Inspiration commences enrollment in second OBI-1 Accur8 clinical trial program for congenital hemophilia A

Nov 28 2011

Inspiration Biopharmaceuticals, Inc. (Inspiration) today announced the initiation of patient enrollment in the second of two pivotal studies in the Company's OBI-1 Accur8 clinical trial program.  In the newly initiated clinical study, OBI-1, an intravenous recombinant porcine factor VIII (FVIII) product, will be evaluated for the treatment of individuals with congenital hemophilia A who have developed inhibitory antibodies (inhibitors) against their human FVIII replacement therapy.  In achieving this milestone, Inspiration has received a $25 million milestone payment from the Ipsen Group (Euronext: IPN; ADR: IPSEY), under a partnership agreement signed with Ipsen in January 2010.  In return, Inspiration has issued Ipsen a convertible note, bringing Ipsen's fully diluted ownership position in Inspiration to 40.7%.

The OBI-1 pivotal clinical study is a prospective, non-randomized, open-label study evaluating the efficacy of OBI-1 for the treatment of serious bleeding episodes, including episodes that are a threat to a patient's life or vital organs. 

Dr. Johnny Mahlangu, MD, Director of the Adult Hemophilia Comprehensive Care Unit at Johannesburg Hospital, and President of the South African Society of Hematology, commented, "There remains a significant unmet medical need when it comes to treating individuals with hemophilia A who have developed inhibitors.  Current therapies do not reach the same level of efficacy as replacement therapy for non-inhibitor patients, leading to potential joint complications, increased risk of bleeding, pain and impact on the patient's quality of life.  Unlike available bypassing agents, OBI-1 facilitates the intrinsic hemostatic pathway, and therefore may allow clinicians to correlate activity and efficacy with FVIII levels (a surrogate for efficacy), enabling them to guide dosing and better predict treatment outcomes.  We are pleased to have enrolled the first patient in this pivotal clinical study."

Inspiration in-licensed OBI-1 from Ipsen as part of their January 2010 partnership agreement, whereby Inspiration is responsible for the clinical development, regulatory approval and commercialization of the product.  Previously, in November 2010, Inspiration initiated the first pivotal study of OBI-1 for the treatment of individuals with acquired hemophilia A, a rare, potentially life-threatening autoimmune bleeding disorder caused by the development of inhibitors against endogenous FVIII.  Results from the first patients in this clinical study were presented in a Scientific Session held in conjunction with the 23rd Congress of the International Society on Thrombosis and Haemostasis (ISTH) in July 2011.  Enrollment in the OBI-1 acquired hemophilia clinical trial is ongoing.

John P. Butler, Chief Executive Officer of Inspiration, commented, "Inspiration is committed exclusively to driving innovation that can bring positive impact to the treatment of people with hemophilia.  We now have OBI-1 in pivotal development for multiple indications.  We also recently announced the filing of a Marketing Authorization Application (MAA) for our other lead program, IB1001, a recombinant factor IX product for the treatment of hemophilia B.  These two late stage products, along with our preclinical programs for factor VIIa and FVIII deficiency, gives Inspiration one of the broadest pipelines for hemophilia treatment in the industry.  As a company, we are committed to developing and commercializing an array of hemophilia products, which will provide value to physicians and to people living with the condition worldwide."