Celtaxsys Begins US Enrollment for Phase 2 Trial of Acebilustat Anti-Inflammatory Therapy for Cystic Fibrosis

Print 08 October 2015

ATLANTA, Oct. 8, 2015 /PRNewswire/ -- Celtaxsys, a clinical stage drug development company focused on advancing care for patients suffering from orphan inflammatory diseases, announced today the commencement of the US arm of an international phase 2 clinical trial for its flagship compound, acebilustat, in patients with cystic fibrosis (CF). The study will be conducted at approximately 60 sites inthe United States and European Union, with the EU sites anticipated to begin enrollment early 2016. This landmark clinical trial testing once-daily oral acebilustat treatment over 48 weeks could be the first to establish proof-of-concept for an anti-inflammatory treatment specifically designed to prevent long-term loss of lung function in CF patients. The study will test once daily oral doses of 50 mg and 100 mg acebilustat against placebo on top of background therapy in adult CF patients.

Acebilustat is a first-in-class neutrophil modulator that controls a key inflammatory signal overexpressed in CF and other orphan inflammatory diseases. Acebilustat has been granted orphan drug status for the treatment of CF in the US and the EU.  Results from initial clinical studies in CF patients demonstrated acebilustat's ability to moderate the over-activated inflammatory response in CF, decreasing neutrophils in the lung by 65% and decreasing damaging neutrophil elastase in just two weeks of treatment. Acebilustat did this without jeopardizing the patient's immune response to infection. Importantly, acebilustat treatment is applicable to all CF patients irrespective of their gene mutation and can be used on top of background therapy, including Kalydeco and Orkambi.

The Phase 2 CF trial was designed in conjunction with and supported by a grant from Cystic Fibrosis Foundation Therapies, Inc. (CFFT), the non-profit drug discovery and development affiliate of the Cystic Fibrosis Foundation. It will include approximately 30 sites in the US under the oversight of Principal Investigator Dr. Stephen Rowe MD MSPH, Professor at the University of Alabama Birmingham School of Medicine, Director of the Cystic Fibrosis Transition Clinic, and Director of the CFFT Development Network Center for CFTR Detection. Said Dr. Rowe, "CF patients are in desperate need of efficacious and safe anti-inflammatory therapies to control progressive lung disease.  Evaluation of the long term effects of acebilustat is a very important step in that direction, given the favorable Phase 1b results in CF patients."

For more information, please visit http://clinicaltrials.gov/show/NCT02443688.

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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