New England Journal of Medicine publishes positive final results from Phase 1/2a study of BIVV001 in people with severe hemop...
10 Setembro 2020 - 2:00AM
- BIVV001 is the first investigational factor VIII therapy
independent of von Willebrand Factor and has the potential to
transform replacement therapy for people with hemophilia A
- It is uniquely designed to deliver near-normal factor activity
levels for the majority of the week, extending bleed protection in
a once-weekly dose
- Results from the Phase 1/2a study showed that a single dose of
BIVV001 achieved high sustained factor activity and a three- to
four-fold increase in half-life when compared to conventional
factor VIII replacement therapies
PARIS and STOCKHOLM –
September 10, 2020 –The New England Journal of Medicine
today published positive final results from the Phase 1/2a trial
evaluating the safety, tolerability and pharmacokinetics of BIVV001
(rFVIIIFc-VWF-XTEN) in adult patients with severe hemophilia A.
BIVV001 is an investigational factor VIII therapy designed to
provide higher bleed protection in a once-weekly prophylactic
treatment regimen. Sanofi and Sobi™ (STO:SOBI) collaborate on the
development and commercialization of BIVV001.
“BIVV001 represents a potential new class of
factor VIII replacement therapies. The sustained factor activity
levels and three- to four-fold increase in half-life observed
underscore its potential to provide near-normal bleed protection
while reducing the dosing frequency of a prophylactic treatment to
once a week,” said Barbara A. Konkle, MD, lead investigator and
Chief Scientific Officer, Bloodworks Northwest and Professor
of Medicine/Hematology, University of Washington. “These results
support the conclusion that BIVV001 may be a significant
advancement for patients and we look forward to exploring this
further in the ongoing Phase 3 study.”
Phase 1/2a study resultsEXTEN-A
is an open-label, multicenter study that evaluated the safety,
tolerability and pharmacokinetics of BIVV001 in both a 25 IU/kg
(n=6) and a 65 IU/kg (n=8) dose cohort of participants aged 19-63
years with severe hemophilia A (NCT03205163). In the trial,
participants received a single dose of conventional recombinant
factor VIII (rFVIII) followed, after a washout period, by either a
single 25 IU/kg or 65 IU/kg dose of BIVV001. Primary endpoints
included occurrence of adverse events and development of
inhibitors. Key findings included:
- BIVV001 was generally well tolerated with no inhibitor
development detected through 28 days post-dose. During the study
period no adverse events of allergic reaction, anaphylaxis, or
clinically meaningful treatment-related adverse events were
reported.
- In the 65 IU/kg dose cohort, a single dose of BIVV001 achieved
a FVIII half-life of 43 hours, a greater than three-fold increase
from the 13-hour half-life observed with rFVIII. Mean factor VIII
activity level was ≥51% and in the normal range for four days, and
17% at seven days post BIVV001 infusion.
- In the 25 IU/kg cohort, a single dose of BIVV001 achieved a
FVIII half-life of 38 hours, a four-fold increase from the 9-hour
half-life observed with rFVIII, with a mean factor activity level
of 5% at seven days post BIVV001 infusion.
Factor activity levels refer to the amount of
factor VIII in a person’s blood and are used to determine the
severity of a person’s disease. Participants enrolled in the
EXTEN-A trial have severe hemophilia A (factor levels of <1%).
Moderate hemophilia A is characterized by factor levels of 1-5%,
and mild hemophilia A is from 5 - 40%.
A potential to transform factor
replacement therapy for hemophilia AThe half-life of
conventional factor VIII therapy is constrained by the von
Willebrand factor’s (VWF) chaperone effect, which is believed to
limit the time the factor remains in the body. BIVV001 is the first
factor VIII therapy under development that has been shown to break
through the VWF ceiling, thus allowing people with hemophilia A to
potentially move toward normal factor activity levels for the
majority of the week.
