Catabasis Pharma (CATB) Announces Presentation of Positive CAT-5571 Data in CF at NACFC

October 27, 2016 11:16 AM EDT

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Catabasis Pharmaceuticals, Inc. (Nasdaq: CATB) announced positive preclinical data presented at the 30th Annual North American Cystic Fibrosis Conference (NACFC), which support CAT-5571 as a potential oral treatment for cystic fibrosis (CF) with potential effects on both the cystic fibrosis transmembrane conductance regulator (CFTR) and on bacterial clearance of Pseudomonas aeruginosa. NACFC is sponsored by the Cystic Fibrosis Foundation and is the largest collaborative forum of its kind to advance research for the treatment and cure of CF.

The data presented demonstrate that CAT-5571 is a novel autophagy activator which, in combination with the current standard of care therapy, increased the cell surface expression and function of CFTR in human bronchial epithelial cells from multiple CF patients with the F508del mutation. When CAT-5571 was used in combination with lumacaftor and ivacaftor, it significantly enhanced the effects of the standard of care combination, both in the amount of the more mature Band C form of the CFTR protein with complex glycosylation, and in the amount of the CFTR protein reaching the cell surface. Importantly, CAT-5571 significantly enhanced the chloride current increase resulting from treatment with lumacaftor and ivacaftor as measured in a functional assay using cultured primary homozygous F508del human bronchial epithelial cells.

Data presented also showed the ability of CAT-5571 to increase bacterial clearance, which was evaluated in preclinical models both in vitro and in vivo. In vitro CAT-5571 pretreatment of human bronchial epithelial cells from a CF patient with the F508del mutation reduced intracellular bacterial load resulting from infection with P. aeruginosa compared to vehicle. In an acute infection model using a lethal challenge of P. aeruginosa in mice, CAT-5571 significantly enhanced the activity of a suboptimal dose of ciprofloxacin, improving the clinical score and overall survival. In a chronic infection model using gut-corrected Cftr-deficient mice infected with P. aeruginosa embedded in agarose beads, treatment with CAT-5571 alone for 10 days after the infection resulted in a significant reduction in the lung bacterial load and neutrophil counts.

“We are excited about these preclinical results and the potential for a treatment for CF that is able to improve CFTR trafficking and function as well as increase bacterial clearance, both through the novel mechanism of autophagy activation,” said Andrew Nichols, Ph.D., Chief Scientific Officer of Catabasis. “We look forward to progressing CAT-5571 in preclinical development. This program further builds our rare disease pipeline in addition to our lead program edasalonexent (CAT-1004) for the potential treatment of Duchenne muscular dystrophy and CAT-4001 for the potential treatment of neurodegenerative diseases such as Friedreich’s ataxia and ALS.”

CAT-5571 is a novel molecule comprising cysteamine covalently conjugated to docosahexaenoic acid (DHA) using the company’s SMART linker drug discovery platform to enhance the intracellular activity of the bioactive components. CAT-5571 allows sustained intracellular delivery of the two bioactive components leading to activation of autophagy through two different pathways. Autophagy is a process that maintains cellular homeostasis and host defense mechanisms, and is known to be impaired in CF. We have found that the level of autophagy activation achieved with CAT-5571 cannot be replicated by administering the bioactive components either individually or in combination, even at much higher concentrations.

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