Opsona, And Pharma Backers, Await Day of Reckoning for Kidney Drug

Xconomy National — 

Few biotech startups out of Dublin have gotten the type of industry backing that Opsona Therapeutics has. The company’s investor list reads like a who’s who of international pharma venture arms—among them Novartis, Amgen, and Roche—and investors close and abroad. It’s raised more than 60 million euros in venture cash to date.

The lure: Opsona offered a promising new way to control the immune system by targeting certain receptors on immune system cells. But promise and deep pockets only get a biotech so far. At some point, some real substantial clinical data is needed to keep the spigot from running dry. For Opsona, that critical day is coming.

Opsona is in the midst of testing its lead—and only—drug candidate, OPN-305, in a mid-stage clinical trial. It’s an antibody drug designed, initially, for patients that have just gotten kidney transplants. The idea is that OPN-305 would help reduce the risk of delayed graft function (DGF)—a common complication of transplants, when there’s a “delay” before the new organ starts working. During such delays, which may last several days, kidney transplant patients must be put on a dialysis machine. Some patients experience outright kidney failure.

Success in the trial, which is expected to wrap up towards the end of next year, could turn Opsona into acquisition fodder for one of its Big Pharma/Big Biotech backers—many of whom have been supporting the Irish biotech for close to a decade. Failure would bring a much tougher road, like trying to salvage the drug for another condition.

“That’s the real moment for the company, when we find out [the results of the trial],” says Opsona co-founder and chief scientific officer Luke O’Neill (pictured above), the chair of Trinity College Dublin’s biochemistry department.

Of course, in the competitive world of biotech even a successful clinical trial doesn’t necessarily mean commercial success. Other deep-pocketed companies are going after the same indication. Cheshire, CT-based Alexion Pharmaceuticals (NASDAQ: ALXN) is testing its blockbuster drug eculizumab, which already approved for rare blood diseases. San Francisco, CA-based Quark Pharmaceuticals is developing an RNA-based drug for the condition as well, though the drug failed a mid-stage trial in June.

But O’Neill believes Opsana has got a leg up on the competition, because of how it’s going after DGF. Opsana’s antibody targets a specific so-called “toll-like receptor” on immune cells called TLR2. These receptors are implicated in inflammation and infection-fighting. And O’Neill says they’re the key driver for the inflammation and injury in a newly transplanted kidney.

“Very early in the cascade these receptors get triggered,” O’Neill says. “So you’re getting in at the ground level, if you like, and stopping all the cascade of events that might happen next.”

O’Neill has been on a roughly 10-year quest to demonstrate the value of targeting the toll-like receptors. Opsona started up in 2004, budding out of the work O’Neill and two other immunologists at Trinity—Kingston Mills and Dermot Kelleher—were doing in inflammation.

It hasn’t been easy, historically, for startup biotechs in Dublin to get the necessary funding to leap from academic research to developing real products—crossing the so-called valley of death. There just wasn’t a critical mass of local venture firms and government support. But in the late 90s and early 2000s, initiatives like Enterprise Ireland (a government agency supporting Irish startups) and Science Foundation Ireland (a non-profit national foundation funding scientific and engineering research) sprouted up to help local startups. Opsona was one of the beneficiaries, landing an initial $20,000 from Enterprise Ireland.

That sum, of course, is a pittance in biotech, where it takes exorbitant amounts of cash to even get a drug into clinical trials, let alone all the way through development. And as O’Neill notes, the Dublin VC community was “sparse, to say the least, at the time.”

But Opsona had two things working in its favor: First, after setting up his lab at Trinity in the early 90s to study the molecular basis of inflammatory diseases, O’Neil worked hard to build relationships with industry. He did contract research and other collaborative work for Big Pharma and established relationships with people involved with biotech companies, like South San Francisco, CA-based Genentech. So after Enterprise Ireland got Opsona started, Seroba BioVentures (now Seroba Kernel) and Genentech joined in and helped back a 6.6 million euro Series A round in February 2005.

“I knew those guys [at Genentech], because I worked with them and collaborated before with them,” O’Neill says. “I was very plugged in internationally, [and] you’ve got to be.”

Second, Opsona was going after a hot target in biotech research—the toll-like receptors. TLRs are important components of what’s known as the innate immune system—the body’s first line of defense, which recognizes foreign invaders attacking the body at entry points just under the skin, or in the lining of the gut. (The adaptive immune system, by comparison, makes antibodies that can remember and develop the ability to destroy certain pathogens.)

Research into TLRs has shown they play a key role in diseases involving … Next Page »

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