One of the hot areas of drug development these days can be filed under the header of “deadly lung scarring condition we don’t understand very well.” But the scientific knowledge of idiopathic pulmonary fibrosis is improving fast, and a new San Diego startup called Genoa Pharmaceuticals is betting it can satisfy Big Pharma’s hunger for new drugs against this disease.
The new company, pronounced JEN-oh-uh, has raised its first $1 million from friends and family to develop an inhalable version of a well-known compound called pirfenidone. Genoa is led by chief scientist Mark Surber, an aerosol drug expert formerly with Mpex Pharmaceuticals and Aires Pharmaceuticals, and chief business officer Mike Kamdar, formerly an executive vice president with San Diego-based VentiRx Pharmaceuticals.
The opportunity for this new drug has become clearer over the past nine months. The disease, probably best known as the scourge that makes it hard for many 9/11 first responders to breathe, is estimated to affect more than 200,000 people in the U.S. and Europe. It kills an estimated 40,000 people in the U.S. each year, roughly on par with breast cancer. Physicians have often struggled to diagnose it, and scientists are looking to understand the inflammation and collagen buildup at its core, but knowledge of the condition is improving as new biomarkers have become linked to the disease.
There are still no approved drugs in the U.S. for this condition, but Brisbane, CA-based InterMune (NASDAQ: [[ticker:ITMN]]) won regulatory approval last year for an oral version of pirfenidone (Esbriet) in Europe. While InterMune hasn’t been able to win U.S. approval, it helped blaze a path in clinical trials that has intrigued other developers. In the last year, Bristol-Myers Squibb paid $325 million in up-front cash to acquire one IPF drug developer, Amira Pharmaceuticals. Then, Biogen Idec spent $75 million up front to acquire Cambridge, MA-based Stromedix.
Partly because of those deals, and partly because of improved biological understanding, there has been a surge in interest among companies looking to treat IPF, says Michael Gilman, the co-founder and former CEO of Stromedix. “Thanks largely to InterMune, we now understand the regulatory hurdles,” Gilman says. “All the failed—but well-run—trials of the last few years have provided valuable data on the natural history of the disease based on the placebo groups, which provides confidence around trial design—duration, number of patients, statistical power, etc.”
Kamdar says he got a strong sense of the enthusiasm among doctors at a medical meeting in December. “You can see there’s a vast unmet need,” he says. “For me, this is about the chance to work on something that could have a meaningful impact on a reasonably large number of people. It got me excited to roll up my sleeves.”
Genoa’s bet is essentially that pirfenidone has already shown some reasonable
