Celtaxsys, a clinical stage drug development company focused on advancing care for patients suffering from orphan inflammatory diseases, announced today the commencement of the US arm of an international phase 2 clinical trial for its flagship compound, acebilustat, in patients with cystic fibrosis (CF). The study will be conducted at approximately 60 sites in the United States and European Union, with the EU sites anticipated to begin enrollment early 2016. This landmark clinical trial testing once-daily oral acebilustat treatment over 48 weeks could be the first to establish proof-of-concept for an anti-inflammatory treatment specifically designed to prevent long-term loss of lung function in CF patients. The study will test once daily oral doses of 50 mg and 100 mg acebilustat against placebo on top of background therapy in adult CF patients.
Acebilustat is a first-in-class neutrophil modulator that controls a key inflammatory signal overexpressed in CF and other orphan inflammatory diseases. Acebilustat has been granted orphan drug status for the treatment of CF in the US and the EU. Results from initial clinical studies in CF patients demonstrated acebilustat's ability to moderate the over-activated inflammatory response in CF, decreasing neutrophils in the lung by 65% and decreasing damaging neutrophil elastase in just two weeks of treatment. Acebilustat did this without jeopardizing the patient's immune response to infection. Importantly, acebilustat treatment is applicable to all CF patients irrespective of their gene mutation and can be used on top of background therapy, including Kalydeco and Orkambi.
The Phase 2 CF trial was designed in conjunction with and supported by a grant from Cystic Fibrosis Foundation Therapies, Inc. (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. It will include approximately 30 sites in the US under the oversight of Principal Investigator Dr. Stephen Rowe MD MSPH, Professor at the University of Alabama Birmingham School of Medicine, Director of the Cystic Fibrosis Transition Clinic, and Director of the CFFT Development Network Center for CFTR Detection. Said Dr. Rowe, "CF patients are in desperate need of efficacious and safe anti-inflammatory therapies to control progressive lung disease. Evaluation of the long term effects of acebilustat is a very important step in that direction, given the favorable Phase 1b results in CF patients."