TAK-755 trumps plasma-based therapy, trial shows

A Phase III trial evaluating Takeda’s TAK-755 (recombinant ADAMTS13) replacement therapy for a rare blood clotting disorder facilitated a five-fold increase in ADAMTS13 activity levels compared to patients receiving plasma-based therapy. 

The first and only recombinant ADAMTS13 protein in development provides a targeted therapy to address an unmet medical need in congenital thrombotic thrombocytopenic purpura (cTTP), the company declared.

Based on positive interim results from the first and only Phase III controlled trial for the prophylactic cTTP treatment, TAK-755 reduced the incidence of thrombocytopenia by 60 percent. This was compared to plasma-based therapy. The treatment demonstrated a favourable safety and tolerability profile, with a potential safety advantage over plasma-based therapies.

What is cTTP?

Utilising TAK-755 (recombinant ADAMTS13) replacement therapy

cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in ADAMTS13 enzyme. Dr Daniel Curran, Head of Rare Genetics & Hematology Therapeutic Area Unit at Takeda highlighted that at present, there is no approved treatment for cTTP.

The therapy works by replacing the missing or deficient ADAMTS13 enzyme, according to a 2017 paper by Scully et al., published in Blood.

Current treatment for the rare condition is based on plasma-based therapy. This is time consuming and can be associated with severe treatment complications, according to research, Takeda noted.

Additional results from the first Phase III controlled trial in cTTP

Treatment-emergent adverse events (TEAEs) were reported in 10.3 percent of patients ages 12-68 receiving TAK-755 during the Phase III trial. This is compared to half of patients who received plasma-based therapy.

No patient had an acute TTP event while receiving TAK-755 prophylactic treatment in the clinical trial, Takeda stated.

In an interim analysis of the Phase IIIb continuation study, there was no evidence of development of neutralising antibodies.

Overall, findings from the “first-of-its-kind clinical trial,” and the Phase IIIb continuation study “suggest that recombinant ADAMTS13 is a promising innovative investigational treatment for patients with cTTP,” commented Dr Marie Scully from the Department of Haematology at University College London Hospitals, UK.

Results were presented in three oral presentations at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress.

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