-- Edasalonexent Substantially Slowed Duchenne
Muscular Dystrophy Disease Progression through 36 Weeks --
-- Improvements Across Multiple Assessments of
Muscle Function and Measures of Muscle Health --
-- Conference Call October 4 at 8:30am ET
--
Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage
biopharmaceutical company, today reported new positive efficacy
results showing sustained disease-modifying effects in the MoveDMD
trial open-label extension following 24 and 36 weeks of treatment
with edasalonexent. Across all key assessments of muscle function,
improvements were observed in the rate of decline after 24 and 36
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. These data provide clinically meaningful
evidence that edasalonexent substantially slowed the progression of
Duchenne muscular dystrophy (DMD).
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Additionally, supportive changes in measures of muscle health
were seen, consistent with positive edasalonexent treatment
effects. Muscle enzymes significantly decreased compared to
baseline at 12 weeks and later time points (p<0.05) and lower
leg muscle MRI T2 rate of change was significantly improved in
comparison to progression during the control period (p≤0.05).
Edasalonexent continued to be well tolerated with no safety signals
observed in the trial.
Based on the consistency of the MoveDMD results and supportive
regulatory input from FDA, Catabasis plans to initiate a single
global Phase 3 trial with edasalonexent in patients with DMD
regardless of mutation type in the first half of 2018 with top-line
results expected in 2020. The data were presented today in a late
breaking session at the World Muscle Society Conference and will be
discussed, along with the Phase 3 clinical trial plan, on a
conference call today, October 4, 2017.
“We are extremely excited to see edasalonexent change the
trajectory of disease in the MoveDMD trial with substantially
slowed disease progression,” said Jill C. Milne, Ph.D., Chief
Executive Officer of Catabasis. “Boys treated with edasalonexent
stabilized; they experienced meaningful improvements in muscle
function compared to the rates of change observed during the
control period. Importantly, other supportive positive measures of
muscle health were observed. We look forward to advancing
edasalonexent as a disease-modifying therapy in a single Phase 3
pivotal trial as soon as possible with the goal of providing a
meaningful impact on disease progression for all boys affected by
Duchenne.”
“Our goal in treating boys with Duchenne is to slow the
progression of the disease. It is tremendously encouraging to see
boys taking edasalonexent stabilize in their functional abilities
and MRI T2 measures along with its continued safety profile,” said
Richard Finkel, M.D., Chief, Division of Neurology, Department of
Pediatrics at Nemours Children’s Health System and a Principal
Investigator for the study. “I look forward to continuing to
investigate edasalonexent as a potential therapy for the many boys
affected by this devastating disease.”
In the MoveDMD trial, a substantial slowing of the disease
progression of DMD was seen in boys treated with edasalonexent
compared to the rates of change during the control period. Through
36 weeks of treatment, the 100 mg/kg/day treatment group showed
clinically meaningful numerical improvements in rates of decline
compared to rates of change during the control period across all
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. Control
period changes were measured prior to boys receiving edasalonexent,
either prior to Phase 2 or in the placebo group, for time periods
averaging 39 weeks. 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. At the time of the
open-label extension data analysis, all 14 boys continuing to
participate had received 100 mg/kg/day for 24 weeks and 11 had
completed 36 weeks of 100 mg/kg/day edasalonexent treatment.
Results are detailed for the 100 mg/kg/day treatment group as all
boys are now taking this dose in the open-label extension.
Additional supportive measures of muscle health were also
consistent with a positive edasalonexent treatment effect. Four
muscle enzymes (creatine kinase, alanine aminotransferase,
aspartate aminotransferase and lactate dehydrogenase) were
significantly decreased compared to baseline following
edasalonexent treatment at 12 weeks and later time points
(p<0.05), consistent with the ability to slow muscle
degeneration and improve muscle integrity. Rate of change in lower
leg MRI T2 significantly improved at 12 weeks and at last
observation on treatment compared to control period (p≤0.05),
consistent with a reduction of inflammation in the muscle.
Edasalonexent continued to be well tolerated with no safety
signals observed to date in the MoveDMD trial. The majority of
adverse events (AEs) have been mild in nature with no serious AEs.
