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In The News

Why BioMarin's Gene Therapy Initially Targets One Type of Hemophilia Instead of Another

When researchers at University College London used  a gene therapy to effectively cure six patients of hemophilia B, BioMarin Pharmaceutical Inc. took notice.

But the San Rafael company (NASDAQ: BMRN), which licensed in the therapy in February 2013 from the college and St. Jude Children's Research Hospital, hasn't stuck to the script. Instead of targeting hemophilia B, BioMarin scientists focused on hemophilia A — and the reasons cut across business and science.

For one, several companies have jumped into the race for next-generation Factor IX drugs to treat hemophilia B patients. Cambridge, Mass.-based Biogen Idec (NASDAQ: BIIB), for example, won Food and Drug Administration approval last month for Alprolix, which can be taken once a week.

"All of these companies are going to Factor IX and they're more advanced than we are," BioMarin CEO Jean-Jacques Bienaimé said, "and it's a smaller market."

There are roughly 14,000 hemophilia A patients in the United States, but only 4,000 people have hemophilia B, according to the National Hemophilia Foundation.

On the science end, BioMarin, which by virtue of its enzyme replacement therapies sits atop the pile of rare disease drug developer contenders and pretenders, has the R&D chops to pull off a switch from hemophilia B to hemophilia A.

BioMarin's vice president of research is Gordon Vehar, while at Genentech Inc. 30 years ago, was the first person to clone Factor VIII, the blood-clotting protein that stanches bleeding in hemophilia A patients.

What's more, Barrie Carter, BioMarin's vice president of vector biology, produced some of the first adeno-associated virus, or AAV, vectors. Those stripped-down viruses act as vehicles that carry a correct gene to the right spot in the body.

There are technical challenges as well, since the gene responsible for Factor IX is much smaller than the gene involved with Factor VIII, "so it's easier to work with VIII," Bienaimé said.

From a patient perspective, of course, a one-shot-and-you're-done gene therapy would do away with three-times-a-week injections — or even the one-time-a-week that Alprolix and other next-generation therapies offer.

"We will deliver the virus into the nucleus of the cell and it will stay there forever," said Carter, who also is president of the American Society of Gene and Cell Therapy.

Also, Vehar added, a cured patient no longer would develop arthritis and other joint diseases that bring the lifetime tally of treating a hemophilia patient to more than $20 million.

BioMarin plans to file an investigational new drug application with the FDA early next year for its hemophilia A gene therapy, dubbed BMN-270. If accepted, human trials would begin next year.

But BioMarin doesn't plan to stop there.

"We've only announced Factor VIII," Vehar said, "but we view this as a platform."

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