Inspiration Biopharmaceuticals Presents Data on Porcine Factor VIII Inhibitor Testing Methods at International Society on Thrombosis and Haemostasis Meeting

June 27, 2012
  • Supports accuracy and precision of testing methods used in the evaluation of OBI-1, an investigational recombinant porcine factor VIII therapy 
  • OBI-1 under investigation for individuals with congenital hemophilia A with inhibitors, and acquired hemophilia 

Liverpool, UK, June 27, 2012 – Inspiration Biopharmaceuticals today presented the results of a non-clinical study evaluating the precision and accuracy of an assay used to monitor the development of inhibitory antibodies to porcine factor VIII.  The in vitro study, which involved fifteen of the international sites participating in Inspiration's Phase 3 Accur8 clinical trial program, found that the degree of variability in results obtained from assays used to monitor for porcine factor VIII inhibitors is similar to that seen with assays for human factor VIII inhibitors.  The results were presented in a poster presentation today at the 58th Annual Meeting of the Scientific and Standardization Committee (SSC) of the International Society on Thrombosis and Haemostasis (ISTH) in Liverpool, UK. 

Inspiration is developing an investigational recombinant porcine factor VIII, OBI-1, for the treatment of bleeding in people with congenital hemophilia A with inhibitors, or acquired hemophilia. 

"The ability to accurately monitor development of anti-porcine antibodies is a key component of our ongoing safety and efficacy studies with OBI-1," said Howard Levy, MD, Chief Medical Officer at Inspiration and a co-author on the study.  "We are pleased with these findings, which suggest that labs around the world who already monitor inhibitors to human factor VIII can successfully adapt their monitoring procedures to assess inhibitors to porcine factor VIII. These study results support the validation of our ability to accurately monitor factor VIII as an indicator of the response to OBI-1 as we continue to advance the OBI-1 clinical development program."

Study Design and Results
Centers participating in the OBI-1 Phase 3 Accur8 Auto-antibody study tested a set of inhibitor samples of a human anti-C1 factor VIII antibody with strong cross-reactivity to porcine factor VIII.  Local laboratories performed a regular Bethesda assay (the assay commonly used to monitor inhibitors to human factor VIII in people resistant to treatment with clotting factor concentrate), substituting a standard OBI-1 preparation pre-diluted in human factor VIII deficient plasma creating a "normal porcine plasma".  Fifteen laboratories in five countries participated in the study.  The inter-laboratory coefficient of variation (CV) ranged from 29.5% to 56.8% for the various samples, comparable to the CV seen with the Bethesda assay measuring inhibitors to human factor VIII.[i]

"OBI-1 is being developed as a potential treatment option for people with congenital hemophilia A, and acquired hemophilia who have developed inhibitors to human factor VIII, which can  render some existing treatments ineffective at controlling their bleeds.  For these inhibitor patients, accurate monitoring of anti-porcine inhibitors is important to determine the overall safety and efficacy profile of this investigational product," said Edward D. Gomperts, MD, Director of Clinical Research, Clinical Investigation Center, Children's Hospital Los Angeles and Consultant to Inspiration Biopharmaceuticals.

About Inhibitor Development in Hemophilia A and Acquired Hemophilia
People with hemophilia A can develop inhibitors (also called antibodies) against the coagulation factor VIII concentrates that are commonly used to treat or prevent bleeding.  This can make treatment of bleeds very difficult.  People with acquired hemophilia develop antibodies, in their case, to the native factor VIII their bodies produce. The antibodies make them unable to effectively use their native factor VIII or respond to human factor VIII concentrate. 

About OBI-1
OBI-1, a recombinant porcine factor VIII designed to avoid recognition by antibodies to human factor VIII, is under investigation for people with congenital hemophilia A with inhibitors, and people with acquired hemophilia. People with inhibitors to human factor VIII may respond to porcine factor VIII, replacing the missing step in their intrinsic coagulation cascade and promoting clotting. Because OBI-1 is a recombinant porcine factor VIII, some people undergoing treatment with OBI-1 may develop anti-porcine antibodies. The safety and efficacy of OBI-1, in addition to the development of antibodies to porcine factor VIII, are currently being evaluated in pivotal Phase 3 clinical trials in both congenital hemophilia A with inhibitors, and acquired hemophilia.

About Inspiration Biopharmaceuticals
As the only biopharmaceutical company dedicated solely to hemophilia, Inspiration is committed to improving the care of people with this condition by broadening treatment choices, expanding global access to care, and advancing innovative therapies.  Founded by two families whose sons have hemophilia, Inspiration is inspired to make a difference in the lives of people impacted by hemophilia around the world. Inspiration's lead product candidates are OBI-1, and IB1001 which is an investigational intravenous recombinant factor IX being developed for the treatment of hemophilia B. For more information about Inspiration Biopharmaceuticals, please visit


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Feinstein Kean Healthcare
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