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Biogen Idec Inc. (BIIB), the third-largest biotechnology company, said its experimental hemophilia B drug helped limit bleeding with fewer infusions in a study, prompting plans to apply for U.S. approval in the first half of 2013.
More than 90 percent of bleeding episodes were controlled by one injection of recombinant Factor IX Fc fusion protein, or rFIXFc, in a 123-patient trial, the Weston, Massachusetts-based company said today. The therapy was well-tolerated in the late- stage study, Biogen said.
With current therapy, patients often get as many as three infusions a week, said Glenn Pierce, Biogen’s chief medical officer for hemophilia, in an interview. The study showed the company’s drug helped some patients when given once every two weeks. Biogen may be able to grab a substantial share of the $1 billion hemophilia B medicine market, said Eric Schmidt, a New York-based analyst with Cowen & Co.
“This really is the No. 1 treatment advance that patients with hemophilia are looking for and looking forward to,” Pierce said by telephone today. “The idea of being able to treat oneself much less often is something that has resonated within the community.”
Hemophilia B is an inherited bleeding disorder in which the blood can’t clot properly. It affects about 4,000 people in the U.S. and 25,000 worldwide, according to Biogen. The company’s drug would compete with Pfizer Inc. (PFE)’s BeneFix, which dominates the market with about $700 million in revenue, according to Mark Schoenebaum, an analyst with ISI Group in New York.
Biogen fell less than 1 percent to $151.62 at the close in New York. The shares have gained 38 percent this year. Swedish Orphan Biovitrum AB (SOBI), Biogen’s partner on the drug, surged 44 percent to close at 37.50 kronor in Stockholm. The gain was the biggest ever for the company, which sold shares in an initial public offering in September 2006.
Biogen’s study examined the drug’s use as a weekly therapy to prevent bleeding, on an individualized interval for that purpose; as a treatment given to control a bleed; and for use in surgery. The most common side effects included joint pain, high blood pressure and flu, with one serious side effect found to be “possibly related” to the drug, Biogen said.
The serious adverse event was resolved and the patient stayed on the therapy, Biogen said. No patients developed inhibitors, antibodies that stop treatment from working, the company said.
Patients in the group receiving preventative therapy once a week had a median bleed rate of 2.95 a year, Biogen said. Those treated for preventative purposes on an individualized basis, who were dosed about once every two weeks for the last six months of the study, had bleed rates of 1.38 a year. Patients treated episodically had bleeds at a rate of 17.69 a year, Biogen said.
“That means that, by and large, these patients were very well protected while they were on study with our product,” Pierce said.
The positive results were expected by most analysts, said Cowen’s Schmidt, who recommends buying Biogen shares. The company’s “body language was quite favorable,” he said in a telephone interview today.
The half-life of Biogen’s drug, a measure of how long it’s active, was 82 hours, compared with 34 hours for Pfizer’s BeneFix.
Biogen also is in late-stage trials of a therapy for hemophilia A, a market about four times the size of that for hemophilia B, Schmidt said. He expects the data from that study to be positive as well.
“This is a slow-switching market, but over time this drug could take a substantial share,” Schmidt said of the hemophilia B therapy.
Biogen, which sells the multiple sclerosis therapies Avonex and Tysabri, is awaiting Food and Drug Administration approval of its MS pill BG-12, expected by the end of the year or early in 2013. The company has also said it anticipates reporting data on its experimental drug for amyotrophic lateral sclerosis, or Lou Gehrig’s disease, by year-end.
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