Catabasis Pharmaceuticals Presents New Edasalonexent Data Showing Significantly Slowed Duchenne Muscular Dystrophy Disease Pr...
April 25 2018 - 5:00PM
Business Wire
-- Statistically Significant Improvements in
MRI T2 Rate of Change Compared to Control Consistent with
Improvements Demonstrated in Assessments of Muscle Function in
MoveDMD® Trial --
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported new positive magnetic
resonance efficacy results showing slowed disease progression in
boys with Duchenne muscular dystrophy (DMD) in the MoveDMD trial
through 48 weeks of edasalonexent treatment. Both magnetic
resonance imaging (MRI) and magnetic resonance spectroscopy (MRS)
were performed in the Phase 2 MoveDMD trial and open-label
extension. Statistically significant improvement in the rate of
change in lower leg composite MRI T2 at 12, 24, 36 and 48 weeks on
oral 100 mg/kg of edasalonexent treatment were observed compared to
the off-treatment control period (p<0.05 for all time points).
Improvements in changes in both soleus and vastus lateralis (VL)
MRS fat fraction through 48 weeks of edasalonexent treatment
compared to the off-treatment control period were also
demonstrated. These data were presented today at the American
Academy of Neurology 70th Annual meeting in Los Angeles, CA. These
improvements in MRI T2 and MRS fat fraction show a slowing of
disease progression and are in addition to the improvements
observed in all assessments of muscle function through more than a
year of edasalonexent treatment.
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“The results reported to date from the MoveDMD trial have been
consistent and support edasalonexent as a disease-modifying
therapy. Overall, in the MoveDMD trial edasalonexent has slowed
progression of this disease based on improvements in multiple
assessments of physical function and biomarkers of muscle health
and inflammation,” said Jill C. Milne, Ph.D., Chief Executive
Officer of Catabasis. “We believe that these effects
ultimately will translate to boys with Duchenne maintaining
functional abilities longer.”
“I am very encouraged to see these emerging data with favorable
MRI changes and results of muscle function assessments with
edasalonexent, which are clearly divergent from the untreated
disease progression that I see in boys in this age range with
Duchenne,” said Richard Finkel, M.D., Chief, Division of Neurology,
Department of Pediatrics at Nemours Children’s Hospital, Orlando
and a Principal Investigator for the study. “There is a strong need
for a therapy with a safety and tolerability profile like
edasalonexent for the many boys affected by this devastating
disease.”
MRI is a non-invasive approach to assess disease progression in
DMD. MRI T2 as well as MRS fat fraction and T2 were performed in
the Phase 2 MoveDMD trial and open-label extension. MRI T2 measures
combined inflammation and fat in one measurement. MRS T2 measures
the inflammatory component independently and MRS fat fraction
measures the amount of fat in the muscle. As boys with DMD get
older, the amount of fat in their muscles increases with consequent
loss of functional abilities. Changes in MRI T2 and MRS fat
fraction are known to correlate with changes in function in boys
with DMD. Increases in both measures strongly correlate with worse
performance on timed function tests and predict future loss of
functional abilities. FDA considers MRI to be an important
supportive early endpoint demonstrating therapeutic effect as
mentioned in the recently released FDA guidance on development in
DMD.
Statistically significant improvement in the rate of change in
lower leg composite MRI T2 at 12, 24, 36 and 48 weeks of oral 100
mg/kg of edasalonexent treatment were observed compared to the
off-treatment control period (p<0.05 at all time points). After
48 weeks of 100 mg/kg of edasalonexent treatment, MRS fat fraction
of the soleus muscle increased by an average of 0.85%, while in the
same boys during the off-treatment control period the average
annualized increase was 2.6% per year. Boys from the ImagingDMD
natural history study who were largely on steroids had an increase
in soleus fat fraction of approximately 3% per year. After 48 weeks
of edasalonexent treatment, MRS fat fraction of the VL (one of the
quadriceps) increased by 5.9%, while in the same boys during the
off-treatment control period the average annualized increase was
10.4% per year. Boys from the ImagingDMD natural history comparison
study who were largely on chronic steroids had an increase in VL
fat fraction of approximately 7% per year. MRS T2 of soleus and VL
decreased from baseline. The changes in the rate of increase in fat
fraction in the soleus and VL muscles demonstrate greater stability
with edasalonexent treatment in the MoveDMD trial with less of an
increase in fat in their muscles compared to the same boys prior to
treatment. The increase in fat fraction observed on edasalonexent
treatment was also less than that observed in natural history in a
different population of boys with DMD.
