More than 20 abstracts focusing on investigational and
branded products showcase CSL Behring's expertise and
commitment to advancing the care of patients with bleeding
disorders
KING OF PRUSSIA, Pa.,
June 16, 2015 /PRNewswire/
-- CSL Behring announced today it will present more than 20
abstracts, including five oral presentations, from across its
hematology portfolio of investigational and branded products at the
2015 International Society on Thrombosis and Haemostasis (ISTH)
Congress, being held in Toronto
June 20-25. The presentations will include pivotal trial data
for two of its late-stage recombinant products – its novel
recombinant factor VIII SingleChain (rVIII-SingleChain) compound
for hemophilia A and its long-acting recombinant factor IX albumin
fusion protein (rIX-FP) for hemophilia B.
Hemophilia is an inherited bleeding disorder, primarily
affecting males, caused by a shortage of or defect in proteins that
prevent the blood from clotting normally. The condition
affects more than 175,000 people worldwide, the majority of whom
have hemophilia A.[1]
"CSL is proud to be sharing a significant amount of new
scientific and clinical research at ISTH's 2015 conference," said
Dr. Andrew Cuthbertson, Chief
Scientific Officer and Director of R&D, CSL Limited. "I am
particularly excited that data from our phase III pivotal studies,
for both rVIII-SingleChain and rIX-FP, will be presented publicly
for the first time. These product candidates hold great
promise and potential, and could offer patients strong and
sustained efficacy and improved convenience with less frequent
dosing, two key areas of unmet need. These data, along with
abstracts for other R&D candidates and products in our
coagulation franchise, reinforce the depth and breadth of CSL's
knowledge and commitment to advancing the care of patients with
serious medical conditions."
In addition to presenting new clinical data, CSL Behring will
host programs at ISTH 2015 focusing on the unmet needs of people
living with hemophilia A and B, as well as other bleeding
disorders. Of note is a satellite symposium, "Pioneering
Therapeutic Proteins in Hemophilia Care Through Innovative
Technologies," being held on Monday, June 22 from 12:15 –
1:45 p.m. EDT (Room 714, Level
700). Representatives of the CSL Behring coagulation
franchise will be available at booth number 1522 throughout
ISTH.
Key abstracts from CSL Behring at ISTH 2015 include:
rVIII-SingleChain
Oral Presentation:
Wednesday,
June 24, 8:30–8:45 a.m. EDT
- Late-breaking abstract # LB008: rVIII-SingleChain,
results of the pivotal phase I/III PK, efficacy and safety clinical
trial in adults and adolescents with severe hemophilia A
Posters:
Tuesday, June
23, 6:00-7:30 p.m.
EDT
- The FVIII plasma activity of rVIII-SingleChain can be measured
in both the one-stage and chromogenic substrate assays. E-Poster #
PO089
- Physicochemical characterization of recombinant single-chain
factor VIII (rVIII-SingleChain). E-Poster # PO146
- Population pharmacokinetic model of recombinant single-chain
factor VIII (rVIII-SingleChain) in patients with hemophilia
A. E-Poster # PO241
- Efficacy and safety of rVIII-SingleChain in surgical
prophylaxis. E-Poster # PO258
- rVIII-SingleChain pharmacokinetics in adults, adolescents and
children. E-Poster # PO262
rIX-FP
Oral Presentations:
Wednesday, June 24, 2:00-3:15p.m. EDT
- Efficacy, pharmacokinetics (PK) and safety results of a phase 3
clinical study of recombinant fusion protein linking coagulation
factor IX with albumin (rIX-FP) in previously treated children with
hemophilia B. Oral Communication # OR346
- Efficacy and safety results of a phase 3 pivotal clinical study
of recombinant fusion protein linking coagulation factor IX with
albumin (rIX-FP) in previously treated patients with hemophilia
B. Oral Communication # OR347
- Population pharmacokinetics (PK) of recombinant fusion protein
linking coagulation factor IX with recombinant albumin (rIX-FP) in
adult and pediatric patients with severe hemophilia B. Oral
Communication # OR350
Posters:
Monday, June 22, 5:15–6:30 p.m.
EDT
- Tissue distribution of rIX-FP after intravenous application to
rodents. E-Poster # PO152
Tuesday, June 23,
6:00–7:30 p.m. EDT
- Structural characterization of recombinant factor IX fusion
protein linked with human albumin (rIX-FP). E-Poster # PO144
Wednesday, June 24,
5:15–6:30 p.m. EDT
- Efficacy and safety of recombinant fusion protein linking
coagulation factor IX with albumin (rIX-FP) in previously treated
patients with hemophilia B undergoing a surgical procedure.
