Celtaxsys receives FDA clearance to begin acebilustat Phase 2 trial in adult CF patients in the US

Celtaxsys, Inc., a clinical stage pharmaceutical development company focusing on anti-inflammatory therapeutics, including those with rare and orphan inflammatory disease indications, announced today that it has gained clearance from the U.S. Food and Drug Administration (FDA) to begin a Phase 2 clinical trial of its flagship drug candidate, acebilustat (CTX-4430), in adult CF patients in the US. The regulatory submissions to the health authorities in Europe will be filed soon after. This groundbreaking clinical study, testing the lung function preservation effects of once-daily oral acebilustat treatment over 48 weeks, was designed in consultation with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT). The trial will be conducted in partnership with the CFFT and is partially funded by the $5M research award granted by the CFFT to Celtaxsys earlier this year.

"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," said Greg Duncan, Celtaxsys CEO. "Once-daily oral acebilustat treatment can potentially address the underlying inflammation that results in irreversible lung function decline and occurs in all CF patients, independent of CFTR genotype."

Added Sanjeev Ahuja, MD and Chief Medical Officer at Celtaxsys, "The interplay between infection and inflammation is the main driver for lung function decline in CF. Finding a proper balance in immune modulation is key to treating the inflammation and resultant decline in lung function without increasing risk of infection. In a previous clinical trial in CF patients, acebilustat reduced important biomarkers of lung and systemic inflammation without apparent increased susceptibility to infection. Anti-inflammatory agents, such as acebilustat, could work by slowing the progression of disease over time, but do require a longer duration of assessment to best judge their long term benefits. Importantly, acebilustat should be complementary to existing CF therapies, including the new CFTR modulators, and may actually help boost their efficacy by reducing the pool of neutrophil elastase enzyme in the lungs that can degrade functional CFTR in these patients."

Steven M. Rowe, MD, MSPH, Professor in the Division of Pulmonary, Allergy and Critical Care Medicine and Director of the Center for CFTR Detection at the University of Alabama at Birmingham School of Medicine, and the US Principal Investigator for this trial, suggested that "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."

This Phase 2 CF trial (NCT02443688) is slated to begin in the US by October this year.