Clinical-stage pharmaceutical development firm Celtaxsys has received approval from the US Food and Drug Administration (FDA) to start a Phase II clinical trial of its drug candidate, acebilustat (CTX-4430), in adult cystic fibrosis (CF) patients in the US.
Acebilustat is a once-daily oral drug candidate and a novel small molecule inhibitor of Leukotriene A4 Hydrolase (LTA4H), the key enzyme in the production of the potent inflammatory mediator Leukotriene B4 (LTB4). It is being tested to treat inflammatory diseases.
Designed in consultation with Cystic Fibrosis Foundation Therapeutics (CFFT), the trial will test the lung function preservation effects of once-daily oral acebilustat treatment over 48 weeks.
The trial will be carried out in partnership with the CFFT and is partly funded by the $5m research award granted by the CFFT to Celtaxsys.
Celtaxsys CEO Greg Duncan said: "We believe this day marks another watershed moment in the development of new therapeutics for CF patients.
"While the advent of important new therapies, including CFTR modulators, has been a true game-changer for many CF patients, lung inflammation continues to be a primary cause of CF patient morbidity and mortality."
Celtaxsys intends to begin the Phase II CF trial (NCT02443688) by October this year.
The trial’s US principal investigator Dr Steven Rowe said: "Anti-inflammatory treatments are clearly needed for CF. Acebilustat is a potentially promising new treatment that, if demonstrated to be efficacious and safe, can become an important addition to the armament of current CF treatments by reducing lung inflammation and slowing decline of lung function."
The company is also planning for regulatory submissions of the acebilustat trial to the health authorities in Europe.
Image: Clubbing of the fingers is a classic feature of cystic fibrosis. Photo: courtesy of Jerry Nick, M.D.