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An investigational recombinant factor VIII (rFVIII) product can safely treat and prevent bleeding in previously treated patients with hemophilia A, according to researchers.
The product, BAX 855, is a pegylated version of ADVATE, a full-length rFVIII product already approved to treat hemophilia A.
BAX 855 was designed to have a longer half-life than ADVATE, thereby allowing for fewer prophylactic infusions without affecting hemostatic efficacy.
Results of a phase 1 study showed that BAX 855 had a longer half-life and mean residence time than ADVATE. And results of a phase 2/3 study showed that twice-weekly prophylactic treatment with BAX 855 lowered the median annualized bleed rate (ABR) when compared to on-demand treatment with BAX 855.
None of the patients who received BAX 855 in either study developed inhibitors, and there were no serious adverse events (AEs) that were considered treatment-related.
Results from both trials were published in Blood. The research was funded by Baxalta, a spin-off of Baxter Healthcare Corporation.
Study characteristics
The phase 1 study included 19 previously treated patients with severe hemophilia A and a median age of 29 (range, 18-60). The patients received single infusions of ADVATE followed by BAX 855 (after a wash-out period) at 30 IU/kg or 60 IU/kg.
In the phase 2/3 trial, researchers evaluated BAX 855 in 137 previously treated patients with hemophilia A. These patients also had a median age of 29 (range, 12-58).
They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with BAX 855. One hundred and twenty-six patients completed the study.
Pharmacokinetics and safety
In the phase 1 study, the mean residence time was higher with BAX 855 than with ADVATE—1.4-fold higher in the 30 IU/kg arm and 1.5-fold higher in the 60 IU/kg arm. The mean half-life was higher with BAX 855 as well—1.4-fold higher in the 30 IU/kg arm and 1.5-fold in the 60 IU/kg arm.
Results were similar in the phase 2/3 trial.
None of the subjects in the phase 1 study experienced a serious AE after their single infusion of BAX 855. Eight patients experienced a total of 11 non-serious AEs, none of which were considered related to BAX 855.
In the phase 2/3 study, there were 171 AEs in 73 patients who received BAX 855 for about 6 months. There were 7 AEs (occurring in 6 patients) that were considered possibly related to BAX 855. These included diarrhea, nausea, headache, and flushing.
There were 5 serious AEs (occurring in 5 patients) that were not considered treatment-related. These included osteoarthritis, herpes zoster infection, humerus fracture, neuroendocrine carcinoma, and muscle hemorrhage.
Efficacy: Prophylaxis and on-demand
The researchers only assessed the efficacy of BAX 855 prophylaxis and on-demand treatment in the phase 2/3 study.
Patients received a median dose of 44.6 IU/kg per prophylactic infusion. The mean reduction in dosing frequency from pre-study prophylaxis was 26.7%, and 70.4% of patients were able to reduce the frequency of dosing by 30% or more. This is roughly equivalent to at least 1 less prophylactic infusion per week.
Patients who received prophylaxis had a 90% reduction in ABR compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
The ABRs for joint bleeds or spontaneous/unknown bleeds were both 0 in the prophylactic arm, compared to 38.1 and 21.6, respectively, in the on-demand treatment arm.
Patients who received on-demand treatment were given a median dose of 30.87 IU/kg per episode and 29.19 IU/kg for the maintenance of hemostasis.
Of the 518 bleeding episodes reported during the study, 95.9% were treated with 1 or 2 infusions of BAX 855. The mean number of infusions required to treat a bleeding episode was 1.2. 
An investigational recombinant factor VIII (rFVIII) product can safely treat and prevent bleeding in previously treated patients with hemophilia A, according to researchers.
The product, BAX 855, is a pegylated version of ADVATE, a full-length rFVIII product already approved to treat hemophilia A.
BAX 855 was designed to have a longer half-life than ADVATE, thereby allowing for fewer prophylactic infusions without affecting hemostatic efficacy.
Results of a phase 1 study showed that BAX 855 had a longer half-life and mean residence time than ADVATE. And results of a phase 2/3 study showed that twice-weekly prophylactic treatment with BAX 855 lowered the median annualized bleed rate (ABR) when compared to on-demand treatment with BAX 855.
None of the patients who received BAX 855 in either study developed inhibitors, and there were no serious adverse events (AEs) that were considered treatment-related.
Results from both trials were published in Blood. The research was funded by Baxalta, a spin-off of Baxter Healthcare Corporation.
Study characteristics
The phase 1 study included 19 previously treated patients with severe hemophilia A and a median age of 29 (range, 18-60). The patients received single infusions of ADVATE followed by BAX 855 (after a wash-out period) at 30 IU/kg or 60 IU/kg.
In the phase 2/3 trial, researchers evaluated BAX 855 in 137 previously treated patients with hemophilia A. These patients also had a median age of 29 (range, 12-58).
They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with BAX 855. One hundred and twenty-six patients completed the study.
Pharmacokinetics and safety
In the phase 1 study, the mean residence time was higher with BAX 855 than with ADVATE—1.4-fold higher in the 30 IU/kg arm and 1.5-fold higher in the 60 IU/kg arm. The mean half-life was higher with BAX 855 as well—1.4-fold higher in the 30 IU/kg arm and 1.5-fold in the 60 IU/kg arm.
Results were similar in the phase 2/3 trial.
