WALTHAM, Mass.--(BUSINESS WIRE)--May 14, 2018--Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, will present data at the World Federation of Hemophilia (WFH) 2018 World Congress, in Glasgow, Scotland, May 20-24, demonstrating the company’s ongoing commitment to transforming the standard of care for people with hemophilia.

“We are committed to making a meaningful, positive impact for people with hemophilia by improving patient outcomes and advancing cutting-edge research,” said Joachim Fruebis, Senior Vice President of Development at Bioverativ. “We look forward to sharing data on BIVV001, an investigational and potentially transformative von Willebrand factor independent therapy that has been developed to further extend protection from bleeds with prophylactic dosing of once weekly or longer for people with hemophilia A.”

BIVV001 (rFVIIIFc-VWF-XTEN) is the first factor VIII therapy in clinical development that is designed to overcome the half-life ceiling imposed by von Willebrand factor, and it builds on the half-life extension provided by Fc fusion technology. Preliminary pharmacokinetic and safety data from a Phase 1/2a study of BIVV001 will be presented as an oral, late-breaking presentation.

Bioverativ will also present real-world data demonstrating improvements in quality-of-life measures, like physical activity and joint pain, in patients treated prophylactically with ELOCTATE ® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for hemophilia A and ALPROLIX ® [Coagulation Factor IX (Recombinant), Fc Fusion Protein], for hemophilia B, when compared to short-acting factor treatments. ELOCTATE and ALPROLIX are the leading extended half-life hemophilia therapies with well-established safety and efficacy profiles and the most real-world experience.

Bioverativ will present a total of six presentations, including a joint presentation with Sobi™.

Bioverativ Presentations

BIVV001 – a novel, weekly dosing, VWF-independent, extended half-life FVIII therapy: first-in-human safety, tolerability, and pharmacokinetics: Monday, May 21, 10:15 – 11:45. Late-breaker Comparisons in physical activity and bleed rate among severe hemophilia A and B patients on prophylactic treatment with rFVIIIFc/rFIXFc vs conventional rFVIII/rFIX: Monday, May 21, 16:30-18:00. Poster #50 Impact of pain on health-related quality of life in persons with hemophilia from the Hemophilia Utilization Group Studies Part VI (HUGS VI): USA Experience: Tuesday, May 22, 16:30-18:00. Oral presentation Distribution of rFIXFc and FIX using in vivo PET imaging analysis in non-human primates: Monday, May 21, 14:15-15:15. Oral presentation Allosteric activation of Factor IXa by an antibody binding to the protease domain: Monday, May 21, 16:30-18:00. Poster #36

Bioverativ and Sobi Joint Presentation

Economic impact of recombinant factor VIII Fc fusion protein (rFVIIIFc) compared to conventional factor VIII for immune tolerance induction (ITI) of Hemophilia A patients with inhibitors: Monday, May 21, 16:30-18:00. Poster #77

All oral and poster presentations can be accessed at the WFH 2018 World Congress website here.

In addition, Bioverativ and Sobi will co-host two scientific symposia at the congress.

. Monday, May 21, 18:15 – 19:45, Hall 3, Scottish Event Campus. The session will be chaired by K. John Pasi, Professor, MD, PhD, Barts and the London School of Medicine and Dentistry, London and will be open to healthcare practitioners only. Tuesday, May 22, 12:30-14:00, Hall 2, Scottish Event Campus. The session will be chaired by Victor Blanchette, MD, MA, MB, Pediatric Thrombosis and Hemostasis Program, The Hospital for Sick Children, Toronto and is open to all congress attendees.

About BIVV001 BIVV001(rFVIIIFc-VWF-XTEN) is a novel, investigational factor VIII therapy that is designed to extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. It is currently the only therapy in clinical development designed to overcome the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. BIVV001 builds on the Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides to potentially extend its time in circulation. BIVV001 was granted orphan drug designation by the Food and Drug Administration in August 2017.

About ELOCTATE ® ELOCTATE ® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] is a recombinant clotting factor therapy developed for hemophilia A using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling ELOCTATE to use a naturally occurring pathway to extend the time the therapy remains in the body. While Fc fusion technology has been used for more than 15 years, Bioverativ and Swedish Orphan Biovitrum AB (publ) (Sobi) have optimized the technology and are the first companies to utilize it in the treatment of hemophilia. ELOCTATE is manufactured using a human cell line in an environment free of animal and human additives.

