Promising new drug for chronic blood clotting disorder

Blood cells

Takeda has revealed favourable interim results from its Phase III crossover trial evaluating the safety and efficacy of TAK-755 (recombinant ADAMTS13) replacement therapy for the prophylactic treatment of congenital thrombotic thrombocytopenic purpura (cTTP).

cTTP is an ultra-rare, chronic and debilitating blood clotting disorder caused by a deficiency in ADAMTS13 enzyme.

Clinical presentation of cTTP lies on a spectrum of severity ranging from severe acute TTP events to chronic, recurring TTP manifestations (e.g., thrombocytopenia, haemolytic activity, headache, abdominal pain). Acute TTP events have a mortality rate of >90%, if left untreated.

In the pivotal trial, no patient had an acute TTP event while receiving TAK-755 prophylactic treatment. TAK-755 also reduced the incidence of thrombocytopenia by 60%, as compared to plasma-based therapy.

Thrombocytopenia, an important marker of disease activity, was the most frequently observed TTP manifestation. In addition, the results show that TAK-755 demonstrated a favourable safety and tolerability profile, with a potential safety advantage over plasma-based therapies.

In the pivotal trial, treatment-emergent adverse events (TEAEs) were reported in 10.3% of patients ages 12-68 receiving TAK-755 compared to 50% of patients receiving plasma-based therapy.

“These findings suggest that recombinant ADAMTS13 is a promising innovative investigational treatment for patients with cTTP,” said Marie Scully, Department of Haematology, University College London Hospitals, London, United Kingdom. “Given the high burden of illness these patients experience, complicated by unpredictable acute episodes and multiple disease-related complications, this is a much-needed option, supported through a first-of-its-kind clinical trial.”

The results were presented at the International Society on Thrombosis and Haemostasis (ISTH) 2023 Congress.

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