Catabasis Pharmaceuticals Announces Collaboration with the University of Texas Southwestern to Explore the
Potential Benefits of Edasalonexent on Cardiac Function in Duchenne and Becker Muscular Dystrophies
-- 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 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 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 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, 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.
Investor and Media Contact
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com
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