Catabasis Pharmaceuticals Announces Collaboration with the University of Texas Southwestern to Explore the Potential Benefits...
November 13 2018 - 7:45AM
Business Wire
-- One-Year Preclinical Study of Edasalonexent,
a Novel Inhibitor of NF-kB, in Animal Models of Duchenne to
Investigate Potential Improved Cardiac Function --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced a collaboration with
Pradeep Mammen, MD, FACC, FAHA, founder and Medical Director of the
Neuromuscular Cardiomyopathy Clinic at the University of Texas
Southwestern (UT Southwestern) Medical Center as well as
Co-Director of the National Institute of Health Sponsored UT
Southwestern Senator Paul D. Wellstone Muscular Dystrophy
Cooperative Research Center. The mission of this center is to
rapidly translate discoveries at the bench into therapies for
Duchenne muscular dystrophy (DMD) in the clinic. The collaboration
is designed to explore the potential of edasalonexent, a novel oral
NF-kB inhibitor, to improve cardiac function in Duchenne and Becker
muscular dystrophies.
Cardiomyopathy is the leading cause of mortality in Duchenne and
Becker muscular dystrophies. Young boys with Duchenne typically
have an elevated heart rate that exceeds the normal resting rate
for age, which is the first cardiac manifestation in boys with DMD.
Preclinical and clinical biomarker data support the potential for
cardiac benefits with edasalonexent in Duchenne and Becker muscular
dystrophies.
The one-year collaboration between Catabasis and Dr. Mammen will
utilize the muscular dystrophy mdx mouse model with reduced
utrophin as these animals display an early and prominent
cardiomyopathy. The study will evaluate both functional and
echocardiographic assessments of the heart as well as histological,
biochemical and molecular assessments to study the potential
benefits of edasalonexent treatment. Results are expected in the
second half of 2019.
“Inhibiting NF-kB with edasalonexent offers a unique mechanism
with the advantage of potentially impacting both skeletal and
cardiac muscle disease in those living with Duchenne and Becker
muscular dystrophies,” said Andrew Nichols, Chief Scientific
Officer at Catabasis Pharmaceuticals. “With the promising
preclinical and clinical biomarker data that we have seen to-date,
we are excited to collaborate with Dr. Mammen to learn from his
extensive experience and further explore the potential beneficial
heart effects of edasalonexent in these muscular dystrophies.”
Dr. Mammen is an expert in advanced heart failure, with a focus
on neuromuscular cardiomyopathy, and is board certified in advanced
heart failure/VAD/transplant cardiology. As a treating physician at
the UT Southwestern Medical Center, Dr. Mammen cares for Duchenne
and Becker muscular dystrophy patients in his role as the Medical
Director of the UT Southwestern’s dedicated Neuromuscular
Cardiomyopathy Clinic, which he founded in 2010. In addition, Dr.
Mammen is also the Director of Translational Research for the
Advanced Heart Failure Program and investigates the molecular
mechanisms underlying heart failure with more than 50 published
journal articles. He holds the Alfred W. Harris, M.D. Professorship
in Cardiology at UT Southwestern.
“There is significant unmet medical need for therapies that
could treat both the skeletal and cardiac muscle disease in
Duchenne and Becker muscular dystrophies,” said Pradeep Mammen,
M.D., Medical Director of the Neuromuscular Cardiomyopathy Clinic
at UT Southwestern. “I have dedicated my career to improving the
lives of patients with heart failure, and I look forward to helping
advance the understanding of edasalonexent and how it could benefit
patients in the future.”
The collaboration builds upon preclinical and clinical biomarker
data supporting the potential for cardiac benefits with
edasalonexent. In the Catabasis Phase 2 MoveDMD clinical trial and
open-label extension, significantly decreased heart rate towards
age-normative values was observed in boys with DMD. Preclinical
data in mdx mice and GRMD dogs, animal models of Duchenne, have
shown substantially decreased cardiac fibrosis with NF-kB
inhibition.
Edasalonexent is currently being studied as a potential
treatment for Duchenne muscular dystrophy in the Phase 3 PolarisDMD
clinical trial, a one-year, randomized, double-blind,
placebo-controlled trial. Catabasis plans to enroll approximately
125 patients in the trial ages 4 to 7 (up to 8th birthday)
regardless of mutation type who have not been on steroids for at
least 6 months. In the clinic, edasalonexent has been shown to
preserve muscle function and substantially slow DMD disease
progression across all four assessments of muscle function (the
North Star Ambulatory Assessment, time to stand, 4-stair climb, and
10-meter walk/run) compared to control. Edasalonexent has been well
tolerated through more than 50 patient-years of treatment with no
safety signals observed.
About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is
an investigational oral small molecule that is being developed as a
potential new standard of care for all patients affected by DMD,
regardless of their underlying mutation. Edasalonexent inhibits
NF-kB, which is a key link between loss of dystrophin and disease
progression in DMD. NF-kB has a fundamental role in skeletal and
cardiac muscle disease in DMD. Catabasis is currently enrolling the
single global Phase 3 PolarisDMD trial to evaluate the efficacy and
safety of edasalonexent for registration purposes. In the clinic,
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. Edasalonexent continues to be
dosed in the open-label extension of the MoveDMD Phase 2 clinical
trial. 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 CatabasisAt 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
development for the treatment of Duchenne muscular dystrophy. The
global Phase 3 PolarisDMD trial is currently enrolling boys
affected by Duchenne. For more information on edasalonexent and our
Phase 3 PolarisDMD trial, please visit www.catabasis.com or
www.twitter.com/catabasispharma.
Forward Looking StatementsAny 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, 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 Quarterly Report on Form 10-Q for the quarter ended June
30, 2018, 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 ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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