AOP ORPHAN, LANTIBIO ANNOUNCE RESULTS FROM CYSTIC FIBROSIS STUDY

AOP Orphan Pharmaceuticals and Lantibio have announced positive final results from their placebo-controlled, double-blind, Phase II clinical study of Moli1901, an experimental compound to treat cystic fibrosis. Moli1901 is being developed by AOP Orphan in Europe under a licensing and commercialization agreement with Lantibio. The study, performed at multiple centers in Europe, was designed to investigate safety and tolerability of aerosolized Moli1901 in adolescents and adults with cystic fibrosis.

Safety and tolerability of the drug were assessed by spirometric evaluation of pulmonary function, vital signs, pulse oximetry, physical examinations, clinical laboratory tests, electrocardiograms and by monitoring adverse events. Efficacy was assessed by pre- and post-treatment pulmonary function evaluation measuring forced expiratory volume in one second (FEV1), forced vital capacity (FVC) and forced expiratory flow 25 to 75 percent of FVC. Exercise tolerance and quality of life were also assessed.

Results of the study demonstrated that subjects over 12 tolerated daily administration of Moli1901 over 28 days by the inhalation route, and analysis of the data did not reveal unexpected safety concerns.

Furthermore, no traces of Moli1901 were detected in the plasma of subjects, indicating that the drug has limited or no systemic absorption. Notably, although the study was not powered to demonstrate efficacy, patients who received 2.5 mg Moli1901 per day demonstrated a statistically significant improvement of lung function as demonstrated by FEV1, according to the company. The median FEV1 change from day one to the final evaluation on day 56 amounted to negative 3 percent in the placebo group and 2 percent in the Moli1901 group.