Genoa Pharma Aims To Satisfy Big Pharma’s Hunger for IPF Drugs
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: 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 degree of effectiveness as an oral pill, and that it might be even more potent if delivered directly to the lungs. The founders noted that the InterMune drug needs to be given in relatively large doses three times a day, which causes nausea and vomiting that ends up prompting half of patients to quit taking it or reduce the dose. An aerosol form, they reasoned, could get more of the active ingredient directly to the lungs, while allowing less of it to get distributed throughout the bloodstream, where it would cause more side effects.
Even though InterMune has spent a lot of time and money on pirfenidone, Genoa believes it is free to make its own version of the drug without violating any patents. Pirfenidone, Kamdar says, is an old generic molecule, and InterMune’s intellectual property mainly protects its proprietary dosing forms, he says.
No other drug in development for IPF is thought to be given via an inhaler, Kamdar says. The Amira compound purchased by Bristol is an oral pill that goes after the new LPA-1 target, while the Biogen Idec/Stromedix drug is an injectable antibody designed to inhibit a pathway known as TGF-beta.
Surber, the technical founder of Genoa, spent almost a year working on 200 different chemical formulations to get the right properties for an inhalable form of pirfenidone, Kamdar says. Genoa has now filed its patents on its lead drug candidate, GP-101. The plan is to deliver it two or three times a day, through 10-minute sessions on a portable nebulizer, for patients with mild-to-moderate IPF, Kamdar says. Going after mild-to-moderate patients is important, Kamdar says, because the scarring of IPF tends to start on the outer areas of the lungs and work in. An inhalable drug, logically, is probably going to work better in milder patients who don’t yet have blocked airways.
“For most part, these IPF patients can move about and work and lead reasonably normal lives,” Kamdar says.
Genoa still has a ways to go before it can get within hailing distance of the clinical trial milestones reached by companies like Amira and Stromedix. But it does have a lead drug candidate, and its plan is to raise some more money to complete animal toxicology tests that it needs to run to get its first clinical trials underway, Kamdar says. If the fundraising comes together, it could be ready to start its first clinical trial in early 2013, he says.
The company may try to raise venture capital, but Kamdar says he’s considering other ways to advance the program in development, such as through a collaboration with a pharma or biotech company that could lead to a structured buyout of Genoa—assuming certain goals are met in clinical trials. That’s easier said than done, but like most entrepreneurs, Kamdar spoke to me with a lot of conviction about the need to go after this condition with gusto.
“The first-line treatment for this disease is a clinical trial. The second line, a lung transplant,” Kamdar says. “We are well aware this is an area with no treatments. There’s a very high unmet medical need.”