#NACFC2016 – Catabasis to Present Data on Potential Treatment for CFTR and Bacterial Infection

Daniela Semedo, PhD avatar

by Daniela Semedo, PhD |

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Catabasis Pharmaceuticals announced that it will present data on CAT-5571, its investigational oral treatment for cystic fibrosis (CF). The drug has potential effectiveness both on improving the working of the cystic fibrosis transmembrane conductance regulator (CFTR), and in treating Pseudomonas aeruginosa infection.

The data will be presented at the 30th Annual North American Cystic Fibrosis Conference (NACFC) now underway in Orlando, Florida (through Oct. 29), the company said in in a press release .

CAT-5571 is a small molecule that activates autophagy — a process that maintains cellular regulation and host defense mechanisms — known to be impaired in CF. The disease is caused by defects on the CFTR gene.

Feng Liu, PhD, principal scientist at Catabasis, will give an oral presentation and present a poster, titled “CAT-5571 as a Novel and Potent Autophagy Activator that Enhances the Trafficking of the F508DEL-CFTR.” The presentations, scheduled for today, highlight the mechanism of action of CAT-5571 on the restoration of autophagy.

Autophagy maintains cellular homeostasis by degrading dysfunctional cellular components, such as misfolded proteins or foreign pathogens, and converting them into useful building blocks, such as amino acids, nucleic acids, and lipids. CF patients with the common F508del mutation in the CFTR gene experience an upregulation in the activity of an enzyme called transglutaminase. This enzyme, in turn, disables a key protein that is needed for autophagy activation.

Once the autophagy process is impaired, there is an increase in a protein called p62. This protein is responsible for regulating the formation of misfolded proteins in the cells, and its accumulation effectively traps the F508del-CFTR. CAT-5571 was developed to reverse this sequence of events, allowing more of the misfolded CFTR to reach the cell surface and go about its work as an ion channel.

Also scheduled for today, Tracey Bonfield, PhD, an assistant professor at Case Western Reserve University School of Medicine, will present the poster, “CAT-5571 as a Novel Autophagy Activator that Enhances the Clearance of Pseudomonas Aeruginosa,

P. aeruginosa, a gram-negative extracellular bacterium, frequently infects CF patients and is increasingly resistant to antibiotic treatment. In the poster, Bonfield will emphasize the mechanism by which CAT-5571 can be effective in clearing this pathogen.