Full Press Release Details
Additional Analyses from Phase 3 A-LONG Study Support Positive Clinical Profile
of ELOCTATE for Haemophilia A
Biogen Idec (NASDAQ: BIIB) and Swedish Orphan Biovitrum AB (publ) (Sobi) (STO:
SOBI) presented new data that support the clinical and safety profile of their
long-lasting recombinant factor VIII candidate ELOCTATE* for haemophilia A. Five
platform and oral presentations at the XXIV International Society on Thrombosis
and Haemostasis (ISTH) Congress in Amsterdam, The Netherlands, highlight the new
FVIII candidate's potential to reduce the number of intravenous injections
people with haemophilia A require, its efficacy in controlling bleeding during
and after surgery, and its efficacy in treating acute bleeding episodes.
"We are excited to share these new data, which include patients' and physicians'
assessment of ELOCTATE's efficacy in treating bleeding episodes and in
controlling bleeding during surgery," said Glenn Pierce, M.D., Ph.D., senior
vice president of Global Medical Affairs and chief medical officer of Biogen
Idec's haemophilia therapeutic area. "The A-LONG data presented at ISTH support
the potential of ELOCTATE to enable longer intervals between prophylactic
(preventative) injections compared to the current standard of care."
Treatment of Bleeding
An evaluation of the treatment of acute bleeding episodes across the prophylaxis
and episodic (on demand) treatment arms of the phase 3 A-LONG study showed that
more than 87% of bleeds were controlled with a single injection of ELOCTATE and
more than 97% of were controlled with two or fewer injections. These data were
showcased in the e-Poster presentation:
* Treatment of Bleeding Episodes in Subjects with Haemophilia A With Long-
Lasting Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) in the Phase 3
Novel Assay Clinical Research
For the first time Biogen Idec shared results of evaluations of the performance
of ELOCTATE when evaluated using two investigational haemostasis assays.
Results from a large scale, global clinical evaluation of ELOCTATE by thrombin
generation assay (TGA), using a standardized sample collection procedure and an
optimized and validated assay performed at a central laboratory showed that,
despite inherent patient-to-patient differences in thrombin generation activity,
ELOCTATE and Advate** showed equivalent thrombin generation potential post-
infusion. ELOCTATE also showed prolonged thrombin generation activity relative
to Advate, correlating with the pharmacokinetic data observed in these subjects.
Results from a rotation thromboelastometry (ROTEM ) analysis conducted on 44
patients treated with ELOCTATE from 13 A-LONG study sites showed that ELOCTATE
was fully active in patient samples. Additionally, the FVIII activity as
measured by clotting time seen for ELOCTATE at 72 hours after dosing was
comparable to the activity of Advate at 48 hours after dosing (mean clotting
times of 1,238 seconds and 1,213 seconds, respectively).
The data presentations were consistent with conventional assay results from the
A-LONG study. These data were showcased in two oral platform presentations:
* Evaluation of The Thrombin Generation Potential of a Recombinant Factor VIII
Fc Fusion Protein in a Phase III Multi-National Clinical Trial
* Evaluation of Whole Blood Clotting Activity of Recombinant Factor VIII Fc
Fusion Protein by ROTEM Analysis in a Multi-Center Phase 3 Clinical Trial
Results from an analysis of the phase 3 A-LONG study showed that ELOCTATE
consistently controlled bleeding during and after 9 major surgeries in 9
patients with haemophilia A. Physicians reported high efficacy levels of
ELOCTATE during surgery, with haemostasis (the stoppage of bleeding) rated as
"excellent" for 8/9 surgeries and "good" for 1/9 surgeries. According to the
investigators' analysis, the results were comparable to that for similar
surgeries in people without haemophilia. These data were showcased in the e-
poster presentation:
* Long-Lasting Recombinant Factor VIII Fc Fusion (rFVIIIFc) for Perioperative
Management of Subjects with Haemophilia A in the Phase 3 A-LONG Study
Population Pharmacokinetics (PK) Analysis
Analysis of a population pharmacokinetics (popPK) model developed for ELOCTATE
demonstrate that the model accurately predicts peak and trough factor VIII
activity levels achieved in the A-LONG clinical study at a variety of ELOCTATE
doses. These data were showcased in the e-poster presentation:
* Population Pharmacokinetic Analysis of Long-Lasting Recombinant Factor VIII
Fc Fusion Protein (rFVIIIFc) in Patients with Severe Haemophilia A
"These new data from the A-LONG study show potential for the application of Fc
fusion technology in haemophilia," said Birgitte Volck, M.D., Ph.D., senior vice
president development and chief medical officer of Sobi. "We are excited to
share these data on ELOCTATE, which provide additional evidence of the potential
of ELOCTATE to make a meaningful difference in the lives of patients with
ELOCTATE Global Regulatory Status
A Biologics License Application (BLA) for Biogen Idec's long-lasting haemophilia
product candidate ELOCTATE is currently under review with the FDA for the
treatment of haemophilia A. If approved, ELOCTATE would be the first major
treatment advance for the haemophilia A community in more than two decades.
A Marketing Application for ELOCTATE has been submitted in Australia for the
treatment of haemophilia A. Additional regulatory filings are planned.
About the Fc Fusion Technology Platform
ELOCTATE is a clotting factor under development using Biogen Idec's novel and
proprietary monomeric Fc fusion technology, which makes use of a naturally
occurring pathway that delays the breakdown of factor in the body and cycles it
back into the bloodstream, resulting in a longer circulating half-life. Fc
fusion technology is used in seven FDA-approved products for the treatment of
chronic diseases including rheumatoid arthritis, psoriasis and platelet
disorders. Biogen Idec is the first and only to apply this proprietary
technology to haemophilia.
Haemophilia A is a rare, inherited disorder in which the ability of a person's
blood to clot is impaired. Haemophilia A occurs in about one in 5,000 male
births annually and is caused by having substantially reduced or no factor VIII
activity, which is needed for normal blood clotting. People with haemophilia A
experience bleeding episodes that can cause pain, irreversible joint damage and
life-threatening haemorrhage. Injections of factor VIII can restore the
coagulation process, control bleeding, and prevent new bleeding episodes. The
Medical and Scientific Advisory Council of the National Haemophilia Foundation
recommends prophylaxis as the optimal therapy for people with severe haemophilia
A. Currently, prophylaxis in haemophilia A typically requires injections three
times per week or every other day to maintain a sufficient circulating level of
About the Biogen Idec and Sobi Collaboration
Biogen Idec and Swedish Orphan Biovitrum (Sobi) are partners in the development
and commercialization of ELOCTATE in haemophilia A and ALPROLIX in haemophilia
B. Biogen Idec leads development, has manufacturing rights, and has