-- Preclinical Data Demonstrate Disease Modifying Effects in Two
Animal Models of Duchenne Muscular Dystrophy --
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 4, 2017--
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 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
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release about future expectations, plans and prospects for the Company,
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“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
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as of any date subsequent to the date of this release.
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Source: Catabasis Pharmaceuticals, Inc.
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com