N30 Pharmaceuticals, a Colorado-based clinical stage biopharmaceutical company, has treated the first patient in Phase Ib/IIa trial of N6022 in cystic fibrosis (CF) patients with two copies of the F508del-CFTR mutation.
The multicentre, double-blind study is designed to evaluate ascending doses of N6022 in patients with the most common and serious form of CF.
The study is being conducted in collaboration with the Cystic Fibrosis Foundation’s Therapeutics Development Network (TDN), and the first patient was dosed at National Jewish Health in Denver.
N30 Pharma president and chief executive officer Dr Charles Scoggin said; "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."
The primary objective of the randomised, placebo-controlled study is to assess the safety of repeat doses of N6022.
The evaluation of N6022 serum levels, lung function and other markers of disease activity in CF are the secondary and exploratory objectives of the study.
N6022 is the first candidate in N30 Pharma’s new class of drugs targeting conservation of endogenous, signalling molecule, S-nitrosoglutathione (GSNO), believed to decrease in CF patients.
The drugs are said to restore GSNO levels by inhibiting S-nitrosoglutathione reductase, the enzyme that breaks down GSNO.
N6022 has previously been studied for the treatment of asthma.