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New Data at ASH Showcase Bioverativ’s Commitment to Transforming Care for People with Hemophilia and Other Rare Blood Disorders

• An oral presentation on BIVV001, an investigational and potentially transformative von Willebrand-independent factor VIII therapy for people with hemophilia A will be given
• Factor replacement is fundamental to the treatment of hemophilia. Data reinforcing the proven long-term efficacy and safety of extended half-life factor therapies ELOCTATE® and ALPROLIX® to be presented in multiple poster sessions
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Thursday, November 1, 2018 9:22 AM

WALTHAM, Mass.--()--Bioverativ Inc., a Sanofi company dedicated to transforming the lives of people with rare blood disorders, today announced it will present data demonstrating its commitment to improving the care of people with hemophilia and other rare blood disorders like sickle cell disease at the 60th Annual Meeting of the American Society of Hematology (ASH), December 1 - 4 in San Diego, California.

“The new data we are presenting at ASH highlight our commitment to protection in hemophilia and our focus on advancing cutting-edge science that has the potential to make a meaningful, positive impact for people with rare blood disorders,” said Tim Harris, Ph.D., DSc, Executive Vice President of Research and Development at Bioverativ.

Highlights will include an oral presentation on the ongoing Phase 1/2a trial for BIVV001 (rFVIIIFc-VWF-XTEN), the first investigational factor VIII therapy with the potential to provide extended protection from bleeds with prophylactic dosing of once weekly or longer. Data presented at the NHF Bleeding Disorders Conference in October showed that a single low dose (25 IU/kg) of BIVV001 extended the half-life of factor VIII to an unprecedented 37 hours, four times longer than the 9.1 hours seen with rFVIII, while achieving high factor activity levels (>5% at seven days). BIVV001 was generally well tolerated with no development of inhibitors. The ASH presentation will include preliminary results from the high-dose cohort of the study.

Results confirming the long-term safety and efficacy of ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] and ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] will also be presented. Of note, final study results from ASPIRE, Bioverativ and Sobi’s long-term extension study of ELOCTATE for hemophilia A, and B-YOND, the companies’ long-term extension study of ALPROLIX for hemophilia B, demonstrating excellent bleed control, joint protection, and adherence over four years will be reported.

Additional poster presentations from Bioverativ’s hemophilia portfolio include an update on the retrospective chart analysis (United States and Canada) on ELOCTATE used for immune tolerance induction (ITI) in severe hemophilia A patients with inhibitors. Treatment of inhibitors remains a significant unmet need in hemophilia. ELOCTATE is not currently approved for ITI in the United States or Canada.

Bioverativ will also present preclinical data on BIVV003, a gene-edited cell therapy candidate for the treatment of people with sickle cell disease, which is being developed in collaboration with Sangamo Therapeutics, Inc. BIVV003 is a non-viral cell therapy that involves gene editing of a patient’s own hematopoietic stem cells (HSCs) using zinc finger nuclease (ZFN) technology. The U.S. Food and Drug Administration accepted the Investigational New Drug application for BIVV003, enabling Bioverativ to initiate a Phase 1/2 clinical trial to assess the safety, tolerability, and efficacy in adults with sickle cell disease. NCT03653247.

Hemophilia

  • BIVV001: The First Investigational Factor VIII Therapy to Break Through the VWF Ceiling in Hemophilia A, with Potential for Extended Protection for One Week or Longer – Poster #636 –December 3 at 10:30 AM -12:00 PM (PT) – Room 30D
  • Real-World Data of Immune Tolerance Induction Using rFVIIIFc in Subjects with Severe Hemophilia A with Inhibitors at High Risk for ITI Failure – Poster #2500 – December 2 at 6:00 – 8:00 PM (PT)
  • Real-World Data on the Use of rFIXFc in Subjects with Hemophilia B for Up to 3.7 Years Demonstrates Improved Bleed Control and Adherence with Reduced Treatment Burden – Poster #2493 – December 2 at 6:00 – 8:00 PM (PT)
  • Changing the Paradigm in Hemophilia Care: Extended Half-Life Products (rFVIIIFc and rFIXFc) - Poster #3514 – December 2 at 6:00 – 8:00 PM (PT)
  • Economic Impact of Immune Tolerance Induction (ITI) with Recombinant Factor VIII Fc Fusion Protein (rFVIIIFc) Compared to Conventional Recombinant Factor VIII (rFVIII) – Poster #3520 – December 2 at 6:00 – 8:00 PM (PT)
  • A Novel Humanized Hemophilia-A Mouse Model to Facilitate Preclinical In Vivo Studies of Human Specific FVIIIa-mimetic Bispecific Antibodies – Poster #2458 - December 2 at 6:00 – 8:00 PM (PT)
  • Phospholipid-Independent Activity of FVIIIa Mimetic Bispecific Antibodies in Plasma – Poster #2461 – December 2 at 6:00 – 8:00 PM (PT)

