Ipsen, a global specialty-driven pharmaceutical company, said that its California-based partner, Inspiration Biopharmaceuticals, Inc. (Inspiration) has started the treatment of the first patient in the second of two pivotal studies from the OBI-1’s Accur8 clinical trial programme.
According to a company press release, in this newly initiated clinical study, OBI-1, an intravenous recombinant porcine factor VIII (FVIII) product, will be evaluated for the treatment of individuals with congenital haemophilia A, who have developed inhibitory antibodies (inhibitors) against their human FVIII replacement therapy.
Under the partnership agreement signed with Inspiration in January 2010, the initiation of this clinical study triggers the subscription by Ipsen to a US$ 25 million convertible note newly issued by Inspiration. Ipsen’s fully diluted share ownership position in Inspiration now reaches about 40.7 per cent.
Marc de Garidel, chairman and CEO of Ipsen said: “The initiation of the second OBI-1 phase III clinical trial in congenital haemophilia A with inhibitors is an important step forward in the development of our haemophilia franchise. Currently, patients suffering from haemophilia A who have developed inhibitors have limited therapeutic options. The advancement of OBI-1 clinical programme paves the way towards potential major improvements in the therapeutic armamentarium within the coming years. We are pleased with the progress made by Inspiration who initiates in due time the second OBI-1 phase III clinical trial after having filed a Marketing Authorization Application (MAA) in Europe for IB1001, a recombinant factor IX.”
The OBI-1 phase III 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. The first patient is being treated at the Johannesburg Hospital in South Africa.
In November 2010, Inspiration initiated the first pivotal study of OBI-1 for the treatment of severe bleedings in individuals with acquired haemophilia A, caused by the development of inhibitors against human 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 haemophilia A clinical trial is ongoing.
OBI-1, a recombinant form of porcine FVIII which may possess low cross reactivity to antihuman FVIII antibodies, is a replacement therapy, activating the intrinsic haemostatic pathway. This should allow clinicians to correlate activity and efficacy with FVIII levels, a surrogate for efficacy in haemophilia, 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.
Haemophilia 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 haemophilia are types A and B. Haemophilia A is caused by a factor VIII deficiency and the congenital form occurs in ~1 out of every 5,000 male births. Haemophilia B is caused by factor IX deficiency and occurs in ~1 out of every 30,000 male births.
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.