Note: This is an edited version of a press release from Biogen. To read the original release, click here.
Newly published clinical data demonstrate that people on extended-interval prophylaxis regimens with ELOCTATE® [Antihemophilic Factor (Recombinant), Fc Fusion Protein] experienced low bleeding rates, Biogen and Swedish Orphan Biovitrum AB (Sobi) announced on Monday, August 10, 2015. The interim results of this Phase 3, open-label extension study called ASPIRE were published in the online edition of Haemophilia, the journal of the World Federation of Hemophilia, the European Association for Haemophilia and Allied Disorders, and the Hemostasis & Thrombosis Research Society.
Study participants completing the Phase 3 A-LONG and Kids A-LONG studies were eligible to participate in ASPIRE. The results to-date show the majority of participants in ASPIRE, maintained or extended their dosing intervals between treatments compared to the A-LONG and Kids A-LONG studies. As of the interim analysis, the median time in the ASPIRE study was 80.9 weeks for adults and adolescents completing the A-LONG study, and 23.9 weeks for children completing the Kids A-LONG study. Inhibitor development is the primary endpoint of ASPIRE and no inhibitors were reported in any treatment groups. Through the interim ASPIRE analysis, adults and adolescents experienced annualized bleeding rates (ABRs) of 0.66, 2.03 and 1.97 in the individualized, weekly and modified prophylaxis arms, respectively. Children on individualized prophylaxis also experienced low bleeding rates, with an overall median ABR of 0.0 in children less than 6 years of age, and 1.54 for children ages 6 to 12. These results were consistent with data from the Phase 3 A-LONG and Kids A-LONG studies.
In addition to efficacy and safety endpoints, the publication also reports changes in prophylactic infusion frequency from the end of the A-LONG study through the interim analysis. Of the adults and adolescents who had previously been treated prophylactically and who remained in the study through the interim analysis (n=128), 72 percent maintained their prophylactic dosing interval and 22 percent lengthened and six percent shortened the time between infusions. Extension study participants could change treatment group at any time.
“The design of the ASPIRE study provides physicians a high degree of dosing flexibility, with the goal of reflecting their real-world treatment practices,” said Guy Young, M.D., Director of the Hemostasis and Thrombosis Center, Children’s Hospital of Los Angeles. “The results suggest prophylaxis with ELOCTATE shows efficacy and safety for the long-term treatment of hemophilia A.”
In ASPIRE, most participants received prophylactic treatment and were able to maintain protection against bleeding episodes with ELOCTATE consumption that was consistent with that observed in A-LONG and Kids A-LONG.
The publication reported cumulative duration of treatment from the beginning of the A-LONG and Kids A-LONG studies through the ASPIRE interim data analysis. The median cumulative duration of treatment was 117.7 weeks for adults and adolescents, and 51.5 weeks for children less than 12 years old.
Across age groups, safety results were consistent with the general hemophilia A population. There were no reports of serious allergic reactions or vascular clots. The most common adverse events (incidence of greater than or equal to five percent) included nasopharyngitis (common cold), arthralgia (joint pain) and upper respiratory infection.
“These published results add to the body of data demonstrating ELOCTATE’s safety profile and ability to provide protection against bleeding episodes,” said Wing-Yen Wong, M.D., vice president, Global Medical, Hematology and Immunology at Biogen. “As a company focused on bringing treatment advances to the hemophilia community, we are committed to gathering comprehensive, long-term clinical data across populations.”
“We remain focused on the goal of elevating hemophilia care globally, and we believe the publication of these data is important clinical research that contributes to the advancement of medical science in hemophilia,” said Birgitte Volck, M.D., Ph.D., senior vice president of Development and chief medical officer of Sobi. “These interim extension data help affirm the known efficacy and safety of ELOCTATE for the treatment of people with hemophilia A.”
ASPIRE is an open-label, non-randomized, multi-year extension study for people who completed the pivotal, Phase 3 A-LONG or Kids A-LONG studies. The study enrolled 211 males, including 150 (98 percent) of those who completed A-LONG and 61 (91 percent) of those who completed Kids A-LONG. The primary endpoint is the development of inhibitors. Secondary endpoints include the annualized number of bleeding episodes per subject, ELOCTATE exposure days and a participant’s assessment of response to treatment of a bleeding episode.