AOP Orphan reveals positive cystic fibrosis drug data

Moli1901 is being developed by AOP Orphan in Europe under a licensing and commercialization agreement with Lantibio. The study was designed to investigate safety and tolerability of aerosolized Moli1901 in adolescents of 12 years of age or older and adults with cystic fibrosis.

Results of the study demonstrated that subjects ages 12 and older tolerated daily administration of Moli1901 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 2.5mg Moli1901 per day demonstrated a statistically significant improvement of lung function as demonstrated by FEV1, a standard measure of lung capacity in cystic fibrosis patients. The median FEV1 change from day 1 to the final evaluation on day 56 amounted to -3% in the placebo group and 2% in the Moli1901 group.

Further statistically significant improvements of the patients receiving Moli1901 were observed in the patients' subjective health perception as measured by the CF-validated Quality of Life Questionnaire.