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Inspire Pharmaceuticals Inc. is a biopharmaceutical company focused on researching, developing and commercializing prescription pharmaceutical products for ophthalmic and pulmonary diseases. The advanced compounds in the clinical pipeline are denufosol tetrasodium (DTS) for cystic fibrosis (CF) and Prolacria for dry eye, both of which are in Phase III development, and AzaSite for blepharitis, which is in Phase II development. The Company's portfolio of products includes AzaSite, Elestat, Restasis, Denufosol tetrasodium, Prolacria,
DTS is very promising drug candidate based on strong science studies. This drug (if successful) could be a breakthrough in CF treatment.

Cystic Fibrosis Disease Information

Cystic fibrosis (CF) is a life-threatening disease involving a genetic mutation that disrupts the cystic fibrosis transmembrane regulator (CFTR) protein, an ion channel. In CF patients, a defect in this ion channel leads to poorly hydrated lungs and severely impaired mucociliary clearance. Chronic secondary infections invariably occur, resulting in progressive lung dysfunction and deterioration. Respiratory infections and complications account for more than 90% of the mortality associated with this disease.
According to the U.S. Cystic Fibrosis Foundation's Patient Registry, the median life expectancy for patients is approximately 38 years. There are approximately 30,000 diagnosed CF patients in the U.S., included in an estimated 70,000 total in the eight major international prescription pharmaceutical markets.

Current CF Treatment Options

The current therapeutic approaches to address cystic fibrosis mainly treat the complications of the disease and are aimed at reducing respiratory infections and breaking up thickened mucous secretions that cause airflow obstruction and harbor bacteria. For example, TOBI® and Cayston® are inhaled antibiotics that treat the infection, and Pulmozyme® is an inhaled protein that breaks up excessive DNA in mucus thereby reducing the thickness and tackiness of the respiratory secretions. While both products are approved for the treatment of cystic fibrosis, neither product is designed to address the underlying ion-transport defect, which results in dehydrated mucus and severely impaired mucociliary clearance.

Drugs of the Future
Drugs Fut 2008, 33(8): 668
ISSN 0377-8282
Copyright 2008 Prous Science
CCC: 0377-8282
DOI: 10.1358/dof.2008.033.08.1237753 Denufosol tetrasodium. P2Y2 agonist, treatment of cystic fibrosis
Cole, P., Serradell, N., Rosa, E., Bolos, J., Castaner, R.

Denufosol tetrasodium is a first-in-class receptor-mediated chloride channel activator that addresses the underlying ion transport defect in the lungs of patients with cystic fibrosis (CF).

About Denufosol Tetrasodium
Denufosol tetrasodium is a first-in-class receptor-mediated chloride channel activator that addresses the underlying ion transport defect in the lungs of patients with cystic fibrosis (CF). Denufosol is designed to enhance airway hydration and mucociliary clearance through receptor-mediated mechanisms that increase chloride secretion, inhibit sodium absorption and increase ciliary beat frequency. These integrated pharmacological actions are important to restoring airway clearance, maintaining lung function, and potentially delaying the progression of CF lung disease. Denufosol is targeted as an early intervention therapy for CF lung disease. This product candidate has been granted orphan drug and fast-track review status by the U.S. Food and Drug Administration (FDA) and orphan drug status by the European Medicines Agency (EMEA).

Study of Denufosol Tetrasodium Inhalation Solution in Patients With Cystic Fibrosis (CF) Lung Disease

Primary Outcome Measures:
Change in lung function [ Time Frame: 48 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
• Pulmonary Exacerbation, Antibiotic Use, Incidence of Hospitalization/ER visits, Health resource utilization, Quality of Life [ Time Frame: 48 weeks ] [ Designated as safety issue: Yes ]

Estimated Study Completion Date: December 2010 Estimated Primary Completion Date: December 2010 (Final data collection date for primary outcome measure)

January 03, 2011 by BiotechInvest

Sad news for ISPH today.

Inspire Pharmaceuticals (ISPH 3.49, -4.92, -58.51%) shares were crushed in premarket trading Monday after the biotech group reported that a Phase III clinical trial for its drug denufosol failed to show it was effective in treating cystic fibrosis. Inspire shares were down nearly 60% at $3.47. Inspire said it plans to analyze the data to determine if it will proceed with further develop the product. The company intends to issue a full corporate update in mid-February.

Well, it's nothing usual for biotech investment. The promising in phase II study drug failed to meet primary and key secondary goals in a late-stage trial.


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