Catabasis Pharmaceuticals Presents Data Supporting Edasalonexent as a Potential Foundational Treatment for Duchenne Muscular Dystrophy

— Boys on Edasalonexent Grew More than Two Inches Taller per Year on
Average —

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis
Pharmaceuticals, Inc.
(NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today shared additional clinical results from
the MoveDMD trial of edasalonexent. In the Phase 2 MoveDMD trial and
open-label extension, boys with Duchenne muscular dystrophy (DMD)
treated with edasalonexent on average grew in line with the growth of
unaffected boys in the same age range. These data were presented
on Sunday, February 17, 2019, at the XVII International Conference on
Duchenne and Becker Muscular Dystrophy in Rome, Italy.

While the primary goal in treating boys with Duchenne is to slow the
progression of the disease, we recognize the significant negative impact
on boys’ quality of life when they do not grow and develop like their
friends. We are pleased to see boys on edasalonexent growing like their
unaffected peers while also demonstrating substantially slowed disease
progression,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of
Catabasis. “These characteristics are what make edasalonexent a great
potential foundational therapy for the treatment of Duchenne.”

In the MoveDMD trial, boys treated with edasalonexent grew an average of
2.1 inches taller per year and gained 2.9 pounds per year, and their
overall body mass index decreased from 70th percentile of
unaffected boys to the 55th percentile over 72 weeks of
treatment, approaching the average body mass index for unaffected boys.
Boys treated with corticosteroids, the standard of care in DMD,
typically experience excess weight gain and curtailed growth.

Using corticosteroids can be very beneficial to many patients with DMD.
However, alternatives are desperately needed as steroids come with a
long list of very unpleasant side effects, which includes stunted
growth. This can affect self-esteem and confidence. We are very
encouraged by this early data suggesting that boys on edasalonexent did
not have their growth affected, and we look forward to seeing more
results,” said Emily Crossley, co-founder and co-CEO, Duchenne UK.

Edasalonexent is being studied in the Phase 3 PolarisDMD trial, which is
actively enrolling patients. The PolarisDMD trial is evaluating the
efficacy and safety of edasalonexent in patients with DMD and is
intended to support an application for commercial registration of
edasalonexent. A total of 17 PolarisDMD clinical trial sites are now
open for enrollment across the United States, Canada and Australia.
Additional PolarisDMD clinical trial sites are expected to open in the
United States, Canada, Europe, Australia and Israel in the next couple
of months. In total, the PolarisDMD trial will include approximately 40
clinical trial sites globally with enrollment expected to be completed
in 2019.

The global Phase 3 PolarisDMD trial is a one-year, randomized,
double-blind, placebo-controlled trial. Catabasis plans to enroll
approximately 125 patients ages 4 to 7 (up to 8th birthday)
regardless of mutation type who have not been on steroids for at least 6
months. Boys on a stable dose of eteplirsen may be eligible to enroll.
The primary efficacy endpoint is change in the North Star Ambulatory
Assessment score after 12 months of treatment with edasalonexent
compared to placebo. Key secondary endpoints include the age-appropriate
timed function tests: time to stand, 4-stair climb and 10-meter
walk/run. Assessments of growth, cardiac and bone health are also
included as important potential areas of differentiation. Two boys will
receive 100 mg/kg/day of edasalonexent for each boy that receives
placebo, and, after 12 months, all boys are expected to receive
edasalonexent in an open-label extension study. The PolarisDMD trial
design was informed by discussions with regulators as well as input from
treating physicians, patient organizations and families of boys affected
by Duchenne.

The Phase 3 PolarisDMD trial is designed to further validate the
positive efficacy seen in the MoveDMD Phase 2 trial and open-label
extension evaluating edasalonexent. In the MoveDMD trial, we observed
that edasalonexent preserved muscle function and substantially slowed
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) through 72 weeks of treatment compared to an
off-treatment control period. Preclinical data and clinical biomarker
data from the MoveDMD Phase 2 trial suggest that edasalonexent could
have potential benefits in skeletal muscle, diaphragm and heart.
Edasalonexent has been well tolerated through more than 55 patient-years
of treatment with no safety signals observed.

More information about the Phase 3 PolarisDMD clinical trial is
available on clinicaltrials.gov
and in a recently recorded webinar
with PPMD. Contact the Catabasis clinical team with any questions at [email protected].

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. We are currently enrolling our global Phase 3 PolarisDMD
trial to evaluate the efficacy and safety of edasalonexent for
registration purposes. In our MoveDMD Phase 2 trial and open-label
extension, 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 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. Our 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 September 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.

Contacts

Andrea Matthews
Catabasis Pharmaceuticals, Inc.
T: (617)
349-1971
[email protected]

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