Catabasis Pharmaceuticals Presents New Data for CAT-5571 as a Novel Potential Oral Treatment for Cystic Fibrosis at the 40th ...
June 08 2017 - 11:00AM
Business Wire
-- 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-5571Catabasis 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 FibrosisCystic 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 CatabasisAt 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 StatementsAny 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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Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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