Catabasis Pharmaceuticals Announces Plans for Edasalonexent Phase 3 POLARIS DMD Trial in Duchenne Muscular Dystrophy
-- 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 --
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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
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
“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
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
About Catabasis
At
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 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
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Source:
Investor and Media Contact
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com