N30 announces cystic fibrosis trial

N30 Pharmaceuticals has administered the first dose in its Phase 1b/2a clinical trial of N6022 in patients with cystic fibrosis.

It is a multi-centre double-blind, placebo-controlled review of ascending doses of the drug in patients who have two copies of the F508del-CFTR mutation, which is the most common and serious form of the disease.

It is being conducted in collaboration with the Cystic Fibrosis Foundation's Therapeutics Development Network, with Scott Donaldson from the University of North Carolina standing as the lead principal investigator in the work.

Secondary and exploratory objectives include the evaluation of N6022 serum levels, lung function and their markers of disease activity.

Cystic fibrosis is the most common genetic disease among Caucasians and impacts around 30,000 people in the US and 70,000 across the world, with around 48 per cent of the patient population living in the US.

CF is characterised by a fault in the chloride channel of human cells, which is known as the cystic fibrosis transmembrane conductance regulator (CFTR).

As a result of the mutation in gene for CFTR, F508del-CFTR,with patients producing a form of CFTR that is broken down and discarded in the cells of the body.

N30 Pharma's new drug hope to rescue the damaged CFTR, reduce inflammation and restore proper hydration to crucial organs, especially the lungs.

As well as this, N30 Pharma's GSNOR inhibitors have multiple effects in cystic fibrosis, as they rescue or correct the abnormal chloride channel function that leads to thick secretions of the lung and other organs.

Furthermore, they are anti-inflammatory and inflammation of the lung can cause major morbidity in patients.

N30 Pharma is a based in Colorado and is the first company to develop small molecules targeting GSNOR.

It is also rapidly advancing an oral dosage form through preclinical testing and IND submission.

Posted by Ben Evans