Status: BLA expected in early 2013

Organizations involved:

Baxter Healthcare Corp. – Manuf.; R&D; Tech.; World mark.

Baxter AG. – Parent

Inspiration Corp. – Manuf.; R&D; Tech.; Former

Octagen Corp. – Former

Ipsen, Inc. – Former I

Ipsen S.A. – Parent; Former

Emory University – Tech.

Cross ref.: See the entries for Hyate:C, a former porcine-derived Factor VIII product (#901) and the Factor VIII Products entry (#715)

Description: OBI-1 is a recombinant porcine (pig) B-domain deleted Factor VIII.

Much like the product that preceded this and replaced by OBI-1, Hyate:C, OBI-1 provides clinicians with a unique, alternative approach to address the needs of individuals who have developed inhibitors (neutralizing antibodies) to Factor VIII.

Nomenclature: Factor VIII, porcine rDNA [BIO]; OBI-1 [SY]; rpFVIII [SY]; Recombinant porcine factor VIII (B domain deleted) [SY] Companies: In July 2008, Ipsen completed the purchase from Octagen Corp.,of all of its assets related to OBI-1. Ipsen paid Octagen an upfront milestone of $10.5 million. Immediately following the effective transfer of all of the assets related to OBI-1, Ipsen redeemed its stake in Octagen.

In Jan. 2010, Inspiration (which was founded by two businessmen with children with hemophilia A) entered into a strategic agreement with Ipsen to develop a broad portfolio of hemophilia products and two products in Phase III -- IB1001 (a Factor VIII product; see related entry) and OBI-1 intravenous recombinant porcine factor VIII (rpFVIII) therapy for the treatment of patients with acquired hemophilia A and congenital hemophilia A who have developed inhibitors against human Factor VIII (including human-derived and recombinant human Factor VIII products). In Aug. 2011, Ipsen and Inspiration extended of 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.

In Aug. 2012, Ipsen and Inspiration renegotiated their 2010 partnership. Ipsen gained commercial rights in key territories. Inspiration remains responsible for the world-wide development and manufacture of OBI-1 and IB1001. Ipsen paid Inspiration an upfront payment $30 million for these rights. Later in August 2012, Ipsen paid Inspiration $7.5 million and received a warrant for 15% of Inspiration’s equity, which Ipsen later terminated in October 2012. Ipsen also agreed to pay Inspiration an additional $12.5 million, if Inspiration had raised third party financing by the contractual deadline of 30 Sept. 30, 2012. Inspiration failed to raise external funding by this contractual deadline.

On Oct. 30, 2012, Inspiration filed for protection under Chapter 11 of the United States Bankruptcy Code. Inspiration was then seeking a strategic buyer through a formal sale process. Ipsen will include the commercialization rights obtained from Inspiration in August 2012 in the sale, as well as Ipsen’s OBI-1 manufacturing facility in Milford, MA. Ipsen agreed to provide Debtor-in-Possession (DIP) financing to Inspiration to fund Inspiration’s operations through the sale process.

In Jan. 2013, Baxter has agreed to pay over $50 million up front for full rights to psen and Inspiration Biopharmaceuticals for OBI-1 and its Boston-area manufacturing facility. Baxter pledged up to $135 million in additional development and commercial milestones, plus a double-digit percentage on potential sales of the candidate known as OBI-1, with Ipsen receiving the majority of royalties in the proposed deal. Read more: Baxter snags hemophilia candidate in $185M deal with Ipsen and broke biotech - FierceBiotech http://www.fiercebiotech.com/story/baxter-snags-hemophilia-candidate-185m-deal-ipsen-and-broke-biotech/2013-01-24#ixzz2Iv7Jqlf6 Subscribe: http://www.fiercebiotech.com/signup?sourceform=Viral-Tynt-FierceBiotech-FierceBiotech FDA class: Biologic BLA

Status: BLA pending.

In Nov. 2012, FDA granted Fast Track designation for OBI-1 in acquired haemophilia A. Inspiration at the time intended to submit a BLA to FDA in the first half of 2013.

OBI-1 has US and EU orphan designations.

Tech. transfer: Dr. Peter Lollar, Emory University, is credited as the inventor of recombinant porcine Factor VIII, and Emory Univ. has exclusively licensed related patents to Octagen/Inspiration. For example, see U.S patents 5,364,771 and 6,180,371 (and related patents) and "High Level Expression of Recombinant Porcine Coagulation Factor VIII," J. of Biol. Chem., 277, 38345-9, Oct. 2012. Emory originally licensed OBI-1 to Octagen Corp., a “homegrown” startup company founded in 1997. Octagen sublicensed the OBI-1 technology to Ipsen Biopharm in 1998.

Trials: 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 has initiated a second pivotal clinical trial in individuals with congenital hemophilia A who have developed inhibitors against human FVIII. Enrollment in the OBI-1 acquired hemophilia A clinical trial was ongoing in late 2012.