Celtaxsys Begins EU Enrollment for CF Lung Function Preservation Trial of Acebilustat Anti-Inflammatory Therapy for Cystic Fibrosis

Print 03 May 2016

ATLANTA, GA-- May 03, 2016 - Celtaxsys, a clinical stage drug development company focused on advancing therapies for patients with rare and orphan inflammatory diseases, announced today the opening of enrollment in Europe of an international phase 2 clinical trial for acebilustat, its flagship treatment for people with cystic fibrosis (CF). The study, termed EMPIRE-CF (Evaluating Modulation of the Pulmonary Inflammatory Response in CF), will include approximately 30 sites across the EU, with sites in the United Kingdom, France, Italy, Germany, and Belgium. The trial will test the safety and efficacy of once-daily oral acebilustat over 48 weeks of treatment and could establish the first proof-of-concept for a novel anti-inflammatory therapy designed to prevent long-term loss of lung function in CF patients. Acebilustat is being tested against placebo on top of background therapy, including CFTR modulators (Kalydeco® and Orkambi®), in adult CF patients irrespective of their specific CF gene mutation. The US sites in the EMPIRE-CF study are already enrolling patients.

Acebilustat is a first-in-class neutrophil modulator that controls a key inflammatory signal overexpressed in CF and other inflammatory diseases. A previous clinical study in CF patients demonstrated acebilustat's ability to modulate the over-activated inflammatory response associated with CF lungs, decreasing neutrophils in the lung by 65% and reducing damaging neutrophil elastase levels in just two weeks of treatment. These results were accomplished without jeopardizing the patient's immune response to lung infection.

This Phase 2 CF trial is supported by a grant from Cystic Fibrosis Foundation Therapeutics (CFFT), the non-profit drug discovery and development affiliate of the CF Foundation. The trial was designed in conjunction with CFFT and the European CF Clinical Trial Network (CTN). The European portion of the EMPIRE-CF study will be under the supervision of Principal Investigator Professor Stuart Elborn, Dean of the School of Medicine, Dentistry and Biomedical Sciences, Queen's University, Belfast.

Professor Elborn said, "Anti-inflammatory therapies (AITs) represent the next wave of treatments for CF. In conjunction with the CFTR modulators and other emerging therapies, AITs like acebilustat have the potential to significantly preserve lung function and extend life expectancy for CF patients."

For more information regarding this trial, please visit https://clinicaltrials.gov/ct2/show/NCT02443688.

About Cystic Fibrosis:

Cystic fibrosis (CF) is a life-threatening disease that affects the lung and digestive system and impacts about 70,000 patients worldwide. CF is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene leading to abnormal CFTR protein functioning, the result of which causes the body to accumulate excessive levels of unusually thick mucus in the lungs. This excessive sticky mucus in the lungs is a site for infections that can require hospitalization. In the pancreas and GI tract CFTR protein dysfunction results in malabsorption of nutrients and sometimes intestinal blockage. Respiratory distress in CF, defined as acute difficulty in breathing, infection and/or hospitalization, is most commonly related to lung infection and inflammation induced lung tissue damage attributable to an overwhelming and dysfunctional response by dysregulated neutrophils. Treatment of this lung inflammation is, therefore, thought to be key to improving CF patient’s lung health and well-being. For more information on cystic fibrosis, go to www.cff.org.

About acebilustat:

Acebilustat is a once-daily oral drug currently undergoing clinical trials for inflammatory diseases. It is a novel small molecule inhibitor of Leukotriene A4 Hydrolase (LTAH4), the key enzyme in the production of the potent inflammatory mediator Leukotriene B4 (LTB4). LTA4H and LTB4 have been strongly implicated in the pathogenesis of cystic fibrosis and other orphan inflammatory diseases.

About Celtaxsys:

Celtaxsys is a privately-held drug discovery and development company focused on advancing medicines to treat patients suffering from orphan inflammatory diseases. The company is building a sustainable pipeline of first-in-class immuno-modulators, the most advanced of which is acebilustat. Our follow-on molecules enable new intellectual property and exhibit differentiated properties that enable optimization for particular indications. For more information, visit www.celtaxsys.com.

Return