Celtaxsys Announces Last Patient, Last Visit in Landmark CF Phase 2b Lung Function Preservation Trial, Clinical Results Expected in July

ATLANTA –  May 17, 2018 – Celtaxsys, Inc., a clinical stage pharmaceutical development company focused on advancing treatments for patients with rare inflammatory diseases, today announced the last patient, last visit in its 200-patient acebilustat Phase 2b lung function preservation trial for the treatment of cystic fibrosis (CF). This trial has been carried out with the scientific and financial support of the CF Foundation.

“Completing this trial represents a significant milestone for the development of our lead medicine, the company and the CF community.  We are excited to explore once daily acebilustat’s potential to reduce excessive lung inflammation as a complement to a CF patient’s current treatment regimen, irrespective of genotype,” said Greg Duncan, chief executive officer of Celtaxsys. “We would like to thank all of the patients and medical professionals who helped execute this groundbreaking program and are very much looking forward to sharing the Phase 2b trial results with the cystic fibrosis community this summer.”

Additionally, a scientific abstract from the trial has been accepted for oral presentation at the 2018 European Cystic Fibrosis Society Conference (ECFS) in Belgrade, Serbia on June 6-9, 2018. The company will also sponsor a satellite symposium focused on the science of CF lung inflammation and the importance of addressing the inflammatory component of CF, including an overview of the latest developments in the field and the design of the acebilustat CF Phase 2b trial.

Details on the sponsored symposium and presentation are as follows:  

Symposium Title: Inflammation in Cystic Fibrosis: The Next Frontier

Moderator: J. Stuart Elborn

Speakers: Steven M Rowe, Marcus Mall, and Jennifer Taylor-Cousar

Date: June 7, 2018
Time: 7:15 – 8:15 a.m. CEST

Location: Amphitheatre

Presentation Title: Demographics of patients in a phase 2 trial of acebilustat in patients with cystic fibrosis (EMPIRE CF)

Presenter: J. Stuart Elborn

Date: June 8, 2018
Time: 4:15-4:30 p.m. CEST

Location: Annex A

Additional details can be found on the ECFS website at: https://www.ecfs.eu/belgrade2018/sponsorship-and-exhibition/satellite-symposia

About acebilustat: Acebilustat is a once-daily oral drug candidate currently in Phase 2 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 neutrophil mediated immune response and inflammation and has been strongly implicated in the pathogenesis of many diseases involving excessive inflammation, including cystic fibrosis (CF). More specifically, an overactive immune response driven by neutrophils results in excessive inflammation in the CF lung. This causes lung clogging and irreversible damage resulting in excessive morbidity and mortality in CF patients. Acebilustat modulates 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 healthy volunteers and CF patients. Acebilustat is the most advanced therapy 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 persistent bacterial infection and inflammation of the lungs. Chronic infection of the lungs in turn elicits an excessive neutrophil driven inflammatory immune response, with the overabundance of neutrophils clogging the lungs, thereby further compromising a patient’s breathing capacity. Excessive production of a neutrophil byproduct, neutrophil elastase, has been shown to be the best predictor of lung damage and dysfunction over the life of a CF patient. Excessive production of neutrophil elastase can also lead to reduced bacterial clearance. Over time, the amplification of this synergistic cycle of infection and inflammation leads to lung function decline and an increase in life-threatening 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 Celtaxsys: 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.