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Press Release

Feb 14, 2018 12:00 UTC

Haemophilia Publishes a Retrospective Analysis Investigating the Use of ELOCTATE® for Immune Tolerance Induction in People with Inhibitors and Severe Hemophilia A

WALTHAM, Mass.--(Business Wire)--Bioverativ Inc. (NASDAQ: BIVV), a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, today announced the publication of a retrospective analysis investigating the use of ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for immune tolerance induction (ITI). These data add to a growing body of evidence supporting the potential of ELOCTATE to induce tolerance in severe hemophilia A patients with inhibitors. This analysis was published online as an early view manuscript in Haemophilia on February 13, 2018.

The development of inhibitors, or antibodies, is one of the most serious complications for people with hemophilia, occurring in approximately 30% of people with severe hemophilia A.Eradication of inhibitors remains the standard of care and ITI therapy is considered to be the only way to eradicate inhibitors in people with hemophilia.2 ITI requires factor therapy to be given regularly over a period of time to train the immune system to accept the treatment without reacting to it. Improvement has typically been seen in 12-18 months, but more difficult cases can take two years or longer.3

In this retrospective analysis, data from 19 patients (7 first-time ITI and 12 rescue patients) treated with ELOCTATE for ITI were evaluated. Findings show that four of the seven first-time ITI patients achieved tolerization in a median of 7.8 months. The remaining three patients have continued on ELOCTATE ITI treatment, with two patients showing a reduction in inhibitor levels. Additionally, in the 12 patients where ITI had been attempted with other factor therapies and failed, seven patients initially achieved a negative inhibitor level in a median 3.3 months, and one patient demonstrated a reduction in inhibitor levels. The remaining four rescue patients did not show a response to ITI with ELOCTATE. At the time of analysis, 16 of 19 patients remained on ELOCTATE prophylaxis treatment or ITI, and no adverse events were reported. Read the study here.

“The development of inhibitors is a tremendous challenge and significant burden for people with severe hemophilia A, and the goal of treatment should be eradication of inhibitors,” said Maha Radhakrishnan, M.D., Senior Vice President of Medical at Bioverativ. “The results of this analysis are encouraging and support the need for additional and ongoing scientific research on ELOCTATE in ITI to determine whether an Fc-based recombinant factor VIII therapy can rapidly tolerize patients with inhibitors. At Bioverativ, we are dedicated to addressing the unmet needs of people with hemophilia.”

Bioverativ, and its collaborator Swedish Orphan Biovitrum AB (publ) (Sobi), have initiated two prospective studies that will further evaluate the use of ELOCTATE in ITI in people with severe hemophilia A and inhibitors (NCT03093480 and NCT03103542).

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, 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 Hemophilia A

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. The World Federation of Hemophilia estimates that approximately 150,000 people are currently diagnosed with hemophilia A worldwide.4

People with hemophilia A experience bleeding episodes that can cause pain, irreversible joint damage and life-threatening hemorrhages. Prophylactic injections of factor VIII can temporarily replace the clotting factor that is needed to control bleeding and prevent new bleeding episodes.5 The World Federation of Hemophilia (WFH) recommends prophylaxis as the optimal therapy as it can prevent bleedings and joint destruction.6

About Bioverativ

Bioverativ is a global biopharmaceutical 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 the potential benefits of ELOCTATE in hemophilia A. These forward-looking statements may be accompanied by such words as “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “intend,” “may,” “plan,” “potential,” “project,” “target,” “will” and other words and terms of similar meaning. You should not place undue reliance on these statements. These statements involve risks and uncertainties that could cause Bioverativ’s actual results to differ materially from those reflected in such statements, including, without limitation, unexpected concerns that may arise from data, findings, analysis or results obtained from research or clinical trials or post hoc analysis of studies, regulatory authorities may require additional information or further studies, regulatory authorities may fail to expand product labeling, and other risks and uncertainties associated with Bioverativ’s drug development and commercialization activities described in the Risk Factors section of Bioverativ’s filings with the Securities and Exchange Commission. These statements are based on Bioverativ’s current beliefs and expectations and speak only as of the date of this press release. Bioverativ does not undertake any obligation to publicly update any forward-looking statements.

1 Witmer and Young, Therapeutic Advances in Hematology. 2013 Feb; 4(1):59-72.
2 Hay and Dimichele. Blood. 2012.
3 World Federation of Hemophilia, About Bleeding Disorders – How does immune tolerance induction work? Available at https://www.wfh.org/en/page.aspx?pid=647. Accessed on December 14, 2017.
4 World Federation of Hemophilia, Annual Global Survey 2016, published in October 2017. Available at: http://www.wfh.org/en/data-collection
5 World Federation of Hemophilia. About Bleeding Disorders – Frequently Asked Questions. Available at: http://www.wfh.org/en/page.aspx?pid=637#Difference_A_B. Accessed on: June 17, 2016
6 Guideline for the management of hemophilia, World Federation of Hemophilia, 2nd edition, http://www1.wfh.org/publications/files/pdf-1472.pdf. Accessed on December 2015

BIOVERATIV
Media:
Marianne McMorrow, +1 781-663-4376
media@bioverativ.com
or
Investor Relations:
Samuel Chase, +1 781-663-4360
IR@bioverativ.com