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Catabasis Pharmaceuticals Reports Positive Results from Open-Label Extension of Phase 2 MoveDMD® Trial Evaluating Edasalonexent in Duchenne Muscular Dystrophy and Plans to Initiate Phase 3 Clinical Trial in First Half 2018


-- Edasalonexent Substantially Slowed Duchenne Muscular Dystrophy
Disease Progression through 36 Weeks

-- Improvements Across Multiple Assessments of Muscle Function and
Measures of Muscle Health

-- Conference Call October 4 at 8:30am ET --

Pharmaceuticals, Inc.
(NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported new positive efficacy results
showing sustained disease-modifying effects in the MoveDMD trial
open-label extension following 24 and 36 weeks of treatment with
edasalonexent. Across all key assessments of muscle function,
improvements were observed in the rate of decline after 24 and 36 weeks
of oral 100 mg/kg/day edasalonexent treatment compared to the rate of
change in the control period for boys prior to receiving edasalonexent
treatment. These data provide clinically meaningful evidence that
edasalonexent substantially slowed the progression of Duchenne muscular
dystrophy (DMD).

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Additionally, supportive changes in measures of muscle health were seen,
consistent with positive edasalonexent treatment effects. Muscle enzymes
significantly decreased compared to baseline at 12 weeks and later time
points (p<0.05) and lower leg muscle MRI T2 rate of change was
significantly improved in comparison to progression during the control
period (p≤0.05). Edasalonexent continued to be well tolerated with no
safety signals observed in the trial.

Based on the consistency of the MoveDMD results and supportive
regulatory input from FDA, Catabasis plans to initiate a single global
Phase 3 trial with edasalonexent in patients with DMD regardless of
mutation type in the first half of 2018 with top-line results expected
in 2020. The data were presented today in a late breaking session at the
World Muscle Society Conference and will be discussed, along with the
Phase 3 clinical trial plan, on a conference call today, October 4, 2017.

"We are extremely excited to see edasalonexent change the trajectory of
disease in the MoveDMD trial with substantially slowed disease
progression," said Jill C. Milne, Ph.D., Chief Executive Officer of
Catabasis. "Boys treated with edasalonexent stabilized; they experienced
meaningful improvements in muscle function compared to the rates of
change observed during the control period. Importantly, other supportive
positive measures of muscle health were observed. We look forward to
advancing edasalonexent as a disease-modifying therapy in a single Phase
3 pivotal trial as soon as possible with the goal of providing a
meaningful impact on disease progression for all boys affected by

"Our goal in treating boys with Duchenne is to slow the progression of
the disease. It is tremendously encouraging to see boys taking
edasalonexent stabilize in their functional abilities and MRI T2
measures along with its continued safety profile," said Richard Finkel,
M.D., Chief, Division of Neurology, Department of Pediatrics at Nemours
Children's Health System and a Principal Investigator for the study. "I
look forward to continuing to investigate edasalonexent as a potential
therapy for the many boys affected by this devastating disease."

In the MoveDMD trial, a substantial slowing of the disease progression
of DMD was seen in boys treated with edasalonexent compared to the rates
of change during the control period. Through 36 weeks of treatment, the
100 mg/kg/day treatment group showed clinically meaningful numerical
improvements in rates of decline compared to rates of change during the
control period across all three timed function tests (10-meter walk/run,
4-stair climb and time to stand), as well as the North Star Ambulatory
Assessment (NSAA), an integrated global assessment of muscle function.
Control period changes were measured prior to boys receiving
edasalonexent, either prior to Phase 2 or in the placebo group, for time
periods averaging 39 weeks. In the 100 mg/kg/day treatment group, 16
boys commenced edasalonexent either at the beginning of Phase 2 or at
the beginning of the open-label extension. At the time of the open-label
extension data analysis, all 14 boys continuing to participate had
received 100 mg/kg/day for 24 weeks and 11 had completed 36 weeks of 100
mg/kg/day edasalonexent treatment. Results are detailed for the 100
mg/kg/day treatment group as all boys are now taking this dose in the
open-label extension.

Additional supportive measures of muscle health were also consistent
with a positive edasalonexent treatment effect. Four muscle enzymes
(creatine kinase, alanine aminotransferase, aspartate aminotransferase
and lactate dehydrogenase) were significantly decreased compared to
baseline following edasalonexent treatment at 12 weeks and later time
points (p<0.05), consistent with the ability to slow muscle degeneration
and improve muscle integrity. Rate of change in lower leg MRI T2
significantly improved at 12 weeks and at last observation on treatment
compared to control period (p≤0.05), consistent with a reduction of
inflammation in the muscle.

