Catabasis Pharmaceuticals Announces Plans for Edasalonexent Phase 3 POLARIS DMD Trial in Duchenne Muscular Dystrophy
July 09 2018 - 8:00AM
Business Wire
-- Global Phase 3 POLARIS DMD Trial Expected to
Initiate in the Second Half of 2018 and Enroll Approximately 125
Patients --
-- Phase 2 MoveDMD® Trial and Open-Label
Extension Showed Substantially Slowed Duchenne Disease Progression
in Patients Treated with Edasalonexent --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced plans for the Phase 3
POLARIS DMD trial with edasalonexent in patients with Duchenne
muscular dystrophy (DMD). Catabasis plans to initiate the global
POLARIS DMD trial in the second half of 2018 with top-line results
expected in the second quarter of 2020.
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The POLARIS DMD trial will evaluate the efficacy and safety of
edasalonexent in patients with DMD and is intended to support an
application for commercial registration of edasalonexent. The trial
design was informed by discussions with the U.S. Food and Drug
Administration (FDA) as well as input from treating physicians and
families of boys affected by Duchenne.
The randomized, double-blind, placebo-controlled POLARIS DMD
trial has many key elements in common with the Phase 2 MoveDMD®
trial, including the patient population and functional endpoints.
Catabasis anticipates enrolling approximately 125 patients between
the ages of 4 and 7 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
will be change in the North Star Ambulatory Assessment score after
12 months of treatment with edasalonexent compared to placebo. Key
secondary endpoints are planned to 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
planned to be included. Two boys will receive edasalonexent for
every boy that receives placebo and after 12 months, all boys are
expected to receive edasalonexent in an open-label extension.
“We have designed a robust study with POLARIS DMD to evaluate
edasalonexent as a potential new treatment for Duchenne. We have
benefited from input from many people that are part of the Duchenne
community and we are well underway with our preparations to begin
the trial,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer
of Catabasis. “We are very excited to advance edasalonexent through
this potentially last phase of clinical development with the hope
of providing a new treatment option to all boys affected by this
disease. We believe that edasalonexent has great potential as a
therapy to be taken on its own as well as in combination with other
treatments.”
Edasalonexent is a potential oral foundational therapy that is
being developed for all patients affected by DMD. Edasalonexent is
being developed for use as monotherapy and in possible combination
with dystrophin upregulation therapies. Edasalonexent has been
shown to preserve muscle function and substantially slow Duchenne
disease progression in the MoveDMD Phase 2 trial and open-label
extension. Preclinical data and clinical biomarker data from the
MoveDMD Phase 2 trial suggest that edasalonexent could have
potential benefits in skeletal muscle, diaphragm and heart.
Edasalonexent has been safe and well tolerated through more than 45
patient-years of treatment.
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, fibrosis and muscle degeneration and suppresses
muscle regeneration. Edasalonexent continues to be dosed in an
open-label extension of the MoveDMD Phase 2 clinical trial, and
Catabasis is preparing to initiate a single global Phase 3 trial in
the second half of 2018 to evaluate the efficacy and safety of
edasalonexent for registration purposes. 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 lead program is edasalonexent, an NF-kB inhibitor in
development for the treatment of Duchenne muscular dystrophy.
Edasalonexent was designed using our SMART (Safely Metabolized And
Rationally Targeted) Linker drug discovery platform that enables us
to engineer molecules that simultaneously modulate multiple targets
in a disease. For more information on edasalonexent or our drug
discovery platform, 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
including, among other things, statements about the Company’s plans
to commence a single global Phase 3 trial in DMD to evaluate the
efficacy and safety of edasalonexent for registration purposes, 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; the Company’s ability to obtain
financing on acceptable terms and in a timely manner to fund the
Company’s planned Phase 3 trial of edasalonexent in DMD for
registration purposes; 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 quarter ended March 31, 2018,
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 ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
Catabasis Pharmaceuticals (NASDAQ:CATB)
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