Bioverativ, a global biopharmaceutical company dedicated to transforming the lives of people with rare blood disorders, announced that it will present data showcasing its work to advance therapies for people affected by hemophilia, cold agglutinin disease (CAgD), sickle cell disease and beta-thalassemia at the 59th Annual Meeting of the American Society of Hematology (ASH), December 9 -12 in Atlanta, Georgia.
Highlights at ASH will include an oral presentation of Phase 1/1b safety and efficacy data on BIVV009, an investigational first-in-class, monoclonal antibody developed to treat cold agglutinin disease (CAgD). Cold agglutinin disease is a poorly understood chronic, autoimmune hemolytic anemia with no approved therapies. The company will also share new research that will help characterize the burden of CAgD and the risk of thromboembolic events, and increase the understanding of morbidity and mortality associated with this rare blood disorder. Bioverativ plans to start two Phase 3 clinical trials of BIVV009 in CAgD by the end of the year.
The company will also have several presentations on its hemophilia therapies, including preclinical findings from a first-of-its-kind study using radiolabeled imaging to understand the impact of extravascular distribution of factor IX therapies on joint health. Data supporting extended prophylactic dosing intervals with ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] for hemophilia A and ALPROLIX® [Coagulation Factor IX (Recombinant), Fc Fusion Protein] for hemophilia B will also be discussed in two joint poster presentations with Sobi.
Bioverativ’s oral and poster presentations:
Hemophilia Abstracts
- Extravascular Distribution of Conventional and EHL FIX Products Using In Vivo SPECT Imaging Analysis in Hemophilia B Mice – Poster #1061 – Saturday, December 9, from 5:30-7:30 PM (ET) – Hall A2
- Clinical Outcomes of Weekly Prophylaxis with rFVIIIFc: Longitudinal Analysis of the A-LONG and ASPIRE Study Population – Poster #2368 – Sunday, December 10, from 6:00-8:00 PM (ET) – Hall A2
- Optimization of Clot Formation Under Blood Flow in a Tissue Engineered Blood Vessel – Poster #2304 – Sunday, December 10, from 6:00-8:00 PM (ET) – Hall A2
- Clinical Outcomes of 14-Day Prophylaxis with rFIXFc: Longitudinal Analysis of the B-LONG and B-YOND Study – Poster #3667 – Monday, December 11, from 6:00-8:00 PM (ET) – Hall A2
Cold Agglutinin Disease Abstracts
- Incidence of Thromboembolic Events Is Increased in a Retrospective Analysis of a Large Cold Agglutinin Disease (CAD) Cohort – Poster #928 – Saturday, December 9, from 5:30-7:30 PM (ET) – Hall A2
- Long Term Efficacy, Safety and PK/PD Profile of the Anti-C1s Antibody (BIVV009) in Primary Cold Agglutinin Disease Patients – Oral Presentation #703 – Monday, December 11 at 2:45 PM (ET) –C208 – C210
Beta-Thalassemia and Sickle Cell Disease Abstract
- ST-400 as a Potential Therapy for Beta-Thalassemia and Sickle Cell Disease – Poster #2066 – Saturday, December 9, from 5:30-7:30 PM (ET) – Hall A2
Bioverativ and Sangamo Therapeutics, Inc. have an exclusive worldwide collaboration to develop and commercialize ZFN-mediated gene-edited cell therapies for the treatment of beta-thalassemia and sickle cell disease.
