Bioverativ Inc. (NASDAQ: BIVV) and Swedish Orphan Biovitrum AB
(publ) (Sobi™) (STO:SOBI) today announce the results of a new,
post-hoc, longitudinal analysis of the pivotal Phase 3 A-LONG study
and ASPIRE long-term extension study, showing that weekly
prophylactic dosing with its extended half-life therapy ELOCTATE®
[Antihemophilic Factor (Recombinant), Fc Fusion Protein], marketed
as Elocta® in Europe and the Middle East, has the potential to
provide improved bleed protection over episodic treatment, resolve
target joints and reduce the treatment burden associated with more
frequent dosing intervals. The analysis is being presented today in
a poster session at the 59th Annual Meeting of the American Society
of Hematology.
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the full release here:
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ELOCTATE was developed using Fc fusion technology to prolong
circulation in the body and its efficacy and safety has been
studied in hemophilia A patients since 2010. This new, post-hoc
analysis supports a growing body of clinical data showing
prophylactic treatment with ELOCTATE can positively impact
long-term joint health. ELOCTATE is currently not indicated for
weekly dosing.
“One of the challenges for people with severe hemophilia A can
be treatment every few days with inadequate bleed protection,” said
Maha Radhakrishnan, M.D., Senior Vice President of Medical at
Bioverativ. “We are committed to improving patient outcomes and
continue to explore how ELOCTATE can meaningfully make a difference
for patients with the potential for longer dosing intervals that
could provide continued joint health improvement.”
Prophylactic treatment with factor therapy is recognized as the
optimal therapy for severe hemophilia A, yet, according to the
World Federation of Hemophilia guidelines, this treatment regimen
traditionally involves injections three times per week with
conventional factor based products. With ELOCTATE’s extended
half-life, patients can extend dosing intervals up to five days
resulting in less frequent injections. Using data spanning four
years from the pivotal Phase 3 A-LONG study, and ASPIRE, the
long-term extension study, researchers examined subjects who were
exposed to a seven-day dosing interval (65 IU/kg/wk) to assess
long-term outcomes as determined by annualized bleeding rates
(ABR), adherence and resolution of target joints.
In the study, 43 adults and adolescents (≥12 years) were exposed
to an ELOCTATE weekly dosing interval for a median study duration
of 3.1 years. Researchers also analyzed results of those who
maintained a weekly dosing interval throughout the study period
(n=19).
For those subjects in the ever-on-weekly dosing group who had
pre-study episodic treatment (n=32), transition to weekly
prophylaxis dosing resulted in a change in median ABR (IQR) of
-23.7 (-35.8, -12.8). For those subjects who were always on a
weekly dosing regimen throughout the study period (n=19), the
median pre-study ABR (IQR) for subjects on a pre-study episodic
regimen was 29 (18, 45) compared to an on-study ABR (IQR) of 1.7
(0.5, 6.7). Subjects experienced protection from spontaneous bleeds
(median ABR (IQR) of 1.2 (0.2, 2.8) for subjects ever-on-weekly
dosing and 0.7 (0, 1.6) for subjects always-on-weekly dosing) and
from spontaneous joint bleeds (median ABR (IQR) of 0.8 (0, 2.5) in
subjects ever-on-weekly dosing and 0.2 (0, 1.0) in subjects
always-on-weekly dosing).
All subjects were highly adherent while on the weekly dosing
regimen (median duration of 3.1 years) and among subjects who chose
to initiate a weekly dosing regimen on ELOCTATE at any point during
the study, the majority stayed on weekly dosing. One hundred
percent of all evaluable target joints in both the ever-on-weekly
dosing group and always-on-weekly dosing group resolved during the
study period. Study findings suggest weekly dosing may be a
reasonable prophylaxis regimen for patients receiving episodic
treatment, who would prefer the benefit of prophylaxis and better
bleed protection, but with minimal treatment burden.
“Together with Bioverativ, we have long been committed to
transforming the care of people with hemophilia through our
treatments and ongoing research,” said Armin Reininger, M.D.,
Ph.D., Head of Medical and Scientific Affairs, Sobi. “These data
show the potential of ELOCTATE to make a difference for patients,
to be able to extend their dosing intervals based on their needs,
with improved joint health, and the possibility to reduce the
burden of chronic treatment in patients with hemophilia.”
About ASPIRE
ASPIRE is an open-label, non-randomized, multi-year extension
study for people who completed the pivotal, Phase 3 A-LONG or Kids
A-Long studies. The study enrolled 211 males, including 150 (98
percent) of those who completed A-LONG and 61 (91 percent) of those
who completed Kids A-LONG. The primary endpoint is the development
of inhibitors. Secondary endpoints include the annualized number of
bleeding episodes per subject, ELOCTATE exposure days and a
participant’s assessment of response to treatment of a bleeding
episode.
About ELOCTATE® /Elocta®
ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion
Protein] is a recombinant clotting factor therapy developed for
hemophilia A using Fc fusion technology to prolong circulation in
the body. It is engineered by fusing factor VIII to the Fc portion
of immunoglobulin G subclass 1, or IgG1 (a protein commonly found
in the body), enabling ELOCTATE to use a naturally occurring
pathway to extend the time the therapy remains in the body. While
Fc fusion technology has been used for more than 15 years,
Bioverativ and Swedish Orphan Biovitrum AB (publ) (Sobi) have
optimized the technology and are the first companies to utilize it
in the treatment of hemophilia. ELOCTATE is manufactured using a
human cell line in an environment free of animal and human
additives.
