AOP Orphan Pharmaceuticals AG, Vienna, Austria, and Lantibio Inc, Chaplel Hill, NC, have released positive final results from their placebo-controlled, double-blind Phase II clinical study of Moli1901, an experimental compound to treat cystic fibrosis (CF).
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 (12 years of age or older) and adults with CF.
Results of the study demonstrated that subjects ages 12 and older tolerated daily administration of Moli1901 over 28 days of daily dosing 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. 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 (Wilcoxon test, P=.02). Statistically significant improvements of the patients receiving Moli1901 were also observed in the patients’ subjective health perception as measured by the CF-validated Quality of Life Questionnaire.
“These early indications of efficacy of a Moli1901-based therapy are extremely encouraging to both cystic fibrosis patients and AOP Orphan,” said Rudolf Widmann, PhD, CEO of AOP Orphan. “We are pleased with the fast progress of our development program that has already entered the next stage with trials designed for confirmatory proof of efficacy and safety.”
Luis Molina, PhD, CEO of Lantibio Inc, added, “We are looking forward to the results of the next studies of AOP Orphan and regulatory steps toward registration in key territories, including the United States.”