Takeda has reported favourable interim results from a Phase III clinical trial of TAK-755 (recombinant ADAMTS13) for the prophylactic treatment of congenital thrombotic thrombocytopenic purpura (cTTP).

The international pivotal crossover, open-label, controlled, randomised study is designed to assess the safety and efficacy of the investigational therapy TAK-755.

It enrolled 36 patients aged 12 years and above to compare the pharmacokinetics (PK) characteristics of TAK-755 after a single infusion (0-168 hours) against plasma-based therapy.

Thrombocytopenia, among other recurring TTPs such as haemolytic activity, headache, and abdominal pain is considered an important marker of disease activity.

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The incidence of thrombocytopenia was reduced by 60% with TAK-755 prophylactic treatment against plasma-based therapy.

A favourable safety and tolerability profile was demonstrated while 10.3% of patients aged 12-68 years receiving TAK-755 showed treatment-emergent adverse events (TEAEs)

A five-fold increase in the therapeutic activity levels was also observed with a Cmax of 100% activity for TAK-755 against 19% activity for plasma-based therapy.

Takeda rare genetics and haematology therapeutic area unit head Daniel Curran said: “We are encouraged by what these positive results mean for people living with this rare disorder, and we look forward to continuing to advance this programme for patients who may benefit from treatment with TAK-755.”

Caused due to deficiency in the ADAMTS13 enzyme, cTTP is an ultra-rare blood clotting disorder characterised based on the severity ranging from severe acute to chronic TTP events.

The safety and efficacy of long-term data of an interim analysis of the Phase IIIb continuation study of TAK-755 prophylaxis in 29 patients were also reported.

Takeda is also evaluating TAK-755 in ongoing Phase IIb and Phase I studies for treating immune-mediated TTP (iTTP) and sickle cell disease.