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Pioneering therapeutic proteins in hemophilia care through innovative technologies
S1 Thrombosis Research 141S3 (2016) S1
Contents lists available at ScienceDirect
Thrombosis Research j o u r n a l h o m e p a g e : w w w. e l s e v i e r . c o m / l o c a t e / t h r o m r e s
Editorial
Pioneering therapeutic proteins in hemophilia care through innovative technologies Ingrid Pabinger-Faschinga,*, Claude Négrierb,* a b
Clinical Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria Hemophilia Comprehensive Care Center and Hematology Department, Louis Pradel University Hospital, Lyon, France
The XXV Congress of the International Society on Thrombosis and Haemostasis (ISTH 2015 Congress) marked a major milestone in the management of patients with hemophilia, with the announcement of key clinical trial results from studies of three innovative new recombinant coagulation factors that have been developed to ease the burden of hemophilia care. Phase III studies of CSL Behring’s recombinant single-chain factor VIII (rVIII-SingleChain) and recombinant fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) have now been completed, and the results were reported for the first time at this congress. Attendees also heard news of the company’s novel recombinant fusion protein linking recombinant coagulation factor VIIa with recombinant albumin (rVIIa-FP), which has now progressed into clinical trials in patients with hemophilia A or B with inhibitors, and in patients with congenital factor VII deficiency. To celebrate these milestones and recognize the vital contribution made by the patients, clinicians, researchers, and other scientists involved in the studies around the world, CSL Behring hosted a scientific symposium at the ISTH 2015 Congress entitled “Pioneering Therapeutic Proteins in Hemophilia Care Through Innovative Technologies”. The articles in this supplement summarize the presentations from that symposium. In the first paper, Professor Ingrid Pabinger-Fasching from the Medical University of Vienna in Austria tells the story of the unique rVIII-SingleChain for the treatment of hemophilia A. The molecule has been engineered to offer increased stability and a higher binding affinity to von Willebrand factor than current treatments, with an improved pharmacokinetic and pharmacodynamic profile in preclinical and clinical studies. In her paper, Prof. Pabinger-Fasching presents an overview of the latest results from a recently completed phase I/III study with rVIII-SingleChain in previously treated patients with hemophilia A, confirming that this molecule has good hemostatic efficacy and is well tolerated. In the second paper, Dr. Elena Santagostino from Maggiore Hospital and the University of Milan, Italy, provides an update on the results from the PROLONG-9FP clinical development program
for rIX-FP, which has generated considerable interest amongst the hemophilia community. Dr. Santagostino describes the highly anticipated results from two phase III studies of rIX-FP in previously treated adults, adolescents, and pediatrics with severe hemophilia B, which were presented for the first time at the ISTH 2015 Congress. These studies have confirmed that rIX-FP is a truly remarkable new treatment that will transform the lives of many patients with hemophilia B, extending the dosing interval for FIX prophylaxis from every 3 or 4 days to every 7 or 14 days. In the final paper, Prof. Claude Négrier from the Louis Pradel University Hospital in Lyon, France, contributes a paper describing the latest findings from the PROLONG-7FP program, which is the clinical development program for CSL Behring’s novel fusion protein, rVIIa-FP. Initial results from a phase I study of rVIIa-FP in healthy volunteers suggests that this new molecule has a markedly extended half-life compared with the currently available recombinant factor VIIa, offering the potential for more sustained hemostasis and more manageable prophylaxis schedules. Studies in patients with hemophilia A or B and inhibitors, and in those with congenital FVII deficiency, are now underway. The papers in this supplement bring together all the latest findings from the comprehensive clinical development programs underway for CSL Behring’s most advanced recombinant coagulation factors. CSL Behring has always been considered to be a pioneer in the plasma therapeutics industry, and we are delighted that the company has committed to developing these – and other – innovative new recombinant technologies from which our patients may soon be able to benefit. Conflict of interest CN has received research support/honoraria, and/or has participated as a member of the scientific advisory board for Alnylam, Baxter, Bayer, Biogen Idec/SOBI, CSL Behring, Inspiration, LFB, Novo Nordisk, Octapharma, and Pfizer. IP-F has received research/travel support, and/or has participated as a member of the scientific advisory board for Amgen, Baxter, Bayer, Boehringer Ingelheim, CSL Behring, Pfizer, and GSK.
