Catabasis Pharmaceuticals Presents Data Supporting MRI T2 as a Potential Marker of Clinical Outcome in Duchenne Muscular Dystrophy at the World Muscle Society Congress
Business Wire 3-Oct-2018 8:00 AM
-- Data from Large Natural History Study Show Strong Correlation of
Lower Leg Composite MRI T2 with DMD Disease Progression, Supporting
Positive Edasalonexent Effects in the MoveDMD®
Trial --
-- Positive MoveDMD Clinical Results Through 72 Weeks of
Edasalonexent Treatment and Phase 3 PolarisDMD Trial Design Also
Presented --
Catabasis
Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported new data supporting magnetic
resonance imaging (MRI) T2 as a potential marker of clinical outcome in
boys with Duchenne Muscular Dystrophy (DMD). Data from ImagingDMD, the
largest natural history database of MRI assessments in boys with DMD,
demonstrate a strong correlation between a composite of lower leg MRI T2
and boys' functional abilities. These results highlight the clinical
importance of the significant improvement observed in the MoveDMD trial
with edasalonexent treatment for lower leg MRI T2 compared to the
off-treatment control period. These data were presented today in the
late breaking session at the 23rd International Congress of
the World Muscle Society in Mendoza, Argentina.
The utility of MRI T2 as a marker to predict disease progression in DMD
was assessed in the ImagingDMD natural history database of more than 150
boys. The lower leg composite MRI T2 was highly correlated with
functional abilities in boys with DMD. The lower leg composite MRI T2
correlated with ability to complete assessments of muscle function, with
2 milliseconds (MRI T2 measurement units) corresponding to clinically
relevant functional changes. The MoveDMD edasalonexent trial also
assessed MRI T2 and saw a statistically significant improvement in the
lower leg composite MRI T2 rate of change following 12, 24, 36 and 48
weeks of edasalonexent treatment compared to the off-treatment control
period, supporting the potential clinical benefit of edasalonexent.
These improvements in MRI T2 are consistent with the improvements
observed in all assessments of muscle function with edasalonexent
treatment compared to the off-treatment control period.
"It is extremely exciting to see the MRI composite correlate with
disease progression in boys with Duchenne in the natural history data as
we have been interested in MRI measures as a potential marker of
clinical outcome in Duchenne to make drug development more efficient,"
said H. Lee Sweeney, Ph.D., Myology Institute Director, University of
Florida and ImagingDMD co-Principal Investigator. "Current assessments
of Duchenne rely on physical tests and invasive procedures. MRI
represents an objective, sensitive and non-invasive way to assess
disease progression in Duchenne and will serve as an incredibly valuable
tool as we look to evaluate and quickly make available new treatment
options for the disease."
"The potential of MRI T2 to predict disease progression and potential
treatment benefit in Duchenne will greatly enhance the ability of
investigators to assess clinical effects of investigational agents and
hopefully lead to improved clinical management of boys living with the
disease," said Joanne Donovan, M.D., Ph.D., Chief Medical Officer of
Catabasis. "This represents a promising step forward to better
understand MRI endpoints and their utility in Duchenne to support our
work to bring hope and life changing therapies to those affected by
Duchenne. We are excited to have recently initiated our Phase 3
PolarisDMD trial for edasalonexent based on the positive results
observed in the MoveDMD trial."
Catabasis also presented new MoveDMD trial data and the Phase 3
PolarisDMD trial design at the International Congress of the World
Muscle Society:
MoveDMD Trial Data through 72 Weeks of Edasalonexent Treatment
-
New positive efficacy and safety results through 72 weeks of
edasalonexent treatment were presented, showing preservation of muscle
function and sustained disease-modifying effects in all assessments of
muscle function in boys with DMD in the MoveDMD trial and open-label
extension compared to the off-treatment control period.
-
Significant decreases in muscle enzymes through 72 weeks were also
seen in boys treated with edasalonexent, supporting the durability of
edasalonexent treatment effects.
-
Significantly decreased heart rate towards age-normative values was
observed and supports the potential beneficial cardiac effects of
edasalonexent. Boys with DMD in this age range typically have resting
tachycardia, a heart rate that exceeds the normal resting rate, and is
the first cardiac manifestation in boys with DMD. Cardiomyopathy is
the leading cause of mortality in DMD.
-
Edasalonexent continued to be well tolerated with no safety signals
observed in the trial. Boys treated with edasalonexent continue to
follow age-appropriate growth curves with age-appropriate increases in
weight and height and overall BMI has trended down to age-normative
values.
PolarisDMD Phase 3 Trial Design for Edasalonexent
-
The recently initiated Phase 3 PolarisDMD trial will evaluate the
efficacy and safety of edasalonexent in patients with DMD and is
intended to support an application for commercial registration of
edasalonexent.
-
The PolarisDMD trial is expected to include approximately 40 clinical
trial sites globally.
-
The Phase 3 PolarisDMD trial is a one-year, randomized, double-blind,
placebo-controlled trial. Catabasis plans to enroll approximately 125
patients ages 4 to 7 (up to 8th birthday) regardless of
mutation type who have not been on steroids for at least 6 months.
-
The primary efficacy endpoint will be change in the North Star
Ambulatory Assessment score after 12 months of treatment with
edasalonexent compared to placebo. Key secondary endpoints include the
age-appropriate timed function tests time to stand, 4-stair climb and
10-meter walk/run. Assessments of growth, cardiac and bone health are
also included.
-
Top-line results from the Phase 3 PolarisDMD trial are expected in the
second quarter of 2020.
About Edasalonexent (CAT-1004)Edasalonexent (CAT-1004) is
an investigational oral small molecule that is being developed as a
potential new standard of care for all patients affected by DMD,
regardless of their underlying mutation. Edasalonexent inhibits NF-kB,
which is the key link between loss of dystrophin and disease pathology
and plays a fundamental role in the initiation and progression of
skeletal and cardiac muscle disease in DMD. Catabasis is currently
enrolling the single global Phase 3 PolarisDMD trial to evaluate the
efficacy and safety of edasalonexent for registration purposes.
Edasalonexent continues to be dosed in an open-label extension of the
MoveDMD Phase 2 clinical trial. 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. For a summary of clinical
results, please visit www.catabasis.com.
About CatabasisAt Catabasis Pharmaceuticals, our mission is
to bring hope and life-changing therapies to patients and their
families. Our lead program is edasalonexent, an NF-kB inhibitor in
development for the treatment of Duchenne muscular dystrophy. The global
Phase 3 PolarisDMD trial is currently enrolling boys affected by
Duchenne. For more information on edasalonexent and our Phase 3
PolarisDMD trial, please visit www.catabasis.com
or www.twitter.com/catabasispharma.
Forward Looking StatementsAny statements in this press
release about future expectations, plans and prospects for the Company,
including statements about future clinical trial plans including, among
other things, statements about the Company's global Phase 3 PolarisDMD
trial in DMD to evaluate the efficacy and safety of edasalonexent for
registration purposes, 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; 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
quarter ended June 30, 2018, 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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