None of the subjects in the phase 1 study experienced a serious AE after their single infusion of BAX 855. Eight patients experienced a total of 11 non-serious AEs, none of which were considered related to BAX 855.
In the phase 2/3 study, there were 171 AEs in 73 patients who received BAX 855 for about 6 months. There were 7 AEs (occurring in 6 patients) that were considered possibly related to BAX 855. These included diarrhea, nausea, headache, and flushing.
There were 5 serious AEs (occurring in 5 patients) that were not considered treatment-related. These included osteoarthritis, herpes zoster infection, humerus fracture, neuroendocrine carcinoma, and muscle hemorrhage.
Efficacy: Prophylaxis and on-demand
The researchers only assessed the efficacy of BAX 855 prophylaxis and on-demand treatment in the phase 2/3 study.
Patients received a median dose of 44.6 IU/kg per prophylactic infusion. The mean reduction in dosing frequency from pre-study prophylaxis was 26.7%, and 70.4% of patients were able to reduce the frequency of dosing by 30% or more. This is roughly equivalent to at least 1 less prophylactic infusion per week.
Patients who received prophylaxis had a 90% reduction in ABR compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
The ABRs for joint bleeds or spontaneous/unknown bleeds were both 0 in the prophylactic arm, compared to 38.1 and 21.6, respectively, in the on-demand treatment arm.
Patients who received on-demand treatment were given a median dose of 30.87 IU/kg per episode and 29.19 IU/kg for the maintenance of hemostasis.
Of the 518 bleeding episodes reported during the study, 95.9% were treated with 1 or 2 infusions of BAX 855. The mean number of infusions required to treat a bleeding episode was 1.2.
An investigational recombinant factor VIII (rFVIII) product can safely treat and prevent bleeding in previously treated patients with hemophilia A, according to researchers.
The product, BAX 855, is a pegylated version of ADVATE, a full-length rFVIII product already approved to treat hemophilia A.
BAX 855 was designed to have a longer half-life than ADVATE, thereby allowing for fewer prophylactic infusions without affecting hemostatic efficacy.
Results of a phase 1 study showed that BAX 855 had a longer half-life and mean residence time than ADVATE. And results of a phase 2/3 study showed that twice-weekly prophylactic treatment with BAX 855 lowered the median annualized bleed rate (ABR) when compared to on-demand treatment with BAX 855.
None of the patients who received BAX 855 in either study developed inhibitors, and there were no serious adverse events (AEs) that were considered treatment-related.
Results from both trials were published in Blood. The research was funded by Baxalta, a spin-off of Baxter Healthcare Corporation.
Study characteristics
The phase 1 study included 19 previously treated patients with severe hemophilia A and a median age of 29 (range, 18-60). The patients received single infusions of ADVATE followed by BAX 855 (after a wash-out period) at 30 IU/kg or 60 IU/kg.
In the phase 2/3 trial, researchers evaluated BAX 855 in 137 previously treated patients with hemophilia A. These patients also had a median age of 29 (range, 12-58).
They were assigned to either twice-weekly prophylaxis (40-50 IU/kg, n=120) or on-demand treatment (10-60 IU/kg, n=17) with BAX 855. One hundred and twenty-six patients completed the study.
Pharmacokinetics and safety
In the phase 1 study, the mean residence time was higher with BAX 855 than with ADVATE—1.4-fold higher in the 30 IU/kg arm and 1.5-fold higher in the 60 IU/kg arm. The mean half-life was higher with BAX 855 as well—1.4-fold higher in the 30 IU/kg arm and 1.5-fold in the 60 IU/kg arm.
Results were similar in the phase 2/3 trial.
None of the subjects in the phase 1 study experienced a serious AE after their single infusion of BAX 855. Eight patients experienced a total of 11 non-serious AEs, none of which were considered related to BAX 855.
In the phase 2/3 study, there were 171 AEs in 73 patients who received BAX 855 for about 6 months. There were 7 AEs (occurring in 6 patients) that were considered possibly related to BAX 855. These included diarrhea, nausea, headache, and flushing.
There were 5 serious AEs (occurring in 5 patients) that were not considered treatment-related. These included osteoarthritis, herpes zoster infection, humerus fracture, neuroendocrine carcinoma, and muscle hemorrhage.
Efficacy: Prophylaxis and on-demand
The researchers only assessed the efficacy of BAX 855 prophylaxis and on-demand treatment in the phase 2/3 study.
Patients received a median dose of 44.6 IU/kg per prophylactic infusion. The mean reduction in dosing frequency from pre-study prophylaxis was 26.7%, and 70.4% of patients were able to reduce the frequency of dosing by 30% or more. This is roughly equivalent to at least 1 less prophylactic infusion per week.
Patients who received prophylaxis had a 90% reduction in ABR compared to those who received on-demand treatment. The median ABR was 1.9 in the prophylactic arm and 41.5 in the on-demand arm.
The ABRs for joint bleeds or spontaneous/unknown bleeds were both 0 in the prophylactic arm, compared to 38.1 and 21.6, respectively, in the on-demand treatment arm.
Patients who received on-demand treatment were given a median dose of 30.87 IU/kg per episode and 29.19 IU/kg for the maintenance of hemostasis.
Of the 518 bleeding episodes reported during the study, 95.9% were treated with 1 or 2 infusions of BAX 855. The mean number of infusions required to treat a bleeding episode was 1.2.