– Individualized and weekly prophylactic regimens resulted in low single-digit median annualized bleeding rates –
– 98% of bleeding episodes were controlled with one or two injections of rFVIIIFc –
– No patients developed inhibitors to rFVIIIFc –
– The primary efficacy and safety objectives were met and
Top-line results from A-LONG, a global, multi-center, Phase 3 clinical study of the companies’ long-lasting recombinant Factor VIII Fc fusion protein (rFVIIIFc), showed that rFVIIIFc was effective in the control and prevention of bleeding, routine prophylaxis and perioperative management. Recombinant FVIIIFc was generally well-tolerated. Additional analyses of the A-LONG study are ongoing, and the companies anticipate presenting detailed results at a future scientific meeting.
“These top-line results demonstrated that rFVIIIFc has the potential to
enhance the care of people living with hemophilia A by offering
protection from bleeding with reduced treatment burden,” said
“We are very encouraged by the positive A-LONG study results, which
support the application of Fc fusion technology in hemophilia A to
prolong factor activity and potentially offer extended protection from
Summary of Key Data from A-LONG
In the A-LONG study, 165 male patients aged 12 years and older were enrolled. The A-LONG study had three treatment arms: individualized prophylaxis, weekly prophylaxis and episodic (on-demand) treatment (Arms 1, 2 and 3, respectively). In a subgroup of patients across treatment arms, rFVIIIFc was evaluated in the perioperative management of patients who required a major surgical procedure during the study.
Overall, 93 percent of patients completed the study. Recombinant FVIIIFc was generally well-tolerated. No inhibitors to rFVIIIFc were detected and no cases of anaphylaxis were reported in any patients, all of whom switched from commercially-available Factor VIII products. No serious adverse events were assessed to be related to drug by the investigator. The most common adverse events (incidence of ≥5 percent) occurring outside of the perioperative management period were nasopharyngitis, arthralgia, headache and upper respiratory tract infection.
The median annualized bleeding rates (ABR), including spontaneous and traumatic bleeds, were 1.6 in the individualized prophylaxis arm, 3.6 in the weekly prophylaxis arm and 33.6 in the episodic treatment arm. In the individualized prophylaxis arm, the median dosing interval was 3.5 days. During the last three months on study, 30 percent of patients in the individualized prophylaxis arm achieved a mean dosing interval of five days.
Control of bleeding was assessed in all patients who experienced a bleeding episode during the study. Overall, 98 percent of bleeding episodes were controlled by one or two injections of rFVIIIFc.
In addition, rFVIIIFc was assessed in the perioperative management of nine patients undergoing nine major surgical procedures. The treating physicians rated the hemostatic efficacy of rFVIIIFc as excellent or good in 100 percent of these surgeries.
A-LONG included pharmacokinetic (PK) analysis of rFVIIIFc in all patients in the study. In a protocol-defined subset of patients with extensive PK sampling, the approximate terminal half-life of rFVIIIFc was 19.0 hours compared to 12.4 hours for Advate® [antihemophilic factor (recombinant), plasma/albumin-free method], consistent with the results obtained in the Phase 1/2a study of rFVIIIFc.
About the A-LONG Study and the rFVIIIFc Program
A-LONG was a global, open-label, multi-center Phase 3 study that
evaluated the efficacy, safety and pharmacokinetics of
intravenously-injected rFVIIIFc. The study was designed to evaluate
rFVIIIFc in the control and prevention of bleeding, routine prophylaxis
and perioperative management in patients with hemophilia
The A-LONG study had three treatment arms. In Arm 1 (individualized prophylaxis; n=118), patients were treated with 25-65 IU/kg of rFVIIIFc, at an interval of every three to five days, which was individualized to maintain factor trough levels sufficient to prevent bleeding. In Arm 2 (weekly prophylaxis; n=24), patients were treated with a weekly dose of 65 IU/kg. In Arm 3 (episodic treatment; n=23), patients received rFVIIIFc as needed for bleeding. In a subgroup of patients across treatment arms, rFVIIIFc was evaluated in the surgical setting.
The primary efficacy and safety measures were the annualized bleeding rate and the incidence of adverse events including inhibitor development in patients studied for up to approximately 52 weeks. Secondary endpoints included response to treatment of bleeding episodes and the pharmacokinetics of rFVIIIFc versus Advate.
Ongoing clinical studies of rFVIIIFc include the Kids A-LONG and ASPIRE studies. Kids A-LONG is a Phase 3, open-label study in previously-treated children with hemophilia A under age 12, which is actively recruiting patients. ASPIRE is a long-term open-label study for patients who completed the A-LONG study or who complete the Kids A-LONG study.
About the Fc Fusion Technology Platform
Recombinant FVIIIFc is a clotting factor developed using
Using the same Fc fusion technology,
About Hemophilia A
Hemophilia A is a rare, inherited disorder in which the ability of a
person's blood to clot is impaired. Hemophilia A occurs in about one in
5,000 male births annually and is caused by having substantially reduced
or no Factor VIII protein, which is needed for normal blood clotting.
People with hemophilia A therefore need injections of Factor VIII to
restore the coagulation process and prevent frequent bleeds that could
otherwise lead to pain, irreversible joint damage and life-threatening
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,"
"potential," "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 or may
delay approval of 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:
Jim Baker, +1-781-464-3260
Senior Manager, Public Affairs
Biogen Idec Investor Relations Contact:
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Director, Investor Relations
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Vice President, Head of Communications and Investor Relations (Interim)