Laguna Niguel-based Inspiration Biophar-maceuticals Inc. is armed with a large war chest and has a growing staff as it works on a new way of treating hemophilia.
The war chest comes from French drug maker Ipsen SA, which earlier this year struck a deal to provide up to $259 million toward Inspiration’s bid to develop products to treat forms of hemophilia.
About a third of the money bought Ipsen a 20% stake in Inspiration. Most of the rest goes toward convertible notes Ipsen will be able to turn into equity over time. That likely would come in chunks and dovetail with certain research and development milestones, eventually giving Ipsen the chance to buy the company.
One milestone has been reached: Late last month Inspiration received a $50 million payment from Ipsen based on the start of clinical trials for one of its products.
Inspiration expects to have 30 workers by year’s end, up from eight a year ago.
Hemophilia strikes mainly males with low levels or an absence of proteins called coagulation factors, which clot the blood.
In the Pipeline
Inspiration has two primary drugs in development. Its lead compound, IB1001, is in late-stage clinical trials for treating patients with congenital hemophilia B, the less common form of the disease.
Congenital hemophilia B occurs in less than 1 of every 30,000 boys. Congenital hemophilia A is six times more likely.
Inspiration’s other major compound, OBI-1, is being tested to treat patients who have developed resistance to traditional hemophilia treatments, which typically consist of intravenous injections of coagulation proteins.
The company also is looking at OBI-1 to treat what’s called acquired hemophilia A, which can happen when patients develop resistance to coagulation proteins and develops in older males and females.
The French drug maker sees Inspiration as a chance to bolster its development of hemophilia treatments.
“Ipsen had one hemophilia product that they were working on and had an interest in developing a larger hemophilia franchise,” said Gordon Busenbark, Inspiration’s chief financial officer. “They also had the financial deep pockets required to bring biotech products, like things that we’re working on, to market.”
The global market for hemophilia treatments is estimated at $8 billion annually.
There are a number of big players. Pfizer Inc. of New York has a Food and Drug Administration-approved human recombinant clotting factor product called Benefix for treating hemophilia B.
If Inspiration’s IB1001 is approved it would become only the second product of its type on the market. The company said that would increase access to care for hemophilia patients worldwide, including the 75% to 80% of those with B type who never get treated for it.
“Benefix is a very good product,” Busen-bark said. “The only problem is it’s limited in terms of product supply and it’s expensive.”
Inspiration is based in Orange County rather than traditional biotechnology haven San Diego because Chief Executive Michael Griffith previously ran a local company called Bluebird Biosciences Inc. that made recombinant protein products.
Griffith’s experience made him the top choice when a couple of business owners with no local ties but very personal interests in battling hemophilia founded Inspiration five years ago.
Common Cause
John Taylor and Scott Martin have sons with hemophilia B.
Taylor, a foreign exchange currency manager who runs an investment fund in New York, is Inspiration’s chairman. He also established the Coalition for Hemophilia B, an advocacy group.
Martin, who is in the oil distribution business in Houston, is Inspiration’s co-chairman.
Taylor and Martin had “been pursuing research in this area for many, many years” and eventually met and made it a common cause, said Griffith.
“What they were looking at was an alternate delivery to intravenous injections,” he said.
Traditional hemophilia treatment involves patients taking intravenous coagulation proteins two to three times a week “and that is very, very painful and problematic for little children especially,” Griffith said. Hemophilia sometimes is diagnosed in children as young as 2, according to Griffith.
Inspiration’s products could be used in what’s called prophylactic hemophilia therapy, a less-invasive effort to prevent or delay the progression of disabling problems that result from bleeding into a patient’s joints.
Taylor and Martin funded Inspiration for its first three years of existence.
Besides Ipsen and its early founder financing, Inspiration struck a funding and collaboration deal with Bermuda-based Celtic Pharma Management LP in 2008 valued up to $35 million.
Inspiration is pre-revenue and still some years from having products on the market. The company hopes to release IB1001 in 2012 and OBI-1 in 2013, with plans to seek regulatory approvals in the U.S. and Europe, Busenbark said.
Inspiration expects that its products will eventually be covered by either private insurance or Medicaid.
