PARIS--(BUSINESS WIRE)-- Regulatory News: Ipsen (Euronext:IPN - News) today announced that its partner, Inspiration Biopharmaceuticals, Inc. (Inspiration), has been informed that the European Medicines Agency (EMA) has validated and accepted the filing of the Marketing Authorization Application (MAA) for Inspiration’s IB1001, a recombinant factor IX (FIX) product for the treatment and prevention of bleeding in individuals with hemophilia B. In doing so, the EMA has verified that it will begin its regulatory review process of the MAA.
The application includes safety and efficacy data from Inspiration’s clinical program for IB1001, which was conducted in the U.S., Europe, Israel and India.
In January 2010, Inspiration and Ipsen entered into a broad strategic partnership to develop and commercialize a unique portfolio of hemophilia products, which included Inspiration in-licensing OBI-1 from Ipsen, as well as Ipsen providing Inspiration with milestone-based funding to support the development of Inspiration’s two lead development programs, including FIX IB1001. Based on this partnership, by receiving the IB1001 MAA submission acceptance for review from the EMA, Inspiration will receive a $35 million milestone payment from Ipsen. In return, Inspiration will issue a convertible note to Ipsen, bringing Ipsen’s fully diluted equity ownership position in Inspiration to approximately 38%. In late August, Ipsen and Inspiration entered into a new agreement to create a hemophilia business unit structure being established within Ipsen’s existing European commercial organization, which will be the exclusive commercial agent for all products marketed under the Inspiration brand in Europe, including IB1001.
Marc de Garidel, Chairman and Chief Executive Officer of Ipsen, stated, “The filing of IB1001 by the European Medicines Agency is an important step forward in the development of our hemophilia franchise. We believe the hemophilia community will positively receive an alternative to the single recombinant Factor IX currently on the market. As we have announced in late August, Ipsen and Inspiration’s newly created Hemophilia Business Unit will actively prepare the marketing, medical and regulatory plans prior to the launch of IB1001 in Europe, pending regulatory authorization expected in late 2012.”
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 has completed the pivotal Phase 3 clinical testing for the European regulatory submission, while clinical testing for the US regulatory submission is ongoing. To date, IB1001 has been well tolerated by patients and pharmacokinetic results have demonstrated non-inferiority to the one approved recombinant FIX product currently available for the treatment of hemophilia B.
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 plans to initiate a second pivotal clinical trial by the end of this year 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. 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 estimated at $8 billion worldwide.
Ipsen is a global specialty-driven pharmaceutical company with total sales exceeding €1.1 billion in 2010. Ipsen’s ambition is to become a leader in specialty healthcare solutions for targeted debilitating diseases. Its development strategy is supported by four franchises: neurology / Dysport®, endocrinology / Somatuline®, uro-oncology / Decapeptyl® and hemophilia. Moreover, the Group has an active policy of partnerships. R&D is focused on innovative and differentiated technological patient-driven platforms, peptides and toxins. In 2010, R&D expenditure totaled more than €220 million, above 20% of Group sales. The Group has total worldwide staff of close to 4,500 employees. Ipsen’s shares are traded on segment A of Euronext Paris (stock code: IPN, ISIN code: FR0010259150) and eligible to the “Service de Règlement Différé” (“SRD”). The Group is part of the SBF 120 index. Ipsen has implemented a Sponsored Level I American Depositary Receipt (ADR) program, which trade on the over-the-counter market in the United States under the symbol IPSEY. For more information on Ipsen, visit www.ipsen.com.
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