ELOCTATE is approved and marketed by Bioverativ in the United States, Japan and Canada. It is also approved in Australia, New Zealand, Brazil and other countries, and Bioverativ has marketing rights in these regions. It is also approved as Elocta® in the European Union, Switzerland, Iceland, Liechtenstein, Norway and other countries where it is marketed by Sobi.

As with any factor replacement therapy, allergic-type hypersensitivity reactions and development of inhibitors may occur in the treatment of hemophilia A. Inhibitor development has been observed with ELOCTATE, including in previously untreated patients. For more information, please see the full U.S. prescribing information for ELOCTATE. Note that the indication for previously untreated patients is not included in the EU Product Information for Elocta.

About ALPROLIX ® ALPROLIX ® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] is a recombinant clotting factor therapy developed for hemophilia B using Fc fusion technology to prolong circulation in the body. It is engineered by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body), enabling ALPROLIX to use a naturally occurring pathway to extend the time the therapy remains in the body (half-life). While Fc fusion technology has been used for more than 15 years, Bioverativ and Sobi have optimized the technology and are the first companies to utilize it in the treatment of hemophilia. ALPROLIX is manufactured using a human cell line in an environment free of animal and human additives.

ALPROLIX is approved and marketed by Bioverativ for the treatment of hemophilia B in the United States, Japan and Canada. It is also approved in Australia, New Zealand, Brazil and other countries, and Bioverativ has marketing rights in these regions. It is also authorized in the European Union, Iceland, Liechtenstein, Norway and Switzerland, where it is marketed by Sobi.

Allergic-type hypersensitivity reactions and development of inhibitors have been observed with ALPROLIX in the treatment of hemophilia B, including in previously-untreated patients. For more information, please see the full U.S. prescribing information for ALPROLIX. Note that the indication for previously-untreated patients is not included in the EU Product Information.

About Hemophilia A and B Hemophilia is a rare, genetic disorder in which the ability of a person's blood to clot is impaired. Hemophilia A occurs in about one in 5,000 male births annually, and more rarely in females. Hemophilia B occurs in about one in 25,000 male births annually, and more rarely in females. The World Federation of Hemophilia estimates that approximately 180,000 people are currently diagnosed with hemophilia A and B worldwide. 1

People with hemophilia A or B experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Prophylactic infusions of factor VIII or IX can temporarily replace the clotting factors that are needed to control bleeding and prevent new bleeding episodes. 2 The World Federation of Hemophilia recommends prophylaxis as the optimal therapy as it can prevent bleedings and joint destruction. 3

About Bioverativ, a Sanofi company Bioverativ, a Sanofi company, is dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development, and commercialization of innovative therapies. Bioverativ is committed to actively working with the blood disorders community, and its hemophilia therapies when launched represented the first major advancements in hemophilia treatment in more than two decades. For more information, visit www.bioverativ.com or follow  @bioverativ  on Twitter.

About the Bioverativ and Sobi Collaboration Bioverativ and Sobi collaborate on the development and commercialization of ALPROLIX and ELOCTATE®/Elocta® [Antihemophilic Factor (Recombinant), Fc Fusion Protein]. Bioverativ has final development and commercialization rights in North America and all other regions in the world excluding the Sobi territory, and has manufacturing responsibility for ELOCTATE and ALPROLIX. Sobi has final development and commercialization rights in the Sobi territory (essentially Europe, North Africa, Russia and most Middle Eastern markets). In September 2014, Sobi elected to add the rFVIIIFc-VWF-XTEN fusion molecule for the potential treatment of hemophilia A to its collaboration agreement with Bioverativ.

1 World Federation of Hemophilia. Annual Global Survey 2015, published in October 2016. Available at:  http://www1.wfh.org/publication/files/pdf-1669.pdf. Accessed on May 23, 2017. 2 World Federation of Hemophilia. About Bleeding Disorders – Frequently Asked Questions. Available at:  http://www.wfh.org/en/page.aspx?pid=637. Accessed on May 23, 2017. 3 World Federation of Hemophilia. Guideline for the management of hemophilia, 2nd edition. Available at:  http://www1.wfh.org/publication/files/pdf-1472.pdf. Accessed on May 23, 2017.

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CONTACT: Bioverativ Inc.

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PUB: 05/14/2018 09:02 AM/DISC: 05/14/2018 09:02 AM

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