Catabasis Pharmaceuticals Presents Edasalonexent, a Potential Foundational Treatment for Duchenne Muscular Dystrophy
05 October 2019 - 10:00PM
Business Wire
-- Differentiating Safety and Tolerability
Profile of Edasalonexent Through More Than 55 Patient Years of
Exposure --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today presented the findings from the
MoveDMD trial of edasalonexent. In the Phase 2 MoveDMD trial and
open-label extension, edasalonexent slowed disease progression
compared to the off-treatment control period and was well tolerated
through more than 55 cumulative patient years of exposure in boys
affected by Duchenne muscular dystrophy (DMD). These data were
presented by Dr. Richard Finkel, M.D., Chief, Division of
Neurology, Department of Pediatrics at Nemours Children’s Health
System, and Principal Investigator for the Phase 2 MoveDMD and
Phase 3 PolarisDMD studies of edasalonexent in DMD at the 24th
International Annual Congress of the World Muscle Society.
“Our goal is to provide a therapy for DMD that slows disease
progression, has a compelling safety profile and can be used in
boys regardless of mutation,” said Joanne Donovan, M.D., Ph.D.,
Chief Medical Officer of Catabasis. “The safety and tolerability
data from the MoveDMD trial support the potential of edasalonexent
to become a foundational therapy for those with Duchenne, from the
time of diagnosis onwards. Edasalonexent has broad potential for
benefit and can be used as a monotherapy as well as potentially
with other therapies. Our hope is to improve the quality of life
for those affected by Duchenne.”
In the MoveDMD trial and open-label extension, edasalonexent
preserved muscle function and substantially slowed disease
progression compared to rates of change in the off-treatment
control period, significantly improved biomarkers of muscle health
and inflammation and was safe and well-tolerated. In more than 55
cumulative patient years of exposure, the majority of adverse
events were mild in nature, and the most common treatment-related
adverse event was diarrhea, generally mild and transient. There
were no serious adverse events observed on treatment, and no
adverse trends in chemistry, hematology, or measures of adrenal
function. Edasalonexent is not a steroid and has not shown the
known side effects of corticosteroids.
Edasalonexent is an investigational oral small molecule designed
to inhibit NF-kB. In DMD the loss of dystrophin leads to chronic
activation of NF-kB, which is a key driver of skeletal and cardiac
muscle disease progression. Edasalonexent is currently being
studied in the Phase 3 PolarisDMD trial, which has fully enrolled
130 boys with DMD, ages 4 to 7 (up to 8th birthday) with any
mutation type and who had not been on steroids for the past 6
months. After the completion of 52 weeks of treatment, all boys and
their eligible siblings are expected to have the option to enroll
in GalaxyDMD, an open-label extension study designed to assess the
long-term safety of edasalonexent. Top-line results from the Phase
3 PolarisDMD trial are expected in the fourth quarter of 2020, and
the trial is anticipated to support an NDA filing in 2021.
About Edasalonexent (CAT-1004) Edasalonexent (CAT-1004)
is an investigational oral small molecule designed to inhibit NF-kB
that is being developed as a potential foundational therapy for all
patients affected by DMD, regardless of their underlying mutation.
In DMD the loss of dystrophin leads to chronic activation of NF-kB,
which is a key driver of skeletal and cardiac muscle disease
progression. Our ongoing global Phase 3 PolarisDMD trial is
evaluating the efficacy and safety of edasalonexent for
registration purposes. Edasalonexent is also being dosed in the
open-label extension trial GalaxyDMD. In our MoveDMD Phase 2 trial
and open-label extension, we observed that edasalonexent preserved
muscle function and substantially slowed disease progression
compared to rates of change in a control period, and significantly
improved biomarkers of muscle health and inflammation. 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. For a summary of clinical results, please visit
www.catabasis.com.
About Catabasis At Catabasis Pharmaceuticals, our mission
is to bring hope and life-changing therapies to patients and their
families. Our lead program is edasalonexent, an NF-kB inhibitor in
Phase 3 development for the treatment of Duchenne muscular
dystrophy. For more information on edasalonexent and our Phase 3
trial, 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
including, among other things, statements about the Company’s
global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and
safety of edasalonexent for registration purposes, including the
anticipated timing for top-line results, potential timing for the
filing of an NDA, 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; whether interim
results from a preclinical or 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 quarterly period ended June 30, 2019, 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 Investor and Media
Contact Andrea Matthews Catabasis Pharmaceuticals, Inc. T:
(617) 349-1971 amatthews@catabasis.com
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