- New patents build on previously granted composition of matter protection for acebilustat, with additional intellectual protection for use in cystic fibrosis, as well as new compositions for second generation anti-inflammatory molecules
- Acebilustat, the most advanced new candidate in the CF anti-inflammatory pipeline, is a first in-class, oral anti-inflammatory medicine currently being assessed in a landmark CF lung function preservation trial, top line results of which will be reported in mid-2018
ATLANTA, GA – Celtaxsys, Inc., a clinical stage pharmaceutical development company focused on advancing treatments for patients with rare inflammatory diseases, announced today the issuance of four new patents, extending its leadership position in development of a robust pipeline of inhibitors of Leukotriene A4 Hydrolase (LTA4H), the key rate limiting enzyme in production of the inflammatory mediator Leukotriene B4 (LTB4). LTB4 is a potent mediator of neutrophil activity and when over-expressed, can lead to hyper-activation of neutrophils, resulting in sustained inflammation, tissue damage and reduced organ function, including in the lungs of CF patients. By modulating the over-production of LTB4, acebilustat and second generation LTA4H inhibitors, have the potential to return neutrophil response to a more healthy state (homeostasis), thereby reducing immune mediated morbidity and mortality. These new patents cover core intellectual property to 2034 before addition of extensions.
Said Andrew Luster, MD PhD, Chief of Rheumatology, Allergy & Immunology at Massachusetts General Hospital, and the Persis, Cyrus and Marlow B. Harrison Professor of Medicine at Harvard Medical School. “The field of LTA4H inhibitors is a largely untapped arena for treatment of human diseases. Emerging science in the field of LTA4H and LTB4 points to potential applications across many serious inflammatory diseases including CF, bronchiectasis, bullous dermatoses, NASH and cancer.”
One new patent (US patent 9,820,974 B2) covers use of the company’s flagship anti-inflammatory drug candidate, acebilustat, for treatment of cystic fibrosis (CF). This patent expands and extends the previously issued intellectual property portfolio surrounding acebilustat composition of matter. Acebilustat is the most advanced anti-inflammatory drug candidate in the CF pipeline. It is currently being studied in a Phase 2b trial (EMPIRE-CF) testing its ability to stem lung function decline and reduce pulmonary exacerbations over 48 weeks of treatment. The trial is fully enrolled and topline results are expected in mid-2018.
Three additional patents (US patents 9,822,106 B2; 9,856,249 B2; and 9,777,006 B2) cover new compositions of matter for second generation LTA4H inhibitors. These second generation compounds bolster the company’s LTA4H inhibitor platform covering novel compositions that provide an expanded array of properties to facilitate development of topical, inhaled and parenteral formulations as well as providing differential selectivity across functions of LTA4H. Additional patent filings are planned as Celtaxsys continues to extend its leadership position in the field of LTA4H inhibition.
“The expansion of our intellectual property platform represents important and continued progress for Celtaxsys, as well as for the 75,000 CF patients who are counting on companies like Celtaxsys to develop a safe and effective treatment for lung inflammation, which is still the leading cause of morbidity and mortality in CF” said Greg Duncan, Celtaxsys CEO.
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. 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. Results from an ongoing acebilustat Phase 2b lung preservation trial, conducted with the scientific and financial support of the CF Foundation, are expected in mid-2018.
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 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. Paradoxically, excessive production of a 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.
Media Contact: Angela Walsh
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