CAMBRIDGE, Mass.--(BUSINESS WIRE)--Mar. 13, 2017--
Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced that it will present data
from Part B of the MoveDMD trial of edasalonexent (CAT-1004) for the
treatment of Duchenne muscular dystrophy (DMD) at the 2017 Muscular
Dystrophy Association Scientific Conference to be held March 19 – 22,
2017, in Arlington, VA, at the Hyatt Regency Crystal City.
Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis, will
deliver oral and poster presentations titled “MoveDMD: Phase 1/2 trial
of Edasalonexent, an NF-kB Inhibitor, in 4- to 7-Year-Old Patients with
Duchenne Muscular Dystrophy.” The oral presentation will take place on
Wednesday, March 22, 2017 from 11:15am – 11:45am ET. The poster
presentations will take place during the poster sessions on Monday,
March 20, 2017 from 6:45pm – 8:45pm and Tuesday, March 21, 2017 from
6:30pm – 8:30pm ET.
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
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, 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 trial evaluated the safety, tolerability and pharmacokinetics of,
and NF-kB target engagement with, edasalonexent in 17 boys with DMD.
Part B of the trial was a double-blind, placebo-controlled evaluation of
the safety and efficacy of edasalonexent over a 12-week period in 31
boys. The primary efficacy end point for Part B was average change from
baseline to week 12 in MRI T2 measures in boys given edasalonexent
compared to placebo. Additional efficacy end points included
age-appropriate timed function tests (10-meter walk/run, 4-stair climb
and time to stand), North Star Ambulatory Assessment (NSAA), the
pediatric outcomes data collection instrument (PODCI) and muscle
strength. Part C is an open-label extension with edasalonexent for 36
weeks beyond Part B and will evaluate longer term safety and efficacy
with the same clinical end points as Part B. From the MoveDMD trial, we
have reported that edasalonexent was well tolerated with no safety
signals. We reported top-line data for Part B that the primary efficacy
end point was not met. The edasalonexent 100 mg/kg/day treatment group
consistently showed numerical improvement versus placebo across multiple
timed function tests and the NSAA, although as expected the changes were
not statistically significant. The 67 mg/kg/day treatment group had
mixed results compared with both the 100 mg/kg/day treatment group and
placebo, which in each case were not statistically significant. Part C
of the trial is ongoing.
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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Source: Catabasis Pharmaceuticals, Inc.
Corporate and Media Contact
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com
