Positive Preclinical Research on the Edasalonexent (CAT-1004) Program, a Potential Disease-Modifying Therapy for Duchenne Mus...
January 04 2017 - 7:00AM
Business Wire
-- Preclinical Data Demonstrate Disease
Modifying Effects in Two Animal Models of Duchenne Muscular
Dystrophy --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced the publication of
preclinical data on the edasalonexent program, a potential
disease-modifying therapy for Duchenne muscular dystrophy (DMD).
The preclinical data demonstrate that edasalonexent (CAT-1004) and
an analog, CAT-1041, oral inhibitors of NF-kB, are effective in
ameliorating the dystrophic process in two animal models of DMD in
an article titled “Disease Modifying Effects of Orally Bioavailable
NF-kB Inhibitors in Dystrophin-Deficient Muscle” in JCI Insight
(JCI Insight 2016 Dec 22;1(21):e90341).
This research was led by H. Lee Sweeney, Ph.D., then at the
University of Pennsylvania. Edasalonexent (CAT-1004) and CAT-1041,
which represent a novel class of NF-kB inhibitors, were evaluated
in both mdx mouse and golden retriever muscular dystrophy (GRMD)
dog models of DMD. Initial studies with edasalonexent and CAT-1041
in mdx mice demonstrated nearly identical in vitro and in vivo
efficacy, and CAT-1041 was selected for further evaluation in the
treatment of dystrophic muscle. In vivo, CAT-1041 effectively
improved the phenotype of mdx mice undergoing voluntary wheel
running, in terms of activity, muscle mass and function, damage,
inflammation, fibrosis and cardiac pathology. The researchers
identified significant increases in dysferlin as a possible
contributor to the protective effect of CAT-1041 against
sarcolemmal damage. Furthermore, CAT-1041 improved the more severe
GRMD phenotype in a canine case study, where muscle mass and
diaphragm function were maintained in a treated GRMD dog.
“There remains a large unmet need in Duchenne for therapies that
can treat all affected boys and slow disease progression. The
orally bioavailable NF-kB inhibitors, edasalonexent and CAT-1041,
improve the severe dystrophic phenotype found in both
mechanically-damaged mdx mice and a GRMD dog and create an
environment that can support more successful muscle regeneration,”
said Dr. Sweeney, currently Myology Institute Director, University
of Florida. “We believe that these in vivo preclinical results
support edasalonexent as a candidate for the treatment of DMD.”
“We very much appreciate the research performed by Dr. Sweeney
and his colleagues,” said Andrew Nichols, Ph.D., Chief Scientific
Officer of Catabasis. “We agree that these data support
edasalonexent as a potential treatment to improve both the quantity
and quality of muscle fibers in boys affected by DMD and look
forward to the Phase 2 clinical trial results with edasalonexent in
the first half of Q1 2017.”
About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is
an oral small molecule that has the potential to be a
disease-modifying therapy for all patients affected by Duchenne
muscular dystrophy (DMD or Duchenne), regardless of their
underlying mutation. Edasalonexent inhibits NF-kB, a protein that
is activated in Duchenne and drives inflammation and fibrosis,
muscle degeneration and suppresses muscle regeneration. In animal
models of DMD, edasalonexent produced beneficial effects in
skeletal, diaphragm and cardiac muscle and improved function. The
FDA has granted orphan drug, fast track and rare pediatric disease
designations and the European Commission has granted orphan
medicinal product designation to edasalonexent for the treatment of
DMD. We have previously reported safety, tolerability and reduction
in NF-kB activity in Phase 1 trials in adults. We are currently
conducting the MoveDMD® trial of edasalonexent in 4-7 year-old boys
affected by Duchenne. From Part A of the MoveDMD trial, we have
reported that edasalonexent was generally well tolerated with no
safety signals observed and we observed NF-kB target
engagement. Pharmacokinetic results demonstrated edasalonexent
plasma exposure levels consistent with those previously observed in
adults, at which inhibition of NF-kB was observed.
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
Catabasis Pharmaceuticals (NASDAQ:CATB)
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