Novo Nordisk today reported positive results from its paradigm™2 Phase III trial for N9-GP (glycopegylated recombinant factor IX), for hemophilia B patients. The multi-center trial assessed the safety and efficacy of N9-GP, the first long-acting Factor IX derivative, for on-demand or prophylactic treatment.
Among patients who randomly received 40 U/kg N9-GP, 99% of bleeding episodes were treated with only one infusion, while two-thirds of the patients experienced complete resolution of bleeding in their target joints. N9-GP patients also reported an improvement in quality of life during the trial.
No patients developed inhibitors to the Factor IX product, and no apparent differences between the treatment groups were observed with respect to adverse events and standard safety parameters, according to Novo Nordisk.
During paradigm 2, 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. Median annualized bleeding rate for patients treated on-demand was 15.6 episodes per year. However, patients on prophylaxis had a median 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.
”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,” Mads Krogsgaard Thomsen, evp and CSO of Novo Nordisk, said in a statement. “We are very excited about the strong results from this trial, which could represent a paradigm shift in the treatment of hemophilia B.”
Novo Nordisk said it expects the two remaining Phase III paradigm™ trials—involving pediatric patients and patients undergoing surgery, respectively, to be completed within the next 12 months. The paradigm trials consist of seven studies designed to investigate the pharmacokinetics, immunogenicity, efficacy, and safety of N9-GP in adult and pediatric hemophilia B patients, as well as in patients undergoing surgery.
By 2015, the company said, it expects to submit N9-GP for marketing approvals “in all major markets,” to enable validation of commercial scale production of the recombinant Factor IX. Novo Nordisk hopes to challenge Pfizer, whose BeneFIX® is the first recombinant Factor IX product marketed for people with Hemophilia B, accounting for its strong sales last year of $775 million, up 12% from 2011. Also looking to bring a recombinant Factor IX to market is Biogen Idec and Swedish Orphan Biovitrum, which last year also published promising Phase III clinical trial results for their product.
N9-GP applies glycopegylation to recombinant Factor IX in order to increase its circulating half-life, allowing for lower bleeding frequency with less frequent intravenous dosing, according to Novo Nordisk.