Ipsen/Inspiration’s Coagulation Factor IX Accepted For Review By EMA

As the two firms try to launch a hemophilia B therapy to compete with Pfizer’s BeneFix, EMA acceptance triggers $35 million milestone payment to Inspiration.

The European Medicines Agency has accepted an marketing authorization application filed by Ipsen Group and Inspiration Biopharmaceuticals Inc. for IB-1001, a recombinant coagulation Factor IX that analysts say could be a lower-priced competitor to Pfizer Inc.' s BeneFix. The companies are developing IB-1001 for both treatment and prevention of bleeding in individuals with hemophilia B.

The filing, announced Oct. 3, triggers a $35 million milestone payment from Ipsen to the venture-backed biotech under a collaboration the two companies signed in 2010. In exchange, Inspiration is issuing a convertible note to Ipsen, which will increase the French pharma company's equity stake in the biotech from 34% to 38%. That percentage could grow to 49% in the next two years if additional milestones are achieved.

Ipsen has high hopes for the 2010 deal, in which it paid $85 million for co-development rights to two hemophilia compounds and a 20% stake in Inspiration. The second compound, OBI-1, is a recombinant porcine Factor VIII for patients with acquired hemophilia and hemophilia A with inhibitory immune reaction to human forms of Factor VIII ('Finding Inspiration: Ipsen Builds Fourth Specialist Franchise With Hemophilia Deal,' 'The Pink Sheet' DAILY, Jan. 21, 2011).

In an unusual move, Ipsen also asked Inspiration to assume commercial responsibility for OBI-1, which currently is in Phase III trials.

Ipsen Holds Option To Buy Out Inspiration

Ipsen also is providing $259 million in R&D funding over the life of the 2010 deal and holds an option to acquire a controlling interest in the biotech if certain undisclosed events occur ([See Deal]).

If Ipsen buys Inspiration, it could provide an exit for Inspiration's primary backer, Celtic Pharmaceutical Holdings. "It's one of several possible exits - keep in mind Ipsen will have an option to acquire the company at market price but not necessarily an obligation" Inspiration Chief Financial Officer Gordon Busenbark said in an interview. "If they choose not to, then we have other possible exits including possibly an IPO or a partnership with another company."

IB-1001 could be positioned a less-expensive alternative to Pfizer's BeneFix, also a recombinant Factor IX product, because Inspiration has lower production costs. The Laguna Niguel, Calif., biotech has proprietary technology that allows for high-yield cell culture manufacturing, Busenbark explained.

"We think that's going to become increasingly important as pricing and price sensitivity becomes an important issue in health care and in hemophilia in particular," he said.

Several market analysts who follow Ipsen expect IB-1001 to gnaw away at Pfizer's market share if it gains EMA approval. Inspiration also plans to file the compound in the U.S. by mid-2012. It is taking longer to complete the FDA filing because the agency requested a larger trial than was required in Europe - 50 patients, rather than 20.

Noting that IB-1001 has demonstrated non-inferiority to BeneFix in clinical trials, Bryan Garnier analyst Cedric Moreau predicted in an Oct. 3 note that the compound will be approved in late 2012 and launched in the first quarter of 2013. That assessment was based on preliminary results, however, because Inspiration says it will not release full clinical data until the trial backing the FDA filing is complete.

"All in all, we think that IB-1001 could achieve globally a 15% market share at peak sales to reach €98 million in 2022 (€51 million in Europe)," he wrote. Moreau projects that Inspiration's entire hemophilia franchise, also including OBI-1, could yield sales totaling €350 million by 2020.

Other analysts are more bullish on IB-1001. Societe Generale, in an Oct. 3 note, predicts the product will achieve 20% global market share in hemophilia B, with peak sales of $200 million, roughly one-third of that in the U.S. and two-thirds in Europe and the rest of the world, while Tara Raveendran of Jefferies International Ltd. projects peak sales of $320 million. She noted that 2010 worldwide sales of BeneFix were roughly $634 million.

Lower price seems to be Inspiration's main competitive advantage in the hemophilia B market.

"The product from a functional point of view is equivalent to BeneFix, what we're really bringing to the market is choice and increased supply," Busenbark said. "In a disease like hemophilia, making sure that there is adequate supply and customer choice is very important. That way, more patients can be treated and have access to prophylaxis, which is the preferred therapy for hemophilia B patients."

Marketing Partnership For Europe, Russia

In August, Ipsen announced an extension of its partnership with Inspiration, under which the two companies will co-market IB-1001 and OBI-1 in Europe. A specialized unit within Ipsen's commercial infrastructure will market and sell the products in the EU, Russia and other parts of Europe ('Ipsen Extends Inspiration Deal, Appoints New CFO And Updates Sales Forecast,' 'The Pink Sheet' DAILY, Aug, 30, 2011).

"In the investment agreement and partnership that we entered in January 2010, Inspiration had full global commercialization rights for all products in our portfolio," Busenbark explained. "But, as we looked at the need to build a commercial infrastructure in Europe, it seemed like a natural extension of our partnership with Ipsen because of their very strong commercial presence in Europe."

Hemophilia is among Ipsen's recently reworked areas of focus - the pharma intends to have four specialty divisions, also including endocrinology, neurology and oncology ( (Also see "In Seeking Focus, Ipsen Turns To Specialty Pharma Model" - In Vivo, 1 Sep, 2011.)

-Joseph Haas ([email protected])