Patients with cystic fibrosis can expect to live until their late 30s or early 40s. It’s a big improvement over the life expectancy they had a decade or two ago. But now a biotech startup in Austin, TX, has put itself into position to find out, in the next year, if it has another way to improve the outlook for patients with this deadly genetic disease.
Savara Pharmaceuticals, a little company that has scraped together $19 million in angel investment since its founding in 2007, is entering a critical proving ground this year in clinical trials to test its idea for treating cystic fibrosis. It is enrolling 80 patients in a clinical trial that will give it a clear idea of whether it has come up with important new inhalable antibiotic, or whether it has to go back to the drawing board. The answer should arrive in less than a year.
“We’ve already shown safety. Once we show efficacy, we believe this will be a game-changer for us,” says Savara co-founder and CEO Rob Neville.
Cystic fibrosis, for those new to the story, is a genetic condition that affects about 30,000 people in the U.S. It’s known for leading to the buildup of thick, sticky mucus in the lungs that makes it hard to breathe. The mucus also becomes a haven for dangerous infections from bugs like pseudomonas aeruginosa and MRSA. Patients used to get intravenous antibiotics in the hospital once they had a severe infection, by which point it was often getting late in the game. Then pharmaceutical companies developed inhalable antibiotics that could be delivered more effectively to the lungs where the bugs reside, and which patients could take themselves on a regular basis at home.
What Savara noticed, about five years ago, was that those innovative products weren’t killing all the bugs that CF patients ought to be worried about. Novartis’ inhalable tobramycin (TOBI) and Gilead Sciences’ inhalable aztreonam (Cayston) won FDA approval for their ability to fight pseudomonas. But no one has come up with an inhalable version of vancomycin, the go-to antibiotic for MRSA. Savara’s big bet is that it can create such a drug, that it will be an important weapon against an infection increasingly viewed as troublesome, and that the rising incidence of MRSA is going to make it a widely prescribed product for CF.
Data from a 2010 study published in the Journal of the American Medical Association suggested that patients die sooner when they have chronic MRSA infections that are left untreated. That has provided some added urgency for the doctors and patients participating in the ongoing study of Savara’s inhalable vancomycin, which it calls AeroVanc.
The Savara story actually traces its roots back to 2007 in Kansas, with technology licensed from Cory Berkland’s lab at Kansas University. But Neville got involved the next year as CEO. He moved the company to Texas, which was where he had his home and a lot of business contacts. The original idea for Savara was to develop a platform technology for making inhalable drugs, which could be broadly applied to many products, but it ultimately didn’t prove commercially viable or scalable, and Savara is in the process of handing it back to KU, Neville says.
While Savara was looking at opportunities for inhalable drugs, it sized up a promising one in cystic fibrosis. The patients, physicians, and insurers were already well versed with the transition from IV to inhalable products, through the past experience with Novartis, Gilead, and others. Those drugs started with killing pseudomonas, which made sense, because it’s dangerous and affects about 60 percent of the cystic fibrosis population. The market appeared smaller for MRSA infections, because as recently as a few years ago, fewer than 20 percent of patients were thought to have this bug. Plus, converting vancomycin to an inhalable form presented technical challenges, and there wasn’t a consensus on how important MRSA was to treat, Neville says.
As Savara was working on its own inhalable form of vancomycin, a key paper in JAMA came out in 2010. It looked at patients in the Cystic Fibrosis Foundation’s database, and saw that those with MRSA infections had a significantly higher risk of death than those without. The study, although retrospective in nature, suggested that life expectancy for MRSA-infected patients was about 31, compared with 37 for those without, Neville says.
Suddenly, there was newfound urgency for
