Positive results for the Phase 3 trial of Cablivi in adults with acquired thrombotic thrombocytopenic purpura (aTTP) have been published in the New England Journal of Medicine.
aTTP is a rare, life-threatening autoimmune blood disorder characterised by extensive clot formation in small blood vessels throughout the body, leading to a very low platelet count, loss of red blood cells through destruction, ischemia and widespread organ damage, especially in the brain and heart.
The current treatment for aTTP consists of daily plasma exchange, in which a patient’s blood plasma is removed and replaced with donor plasma, and immunosuppression.
Even with currently available treatments, patients continue to be at risk of developing acute blood clotting conditions, such as stroke and heart attack, as well as recurrence of disease.
HERCULES is a Phase 3 randomised, double-blind, placebo-controlled study of Cablivi in patients with aTTP.
In the study, 145 patients were randomly assigned to Cablivi or placebo in conjunction with plasma exchange and immunosuppression.
Cablivi (caplacizumab) demonstrated a safety profile consistent with what has been previously reported and in line with its mechanism of action; this included an increased risk of bleeding.
The most frequently reported bleeding-related adverse events were epistaxis and gingival bleeding.
“These results demonstrate that Cablivi has the potential to address a major unmet medical need and to help those facing the potentially devastating consequences of this disorder,” said Marie Scully, professor of haematology at University College London Hospitals, and lead author of the HERCULES study.