Takeda Pharmaceutical has reported positive data from Phase III clinical trial assessing recombinant von Willebrand factor (rVWF) prophylaxis for von Willebrand disease (VWD).
The prospective, open-label, global, multi-centre, 12-month trial had 23 patients with severe VWD, a common inherited bleeding disorder that develops due to VWF deficiency or dysfunction.
It involved the prior on-demand (OD) arm, which recruited subjects who previously took OD VWF, and the switch arm of subjects previously on prophylactic plasma-derived von VWF (pdVWF) therapy, LPB0128.
According to the trial results, rVWF prophylaxis decreased spontaneous, treated annualised bleeding rates (sABRs) in the prior OD arm and the same haemostatic control level was observed in the switch arm, thus meeting the trial’s endpoint.
Compared with historical sABR in prior OD subjects, a 91.5% sABR reduction was seen in those on prophylactic rVWF and the rates were maintained on-study in switch subjects.
The data did not show any new risks or serious adverse events associated with rVWF.
These Phase III trial results are one of the 12 abstracts that Takeda is presenting at the International Society on Thrombosis and Haemostasis (ISTH) Virtual Congress 2021.
Most of the abstracts analysed the use of prophylaxis in line with the company’s aim to enhance outcomes for rare bleeding disorder patients.
Takeda Pharmaceutical Haematology Global Medical Affairs head Wolfhard Erdlenbruch said: “At Takeda, we are committed to helping make a difference to the lives of patients with rare haematological disorders, both today through our broad portfolio of treatments, and in the future by developing innovative solutions to address unmet needs.
“The data presented at ISTH 2021 highlights the potential for rVWF to address unmet needs in VWD, as well as helping us to understand the clinical and resource utilisation impact when individualising treatment through PK-guided prophylaxis in haemophilia.”
The company also highlighted the Phase III trial of TAK-755, a recombinant ADAMTS13 replacement therapy candidate intended for prophylactic and OD treatment of severe congenital thrombotic thrombocytopenic purpura.
In May 2021, Takeda reported that its vaccine candidate, TAK-003, offered protection against dengue illness and hospitalisation in the Phase III TIDES trial.