Press Release: AOP Orphan Pharmaceuticals, Lantibio Announce Positive Phase II Study
AOP Orphan Pharmaceuticals and Lantibio Announce Positive Phase II Study with Novel Cystic Fibrosis Therapy VIENNA, Austria and CHAPEL HILL, N.C., US, 07.03.2007 - AOP Orphan Pharmaceuticals AG and Lantibio, Inc. today announced positive final results from their placebo controlled, double-blinded 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. The study was first populated with 9 subjects aged 16 years and older (Phase I), followed with 9 subjects aged 12-16 years (Phase II). Safety and tolerability were assessed by spirometric evaluation of pulmonary function, vital signs, pulse oximetry, physical examinations, clinical laboratory tests, electrocardiogram (ECG), and by monitoring adverse events. Efficacy was assessed by pre-treatment and post-treatment pulmonary function evaluation measuring forced expiratory volume in one second (FEV1), forced vital capacity (FVC), and forced expiratory flow 25-75% of FVC. Exercise tolerance and quality of life were also assessed. 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, a standard measure of lung capacity in CF 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 (Wilcoxon test, p=0.0217). 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. "These early indications of efficacy of a Moli1901-based therapy are extremely encouraging to both cystic fibrosis patients and AOP Orphan," said Dr. Rudolf Widmann, CEO of AOP Orphan Pharmaceuticals AG. "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." "We are pleased with the outcome of this study. 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," said Dr. Luis Molina, CEO of Lantibio, Inc. About Moli1901 In CF, absence or dysfunction of the chloride channel CFTR causes reduced chloride ion and water transport in respiratory epithelial cells. This results in an altered water content in the airway surface liquid (ASL), leading to thick mucus secretion, inflammation and infection in the CF lung. Moli1901 is a stable 19-residue polycyclic peptide produced by fermentation of Streptomyces cinnamoneum. In previous published studies, Moli1901 has been shown to increase chloride transport and fluid secretions when applied to airway epithelium in-vitro, and increases chloride permeability in nasal epithelium of healthy individuals and subjects with CF. In addition, animal experiments have shown that Moli1901 increased the volume of ASL, was slowly eliminated from the lung, and did not accumulate substantially in other tissues. Results of a previous clinical study performed by AOP Orphan in Europe presented in oral and poster format at the 20th Annual North American Cystic Fibrosis Conference, Denver, Colorado, November 2-5, 2006, indicated that dosing of Moli1901 for 5 consecutive days by the inhalation route improved lung function in a dose-dependent manner. About AOP Orphan AOP Orphan Pharmaceuticals AG is a privately held corporation founded in Vienna in 1997. The company is dedicated to developing, marketing and distributing medicines in niche indications on a Pan-European level with special focus on Central and Eastern Europe, Middle East and Southern Asia. About Lantibio Lantibio, Inc. is a development pharmaceutical company focused on discovery, development, and marketing of products for the treatment of ocular disease and serious respiratory conditions. Lantibio's lead product, Moli1901 holds an IND in CF in the US as well as Orphan Drug status in that indication. This press release might contain forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or unknown risks or uncertainties materialize, actual results could vary materially from AOP Orphan's and/or Lantibio's expectations and projections. Risks and uncertainties include success of clinical trials for AOP Orphan's and/or Lantibio's products, intellectual property risks, need for additional financing or capital, ability to develop and successfully commercialize our products, ability to enter into licensing agreements, and the ability of the competition to render AOP Orphan's and/or Lantibio's product candidates or technologies obsolete or noncompetitive. AOP Orphan and Lantibio assume no obligation to update any forward-looking statements as a result of new information or future events or developments, except as required by law.