CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that Catabasis will present the MoveDMD trial of edasalonexent for the treatment of Duchenne muscular dystrophy (DMD) at the American Neurological Association’s (ANA) 2016 Annual Meeting. The ANA 2016 Annual Meeting is being held October 16 – October 18, 2016, in Baltimore, MD, at the Baltimore Marriott Waterfront Hotel.
Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis, will present the poster “CAT-1004, an oral agent targeting NF-kB: MoveDMD trial results in Duchenne muscular dystrophy.” The presentation will take place in Poster Session II and Wine & Cheese Reception on Monday, October 17, from 5:30pm – 7:00pm local time in the Harborside Ballroom, Fourth Floor, of the Baltimore Marriott Waterfront Hotel.
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 their 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 produced beneficial
effects in skeletal, diaphragm and cardiac muscle and improved function.
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 we observed
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®
The MoveDMD trial is a
three-part clinical trial investigating the safety and efficacy of
edasalonexent in boys ages 4 – 7 affected with DMD (any confirmed
mutation). Part A of the MoveDMD trial evaluated the safety,
tolerability and pharmacokinetics of, and NF-kB target engagement
with, edasalonexent and showed positive results. Sixteen of the 17 boys
enrolled in Part A continued to Part B of the trial, which is a Phase 2
trial to evaluate the safety and efficacy of edasalonexent in DMD over a
12-week period in approximately 30 boys. The primary end point is change
in MRI of the lower 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 instrument (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 (Part B) 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.