Takeda therapy shows promise in rare blood clotting disorder

Takeda has announced positive phase 3 results supporting the efficacy and safety of TAK-755 as an enzyme replacement therapy for congenital thrombotic thrombocytopenic purpura (cTTP).

cTTP is an ultra-rare subtype of thrombotic thrombocytopenic purpura (TTP), a rare, chronic and debilitating blood clotting disorder caused by a deficiency in ADAMTS13 protease.

The disorder has both acute and chronic manifestations, including cardiovascular disease and stroke, and is associated with a significant disease burden.

Patients with cTTP experience a significantly reduced quality of life and lifespan compared to the general population as a result of ongoing widespread organ damage and other co morbidities associated with an ADAMTS13 deficiency.

The current standard of care (SoC) for cTTP is plasma therapy, which Takeda describes as ‘insufficient in restoring ADAMTS13, time-consuming, and costly’.

TAK-755 is the first and only recombinant ADAMTS13 protein in development, providing targeted therapy to patients with TTP by replacing the missing or deficient ADAMTS13 enzyme.

Results from a pre-planned interim analysis of the phase 3 trial showed TAK-755 reduced the incidence of thrombocytopenia events by 60%, an important marker of disease activity in cTTP, as compared to plasma-based therapies.

The proportion of patients experiencing treatment-related adverse events was also significantly lower in the TAK-755 treatment group, at 8.9%, compared to 47.7% of patients receiving SoC therapy.

The company said it aims to seek marketing authorisation for TAK-755 as the first recombinant ADAMTS13 replacement therapy for cTTP based on these results.

Daniel Curran, head, rare genetics and haematology therapeutic area unit at Takeda, said: “We are committed to advancing treatment options for those living with cTTP, who currently have no therapies approved specifically to manage their condition.

“The results of the trial are very encouraging, and we look forward to continuing to engage with global regulatory bodies with the aim of bringing TAK-755 to patients as rapidly as possible.”

As well as the cTTP clinical development programme, TAK-755 is also being investigated in immune-mediated TTP and sickle cell disease.

The enzyme replacement therapy was granted orphan drug designation by the US Food and Drug Administration (FDA) for the treatment and prevention of TTP including its congenital, acquired idiopathic and secondary forms, and by the European Medicines Agency and Japan’s Ministry of Health, Labour and Welfare for the treatment of TTP.

The FDA has also granted TAK-755 Fast Track Designation for the treatment, prevention and routine prophylaxis of acute episodes of TTP in patients with hereditary (congenital) ADAMTS13 deficiency.