New data from a post hoc analysis of the Phase II SUSTAIN study of crizanlizumab -- a once-a-month, humanized anti-P-selectin monoclonal antibody infusion being investigated for the treatment of sickle cell disease (SCD) -- shows greater reductions of vaso-occlusive crises (VOCs) in patients who were adherent to the treatment protocol. The data were presented during the 60th Annual Meeting of the American Society of Hematology (ASH) in San Diego.
Sickle cell VOCs are painful complications of the disease and the main reason why patients seek medical care in hospitals,. These crises are triggered by multi-cell adhesion, or clusters of cells that block blood flow, and are associated with increased morbidity and mortality,. Currently, treatment options to prevent VOCs are limited. By targeting P-selectin, crizanlizumab reduces multicellular adhesion,.
"Patients with sickle cell disease experience recurrent and severe episodes of debilitating pain that often require medical attention and emergency medical care," said Kenneth Ataga, MD, Director of the Center for Sickle Cell Disease at the University of Tennessee Health Science Center at Memphis, and Principal Investigator of the SUSTAIN analysis. "It is encouraging that these data show treatment per protocol not only reduced the frequency of painful crises, but also increased the number patients with no crises at all. These findings underscore the potential of crizanlizumab and the importance of proactive management of sickle cell disease."
In the analysis of the per protocol population of the 52-week SUSTAIN study, which compared the P-selectin inhibitor crizanlizumab with placebo in patients with sickle cell disease, crizanlizumab (5.0 mg/kg) significantly:
Increased the percentage of patients who did not experience any VOCs vs placebo (37.5% vs. 12.2%, p=0.008) during treatment
More than doubled the median time to first on-treatment VOC (6.55 vs 1.58 months, p < 0.001) and
Decreased the annual rate of VOCs (1.04 vs 2.18, p=0.02).
SUSTAIN is part of the SENTRY clinical trial program including seven active or planned clinical studies designed to generate an array of additional data on the role crizanlizumab plays in the management of sickle cell disease. More studies may be added as plans are finalized.
Major active trials in the SENTRY program include:
SOLACE-adults (A2202) Phase II study investigating the pharmacological properties and safety of crizanlizumab in patients with sickle cell disease aged 16 and above
SOLACE-kids (B2201) Phase II study investigating the safety and efficacy of crizanlizumab in pediatric patients with sickle cell disease
STAND (A2301) Phase III study investigating the efficacy and safety of crizanlizumab in sickle cell disease patients aged 12 and above
SUCCESSOR retrospective cohort study among adult sickle cell disease patients in the US
"The SENTRY program emphasizes our long-term commitment to reimagining sickle cell disease treatment for as many people as possible," said Samit Hirawat, MD, Head, Novartis Oncology Global Drug Development. "The outcomes of these trials, alongside our analyses of SUSTAIN, will increase our understanding of the disease and, we hope, take us a step forward in our aspiration to reduce the burden of sickle cell pain crises."