Ipsen announced that its partner Inspiration Biopharmaceuticals, Inc. presented data from its clinical development program for OBI-1, an intravenous (IV) recombinant porcine factor VIII product (rpFVIII), intended for the treatment of bleeding in people with hemophilia A with inhibitors and in people with acquired hemophilia. The data were presented in a Scientific Session held in conjunction with the 23rd Congress of the International Society on Thrombosis and Haemostasis (ISTH), which was chaired by Amy Shapiro, M.D., Co-Medical Director at the Indiana Hemophilia and Thrombosis Center (IHTC).
During the Scientific Session, Anne Greist, M.D., Co-Medical Director at IHTC, presented interim results from the first registration study in the OBI-1 Accur8 clinical trial program. A total of three patients with acquired hemophilia, who had experienced severe bleeds not controlled with by-passing agents, were treated with OBI-1; in all three patients, treatment with OBI-1 stopped the bleeding. Further data on hemostatic efficacy and safety are being collected as part of the Accur8 program, designed to study OBI-1 in acquired hemophilia. According to Inspiration, a second study in individuals with congenital hemophilia A who have developed inhibitors against FVIII is set to commence later this year.
Additional reports from the Scientific Session confirmed findings from the Phase 2 study in congenital hemophilia A with inhibitors; that OBI-1 effectively resulted in hemostasis, and controlled all non-life/non-limb threatening bleeding episodes (minor bleeds) in those patients, even in the presence of high inhibitor levels against hFVIII. All 25 bleeds in the study were controlled, and 20 out of 25 bleeds (80%) were controlled with two infusions. In over 40 infusions administered, OBI-1 was well tolerated by all participants and no drug-related serious adverse events were observed.
Dr. Greist commented, “Acquired hemophilia can be a life-threatening crisis in which individuals develop antibodies against their own coagulation factor. Unfortunately, current therapies for inhibitors do not provide the same level of hemostatic efficacy as do human FVIII therapies for non-inhibitor patients. Further limitations include the inability to guide dosing and monitor treatment efficacy using established laboratory parameters. OBI-1 represents a potential alternative treatment, which allows established laboratory parameters to guide dosing and monitor efficacy, in addition to clinical outcome. I am happy to report the progress that has been seen in this development program.”
Nonclinical findings were also presented at the ISTH Congress, showing that OBI-1 corrected biomarkers of blood coagulation in vitro in a dose-dependent, anti-OBI-1 inhibitor titer-dependent fashion, including in plasma taken from individuals with congenital hemophilia A who have inhibitors. The data were presented in Poster # No. 01829: “In vitro correction of thrombin generation and improvement of clot structure by recombinant porcine factor VIII in plasma containing anti-factor VIII inhibitory antibodies.”
According to Claude Négrier, M.D., author on the poster and head of the Hematology Department at Edouard Herriot University Hospital (Lyon, France), “The study demonstrated that recombinant porcine FVIII has the potential to correct surrogate markers of haemostasis, depending on the anti-porcine FVIII titre and on dose, which would likely translate into in vivo effectiveness.”
Date: July 28, 2011
Filed Under: Drug Discovery