Up until earlier this year, Horizon Therapeutics was a stealth biotech. The biotech company wasn’t after a low profile--it aimed for complete anonymity. No press releases. No big news about its drug candidates.

Nada.

“We didn’t even have a website,” says CEO George Tidmarsh, MD, PhD. “We didn’t answer the phone, basically. Our goal was to stay under the radar screen until our first patent was published. We felt this area would be a bit of a foot race, and we didn’t want to give anyone an idea of what we were doing.”

Now, with a lead therapy in Phase III and $30 million in venture funds committed to its progress, Horizon sees good reason to start raising its profile considerably.

The biotech was born, explains Tidmarsh, around the time that Vioxx was pulled from the market. Tidmarsh, who has about 20 years of biotech experience including a stint as CMO of Coulter and founding president of Threshold, vividly recalls his scientific assessment:

“Wow! A huge market need exists here.

“We correctly surmised that doctors would go back to ibuprofen, away from the Cox-2 inhibitors, and that’s what happened,” says Tidmarsh. But the surge in popularity of ibuprofen was also likely to create higher risks of gastrointestinal toxicity--which is just what the Cox-2 drugs had set out to avoid. As it turned out, of course, that Cox-2 advantage was also tied to an increased risk of cardiovascular disease in Vioxx.

Horizon Therapeutics’ strategy was to try something very popular in drug development: Marry two existing therapies with clear safety profiles into a new compound--HZT-501--that could theoretically gain relatively swift approval and capture a significant market.

Says Tidmarsh: "We combined two approved agents: Famotidine, which lowers acid in the stomach, with ibuprofen, for pain relief, in one single pill."

Strategically, there was also an additional advantage in combining the drugs--increased compliance among patients who needed co-prescriptions.

“The seemingly simple concept of combining them into one pill leads to a dramatic improvement in compliance there,” says Tidmarsh.

Working with well known drugs, Horizon scooted right past the animal toxicity studies.

“We had very good Phase II proof of concept data with the two pills separately,” says the CEO. “So we did three Phase I studies to show no change in the way the body handles them.” The company also wanted to show the impact of more frequent dosing of famotidine on gastric pH (a measure of acidity). The next step was demonstrating bioequivalence of the combinatorial pill and then moved straight into Phase III.

His approach has won some big venture backing. In late July, Horizon announced a $30 million Series C. And that’s on top of the $21 million they had garnered earlier. Scale Venture Partners, Pequot Ventures, Sutter Hill Ventures and Essex Woodlands Health Ventures put up the funds. That money, adds Tidmarsh, is enough to get HZT-501 to an NDA and HZT-602 to Phase III.

By most standards, Horizon should already be beating the bushes for a collaborator. Common wisdom dictates that you would need to have a big outfit with a sizeable sales force if you wanted to claim market share for a drug that would have to be widely prescribed by primary care doctors. But Tidmarsh takes a contrarian view to the standard question he fields about a Big Pharma partner. He’s not ruling it out, but Tidmarsh sounds supremely confident when he says it’s not required.

“We do believe that this is the type of product that can be marketed by a younger, smaller, aggressive company.”

Actually, with five employees on the payroll, Horizon could charitably be described as tiny. But that hasn’t stopped Tidmarsh from thinking big.

“I literally was involved in Gilead when it was less than 5 people. My wife was the company’s third employee. I knew it when it was in a warehouse in San Carlos, and I know people there today. It grew from nothing to be a successful company, and I’m certain it can be done.”

But this is more than a story about a little biotech that thinks it could.
Tidmarsh is convinced that the revolution in new technology makes it
possible for smaller outfits to handle jobs once considered far beyond their reach.

Starting this fall, Tidmarsh also vows to start making the rounds, offering presentations and raising the company’s profile. When Phase III data arrives around mid-2008, Horizon may find itself in the industry’s spotlight.