Pharma, Startups

An encouraged Savara Pharmaceuticals preps for phase 2 study of inhaled antibiotic for CF patients

Friday’s U.S. Food and Drug Administration approval of the TOBI Podhaler was great news for […]

Friday’s U.S. Food and Drug Administration approval of the TOBI Podhaler was great news for drugmaker Novartis and for the cystic fibrosis community. It was also welcome news for Texas drug startup Savara Pharmaceuticals, whose CEO Rob Neville said he was “delighted that the FDA has approved a very necessary product.”

Novartis’s drug is an inhalable formulation of the antibiotic tobramycin that’s administered with a hand-held device. It was approved to treat Pseudomonas aeruginosa, the most common source of lung infection in patients with CF. Meanwhile, Neville and his team at Savara have spent the past several years developing a drug they hope will someday be used in the same way to treat another common bacterial infection that inhibits the lungs of patients with CF, methicillin-resistant Staphylococcus aureus.

In patients with the genetic disease cystic fibrosis, a characteristic thick, sticky mucus clogs the lungs and acts as a breeding ground for bacteria to grow and makes them prone to frequent lung infections that result in a loss of lung function. Respiratory failure, in fact, is the most common cause of death in these patients.

MRSA has been growing in prevalence among CF patients and it’s also been associated with a quicker rate of decline in lung function.

Savara’s drug, AeroVanc, is a dry powder formulation of the antibiotic vancomycin, which is usually administered intravenously. Although vancomycin is typically the preferred treatment for MRSA infections that occur in CF patients, it’s been linked to serious side effects such as heart and kidney damage.

Savara thinks it can skirt some of those negative effects and improve the drug’s efficacy by delivering it straight to the lungs. AeroVanc comes in a capsule-based device that’s designed for use as a once- or twice-daily, self-administered treatment for a MRSA infection.

It’s already demonstrated strong safety and pharmacokinetic profiles in phase 1 studies, and now Savara is hoping it will show the desired efficacy in an upcoming phase 2a study. Set to begin enrolling soon, Neville said the phase 2 study will involve 80 CF patients with chronic MRSA infections in the U.S. and Canada. With the placebo-controlled study, Savara hopes to show AeroVanc can reduce the bacterial load in the lungs and lead to other improvements in outcome, he said.

To get through the study, Savara recently closed the second tranche of a $16 million series B that started last year. That round saw huge buy-in from angel investor networks including Keiretsu Forum, the Tech Coast Angels, the North Texas Angel Network and the Texas Angel Network. Savara has also received funding and grants from the Texas Emerging Technology Fund, Qualifying Therapeutic Discovery Program, the National Institute for Allergies and Infectious Diseases and the National Institutes of Health, and went through the Austin Technology Incubator.

Advances in medicine have greatly extended the life expectancies of patients with CF, which was previously considered a fatal disease of childhood. While they may not be “game changers” in addressing the underlying cause of the disease, inhaled antibiotics stand to improve the lives of patients who have traditionally been bound to long and side effect-inducing nebulizer and IV treatments. In addition to Novartis’s recent approval, Forest Laboratories is also undergoing regulatory review of dry powder treatment for P. aeruginosa called Colobreathe.

Founded in 2007, Austin-based Savara was granted orphan drug status last year. Neville said data from the upcoming  study is expected around this time next year.

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