Emerging Treatments for Cold Agglutinin Disease

• Unmet medical need due to the high frequency of persistent anemia/hemolysis; immunochemotherapy is unsuccessful in at least 25% of cases because of treatment failure or toxicity; and small B-cell clone shows low proliferation activity and is difficult to target efficiently
• Need for rapid acting therapy, especially in specific clinical settings, such as acute and severe exacerbations due to infections, major surgery, trauma, cardiac surgery
• As of June 2021, there were six emerging treatments in trials, including Eculizumab, Bortezomib, Sutimlimab (BIVV009) in the CARDINAL and CADENZA trials, Pegcetacoplan (APL-2) and BIVV020 (both multiple and single IV doses)
• The CADENCE Registry, a global patient registry launched in 2019, is an important tool in treating CAD and will provide perspective longitudinal data to advance understanding of patient demographics, clinical presentation and characteristics, co-morbidities and disease burden, patterns and use of CA