Novo Nordisk recently announced completion of its first Phase III trial for its hemophilia B candidate, N9-GP (glycopegylated recombinant factor IX).
Paradigm™ 2 evaluated N9-GP, a long-active FIX derivative, for the safety and efficacy of the drug when used for on-demand or prophylactic treatment in patients with hemophilia B. The trial consisted of 74 patients who were treated for six months on-demand, or 12 months by prophylactic regimen of 40 U/kg or 10 U/kg N9-GP once-weekly. Results found that the median bleeding rate for patients who were treated on-demand was 15.6 episodes per year. Patients on prophylaxis had a median annualized bleeding rate of 1.0 for patients receiving the 40 U/kg, and 2.9 episodes per year for patients receiving the 10 U/kg.
Additionally, 99 percent of bleeding episodes were treated with only one infusion for patients receiving 40 U/kg N9-GP, and two-thirds of the patients experienced complete resolution of bleeding in their targeted joints. According to the company, patients in this dose group reported an improvement in quality of life during the trial.
“We are very excited about the strong results from this trial, which could represent a paradigm shift in the treatment of hemophilia B,” Mads Krogsgaard Thomsen, Executive Vice President and Chief Scientific Officer of Novo Nordisk stated. “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.”
The drug was safe and well-tolerated in the trial. There were no apparent differences of adverse events and standard safety parameters observed between treatment groups. In the trial, no patients developed inhibitors.
According to the company, the two remaining phase III trials in the paradigm™ program involving pediatric patients and patients undergoing surgery are expected to be completed in the next year. Additionally, Novo Nordisk expects to submit regulatory application in all major markets in 2015.
Source: Novo Nordisk A/S
Last Updated: 5/20/13; 12:00PM EST