Takeda dropped a trial of data breadcrumbs in January as the run to regulators for an enzyme replacement therapy began, but now the Japanese pharma is cutting off a more substantial slice from the whole loaf, showing a complete reduction in deadly blood clot attacks in patients with an ultra-rare disease.
Previously, Takeda reported that the ADAMTS13 replacement therapy TAK-755 produced a 60% reduction in the incidence of thrombocytopenia events in patients with congenital thrombotic thrombocytopenic purpura (cTTP) compared to the plasma-based therapies that are the current standard of care.
Now, the company has more to share. Interim results from a phase 3 trial of TAK-755 showed that patients taking the drug as a preventative measure did not have any acute TTP attacks. The study was an open-label, randomized crossover trial evaluating the safety of TAK-755. Takeda did not share data on the rate of less serious events.
TTP is a rare disorder that causes blood clots to form in small blood vessels throughout the body, blocking blood flow to the major organs and preventing them from working properly. The disorder can be fatal and is characterized by sudden attacks, which have a fatality rate of 90% if left untreated.
TAK-755 also had a favorable safety profile that could be better than the standard of care, Takeda said in a Sunday release from the International Society on Thrombosis and Haemostasis 2023 Congress in Montréal. The study saw treatment-emergent adverse events in 10% of patients, aged 12 to 68, who received the therapy, compared to half of patients who received the standard of care.
Takeda also revealed an interim analysis from a long-term phase 3b continuation study of TAK-755 in 29 patients, showing a favorable safety profile and no development of neutralizing antibodies that could complicate treatment. There were zero acute TTP attacks during the study period. The rate of less serious attacks was similar to the data recorded in the larger pivotal study.
TAK-755, acquired by Takeda from Shire, has been granted a priority review in cTTP from the FDA and a rare pediatric disease tag.