Inspiration Biopharmaceuticals Demonstrates Non-Inferiority of IB1001, Its Recombinant Factor IX for Hemophilia B

GENEVA, Feb. 3, 2011 /PRNewswire/ -- Inspiration Biopharmaceuticals, Inc. (Inspiration) today presented pharmacokinetic (PK) data on its lead product, IB1001, a recombinant factor IX (FIX) for the treatment and prevention of bleeding in individuals with hemophilia B.  Results of the Phase 1 portion of an ongoing IB1001 clinical study demonstrated non-inferiority of IB1001 in achieving overall levels of replacement factor compared to BeneFIX®, the only approved recombinant FIX product for the treatment of hemophilia B.  Currently, IB1001 is in Phase 3, with safety and efficacy results expected later this year.

The clinical results were presented at the 4th Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) in Geneva, Switzerland, in a poster presentation titled, "Pharmacokinetics of IB1001, a New Recombinant Factor IX".

Ed Gomperts, M.D., Medical Director of Inspiration Biopharmaceuticals, commented, "We are very encouraged by the clinical results for IB1001, which showed PK results at least as good as the existing, marketed recombinant FIX product.  Further, the findings were similar in terms of half-life and in vivo recovery, which is fundamental for the determination of dose for the prevention and treatment of spontaneous bleeds, as well as trauma associated with surgical procedures."

Dr. Gomperts continued, "There remains a large gap in hemophilia treatment, with access to care primarily limited by availability and cost of therapy.  New initiatives are greatly needed globally.  Pending approval, a new recombinant FIX product such as IB1001 will increase product availability and thus should improve access to care, including prophylactic therapy of hemophilia B in a market where recombinant FIX is limited to only one approved product."

"We are pleased with the continued progress of the IB1001 program," stated Michael Griffith, Ph.D., President and CEO of Inspiration.  "In the past 12 months, Inspiration has made great strides in product development for the treatment of hemophilia, including the initiation of pivotal testing in its two lead programs.  In addition to IB1001, we are developing OBI-1, a recombinant porcine factor VIII (FVIII) for individuals who have developed inhibitors against human FVIII and for those with acquired hemophilia A.  OBI-1 specifically addresses the unmet medical need in inhibitor therapy for individuals with hemophilia."

The randomized, double-blind, cross-over PK protocol was designed to show non-inferiority of IB1001 compared to the only marketed recombinant FIX product.  The study enrolled 32 individuals with severe hemophilia B.  Individuals in the study were infused with either IB1001 or the comparator, and their FIX levels were assessed at different time intervals.  After a predetermined time period, individuals received the second product and FIX levels were assessed at the same time points.

Authors of the poster presentation were Uri Martinowitz, M.D., of the Israeli National Hemophilia Center; Amy D. Shapiro, M.D., of the Indiana Hemophilia and Thrombosis Center; Doris V. Quon, M.D., Ph.D., of the Hemophilia Treatment Center at Orthopaedic Hospital (Los Angeles, CA); Miguel A. Escobar, M.D., of the University of Texas Houston Health Sciences Center; Christine Kempton, M.D., of Emory University School of Medicine (Atlanta, GA); Peter W. Collins, M.D., of the Arthur Bloom Haemophilia Centre University Hospital of Wales School of Medicine, Cardiff University (Cardiff, UK); Pratima Chowdary, M.D., of the Royal Free Hospital (London, UK); Michael Makris, M.D., of the Royal Hallamshire Hospital (Sheffield, UK); Pier M. Mannucci, M.D., of the University of Milan Haemophilia & Thrombosis Center; Massimo Morfini, M.D., of Azienda Ospedaliero - Universitaria Careggi (Florence, Italy); Leonard A. Valentino, M.D., of Rush University Medical Center (Chicago, IL); and Ed Gomperts, M.D., and Martin Lee, Ph.D., both of Inspiration Biopharmaceuticals.

Inspiration is continuing to recruit patients for the Phase 3 portion of the Company's IB1001 study, including for the treatment of patients undergoing major surgical procedures.  For more information on the ongoing study, please visit http://www.clinicaltrials.gov/ct2/show/NCT00768287.

About Hemophilia

Hemophilia is a bleeding disorder caused by low levels or 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 $7.5 billion worldwide.

About IB1001

IB1001, Inspiration's lead product candidate, is an intravenous recombinant FIX product being developed for the treatment and prevention of bleeding in individuals with hemophilia B.  IB1001 currently is in pivotal Phase 3 clinical testing.  To date, IB1001 has been well-tolerated, and pharmacokinetic results have demonstrated non-inferiority to the one approved recombinant FIX product for the treatment of hemophilia B.  Pending results from clinical studies, regulatory approval and commercialization, IB1001 is expected to be the second recombinant FIX product to market, and thereby will increase product supply and access to care worldwide.

About Inspiration Biopharmaceuticals

Inspiration Biopharmaceuticals is dedicated exclusively 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 who have developed inhibitors against human FVIII and for individuals with acquired hemophilia A.  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, regulatory approval 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 addition, in January 2010, Inspiration entered into a strategic partnership with Ipsen SA, leveraging the combined expertise and resources of the two companies.  For further information on Inspiration, please visit http://www.inspirationbio.com.

SOURCE Inspiration Biopharmaceuticals, Inc.