Novo Nordisk presented clinical study data on two new investigational therapies, one to treat individuals with hemophilia A and B with inhibitors to infused factor VIII and IX, and the other to treat factor XIII (FXIII) deficiency, a rare inherited bleeding disorder. The data were reported at the 52nd Annual Meeting of the American Society of Hematology in Orlando, FL, on December 6, 2010.
The company presented data from a phase 2 trial evaluating the safety, pharmacokinetics (processing and duration of drug’s effect in the body) and efficacy of NN1731, a recombinant factor VIIa (rFVIIa) therapy to treat hemophilia A or B inhibitor patients. The new treatment is a faster-acting inhibitor therapy than the company’s existing rFVIIa product, NovoSeven®. The results showed that NN1731 was safe and no inhibitor antibodies were reported. In addition, when patients received the therapy at the two highest dose levels, 96% of the joint bleeds were well controlled.
“There were no safety concerns observed in patients at any dose level of NN1731,” said Erich de Paula, MD, of the State University of Campinas in São Paulo, Brazil, who presented the trial data. “Additionally, the phase 2 trial results demonstrated the potential of the rFVIIa analogue to stop joint bleeds quickly and effectively. These results further support the distinct fast action profile of the rFVIIa analogue in treating bleeds in hemophilia patients with inhibitors.”
Novo Nordisk also presented data from a phase 3 trial measuring the efficacy and safety of mentor™1, a rFXIII therapy for the prevention of bleeding in patients with FXIII deficiency. According to the Novo Nordisk release, there are an estimated 600–1,000 people in the world with FXIII deficiency. Current treatment for FXIII deficiency is the use of plasma products, which carry a risk of infection.
The trial included 41 patients with FXIII deficiency who were given the monthly intravenous therapy for a year. Patients with no previous history of FXIII treatment were used as a control. The results showed that this monthly regimen “significantly decreased” the number of bleeding episodes requiring treatment when compared to the control group. Five bleeding episodes were observed in four patients, all associated with trauma and not related to low FXIII levels. No blood clots or fatal adverse events were reported.
“These data show the potential for rFXIII to become a safe and effective treatment option for patients who would otherwise use treatments at risk for contamination,” said Professor Aida Inbal, MD, of the Hemostasis Unit and Hematology Clinic in the Institute of Hematology at Rabin Medical Center in Tel Aviv, Israel. “We think this is an extremely important milestone in the development of a treatment that is not sourced from human plasma for patients suffering from congenital FXIII deficiency.”
Novo Nordisk plans to file for US Food and Drug Administration approval of the rFXIII therapy in the first half of 2011.Source: Novo Nordisk press release dated December 7, 2010