Market Overview

Catabasis Pharmaceuticals Presents New Edasalonexent Clinical Biomarker Data Showing NF-kB Inhibition and Target Engagement in the MoveDMD® Trial


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

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

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.

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