Biopharmaceutical company, Ablynx, has announced the completion of patient recruitment for its Phase III HERCULES study of caplacizumab for the treatment of acquired thrombotic thrombocytopenic purpura (aTTP).
Patient recruitment
A total of 145 patients have been enrolled into the multinational, double-blind, placebo-controlled trial. Data from the study are expected to be reported on in the second half of the year and should be used to support the marketing authorisation application (MAA) recently submitted to the EMA as well as the planned biologics licence application (BLA) filing in the US next year.
“This is the largest randomised, double-blind, placebo-controlled study ever conducted in patients with aTTP,” highlighted Dr Robert K. Zeldin, chief medical officer at Ablynx. “Recruiting 145 patients in just 20 months is an indication of the interest and enthusiasm of clinicians and their patients. Their commitment is enabling us to evaluate the role of caplacizumab in improving the current standard-of-care for this very severe disease. We thank everyone involved in this trial for their contribution and look forward to reporting the data later this year.”
Caplacizumab is a potent and selective bivalent anti-von Willebrand factor (vWF) Nanobody that blocks the interaction ultra large vWF multimers with platelets, immediately affecting platelet aggregation and the ensuing formation and accumulation of the microclots that cause the severe thrombocytopenia, tissue ischaemia and organ dysfunction in aTTP. If approval for this treatment is received from the regulatory authorities, it will be the first therapeutic specifically indicated for the treatment of aTTP.