Consistent with these magnetic resonance data, in the Phase 2
MoveDMD trial and open-label extension, a preservation of muscle
function and slowing of DMD disease progression was seen in boys
treated with edasalonexent compared to the rates of change during
the control period prior to receiving edasalonexent. Through a year
of treatment, the 100 mg/kg/day treatment group showed consistent
and clinically meaningful improvements in rates of decline compared
to rates of change during the control period across all four
assessments of muscle function: the three timed function tests
(10-meter walk/run, 4-stair climb and time to stand), as well as
the North Star Ambulatory Assessment (NSAA), an integrated global
assessment of muscle function.
Edasalonexent is being developed as a potential foundational
disease-modifying therapy for all patients affected by DMD,
regardless of their underlying mutation. No evidence of side
effects or safety issues common with the current DMD standard of
care has been observed after more than 37 patient-years of exposure
to edasalonexent. Catabasis is preparing for a single global Phase
3 trial to evaluate the efficacy and safety of edasalonexent for
registration purposes, dependent on raising capital.
About the MoveDMD TrialThe MoveDMD trial is
investigating the safety and efficacy of edasalonexent
in steroid-naïve boys enrolled at ages 4 – 7 affected
with DMD (any confirmed mutation). The trial is comprised of
three parts - Phase 1, Phase 2 and open-label extension. Phase 2
was a randomized, double-blind, placebo-controlled 12-week portion
with 31 ambulatory boys across a range of dystrophin mutations. The
12-week MRI T2 primary endpoint for treated boys compared to
placebo was directionally positive although not statistically
significant. The open-label extension evaluated longer term safety
and efficacy using pre-specified analyses comparing the rates of
change in boys receiving edasalonexent treatment and prior to
treatment. In the Phase 2 and open-label extension of the MoveDMD
trial, edasalonexent substantially slowed DMD disease progression
in boys on 100 mg/kg/day up to 60 weeks of treatment. Across all
assessments of muscle function, consistent improvements were
observed in the rate of decline after 12, 24, 36, 48 and 60 weeks
of oral 100 mg/kg/day edasalonexent treatment compared to the rate
of change in the control period for boys prior to receiving
edasalonexent treatment. Statistically significant improvements
were also seen in non-effort based measures of muscle health
(muscle enzymes and c-reactive protein and MRI T2 compared to
control). In the 100 mg/kg/day treatment group, 16 boys commenced
edasalonexent either at the beginning of Phase 2 or at the
beginning of the open-label extension. Edasalonexent has been well
tolerated with no safety signals observed in the trial.
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
DMD, regardless of their underlying mutation. Edasalonexent
inhibits NF-kB, a protein that is activated in DMD and drives
inflammation and fibrosis, muscle degeneration and suppresses
muscle regeneration. Edasalonexent continues to be dosed in the
open-label extension of the MoveDMD Phase 2 clinical trial and
Catabasis is preparing for a single global Phase 3 trial to
evaluate the efficacy and safety of edasalonexent for registration
purposes, dependent on raising capital. 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 reported to-date, 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 SMART (Safely Metabolized And Rationally Targeted)
Linker drug discovery platform enables us to engineer molecules
that simultaneously modulate multiple targets in a disease. Our
lead program in development is edasalonexent for the treatment of
Duchenne muscular dystrophy. For more information on edasalonexent
and our 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
including, among other things, statements about the Company’s plans
to commence a single global Phase 3 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; the Company’s ability to obtain
financing on acceptable terms and in a timely manner to fund the
Company’s planned Phase 3 trial of edasalonexent in DMD for
registration purposes; 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
Annual Report on Form 10-K for the year ended December 31, 2017,
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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Investor and Media ContactCatabasis
Pharmaceuticals, Inc.Andrea Matthews,
617-349-1971amatthews@catabasis.com
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