Catabasis Pharmaceuticals Announces Favorable Results for Functional Assessments in the MoveDMD® Trial for Edasalonexent in ...
April 25 2017 - 8:00AM
Business Wire
-- 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 --
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 CatabasisAt 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 StatementsAny 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.
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Investor and Media
Contact:Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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