Celtaxsys Presents Clinical Data on Oral CTX-4430 at 2015 European Cystic Fibrosis Conference: Positive Impacts on Key Biomarkers in Patients with CF

Atlanta, GA. (June 10, 2015) – Celtaxsys, a clinical stage drug development company focused on
advancing care for patients suffering from inflammatory diseases, including those with rare and
orphan inflammatory diseases, announced today data from Phase 1 clinical trials of CTX-4430
will be presented at the 2015 Meeting of the European Cystic Fibrosis Society in Brussels on
Friday, Jun 12, 2015.
The poster presentations are the following:
– A Phase 1 Clinical Study of CTX-4430 in Cystic Fibrosis Patients (Poster #126)
– Comparative Pharmacokinetics and Pharmacodynamics of CTX-4430 in Healthy
Volunteers and CF Patients (Poster #127)
– A Phase 1 Drug-Drug Interaction Study of CTX-4430 Assessing CYP3A4 Induction
(Poster #129)
These posters will be presented under the “New Therapies” section on Friday, Jun 12. There will
be a guided poster tour from 2-3 pm CET.
“We believe the data from our Phase 1 trials of once daily oral CTX-4430 clearly establish proof
of mechanism by showing encouraging correlated reductions in both blood and sputum
biomarkers of inflammation in CF patients after just 15 days of treatment. In particular,
reductions in LTB4 levels in both blood and sputum were accompanied by reductions in sputum
neutrophil counts, elastase and DNA, all important biomarkers of CF associated lung
inflammation. Importantly, these apparent reductions in lung inflammation did not result in
changes in sputum microbiology. Taken together the Phase 1 data show that CTX-4430 has the
potential to improve clinical outcomes for patients with CF by addressing the underlying
inflammation and we hope to demonstrate this in an upcoming Phase 2 study,” said Greg
Duncan, Chief Executive Officer of Celtaxsys. “Also, the fact that CTX-4430 does not induce
CYP3A4 shows that the molecule can be administered without adversely impacting the levels of
some of the newly introduced drugs for patients with CF,” added Sanjeev Ahuja, MD, Celtaxsys
Chief Medical Officer.
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 wellbeing.
For more information on cystic fibrosis, go to
About CTX-4430: CTX-4430 is a once-daily oral drug candidate 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
many diseases involving inflammation, including cystic fibrosis.
About Celtaxsys: Celtaxsys is a privately-held drug discovery and development company
focused on advancing medicine to treat patients suffering from serious inflammatory diseases.
The company is building a sustainable pipeline of first-in-class immuno-modulators, the most
advanced of which is CTX-4430. Our follow-on molecules enable new intellectual property and
exhibit differentiated properties that enable optimization for alternate routes of administration.
For more information, visit