Acebilustat

Description: Acebilustat, also known as ZK322, is a novel, oral, potent and selective leukotriene A4 hydrolase inhibitor that is a promising new once-daily oral antiinflammatory drug in development for treatment of cystic fibrosis (CF) and other diseases. In a Phase I study, seventeen patients with mild to moderate cystic fibrosis were enrolled and randomized into groups receiving placebo or doses of 50 mg or 100 mg acebilustat administered orally, once daily for 15 days. Sputum neutrophil counts were reduced by 65% over baseline values in patients treated with 100 mg acebilustat. A modestly significant 58% reduction vs. placebo in sputum elastase was observed with acebilustat treatment. Favorable trends were observed for reduction of serum C-reactive protein and sputum neutrophil DNA in acebilustat-treated patients. No changes in pulmonary function were observed. Acebilustat was safe and well tolerated. The results of this study support further clinical development of acebilustat for treatment of cystic fibrosis.

References: Clin Transl Sci. 2017 Jan; 10(1):28-34; Clin Transl Sci. 2017 Jan; 10(1):20-27.