N30 Pharmaceuticals Initiates Phase 1 Clinical Trial for Novel Cystic Fibrosis Therapy

Boulder, Colo., March 12, 2014 -- N30 Pharmaceuticals, Inc. (“N30 Pharma”) announced today that it has begun oral dosing of N91115 in a Phase 1 clinical trial. N91115, a novel inhibitor of S-nitrosoglutathione reductase (GSNOR), is being developed for the treatment of cystic fibrosis(CF).

In preclinical studies, N91115 has been shown to rescue or “correct” F508del-CFTR, the most commonly occurring mutation in CF.

It is the first oral GSNOR inhibitor to enter clinical development, and represents an important step forward in the development of more effective treatments for this serious disease.

The randomized, double-blind, placebo-controlled, Phase 1 study of orally administered N91115 will evaluate the safety, tolerability and pharmacokinetics of multiple ascending doses in healthy volunteers. The trial will assess several dose levels in up to 40 healthy adults.

N30 Pharma is also conducting a multicenter, dose escalation, safety study of N6022, its intravenous GSNOR inhibitor, in patients who are homozygous for F508del-CFTR. That study is currently enrolling the final cohort, at the highest dose, with completion expected in the second quarter of this year.

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 CF patients in the United States have two copies of the F508del-CFTR mutation, and an additional 39% have one copy of this 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 hydration in airways and other organs of the body. As a consequence of a mutation in the F508del-CFTR gene, patients produce a defective form of CFTR protein that is broken down and discarded in the cells of the body. N30 Pharma’s new drugs aim to correct 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 dedicated to making a difference in the lives of patients with cystic fibrosis and their families.

Source: N30 Pharmaceuticals, Inc.

Posted: March 2014

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