US-based clinical stage drug discovery and development firm Celtaxsys has started enrolling patients for its first trial of its lead clinical stage drug candidate, CTX-4430, to treat patients with cystic fibrosis (CF) lung disease.
Safety and tolerability of the drug at several dose levels when directed orally once-daily for two weeks will be determined during the trial.
The trial will also include additional assessments such as pharmacokinetics and biomarkers, as well as effects on lung function.
Celtaxsys CMO Ed Philpot said safety and pharmacokinetics were confirmed in CTX-4430 from a recently completed first-in-human trial conducted by the company with healthy adult volunteers.
"This trial in cystic fibrosis patients is designed to help us optimise the dose level for our upcoming proof-of-concept study and to give us an early indication of potential effects on lung function in CF," Philpot said.
A total of 36 adult CF patients will be given four dose levels of oral once-daily CTX-4430 treatment in the study, which will be carried out at three sites in the UK under the supervision of Professor Stuart Elborn of Queens University, Belfast.
As part of the study, pulmonary function and exploratory lung clearance index (LCI) measurements will be taken after multiple dose administration of CTX-4430.
The primary outcome measures are assessment of the safety and tolerability of multiple oral doses of CTX-4430 when administered to CF patients once-daily.
The company expects to complete data collection for primary outcome measures by February 2014.
Celtaxsys CSO Eric Springman said by measuring biomarkers in blood and lung, the company can more precisely gauge the dose level of CTX-4430 required for therapeutic effect.
"The biomarkers also demonstrate effects of CTX-4430 on important mediators of CF lung disease and help set an expectation of therapeutic effect in the upcoming proof-of-concept study," Springman said.
CTX-4430 is a novel small molecule inhibitor of Leukotriene A4 Hydrolase (LTA4H), the key enzyme in production of the potent inflammatory mediator Leukotriene B4 (LTB4).