Savara Pharmaceuticals, an Austin based pharmaceutical company specializing in innovative drug development and delivery methods to treat life-threatening and rare respiratory diseases, recently announced that their leading product, AeroVanc, has showed promising results from a Phase 2 clinical trial. AeroVanc is the first of its kind inhaled antibiotic designed to treat an antibiotic-resistant lung infection in patients with cystic fibrosis (CF).
- Methicillin-resistant Staphylococcus aureus (MRSA): a type of bacteria that is highly resistant to most currently used antibiotics. Patients can be chronically infected as well as carriers of the organism
- Vancomycin: currently the antibiotic of choice to treat infections caused by MRSA, for it has an inhibitory effect on the organism. It is most often administered by intravenous (IV) methods
- Phase 2 Clinical Trial: Before a drug is given FDA approval it must go through many clinical tests to assess its safety and effectiveness. A Phase 1 trial tests the safety of a drug and once the safety has been established the next step is to conduct a Phase 2 trial which establishes how well the drug works, as well as to continue Phase I safety assessments
CF and MRSA: What You Need To Know:
CF is a chronically progressive life-threatening disease, caused by a genetic mutation. According to the CDC, in the US approximately 1000 new cases are diagnosed annually, with more than 75% of those patients under the age of two. An estimated 30,000 children and adults in the US and 70,000 worldwide have the disease. Patients diagnosed with CF have a lifespan of approximately 30 years, with many patients living into their forties.
The genetic mutation that causes CF affects the normal functioning of the lungs and digestive system, by creating abnormal amounts of very sticky mucus that covers the lungs, pancreas and other important organs of the respiratory and digestive systems. This sticky mucus attracts foreign pathogens, such as viruses and bacteria, making patients more susceptible to infections such as MRSA.
MRSA has become a clinically relevant pathogen, with prevalence close to 26% in the US CF patient population. Studies have shown that the detection of MRSA in the respiratory tract of CF patients leads to lower survival rates. AeroVanc, is an inhaled form of Vancomycin that is specifically designed to treat and prevent the ill-effects that chronic MRSA infection has on CF patients.
The AeroVanc Phase II clinical trial is a randomized, double-blind, placebo-controlled study of 80 CF patients with persistent MRSA lung infection. Its primary and secondary objectives are:
- to evaluate AeroVanc’s efficacy in reducing the quantity of MRSA colony forming units in sputum cultures.
- evaluation of the safety of repeat dosing with AeroVanc and efficacy of AeroVanc in improving lung function, reducing respiratory symptoms, and prolonging the time to pulmonary exacerbations and the need for other antibiotics
Patients included in the study received either 64 mg or 128 mg doses of AeroVanc, and were compared to a control group that received a placebo for any observable differences over a 28 day period.
In a press release about the trial, Rob Neville, Chief Executive Officer and founding member of Savara Pharmaceuticals, stated, “It is well known that people with CF who suffer from chronic Pseudomonas lung infection greatly benefit from inhaled antibiotics, such as TOBI. MRSA prevalence has increased dramatically over the past decade, but there is no inhaled antibiotic available for chronic treatment. Based on the observed clinical benefits in our study, we believe that AeroVanc can be equally successful for treatment of MRSA as the current inhaled antibiotics have been for Pseudomonas.”
The clinical benefits he is referring to included:
- A statistically significant reduction in MRSA density in sputum as compared to placebo.
- Improvements in pulmonary function and respiratory symptoms.
- Improvements in the time from infection to worsening of symptoms.
- A reduction in the need for other antibiotics.
This means that the study’s primary endpoint was met and also, that the secondary objectives showed promising results.
The importance of these results can be better understood from a previous statement made by Dr. Bonnie W. Ramsey, Director of the Center for Clinical and Translational Research at Seattle Children’s Research Institute, and one of the pioneers and lead investigators of TOBI, an inhaled form of tobramycin used to treat chronic Pseudomonas aeruginosa infection in CF: “Savara’s AeroVanc is a much needed investigational product, as none of the current inhaled antibiotics address the growing problem of MRSA infection. TOBI has become the cornerstone of treatment in CF, and AeroVanc has the potential to become analogous to TOBI for MRSA.”
Savara will be presenting the results of its Phase 2 clinical study of AeroVanc at the 2015 North American Cystic Fibrosis Conference in Phoenix, AZ October 8-10, 2015