Catabasis Pharmaceuticals Presents Information on Edasalonexent, a Potential Foundational Therapy for Duchenne Muscular Dystr...
September 28 2020 - 8:00AM
Business Wire
-- New Preclinical Research Indicates that
Edasalonexent Could Have Positive Effects on Cardiac Function and
Preserve Bone Health --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today shared information on the
edasalonexent program in Phase 3 development for Duchenne muscular
dystrophy (DMD) in poster presentations at the Virtual 25th
International Congress of the World Muscle Society. The posters
include new preclinical research findings supporting potential
positive effects on cardiac function and preservation of bone
health with edasalonexent in mouse models of DMD. Information from
clinical trials with edasalonexent was also presented with baseline
assessments from the Phase 3 PolarisDMD trial and long-term safety
results from the Phase 2 MoveDMD trial and open-label
extension.
“As we approach top-line Phase 3 PolarisDMD results, which we
expect to report in the fourth quarter of this year, we are pleased
to share new findings from preclinical studies with edasalonexent
that could support its potential as a foundational therapy for
those affected by Duchenne,” said Andrew Nichols, Ph.D., Chief
Scientific Officer of Catabasis. “Cardiac function and bone health
are critical components of Duchenne care and we are excited to
learn more about these key areas that are important to patients,
their caregivers and physicians.”
Persistent activation of NF-kB in DMD can drive cardiac
dysfunction, which is the leading cause of mortality in DMD. In a
preclinical study performed in the laboratory of Pradeep Mammen,
M.D., Medical Director of the Neuromuscular Cardiomyopathy Clinic
and Director of Translational Research for the Advanced Heart
Failure and Transplant Cardiology Program at UT Southwestern,
edasalonexent prevented the development of DMD-associated
cardiomyopathy in the mdx:Utrn+/- mouse model of DMD. Edasalonexent
reduced the cardiac hypertrophy apparent in these mice, reduced
myocardial fibrosis and prevented the development of DMD-associated
cardiomyopathy.
In DMD, NF-kB activation also drives inflammation and fibrosis,
leading to loss of skeletal muscle function and disease
progression. Reduced skeletal muscle function results in reduced
bone strength. Steroids, which activate the glucocorticoid receptor
(GR), can further negatively impact bone health. In a preclinical
study led by Frank Rauch, M.D., of Shriners Hospitals for Children
- Canada, edasalonexent was seen to maintain bone density and bone
strength in mdx mice. Consistent with these results, edasalonexent
treatment in cells inhibited NF-kB and as expected did not impact
the GR, while the steroid prednisolone strongly activated the
GR.
Information from the edasalonexent clinical program was also
presented at the Congress of the World Muscle Society. The primary
endpoint of the Phase 3 PolarisDMD trial for edasalonexent is the
North Star Ambulatory Assessment (NSAA), a validated measure of
physical function designed for use in ambulatory boys affected by
DMD. In an analysis of baseline characteristics of the patients
enrolled in the Phase 3 trial (boys affected by DMD, ages 4 to 7,
up to 8th birthday, not on steroids), Catabasis saw reliable and
consistent NSAA scores between the two pre-treatment measures at
screening and baseline, which indicate no significant learning
effect between visits.
An additional evaluation of data from patients at baseline
(prior to any treatment) in the Phase 3 PolarisDMD trial was shared
by Leanne Ward, M.D., FRCPC, Scientific Director of Ottawa
Pediatric Bone Health Research Group. This analysis showed that
lean body mass index (a marker for muscle mass) was reduced in
young boys affected by DMD and correlated with reduced muscle
function. An analysis of the same patients was presented earlier
this month at the American Society for Bone and Mineral Research
2020 Annual Conference and showed that DMD has a negative impact on
bone strength, including low bone density and mild vertebral
fractures at an early stage of disease course prior to any
treatment.
As Catabasis has previously presented, the Phase 2 MoveDMD trial
and open-label extension was also shared showing that edasalonexent
was well-tolerated for up to 150 weeks and associated with
favorable growth patterns. The most common related adverse effect
was diarrhea, generally mild and transient.
The posters are available to registered attendees during the
conference and for three months afterwards. Select posters are
available in the “Our Science” section of 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. The
ongoing global Phase 3 PolarisDMD trial is evaluating the efficacy
and safety of edasalonexent for registration purposes.
Edasalonexent is also being evaluated in the GalaxyDMD open-label
extension trial. In the MoveDMD Phase 2 trial and open-label
extension, the Company 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 the
clinical program of edasalonexent, including the expected timing of
release of Phase 3 data, edasalonexent potentially being a
foundational therapy for DMD patients, and edasalonexent
potentially having positive effects on cardiac function and
preservation of bone health, along with other statements containing
the words “believes,” “anticipates,” “plans,” “expects,” “may” and
similar expressions, constitute forward-looking statements within
the meaning of applicable securities regulations and laws. Actual
results may differ materially from those indicated by such
forward-looking statements as a result of various important
factors, including risks and uncertainties: inherent in the
initiation and completion of clinical trials and clinical
development; related to whether the results of pre-clinical studies
will be predictive of results of clinical trials; related to
whether the results of earlier stage clinical trials will be
predictive of the results of later stage trials; related to the
regulatory review and approval process; inherent in the
commercialization of marketed products; related to competitive
products, including those already approved and those in
development; related to other matters that could affect the
clinical development, regulatory status, availability or commercial
potential of edasalonexent, as well as the risks and uncertainties
discussed in the “Risk Factors” section of the Company’s Quarterly
Report on Form 10-Q for the period ended June 30, 2020, 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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version on businesswire.com: https://www.businesswire.com/news/home/20200928005152/en/
Investor relations: Andrea Matthews
investors@catabasis.com
Media: Elizabeth Higgins media@catabasis.com
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