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
-- 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 --
“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
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
From Part A of the MoveDMD trial, the Company reported in
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
About Catabasis
At
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
View source version on businesswire.com: http://www.businesswire.com/news/home/20170425005573/en/
Source:
Investor and Media Contact:
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com