-- Prophylactic regimens resulted in low single-digit annualized bleeding rates --
-- Median dosing interval was 14 days in the individualized interval prophylaxis arm during the last 6 months on study --
-- Greater than 90% of bleeding episodes were controlled by a single injection of rFIXFc --
-- No patients developed inhibitors to rFIXFc --
-- The primary efficacy and safety objectives were met and
Top-line results from B-LONG, a global, multi-center, Phase 3 clinical study of the companies’ long-lasting recombinant Factor IX Fc fusion protein (rFIXFc), showed that rFIXFc was effective in the control and prevention of bleeding, routine prophylaxis, and perioperative management. Recombinant FIXFc was generally well-tolerated. Additional analyses of the B-LONG study are ongoing and the companies anticipate presenting further results at a future scientific meeting.
“The results of the B-LONG study offer the potential for longer-lasting
protection from bleeding for patients with hemophilia B,” said
“Our companies are pioneering the application of Fc fusion technology to
extend the half-life of clotting factors. Fc fusion technology utilizes
a naturally-occurring recycling pathway that has been successfully
employed in other therapeutic areas. This approach holds promise for
combining more consistent protection with fewer injections,” said
Summary of Key Data from B-LONG
In the B-LONG study, 123 male patients aged 12 years and older were enrolled. The B-LONG study had four treatment arms: weekly prophylaxis, individualized interval prophylaxis, episodic treatment and perioperative management (Arms 1, 2, 3 and 4, respectively).
Overall, 93.5 percent of patients completed the study. Recombinant FIXFc was generally well-tolerated. No inhibitors to rFIXFc were detected and no cases of anaphylaxis were reported in any patients, all of whom switched from commercially-available Factor IX products. One serious adverse event was assessed to be possibly related to drug by the investigator. The patient experienced obstructive uropathy in the setting of hematuria; he continued rFIXFc treatment and the event resolved with medical management.
The most common adverse events (incidence of ≥5 percent) occurring outside of the perioperative management arm (i.e., Arms 1, 2 and 3, but not Arm 4) were nasopharyngitis, influenza, arthralgia (joint pain), upper respiratory infection, hypertension and headache.
The overall median annualized bleeding rates (including spontaneous and traumatic bleeds) were 2.95 in the weekly prophylaxis arm, 1.38 in the individualized interval prophylaxis arm, and 17.69 in the episodic treatment arm. In the individualized interval prophylaxis arm, the median dosing interval during the last 6 months on study was 14 days.
Control of bleeding was assessed in all patients who experienced a bleeding episode during the study. Overall, 90.4 percent of bleeding episodes were controlled by a single injection of rFIXFc.
Recombinant FIXFc was assessed in the perioperative management of 12 patients undergoing 14 major surgical procedures. The treating physicians rated the hemostatic efficacy of rFIXFc as excellent or good in 100 percent of these surgeries.
B-LONG included a pharmacokinetic (PK) analysis of rFIXFc in all patients in the study. In a protocol-defined subset of patients with extensive PK sampling, the approximate terminal half-life of rFIXFc was 82 hours compared to 34 hours for BeneFIX® [Coagulation Factor IX (Recombinant)].
About the B-LONG Study and the rFIXFc Program
B-LONG was a global, open-label, multi-center Phase 3 study that
evaluated the efficacy, safety and pharmacokinetics of
intravenously-injected rFIXFc. The study was designed to evaluate rFIXFc
in the control and prevention of bleeding, routine prophylaxis and
perioperative management in patients with hemophilia
The B-LONG study had four treatment arms. In Arm 1 (weekly prophylaxis; n=63), patients were treated weekly with a starting dose of 50 IU/kg, which was adjusted to maintain trough factor levels sufficient to prevent bleeding. In Arm 2 (individualized interval prophylaxis; n=29), patients were treated with 100 IU/kg, at an initial interval of 10 days, which was subsequently individualized to maintain trough factor levels sufficient to prevent bleeding. In Arm 3 (episodic treatment; n=27), patients received rFIXFc episodic treatment as needed for bleeding. In Arm 4 (perioperative management; n=12 patients), rFIXFc was evaluated in the surgical setting; 8 patients in the surgery arm were also enrolled in other treatment arms.
The primary efficacy and safety measures were the annualized bleeding rate and the incidence of adverse events and inhibitor development in patients studied for up to 77 weeks. Secondary endpoints included response to treatment of bleeding episodes and the pharmacokinetics of rFIXFc versus BeneFIX®.
Ongoing clinical studies of rFIXFc include the Kids B-LONG and the B-YOND studies. Kids B-LONG is a Phase 3, open-label study in previously treated children with hemophilia B under age 12, which is actively recruiting patients. B-YOND is a long-term open-label extension study for patients who completed the B-LONG study or who complete the Kids B-LONG study.
About the Fc Fusion Technology Platform
Recombinant FIXFc is a clotting factor developed using
Using the same Fc fusion technology,
About Hemophilia B
Hemophilia B is a rare, inherited disorder in which the ability of a
person's blood to clot is impaired. Hemophilia B occurs in about one in
25,000 male births annually and is caused by having substantially
reduced or no Factor IX activity, which is needed for normal blood
clotting. People with hemophilia B therefore need injections of Factor
IX to restore the coagulation process and prevent frequent bleeds that
could otherwise lead to pain, irreversible joint damage and
Through cutting-edge science and medicine,
Sobi is an international healthcare company dedicated to bringing
innovative therapies and services to improve the lives of rare disease
patients. The product portfolio is primarily focused on inflammation and
genetic diseases, with three late stage biological development projects
within hemophilia and neonatology. Sobi also markets more than 40
products for companies in the specialty and rare disease space. In 2011,
Sobi had revenues of
This press release contains forward-looking statements, including
statements about the development and commercialization of long-lasting
hemophilia therapies and regulatory filings. These statements may be
identified by words such as "believe," "expect," "may," "plan," "will"
and similar expressions, and are based on the companies' current beliefs
and expectations. Drug development and commercialization involve a high
degree of risk. Factors which could cause actual results to differ
materially from the companies' current expectations include the risk
that unexpected concerns may arise from additional data or analysis,
regulatory authorities may require additional information or further
studies, or may fail to approve our drug candidates, or the companies
may encounter other unexpected hurdles. For more detailed information on
the risks and uncertainties associated with
The information above has been published pursuant to the Swedish
Securities Market Act and/or the Financial Instruments Trading Act. The
information was released for public distribution on
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Biogen Idec Media Contact:
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Senior Manager, Public Affairs
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Head of Communications and Investor Relations