One-on-One with Raptor Pharmaceutical CEO Chris Starr

For every biotech company with more than a dozen years under its belt and $1 billion or more burned — and still no drug on the market — there’s a Raptor Pharmaceutical Corp.
OK, maybe not. There are many more companies that have slaved for years toward bringing a drug to market than there are speedy companies like Novato-based Raptor (NASDAQ: RPTP).
The 18-employee was founded a mere six years ago by a couple of BioMarin Pharmaceutical research veterans, Chris Starr and Todd Zankel. It has netted $111.5 million from fundraising and it could have its first drug approved by the end of the year. It filed a new drug application, or NDA, last month with the Food and Drug Administration.
Raptor’s RP-103, however, is no blockbuster. It is a delayed-release version of cysteamine, a drug approved in the 1990s to extend the lives of the fewer than 1,000 patients worldwide with the potentially fatal orphan disease cystinosis. Many of those patients are infants who, if they don’t take the drug, lose kidney function, require a kidney transplant and often die by the time they are teenagers.
I spoke with Starr about the drug, cystinosis and the company’s steady march toward a drug approval.

Industry view: Changes to business model come slowly to biotech

Entering the fifth year of a funding crisis, biotech’s business model is changing.
Slowly.
New sources of early-stage cash are stepping into the void left by fleeing venture capitalists. But whether those relatively small investments — or corporate venture capital, or grants from disease-specific foundations — are enough to build sustainable companies is an open question.
(San Francisco Business Times subscription required.)

Raptor drug's big selling point — no stink, better compliance

On the verge of submitting a rare-disease drug for approval, Raptor Pharmaceuticals Corp. is counting on one major selling point: Its drug doesn’t stink.
The 14-person Novato company will file a new drug application early in first-quarter 2012 for delayed-release cysteamine, a treatment for a deadly inherited disease known as cystinosis. The drug uses the same active ingredient as Cystagon, approved in 1994 by the Food and Drug Administration.
The knock against Cystagon has been a putrid rotten egg smell and its every-six-hours dosing. That’s no small thing in a disease that can kill children by the age of 9 — parents must wake a child during the night to give the powder-filled capsule.

Raptor rare disease trial has good results, but stock drops

Shares of Raptor Pharmaceutical Corp.plummeted more than 28 percent Monday despite the Novato company reporting that its treatment for a rare metabolic disorder was as effective in a late-stage clinical trial as a currently marketed treatment.
Raptor (NASDAQ: RPTP) is betting that its drug, called Cysteamine or RP-103, is at least as effective as Mylan Laboratories Inc.’s (NASDAQ: MYN) Cystagon for the rare and potentially fatal genetic disease nephropathic cystinosis.