ATLANTA – October 22, 2018 – Celtaxsys, Inc., a clinical stage pharmaceutical development company focused on advancing treatments for patients with rare inflammatory diseases, today announced that results from the EMPIRE-CF Phase 2 study evaluating its investigational oral, once daily product, acebilustat, for the treatment of cystic fibrosis (CF) in 200 patients were presented at the North American Cystic Fibrosis Conference (NACFC) held October 18-20, 2018 in Denver, Colorado.
These results showed that acebilustat-treated patients (n = 133) exhibited a 19% reduction in pulmonary exacerbations (PEx) and a 22% reduced risk in progressing to first PEx versus placebo on a per protocol basis. Patients with less severe impairment of lung function (FEV1pp >75, n = 47) achieved the largest benefit from acebilustat treatment, achieving a 35% reduction in PEx rate, a 43% reduction in risk of experiencing their first exacerbation and a 96% increased likelihood of being free of exacerbations after 48 weeks of treatment versus placebo. Furthermore, patients concomitantly treated with CFTR modulator therapy (n = 43) exhibited a clinically meaningful 20% reduction in PEx, a 29% increased time to first exacerbation and a 47% higher likelihood of no exacerbations compared to patients treated with CFTR modulators and placebo.
In acebilustat treated patients, the majority of adverse events were mild or moderate in severity, with the most common being infective pulmonary exacerbation of CF, cough, hemoptysis, nasopharyngitis, headache, and increased sputum. There was a low discontinuation rate from adverse events among patients treated with acebilustat.
“The North American Cystic Fibrosis Conference provided an ideal opportunity for Dr. Steven Rowe and Dr. Stuart Elborn, both principal investigators for our Phase 2 trial of acebilustat, to present the comprehensive results from the trial to the CF community. These data showed that acebilustat has the potential to reduce the rate of pulmonary exacerbations in CF patients, including patients on a CFTR modulator. This is a critically important health outcome for the CF population that we expect to explore further,” said Greg Duncan, CEO of Celtaxsys. “We would like to thank Dr. Rowe and Dr. Elborn for their expert guidance throughout the trial and their participation in these presentations. We are also grateful to the patients and investigators who participated in the study.”
Celtaxsys, with continued support from the CF Foundation, has commenced preparations for designing and executing the Phase 3 clinical program of acebilustat.
Acebilustat is a once-daily oral drug candidate progressing to Phase 3 development. 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). LTB4 can create an over activation of the neutrophil mediated immune response and inflammation and has been strongly implicated in the pathogenesis of many diseases involving excessive inflammation, including cystic fibrosis (CF). Inflammation in the CF lung leads to pulmonary exacerbations and irreversible damage resulting in excessive morbidity and mortality in these patients. Acebilustat is designed to modulate the neutrophil driven immune response bringing the inflammation to homeostasis, preventing overactive inflammation from occurring, and thus could be potentially helpful in CF patients. By contrast, pro-resolving agents theoretically tone down inflammation once it is overactive and already contributing to lung damage in patients. Furthermore, unlike immunosuppressive treatments, such as corticosteroids, acebilustat has not demonstrated any evidence of immunosuppression in preclinical studies or in clinical trials in humans, including in healthy volunteers and CF patients. Acebilustat is the most advanced investigational product in development in the CF anti-inflammatory pipeline.
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, pancreas, and GI tract. Thickened mucus clogs the lungs and serves as a perfect environment to catalyze inflammation and infection of the lungs. This inflammation is primarily mediated by neutrophils. Over time, the amplification of the synergistic cycle of inflammation and infection leads to pulmonary exacerbations that could ultimately lead to lung function decline. Lung inflammation is still the leading cause of morbidity and mortality associated with CF.
Celtaxsys is a privately-held drug discovery and development company focused on advancing treatments for serious inflammatory diseases. The company is building a sustainable pipeline of first-in-class immuno-modulators, the most advanced of which is acebilustat. For more information, visit www.celtaxsys.com.
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