Catabasis Pharmaceuticals & the Jain Foundation Announce a Preclinical Research Collaboration to Study Edasalonexent in Dysfe...
September 26 2019 - 8:00AM
Business Wire
-- Edasalonexent Inhibits NF-kB, a Potential
Driver of Disease Progression in Dysferlinopathy --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, and the Jain Foundation, a non-profit
foundation whose mission is to cure muscular dystrophies caused by
dysferlin protein deficiency, announced a preclinical research
collaboration to study edasalonexent (CAT-1004) in Dysferlinopathy.
Dysferlinopathy (Limb-girdle muscular dystrophy type 2B / Miyoshi
myopathy) is a serious rare disease that causes progressive muscle
weakness for which there are currently no approved treatment
options. Edasalonexent is in Phase 3 development for the treatment
of Duchenne muscular dystrophy (DMD) and has the potential to
benefit patients with other diseases, such as Dysferlinopathy.
In Dysferlinopathy, muscles lack dysferlin and as a result NF-kB
is chronically activated. Edasalonexent, an oral small molecule
designed to inhibit NF-kB, has the potential to slow disease
progression in dysferlin-deficient populations. Under this
collaboration, Catabasis and the Jain Foundation are conducting a
preclinical study to evaluate the potential of edasalonexent as a
therapeutic intervention for Dysferlinopathy by measuring disease
progression in dysferlin-deficient mice treated with edasalonexent.
The study will utilize magnetic resonance imaging (MRI) and
magnetic resonance spectroscopy (MRS) to measure muscle volume, fat
accumulation, and other changes in the dysferlin-deficient mice.
Initial results are expected in the first half of 2020.
“We look forward to working with Catabasis to advance research
for Dysferlinopathy,” said Laura Rufibach, Ph.D., and Doug
Albrecht, Ph.D., Co-Presidents of the Jain Foundation. “Patients
with Dysferlinopathy (LGMD2B / Miyoshi myopathy) experience a
progressive and debilitating decline in muscle function which
significantly impacts their lives. As there are currently no
treatment options, we are excited to explore the potential of
edasalonexent to benefit those living with this disease.”
“The chronic activation of NF-kB is a key driver in many
neuromuscular disorders, including Duchenne muscular dystrophy.
Evidence of NF-kB activation in Dysferlinopathy suggests a similar
disease mechanism and opportunity for intervention. In the MoveDMD
trial and open-label extension, treatment with edasalonexent slowed
disease progression compared to the off-treatment control period.
Through this collaboration, we look forward to learning more about
the potential of edasalonexent in Dysferlinopathy, where, similar
to Duchenne, there is also a significant unmet need,” said Andrew
Nichols, Ph.D., Chief Scientific Officer at Catabasis
Pharmaceuticals.
About Edasalonexent 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. We are currently enrolling our global Phase 3
PolarisDMD trial to evaluate the efficacy and safety of
edasalonexent for registration purposes. Edasalonexent is also
being dosed in the open-label extension trial GalaxyDMD. 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
development for the treatment of Duchenne muscular dystrophy. The
global Phase 3 PolarisDMD trial is currently enrolling boys
affected by Duchenne. For more information on edasalonexent and our
Phase 3 PolarisDMD trial, please visit www.catabasis.com.
About the Jain Foundation The Jain Foundation is a
non-profit foundation whose mission is to cure muscular dystrophies
caused by dysferlin protein deficiency, which includes the clinical
presentations Limb-girdle muscular dystrophy type 2B (LGMD2B) and
Miyoshi muscular dystrophy 1 (MMD1). Collectively these are
referred to as Dysferlinopathy. The foundation’s focused strategy
includes financing and actively directing the progress of projects
in key pathways towards a cure. These include identification and
assessment of promising drug candidates, the development and
maintenance of a global patient registry (the Dysferlin Registry),
as well as a natural history study of patients with Dysferlinopathy
to better understand disease progression and identify the best
outcome measures for use in clinical trials. For more information
about the Jain Foundation, the Dysferlin Registry, and the projects
we support, please visit www.jain-foundation.org.
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 Company’s
global Phase 3 PolarisDMD trial in DMD to evaluate the efficacy and
safety of edasalonexent for registration purposes, including the
anticipated timing for top-line results, potential timing for the
filing of an NDA, 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 preclinical or 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
Quarterly Report on Form 10-Q for the year ended June 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20190926005078/en/
Catabasis Investor and Media
Contact Andrea Matthews Catabasis Pharmaceuticals, Inc. T:
(617) 349-1971 amatthews@catabasis.com Jain
Foundation Contacts Doug Albrecht or Laura Rufibach Jain
Foundation Inc. T: (425) 882-1492 admin@jain-foundation.org
Catabasis Pharmaceuticals (NASDAQ:CATB)
Historical Stock Chart
From Aug 2024 to Sep 2024
Catabasis Pharmaceuticals (NASDAQ:CATB)
Historical Stock Chart
From Sep 2023 to Sep 2024