Data Presented during Late Breaking Session at the
2015 ISTH Congress showed:
- Patients using rVIII-SingleChain prophylactically to
prevent bleeding were well controlled when dosed only two or three
times weekly
- Prophylactic dosing resulted in low annualized spontaneous
bleeding rates and the majority of treated bleeds were controlled
with only one dose
- rVIII-SingleChain had improved pharmacokinetic parameters
compared with octocog alfa, the comparator
TORONTO, June 24, 2015 /PRNewswire/ -- CSL Behring
today presented data from a Phase I/III study on the efficacy and
safety of its novel investigational recombinant factor VIII single
chain (rVIII-SingleChain) in adolescents and adults with hemophilia
A during a late breaking abstract session at the 2015 International
Society on Thrombosis and Haemostasis (ISTH) Congress.
Overall, patients using rVIII-SingleChain to prevent bleeding
(prophylaxis) were well controlled with two to three infusions per
week and developed no inhibitors.
Patients using rVIII-SingleChain to prevent bleeding had low
annualized bleeding rates (median ABR of 1.14), and an annualized
spontaneous bleeding rate (AsBR) of 0.00. rVIII-SingleChain had
improved pharmacokinetic parameters compared with octocog alfa, the
comparator. Of 848 bleeds treated in the study, 94 percent
were successfully controlled with no more than two infusions of
rVIII-SingleChain, with 81 percent controlled by only one
infusion. The majority of bleeding events treated with
rVIII-SingleChain and assessed by investigators (94 percent of 835
assessed bleeding events) were rated as excellent or good. The data
are part of the AFFINITY Phase I/III study, an open-label,
multi-center trial examining the safety, efficacy and
pharmacokinetics of rVIII-SingleChain compared with recombinant
human antihemophilic factor VIII (octocog alfa). Study design
details for rVIII-SingleChain (CSL627) are available at
clinicaltrials.gov.
"In this large-scale study, we observed relatively low
annualized bleeding rates and a median of zero spontaneous bleeding
events with rVIII-SingleChain for routine prophylaxis for patients
with hemophilia A," said Professor Ingrid
Pabinger-Fasching, M.D., of the Medical University of
Vienna, Austria and lead
investigator of the pivotal trial. "As the first and only single
chain recombinant factor product, rVIII-SingleChain has the
potential to offer improved protection from bleeding with less
frequent dosing, and an excellent safety profile thus far."
Results presented in the late breaking session included data on
more than 14,000 exposure days in 146 patients on prophylaxis and
27 patients treated on demand for a bleeding event. In total,
120 patients were treated for more than 50 days of exposure; 52 had
more than 100 days of exposure. Among patients in the prophylaxis
group, 32 percent were dosed twice weekly and 54 percent received
treatment three times per week. The most common adverse events were
naso-pharyngitis, arthralgia, and headache. Overall,
rVIII-SingleChain was well tolerated and no inhibitors have been
reported.
"Our novel rVIII-SingleChain was specifically designed to
improve the stability and provide longer-lasting hemostatic
efficacy of factor VIII, thereby addressing the need to provide
hemophilia A patients with a treatment that may require fewer
infusions while maintaining its therapeutic effect," said Dr.
Andrew Cuthbertson, Chief Scientific
Officer and Director of R&D, CSL Limited. "These pivotal
data are promising and are supportive of CSL Behring's commitment
to bringing this therapy to the market, and to helping improve the
care of people living with hemophilia A."
About rVIII-SingleChain
rVIII-SingleChain is a novel recombinant single-chain factor
VIII (FVIII) construct specifically designed for greater molecular
stability. It uses a covalent bond that forms one structural
entity, a single chain, to improve the stability of FVIII and
provide longer-lasting FVIII activity.
About Hemophilia A
Hemophilia A (congenital factor VIII deficiency) is caused by
deficient or defective factor VIII. The condition is characterized
by prolonged or spontaneous bleeding, especially into the muscles,
joints, or internal organs. Affecting approximately 1 in 5,000 to
10,000 people, hemophilia A is the most common form of hemophilia.
Nearly all hemophilia A patients are male.
About CSL Behring
The people and science of CSL Behring save lives around the
world. We develop and deliver innovative specialty biotherapies,
driven by our 100-year promise to help people with life-threatening
conditions live full lives. With 14,000 employees and operations in
30 countries, CSL Behring applies world-class R&D, high-quality
manufacturing and patient-centered management.
CSL Behring therapies are used around the world to treat
coagulation disorders including hemophilia and von Willebrand
disease, primary immune deficiencies, hereditary angioedema and
inherited respiratory disease, and neurological disorders in
certain markets. The company's products are also used in cardiac
surgery, organ transplantation, burn treatment and to prevent
hemolytic disease of the newborn.
CSL Behring operates one of the world's largest plasma
collection networks, CSL Plasma. CSL Behring is a global
biopharmaceutical company and a member of the CSL Group of
companies. The parent company, CSL Limited (ASX:CSL), is
headquartered in Melbourne,
Australia. For more information, visit
www.cslbehring.com.
Contact:
Greg Healy
CSL Behring
Office: 610-878-4841
Mobile: 610-906-4564
Greg.Healy@CSLBehring.com
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