Aradigm Corporation announced the dosing of the first patient in a Phase III pivotal clinical trial that is testing Pulmaquin, an inhaled ciprofloxacin formula. ORBIT-4 is the second of two trials that the company is conducting in order to asses the efficiency of the drug for non-cystic fibrosis bronchiectasis.
“Pulmonary exacerbations in bronchiectasis patients are always a concern as they have an immediate adverse impact on their lives and further worsen their health long term. A treatment that would cause a reduction in the frequency and severity of pulmonary exacerbations in this disease would be highly desirable,” explained Dr. Shari Brazinsky, who dosed the first patient of ORBIT-4 at the Institute of HealthCare Assessment in San Diego.
The Phase III clinical exams are being performed in two worldwide, double-blind, placebo-controlled pivotal trials (ORBIT-3 and ORBIT-4), which are similar. However, one of the trials also contains a pharmacokinetics sub-study. Each of the trials involve about 255 patients and will feature 48-week double blind periods of six cycles of 28 days. The periods of Pulmaquin or placebo treatment are followed by 28 days of regular treatment, in which all patients are treated with Pulmaquin.
After an overall study period of approximately one year, researchers will assess the drug in terms of the time to first pulmonary exacerbation (primary endpoint), as well as other key secondary endpoints, which will include a reduction in the number of pulmonary exacerbations and improvements in quality of life measures. As a safety indicator, lung function will also being monitored.
In April, Aradigm dosed the first patient of the ORBIT-3 study and received a $5 million milestone payment from Grifols S.A. for the launch of their clinical trial. The project has been given the Qualified Infectious Disease Product (QIDP) designation from the U.S. Food and Drug Administration (FDA), which allows Pulmaquin and the company to be eligible for drug development incentives from the FDA for new antibiotics through their Generating Antibiotic Incentives Now Act (GAIN Act). It also means that Pulmaquin may be awarded an additional five-year exclusivity period if Pulmaquin wins approval from the FDA.
“Both of our Phase III trials are now globally proceeding. The QIDP Designation will provide us with the opportunity for expedited regulatory interactions to make a potential new treatment for non-cystic fibrosis bronchiectasis more expeditiously available to many patients with this severe condition,” said Juergen Froehlich, MD, Chief Medical Officer of Aradigm.
“Our experience from recent international meetings confirms that non-cystic fibrosis bronchiectasis is a serious, growing healthcare problem. The pulmonary infections with Pseudomonas aeruginosa are of particular concern in this patient population. We have established a network of multi-national international collaborations to help us in the execution of the ORBIT-3 and 4 trials,” said Igor Gonda, President and CEO of Aradigm.
Non-cystic fibrosis bronchiectasis causes an abnormal dilatation of the bronchi and bronchioles, but sometimes also chronic lung infections, and is a severe, chronic, and rare disease that represents an unmet medical need, since there is no drug approved to treat it. Vicious cycles of inflammation, frequent infections, and damage make it a deadly disease with a mortality that affects more than 110,000 people in the U.S.