Celtaxsys Announces Publication of Results Demonstrating the Safety, Tolerability and Optimal PK/PD Profile of First-In-Class Anti-Inflammatory Medicine, Oral Acebilustat, in Phase 1 Clinical Trials Including Cystic Fibrosis Patients

Print 28 October 2016

ATLANTA, GA- October 28, 2016 - Celtaxsys, Inc., a clinical stage drug development company focused on advancing therapies for patients with rare inflammatory diseases, announced today the publication of the first in a series of papers detailing the results from the 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 first paper details pharmacokinetics (PK), pharmacodynamics (PD) and drug-drug interaction results, as well as initial safety and tolerability, from three Phase 1 studies of acebilustat in healthy volunteers and patients with CF.

Acebilustat was observed to be safe and well tolerated in the Phase 1 studies. The PK and PD support Phase 2 development of oral acebilustat in once-daily doses of 50 mg and 100 mg, demonstrating a rapid and sustained effect on modulating leukotriene B4, the pharmacologic target of this first-in-class leukotriene A4 hydrolase inhibitor. Importantly, no difference was observed in drug exposures between healthy volunteers and CF patients, and there was no difference in exposure when given under fasting conditions or after consuming a high fat meal. Finally, acebilustat did not induce CYP3A4, indicating that it may be suitable for use in combination with marketed CFTR modulators (Kalydeco® and Orkambi®).

The article will be published online in the peer-reviewed journal Clinical and Translational Science (CTS), a publication of the American Society for Clinical Pharmacology and Therapeutics (ASCPT). Follow this link to visit the CTS homepage: http://ascpt.onlinelibrary.wiley.com/hub/journal/10.1111/(ISSN)1752-8062.

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. 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.

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, causing severe infections that can 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, infection and/or 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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