N30 Pharmaceuticals Announces First Patient Treated in Clinical Trial of N6022 in Cystic Fibrosis

BOULDER, Colo., March 13, 2013 /PRNewswire/ -- N30 Pharmaceuticals, Inc. ("N30 Pharma") a leader in the development of therapeutics that target S-nitrosoglutathione reductase (GSNOR), announced today that it has administered the first dose in its Phase 1b/2a clinical trial of N6022 in patients with cystic fibrosis (CF).  The study is a multicenter, double-blind, randomized, placebo-controlled evaluation of ascending doses of N6022 in patients who have two copies of the F508del-CFTR mutation, the most common and serious form of CF.

The clinical study announced today is being conducted in collaboration with the Cystic Fibrosis Foundation's Therapeutics Development Network (TDN), and the lead principal investigator for the study is Scott Donaldson, M.D., at The University of North Carolina, Chapel Hill.  The first patient was dosed this week at National Jewish Health in Denver.  The primary objective of the study is to evaluate the safety of repeat doses of N6022.  Secondary and exploratory objectives include the evaluation of N6022 serum levels, lung function, and other markers of disease activity in CF.

"We are indebted to the cystic fibrosis clinicians and nurses who have given us such valuable advice, to the members of the TDN who have been instrumental in getting this first trial started, and to the patients who will be volunteering for participation. With this first trial, we look forward to paving the way to a new class of therapy for cystic fibrosis," said Charles Scoggin, M.D., President and Chief Executive Officer of N30 Pharma.

About Cystic Fibrosis

Cystic fibrosis 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 N6022

N6022 is the first of a novel class of N30 Pharma's drugs aimed at conservation of S-nitrosoglutathione (GSNO) a key, endogenous, signaling molecule that has been shown to be reduced in cystic fibrosis patients.  N30 Pharma's drugs restore GSNO levels by inhibiting GSNOR, the enzyme in the body that breaks down GSNO.

N30 Pharma's GSNOR inhibitors have dual effects in cystic fibrosis.  First, in experimental models of cystic fibrosis, they rescue or "correct" the abnormal chloride channel function that causes the thick, tenacious secretions of the lung and other organs characteristic of cystic fibrosis.  Secondly, they are potently anti-inflammatory.  This is potentially important because inflammation, particularly of the lung, is the cause of major morbidity in CF patients.

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.  In addition to N6022, an injectable formulation, N30 Pharma is rapidly advancing an oral dosage form, N91115, through preclinical testing and IND submission.  Clinical trials of N91115 are targeted to begin 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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