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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 . Scalper1 News
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