Catabasis Pharmaceuticals Announces Top-Line Results for Part B of the MoveDMD® Trial for Edasalonexent (CAT-1004) in Duchenne Muscular Dystrophy
-- Conference Call Today at
Thirty-one boys enrolled in Part B and all completed the trial. Both dose levels of edasalonexent evaluated were well tolerated with no safety signals observed. The majority of adverse events were mild in nature and the most common treatment-related adverse events were gastrointestinal, primarily mild diarrhea and vomiting. There were no treatment-related serious adverse events, no drug discontinuations and no dose reductions. Edasalonexent plasma exposure in Part B of the MoveDMD trial was consistent with that observed in Part A.
Additional efficacy end points included three age-appropriate timed function tests (10-meter walk/run, 4-stair climb and time to stand), the North Star Ambulatory Assessment (NSAA) and additional MRI measures. The trial was not powered to detect statistically significant changes in the timed function tests or NSAA and no significant changes were detected in these measures for those dosed with edasalonexent versus placebo. For all three timed function tests, the change in speed of performance from baseline to week 12 was numerically better for the 100 mg/kg/day treatment group compared to placebo. The change in NSAA was also numerically better for the 100 mg/kg/day treatment group compared to placebo. In addition to the lower leg, MRI T2 was also measured for the upper leg. The results in MRI T2 score for change from baseline to week 12 for the vastus lateralis (a large component of the quadriceps) were not statistically significant but showed a numerical improvement in the 100 mg/kg/day treatment group compared to placebo. Compared to the placebo group, patients in the edasalonexent 100 mg/kg/day group had characteristics of more advanced disease at baseline. Patients in this treatment group had been diagnosed at a younger age and, at baseline, did not perform as well on function tests.
“Although we did not meet the MRI T2 composite end point, the continued
safety, tolerability and plasma exposure data in Part B of the MoveDMD
trial are reassuring. We observed potential treatment-associated effects
at 12 weeks in the 100 mg/kg/day treatment group, which we believe
warrant further evaluation to see if the signals strengthen in the
longer-term data from the ongoing open-label extension. Following
additional data analysis from the open-label extension, we will
determine the next steps for edasalonexent in DMD,” said
“The top-line data results from Part B of the MoveDMD trial provide us
with an early snapshot of the effects of edasalonexent in boys with DMD
over a 12-week period. Continuing the open-label extension of the
MoveDMD trial will allow us to further evaluate the potential for
edasalonexent to provide benefit in DMD,” said
“We in the DMD community continue to learn from each completed clinical
trial and appreciate the effort of Catabasis in studying a potential
therapy that could be applicable for all of those affected by DMD
regardless of the underlying mutation type,” said
About Part B of the MoveDMD Trial
Part B of the MoveDMD trial was a randomized, double-blind, placebo-controlled trial with 31 ambulatory boys between ages 4 and 7 with a genetically confirmed diagnosis of DMD across a range of dystrophin mutations. The boys were steroid naive. This portion of the trial was conducted at five sites in the U.S., and assessed the safety and efficacy of edasalonexent in patients at two dosing levels (67 mg/kg/day and 100 mg/kg/day) or placebo before and after 12 weeks of dosing. The 67 mg/kg/day treatment group was dosed 33 mg/kg twice per day and the 100 mg/kg/day treatment group was dosed 33 mg/kg three times per day. Sixteen of the 17 boys who participated in the first part of the MoveDMD trial (Part A) participated in Part B. The primary efficacy end point in Part B for which the trial was powered was average change from baseline to week 12 in MRI T2 measure for a composite of five lower leg muscles for the pooled edasalonexent treatment groups, 67 mg/kg/day and 100 mg/kg/day, compared to placebo. MRI T2 is a measure of the composition of muscle and an indicator of muscle inflammation. Safety and tolerability were also evaluated. Additional assessments were measured; however, the trial was not powered for statistical significance for these assessments. The additional assessments include timed function tests (10-meter walk/run, 4-stair climb and time to stand), the North Star Ambulatory Assessment (NSAA), muscle strength measures and the pediatric outcomes data collection instrument (PODCI).
The open-label extension (Part C) was initiated in July and includes
dosing with edasalonexent for 36 weeks beyond Part B and will evaluate
longer term safety and efficacy with the same clinical end points as in
Part B. PPMD and the
Catabasis today also announced that
Conference Call Dial-In Information:
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|
Please specify to the operator that you would like to join the “Catabasis MoveDMD Part B 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 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 Duchenne muscular dystrophy (DMD or Duchenne), regardless of their underlying mutation. Edasalonexent inhibits NF-kB, a protein that is activated in Duchenne and drives inflammation and fibrosis, muscle degeneration and suppresses muscle regeneration. In animal models of DMD, edasalonexent produced beneficial effects in skeletal, diaphragm and cardiac muscle and improved function. 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 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; availability and timing of results from preclinical studies and clinical trials; 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 period ended
Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971