Catabasis Pharmaceuticals Announces Top-Line Results for the Phase 3 PolarisDMD Trial of Edasalonexent in Duchenne Muscular D...
October 26 2020 - 5:00PM
Business Wire
-- PolarisDMD Trial Did Not Achieve Primary or
Secondary Endpoints --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced that the Phase 3
PolarisDMD trial of edasalonexent in Duchenne muscular dystrophy
(DMD) did not meet the primary endpoint, which was a change from
baseline in the North Star Ambulatory Assessment (NSAA) over one
year of edasalonexent compared to placebo. The secondary endpoint
timed function tests (time to stand, 10-meter walk/run and 4-stair
climb) also did not show statistically significant improvements.
Edasalonexent was observed to be generally safe and well-tolerated
in this trial. Catabasis is stopping activities related to the
development of edasalonexent including the ongoing GalaxyDMD
open-label extension trial. The Company plans to work with external
advisors to explore and evaluate strategic options going
forward.
“We are deeply saddened and disappointed by the results of our
Phase 3 PolarisDMD trial,” said Jill C. Milne, Ph.D., Chief
Executive Officer of Catabasis. “I want to sincerely thank all of
the boys, their families and caregivers, investigators and the
trial sites that participated in and enabled this program. The
entire Catabasis team has worked tirelessly to find a treatment for
this progressive disease. We hope that our data and work to date
can be used to benefit ongoing and future research in DMD.”
The Phase 3 trial was a one-year placebo-controlled trial
designed to evaluate the safety and efficacy of edasalonexent in
boys ages 4-7 (up to 8th birthday) with DMD. The global trial
enrolled 131 boys across eight countries, with any mutation type,
who were not on steroids. Edasalonexent was well-tolerated,
consistent with the safety profile seen to date. The majority of
adverse events were mild in nature and the most common
treatment-related adverse events were diarrhea, vomiting, abdominal
pain and rash. There were no treatment-related serious adverse
events and no dose reductions. The global COVID-19 pandemic had no
meaningful impact on the trial or its results. Data from the
PolarisDMD trial will be further analyzed and are expected to be
presented at an upcoming scientific conference and published.
“These results are disheartening for the Duchenne community, and
specifically for the boys who participated in this trial and their
families. However, the results contribute to the natural history
data of Duchenne and add to the knowledge base that will one day
produce a foundational, long-term therapy for this disease,” said
Pat Furlong, Founding President and Chief Executive Officer of
Parent Project Muscular Dystrophy (PPMD). “The continued
advancement of research and the development of possible treatment
options will remain of critical importance to our community. We
appreciate Catabasis’ efforts and commitment to every family that
is or has ever been affected by Duchenne.”
The Company expects to report Q3 2020 financials in November of
2020. As of September 30, 2020, Catabasis had cash and cash
equivalents of approximately $52.9 million.
About Catabasis
Catabasis Pharmaceuticals is a clinical-stage biopharmaceutical
company. Our mission is to bring hope and life-changing therapies
to patients and their families.
Forward Looking Statements
Any statements in this press release about future expectations,
plans and prospects for the Company, including statements about its
exploration and evaluation of strategic options, the termination of
activities related to the edasalonexent program, including the
ongoing GalaxyDMD open-label extension trial, and plans to publish
data from the Phase 3 PolarisDMD trial, and other statements
containing the words “believes,” “anticipates,” “plans,” “hopes,”
“expects,” and similar expressions, constitute forward-looking
statements within the meaning of applicable securities laws and
regulations. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors, including risks and uncertainties related to:
the impact of the COVID-19 pandemic and the effectiveness of the
steps we have implemented to address the pandemic; the availability
of funding sufficient for the Company’s foreseeable and
unforeseeable operating expenses and capital expenditure
requirements; unexpected costs or expenses that arise during the
termination of activities related to the edasalonexent program;
risks inherent in the Company’s exploration, evaluation and
implementation of its review of strategic options; and general
market and economic 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, 2020, 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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Catabasis: Investor
relations: Andrea Matthews investors@catabasis.com
Media: Elizabeth Higgins media@catabasis.com
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