Novo Nordisk said Monday that it is suspending the Phase III explorer7 and explorer8 trials as well as the Phase II explorer5 study evaluating prophylactic use of concizumab in haemophilia A and B patients regardless of inhibitor status, citing the occurrence of non-fatal thrombotic events in three patients enrolled in the late-stage programme. The company said it along with an independent data monitoring committee are in the process of "assessing the relevance of the events to the continuation of the programme and no conclusions have yet been made."
Mads Krogsgaard Thomsen, chief science officer at Novo Nordisk, said that while it is "disappointing" to pause the trials, "together with relevant authorities, we will now carefully evaluate all available data and decide how to best move forward."
The company initiated explorer7 last October to assess concizumab in patients with haemophilia A or B with inhibitors towards factors VIII or IX, followed by explorer8, which enrolled patients without inhibitors. The trials, which were expected to enrol a total of about 293 patients, evaluated once-daily prophylactic concizumab delivered subcutaneously via a pen device to reduce the number of bleeding episodes. Meanwhile, the mid-stage explorer5 study, launched in 2017, currently includes 15 patients and is assessing the ability of concizumab to reduce the number of bleeds in haemophilia A patients. Novo Nordisk noted that approximately 109 patients are currently being treated with the anti-TFPI antibody.
Last year, the Danish drugmaker unveiled Phase II data at the International Society on Thrombosis and Haemostasis (ISTH) Congress from explorer5, as well as from the explorer4 trial investigating concizumab in patients with haemophilia A/B with inhibitors. It said at the time that results at 24 weeks demonstrated the drug's potential as a "safe and efficacious subcutaneous prophylaxis treatment for all patients with haemophilia."
The FDA granted a breakthrough therapy designation for concizumab last year to treat patients with haemophilia B and inhibitors.
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