Ambitious Regeneron unveils mixed PhIII results for gout drug

Inspired by a string of development failures, Tarrytown, NY-based Regeneron Pharmaceuticals is endeavoring to find out if it can duplicate the ambitious R&D strategy that made Genentech a star in the biotech world. And if the developer can bring in approvals on all three of its drug programs now in late-stage development, analysts estimate the potential reward at $2 billion in annual revenue.

This morning, Regeneron Pharmaceuticals announced that its gout drug Arcalyst hit its goals in a late-stage study examining its ability to prevent new attacks of the disease but flunked a separate Phase III designed to demonstrate its effectiveness at reducing gout-related pain.

Patients enrolled in the gout attack trial demonstrated an 80 percent reduction in flare-ups compared to placebo when given a 160 mg dose; there was a 73 percent reduction for subjects taking an 80 mg dose. In the separate trial, Arcalyst failed to demonstrate an ability to reduce pain once the condition was triggered. And Regeneron says it will now wait for two more rounds of late-stage data on the drug before it pursues an FDA approval in mid-2011.

Regeneron's gout trials are being pursued at the same time the biotech company has several other Phase IIIs underway for a new cancer medication as well as an eye disease therapy--a broad range of disease targets and studies for a relatively small biotech company to undertake at one time. Regeneron has data from five more late-stage trials due over the next 12 months.

"We don't want the company to sink or swim on the back of any one thing, which is why we're trying to move an army of ideas forward," said CEO Len Schleifer, in an interview with Bloomberg. "We've modeled ourselves on the companies like Genentech that had a cadre of people and technologies."

- see Regeneron's release
- check out the story from Reuters
- read the report from Bloomberg
- here's Bloomberg's article on Regeneron's development strategy