Novo Nordisk has announced its latest positive set of phase 3 results from the Explorer7 study, evaluating the efficacy and safety of concizumab for those living with haemophilia A or B.
The company shared the data from the primary analysis at the International Society of Thrombosis and Haemostasis Annual Congress (ISTH 2022) in London, UK.
Concizumab is an anti-tissue factor pathway inhibitor (TFPI) antibody in development for a once-daily treatment to prevent prolonged and spontaneous bleeding.
Haemophilia is a rare disease affects around 1,125,000 people globally. For those living with haemophilia, their ability to produce blood clots is impaired. Clotting is necessary to stop bleeding following an injury.
Haemophilia A and B are more common in males than in females, with ~ 88% of people diagnosed with haemophilia worldwide being male. Some people with the condition may also develop inhibitors, which are produced as an immune system response to the clotting factors in replacement therapy that cause the treatment to stop working.
Currently, it is estimated that 30% of people living with haemophilia A and 1-3% of people living with haemophilia B have inhibitors.
The Explorer7 study is part of the ongoing clinical trial programme for concizumab, which is focused on assessing the efficacy and safety of concizumab for people living with haemophilia A or B with or without inhibitors.
The results demonstrated an 86% reduction in treated spontaneous and traumatic bleeds when on concizumab prophylaxis (prevention). Of those treated with concizumab, 21 (63.6%) had no treated bleeds, compared to two (10.5%) on no prophylaxis. The safety and tolerability profile of concizumab in this study was within the expected range, with no thromboembolic events reported after treatment restart.
Martin Lange, executive vice president and head of Development at Novo Nordisk, said: “Concizumab offers the potential for everyday protection for people living with haemophilia and provides an important potential addition to our haemophilia offering, especially in the haemophilia B with inhibitor population who currently have limited treatment options.”
The use of concizumab is investigational and is currently not approved by regulatory authorities. However, Novo Nordisk plans to submit concizumab for regulatory approval for this indication in the second half of 2022 in the US and Japan, and then in 2023 in the EU and the UK.