There have been no dose reductions and no drug-related
discontinuations. The most common AEs were gastrointestinal,
primarily mild and transient diarrhea. Height, weight and BMI
growth patterns were similar to standard growth curves for
unaffected boys in the age range of MoveDMD subjects. Boys with DMD
in this age range typically have resting tachycardia, a heart rate
that exceeds the normal resting rate, and heart rate of the boys
treated with edasalonexent decreased toward age-normative values
during treatment. We believe these clinical heart rate observations
are intriguing and warrant follow-up.
Catabasis plans to commence a single global Phase 3 trial in DMD
in the first half of 2018 to evaluate the efficacy and safety of
edasalonexent for registration purposes. The planned design of the
randomized, double-blind, placebo-controlled trial is informed by
discussions with FDA. Catabasis plans for the Phase 3 trial to have
many elements in common with the Phase 2 trial including the
patient population and endpoints. The trial is anticipated to
enroll approximately 125 patients ages 4 to 7 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 are planned to include age-appropriate timed
function tests. Catabasis expects to report top-line results from
this trial in 2020.
“We are excited to see positive effects on muscle function with
edasalonexent, as we know from research that effects on muscle
function are the most important aspect of a therapy for Duchenne
for the affected patients and their families,” said Pat Furlong,
Founding President and Chief Executive Officer of Parent Project
Muscular Dystrophy (PPMD). “We look forward to learning more about
edasalonexent as Catabasis begins its Phase 3 trial. With the
disease-modifying effects and safety and tolerability profile
observed to date for edasalonexent, it has the potential to be a
foundational therapy for all people affected by Duchenne.”
Conference Call and Webcast
Catabasis will host a conference call and webcast today, October
4, 2017, at 8:30am ET to discuss the open-label extension results
from the MoveDMD trial and the Phase 3 clinical trial plan for
edasalonexent.
Participant Toll-Free Dial-In Number:
(877) 388-2733
Participant International Dial-In Number: (541)
797-2984
Pass Code:
90435261
Please specify to the operator that you would like to join the
“Catabasis MoveDMD Results Call.”
Interested parties may access a live audio webcast of the
conference call via the investor section of the Catabasis website,
www.catabasis.com. Please connect to the Catabasis website several
minutes prior to the start of the broadcast to ensure adequate time
for any software download that may be necessary. The webcast will
be archived for 90 days.
About the MoveDMD Phase 2 Trial
The MoveDMD trial included a randomized, double-blind,
placebo-controlled Phase 2 trial with 31 ambulatory boys enrolled
between ages 4 and 7 years with a genetically confirmed diagnosis
of DMD across a range of dystrophin mutations. The boys were all
steroid naive. The open-label extension was initiated in July 2016
and is evaluating longer term safety and efficacy with the same
clinical endpoints as the placebo-controlled portion. Based on
Phase 2 results, all boys who were on the lower dose (67 mg/kg/day)
were transitioned to the higher dose (100 mg/kg/day) because a
greater treatment effect was observed with the higher dose,
consistent with a dose response. Catabasis expects to submit
additional data for presentation to scientific meetings in
2018.
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. We are currently
conducting the MoveDMD trial, a three-part clinical trial
investigating the safety and efficacy of edasalonexent in boys
enrolled at ages 4 – 7 affected with DMD (any confirmed mutation).
The third part of the trial, an open-label extension with
edasalonexent, is ongoing. 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 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
LinkerSM 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 Statements
Any 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 our plans to commence a single global Phase 3
trial in DMD in the first half of 2018 to evaluate the efficacy and
safety of edasalonexent for registration purposes, our plans to
report top-line results from this trial in 2020 and our plans to
continue to evaluate data from the open-label extension of our
MoveDMD® clinical trial of edasalonexent for the treatment of DMD,
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, including the final trial design
of our planned Phase 3 trial in DMD; availability and timing of
results from preclinical studies and clinical trials, including the
availability of top-line results from our planned Phase 3 trial in
DMD in 2020; 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; our ability to obtain financing on
acceptable terms and in a timely manner to fund our planned Phase 3
trial in DMD to evaluate the efficacy and safety of edasalonexent
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
Quarterly Report on Form 10-Q for the period ended June 30, 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
Catabasis Pharmaceuticals (NASDAQ:CATB)
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