NEJM publishes Phase III Adzynma data

The prestigious New England Journal of Medicine (NEJM) has published interim results from the Phase III trial of Japanese drug major Takeda Pharma’s (TYO: 4502) Adzynma (apadamtase alfa/cinaxadamtase alfa; code name TAK-755) in patients with congenital thrombotic thrombocytopenic purpura (cTTP).

cTTP is an ultra-rare, chronic blood clotting disorder caused by a deficiency in the ADAMTS13 enzyme. When left untreated, acute thrombotic thrombocytopenic purpura (TTP) events have a mortality rate of >90%.

The randomized, open-label, crossover Phase III trial was comprised of two cohorts: a prophylactic cohort and an on-demand cohort. In the prophylactic cohort, patients received 40 IU/kg Adzynma IV or plasma-based therapy every other week or weekly based on prior regimen for six months (period 1), crossing over to the alternate treatment for six months (period 2), and all patients received Adzynma for an additional six months (period 3).