Aptalis Pharmaceutical Technologies Announces Results of Phase 3 Studies of Aeroquin (Levofloxacin Solution for Inhalation) Among Patients With Cystic Fibrosis and Chronic Lung Infection

Published: Jan 07, 2013

BRIDGEWATER, NJ--(Marketwire - January 07, 2013) - Aptalis Pharma, Inc., a global specialty pharmaceutical company focused on gastrointestinal diseases and cystic fibrosis (CF), announced the results of two global Phase 3 studies of Aeroquin™ (levofloxacin inhalation solution). The two studies were conducted in cystic fibrosis patients with stable chronic lung infection with Pseudomonas aeruginosa. They followed a successful placebo-controlled dose-finding study that demonstrated a dose-dependent pronounced reduction in the sputum density of Pseudomonas aeruginosa and improvement of lung function in the same patient population [Geller DE et al, Am J Respir Crit Care Med. 2011 Jun 1;183(11):1510-6].

In one Phase 3 study (Study 207) conducted in 330 patients, Aeroquin, delivered by a customized nebulizer using eFlow technology (PARI Pharma GmbH) at a dose of 240 mg twice daily, was compared to placebo in a double-blind, randomized single treatment-cycle of 28 days. In the second Phase 3 study (Study 209) conducted in 282 patients, the same dose of Aeroquin was compared to tobramycin for inhalation solution (TIS) in a randomized open-label study of three 28-day on/off treatment cycles. In both studies, patients had been heavily pretreated with inhaled antibiotics prior to enrollment.

In the TIS-comparator study, the primary endpoint -- non-inferiority of relative change from baseline in percent predicted Forced Expiratory Volume in 1 Second (FEV1) after the first treatment cycle of 28 days -- was met. This effect with Aeroquin on lung function was maintained over all three treatment cycles (i.e., through Day 168). For other clinically relevant secondary endpoints, such as quality-of-life measures and time to pulmonary exacerbation, the effect of Aeroquin was measurably similar or superior to TIS. An open-label extension study of Study 209 is ongoing.

In Study 207, the placebo-controlled single-cycle study, the differences between Aeroquin and placebo groups in the predefined primary endpoint of time to first pulmonary exacerbation were not statistically significant, however efficacy among certain secondary endpoints (lung function, reduction in Pseudomonas aeruginosa from sputum) in Aeroquin treated patients was demonstrated.

Tolerability and safety of Aeroquin was consistent across all three studies and showed similarity to TIS and placebo, respectively, with the exception of dysgeusia being observed more frequently in the Aeroquin group, attributable to the metallic taste of the active ingredient, levofloxacin.

Frank Verwiel, M.D., President and Chief Executive Officer of Aptalis Pharma, stated, "We are pleased with these results, which suggest that Aeroquin is potentially an important new treatment option for cystic fibrosis patients with chronic airways infection. Additional classes of inhaled antimicrobials are needed to ensure such options are available. We are looking forward to taking the next steps toward a regulatory filing for Aeroquin as part of our broader mission: to improve health and quality of care by providing specialty therapies for patients around the world."

About Cystic Fibrosis
Patients with CF suffer from chronic infections of the lower respiratory tract that can be caused by multiple bacteria, including Pseudomonas aeruginosa. Chronic pulmonary infection is associated with a decrease in lung function over time caused by inflammation arising from bacteria and their toxins. Periodic exacerbations in the lung result from bacterial infection (requiring antibacterial intervention), and these exacerbations are implicated as a major cause of progressive loss of lung function, morbidity and mortality in CF patients.

About Aeroquin
Aeroquin is a proprietary formulation of levofloxacin that has been optimized for aerosol delivery using an optimized Investigational eFlow Nebulizer System (PARI Pharma GmbH). Levofloxacin is a fluoroquinolone antibiotic that has been widely used in a variety of indications for over a decade and has established safety and efficacy when administered orally or intravenously against many bacterial pathogens, including Pseudomonas aeruginosa. Administration of Aeroquin with a high efficiency nebulizer to the lungs allows for the delivery of high concentrations of active drug directly to the site of infection in approximately five minutes, while minimizing systemic exposure.

About Aptalis
Aptalis Pharma is a privately held, leading specialty pharmaceutical company providing innovative, effective therapies for unmet medical needs including cystic fibrosis and gastrointestinal disorders. Aptalis has manufacturing and commercial operations in the United States, the European Union and Canada, and its products include ZENPEP®, CANASA®, CARAFATE®, PYLERA®, RECTIV®, VIOKACE™, ULTRESA™ LACTEOL®, DELURSAN®, PANZYTRAT®, and SALOFALK®. Aptalis also formulates and clinically develops enhanced pharmaceutical and biopharmaceutical products for itself and others using its proprietary technology platforms including bioavailability enhancement of poorly soluble drugs, custom release profiles, and taste-masking/orally disintegrating tablet (ODT) formulations. For more information, visit www.aptalispharma.com.

Forward-Looking Statements
This release contains forward-looking statements within the meaning of the U.S. federal securities laws, including statements regarding the expectations for the commercialization and marketing of AEROQUIN™. Forward-looking statements include those which express plan, anticipation, intent, contingency, goals, targets or future development and/or otherwise are not statements of historical fact. The words "expects," "potentially," "anticipates," "could," "calls for" and similar expressions also identify forward-looking statements. These statements are based upon Aptalis' current expectations and are subject to risks and uncertainties which could cause actual results and developments to differ materially from those expressed or implied in such statements. Factors that could affect actual results and developments include the results, consequences, effects or timing of any inquiry or investigation by any regulatory authority or any legal or administrative proceedings, and the successful preparation and implementation of an effective marketing plan. Investors should evaluate any statement in light of these important factors. Forward-looking statements contained in this press release are made as of this date, and, other than as required by applicable law, Aptalis undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Actual events could differ materially from those anticipated in the forward-looking statements.