CAMBRIDGE, Mass., April 17, 2012 /PRNewswire/ -- Inspiration Biopharmaceuticals, Inc. today announced that it has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for the approval of IB1001, an intravenous recombinant factor IX (rFIX) protein for the treatment and prevention of bleeding in individuals with hemophilia B.
Based on the terms of its agreement with Ipsen (Euronext: IPN; ADR: IPSEY), Inspiration will receive a $35 million milestone payment associated with its filing of the BLA. In return, Inspiration will issue a convertible note to Ipsen, bringing Ipsen's fully diluted equity ownership position in Inspiration to approximately 43.5%.
Approval of IB1001 would represent a significant advance for the hemophilia community, including the estimated 75% of people living with hemophilia worldwide who do not have adequate access to currently available treatments. IB1001 would be the first recombinant therapy for people with hemophilia B introduced in more than 15 years.
Regulatory review is now pending in both the U.S. and Europe. Inspiration's Marketing Authorization Application for IB1001 was accepted by the European Medicines Agency in September 2011. The company is currently finalizing plans for additional regulatory filings and preparing for the commercial launch of IB1001.
"The introduction of recombinant factor IX was an important step forward in the treatment of hemophilia B, but a single product is not sufficient to meet the needs of all people affected by this disease," said John P. Butler, Inspiration's Chief Executive Officer. "At Inspiration, we are solely focused on the needs of people with hemophilia, and we are proud of the progress we have made towards bringing forward a new choice of treatment for patients. We plan to work closely with regulatory authorities both here in the U.S. and around the world to make IB1001 available as broadly and rapidly as possible."
The IB1001 BLA filing includes a comprehensive set of pharmacokinetics safety, and efficacy data from a Phase 3 clinical trial in people affected by hemophilia B. A surgery substudy was also included.
John Taylor, Co-Founder and Chairman of Inspiration, stated, "With sons affected by hemophilia B, Inspiration co-founder Scott Martin and I are acutely aware of the impact of limited treatment options on families. We believe more choice can help us achieve our mission to broaden access to care—increasing the supply of products, driving healthy competition, and supporting prophylaxis, which is rapidly becoming the standard of care in hemophilia. We believe that the approval of IB1001 will be the first step in our journey to make broader access to care a reality for thousands of individuals with hemophilia."
IB1001 is an intravenous rFIX product being developed for the treatment and prevention of bleeding in individuals with hemophilia B. IB1001 has completed pivotal Phase 3 clinical studies that support the filling of the BLA, conducted at study sites in the U.S., Europe and India.
To date, IB1001 has been well tolerated by patients and pharmacokinetic (PK) analyses have demonstrated non-inferiority to the one approved rFIX product currently marketed for the treatment of hemophilia B. Confirmatory PK studies reported at the 53rd Annual Meeting of the American Society of Hematology showed no evidence of development of inhibitors during treatment periods ranging from 4 to 18 months.
Results recently presented at the 5th Annual Congress of the European Association for Haemophilia and Allied Disorders demonstrated that IB1001 provided effective surgical hemostasis in study participants with hemophilia B undergoing major surgical procedures.
About Inspiration Biopharmaceuticals
Inspiration Biopharmaceuticals is exclusively dedicated to developing treatments for hemophilia, with a primary mission to broaden access to care by providing safe and effective recombinant therapies and advancing innovation for people living with hemophilia. Inspiration has a broad portfolio of recombinant hemophilia product candidates, which includes one under review by the FDA and EMA for marketing approval in the U.S. and Europe, one in late-stage clinical development, and two preclinical programs.
Inspiration's lead product candidates are IB1001 and OBI-1, an intravenous recombinant porcine factor VIII (FVIII) product being developed for the treatment of individuals with congenital hemophilia A who have developed inhibitors against human FVIII, and for individuals with acquired hemophilia. Inspiration has earlier-stage development programs focused on human recombinant factor VIIa (rFVIIa) for individuals with either hemophilia A or hemophilia B who have developed inhibitors or those with factor VII deficiency, and human recombinant FVIII for individuals with hemophilia A.
Inspiration's senior management team has broad experience and expertise in hemophilia product development, biologics manufacturing and the successful commercialization of products to treat hemophilia and other rare diseases.
In January 2010, Inspiration entered into a strategic agreement with Ipsen (EURONEXT: IPN; ADR: IPSEY), leveraging the combined expertise and resources of the two companies, to develop a broad portfolio of hemophilia products. As announced in late August 2011, Ipsen and Inspiration extended 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. For further information on Inspiration, please visit http://www.inspirationbio.com.
SOURCE Inspiration Biopharmaceuticals, Inc.