Catabasis Pharmaceuticals presents data from Part A of MoveDMD trial
Catabasis Pharmaceuticals announced that Catabasis is presenting data from Part A of the MoveDMD trial of edasalonexent for the treatment of Duchenne muscular dystrophy at the World Muscle Society Congress. The World Muscle Society Congress is being held October 4 - October 8, 2016, in Granada, Spain, at the Palacio de Congresos de Granada. Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis, will present the poster "CAT-1004, an oral agent targeting NF-kB: MoveDMD trial results in Duchenne muscular dystrophy" and Pradeep Bista, Ph.D., Principal Scientist at Catabasis, will present the poster "Serum pro-inflammatory proteins have potential utility as biomarkers for NF-kB targeting approaches in DMD." Both poster presentations will take place in Poster Presentations 3: Parallel Sessions today from 3:00pm - 4:30pm local time in the Poster area of the Palacio de Congresos de Granada. Catabasis has previously announced positive biomarker results from Part A of the MoveDMD clinical trial demonstrating successful NF-kB target engagement and detailed data are being presented at the World Muscle Society Congress. After one week of dosing with edasalonexent in 4-7-year-old boys affected by DMD, a pre-selected NF-kB gene set was significantly inhibited in whole blood mRNA at each of the two higher doses. The evaluated gene set is 200 expressed genes known to be regulated by NF-kB that was curated by the Broad Institute. This gene set was significantly decreased when compared to all other expressed genes. In addition to gene expression, serum proteins were also evaluated. In the combined group of patients on 67 mg/kg and 100 mg/kg, levels of NF-kB-regulated proteins, IL-12 and osteopontin, were significantly reduced following 7 days of dosing with edasalonexent. These data further support the ongoing evaluation of the 67 and 100 mg/kg edasalonexent doses in the Phase 2 MoveDMD clinical trial. Baseline data for boys from Part A of the trial who are also participating in Part B are being presented at the World Muscle Society Congress as well. Timed function tests were assessed before the boys had started dosing with edasalonexent in Part A of the trial as well as at baseline before they started dosing with edasalonexent in Part B of the trial. For all three of the timed function tests that we are evaluating, 10-meter walk/run, 4-step climb and time-to-stand, there was an increase in the average time to complete the tests from Part A baseline to Part B baseline. The time elapsed between Part A baseline and Part B baseline ranged from 4 to 11 months for these 15 boys. Although the MoveDMD trial is not powered to show statistically significant changes in these three timed function tests in Part B, the observations provide data on disease progression within the MoveDMD trial that shows a decline in function when these boys are predominantly not on treatment and may provide additional context for assessing these secondary end points during the on-treatment part of the trial. The primary end point of Part B of the MoveDMD trial is change in MRI T2 signal of the lower leg muscles for edasalonexent as compared with placebo.