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Press Release

Catabasis Pharmaceuticals Presents Positive Data for CAT-5571, a Novel Activator of Autophagy, as a Potential Oral Treatment for Cystic Fibrosis at the 30th Annual North American Cystic Fibrosis Conference

Catabasis Pharmaceuticals,Inc.
Posted on: 27 Oct 16

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today 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.

About CAT-5571
Catabasis is developing 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 the bacterial clearance of Pseudomonas aeruginosa. CAT-5571 is a small molecule that activates autophagy, a process that maintains cellular homeostasis and host defense mechanisms, and is known to be impaired in CF. Catabasis has shown that CAT-5571, in combination with lumacaftor/ivacaftor, enhances cell-surface trafficking and function of CFTR with the F508del mutation. Catabasis has also shown that CAT-5571 enhances the clearance of P. aeruginosa infection in preclinical models of CF, irrespective of CFTR mutation status.

About Cystic Fibrosis
Cystic fibrosis (CF) is a rare, chronic, genetic, life-shortening disease that affects over 70,000 patients worldwide, predominantly in the Caucasian population. In CF, a malfunctioning cystic fibrosis transmembrane conductance regulator (CFTR) ion channel impairs chloride secretion, with deleterious effects on multiple organs, and particularly devastating effects on pulmonary, intestinal and pancreatic function. Patients affected with CF are also predisposed to respiratory failure caused by persistent lung infections that are difficult to treat with standard antibiotics. Advancements in research and treatments have extended the life expectancy for those living with CF, however there is currently no cure.

About Catabasis
At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. Our SMART (Safely Metabolized And Rationally Targeted) linker drug discovery platform enables us to engineer molecules that simultaneously modulate multiple targets in a disease. We are applying our SMART linker platform to build an internal pipeline of product candidates for rare diseases and plan to pursue partnerships to develop additional product candidates. For more information on the Company's drug discovery platform and pipeline of drug candidates, please visit

Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about future preclinical development plans and statements containing the words “believes,” “anticipates,” “plans,” “expects,” “may” and similar expressions, constitute forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties inherent in the initiation and completion of preclinical studies and clinical trials and clinical development of the Company’s product candidates; availability and timing of results from preclinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products; availability of funding sufficient for the Company’s foreseeable and unforeseeable operating expenses and capital expenditure requirements; other matters that could affect the availability or commercial potential of the Company’s product candidates; and general economic and market conditions and other factors discussed in the “Risk Factors” section of the Company’s Quarterly Report on Form 10-Q for the period ended June 30, 2016, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this press release. The Company anticipates that subsequent events and developments will cause the Company’s views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this release.

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Last updated on: 27/10/2016

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