Dive Brief:
- On Dec. 1, Aradigm announced mixed results in its two Phase 3 clinical trials evaluating safety and efficacy of its once-daily inhaled antibiotic, Pulmaquin, for treating people with a rare, severe chronic lung disorder that permanently enlarges parts of airways. Pulmaquin contains liposome encapsulated and unencapsulated ciprofloxacin, a widely used antibiotic.
- Primary endpoint for both late-stage studies was an increase in the median time until patients with non-cystic fibrosis bronchiectasis with lung Infections with Pseudomonas aeruginosa had their first pulmonary exacerbation. In one study, Pulmaquin, outperformed placebo in a statistically significant way; in the other study, it did not.
- Pulmaquin was safe and well tolerated in both 48-week treatment trials, the California-based specialty pharmaceutical firm said. Eight patients died in one trial, including three on placebo, and six died in the other trial (four on placebo), but Aradigm said no deaths were related to Pulmaquin or placebo.
Dive Insight:
The Food and Drug Administration granted fast track status to Aradigm's Pulmaquin in September 2014. Aradigm also has orphan drug designation for Pulmaquin for non-CF BE patients in the U.S., and the FDA has designated it as a Qualified Infectious Disease Product (QIDP) for treating patients with non-CF BE who also have P. aeruginosa lung infections: a particularly severe form of the disease.
According to Aradigm, both Pulmaquin studies (almost identical in design) showed a statistically significant reduction in P. aeruginosa density at Day 28 when the first treatment period ended. Also in each study, the antibiotic's effect persisted through all on-treatment periods. After 48 weeks of treatment ended, about nine in 10 patients in both studies opted to receive Pulmaquin in a 28-day open-label extension period.
Anne O'Donnell, professor of medicine and chief of the division of pulmonary, critical care and sleep medicine at Georgetown University Medical Center in Washington, D.C., and principal investigator for the North American segment of Aradigm's ORBIT-3 and ORBIT-4 trials, said that "after many setbacks in the development of inhaled antibiotics to treat [non-CF BE chronically infected with P. Aeruginosa] patients, we are finally seeing results with good safety and efficacy."
But investors disagreed and saw Aradigm's Pulmaquin trial results as disappointing. After Aradigm's announcement of the results, the company's shares fell nearly 54% in pre-market trading.