Celtaxsys announces full enrollment of its landmark EMPIRE-CF phase 2b clinical trial assessing the potential of novel anti-inflammatory investigational therapy, oral acebilustat, to preserve lung function in CF patients

ATLANTA, GA--(Marketwired - May 17, 2017) - Celtaxsys, Inc., a clinical stage pharmaceutical development company focused on advancing treatments for patients with rare pulmonary diseases, announced today the full enrollment of its phase 2b clinical trial testing its flagship anti-inflammatory drug candidate, once-daily oral acebilustat, in adult CF patients (NCT02443688). The study is investigating the potential of acebilustat to reduce lung inflammation and preserve lung function over the course of 48 weeks in CF patients at a high risk for rapid lung function decline. Lung inflammation is still the leading cause of excessive morbidity and premature mortality associated with CF and there are no drugs approved for the treatment of CF lung inflammation. Topline results are expected in mid-2018.

In total, 200 CF patients of all CFTR genotypes across North America and Europe have been enrolled for 48 weeks of treatment comparing 50 mg and 100 mg doses of once-daily oral acebilustat against placebo. In this trial, acebilustat or placebo are being given concomitantly with standard CF care, including CFTR modulator therapies (Orkambi® or Kalydeco®). In addition to investigating the effect of acebilustat on lung function (FEV1 percent predicted, a standard efficacy readout for CF trials), this trial will also assess the effect of acebilustat on pulmonary exacerbations and patient reported outcomes.

"This acebilustat phase 2b trial has the potential to take the next crucial step in transforming the way we treat underlying pulmonary inflammation in CF patients, which is not currently adequately addressed, even by the best available care today," said Steven Rowe, M.D., Professor of Medicine and the Director of the Cystic Fibrosis Research Center at the University of Alabama at Birmingham, the US Principal Investigator for the study.

The design of this trial enables evaluation of outcomes stratified by patient baseline FEV1 percent predicted, number of pulmonary exacerbations experienced in the year prior to enrollment, and whether the patient is taking concomitant CFTR modulators. The study will also assess inflammation biomarkers closely tied to acebilustat's immunomodulatory mechanism of action, including sputum neutrophil elastase and serum C-reactive protein. Reductions in these inflammatory markers were observed in an earlier trial of once-daily oral acebilustat in CF patients. "If acebilustat exhibits adequate safety and beneficial effects in preserving lung function, the outcome of this trial could herald the coming of a new era for anti-inflammatory treatment of CF patients, one which could change CF patient treatment standards moving forward," said Greg Duncan, Celtaxsys CEO. "The efforts of all the investigators, site staff, and, in particular, the patients enrolled in the study are highly appreciated."

This trial has been made possible by financial and operational support from Cystic Fibrosis Foundation (CFF) Therapeutics, the CFF's nonprofit drug discovery and development affiliate focused on advancing novel therapies for CF patients.

About Cystic Fibrosis: Cystic fibrosis (CF) is a life-threatening disease that affects the lung and digestive system of 70,000 patients worldwide. CF is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene leading to abnormal CFTR protein functioning, which causes excessively high levels of thick mucus to accumulate in the lungs. Thickened mucus clogs the lungs and serves as a perfect environment to catalyze persistent bacterial colonization of the lungs. Chronic bacterial colonization of the lungs in turn elicits an excessive neutrophil driven inflammatory immune response with the overabundance of neutrophils in the lungs further compromising a patient's breathing capacity. Somewhat paradoxically, excessive production of a neutrophil byproduct, neutrophil elastase, leads to reduced bacterial clearance. Most importantly, elastase is the key driver of irreversible damage to the lungs. Over time, this cycle of infection and inflammation leads to lung function decline and increased pulmonary exacerbations. Lung inflammation is still the leading cause of morbidity and mortality associated with CF leading the CF Foundation to identify development of safe and effective anti-inflammatory therapies as a key research priority. For more information about CF, visit: www.cff.org.

About acebilustat (formerly CTX-4430): Acebilustat is a once-daily oral immunomodulatory 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. Acebilustat is presently being evaluated in a CF phase 2b lung function preservation trial, which is supported by the CF Foundation. This trial is assessing acebilustat's potential to preserve CF patient breathing capacity by enhancing airway clearance and reducing the irreversible damage associated with an overactivated neutrophil driven inflammatory immune response.

About Celtaxsys: Celtaxsys, Inc. is a privately-held pharmaceutical discovery and development company focused on advancing medicine to treat patients suffering from rare, orphan designated diseases. The company is developing a sustainable pipeline of first-in-class anti-inflammatory immunomodulators, including its flagship compound, acebilustat (formerly CTX-4430). Our platform of follow-on candidates are covered separately under new intellectual property and exhibit differentiated properties that enable optimization of treatment for other inflammatory diseases. For more information, please visit: www.celtaxsys.com.