Catabasis Pharmaceuticals Research on CAT-5571, a Novel Activator of Autophagy & Potential Oral Treatment for Cystic Fibrosis...
January 05 2017 - 8:00AM
Business Wire
-- Preclinical Data Demonstrate that Fatty Acid
Cysteamine Conjugates Are Autophagy Activators that Enhance the
Correction of Misfolded F508del-CFTR --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced the publication of
preclinical data on CAT-5571, a novel activator of autophagy and
potential oral treatment for cystic fibrosis (CF). The preclinical
data demonstrate an increase in CF transmembrane conductance
regulator (CFTR) activity and trafficking which are detailed in an
article titled “Fatty Acid Cysteamine Conjugates as Novel and
Potent Autophagy Activators that Enhance the Correction of
Misfolded F508del-CFTR” in the Journal of Medicinal Chemistry (J
Med Chem. 2016 Dec 23. doi: 10.1021/acs.jmedchem.6b01539).
The publication describes the synthesis and biology of the
CAT-5000 series of molecules developed using the proprietary
Catabasis SMART linker drug discovery platform. The data
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 bronchial
epithelial cells isolated from multiple CF patients with the
F508del mutation. When CAT-5571 was used in combination with
lumacaftor and ivacaftor, it significantly enhanced the effects on
the cells of the standard of care combination, both in the amount
of the more mature C-band 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
lumacaftor/ivacaftor-mediated chloride current increase in cultured
primary homozygous F508del human bronchial epithelial cells.
“We are excited about these preclinical results and the
potential of autophagy activation by CAT-5571 as a treatment for CF
that is able to improve CFTR trafficking and function. We look
forward to progressing CAT-5571 in preclinical development and
toward clinical development,” said Andrew Nichols, Ph.D., Chief
Scientific Officer of Catabasis. “This program further builds our
rare disease pipeline and shows the capability of the proprietary
Catabasis SMART linker drug discovery platform.”
Catabasis expects to initiate a Phase 1 clinical trial with
CAT-5571 for the potential treatment of CF in Q4 2017 or Q1 2018.
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-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, regardless of CFTR mutation
status.
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 September 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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Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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