Hemophilia with Sobi
Bioverativ and Sobi collaborate on the development and commercialization of ALPROLIX and ELOCTATE, which is marketed as Elocta®in Europe.

  • ASPIRE Final Results Confirm Established Safety and Sustained Efficacy for Up to 4 Years of Treatment with rFVIIIFc in Previously Treated Subjects with Severe Hemophilia A – Poster #1192 – December 1 at 6:15 – 8:15 PM (PT)
  • B-YOND Final Results Confirm Established Safety, Sustained Efficacy, and Extended Dosing Interval for Up to 4 Years of Treatment with rFIXFc in Previously Treated Subjects with Severe Hemophilia B – Poster #1214 – December 1 at 6:15 – 8:15 PM (PT)

Sickle Cell Disease
Bioverativ and Sangamo Therapeutics, Inc. have an exclusive worldwide collaboration to develop and commercialize ZFN-mediated gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease.

  • Ex Vivo Gene-Edited Cell Therapy for Sickle Cell Disease: Disruption of the BCL11A Erythroid Enhancer with Zinc Finger Nucleases Increases Fetal Hemoglobin in Plerixafor Mobilized Human CD34+ Cells – Poster # 2190 - December 1 at 6:15 to 8:15 PM (PT)
  • LC-MS Analysis of Anti-Sickling Compounds in Cord Blood Derived RBCs Demonstrates Modification of Fetal Hemoglobin and Globin Chain Binding Preferences – Poster #1074 – December 1 at 6:15 – 8:15 PM (PT)

Product Theaters:
In addition to the data being presented at the Congress, Bioverativ has sponsored two product theaters that will highlight the latest research in hemophilia and cold agglutinin disease. These events are open to all attendees:

  • Uncovering the Risks Associated with C1-activated Hemolysis in Cold Agglutinin Disease (CAD). December 1 at 11:30 AM - 12:30 PM in Room 2, Upper Level of the San Diego Convention Center
  • Think Again: Evaluating Hypotheses and the Future of Treatment in Hemophilia. December 3 at 12:15 – 1:15 PM in Room 2, Upper Level of the San Diego Convention Center

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

About ELOCTATE® /Elocta®
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, Saudi Arabia, Kuwait 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/Elocta, 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 its collaboration partner, Swedish Orphan Biovitrum AB (publ) (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 other countries, 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 US 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 Sickle Cell Disease
Globally, 300,000 people are born with sickle cell disease every year, and approximately 100,000 people are living with sickle cell disease in the United States.4 People with sickle cell disease have a mutation that alters hemoglobin, the protein in red blood cells that carries oxygen to cells throughout the body. The sickle mutation causes red blood cells to have an abnormal sickle or crescent shape, which makes them inefficient in their oxygen-carrying capacity and leads to chronic anemia, vaso-occlusive crises with severe pain, multi-organ damage, complications like stroke, and a shortened life expectancy.

About the Bioverativ and Sobi Collaboration
Bioverativ and Sobi collaborate on the development and commercialization of ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] and ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein], which is marketed as Elocta® in Europe. 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).

About the Bioverativ and Sangamo Collaboration
Bioverativ and Sangamo have an exclusive worldwide collaboration to develop and commercialize ZFN-mediated gene-edited cell therapies for the treatment of beta thalassemia and sickle cell disease. Based on the terms of the agreement, Bioverativ is responsible for conducting the BIVV003 Phase 1/2 clinical trial and subsequent worldwide clinical development, manufacturing, and commercialization.

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.

References

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.
4 World Health Organization; Piel et al. 2013. Lancet 381: 142-51.

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Marianne McMorrow, +1 781-663-4376
media@bioverativ.com