Edasalonexent continued to be well tolerated with no safety signals
observed to date in the MoveDMD trial. The majority of adverse events
(AEs) have been mild in nature with no serious AEs. There have been no
dose reductions and no drug-related discontinuations. The most common
AEs were gastrointestinal, primarily mild and transient diarrhea.
Height, weight and BMI growth patterns were similar to standard growth
curves for unaffected boys in the age range of MoveDMD subjects. Boys
with DMD in this age range typically have resting tachycardia, a heart
rate that exceeds the normal resting rate, and heart rate of the boys
treated with edasalonexent decreased toward age-normative values during
treatment. We believe these clinical heart rate observations are
intriguing and warrant follow-up.

Catabasis plans to commence a single global Phase 3 trial in DMD in the
first half of 2018 to evaluate the efficacy and safety of edasalonexent
for registration purposes. The planned design of the randomized,
double-blind, placebo-controlled trial is informed by discussions with
FDA. Catabasis plans for the Phase 3 trial to have many elements in
common with the Phase 2 trial including the patient population and
endpoints. The trial is anticipated to enroll approximately 125 patients
ages 4 to 7 who have not been on steroids for at least 6 months. The
primary efficacy endpoint will be change in the North Star Ambulatory
Assessment score after 12 months of treatment with edasalonexent
compared to placebo. Key secondary endpoints are planned to include
age-appropriate timed function tests. Catabasis expects to report
top-line results from this trial in 2020.

"We are excited to see positive effects on muscle function with
edasalonexent, as we know from research that effects on muscle function
are the most important aspect of a therapy for Duchenne for the affected
patients and their families," said Pat Furlong, Founding President and
Chief Executive Officer of Parent Project Muscular Dystrophy (PPMD). "We
look forward to learning more about edasalonexent as Catabasis begins
its Phase 3 trial. With the disease-modifying effects and safety and
tolerability profile observed to date for edasalonexent, it has the
potential to be a foundational therapy for all people affected by

Conference Call and Webcast

Catabasis will host a conference call and webcast today, October 4,
2017, at 8:30am ET to discuss the open-label extension results from the
MoveDMD trial and the Phase 3 clinical trial plan for edasalonexent.

Participant Toll-Free Dial-In Number:       (877) 388-2733

Participant International Dial-In Number:  (541) 797-2984

Pass Code:                                               90435261

Please specify to the operator that you would like to join the
"Catabasis MoveDMD Results Call."

Interested parties may access a live audio webcast of the conference
call via the investor section of the Catabasis website,
Please connect to the Catabasis website several minutes prior to the
start of the broadcast to ensure adequate time for any software download
that may be necessary. The webcast will be archived for 90 days.

About the MoveDMD Phase 2 Trial

The MoveDMD trial included a randomized, double-blind,
placebo-controlled Phase 2 trial with 31 ambulatory boys enrolled
between ages 4 and 7 years with a genetically confirmed diagnosis of DMD
across a range of dystrophin mutations. The boys were all steroid naive.
The open-label extension was initiated in July 2016 and is evaluating
longer term safety and efficacy with the same clinical endpoints as the
placebo-controlled portion. Based on Phase 2 results, all boys who were
on the lower dose (67 mg/kg/day) were transitioned to the higher dose
(100 mg/kg/day) because a greater treatment effect was observed with the
higher dose, consistent with a dose response. Catabasis expects to
submit additional data for presentation to scientific meetings in 2018.

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 and fibrosis, muscle degeneration and suppresses
muscle regeneration. We are currently conducting the MoveDMD trial, a
three-part clinical trial investigating the safety and efficacy of
edasalonexent in boys enrolled at ages 4 – 7 affected with DMD (any
confirmed mutation). The third part of the trial, an open-label
extension with edasalonexent, is ongoing. 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 SMART
(Safely Metabolized And Rationally Targeted) Linker drug discovery
platform enables us to engineer molecules that simultaneously modulate
multiple targets in a disease. We are applying our SMART LinkerSM
platform to build an internal pipeline of product candidates for rare
diseases and plan to pursue partnerships to develop additional product
candidates. For more information on the Company's drug discovery
platform and pipeline of drug candidates, 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 our
plans to commence a single global Phase 3 trial in DMD in the first half
of 2018 to evaluate the efficacy and safety of edasalonexent for
registration purposes, our plans to report top-line results from this
trial in 2020 and our plans to continue to evaluate data from the
open-label extension of our MoveDMD® clinical trial of
edasalonexent for the treatment of DMD, 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, including the final trial design of our
planned Phase 3 trial in DMD; availability and timing of results from
preclinical studies and clinical trials, including the availability of
top-line results from our planned Phase 3 trial in DMD in 2020; 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; our
ability to obtain financing on acceptable terms and in a timely manner
to fund our planned Phase 3 trial in DMD to evaluate the efficacy and
safety of edasalonexent 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 period ended June 30,
2017, 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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