“As part of our overall commitment to the
hemophilia community, we are excited by the clinical potential of
BIVV001 to overcome the limitations of current factor VIII
therapies,” said Dietmar Berger, Global Head of Development and
Chief Medical Officer at Sanofi. “The New England Journal of
Medicine’s publication of these early results support the
possibility of BIVV001 to provide people with hemophilia A with
higher protection for longer, which could allow them to lead
a more active life. We look forward to providing future updates as
we continue to evaluate BIVV001 in Phase 3 development.”
“Factor VIII replacement therapy remains a
cornerstone of care in hemophilia A and is a single therapy that
can be used across numerous treatment scenarios including,
prophylaxis, acute bleed control and perioperative management,”
said Ravi Rao, Head of R&D and Chief Medical Officer at Sobi.
“BIVV001 has the potential to advance factor replacement therapy
further by offering patients and physicians near-normal factor
levels for the majority of the week whilst reducing treatment
burden. We look forward to exploring this further in the Phase 3
study.”
Phase 3 XTEND-1 studyThe safety
and efficacy of BIVV001 is currently being evaluated in the ongoing
Phase 3 XTEND-1 study in previously treated patients ≥12 years of
age (n=150) with severe hemophilia A. XTEND-1 is an open-label,
non-randomized interventional study with two parallel assignment
arms. Participants in the prophylaxis arm will receive a weekly
prophylactic 50 IU/kg dose of BIVV001 for 52 weeks. Participants in
the on-demand arm will receive BIVV001 (50 IU/kg) on demand for 26
weeks followed by a switch to BIVV001 weekly prophylaxis for
another 26 weeks.
About BIVV001BIVV001
(rFVIIIFc-VWF-XTEN) is a novel and investigational recombinant
factor VIII therapy that is designed to extend protection from
bleeds with once-weekly prophylactic dosing for people with
hemophilia A. BIVV001 builds on the innovative Fc fusion technology
by adding a region of von Willebrand factor and XTEN polypeptides
to extend its time in circulation. It is the first investigational
factor VIII therapy that has been shown to break through the von
Willebrand factor ceiling, which imposes a half-life limitation on
current factor VIII therapies. BIVV001 was granted orphan drug
designation by the US Food and Drug Administration in August 2017
and the European Commission in June 2019. BIVV001 is currently
under clinical investigation and its safety and efficacy have not
been reviewed by any regulatory authority.
About Sobi
Sobi is a specialised international
biopharmaceutical company transforming the lives of people with
rare diseases. Sobi is providing sustainable access to innovative
therapies in the areas of haematology, immunology and specialty
indications. Today, Sobi employs approximately 1,400 people across
Europe, North America, the Middle East, Russia and North Africa. In
2019, Sobi's revenues amounted to SEK 14.2 billion. Sobi's share
(STO:SOBI) is listed on Nasdaq Stockholm. You can find more
information about Sobi at www.sobi.com.
About Sanofi Sanofi is dedicated to
supporting people through their health challenges. We are a global
biopharmaceutical company focused on human health. We prevent
illness with vaccines, provide innovative treatments to fight pain
and ease suffering. We stand by the few who suffer from rare
diseases and the millions with long-term chronic
conditions. With more than 100,000 people in 100 countries,
Sanofi is transforming scientific innovation into healthcare
solutions around the globe. Sanofi, Empowering Life |
Sanofi Media Relations Contact Sally BainTel.: +1
(781) 264
1091Sally.Bain@sanofi.com Sobi
Media RelationsLinda Holmström+ 46 708 734
095linda.holmstrom@sobi.com |
Sanofi
Investor Relations Contact Investor Relations – Contacts
Paris Eva Schaefer-JansenArnaud DelepineYvonne
Naughton Investor Relations – Contacts North
AmericaFelix LauscherFara BerkowitzSuzanne
Greco Investor Relations Main LineTel.: +33
(0)1 53 77 45
45ir@sanofi.comhttps://www.sanofi.com/en/investors/contact Sobi
Investor RelationsPaula Treutiger+ 46 733 666
599paula.treutiger@sobi.com |
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