Inspiration Biopharmaceuticals Inc. has received Fast Track designation from the US Food and Drug Administration (FDA) for OBI-1 in acquired haemophilia A. OBI-1, an intravenous recombinant porcine factor VIII (FVIII), is being evaluated for the treatment of individuals with acquired haemophilia A, who have developed inhibitory antibodies (inhibitors) against their innate FVIII.

Fast track is a designation that the FDA reserves for a drug intended to treat a serious disease and has a potential to fill an unmet medical need. Fast track designation is designed to facilitate the development and expedite the review of new drugs. Marketing applications for fast track development programmes are likely to be considered appropriate for priority review, which implies an abbreviated review time of eight months. Inspiration intends to submit a biologics license application (BLA) to FDA in the first half of 2013.

OBI-1 received Orphan Drug Designation from the FDA in March 2004 and from the European Medicines Agency (EMA) in October 2010.  

In November 2010, Inspiration initiated the first pivotal study of OBI-1 for the treatment of severe bleeding in individuals with acquired hemophilia A.  The pivotal phase III study is a prospective, nonrandomized, open-label study evaluating the efficacy of OBI-1 for the treatment of serious bleeding episodes in individuals with acquired hemophilia  caused by development of autoimmune inhibitory antibodies to human FVIII.  Serious bleeding episodes include those that are a threat to a patient’s life or vital organs or limbs, or which require a blood transfusion.

Results from the first 12 subjects in this clinical study were presented during the World Federation of Hemophilia (WFH) Meeting in July 2012.Enrollment in the OBI-1 acquired hemophilia A clinical trial is ongoing.

In the fourth quarter of 2010, OBI-1 entered late-stage clinical testing in individuals with acquired hemophilia, a rare, potentially life-threatening bleeding disorder, which, unlike congenital hemophilia, typically affects older adults and occurs equally in both males and females. Further, Inspiration has initiated a second pivotal clinical trial in individuals with congenital hemophilia A who have developed inhibitors against human FVIII. OBI-1 provides clinicians with a unique, alternative approach to address the needs of individuals who have developed inhibitors to FVIII, and has been greeted with enthusiasm by the medical community.

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. Acquired hemophilia A, a rare condition, is caused by autoantibodies formed against the person’s own FVIII, often brought on by an underlying medical condition mostly in the elderly population. Hemophilia B is caused by a deficiency of coagulation factor IX and occurs in ~1 out of every 30,000 male births.

In January 2010, Inspiration entered into a strategic agreement with Ipsen, leveraging the combined expertise and resources of the two companies, to develop a broad portfolio of hemophilia products and two products in phase III. IB1001,  an investigational intravenous recombinant factor IX (rFIX) therapy for the treatment and prevention of bleeding episodes in people with hemophilia B, and OBI-1, an investigational intravenous recombinant porcine factor VIII (rpFVIII) therapy for the treatment of patients with i) acquired hemophilia A and ii) congenital hemophilia A who have developed inhibitors against human FVIII.

In August 2011, Ipsen and Inspiration announced the extension of their agreement to create a hemophilia business unit structure that will act as the exclusive sales organization for all hemophilia products commercialized under the Inspiration brand in Europe. On 21 August 2012, Ipsen and Inspiration renegotiated their 2010 partnership. This agreement aimed to establish an effective structure whereby Ipsen gained commercial rights in key territories. Inspiration remains responsible for the world-wide development of OBI-1 and IB1001. Ipsen paid Inspiration an upfront payment $30 million for these rights, providing Inspiration with time to secure independent third party financing and Ipsen with time to assess potential ways forward.

On 31 August 2012, Ipsen paid Inspiration $7.5 million and received a warrant for 15% of Inspiration’s equity, which Ipsen later terminated in October 2012.  Ipsen had agreed to pay Inspiration an additional $12.5 million if Inspiration had raised third party  financing by the contractual deadline of 30 September 2012. Inspiration did not manage to raise external funding by this contractual deadline. On 30 October 2012, Inspiration filed for protection under Chapter 11 of the United States Bankruptcy Code.  Inspiration is seeking a strategic buyer through a formal sale process.  Ipsen will include the commercialization rights obtained from Inspiration in August 2012 in the sale, as well as Ipsen’s OBI-1 manufacturing facility in Milford, Massachusetts.