CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced the initiation of an open-label extension for the Phase 2 portion (Part B) of the MoveDMD trial studying edasalonexent (CAT-1004), an investigational therapy, in boys with Duchenne muscular dystrophy (DMD). The Phase 2 portion of the MoveDMD trial is a randomized, double-blind, placebo-controlled 12-week trial to assess the efficacy and safety of two doses of oral edasalonexent with the primary end point being change in magnetic resonance imaging (MRI). Top-line results are expected late 2016. In the open-label extension, all patients will receive edasalonexent for 36 weeks beyond the 12-week placebo-controlled period. During the open-label extension, safety will be monitored and additional assessments including MRI, timed function tests, muscle strength measures, the North Star Ambulatory Assessment and the pediatric outcomes data collection instrument (PODCI) will be performed. All patients who complete the 12-week Phase 2 portion of the MoveDMD trial will be eligible to enroll in the open-label extension. Catabasis has previously reported favorable safety, tolerability and pharmacokinetics as well as positive biomarker results demonstrating NF-kB target engagement from the initial 7-day dose-ranging portion of the MoveDMD trial.
“We are pleased to extend edasalonexent dosing in the MoveDMD trial based on the acceptable safety and tolerability data seen to date,” said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis. “The MoveDMD open-label extension is expected to inform on safety and efficacy of edasalonexent when administered for up to 48 weeks, and is important to our overall development strategy. We very much appreciate the support that we have received from the DMD community for the MoveDMD trial.”
“I am pleased to see the progress with this clinical trial and to offer additional dosing of this novel investigational therapy to boys affected by Duchenne when they complete the 12-week placebo-controlled portion of the trial,” said Erika Finanger, M.D., Assistant Professor, Pediatrics & Neurology, Oregon Health & Sciences University and Consulting Neurologist, Shriners Hospitals for Children and a principal investigator in the MoveDMD trial. “We need therapies that have the potential to make a meaningful difference in boys affected by DMD regardless of the underlying mutation.”
In the first portion of the MoveDMD trial (Part A), 17 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations received edasalonexent. The boys were steroid naive or had not used steroids for at least six months prior to the trial. This portion of the trial was conducted at three sites in the U.S., and assessed the safety, tolerability and pharmacokinetics of edasalonexent in patients at three dosing levels (33 mg/kg/day, 67 mg/kg/day and 100 mg/kg/day) during seven days of dosing. Phase 2 of the MoveDMD trial (Part B) is a randomized, double-blind, placebo-controlled trial to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week period at 5 clinical trial sites in the U.S. at two dosing levels, 67 mg/kg/day and 100 mg/kg/day. The boys that participated in the first part of the MoveDMD trial that remain eligible are asked to participate in Phase 2 and additional participants are expected to be enrolled for a total of approximately 30 boys. The open-label extension (Part C) includes dosing with edasalonexent for 36 weeks beyond the 12-week placebo-controlled portion of the trial and will evaluate longer term safety and efficacy with the same clinical end points. We are currently identifying additional patients who are interested in participating in the Phase 2 trial. Entry criteria are similar to those in the first portion of the trial. The Parent Project Muscular Dystrophy and the Muscular Dystrophy Association are providing funding to support participant travel for the MoveDMD trial.
More information about the MoveDMD trial can be found on the clinical trials page of the Catabasis website and on ClinicalTrials.gov under trial identifier NCT02439216.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is
an oral small molecule that has the potential to be a disease-modifying
therapy for all patients affected by Duchenne muscular dystrophy (DMD or
Duchenne), regardless of the underlying mutation. Edasalonexent inhibits
NF-kB, a protein that is activated in Duchenne and drives inflammation
and fibrosis, muscle degeneration and suppresses muscle regeneration. In
animal models of DMD, edasalonexent inhibited NF-kB, reduced muscle
degeneration and improved muscle regeneration and function, and
beneficial effects were observed in skeletal, diaphragm and cardiac
muscle. 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.
We have previously reported safety, tolerability and reduction in NF-kB
activity in Phase 1 trials in adults. We are currently conducting the
MoveDMD® trial of edasalonexent in 4-7 year-old boys affected
by Duchenne. From Part A of the MoveDMD trial, we have reported that
edasalonexent was generally well tolerated with no safety signals
observed and successful NF-kB target engagement. Pharmacokinetic results
demonstrated edasalonexent plasma exposure levels consistent with those
previously observed in adults at which inhibition of NF-kB was observed.
About MoveDMD®
MoveDMD is a Phase 1 / 2
clinical trial of edasalonexent (CAT-1004) in boys ages 4-7 affected
with DMD (any confirmed mutation). The MoveDMD trial is a three-part
clinical trial investigating the safety and efficacy of edasalonexent in
DMD. Part A of the MoveDMD trial evaluated the safety, tolerability and
pharmacokinetics of, and NF-kB target engagement with,
edasalonexent and showed positive results. The boys in Part A of the
trial are asked to participate, if eligible, in Part B of the trial.
Part B of the trial is a Phase 2 trial to evaluate the safety and
efficacy of edasalonexent in DMD over a 12-week period and will enroll
approximately 30 boys. The primary end point is changes in MRI of the
leg muscles, and the secondary end points are age-appropriate timed
function tests: 10-meter walk/run, 4-stair climb and time to stand.
Additional assessments include muscle strength, the North Star
Ambulatory Assessment and the pediatric outcomes data collection tool
(PODCI). Part C is an open-label extension that includes dosing with
edasalonexent for 36 weeks beyond the 12-week placebo-controlled portion
of the trial and will evaluate longer term safety and efficacy with the
same clinical end points as Part B.
About MRI
Magnetic resonance imaging (MRI) is a non-invasive
imaging technique that can assess muscle structure and composition and
measure disease status in children with DMD. Two MRI measures used in
Duchenne to indicate muscle degeneration are T2 and fat fraction. MRI is
sensitive to changes in muscle structure and composition induced by
disease processes such as the inflammation, edema, muscle damage and fat
infiltration that occur in Duchenne. Changes in T2 may be seen in less
than 12 weeks while changes in fat fraction may take longer. Changes in
these MRI measures have been correlated with longer-term changes in
clinically meaningful measures of functional activity. Changes in MRI
can show the effects of an investigational therapy on disease
progression in Duchenne in an objective and quantifiable manner.
About Catabasis
At Catabasis Pharmaceuticals, our mission is
to bring hope and life-changing therapies to patients and their
families. Our SMART (Safely Metabolized And Rationally Targeted) linker
drug discovery platform enables us to engineer molecules that
simultaneously modulate multiple targets in a disease. We are applying
our SMART linker platform to build an internal pipeline of product
candidates for rare diseases and plan to pursue partnerships to develop
additional product candidates. For more information on the Company's
drug discovery platform and pipeline of drug candidates, please visit www.catabasis.com.
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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 period ended March 31,
2016, which is on file with the Securities and Exchange Commission, and
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