* Corresponding authors at: Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie, Währinger Gürtel 18–20 A-1090, Wien, Austria. Tel.: +43 1 40400 4952; fax: +43 1 40402 6930. E-mail address: [email protected] (I. Pabinger-Fasching); Centre de Référence de l’Hémophilie, Hôpital Louis Pradel – Hospices Civils de Lyon, Université Claude Bernard, Lyon, France. Tel.: +33 4 7211 8821; fax: +33 4 7211 8817. E-mail address: [email protected] (C. Négrier). 0049-3848/© 2016 Elsevier Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)
Contents lists available at ScienceDirect
Thrombosis Research j o u r n a l h o m e p a g e : w w w. e l s e v i e r . c o m / l o c a t e / t h r o m r e s
Editorial
Pioneering therapeutic proteins in hemophilia care through innovative technologies Ingrid Pabinger-Faschinga,*, Claude Négrierb,* a b
Clinical Division of Hematology and Hemostaseology, Medical University of Vienna, Vienna, Austria Hemophilia Comprehensive Care Center and Hematology Department, Louis Pradel University Hospital, Lyon, France
The XXV Congress of the International Society on Thrombosis and Haemostasis (ISTH 2015 Congress) marked a major milestone in the management of patients with hemophilia, with the announcement of key clinical trial results from studies of three innovative new recombinant coagulation factors that have been developed to ease the burden of hemophilia care. Phase III studies of CSL Behring’s recombinant single-chain factor VIII (rVIII-SingleChain) and recombinant fusion protein linking recombinant coagulation factor IX with recombinant albumin (rIX-FP) have now been completed, and the results were reported for the first time at this congress. Attendees also heard news of the company’s novel recombinant fusion protein linking recombinant coagulation factor VIIa with recombinant albumin (rVIIa-FP), which has now progressed into clinical trials in patients with hemophilia A or B with inhibitors, and in patients with congenital factor VII deficiency. To celebrate these milestones and recognize the vital contribution made by the patients, clinicians, researchers, and other scientists involved in the studies around the world, CSL Behring hosted a scientific symposium at the ISTH 2015 Congress entitled “Pioneering Therapeutic Proteins in Hemophilia Care Through Innovative Technologies”. The articles in this supplement summarize the presentations from that symposium. In the first paper, Professor Ingrid Pabinger-Fasching from the Medical University of Vienna in Austria tells the story of the unique rVIII-SingleChain for the treatment of hemophilia A. The molecule has been engineered to offer increased stability and a higher binding affinity to von Willebrand factor than current treatments, with an improved pharmacokinetic and pharmacodynamic profile in preclinical and clinical studies. In her paper, Prof. Pabinger-Fasching presents an overview of the latest results from a recently completed phase I/III study with rVIII-SingleChain in previously treated patients with hemophilia A, confirming that this molecule has good hemostatic efficacy and is well tolerated. In the second paper, Dr. Elena Santagostino from Maggiore Hospital and the University of Milan, Italy, provides an update on the results from the PROLONG-9FP clinical development program
for rIX-FP, which has generated considerable interest amongst the hemophilia community. Dr. Santagostino describes the highly anticipated results from two phase III studies of rIX-FP in previously treated adults, adolescents, and pediatrics with severe hemophilia B, which were presented for the first time at the ISTH 2015 Congress. These studies have confirmed that rIX-FP is a truly remarkable new treatment that will transform the lives of many patients with hemophilia B, extending the dosing interval for FIX prophylaxis from every 3 or 4 days to every 7 or 14 days. In the final paper, Prof. Claude Négrier from the Louis Pradel University Hospital in Lyon, France, contributes a paper describing the latest findings from the PROLONG-7FP program, which is the clinical development program for CSL Behring’s novel fusion protein, rVIIa-FP. Initial results from a phase I study of rVIIa-FP in healthy volunteers suggests that this new molecule has a markedly extended half-life compared with the currently available recombinant factor VIIa, offering the potential for more sustained hemostasis and more manageable prophylaxis schedules. Studies in patients with hemophilia A or B and inhibitors, and in those with congenital FVII deficiency, are now underway. The papers in this supplement bring together all the latest findings from the comprehensive clinical development programs underway for CSL Behring’s most advanced recombinant coagulation factors. CSL Behring has always been considered to be a pioneer in the plasma therapeutics industry, and we are delighted that the company has committed to developing these – and other – innovative new recombinant technologies from which our patients may soon be able to benefit. Conflict of interest CN has received research support/honoraria, and/or has participated as a member of the scientific advisory board for Alnylam, Baxter, Bayer, Biogen Idec/SOBI, CSL Behring, Inspiration, LFB, Novo Nordisk, Octapharma, and Pfizer. IP-F has received research/travel support, and/or has participated as a member of the scientific advisory board for Amgen, Baxter, Bayer, Boehringer Ingelheim, CSL Behring, Pfizer, and GSK.
* Corresponding authors at: Universitätsklinik für Innere Medizin I, Klinische Abteilung für Hämatologie und Hämostaseologie, Währinger Gürtel 18–20 A-1090, Wien, Austria. Tel.: +43 1 40400 4952; fax: +43 1 40402 6930. E-mail address: [email protected] (I. Pabinger-Fasching); Centre de Référence de l’Hémophilie, Hôpital Louis Pradel – Hospices Civils de Lyon, Université Claude Bernard, Lyon, France. Tel.: +33 4 7211 8821; fax: +33 4 7211 8817. E-mail address: [email protected] (C. Négrier). 0049-3848/© 2016 Elsevier Ltd. This is an open access article under the CC BY-NC-ND license (http://creativecommons.org/licenses/by-nc-nd/4.0/)