Danish drug maker Novo Nordisk A/S (NVO) Friday announced positive results from the first Phase-3 trial with a long-acting FIX derivative, N9-GP, in the treatment of haemophilia B patients.
Haemophilia B a blood clotting disorder caused by a mutation of the Factor IX gene, leading to a deficiency of Factor IX.
Novo Nordisk's first Phase-3 trial, paradigm 2, is a multi-centre, blinded study evaluating the safety and efficacy of N9-GP (glycopegylated recombinant factor IX) when used for on-demand or prophylactic treatment in patients with haemophilia B.
In the trial, 74 patients were treated for six months on-demand, or 12 months by a prophylactic regimen of 40 U/kg or 10 U/kg N9-GP once weekly.
The median bleeding rate for patients treated on-demand was 15.6 episodes per year. Patients on prophylaxis had a median annualised bleeding rate of 1.0 and 2.9 episodes per year, when treated with weekly doses of 40 U/kg and 10 U/kg, respectively.
Among patients randomised to receive 40 U/kg N9-GP, 99% of bleeding episodes were treated with only one infusion, and two-thirds of the patients experienced complete resolution of bleeding in their target joints. Patients in this dose group also reported an improvement in quality of life during the trial.
Pharmacokinetic data documented a steady state half-life of 110 hours.
In the trial, N9-GP appeared to have a safe and well-tolerated profile. No patients in the trial developed inhibitors, and no apparent differences between the treatment groups were observed with respect to adverse events and standard safety parameters.
"The trial demonstrated that once-weekly prophylactic administration of N9-GP can reduce the risk of bleeds by more than 90% compared to on-demand treatment and enable 99% of the few occurring bleeds to be stopped with a single dose," said Mads Krogsgaard Thomsen, executive vice president and chief science officer of Novo Nordisk.
Novo Nordisk is expecting the two remaining phase 3 trials in the paradigm program involving paediatric patients and patients undergoing surgery respectively to be completed within the next 12 months. Regulatory submission of N9-GP in all major markets is expected in 2015 to enable validation of the commercial scale production.
For comments and feedback: editorial@rttnews.com