WALTHAM, Mass. & STOCKHOLM--(BUSINESS WIRE)--Bioverativ Inc. (NASDAQ: BIVV) and Swedish Orphan Biovitrum AB (publ) (Sobi™) (STO: SOBI) today announced that new data from their extended half-life therapies, ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] and ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein], marketed as Elocta® in Europe and the Middle East, will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2017 Congress, taking place in Berlin, Germany, from July 8-13, 2017.
Researchers and investigators will present updated analyses of long-term follow-up data from the ASPIRE and B-YOND extension studies in adults, adolescents and children, further supporting the existing safety and efficacy of ELOCTATE and ALPROLIX. Data from the first and only longitudinal study of joint health in patients treated prophylactically with extended half-life therapies will also be presented. These data and the additional findings reported on joint health and long-term quality-of-life outcomes reflect the companies’ commitment to addressing areas of unmet need in hemophilia.
“Debilitating joint disease is a common and challenging complication for people with hemophilia,” said Maha Radhakrishnan, MD, Senior Vice President of Medical at Bioverativ. “New data supporting ELOCTATE’s and ALPROLIX’s impact on long-term joint health when used prophylactically will be presented at ISTH, and reflect our continued focus on improving the care and clinical outcomes for people living with hemophilia.”
Most bleeding events for people with severe hemophilia A and B occur in joints, with joint damage being the most common complication of the condition.1 Data presented at ISTH will demonstrate improvements in modified hemophilia joint health scores, low target joint annualized bleed rates (ABRs) and target joint resolution for people receiving prophylactic treatment with ELOCTATE and ALPROLIX. The long-term safety and efficacy of these products will also be presented at the Congress.
“New data will reinforce that ELOCTATE and ALPROLIX treatment can maintain low ABRs, therefore helping improve the long-term quality-of-life for people with hemophilia A and B,” said Krassimir Mitchev, MD, PhD, Vice President and Medical Therapeutic Area Head of Haemophilia at Sobi. “Based on hemophilia-related quality-of-life measures, findings will show that improvements were seen in patients utilizing these therapies over several years, with the most pronounced improvement in areas including physical health and sports and leisure.”
The development of inhibitors to factor replacement therapy is one of the most serious complications for people with hemophilia, and immune tolerance induction (ITI) treatment can represent a significant burden for patients and their quality of life. A retrospective chart analysis (United States and Canada) on ELOCTATE used for ITI in severe hemophilia A patients with inhibitors will also be discussed in a late-breaking poster presentation. ELOCTATE is not currently approved for immune tolerance induction in the United States or Canada.
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, plus real-world experience with initiating and continuing treatment with ELOCTATE and ALPROLIX and their impact on long-term treatment goals.
Presentation highlights include:
Immune Tolerance Induction Late-Breaking Abstract
- rFVIIIFc For Immune Tolerance Induction in Severe Hemophilia A Patients With Inhibitors – A Retrospective Analysis – Poster LB 04 - Monday, July 10 from 12:00 – 13:15 CEST
Joint Health-Focused Abstracts
- Long-Term Impact of rFVIIIFc Prophylaxis in Paediatric, Adolescent, and Adult Subjects with Target Joints and Severe Haemophilia A – Poster #212 – Monday, July 10, 12:00 -13:15 CEST
- Longitudinal Modified Hemophilia Joint Health Scores (mHJHS) in Children, Adolescents, and Adults with Severe Hemophilia A with Long-term rFVIIIFc Prophylaxis – Poster #946 – Tuesday, July 11, 12:00-13:15 CEST
- Target Joint Outcomes with Prophylaxis with rFIXFc in Adults and Adolescents with Hemophilia B: Updated Results from B-YOND – Poster #961 – Tuesday, July 11, 12:00-13:15 CEST
Quality-of-Life Outcomes-Focused Abstracts
- Long-Term Quality-of-Life Outcomes with rFIXFc in Adults with Hemophilia B: Results from B-LONG and B-YOND – Poster #968 – Tuesday, July 11, 12:00-13:15 CEST
- Long-Term Quality-of-Life Outcomes with rFVIIIFc Prophylaxis in Adult Subjects with Severe Hemophilia A – Poster #1783 – Wednesday, July 12, 12:00-13:15 CEST
Long-Term Efficacy and Safety-Focused Abstract
- Efficacy and Safety of rFVIIIFc Prophylaxis in Pediatric, Adolescent, and Adult Subjects with Severe Hemophilia A Over 3-4 Years: The ASPIRE Study – Poster #1774 – Wednesday, July 12, 12:00-13:15 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® /Elocta®
ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] is a recombinant clotting factor therapy developed for haemophilia 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 haemophilia. 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 haemophilia 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.
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 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).
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.
Sobi is an international specialty healthcare company dedicated to rare diseases. Sobi’s mission is to develop and deliver innovative therapies and services to improve the lives of patients. The product portfolio is primarily focused on Haemophilia, Inflammation and Genetic diseases. Sobi also markets a portfolio of specialty and rare disease products across Europe, the Middle East, North Africa and Russia for partner companies. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2016, Sobi had total revenues of SEK 5.2 billion (USD 608 M) and about 760 employees. The share (STO: SOBI) is listed on Nasdaq Stockholm. More information is available at www.sobi.com.
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. 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.
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.