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.