The 2017 International Society of Thrombosis and Hemostasis Congress was held July 8th through the 13th in Berlin, Germany.

Clinicians and researchers attended the conference to gain new insights into the immediate and future scientific and clinical opportunities and challenges related to thrombosis, hemostasis, and hematology.

A recent report highlighted how the market for hemophilia treatments is expected to experience modest growth over the next few years due to factors such as higher rates of routine prophylaxis in children and adult patients diagnosed with hemophilia A and B well as the introduction and uptake of novel drugs like long-acting factors and alternative coagulation promoters (ACPs).

Still, pharmaceutical companies made numerous presentations highlighting the progress they were making in this treatment space.

Bioverativ was one of notable companies unveiling a series of poster presentations on their pipeline of blood disorder drugs. The company,spun off from pharmaceutical firm Biogen in February 2017, purely focuses on treatments for hemophilia and rare blood diseases.

Drug Discovery & Development interviewed Tim Harris, Ph.D., DSc, the Executive Vice President of Research and Development at Bioverativ via email regarding clinical data for lead candidates Eloctate and Alprolix as well insights into other drugs in the firm’s pipeline.

DD&D: How has spinning off from Biogen and becoming an independent company been helpful to the clinical development goals? Have there been any drawbacks?

Tim Harris: As an independent company solely focusing on hemophilia and rare blood disorders, it allows us to bring a greater focus on the hemophilia community, including advancing our pipeline of novel hemophilia therapies and leading extended half-life therapies for people with hemophilia A and B.

Since our launch, we have continued to expand global access to Eloctate and Alprolix, our leading extended half-life hemophilia therapies, while also advancing our novel pipeline of programs for rare blood disorders, including investigational BIVV001 for hemophilia A, which is on track to enter Phase 1/2a trials later this year.

BIVV001 is an investigational factor VIII therapy designed to extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A.  We have also strengthened our pipeline of innovative therapies with the recent acquisition of True North Therapeutics, a private, clinical-stage biotechnology working in the field of rare, benign hematology.

DD&D: What is unique about the results from the ASPIRE and B-YOND extension studies? Essentially, what edge does this data give Eloctate and Alprolix when it comes to treating hemophilia?

Harris: Bioverativ has 10 poster presentations at ISTH highlighting new data from the ASPIRE (Eloctate) and B-YOND (Alprolix) extension studies, including the first and only longitudinal study on joint health in the field of extended half-life therapies (Eloctate). These collective data demonstrate favorable outcomes in joint health with prophylactic use of Eloctate and Alprolix in severe hemophilia A or B.

DD&D: What are the next steps for testing Eloctate and Alprolix?

Harris: Bioverativ is focused on advancing Eloctate and Alprolix, as well as our pipeline programs, to help further address areas of unmet medical need in hemophilia. We are focused on advancing research on meaningful clinical outcomes, including long-term joint health, and we are also studying the formation and treatment of inhibitors, the single greatest challenge for people with hemophilia.

DD&D: How does the partnership with Sobi work? What role do they play in the development process? Will you seek out other companies to partner with when it comes to developing the other treatments in your pipeline?

Harris: Bioverativ and Swedish Orphan Biovitrum (Sobi) are collaborators in the development and commercialization of Eloctate (Elocta) for hemophilia A and Alprolix for hemophilia B. Bioverativ leads development for the two drugs, has responsibility for manufacturing, and has commercialization rights in North America and all other regions in the world excluding the Sobi territory. Sobi has the rights to final development and commercialization of Eloctate and Alprolix for the Sobi territories (essentially, Europe, North Africa, Russia and certain Middle Eastern markets).

DD&D: Discuss the other products in your pipeline. What type of data are you hoping to see from continued clinical development? Also, how will other products in your pipeline compliment Eloctate and Alprolix?

Harris: Our lead hemophilia pipeline candidate, BIVV001 (also known as rFVIIIFc-VWF-XTEN), is a novel, investigational factor VIII therapy designed to potentially extend protection from bleeds with prophylaxis dosing of once weekly or longer for people with hemophilia A. BIVV001 builds on the company’s existing Fc fusion technology by adding a region of von Willebrand factor and XTEN polypeptides with the goal to further extend the time of factor VIII in circulation. It is the only investigational factor VIII therapy in development that is designed to overcome the von Willebrand factor ceiling, which is believed to impose a half-life limitation on current factor VIII therapies. The company’s Investigational New Drug (IND) application for BIVV001 was accepted in June and is on track to begin Phase 1/2a trials in the second half of this year.

Bioverativ has several preclinical hemophilia programs, including BIVV002, an investigational, recombinant factor IX therapy designed for potential subcutaneous dosing once weekly or longer for people with hemophilia B.

With the acquisition of True North Therapeutics, Bioverativ strengthens its pipeline with the addition of BIVV009 (formerly TNT009), a first-in-class monoclonal antibody in development for cold agglutinin disease (CAgD). There are no approved treatments for CAgD, which is a rare and chronic autoimmune hemolytic condition that often leads to severe anemia. People living with CAgD can suffer with a significant disease burden, including frequent blood transfusions, crippling fatigue, and an increased risk of life-threatening thrombotic events such as pulmonary embolism and stroke.

BIVV009 has received breakthrough therapy designation from the U.S. FDA for the treatment of hemolysis in patients with primary CAgD, and orphan drug designation from the FDA and the European Medicines Agency. Late-stage clinical development planning for BIVV009, including a registrational program, is underway.

DD&D: Finally, what are your thoughts on the hemophilia market and blood disease space overall?

Harris: There have been a lot of great advances in hemophilia over the past few years, and Bioverativ was a pioneer with the development of the first extended half-life factors for hemophilia A and B. However, significant unmet needs still remain, and we intend to address that. The development of inhibitors is particularly important challenge and, based on the data presented as a late-breaker at ISTH, we will begin two phase IV studies later this year. The exciting and innovative science that is coming along and the development of new factor therapies, including further extending half-life of products, will potentially change the treatment and care of patients in the next few years. Of course, gene therapy looks promising, although we expect that is still a number of years away as there as still many unknowns to address, including level of gene expression.

As Bioverativ grows, we are working on unlocking answers to many rare blood disorders, including sickle cell disease and CAgD, disorders that have inadequate treatment options. We strive to develop innovative therapies, grounding ourselves in the science, as we strongly believe that science matters, because patients matter.