Miércoles 13 de Diciembre 2017

Catabasis Pharmaceuticals Announces Favorable Results for Functional Assessments in the MoveDMD® Trial for Edasalonexent in Duchenne Muscular Dystrophy at the American Academy of Neurology 69th Annual Meeting

Guía de Regalos

— Prespecified Analysis of Part B Data Shows Improvement in Rates of
Change Across Five Functional Assessments —

— Part C Interim Results to be Announced in Q3 2017 —

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Catabasis
Pharmaceuticals, Inc.
(NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced additional favorable results
across multiple functional assessments in the MoveDMD trial at the
American Academy of Neurology 69th Annual Meeting. In Part B
of the MoveDMD trial, designed to evaluate the potential of
edasalonexent in the treatment of Duchenne muscular dystrophy (DMD),
numerical improvements were seen in prespecified rate change analyses
across five functional assessments. These results are in addition to and
consistent with the numerical improvements in the same functional
assessments with edasalonexent compared to placebo after 12 weeks of
edasalonexent treatment.

Following our completed analysis of the rate of change data from Part B
of the MoveDMD trial, we are encouraged by the consistency of the
possible treatment effects across the range of functional assessments
after only 12 weeks of dosing as well as the numerical improvements in
functional assessments compared to placebo. These functional assessments
are meaningful to boys affected by Duchenne and are known to correlate
with loss of milestones and disease progression,” said Joanne Donovan,
M.D., Ph.D., Chief Medical Officer of Catabasis. “Coupled with the
reassuring safety, tolerability and plasma exposure data in patients
affected by Duchenne, we are optimistic about edasalonexent’s potential
and look forward to continuing to evaluate it as a novel treatment for
this devastating disease.”

In the MoveDMD trial, functional assessments were performed at baseline
of Part A, at baseline of Part B, which was on average 8 months later,
and at the endpoint of Part B, which was following 12 weeks of
treatment. This design enabled the comparison of changes in functional
ability between an extended off-treatment period and 12 weeks of
treatment with edasalonexent. The MoveDMD trial was not powered for
functional assessments and these analyses were generally not
statistically significant.

  • During the off-treatment period there were declines in average speeds
    of timed function tests (10-meter walk/run, 4-stair climb and time to
    stand) as well as the North Star Ambulatory Assessment (NSAA) and
    Pediatric Outcomes Data Collection Instrument (PODCI) assessment
  • During edasalonexent treatment, positive numerical changes were
    observed across the five functional assessments compared with the
    off-treatment period:

    • Rate of decline slowed by 50% for 10-meter walk/run
    • Rate of decline slowed by 45% for time to stand
    • Rate of decline slowed by more than 50% for 4-stair climb with no
      decline in function
    • Rate of decline in score slowed by more than 50% for NSAA with
      positive improvement seen
    • Rate of decline in score slowed by more than 50% for PODCI
      (p=0.01) with positive improvement seen

The edasalonexent 100 mg/kg/day treatment group also showed numerical
improvement versus placebo across these five functional assessments in
Part B of the trial. Functional assessments included in this trial have
precedence as endpoints for pivotal trials in DMD.

Catabasis’ MoveDMD trial is a three-part clinical trial investigating
the safety and efficacy of edasalonexent in boys ages 4 – 7 affected
with DMD (any confirmed mutation). The planned prespecified analyses
from Part B of the MoveDMD trial included a cross-over comparison to
evaluate the rate of change for the functional assessments while the
patients were largely off-treatment (Part A baseline to Part B baseline)
to the rate of change while on edasalonexent treatment for 12 weeks in
Part B. This comparison included the twelve boys that participated in
Part A and then crossed over to edasalonexent treatment in Part B. In
January 2017, Catabasis reported that the primary efficacy endpoint of
MRI T2 was not met and numerical improvements were observed for the
functional assessments with the placebo-controlled comparisons in Part B
of the trial.

From Part A of the MoveDMD trial, the Company reported in January 2016
that edasalonexent was generally well tolerated with no safety signals
observed and NF-kB target engagement was observed. Consistent with Part
A, there were no safety signals and edasalonexent was well tolerated in
Part B of the trial. There were no treatment-related serious adverse
events, no drug discontinuations and no dose reductions.

Catabasis intends to report results from Part C, the open-label
extension part of the MoveDMD trial, in 2017. To allow for all the boys
participating in Part C to complete 24 weeks of dosing with
edasalonexent, an interim update on Part C results is now planned for Q3
2017. Following additional data analysis on functional assessments from
Part C, the Company will determine the next steps for edasalonexent in
DMD.

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 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 www.catabasis.com.

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 linker 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 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 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; availability and timing
of results from preclinical studies and clinical trials; 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 Annual Report on Form 10-K for the year ended December 31,
2016, 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

Investor and Media Contact:
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com