Celtaxsys Announces Publication of Clinical Trial Results Demonstrating Its Novel Anti-inflammatory Medicine, Oral Acebilustat, Markedly Reduced Lung Inflammation Without Increased Risk of Immunosuppression, in Patients with Cystic Fibrosis (CF)

Print 03 November 2016

Acebilustat clinical trial results are especially encouraging given lung inflammation continues to be the leading cause of morbidity and premature mortality associated with CF

ATLANTA, GA--(Marketwired - November 03, 2016) - Celtaxsys, Inc., a clinical stage drug development company advancing therapies for patients with rare inflammatory diseases, announced today the publication of the second paper detailing the results from Phase 1 clinical trials for its flagship drug, acebilustat. Acebilustat is a novel once-daily oral anti-inflammatory drug in development for treatment of cystic fibrosis (CF) and other rare inflammatory diseases. This second paper details the effect of acebilustat on lung and systemic inflammatory biomarkers in a Phase 1B study in adult CF patients.

Acebilustat doses of 50 mg and 100 mg demonstrated reductions in markers of lung and systemic inflammation after 15 days of treatment in patients with CF. Sputum neutrophil count was reduced by 65% from baseline values in patients treated with 100 mg acebilustat, suggesting the potential for acebilustat treatment to enhance airway clearance. Notably, the combined treated group demonstrated a 58% reduction in sputum neutrophil elastase versus the placebo group. Neutrophil elastase is the most predictive biomarker for future decline in lung function in patients with CF. Reductions in serum C-reactive protein and sputum neutrophil DNA, two additional biomarkers strongly associated with CF lung inflammation, were also observed in acebilustat-treated patients. Both dose levels were observed to be safe and well-tolerated and no negative trends were observed in lung function. Importantly, even in the presence of reduced sputum neutrophil counts, bacterial colonization of the lung, as measured by sputum bacterial load, remained unchanged.

A Phase 2 study in CF patients (EMPIRE-CF) is currently enrolling in North America and Europe to test the ability of once-daily oral doses of 50 mg and 100 mg acebilustat to stem the decline in lung function and, potentially improve airway clearance, over 48 weeks of treatment in these patients. The ongoing Phase 2 study allows CF patients with any underlying genetic mutation to receive anti-inflammatory treatment with acebilustat in conjunction with their usual treatment which could include CFTR modulators (Kalydeco® and Orkambi®). This program is supported by a research grant from Cystic Fibrosis Foundation Therapeutics. For more information about this Phase 2 study, please visit: https://clinicaltrials.gov/ct2/show/NCT02443688.

The article is published online in the peer-reviewed journal Clinical and Translational Science (CTS), a publication of the American Society for Clinical Pharmacology and Therapeutics (ASCPT). The full article can be downloaded from CTS via the following link: http://onlinelibrary.wiley.com/doi/10.1111/cts.12428/full.

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, causing the body to accumulate excessive levels of unusually thick mucus in the lungs, leading to inflammation and severe infections that can reduce lung function and require hospitalization. CFTR protein dysfunction also results in malabsorption of nutrients and sometimes intestinal blockage. Respiratory distress in CF, defined as acute difficulty in breathing and infection with or without hospitalization, is most commonly related to lung infection and inflammation induced lung tissue damage resulting from 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 wellbeing. For more information on CF, visit: 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 rare 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: www.celtaxsys.com.

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