Bayer Presents Five-Year Outcomes Data from the PROTECT VIII Extension Study

Bayer announced findings from an analysis of the PROTECT VIII extension in which patients who received five years of prophylactic treatment with Jivi (BAY 94-9027, antihemophilic factor [recombinant] PEGylated-aucl), maintained a safety and efficacy profile that was consistent with prior analysis of the pivotal Phase III PROTECT VIII main study. These data were presented at the Scientific Sessions of the 60th American Society of Hematology (ASH) annual meeting and Exposition in San Diego, CA. “These findings provide insight into the long-term efficacy and safety of Jivi in previously treated hemophilia A patients 12 years and older,” said Mark Reding, M.D., associate professor of medicine at the University of Minnesota and lead investigator of the PROTECT VIII study. “This additional information can be instrumental for clinical decision-making and disease management regarding the long term treatment of hemophilia A.”

The PROTECT VIII main study was a partially randomized, open-label trial of 134 males aged 12–65 years with severe hemophilia A.  Prophylaxis patients received Jivi (BAY 94-9027) 25 IU/kg twice weekly for a 10-week run-in period. Patients with ≤ 1 spontaneous, joint or muscle bleed during this period were randomized to 45–60 IU/kg every 5 days (Q5D) or 60 IU/kg every 7 days (Q7D) for the main 26-week period, patients enrolling after the randomization arms were full, or with ≥ 2 bleeds in the run-in period, received 30–40 IU/kg twice-weekly (2×W). Treatment success in the every 7-day arm was not established. Twenty patients received Jivi on demand as they had been receiving on-demand FVIII treatment prior to study entry. Following completion of the PROTECT VIII main study, patients could enter an optional extension, continuing Jivi on any regimen used in the main study. Of the 126 patients who completed the main study, 121 entered the extension, of which 107 continued prophylaxis with Jivi.  The data presented were from 33 patients who have completed five years of treatment with Jivi at the time of this analysis. Annualized bleeding rate (ABR) and joint ABR were analyzed, along with safety outcomes.

Study data found the previously demonstrated efficacy of Jivi in the main PROTECT VIII study was maintained for a median (range) follow-up period of 3.9 years (0.8-5.4) with a data cut-off date of January 2018. In the last year of the extension study, one third of patients were entirely free from bleeds. Consistent with prior analyses of PROTECT VIII, there were few study drug-related AE and no evidence of any long-term toxicity of PEGylated product exposure. Drug-related adverse events (AE) occurred in 6 of the 33 patients (18.2 percent) and none of these patients had a study drug-related serious AE. There were no discontinuations for AEs. No patients had confirmed FVIII inhibitors (titer ≥0.6 Bethesda units). The U.S. Food Drug Administration (FDA) based its recent approval of Jivi on results from the PROTECT VIII main study comprised of prophylactic dosing, on-demand treatment and perioperative management in previously treated adults and adolescents 12 years of age and older with severe hemophilia A. – Medical Buyer Bureau