New haemophilia data at ISTH congress to highlight extension study outcomes and paediatric use

- Full results from Kids B-LONG Alprolix® paediatric study to be released for the first time -

- Interim findings to be presented from ASPIRE, an open label extension study with Eloctate®/Elocta® in adults and children with haemophilia A -

Swedish Orphan Biovitrum AB (publ) (Sobi) and collaboration partner Biogen will present 23 company-sponsored platform and poster presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2015 congress taking place in Toronto, Canada, 20-25 June 2015. The data presented underscore the companies’ ongoing commitment to haemophilia through continued research within the joint development programmes in haemophilia.

Data presented will include a late-breaking platform presentation from the Kids B-LONG study detailing the safety and efficacy of Alprolix/rFIXFc [Coagulation Factor IX (Recombinant), Fc Fusion Protein] in children with haemophilia B. This is the first time full results from the Kids B-LONG study will be publicly presented. An interim analysis from the ASPIRE study will also be presented focussing on the long-term safety and efficacy of Eloctate/Elocta/rFVIIIFc [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for the prevention and treatment of bleeding in previously treated adults and adolescents with haemophilia A. ASPIRE is a multi-year extension study for people who completed the pivotal, phase 3 A-LONG or Kids A-LONG studies.

“Our presentations at ISTH will contribute to advance medical scientific insight in the haemophilia community,” said Birgitte Volck, senior vice president, Chief Medical Officer at Sobi. “We believe that the comprehensive, growing body of Eloctate/Elocta and Alprolix clinical evidence will provide clinicians with a greater understanding of the potential these product candidates may have to improve care for people with haemophilia.”

Eloctate and Alprolix have the potential to improve clinical outcomes and extend the interval between prophylactic injections.[1]^,[2] They were developed using a process called Fc fusion, which was designed to prolong a therapy’s circulation in the body using a naturally occurring pathway. While Fc fusion has been used for more than 15 years, Biogen and Sobi are the only companies to apply it in the haemophilia space.

Highlights of Sobi and Biogen’s data at the meeting:

Eloctate/Elocta-focused abstracts

• •Safety and efficacy of recombinant factor VIII fusion protein (rFVIIIFc) for the prevention and treatment of bleeding in previously-treated adult and adolescent subjects with haemophilia A: Interim analysis of the ASPIRE study – Poster #235 – Monday, 22 June, 17:30-18:15 EST
  
  
• •Treatment of bleeding with recombinant factor VIII Fc fusion protein in previously-treated paediatric subject with haemophilia A in the phase 3 Kids A-LONG study – Poster #239 – Monday, 22 June, 17:30-18:15 EST
  
  
• •Indirect comparisons of factor consumption, bleeding rates, and infusion frequencies during routine prophylaxis with recombinant factor VIII Fc fusion protein and other recombinant factor VIII products – Poster #170 – Monday, 22 June, 17:30-18:15 EST

Alprolix-focused abstracts

• •Study of recombinant factor IX Fc fusion protein in children with haemophilia B – late breaking oral session #009 – Wednesday, 24 June, 08:45-09:00 EST
• •Indirect comparisons of factor consumption, bleeding rates, and infusion frequencies during routine prophylaxis with recombinant factor IX Fc fusion protein and other recombinant factor IX products – Poster #171 – Monday, 22 June, 17:30-18:15 EST

Clinical haemophilia abstract

• •A retrospective study on the current treatment practice of haemophilia A and B in the United Kingdom – Poster #299 – Wednesday, 24 June, 17:30-18:15 EST

Full session details and data presentation listings for the 2015 Congress can be found on the ISTH website at https://www.isth.org/page/2015Microsite/.

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About Haemophilia A and B

Haemophilia is a rare, genetic disorder in which the ability of a person’s blood to clot is impaired. Haemophilia A occurs in about one in 5,000 male births annually and haemophilia B occurs in about one in 25,000 male births annually, both more rarely in females. Worldwide, it is estimated that more than 400,000 people are living with haemophilia.

Haemophilia A is caused by having substantially reduced or no factor VIII activity, while haemophilia B is caused by having substantially reduced or no factor IX activity; factor VIII and factor IX are needed for normal blood clotting. People with haemophilia A or B experience prolonged bleeding episodes that can cause pain, irreversible joint damage and life-threatening haemorrhages. Prophylactic injections of factor VIII or IX can temporarily replace the missing clotting factors that are needed to control bleeding and prevent new bleeding episodes.

About Eloctate/Elocta (rFVIIIFc)

rFVIIIFc is a long-acting recombinant factor VIII Fc fusion protein product candidate for people with haemophilia A. Eloctate [Antihemophilic Factor (Recombinant), Fc Fusion Protein], is the first recombinant clotting factor VIII therapy with prolonged circulation in the body for adults and children with haemophilia A approved in the United States, Canada, Australia and Japan. Elocta was submitted to the European Medicines Agency (EMA) for regulatory approval in Europe in October 2014. Eloctate/Elocta was developed by fusing B-domain deleted factor VIII to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). It is believed that this enables Eloctate/Elocta to use a naturally occurring pathway to prolong the time the therapy remains in the body.

About Alprolix (rFIXFc)

rFIXFc is a long-acting recombinant factor IX Fc fusion protein product candidate for people with haemophilia B. Alprolix [Coagulation Factor IX (Recombinant), Fc Fusion Protein], is the first recombinant, clotting factor therapy with prolonged circulation in the body for adults and children with haemophilia B, approved in the United States, Canada, Australia and Japan. Alprolix was submitted to EMA for regulatory approval in Europe in June 2015. Alprolix was developed by fusing factor IX to the Fc portion of immunoglobulin G subclass 1, or IgG1 (a protein commonly found in the body). It is believed that this enables Alprolix to use a naturally occurring pathway to prolong the time the therapy remains in the body.

About the Sobi and Biogen collaboration

Sobi and Biogen are collaboration partners in the development and commercialisation of Elocta/Eloctate/rFVIIIFc for haemophilia A and Alprolix/rFIXFc for the treatment of haemophilia B. Sobi has final development and commercialisation rights for Elocta/rFVIIIFc in the Sobi territories (Europe, North Africa, Russia and certain Middle Eastern markets), as well as the opt-in right to assume final development and commercialisation of Alprolix/rFIXFc in Europe, Russia, certain countries in the Middle East and North Africa. Biogen leads development for Elocta/Eloctate/rFVIIIFc and Alprolix/rFIXFc, has manufacturing rights, and has commercialisation rights in North America and all other regions in the world excluding the Sobi territories.

About Sobi

Sobi is an international specialty healthcare company dedicated to rare diseases. Our 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. We also market a portfolio of specialty and rare disease products for partner companies across Europe, the Middle East, North Africa and Russia. Sobi is a pioneer in biotechnology with world-class capabilities in protein biochemistry and biologics manufacturing. In 2014, Sobi had total revenues of SEK 2.6 billion (USD 380 M) and about 600 employees. The share (STO: SOBI) is listed on NASDAQ OMX Stockholm. More information is available at www.sobi.com.

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[1] Shapiro et al. J Thromb Haemost. 2014; 12(11):1788-1800.

[2]Powell et al. Br J Haematol. 2015; 168(1):113-23.