Celtaxsys, Inc. Gains FDA Clearance for Landmark Phase 2 Trial of Anti-Inflammatory Treatment for Cystic Fibrosis (CF)

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Forty-eight week trial to target lung inflammation, still the main cause of morbidity and mortality associated with CF 
ATLANTA, July 6, 2015 /PRNewswire/ — 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. For more information regarding this clinical 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 patients’ lung health and well-being. For more information on cystic fibrosis, go to www.cff.org.  
About acebilustat (formerly CTX-4430): Acebilustat is a once-daily oral drug candidate being tested for the treatment of inflammatory diseases. It is a novel small molecule inhibitor of Leukotriene A4 Hydrolase (LTA4H), 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 many diseases involving inflammation, including cystic fibrosis.  
About Celtaxsys: Celtaxsys, Inc. is a privately-held pharmaceutical discovery and development company focused on advancing medicine to treat patients suffering from serious inflammatory diseases. The company is developing a sustainable pipeline of first-in-class immune-modulators, including its flagship compound acebilustat (formerly CTX-4430). Our follow-on molecules enable new intellectual property and exhibit differentiated properties that allow optimization for alternate routes of administration. For more information, please visit: