Catabasis Pharmaceuticals Presents Edasalonexent, a Potential Foundational Therapy for Duchenne Muscular Dystrophy
May 12 2020 - 8:00AM
Business Wire
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today shared information on the
edasalonexent program in Phase 3 development for the treatment of
Duchenne muscular dystrophy (DMD) in poster presentations at the
2020 Muscular Dystrophy Association (MDA) Virtual Poster Session.
The three posters include an analysis of the baseline
characteristics of patients enrolled in the Phase 3 PolarisDMD
trial, age-normative growth and normal adrenal function seen in the
Phase 2 MoveDMD trial and open-label extension, and experience with
capsule swallowing in both clinical trials for edasalonexent in
boys with DMD. Top-line results from the Phase 3 trial are expected
in Q4 2020.
“As we advance our Phase 3 PolarisDMD trial, we gain valuable
insights into the full potential of edasalonexent,” said Joanne
Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “We are
pleased to share additional information supporting the design of
the Phase 3 PolarisDMD trial and showing long-term safety of
edasalonexent. In addition to being well-tolerated in over 100
cumulative years of patient exposure, edasalonexent has shown
potential to both preserve muscle function as well as positive
cardiac effects. Those living with Duchenne have limited treatment
options, and our goal is for edasalonexent to be a new foundational
therapy.”
An analysis of the baseline characteristics of the patients
enrolled in the Phase 3 PolarisDMD trial was performed compared to
the patients enrolled in the previous Phase 2 MoveDMD trial and
found overall similar baseline characteristics in the patient
populations in the two trials. Both the Phase 3 PolarisDMD trial
and the Phase 2 MoveDMD trial enrolled boys affected by DMD ages 4
to 7 (up to 8th birthday) with any mutation type who had not been
on steroids for the previous 6 months. There were no significant
differences between the two trials in baseline age, North Star
Ambulatory Assessment (NSAA) score and timed function test values
(time to stand, 4-stair climb, and 10-meter walk/run). These
findings are believed to support the assumptions on which the Phase
3 trial was powered.
Catabasis also shared long-term safety and tolerability data
from the MoveDMD trial and open-label extension. Edasalonexent was
associated with age-normative growth, without negative impact on
bone health, and normal adrenal function in boys with DMD as it
does not impact the glucocorticoid receptor. There was no evidence
of adrenal insufficiency for up to 150 weeks of edasalonexent
treatment, and no clinically significant changes in cortisol or
ACTH. Additionally, edasalonexent treatment in a mdx mouse model of
DMD preserved bone length and bone density. In more than 100 years
of cumulative patient exposure, edasalonexent has been
well-tolerated, with no serious adverse events on treatment, and
without the adverse effects associated with high-dose steroids. In
both the Phase 2 MoveDMD trial and the ongoing Phase 3 PolarisDMD
trial, 97% of 4 to 7 year-old boys with DMD screened for the
studies were able to swallow soft-gel study drug capsules. There
have been no discontinuations due to capsule burden. This
demonstrates that edasalonexent has the potential to be broadly
adopted by those affected by DMD and supports the long-term safety
and tolerability of edasalonexent.
Posters from the MDA Virtual Poster Session are available under
“Our Science” at www.catabasis.com.
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
GalaxyDMD open-label extension trial. 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
PolarisDMD 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, the Company’s global Phase 3 PolarisDMD trial in DMD to
evaluate the efficacy and safety of edasalonexent for registration
purposes, anticipated timing for top-line results 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 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 Annual Report on Form 10-K for the year ended December
31, 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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Investor and Media Contact Andrea
Matthews Catabasis Pharmaceuticals, Inc. T: (617) 349-1971
amatthews@catabasis.com
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