Catabasis Pharmaceuticals Presents New Edasalonexent Clinical Biomarker Data Showing NF-kB Inhibition and
Target Engagement in the MoveDMD ® Trial
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today reported new
NF-kB inhibition biomarker results showing edasalonexent target engagement in the MoveDMD Phase 2 trial and open-label extension in
boys affected by Duchenne muscular dystrophy (DMD). NF-kB is a fundamental driver of disease progression in DMD. These results add
further support to the significant NF-kB biomarker results observed in Phase 1 of the MoveDMD trial and are consistent with
significantly decreased C-reactive protein (CRP) with edasalonexent treatment. These data were presented today at the New
Directions in Biology and Disease of Skeletal Muscle Conference in New Orleans, LA.
The NF-kB pathway is the key link between loss of dystrophin and disease manifestation and progression in DMD. Lack of
dystrophin combined with mechanical stress activates NF-kB, which promotes inflammation and fibrosis, suppresses muscle
regeneration and drives muscle degeneration. Edasalonexent is an oral small molecule that inhibits NF-kB and improves skeletal,
diaphragm and cardiac disease in mouse and dog models of DMD.
During the off-treatment control period when boys were not receiving edasalonexent, transcripts of NF-kB-regulated genes
increased in whole blood, consistent with systemic inflammation in DMD. Gene expression analysis was performed following 12 and 24
weeks of 100 mg/kg of edasalonexent treatment and transcripts of NF-kB-regulated genes significantly decreased by 2-fold
(p<0.005), showing decreased NF-kB signaling in boys receiving edasalonexent and consistent with edasalonexent reducing
inflammation.
“We are pleased to see additional clinical evidence demonstrating the activity of edasalonexent in inhibiting NF-kB in boys
affected by Duchenne, consistent with the observed substantial slowing of Duchenne disease progression in the assessments of muscle
function and magnetic resonance,” said Andrew Nichols, Ph.D., Chief Scientific Officer of Catabasis. “Building on the results
previously reported for edasalonexent treatment in patients in the MoveDMD trial, these new biomarker data further support the
consistent positive edasalonexent results. We look forward to advancing edasalonexent in a single global Phase 3 trial this year
with the goal of improving the quality and length of life for those affected by Duchenne.”
The decrease in NF-kB-regulated transcripts with edasalonexent treatment is consistent with biomarker results that showed CRP
was significantly decreased with edasalonexent at 12, 24, 36 and 48 weeks compared to baseline in the 100 mg/kg treatment group
(p≤0.001). CRP is a well-characterized blood marker that provides a global assessment of inflammation, and CRP is elevated in boys
affected by DMD. The significant decrease observed in CRP supports the biological activity of NF-kB inhibition by edasalonexent
treatment decreasing inflammation.
In the Phase 2 and open-label extension of the MoveDMD trial, edasalonexent substantially slowed DMD disease progression in boys
on 100 mg/kg through more than a year of treatment compared to the rate of change in the off-treatment control period. Across all
assessments of muscle function, consistent improvements were observed in the rate of decline after 12, 24, 36, 48 and 60 weeks of
oral 100 mg/kg edasalonexent treatment. Edasalonexent was well tolerated with no safety signals observed in the trial. The MoveDMD
trial investigated the safety and efficacy of edasalonexent and enrolled boys not on steroids ages 4 to 7 affected with DMD
regardless of mutation type.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an investigational oral small molecule that is being developed as a potential disease-modifying therapy
for all patients affected by DMD, regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a protein that is
activated in DMD and drives inflammation, fibrosis and muscle degeneration and suppresses muscle regeneration. Edasalonexent
continues to be dosed in an open-label extension of the MoveDMD Phase 2 clinical trial, and Catabasis is preparing to initiate a
single global Phase 3 trial in the second half of 2018 to evaluate the efficacy and safety of edasalonexent for registration
purposes. 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 reported
to-date, 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. Edasalonexent was
designed using our SMART (Safely Metabolized And Rationally Targeted) Linker drug discovery platform that enables us to engineer
molecules that simultaneously modulate multiple targets in a disease. For more information on edasalonexent or our drug discovery
platform, 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 plans to commence a single global Phase 3
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; the Company’s ability to obtain financing on acceptable terms and
in a timely manner to fund the Company’s planned Phase 3 trial of edasalonexent in DMD for registration purposes; 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 March 31, 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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