Biopharmas bonded by blood

Ipsen and Inspiration Biopharmaceuticals to create hemophilia franchise

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PARIS—Striving toward providing more economical and higher-quality treatments for bleeding disorders, global specialty pharmaceutical group Ipsen has joined hands with Laguna Niguel, Calif.-based Inspiration Biopharmaceuticals to create a hemophilia franchise. The combined portfolio will cover all major hemophilia markets, with worldwide sales projected to reach $1 billion by 2020.

The partnership, announced Jan. 21, leverages the combined expertise and resources of the two companies to advance a broad portfolio of recombinant proteins, which address all major hemophilia disorders by focusing on two significant unmet needs: wider access to treatment with coagulation factors and treatment for inhibitor complications.

Jean-Luc BÈlingard, chairman and CEO of Ipsen, says Inspiration has both a "strong development experience and broad pipeline that will provide the opportunity for new growth on our way to building a new, multi-product global franchise in hematology."

The two lead product candidates scheduled to begin Phase III clinical testing in 2010 are Ipsen's recombinant porcine factor VIII, OBI-1, for the treatment of patients with acquired hemophilia and hemophilia A who have developed an inhibitory immune reaction to human forms of factor VIII, and Inspiration's recombinant factor IX product, IB1001, for the acute and preventative treatment of bleeding in patients with hemophilia.

The partnership calls for Ipsen to exclusively sublicense OBI-1 to Inspiration in exchange for $50 million in convertible notes and a 27.5 percent royalty on future OBI-1 sales. Inspiration will also enter a separate agreement with Ipsen for supply of the OBI-1 product and Ipsen will provide up to $259 million of funding to Inspiration. The proceeds will be used for the development and commercialization of its hemophilia pipeline, including OBI-1.

Ipsen will also make an upfront investment of $85 million in Inspiration in exchange for shares of a new class of preferred stock constituting 20 percent of Inspiration's fully diluted equity, the agreement states. Ipsen will also be entitled to appoint one individual to the seven-member Inspiration board of directors, and will pay milestone payments up to $174 million to Inspiration based on the successful development of IB1001 and OBI-1.

Hemophilia, congenital or acquired, is a bleeding disorder caused by low levels or absence of a protein called a coagulation factor, essential for blood clotting. There are two common forms. Hemophilia A is caused by a factor VIII deficiency and occurs in  less than1 out of every 5,000 male births. Hemophilia B is caused by factor IX deficiency and occurs in  less than 1 out of every 25,000 male births.

Approximately 60 percent of individuals with hemophilia have a severe condition, which results in frequent spontaneous bleeding episodes as well as serious bleeding after injuries. The market for hemophilia treatment is estimated at $7.5 billion annually.

"Inspiration's goal is to bring a better quality of life to patients suffering from
hemophilia—and we believe Ipsen is the right partner to help make that happen," says John Taylor, Inspiration co-founder and chairman. Ipsen "adds a valuable late stage product to our pipeline, but it will benefit from our existing development infrastructure and future specialty sales force. With the addition of OBI-1, we will be a worldwide leader in hemophilia providing a broad range of treatment options for individuals suffering from bleeding disorders."

Taylor and co-founder Scott Martin were inspired to start the company in 2004, when Taylor's son was 15 and Martin's son was two years old. Both of their boys have had hemophilia and led normal lives because their fathers' could afford the care. But both knew of many families who could not.

The cost of prophylactic infusions three times a week, crucial for a child with hemophilia to lead the life of a typical boy, is $400,000 a year, says Taylor, whose health insurance covered the expense. Parents who do not have the insurance or other means rely on infusions provided for emergencies, he says.

But it is the internal bleeding in hemophilia that could be more dangerous, leaking into and eroding the joints and cutting lives short, Taylor points out.

Although 25,000 people suffer from hemophilia in the United States, hundreds of thousands around the world have hemophilia, but may not survive if they are untreated, Taylor says. Royal members of the Hapsburg Dynasty suffered from blood disorders, and today, many children in areas of Hungary and the Czech Republic live their lives cut short by hemophilia.

The key to effectively treating hemophilia is having affordable access to prophylactic care, shown to reduce complications of the disease and enhance patients' long-term health and quality of life, says Taylor, who believes costs will come down when there is more competition in the field.

Inspirations' lead product candidate, IB1001, is an intravenous recombinant factor IX product for the acute and preventative treatment of bleeding in patients with hemophilia B. Its earlier stage coagulation factor product candidates have been partially funded to date by Celtic Pharma, a global private equity and drug development firm.

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