CAMBRIDGE, Mass.--(BUSINESS WIRE)--
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.
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