Our Future

As part of our vision to broaden the choices available for hemophilia treatment, we are excited to be working on several products that we believe will positively impact the lives of people living with hemophilia and related complications. Two of our product candidates are in advanced clinical development:

Inspiration's first product, IB1001, is an intravenous recombinant factor IX (rFIX) product being developed for the treatment and prevention of bleeding in people with hemophilia B. In September 2011, Inspiration submitted the first marketing application for IB1001 in the EU, with a future submission planned for the US Food and Drug Administration (FDA).

Upon licensure, IB1001 is expected to be the second rFIX treatment to market, which will increase treatment choices for people with hemophilia. More information about IB1001 is expected to become available later in 2011.

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.

Approximately one-third of people with hemophilia A develop inhibitors, which means that the immune system has developed antibodies that prevent the proteins in standard hemophilia therapies from working.¹ The development of inhibitors makes their normal treatment regimen ineffective and severely limits lifestyle choices for people with hemophilia.

Inspiration's second product, OBI-1, is an intravenous recombinant porcine factor VIII (rpFVIII) intended for the treatment of bleeding in people with hemophilia A with inhibitors and acquired hemophilia A. OBI-1 works differently than currently available inhibitor treatments because it allows the natural clotting pathway to be functional.² Due to this unique characteristic, factor VIII levels can be measured to monitor treatment and adjust the dosing if necessary, with the goal of better treatment outcomes for people with inhibitors.

OBI-1 presents a unique and alternative approach to address the needs of people who have developed inhibitors. This development program has been greeted with a great deal of enthusiasm by the medical community.

OBI-1 has been evaluated in early-stage clinical testing in people with congenital hemophilia A who have developed inhibitors and who presented with a non–life-/non–limb-threatening bleed. This trial demonstrated that OBI-1 was well tolerated and can be given over a short infusion time.³

In November 2010, OBI-1 entered late-stage clinical testing in people with acquired hemophilia A. By the end of 2011, Inspiration plans to initiate a second late-stage trial in people with congenital hemophilia A who have developed inhibitors to human FVIII.