Catabasis Pharmaceuticals Provides Update on Global Phase 3 PolarisDMD Trial for Edasalonexent at Parent Project Muscular Dys...
June 27 2019 - 8:00AM
Business Wire
-- Patient Identification Nearing Completion
for Global Phase 3 PolarisDMD Trial for Edasalonexent in Duchenne
Muscular Dystrophy --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, shared an update on the clinical
development of edasalonexent, a novel NF-kB inhibitor for the
treatment of Duchenne muscular dystrophy (DMD) this week at the
Parent Project Muscular Dystrophy (PPMD) 25th Annual
Conference.
Catabasis shared progress in the edasalonexent Phase 3
PolarisDMD trial:
- Screening is ongoing for the randomized, double-blind,
placebo-controlled trial in the United States, Canada, Australia,
the United Kingdom, Ireland, Sweden, Germany and Israel and making
strong progress.
- Clinical trial sites globally are enrolling quickly and sites
in the United Kingdom, Ireland, Sweden, Germany and Israel are at
capacity and no longer accepting additional patients.
- The trial is active in all planned countries with 37 sites open
for enrollment.
- Top-line results from the study are expected in the second half
of 2020 and are anticipated to support an NDA filing in early
2021.
“We are very pleased with the progress made in our Phase 3
PolarisDMD trial for edasalonexent in Duchenne. There has been
significant interest from families and enthusiasm from
investigators globally, and enrollment is going very well with some
countries already at capacity,” said Joanne Donovan, M.D., Ph.D.,
Chief Medical Officer of Catabasis. “We appreciate the hard work of
our study sites and the commitment of participating families as we
look to develop a new treatment to benefit all boys affected by
Duchenne.”
“There is clear need for a therapy that could benefit all boys
affected by Duchenne, regardless of mutation type, by slowing
disease progression while being well tolerated. We are glad to be
participating in the Phase 3 PolarisDMD trial for edasalonexent and
are pleased that Kennedy Krieger is a top enrolling site,” said
Kathryn Wagner, M.D., Ph.D., Director of the Center for Genetic
Muscle Disorders at the Kennedy Krieger Institute, a Professor of
Neurology and Neuroscience at the Johns Hopkins School of Medicine
and a Principal Investigator in the Phase 3 PolarisDMD trial for
edasalonexent. “We have found the PolarisDMD trial to be
thoughtfully designed to be family friendly as well as
straightforward for our clinical team.”
Catabasis also shared insights from a recent blinded qualitative
research project that collected information from in-depth
interviews with approximately 30 physicians treating boys affected
by DMD, caregivers and patient advocacy representatives in the
United States that was sponsored by Catabasis. A key observation
from the research was that members of the DMD community prioritized
treatments that will go beyond skeletal muscle and positively
impact additional important aspects of DMD, including cardiac and
pulmonary effects. Participants also shared their hope that
treatments will provide durable benefits and improve quality of
life. Additionally, physicians predicted that the majority of boys
will receive combination therapy for the treatment of DMD within
the next few years.
Data from the edasalonexent MoveDMD Phase 2 open-label extension
study were also presented at the PPMD conference. Through 72 weeks
of treatment compared to an off-treatment control period, patients
treated with edasalonexent demonstrated preserved muscle function
and substantially slowed DMD disease progression across all four
assessments of muscle function (the North Star Ambulatory
Assessment, time to stand, 4-stair climb and 10-meter walk/run).
Preclinical data and clinical biomarker data from the MoveDMD trial
suggest that edasalonexent could have potential benefits in
skeletal muscle, diaphragm and heart. Edasalonexent has been well
tolerated through more than 55 patient-years of treatment.
In the Phase 3 PolarisDMD trial, Catabasis plans to enroll
approximately 125 patients ages 4 to 7 (up to 8th birthday)
regardless of mutation type who have not been on steroids for at
least 6 months. Boys on a stable dose of eteplirsen may be eligible
to enroll. Boys from the MoveDMD open-label extension and their
eligible siblings can now enroll in the GalaxyDMD open-label
extension trial. Boys who complete the Phase 3 PolarisDMD trial as
well as their eligible siblings will also have the opportunity to
participate in the GalaxyDMD open-label extension trial. Boys can
begin or continue treatment with an approved exon skipping therapy
in the GalaxyDMD trial. The GalaxyDMD trial has a streamlined
schedule with visits to trial sites every six months and is
designed to collect long-term safety data to support registration
filings. The trial is also monitoring assessment of muscle function
and bone health.
About Edasalonexent (CAT-1004) Edasalonexent (CAT-1004)
is an investigational oral small molecule that is being developed
as a potential therapy for all patients affected by DMD, regardless
of their underlying mutation. Edasalonexent inhibits NF-kB, which
is a key link between loss of dystrophin and disease progression in
DMD. NF-kB has a fundamental role in skeletal and cardiac muscle
disease in DMD. 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 Phase 3 PolarisDMD Trial The global Phase 3
PolarisDMD trial is a one-year, randomized, double-blind,
placebo-controlled trial evaluating the efficacy and safety of
edasalonexent in patients with DMD. Catabasis plans to enroll
approximately 125 patients ages 4 to 7 (up to 8th birthday)
regardless of mutation type who have not been on steroids for at
least 6 months. Boys on a stable dose of eteplirsen may be eligible
to enroll. The primary efficacy endpoint is change in the North
Star Ambulatory Assessment score after 12 months of treatment with
edasalonexent compared to placebo. Key secondary endpoints include
the age-appropriate timed function tests: time to stand, 4-stair
climb and 10-meter walk/run. Assessments of growth, cardiac and
bone health are also included as important potential areas of
differentiation. Two boys are receiving 100 mg/kg/day of
edasalonexent for each boy that receives placebo, and, after 12
months, all boys are expected to receive edasalonexent in the
open-label extension study GalaxyDMD. The PolarisDMD trial design
was informed by discussions with regulators as well as input from
treating physicians, patient organizations and families of boys
affected by Duchenne. Top-line results from the Phase 3 PolarisDMD
trial are expected in the second half of 2020. More information
about the Phase 3 PolarisDMD clinical trial is available on
clinicaltrials.gov and contact the team with any questions at
DMDtrials@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. Our
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.
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 completion of enrollment and 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 March 31, 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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Investor and Media Contact Andrea
Matthews Catabasis Pharmaceuticals, Inc. T: (617) 349-1971
amatthews@catabasis.com
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