CSL Behring to Present Pivotal Data for rVIII-SingleChain and rIX-FP at 2015 ISTH Congress

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Jun 16, 2015, 09:00 ET

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
[email protected]

[1] World Federation of Hemophilia. Report on the Annual Global Survey 2013

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