New Data To Be Presented At Isth Underscore Bioverativ’s Commitment To Advance Hemophilia Care

WALTHAM, Mass.--(BUSINESS WIRE)--Bioverativ Inc. (NASDAQ: BIVV), a global biotechnology company focused on the discovery, development and commercialization of innovative therapies for hemophilia and other rare blood disorders, today announced clinical data from its leading extended half-life therapies ALPROLIX^® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] and ELOCTATE^® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] and preclinical data from its hemophilia pipeline, including BIVV001 for hemophilia A, will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, taking place in Berlin, Germany, from July 8-13, 2017.

“We are excited to attend our first ISTH Congress as Bioverativ, and the breadth of data we will be presenting underscores our commitment to scientific innovation for people with hemophilia and demonstrates our growing leadership in this field,” said Tim Harris, Ph.D., D.Sc., Executive Vice President of Research and Development at Bioverativ. “Our data at ISTH will also highlight our work to develop a pipeline of novel therapies for hemophilia, including several presentations on BIVV001, our potential next-generation factor VIII therapy designed to extend protection from bleeds with prophylaxis dosing of once weekly or longer.”

Bioverativ will present a total of 19 hemophilia-related posters, including nine joint presentations with Swedish Orphan Biovitrum AB (publ) (Sobi™) during the Congress. Research supporting Bioverativ’s pipeline programs will be presented in multiple poster and oral presentations including data on the investigational compound BIVV001 (rFVIIIFc-VWF-XTEN), which is the first molecule of its kind to fuse four different proteins for the treatment of hemophilia A. The U.S. Food and Drug Administration recently accepted the Investigational New Drug application for BIVV001 and Bioverativ is on track to initiate a Phase 1/2a trial in the second half of the year.

In addition, new findings from the ASPIRE and B-YOND extension studies will support ELOCTATE’s and ALPROLIX’s potential to deliver improved long-term joint-health and quality-of-life outcomes for people with hemophilia A and B, respectively. A late-breaking poster presentation on the use of ELOCTATE for immune tolerance induction will also be presented.

Symposium:
In addition to the data being presented at the Congress, Bioverativ and Sobi will co-host a company-sponsored scientific symposium, “Fc Extended Half-Life Recombinant Factor Concentrates: Recent Updates on Haemophilia Management and Treatment Goals,” on July 10 at 13:15 -14:30 CEST. Supported by global patient case reports, the session will focus on features of Fc fusion factors, evolving technologies and methodologies in hemophilia management, and real-world experience with initiating and continuing treatment with ELOCTATE and ALPROLIX and their impact on long-term treatment goals.

Highlights of Bioverativ and Sobi’s joint presentations on ALPROLIX and ELOCTATE at ISTH can be found here.

Highlights from Bioverativ’s preclinical presentations include:
BIVV001 (rFVIIIFc-VWF-XTEN)-Focused Abstracts

• Recombinant FVIIIFc-VWF-XTEN (BIVV001) Demonstrates Equivalent Thrombin Generation and Whole Blood Clotting Profiles to Advate – Poster PB 147 – Monday, July 10, 2017 from 12:00 – 13:15 CEST
• Evaluation of Recombinant FVIIIFc-VWF-XTEN (BIVV001) Activity in One-stage Clotting and Chromogenic Assays and its Correlation with in vivo Efficacy in Hemophilia A Mice – Poster PB 149 – Monday, July 10, from 12:00 – 13:25 CEST
• Recombinant FVIIIFc-VWF-XTEN Promotes Normal Fibrin Formation, Structure, and Stability – Poster PB 139 – Monday, July 10 from 12:00 – 13:25 CEST
• rFVIIIFc-VWF-XTEN Demonstrates Comparable Efficacy to Recombinant Human FVIII in Mice by Acute Bleeding and Intravital Microscopy Models – Poster PB 143 – Monday, July 10 from 12:00 – 13:25 CEST

BIVV002 (rFIXFc-XTEN)-Focused Abstract
BIVV002 is an investigational, recombinant factor IX therapy designed for potential subcutaneous dosing once weekly or longer for people with hemophilia B.

• The Pharmacokinetic Profiles of Intravenously and Subcutaneously Administered Recombinant Factor IX Fc-XTEN in Cynomolgus Monkeys – Oral Presentation OC 40.2 – Tuesday, July 11 from 17:45 – 19:00 CEST

FVIII Mimetic Bispecific Antibody Program-Focused Abstracts
A preclinical-stage pipeline program focused on the development of a non-factor bispecific antibody for the treatment of people hemophilia A with or without inhibitors.

• Identification of FIXa- and FX-specific Antibodies for the Generation of Bispecific Antibodies with FVIIIa-like Activity – Oral Presentation #OC 47.5 – Tuesday, July 11, from 17:45 – 19:00 CEST
• Challenges in Quantifying FVIIIa-mimetic Bispecific Antibody Activity Relative to FVIII for Hemophilia A Treatment – Oral Presentation OC 47.3 - Tuesday, July 11, from 17:45 – 19:00 CEST
• Effect of Phospholipid Vesicle Composition on Activity of FVIIIa-mimetic Bispecific Antibodies – Poster PB 1127 – Tuesday, July 11 from 12:00 – 13:25 CEST

Gene Therapy-Focused Abstract
A collaboration program with the San Raffaele Telethon Institute for Gene Therapy (SR-TIGET) to jointly develop gene therapies for the treatment of both hemophilia A and B.

• Persistent Expression of FVIII Following Intravenous Administration of Lenti-viral Vectors in Neonatal Hemophilia A Mouse and Dog Models – Oral Presentation OC 75.2 – Thursday, July 13 from 09:30 – 10:45 CEST

Abstracts are available through the ISTH 2017 website.

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 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 Bioverativ
Bioverativ is a global biotechnology company dedicated to transforming the lives of people with hemophilia and other rare blood disorders through world-class research, development and commercialization of innovative therapies. Launched in 2017 following separation from Biogen Inc., Bioverativ builds upon a strong heritage of scientific innovation and is committed to actively working with the blood disorders community. The company’s mission is to create progress for patients where they need it most 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.

Bioverativ Safe Harbor
This press release contains forward-looking statements, including statements about early stage research and any potential benefits or clinical effects relating thereto, and timing of anticipated clinical trials and enrollment thereof. These statements may be identified by words such as “believe,” “expect,” “may,” “plan,” “potential,” “will” and similar expressions, and are based on Bioverativ’s current beliefs and expectations. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early stage research or clinical trials may not be indicative of full results or results from clinical trials and do not ensure regulatory approval. Factors which could cause actual results to differ materially from Bioverativ’s current expectations include uncertainties relating to the initiation, enrollment and completion of stages of clinical trials, unexpected concerns may arise from data, findings, analysis or results obtained during research and clinical trials, regulatory authorities may require additional information or further studies, or may fail or refuse to approve or may delay approval of product candidates, or Bioverativ may encounter other unexpected hurdles. For more detailed information on the risks and uncertainties associated with Bioverativ’s drug development and commercialization activities, please review the Risk Factors section of Bioverativ’s most recent annual or quarterly report filed with the Securities and Exchange Commission. Any forward-looking statements speak only as of the date of this press release and Bioverativ assumes no obligation to update any forward-looking statements.

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.