- Results from a Phase 2 clinical trial, SUSTAIN, assessing Novartis' (NYSE:NVS) crizanlizumab (SEG101) for the treatment of sickle cell-related pain crises (SCPC) showed a significant treatment benefit. The data were presented at the 58th American Society of Hematology (ASH) annual meeting in San Diego and published simultaneously in The New England Journal of Medicine.
- The 198-subject three-arm study met its primary endpoint by demonstrating a statistically significant reduction in the annual rate of SCPC compared to placebo. In the high-dose (5.0 mg/kg) arm, the relative reduction in SCPC was 45.3% (p=0.01) and 32.6% in the low-dose (2.5 mg/kg) arm (p=0.18). Time-to-first SCPC was 2.9x and 2.0x longer, respectively, in the two arms compared to placebo.
- Treatment-related adverse events that were elevated at least 2x over placebo were arthralgia, pruritis, vomiting, chest pain, diarrhea, fatigue, myalgia, musculoskeletal chest pain, abdominal pain, influenza, oropharyngeal pain and road traffic accident.
- Crizanlizumab is a humanized monoclonal antibody that binds to P-selectin, a protein found on the surface of endothelial cells and platelets. P-selectin drives the vaso-occlusive process, a painful complication of sickle cell disease that occurs when the circulation of blood vessels is obstructed by sickled red blood cells. One of the most severe forms of SCPC is acute chest syndrome which occurs when blood flow to the lungs is restricted.
- The company obtained the rights to the product candidate via its recent acquisition of Selexys Pharmaceuticals, following through on an option to buy the company it held since 2012. Its development is ongoing.