Catabasis Pharmaceuticals Presents Positive Data from Part A of the MoveDMD® Trial of Edasalonexent (CAT-1004), a Potential ...
October 06 2016 - 08:00AM
Business Wire
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today announced that Catabasis is
presenting positive data from Part A of the MoveDMD trial of
edasalonexent for the treatment of Duchenne muscular dystrophy
(DMD) at the World Muscle Society Congress. The World Muscle
Society Congress is being held October 4 – October 8, 2016, in
Granada, Spain, at the Palacio de Congresos de Granada.
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 (DMD)” and
Pradeep Bista, Ph.D., Principal Scientist at Catabasis, will
present the poster “Serum pro-inflammatory proteins have potential
utility as biomarkers for NF-kB targeting approaches in DMD.” Both
poster presentations will take place in Poster Presentations 3:
Parallel Sessions today from 3:00pm – 4:30pm local time in the
Poster area of the Palacio de Congresos de Granada.
Catabasis has previously announced positive biomarker results
from Part A of the MoveDMD clinical trial demonstrating successful
NF-kB target engagement and detailed data are being presented at
the World Muscle Society Congress. After one week of dosing with
edasalonexent in 4-7-year-old boys affected by DMD, a pre-selected
NF-kB gene set was significantly inhibited in whole blood mRNA at
each of the two higher doses (67 mg/kg and 100 mg/kg). The
evaluated gene set is 200 expressed genes known to be regulated by
NF-kB that was curated by the Broad Institute. This gene set was
significantly decreased when compared to all other expressed genes.
In addition to gene expression, serum proteins were also evaluated.
In the combined group of patients on 67 mg/kg and 100 mg/kg, levels
of NF-kB-regulated proteins, IL-12 and osteopontin, were
significantly reduced following 7 days of dosing with
edasalonexent. These data further support the ongoing evaluation of
the 67 and 100 mg/kg edasalonexent doses in the Phase 2 MoveDMD
clinical trial.
Baseline data for boys from Part A of the trial who are also
participating in Part B are being presented at the World Muscle
Society Congress as well. Timed function tests were assessed before
the boys had started dosing with edasalonexent in Part A of the
trial as well as at baseline before they started dosing with
edasalonexent in Part B of the trial. For all three of the timed
function tests that we are evaluating, 10-meter walk/run, 4-step
climb and time-to-stand, there was an increase in the average time
to complete the tests from Part A baseline to Part B baseline. The
time elapsed between Part A baseline and Part B baseline ranged
from 4 to 11 months for these 15 boys. Although the MoveDMD trial
is not powered to show statistically significant changes in these
three timed function tests in Part B, the observations provide data
on disease progression within the MoveDMD trial that shows a
decline in function when these boys are predominantly not on
treatment and may provide additional context for assessing these
secondary end points during the on-treatment part of the trial. The
primary end point of Part B of the MoveDMD trial is change in MRI
T2 signal of the lower leg muscles for edasalonexent as compared
with placebo.
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 MRIMagnetic 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 CatabasisAt 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.
Forward Looking StatementsAny statements in this press
release about future expectations, plans and prospects for the
Company, including statements about future clinical trial plans 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; availability and timing of results from
preclinical studies and clinical trials; 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;
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 period ended June 30, 2016, which is on file with
the Securities and Exchange Commission, and in other filings that
the Company may make with the Securities and Exchange Commission in
the future. In addition, the forward-looking statements included in
this press release represent the Company’s views as of the date of
this press release. The Company anticipates that subsequent events
and developments will cause the Company’s views to change. However,
while the Company may elect to update these forward-looking
statements at some point in the future, the Company specifically
disclaims any obligation to do so. These forward-looking statements
should not be relied upon as representing the Company’s views as of
any date subsequent to the date of this release.
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version on businesswire.com: http://www.businesswire.com/news/home/20161006005194/en/
Catabasis Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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