Catabasis Pharmaceuticals & Duchenne UK Announce Partnership to Evaluate Edasalonexent in a Phase 2 Non-Ambulatory Duchenne M...
January 08 2020 - 8:00AM
Business Wire
-- Duchenne UK Grants Over $600,000 in Funding
to Support Patient and Clinical Trial Site Costs --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, and Duchenne UK, a charity that seeks to
fund and accelerate treatments and a cure for Duchenne muscular
dystrophy (DMD), have entered into a partnership for a Phase 2
trial of edasalonexent, a novel NF-kB inhibitor, in non-ambulatory
DMD patients. This exploratory Phase 2 trial, which is subject to
the receipt of adequate funding, is designed to assess safety,
pharmacokinetics and exploratory measures of function including
cardiac, skeletal muscle and pulmonary function in non-ambulatory
DMD patients.
“We are thrilled to announce plans to expand our knowledge of
edasalonexent to non-ambulatory boys and men affected by Duchenne.
We recognize the urgent need for a well-tolerated treatment like
edasalonexent with the potential to slow disease progression and
preserve muscle function by benefitting both skeletal muscle as
well as cardiac function,” said Joanne Donovan, M.D., Ph.D., Chief
Medical Officer of Catabasis. “We are incredibly fortunate to have
the opportunity to partner with Duchenne UK for this important work
and appreciate their deep commitment as we work together to bring
treatment options to all patients.”
“We first approached Catabasis last year to ask if we could
encourage them to advance a trial to look at the non-ambulant
patient population and we are delighted to be able to announce this
collaboration today,” said Emily Crossley and Alex Johnson,
Duchenne UK cofounders. “Duchenne UK is committed to developing
medicines for all boys and men with DMD, regardless of their
physical stage, mutation or age. This trial will represent an
important step in that direction.”
The Phase 2 trial is designed to be a one-year, randomized,
double-blind, placebo-controlled trial evaluating safety,
pharmacokinetics and exploratory measures of function with
edasalonexent in non-ambulatory boys and men affected by DMD. The
trial expects to enroll approximately 16 non-ambulatory patients
ages 10 and older regardless of mutation type who have not been on
steroids for at least 6 months at clinical trial sites in the
United Kingdom. The exploratory functional endpoints are
anticipated to include assessments of cardiac function, upper limb
skeletal muscle function and pulmonary function. In addition, the
trial is also expected to explore patient reported outcomes. The
intention is that upon completing this trial, patients will have
the option to transition to the GalaxyDMD open-label extension
trial and receive edasalonexent.
About Edasalonexent (CAT-1004) Edasalonexent (CAT-1004)
is an investigational oral small molecule designed to inhibit NF-kB
that is being developed as a potential foundational therapy for all
patients affected by DMD, regardless of their underlying mutation.
In DMD the loss of dystrophin leads to chronic activation of NF-kB,
which is a key driver of skeletal and cardiac muscle disease
progression. Our ongoing global Phase 3 PolarisDMD trial is
evaluating the efficacy and safety of edasalonexent for
registration purposes. Edasalonexent is also being dosed in the
GalaxyDMD open-label extension trial. In our MoveDMD Phase 2 trial
and open-label extension, we observed that edasalonexent preserved
muscle function and substantially slowed disease progression
compared to rates of change in a control period, and significantly
improved biomarkers of muscle health and inflammation. 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, 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 lead program is edasalonexent, an NF-kB inhibitor in
Phase 3 development for the treatment of Duchenne muscular
dystrophy. For more information on edasalonexent and our Phase 3
PolarisDMD trial, 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
including, among other things, statements about the potential
commencement of the Company’s planned Phase 2 trial in
non-ambulatory patients, the Company’s global Phase 3 PolarisDMD
trial in DMD to evaluate the efficacy and safety of edasalonexent
for registration purposes and the GalaxyDMD open-label extension
trial, including the anticipated timing for top-line results, the
potential timing for the filing of an NDA, the Company's cash
expectations, the Company’s planned transition to a
commercial-stage organization 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; 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-Q for the year ended September 30, 2019, 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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Andrea Matthews Catabasis Pharmaceuticals, Inc. T: (617)
349-1971 amatthews@catabasis.com
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