Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company focused on bringing transformative therapies
to patients with rare diseases, today announced that Catabasis will
present CAT-1004 for the treatment of Duchenne muscular dystrophy (DMD)
and the MoveDMDSM trial design at the XIV International
Conference on Duchenne and Becker Muscular Dystrophy. The conference
will be held February 13 – 14, 2016, in Rome, Italy, at the Church
Palace.
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Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis, will
give a presentation titled “CAT-1004, an Oral Agent Targeting NF-kB in
Development for Treatment of Duchenne Muscular Dystrophy.” The
presentation will take place as part of a session on Sunday, February
14, 2016, from 8:40am – 11:00am local time.
About CAT-1004
CAT-1004 is an oral small molecule that has
the potential to be a disease-modifying therapy for all patients
affected with Duchenne muscular dystrophy (DMD or Duchenne), regardless
of the underlying mutation. CAT-1004 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, CAT-1004 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
CAT-1004 for the treatment of DMD. We are currently conducting the
MoveDMDSM trial of CAT-1004 in 4-7 year-old boys affected by
Duchenne and have previously reported safety, tolerability and reduction
in NF-kB activity in Phase1 trials.
About MoveDMD
MoveDMDSM is a Phase 1 / 2 clinical
trial of CAT-1004 in boys ages 4-7 affected with DMD (any confirmed
mutation). The MoveDMD trial is a two-part clinical trial investigating
the safety and efficacy of CAT-1004 in DMD. Part A of the MoveDMD trial
evaluated the safety, tolerability and pharmacokinetics of CAT-1004. In
addition, the Company collected data at baseline on the muscles of the
lower and upper legs using MRI, physical function (including timed
function tests), and muscle strength. The boys in Part A of the trial
will be asked to participate, if eligible, in Part B of the trial. Part
B of the trial will be planned to evaluate the safety and efficacy of
CAT-1004 in DMD over a 12-week treatment period. Additional details of
Part B of the trial will be available once the protocol is finalized.
About MRI
Magnetic resonance imaging (MRI) is a non-invasive
imaging technique that can visualize 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
when used in conjunction with clinical measures 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 corporate focus is to discover, develop and commercialize
transformative therapies for rare diseases. 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 in rare diseases and aim to
target broader disease opportunities via external collaborations. For
more information on the Company's drug discovery platform and pipeline
of drug candidates, please visit www.catabasis.com.
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