Catabasis Pharmaceuticals Reports Edasalonexent Preserved Muscle Function and Substantially Slowed Duchenne Muscular Dystrophy Disease Progression Through More Than One Year of Treatment
-- Consistent Improvements Sustained Across All Assessments of Muscle Function Through 48 and 60 Weeks of Edasalonexent Treatment in MoveDMD® Trial --
-- Single Global Phase 3 Trial Expected to Begin in First Half of 2018 --
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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. Edasalonexent is a potential oral foundational therapy that is being developed for all patients affected by DMD.
“We are thrilled to see this preservation of muscle function and
substantial slowing of disease progression in boys following more than a
year of edasalonexent treatment. This effect has the potential to be
extremely impactful for boys affected by Duchenne,” said
“As this study matures and we see the effect of edasalonexent treatment
through 48 and 60 weeks, it is compelling that all of the assessments of
muscle function are demonstrating stabilization at an age when boys with
DMD have a predictable decline,” said
In Phase 2 and the open-label extension of the MoveDMD trial, 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 60 weeks 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
Additional supportive measures of muscle health also reinforce positive edasalonexent treatment effects in the 100 mg/kg/day treatment group. 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 through 60 weeks (p<0.05), consistent with the ability to slow muscle degeneration and improve muscle integrity. Biomarker results showed that CRP was significantly decreased with edasalonexent at 12, 24, 36 and 48 weeks compared to baseline in the 100 mg/kg/day treatment group (p≤0.001). CRP is a well-characterized blood test marker that provides a global assessment of inflammation, and CRP is elevated in boys affected by DMD. The significant decrease observed in CRP supports the biological activity of NF-kB inhibition by edasalonexent treatment decreasing inflammation.
Edasalonexent continued to be well tolerated with no clinical safety signals observed to date. The majority of adverse events (AEs) have been mild in nature with no serious AEs. The most common related AEs were gastrointestinal, primarily mild and transient diarrhea. Boys with DMD in this age range typically have resting tachycardia, a heart rate that exceeds the normal resting rate, and the heart rate of the boys treated with edasalonexent decreased toward age-normative values during treatment.
Catabasis plans to initiate 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 design of the randomized, double-blind,
placebo-controlled trial has been informed by discussions with
About the MoveDMD Trial
The 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 through 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 across available non-effort based measures of muscle health. 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 was well tolerated with no safety signals observed in the trial. Catabasis expects to present additional data at 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. Edasalonexent continues to be dosed in the open-label extension of the MoveDMD Phase 2 clinical trial and Catabasis plans to initiate a single global Phase 3 trial to evaluate the efficacy and safety of edasalonexent for registration purposes in the first half of 2018. The
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 the Company’s 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 Company’s plans to report top-line results from this trial in 2020, 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 the Company’s 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 the Company’s 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; 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 Quarterly Report on Form 10-Q for the period ended
Investor and Media Contact
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971