Catabasis Pharmaceuticals Reports Positive Results from Open-Label Extension of Phase 2 MoveDMD® Trial Evaluating Edasalonexent in Duchenne Muscular Dystrophy and Plans to Initiate Phase 3 Clinical Trial in First Half 2018
-- Edasalonexent Substantially Slowed Duchenne Muscular Dystrophy Disease Progression through 36 Weeks --
-- Improvements Across Multiple Assessments of Muscle Function and
Measures of
-- Conference Call
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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
“We are extremely excited to see edasalonexent change the trajectory of
disease in the MoveDMD trial with substantially slowed disease
progression,” said
“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
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
“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
Conference Call and Webcast
Catabasis will host a conference call and webcast today,
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
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
About Catabasis
At
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
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Source:
Investor and Media Contact
Catabasis
Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com