Inspiration Biopharmaceuticals Announces Data from OBI-1 Pivotal-Stage Program in Hemophilia at 23rd ISTH Congress

KYOTO, Japan, July 28, 2011 /PRNewswire/ -- Inspiration Biopharmaceuticals, Inc. (Inspiration) today announced 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 clinical trial program, designed to study OBI-1 in acquired hemophilia.  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 in individuals with congenital hemophilia A and inhibitors, even in the presence of high inhibitor levels. All 25 bleeds in the study were controlled, and twenty 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 Negrier, 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."

About Hemophilia and Acquired Hemophilia

Hemophilia is a bleeding disorder caused by low levels or the absence of a protein called a coagulation factor, essential for blood clotting.  The two most common forms of hemophilia are types A and B.  Hemophilia A is caused by a factor VIII deficiency and the congenital form occurs in - 1 out of every 5,000 male births. Hemophilia B is caused by factor IX deficiency and occurs in - 1 out of every 30,000 male births. Approximately 60% of persons with hemophilia have a severe condition, which results in frequent spontaneous bleeding episodes, in addition to serious bleeding after injuries. The annual market for hemophilia treatments is estimated at $8 billion worldwide.

Acquired hemophilia is a rare, though potentially life-threatening bleeding disorder caused by the development of autoantibodies (inhibitors) against coagulation factors. Unlike congenital hemophilia, acquired hemophilia is typically a disorder of older adults, and occurs equally in both males and females.  Also, the pattern of bleeding seen in acquired hemophilia is different from that observed in the more common congenital form. In acquired hemophilia, individuals typically bleed into the skin, muscles and soft tissues, as opposed to bleeding into joints, which is more typical in congenital hemophilia.

In addition, approximately one-third of individuals with hemophilia A develop an immune reaction (inhibitors) to human FVIII (hFVIII), and can no longer respond to replacement treatment with the coagulation factor. Current therapies, specifically human factor VIIa (NovoSeven®) and FEIBA, work by bypassing the natural hemostatic pathway and forcing coagulation with much higher levels of FVIIa than normal.

About OBI-1 and the Clinical Development Program

In the fourth quarter of 2010, the Accur8 clinical trial program started, and OBI-1 entered pivotal clinical testing in individuals with acquired hemophilia (Accur8 Auto-antibody clinical trial). Inspiration plans to initiate a second Phase 3 clinical trial before the end of this year, in individuals with congenital hemophilia A who have developed inhibitors against FVIII (Accur8 Allo-antibody clinical trial).

OBI-1, a recombinant form of porcine FVIII which typically possesses low cross reactivity to anti-human FVIII antibodies, is a replacement therapy, activating the natural hemostatic pathway.  This should allow clinicians to correlate activity and efficacy with a biomarker, and therefore guide dosing to better monitor and predict treatment outcomes. OBI-1 presents a unique and alternative approach to address the needs of individuals who have developed inhibitors to FVIII and is highly desired by the medical community.

For more information about enrolling in one of Inspiration's clinical trials, please visit www.hemophiliaregistry.com or call 1-800-361-3227. For more information on the ongoing clinical studies, please visit http://www.clinicaltrials.gov.

About Inspiration Biopharmaceuticals

Inspiration Biopharmaceuticals is the only company exclusively dedicated to developing treatments for hemophilia, with a primary mission to broaden access to care, including prophylactic therapy, and to improve the treatment of individuals with inhibitor complications.  Inspiration has a broad portfolio of recombinant hemophilia products, which includes two late-stage products in clinical development and two pre-clinical programs.

Inspiration's lead product candidates are IB1001, an intravenous recombinant factor IX (FIX) for the treatment and prevention of bleeding in individuals with hemophilia B, and OBI-1, an intravenous recombinant porcine factor VIII (FVIII) for the treatment of individuals with hemophilia A who have developed inhibitors against human FVIII, and for individuals with acquired hemophilia.  Both products are in clinical development.  Earlier-stage preclinical programs at Inspiration are focused on human recombinant factor VIIa (FVIIa), for individuals with either hemophilia A or hemophilia B who have developed inhibitors, and for individuals with factor VII deficiency; and human recombinant FVIII, to treat individuals with hemophilia A.

Inspiration has extensive expertise and experience in hemophilia product development, biologics manufacturing, and global commercialization. The Company's senior leadership was directly responsible for the development and commercialization of the majority of hemophilia products currently on the market.  In January 2010, Inspiration entered into a long-term strategic partnership with Ipsen SA, leveraging the combined expertise and resources of the two companies. As part of that transaction, Inspiration in-licensed OBI-1 from Ipsen. For further information on Inspiration, please visit http://www.inspirationbio.com.

SOURCE Inspiration Biopharmaceuticals, Inc.