Data to Be Presented at ERS Conference Highlights Potential for Denufosol to Benefit Cystic Fibrosis Patients on Minimal Pharmacotherapy
DURHAM, N.C.--(BUSINESS WIRE)--Sep 17, 2010 - Inspire Pharmaceuticals, Inc. (NASDAQ: ISPH) announced today that data will be presented on denufosol tetrasodium, an investigational therapy for cystic fibrosis (CF), during oral and poster presentations at the European Respiratory Society (ERS) Annual Congress September 18 - 22, 2010 in Barcelona, Spain. The data from Inspire's first Phase 3 trial with denufosol (TIGER-1) suggest that denufosol, a novel inhaled ion channel regulator, has the potential to provide benefit in lung function for those patients on minimal pharmacotherapy.
“Small airways obstruction in CF disease begins early in life and is progressive. Current therapies are directed at treating downstream complications of the disease leading to a large treatment burden. There is a need for novel, disease-modifying therapies that delay onset and progression of disease through early initiation and targeted delivery to the small airways. In the TIGER-1 trial, treatment with denufosol yielded improvement in lung function with limited systemic exposure in stable CF patients taking minimal concomitant medications. Denufosol has potential as an early disease state intervention that may delay CF lung disease progression,” stated Felix Ratjen, M.D., Ph.D., Professor of Pediatrics and Division Chief, Respiratory Medicine, University of Toronto, and lead principal investigator of TIGER-2.
The oral presentation, “Denufosol Targets Small Airways and Elicits Improvements in Mild Cystic Fibrosis Patients on Minimal Pharmacotherapy” (F. Accurso, T. Navratil, A. Schaberg, C. Evans, W. Tian, T. Durham, H. Tiddens, F. Ratjen), is being presented by Dr. Ratjen during the session, “Cystic Fibrosis: New Targets for Therapy and Impact of Viral Infections,” on Sunday, September 19 from 10:45 am - 12:45 pm local time (4:45 - 6:45 am ET). The presentation highlights a post-hoc analysis of subgroup data from patients in the TIGER-1 trial who were taking zero to two classes of concomitant medications for CF lung disease at baseline (n=71, ITT=352). These patients had a mean baseline FEV1 (Forced Expiratory Volume in One Second) of approximately 92%, which was comparable to the ITT population. The treatment effects for denufosol compared to placebo in the subgroup were 100 mL for the change from baseline in FEV1 (p=0.059), 6.4% for the relative change in percent predicted FEV1 (p=0.011) and 234 mL/sec for FEF25%-75% (Forced Expiratory Flow) (p=0.041), a measure of small airways function, at the Week 24 Endpoint.
A poster E-presentation entitled, “Denufosol: A Novel Ion Channel Regulator and Investigational Early Disease State Intervention Therapy for Cystic Fibrosis” (M.J. Stutts, M.S. Cowlen, S.F. Okada, R.C. Boucher), will also be presented during the ERS Annual Congress. This presentation highlights data from pre-clinical studies related to the mechanism of action of denufosol. The data suggest that denufosol increases chloride secretion, inhibits sodium absorption and normalizes airway surface liquid depth in primary epithelial cell cultures from CF patients. These actions support normal airway surface hydration and enhanced mucociliary clearance.
The presentations will be available on Inspire's website, www.inspirepharm.com, following the conference.
About the Denufosol Tetrasodium Clinical Program
Inspire is currently conducting TIGER-2, its second Phase 3 clinical trial with denufosol, and expects top-line results from this trial 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 TIGER-2. This three year trial will evaluate the potential disease-modifying impact of denufosol on rate of lung function decline. In August 2010, Inspire also initiated 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.
About Denufosol Tetrasodium
Denufosol is a first-in-class ion channel regulator targeted as an early disease state intervention treatment that potentially corrects the ion transport defects in patients regardless of CFTR genotype. 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.
Inspire is a biopharmaceutical company focused on researching, developing and commercializing prescription pharmaceutical products for ophthalmic and pulmonary diseases. Inspire's goal is to build and commercialize a sustainable portfolio of innovative new products based on its technical, scientific and commercial expertise. Inspire's clinical pipeline includes denufosol tetrasodium for cystic fibrosis in Phase 3 development and AZASITE® (azithromycin ophthalmic solution) 1% for blepharitis in Phase 2 development. Inspire receives revenues related to the promotion of AZASITE for bacterial conjunctivitis, the co-promotion of ELESTAT® (epinastine HCl ophthalmic solution) 0.05% for allergic conjunctivitis and royalties based on net sales of RESTASIS® (cyclosporine ophthalmic emulsion) 0.05% for dry eye. For more information, visit www.inspirepharm.com.
The forward-looking statements in this news release relating to management's expectations and beliefs are based on preliminary information and management assumptions. Specifically, no assurances can be made with respect to: denufosol's potential to provide benefit in lung function for those patients on minimal pharmacotherapy; denufosol's potential as an early disease state intervention that may delay CF lung disease progression; denufosol's ability to reach the small airway in the lung; denufosol's ability to elicit improvements in mild CF patients on minimal pharmacotherapy; denufosol's ability to increase chloride secretion, inhibit sodium absorption and normalize airway surface liquid depth in primary epithelial cell cultures from CF patients; the timing and outcome of TIGER-2 results in the first quarter of 2011, or otherwise; the ability to undertake, or the timing or outcome of, a U.S. commercial launch of denufosol in 2012, or otherwise; whether or not the results of TIGER-2 will be positive; that Inspire will file a New Drug Application (NDA) for denufosol with the FDA in the second half of 2011; that the FDA will review any denufosol NDA under a priority review timeline; that the FDA will approve denufosol, even if a priority review is undertaken; that denufosol could potentially correct the ion transport defects in patients regardless of CFTR genotype; and that Inspire could build and commercialize a sustainable portfolio of innovative new products based on its technical, scientific and commercial expertise. Such forward-looking statements are subject to a wide range of risks and uncertainties that could cause results to differ in material respects, including those relating to product development, revenue, expense and earnings expectations, the timing of a launch of a generic form of ELESTAT, intellectual property rights, competitive products, results and timing of clinical trials, success of marketing efforts, the need for additional research and testing, delays in manufacturing, funding, and the timing and content of decisions made by regulatory authorities, including the U.S. Food and Drug Administration. Further information regarding factors that could affect Inspire's results is included in Inspire's filings with the SEC. Inspire undertakes no obligation to publicly release the results of any revisions to these forward-looking statements that may be made to reflect events or circumstances after the date hereof.
Contact: Inspire Pharmaceuticals, Inc.
Jenny Kobin, 919-941-9777, Extension 219
VP, Investor Relations and Corporate Communications
Cara Amoroso, 919-941-9777, Extension 266
Assoc. Director, Corporate Communications
Posted: September 2010