Tag Archives: biotech

BioMarin Stock Up As Drug Slows Progress Of Deadly Rare Disease

Shares of rare-disease specialist BioMarin Pharmaceutical ( BMRN ) rose Thursday after the company reported late Wednesday that its drug for Batten disease was on track for approval after a successful trial. BioMarin presented data from its pivotal study of 24 children taking its drug cerliponase alfa for the rare genetic condition, which causes most of its sufferers to lose the ability to walk and talk by the age of six and to die before they reach puberty. Though at least 20 genes have been associated with Batten disease, cerliponase alfa targets patients with a CLN2 mutation, who BioMarin estimates number 1,200 to 1,600 in its commercial territories. BioMarin said patients taking the drug showed 80% less than the expected rate of decline in function for the untreated population, and that the treatment was generally safe and well-tolerated. MRI measurement also showed a slower rate of brain deterioration. The company said it plans to submit the drug for approval with the FDA and the European Medicines Agency around midyear. Cerliponase alfa has already been designated an orphan drug, which means it will be protected from competition in the near term if it is approved. “Whether FDA will approve this is not clear, but stabilization is a major treatment effect in these very sick children, and they’ve had ‘very fulsome dialogue about requirements to support registration’ with agencies,” wrote RBC Capital Markets analyst Michael Yee in a research note. “They haven’t communicated specifically what the numerical benchmark is to get approved, but agencies are aware of the natural-history evidence.” Yee estimates peak annual sales of at least $250 million for the drug, while Evercore ISI analyst Mark Schoenebaum reckons the opportunity at double that number. BioMarin stock was up nearly 2% in late-morning trading on the stock market today , near 89. The stock has been recovering since hitting an 18-month low on Feb. 10, as it suffered both from the market sell-off and the FDA’s rejection of its muscular dystrophy drug Kyndrisa in January. Other MD drug candidates haven’t been faring much better: The FDA put off making a decision on Sarepta Therapeutics ‘ ( SRPT ) eteplirsen until May after issuing a scathing assessment of the drug in its briefing documents, while PTC Therapeutics ( PTCT ) said Monday that the FDA had refused to receive its application for ataluren, despite its having already been approved in Europe.

Bluebird Bio Starts First CAR-T Cancer Trial; Juno Gets An Upgrade

Biotech Bluebird Bio ( BLUE ) was trading up sharply Wednesday after the company said it started testing its cell therapy in blood cancer, triggering a $10 million payment from partner Celgene ( CELG ). And another Celgene partner, Juno Therapeutics, also was up Wednesday, after getting an upgrade. Last June, Bluebird and Celgene agreed to a three-year collaboration to develop Bluebird’s chimeric antigen receptor T cell (CAR-T) therapies targeting the B-cell maturation antigen (BCMA) to help the body fight cancer. On Wednesday, Bluebird said the first patient of its phase-one study of its candidate bb2121 in multiple myeloma had been treated, yielding a $10 million option-to-exercise payment from Celgene. Bluebird stock was up more than 7% in afternoon trading on the stock market today , near 54. “While Celgene’s opt-in decision doesn’t come entirely unexpected, we view the decision positively since it further validates Bluebird’s CAR-T effort,” wrote Leerink analyst Michael Schmidt in a research note. “While several other groups have been working on myeloma CAR-T programs, anti-BCMA CAR-T data from Bluebird’s collaborator at the NCI (National Cancer Institute) . . . has been most promising to date in our view.” Celgene’s lead drug Revlimid is currently one of the most popular treatments for multiple myeloma, but since it’s due to lose patent protection in a few years the company has been looking at newer technologies. Meanwhile, another Celgene partner in CAR-T, Juno Therapeutics ( JUNO ), got an upgrade from Guggenheim on Wednesday based on both valuation and a reassessment of its spending habits. “Juno has been steadily amassing an arsenal of tools to increase efficacy and reduce toxicity of T cell therapies,” wrote analyst Tony Butler as he upgraded the stock to buy from neutral and set the price target at 41. “It is a pay-me-now-or-pay-me-later scenario. . . . While not all of these investments may pay out in the future, it is likely that one or two will, and they could provide Juno the opportunity to surpass the competition or, at the very least, to stay at the vanguard of T cell therapies.” Juno stock was up more than 6.7% in afternoon trading, near 34.

Intercept Pharma Spikes On Report That It’s Exploring A Sale

Shares of biotech Intercept Pharmaceuticals ( ICPT ) spiked 30% in early-afternoon trading Friday on a report that the company is exploring a sale. Reuters cited anonymous sources in its report that Intercept has been working with investment bankers this week after it received interest from other companies. Reuters did not name any of the suitors, but Intercept has been a perpetual source of buyout speculation. Gilead Sciences ( GILD ) is a popular choice of buyer due to its overlapping work in liver diseases, and so are big pharmas working in that space such as Bristol-Myers Squibb ( BMY ), Johnson & Johnson ( JNJ ) and Merck ( MRK ). Intercept shares were up about 31% on the stock market today , near 123. An imminent deal would be oddly timed, however, given that Intercept’s lead drug is tied up in a long and uncertain FDA review. Initially the agency set a deadline of Feb. 29 to decide whether to approve Intercept’s obeticholic acid (OCA) for primary biliary cirrhosis (PBC). In December, however, the FDA pushed the deadline out to May 29 to provide time for an advisory committee, or adcom, to review and vote on the application on April 7. The FDA generally calls such committees when it has unresolved issues with the data. Intercept is also studying OCA for the potentially much bigger market in nonalcoholic steatohepatitis (NASH), but safety issues have arisen in its trials. “We doubt that Intercept could or would be acquired before at least an adcom panel to discuss the risk/benefit of the drug for PBC approval, and any indications of interest by an acquirer would be more to just do due diligence and explore scenarios and valuations first,” wrote RBC Capital Markets analyst Michael Yee in a research note. Yee added that an acquirer would likely have to pay up to $5 billion for Intercept but wouldn’t be able to make money off the deal for years since the drug is expected to ramp slowly. This uncertainty over the last few months has helped tamp down Intercept’s stock even more than other biotechs’, as it’s trading at only about a third of its May high of 314.88. Image provided by Shutterstock .