Inspiration Biopharmaceuticals Reports Positive Interim Results from the Accur8 Phase 2/3 Study of OBI-1 in Acquired Hemophilia A at WFH 2012 World Congress
Inspiration Biopharmaceuticals, Inc. (Inspiration) today reported data from its ongoing Phase 2/3 study evaluating the efficacy of OBI-1, an investigational recombinant porcine factor VIII (FVIII), for the treatment of serious bleeds in people with acquired hemophilia A. According to the interim analysis, seven out of seven trial participants receiving OBI-1 experienced control and subsequent resolution of their bleeds. Edward Gomperts, MD, Attending Hematologist and Director of Clinical Research, Clinical Investigation Center at Children's Hospital Los Angeles and Consultant to Inspiration, presented the findings in an oral presentation at the 2012 World Federation of Hemophilia (WFH) World Congress in Paris.
Acquired hemophilia A is a rare, sometimes life-threatening bleeding disorder that is caused by the development of autoantibodies (inhibitors) against FVIII, a key protein contributing to normal coagulation of blood. The antibodies render the native factor VIII ineffective in promoting clotting. Treatment of acquired hemophilia can be challenging, because the antibodies will attack any human form of FVIII. OBI-1 is designed to avoid recognition by antibodies to human FVIII. People with inhibitors to human FVIII may respond to porcine FVIII, replacing the missing step in their coagulation cascade.
"People with acquired hemophilia currently have limited treatment options to control serious bleeding episodes. These preliminary findings are encouraging and suggest that further investigation of the efficacy and safety of OBI-1 in this population is warranted," said Dr. Gomperts.
A second Accur8 Phase 2/3 clinical trial is also underway to evaluate OBI-1's ability to control bleeding in people with congenital hemophilia A with inhibitors and the safety of this drug in this population.
As part of Inspiration's expanded presence at the WFH World Congress, the company will host a symposium tomorrow entitled, "Hemophilia B: The Neglected Hemophilia," featuring a panel of experts exploring advancements in research, treatment and advocacy for hemophilia B, which is far less prevalent than hemophilia A.
Inspiration has a second product under development, which is a recombinant factor IX therapy being evaluated for the prevention and treatment of bleeding in people with hemophilia B and its safety profile in this population. This product is currently under regulatory review in both the U.S. and the EU.
"We are thrilled to be growing our presence and involvement at the WFH annual congress, the premier global scientific gathering for the hemophilia community," said John P. Butler, Chief Executive Officer of Inspiration. "Our goal as a company centers on broadening treatment choices, expanding global access to therapies and advancing innovation for people with all types of hemophilia. This meeting gives us an opportunity to connect face-to-face with leaders in the global hemophilia community, discuss their ongoing needs and determine the role Inspiration can play in improving the lives of people with hemophilia around the world."
Study Design and Results
The Accur8 Auto-antibody trial is an ongoing prospective, open label, Phase 2/3 study. The primary objective of the study is to evaluate the efficacy of OBI-1 treatment for serious (life- or limb-threatening) bleeds in individuals age 18 and older with acquired hemophilia A, as measured by the control of bleeding 24 hours after initial OBI-1 dose administration.
The interim analysis presented today included data from the first seven trial participants with severe bleeds not controlled with bypassing agents who were enrolled and treated with OBI-1. To date, 15 subjects have been enrolled and completed treatment in the clinical trial. Participants received 200U/kg of OBI-1 on first injection and were subsequently dosed based on target FVIII levels. All seven subjects experienced successful control of bleeds at 24 hours and subsequent resolution of their bleeds. Therapeutic FVIII activity levels were achieved and maintained with intermittent OBI-1 administration based on FVIII levels. Of the adverse events reported during the study thus far five were serious, but these were not considered by investigators to be treatment-related. As part of the ongoing Accur8 Auto-antibody trial, adverse event data continues to be collected including the evaluation of inhibitors to porcine factor VIII.
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