E-Poster # PO253
rVIIa-FP
Oral
Presentation:
Monday, June
22, 4:15–4:30
p.m. EDT
- A recombinant fusion protein linking activated coagulation
factor VIIa with albumin (rVIIa-FP) binds to neonatal Fc receptor
and tissue factor in vitro. Oral Communication # AS016
Posters
Tuesday, June
23, 6:00–7:30 p.m. EDT
- Dosing of rVIIa-FP in clinical studies in hemophilia with
inhibitors and factor VII deficiency. E-Poster # PO257
Wednesday, June 24,
5:15–6:30 p.m. EDT
- The recombinant fusion protein linking activated factor VIIa to
human albumin (rVIIa-FP) provides superior bleeding protection
compared to recombinant FVIIa (rFVIIa) in a novel monkey model of
acquired factor VIII inhibitors. E-Poster # PO596
Plasma-Derived Factor
VIII/VWF
Posters:
Monday, June 22, 5:15–6:30 p.m. EDT
- An open-label, multi-center extension study to assess the
efficacy and safety of a plasma-derived von Willebrand
factor/factor VIII (VWF/FVIII) concentrate in pediatric,
adolescent, and adult subjects with von Willebrand disease.
E-Poster # PO643
- Plasma-derived, purified, pasteurized von Willebrand
factor/factor VIII concentrate in the treatment of patients with
von Willebrand disease and haemophilia A: update of a long-term
observational study. E-Poster # PO647
Tuesday, June 23,
6:00–7:30 p.m. EDT
- A phase III, open-label, multicenter study to evaluate
pharmacokinetics of a plasma-derived von Willebrand factor/factor
VIII (VWF/FVIII) concentrate in pediatric subjects with haemophilia
A (SWIFTLY-HA study). E-Poster # PO206
- A phase III open-label, multi-center study with a
plasma-derived von Willebrand factor/factor VIII concentrate to
assess the pharmacokinetics, efficacy, and safety in pediatric
subjects with von Willebrand disease (SWIFTLY-VWD study). E-Poster
# PO629
Wednesday, June 24,
5:15–6:30 p.m. EDT
- Molar specific activity of Factor VIII concentrates. E-Poster #
PO194
- High-purity, plasma-derived, pasteurized factor VIII
concentrate in the treatment of patients with haemophilia A: update
of a long-term observational study. E-Poster # PO195
- A phase III, open-label, multicentre study to evaluate efficacy
and safety of a plasma-derived von Willebrand factor/factor VIII
concentrate in pediatric subjects with hemophilia A (SWIFTLY-HA
study). E-Poster # PO249
rVWF-FP
Poster:
Tuesday, June 23, 6:00–7:30 p.m.
EDT
- Improved resolution of high molecular weight multimers of
recombinant von Willebrand factor–albumin fusion product by agarose
electrophoresis/western blotting. E-Poster # PO673
Pasteurization
Poster:
Wednesday, June 24, 5:15–6:30 p.m.
EDT
- Inactivation of emerging viruses by pasteurization in
plasma-derived medicinal products. E-Poster # PO605
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 and half-life of FVIII.
The Phase III trial, a part of the AFFINITY clinical development
program, is an open-label, non-randomized, multicenter study
evaluating the efficacy, safety and pharmacokinetics of
rVIII-SingleChain. Study design details for rVIII-SingleChain
(CSL627) are available at clinicaltrials.gov.
About rIX-FP
CSL Behring engineered rIX-FP to extend
the half-life of recombinant factor IX through genetic fusion with
recombinant albumin. CSL Behring selected albumin as its
recombinant genetic fusion partner for its coagulation factor
proteins due to its long physiological half-life. In addition,
albumin has been shown to have a good tolerability profile, low
potential for immunogenic reactions and a well-known mechanism of
clearance. The cleavable linker connecting recombinant factor IX
and recombinant albumin has been specifically designed to preserve
the native function of the coagulation factor in the fusion
protein, while benefiting from recombinant albumin's long
physiological half-life.
In February 2015, the U.S. Food and Drug Administration accepted
for review CSL Behring's Biologics License Application (BLA) for
rIX-FP. In March 2015, the European Medicines Agency (EMA)
started the Centralized Procedure for reviewing CSL Behring's
Marketing Authorization Application (MAA) for rIX-FP.
The PROLONG-9FP clinical development program for rIX-FP covers
patients from the age of 1 to 61 years. Studies in the program were
conducted as open-label, multicenter, safety and efficacy studies
of rIX-FP in previously treated patients with hemophilia B
(FIX </= 2%).
Study design details for rIX-FP (CSL654) are available at
clinicaltrials.gov.
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
[1] World Federation of Hemophilia. Report on the Annual
Global Survey 2013
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