Hyperion Hits Goal in Trial for Rare-Disease Drug, Prepping FDA Application

Hyperion Therapeutics raised $60 million in venture capital a year ago, and it should be in a position fairly soon to generate some return on that investment.

The South San Francisco-based biotech company said yesterday its experimental drug met its primary goal in a pivotal study including 46 patients with a rare phenomenon known as urea cycle disorders. The pivotal study, which the FDA signed off on in advance, should enable the company to file an application for U.S. approval of the drug before the end of next September, says Christine Nash, Hyperion’s vice president of marketing.

Treatments for rare diseases have come back into fashion this year, which my colleague Ryan described earlier this year. Even in the era of cost containment and healthcare reform, drugs for diseases affecting only a few thousand people, but which really do improve people’s lives, can still command huge payments from insurers. It’s also harder for generic competitors to swoop in to grab market share with rare-disease drugs, like they often do with conventional mass-market pharmaceuticals. Drug giant Sanofi-Aventis, knowing this all too well, has moved aggressively to acquire the standard-bearer for rare-disease companies—Cambridge, MA-based Genzyme—for $18.5 billion. Genzyme has said that price is too low.

For its part, Hyperion has developed a new drug for urea cycle disorders, which it estimates afflict about 2,000 people in the U.S., about half of whom have been diagnosed and treated. The market for this treatment is thought to be in the “$200 million-ish” range, Nash says.

Before diving into the weeds with the clinical trial data, I first wanted to know more about the company’s history, the medical problem it’s tackling, the standard of care today, and what Hyperion has that’s really any different. I figured it was worth checking in, since some familiar investors—including Bay City Capital, Sofinnova Ventures, Panorama Capital, Highland Capital Partners, New Enterprise Associates, and WRF Capital—have all thrown capital behind the idea.

Hyperion was founded in 2007 when it acquired the rights to glycerol phenybutyrate (HPN-100) from Ucyclyd Pharma, a subsidiary of Scottsdale, AZ-based Medicis Pharmaceutical (NYSE: MRX), a company better known for the anti-wrinkle injection Restylane than for the treatment of rare diseases.

The problem with urea cycle disorders, in concept anyway, is pretty to understand. These patients lack certain enzymes that clear waste … Next Page »

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