Catabasis Pharmaceuticals Presents New Data for CAT-5571 as a Novel Potential Oral Treatment for Cystic
Fibrosis at the 40 th European Cystic Fibrosis Society Conference
-- CAT-5571 Restores Autophagy, a Host Defense Mechanism, Which Is Known to be Impaired in Cystic Fibrosis
--
-- Data Demonstrate Improved Intracellular Clearance of Bacteria of Importance in Patients with Cystic
Fibrosis --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, announces positive
preclinical data on CAT-5571, supporting its potential as an oral treatment for cystic fibrosis (CF). These data are being
presented today at the 40th Annual European Cystic Fibrosis Society (ECFS) Conference in Seville, Spain.
Patients with CF suffer from persistent lung infections with opportunistic pathogens such as Pseudomonas aeruginosa and
Burkholderia cenocepacia causing chronic infections that are difficult to eradicate from lung tissue. Approximately 50% of
CF patients are known to suffer from chronic infection of P. aeruginosa. B. cenocepacia is often resistant to all
available antibiotics and can be fatal for CF patients. These intracellular pathogens are typically restricted via autophagy in
people not affected by CF. CAT-5571 activates autophagy, a host defense mechanism, and the data presented demonstrate that CAT-5571
improves bacterial clearance of the chronic intracellular pathogens P. aeruginosa and B. cenocepacia.
“We are excited about the potential of CAT-5571 to address impaired autophagy, a host defense mechanism, in CF patients,
strengthening their ability to clear persistent serious lung infections. These data demonstrating CAT-5571’s ability to
significantly reduce the intracellular bacterial load of P. aeruginosa and B. cenocepacia suggest that CAT-5571 could
play an important role in improving clinical outcomes in combination with current CF therapies,” 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 which also includes our lead program edasalonexent for the potential treatment of Duchenne muscular
dystrophy and CAT-4001 for the potential treatment of neurodegenerative diseases.”
The data presented demonstrate that CAT-5571 activates autophagy, a host defense mechanism, in a cystic fibrosis animal model.
In cftr F508del/F508del mice, treatment with CAT-5571 restored the depressed autophagy markers Beclin-1 and LC3B, which are
critical components of the host defense system. The data presented also demonstrate an impact on the intracellular clearance of two
different types of bacteria. In vitro studies using normal or homozygous F508del human bronchial epithelial cells infected
with P. aeruginosa showed that CAT-5571 treatment caused a significant reduction in the intracellular bacterial
load. Similarly, in macrophages isolated from cftr F508del/F508del mice, in vitro treatment with CAT-5571
reduced the intracellular bacterial load of B. cenocepacia.
CAT-5571 is a novel molecule comprising cysteamine covalently conjugated to docosahexaenoic acid (DHA) that was engineered 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. Catabasis is conducting IND-enabling
activities for CAT-5571 and expects to initiate a Phase 1 clinical trial in 2018.
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 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 in preclinical studies 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.
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 www.catabasis.com.
Forward Looking Statements
Any statements in this press release about future expectations, plans and prospects for the Company, including statements about
future clinical trial plans and other 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 March 31, 2017,
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.
Investor and Media Contact
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com
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