ELOCTATE is approved and marketed by Bioverativ in the United
States, Japan and Canada. It is also approved in Australia, New
Zealand, Brazil and other countries, and Bioverativ has marketing
rights in these regions. It is also approved as Elocta® in the
European Union, Switzerland, Iceland, Liechtenstein, Norway and
other countries where it is marketed by Sobi.
As with any factor replacement therapy, allergic-type
hypersensitivity reactions and development of inhibitors may occur
in the treatment of hemophilia A. Inhibitor development has been
observed with ELOCTATE/Elocta, including in previously untreated
patients. For more information, please see the full U.S.
prescribing information for ELOCTATE. Note that the indication
for previously untreated patients is not included in the EU
Product Information for Elocta.
About Hemophilia A
Hemophilia is a rare, genetic 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 more rarely in females. The
World Federation of Hemophilia estimates that approximately 150,000
people are currently diagnosed with hemophilia A worldwide1.
People with hemophilia A experience bleeding episodes that can
cause pain, irreversible joint damage and life-threatening
hemorrhages. Prophylactic injections of factor VIII can temporarily
replace the clotting factor that is needed to control bleeding and
prevent new bleeding episodes2. The World Federation of Hemophilia
(WFH) recommends prophylaxis as the optimal therapy as it can
prevent bleedings and joint destruction3.
About Bioverativ
Bioverativ (NASDAQ: BIVV) is a global biopharmaceutical company
dedicated to transforming the lives of people with hemophilia and
other rare blood disorders through world-class research,
development and commercialization of innovative therapies. Launched
in 2017 following separation from Biogen Inc., Bioverativ builds
upon a strong heritage of scientific innovation and is committed to
actively working with the blood disorders community. The company’s
mission is to create progress for patients where they need it most
and its hemophilia therapies when launched represented the first
major advancements in hemophilia treatment in more than two
decades. For more information, visit www.bioverativ.com or follow
@bioverativ on Twitter.
About Sobi™
Sobi is an international specialty healthcare company dedicated
to rare diseases. Sobi’s mission is to develop and deliver
innovative therapies and services to improve the lives of patients.
The product portfolio is primarily focused on Haemophilia,
Inflammation and Genetic diseases. Sobi also markets a portfolio of
specialty and rare disease products across Europe, the Middle East,
North Africa and Russia for partner companies. Sobi is a pioneer in
biotechnology with world-class capabilities in protein biochemistry
and biologics manufacturing. In 2015, Sobi had total revenues of
SEK 3.2 billion (USD 385 M) and about 700 employees. The share
(STO:SOBI) is listed on Nasdaq Stockholm. More information is
available at www.sobi.com.
About the Bioverativ and Sobi Collaboration
Bioverativ and Sobi collaborate on the development and
commercialization of ALPROLIX and ELOCTATE® [Antihemophilic Factor
(Recombinant), Fc Fusion Protein], which is marketed as Elocta® in
Europe. Bioverativ has final development and commercialization
rights in North America and all other regions in the world
excluding the Sobi territory, and has manufacturing responsibility
for ELOCTATE and ALPROLIX. Sobi has final development and
commercialization rights in the Sobi territory (essentially Europe,
North Africa, Russia and most Middle Eastern markets).
Bioverativ Safe Harbor
This press release contains forward-looking statements,
including statements about the potential benefits of ELOCTATE in
hemophilia A patients, including with weekly prophylactic dosing.
These forward-looking statements may be accompanied by such words
as “anticipate,” “believe,” “could,” “estimate,” “expect,”
“forecast,” “intend,” “may,” “plan,” “potential,” “project,”
“target,” “will” and other words and terms of similar meaning. You
should not place undue reliance on these statements. These
statements involve risks and uncertainties that could cause
Bioverativ’s actual results to differ materially from those
reflected in such statements, including, without limitation, risks
related to development of early stage programs, unexpected concerns
that may arise from additional data or analysis, regulatory
authorities may require additional information or further studies,
regulatory authorities may fail to expand product labeling, and
other risks and uncertainties associated with Bioverativ’s drug
development and commercialization activities described in the Risk
Factors section of Bioverativ’s filings with the Securities and
Exchange Commission. These statements are based on Bioverativ’s
current beliefs and expectations and speak only as of the date of
this press release. Bioverativ does not undertake any obligation to
publicly update any forward-looking statements.
References
1 World Federation of Hemophilia, Annual Global Survey 2016,
published in October 2017. Available at:
http://www.wfh.org/en/data-collection
2 World Federation of Hemophilia. About Bleeding Disorders –
Frequently Asked Questions. Available at:
http://www.wfh.org/en/page.aspx?pid=637#Difference_A_B. Accessed
on: June 17, 2016
3 Guideline for the management of hemophilia, World Federation
of Hemophilia, 2nd edition,
http://www1.wfh.org/publications/files/pdf-1472.pdf. Accessed on
December 2015
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BIOVERATIVMedia:Marianne McMorrow, +1
781-663-4376media@bioverativ.comorInvestor Relations:Susan
Altschuller, +1
781-663-4360IR@bioverativ.comorSOBIMedia:Linda Holmstr�m, +
46 708 73 40 95linda.holmstrom@sobi.comorInvestor
Relations:J�rgen Winroth, +1
347-224-0819jorgen.winroth@sobi.com
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