Inspire Pharmaceuticals, Inc. announced today that the results from its first Phase 3 clinical trial with denufosol tetrasodium for cystic fibrosis (CF), TIGER-1, have been published in the peer-reviewed publication, American Journal of Respiratory and Critical Care Medicine (AJRCCM). Denufosol is an investigational, inhaled, novel ion channel regulator currently in Phase 3 clinical development for the treatment of CF.
The article entitled, "Denufosol Tetrasodium in Patients with Cystic Fibrosis and Normal to Mildly Impaired Lung Function" (Frank J. Accurso, M.D.; Richard B. Moss, M.D.; Robert W. Wilmott, M.D.; Ran D. Anbar, M.D.; Amy E. Schaberg, BSN, RN; Todd A. Durham, M.S.; Bonnie W. Ramsey, M.D.; and the TIGER-1 Investigator Study Group), appeared today online ahead of the print edition (ajrccm.atsjournals.org).
Frank J. Accurso, M.D., Head of Pulmonary Medicine, Director of Cystic Fibrosis Center at University of Colorado, Denver, and the lead principal investigator for TIGER-1 stated, "These exciting data suggest that denufosol may have promise in this mild patient population. The improvement in lung function observed during the placebo-controlled portion and the open-label extension of the TIGER-1 trial is notable given that the patient population studied had little to no pulmonary function impairment."
The TIGER-1 trial included a 24-week placebo-controlled portion, followed by a 24-week open-label safety extension. The placebo-controlled portion was a double-blind, randomized trial comparing 60 mg of denufosol to placebo, administered three times daily by jet nebulizer, in 352 patients with mild CF lung disease (baseline FEV1, or Forced Expiratory Volume in One Second, greater-than or equal to 75% of predicted normal). The TIGER-1 trial demonstrated statistical significance for its primary efficacy endpoint of change in FEV1 from baseline compared to placebo at the Week 24 Endpoint (45 mL treatment group difference, p=0.047). In the open-label extension, patients experienced a progressive improvement in FEV1. Detailed analyses from the trial are included in the AJRCCM publication.
Adrian Adams, President and CEO of Inspire stated, "We believe this publication represents valuable information for the CF medical community because it puts the data from TIGER-1, the first large Phase 3 trial of an ion channel regulator, into context in the current treatment landscape. The results from our second Phase 3 trial with denufosol, TIGER-2, are expected in the first quarter of 2011, which will provide further information."
About Denufosol Tetrasodium
Denufosol is a novel ion channel regulator that potentially corrects ion transport in patients independent of the class of CFTR defect. Denufosol is designed to enhance airway hydration and mucociliary clearance by increasing chloride secretion, inhibiting sodium absorption and increasing ciliary beat frequency. These integrated pharmacological actions and the potential to reach the small airways are key to maintaining lung function and potentially delaying the progression of lung disease.
About the Denufosol Tetrasodium Clinical Program
TIGER-1, the first Phase 3 trial with denufosol for the treatment of CF, included a 24-week placebo-controlled portion, followed by a 24-week open-label safety extension. The placebo-controlled portion was a double-blind, randomized trial comparing 60 mg of denufosol to placebo, administered three times daily by jet nebulizer, in 352 patients with mild cystic fibrosis lung disease (baseline FEV1 greater-than or equal to 75% of predicted normal).
Inspire expects top-line results from TIGER-2, its second Phase 3 clinical trial with denufosol, in the first quarter of 2011. Inspire is targeting a potential U.S. commercial launch for denufosol in the 2012 timeframe assuming the results of TIGER-2 are positive, that Inspire subsequently files a New Drug Application (NDA) for denufosol with the FDA in the second half of 2011 and that the FDA approves such NDA under a priority review timeline.
Inspire is conducting additional clinical trials in connection with its evolving denufosol franchise development plans and activities. Inspire is conducting DEFY (Denufosol Efficacy Over Four Years), a denufosol open-label clinical trial open to eligible patients that complete the year-long TIGER-2 trial. This three year trial will evaluate the potential disease-modifying impact of denufosol on rate of lung function decline. Inspire expects top-line results from REACH (Researching an Early Approach to Cystic Fibrosis Health), a small safety and tolerability clinical trial of denufosol in CF patients ages 2 - 4 years old, in the first quarter of 2011.
Source: Inspire Pharmaceuticals Inc.