N30 Pharma Announces Presentation of Preclinical Data at the Basic Science Meeting of the European Cystic Fibrosis Society

BOULDER, Colo., April 2, 2013 /PRNewswire/ -- N30 Pharmaceuticals, Inc., a clinical stage, biopharmaceutical company focused on the development of therapies for cystic fibrosis (CF), announced today the presentation of key preclinical data for its novel inhibitors of S-nitrosoglutathione reductase (GSNORi). The data were presented at the 10th Basic Science Meeting of the European Cystic Fibrosis Society held in Malaga, Spain (20^th – 24^th March, 2013), and are available on the N30 Pharma website at www.n30pharma.com

The first of the presentations described data from various cell-based models relevant to the function of the mutant CFTR protein in CF.  These studies showed that N30 Pharma's GSNORi improved F508del-CFTR protein expression in transfected cells that overexpress F508del-CFTR as well as improving chloride channel activity in human airway cells derived from CF patients carrying two copies of the F508del-CFTR mutation. These findings are important because they show that GSNORi increase both the expression level and channel function of the mutant CFTR protein.

The second of the presentations described data showing the effects of GSNORi in rodent models of i) CF, ii) chronic obstructive pulmonary disease (COPD) and asthma, and  iii) airway tone. Collectively, these studies showed that N30 Pharma's GSNORi corrected CFTR-mediated chloride channel activity in F508del-CFTR mice, attenuated lung inflammation, and produced a relaxant effect on pre-contracted airway smooth muscle in isolated rat trachea.  These findings are important because they demonstrate that GSNORi have the potential to affect CF lung disease by improving a number of factors including: chloride channel function, inflammation, and lung perfusion.

Targeting GSNOR in CF

N30 Pharma has developed a novel portfolio of GSNOR inhibitor drugs that conserve S-nitrosoglutathione (GSNO), an endogenous, signaling molecule that has been shown to modulate the function of key proteins involved in the pathophysiology of CF through S-nitrosation.  N30 Pharma's drugs restore GSNO levels by inhibiting GSNOR, the enzyme in the body that breaks down GSNO.  Levels of S-nitrosothiols, including GSNO, have been reported to be reduced in the lungs of CF patients.  Furthermore, GSNOR activity and expression have also been reported to be increased in airway cells from patients with CF compared with normal cells.

N30 Pharma's GSNOR inhibitors have a number of potential effects in CF.  In experimental models of CF, they rescue or "correct" the abnormal chloride channel function that causes the thick, tenacious secretions of the lung and other organs characteristic of CF.  They also have anti-inflammatory and bronchodilatory effects that are relevant to the pathogenesis of CF lung disease.

About Cystic Fibrosis

CF is the most common, life-threatening, genetic disease of Caucasians, affecting approximately 30,000 people in the United States and 70,000 people worldwide.  Approximately 48% of the total CF patient population in the United States have two copies of the F508del mutation, and an additional 39% of the total CF patient population have one copy of the F508del mutation.

CF is characterized by a defect in the chloride channel of human cells.  This channel, known as the "cystic fibrosis transmembrane conductance regulator", or CFTR, is critical for adequate water coating, or "hydration" in airways and other organs of the body. As a consequence of a mutation in the gene for CFTR, F508del-CFTR, patients produce a form of CFTR that is broken down and discarded in the cells of the body.  N30 Pharma's new drugs aim to rescue the damaged CFTR, decrease inflammation, and restore proper hydration to critical organs, particularly the lung. 

About N30 Pharmaceuticals, Inc.

N30 Pharma is a privately held, clinical stage, biopharmaceutical company headquartered in Boulder, Colorado. It is the first company to discover and develop small molecules targeting GSNOR.  N30 Pharma is conducting a clinical trial of its lead compound, N6022 (an injectable formulation) in patients with two copies of the F508del mutation.  In parallel, the company is rapidly advancing an oral dosage form, N91115, through preclinical testing and plans to begin clinical trials of N91115 in the first half of 2014.

N30 Pharma is dedicated to making a difference in the lives of patients with cystic fibrosis and their families. 

SOURCE N30 Pharmaceuticals, Inc.

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