Celtaxsys to Present Clinical Data on Oral Acebilustat at North American Cystic Fibrosis Conference: Key Inflammation Biomarkers Reduced in CF Patients

Print 01 October 2015

Atlanta, GA. (Oct 1, 2015) – Celtaxsys, a clinical stage drug development company focused on advancing care for patients suffering from orphan inflammatory diseases, announced today that results from a clinical trial of acebilustat in CF patients will be presented at the 29th North American Cystic Fibrosis Conference (NACFC) in Phoenix Oct 8-10, 2015.

The presentation covers data from the first study of acebilustat in CF patients, in which 100 mg once daily oral acebilustat reduced neutrophil counts in the sputum of CF patients by 65% after only two weeks of treatment. This reduction in sputum neutrophil counts was accompanied by reductions in elastase and DNA in the sputum and a reduction in C-Reactive Protein (CRP) in the blood. Sputum neutrophils, elastase and DNA are important markers of lung inflammation in CF patients, while CRP is a marker of systemic inflammation. Importantly, the results also showed no changes in circulating neutrophils in the blood or bacterial load in the lungs of CF patients.

Acebilustat therapy selectively modulates neutrophil over-activation that drives CF lung inflammation, still the main cause of CF patient lung function decline and pre-mature death. Oral acebilustat has the potential to be beneficial to all CF patients regardless of CF Transmembrane Receptor (CFTR) genotype. A prior clinical study demonstrated that acebilustat treatment does not interfere with the enzyme system responsible for the clearance of other CF therapies such as Kalydeco® and Orkambi®, indicating that acebilustat can be used in conjunction with these important drugs.

Based on these promising initial results and with the endorsement of the Cystic Fibrosis Foundation (CFF), the company is initiating an international Phase 2 proof-of-concept trial to test the ability of once-daily oral acebilustat to preserve lung function in CF patients, including those receiving approved CFTR-targeted drugs and patients of all CFTR genotypes.

“We are excited by the results from our initial study in CF patients, which we believe clearly establishes proofof-mechanism for reduction of lung inflammation by acebilustat,” said Celtaxsys CEO Greg Duncan. “By treating underlying lung inflammation, we believe acebilustat treatment may have the potential to substantially improve lung function over time.”

Added Celtaxsys CMO Sanjeev Ahuja MD, “It is important to note that the inflammatory biomarkers reduced by acebilustat in our first CF study are highly correlated with important aspects of CF lung disease, such as lung function decline and airway obstruction. Acebilustat is a once-daily oral capsule that can be administered concomitantly with existing CF therapies and for all CFTR genotypes, which means that acebilustat has the potential to treat all CF patients.”

About Cystic Fibrosis: Cystic fibrosis (CF) is a life-threatening disease that affects the lung and digestive system and impacts about 70,000 patients worldwide. CF is caused by mutations in the Cystic Fibrosis Transmembrane conductance Regulator (CFTR) gene leading to abnormal CFTR protein functioning, the result of which causes the body to accumulate excessive levels of unusually thick mucus in the lungs. This excessive sticky mucus in the lungs is a site for infections that can require hospitalization. In the pancreas and GI tract CFTR protein dysfunction results in malabsorption of nutrients and sometimes intestinal blockage. Respiratory distress in CF, defined as acute difficulty in breathing, infection and/or hospitalization, is most commonly related to lung infection and inflammation induced lung tissue damage attributable to an overwhelming and dysfunctional response by dysregulated neutrophils. Treatment of this lung inflammation is, therefore, thought to be key to improving CF patient’s lung health and well-being. For more information on cystic fibrosis, go to www.cff.org.

About acebilustat: Acebilustat is a once-daily oral drug currently undergoing clinical trials for inflammatory diseases. It is a novel small molecule inhibitor of Leukotriene A4 Hydrolase (LTAH4), 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 cystic fibrosis and other orphan inflammatory diseases.

About Celtaxsys: Celtaxsys is a privately-held drug discovery and development company focused on advancing medicines to treat patients suffering from orphan inflammatory diseases. The company is building a sustainable pipeline of first-in-class immuno-modulators, the most advanced of which is acebilustat. Our follow-on molecules enable new intellectual property and exhibit differentiated properties that enable optimization for particular indications. For more information, visit www.celtaxsys.com

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