tumor shrinkage. At the same time, the Times of London reported that big British pharma AstraZeneca ( AZN ) is said to be preparing a bid for Medivation , which currently holds a stellar IBD Composite Rating of 97 thanks to strong growth of its sole marketed product, prostate-cancer drug Xtandi. Late last month, Medivation reportedly hired bankers to deal with buyout attempts, and last week it was said to have rebuffed a bid from Sanofi ( SNY ). Medivation stock rose 4%, above 53, near midday trading on the stock market today . It hit 54.55 intraday. The last time it cracked 54 was back in early August, before biotech stocks crashed. IBD’s Take: How healthy is Medivation’s stock, and how does it stack up vs. rivals? Stifel analyst Thomas Shrader wrote that the latest cancer data will likely make Medivation play even harder to get. “It’s hard to draw too much out of such a small data set from a single center — but these data will only make the company less interested in being acquired without very significant upside to the current value of the Xtandi franchise and at least conditional upside to the current value of the talazoparib franchise,” Shrader wrote in a research note.

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.”