CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that Catabasis will present the MoveDMD trial of edasalonexent for the treatment of Duchenne muscular dystrophy (DMD) at the American Neurological Association’s (ANA) 2016 Annual Meeting. The ANA 2016 Annual Meeting is being held October 16 – October 18, 2016, in Baltimore, MD, at the Baltimore Marriott Waterfront Hotel.
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.” The presentation will take place in Poster Session II and Wine & Cheese Reception on Monday, October 17, from 5:30pm – 7:00pm local time in the Harborside Ballroom, Fourth Floor, of the Baltimore Marriott Waterfront Hotel.
About Edasalonexent (CAT-1004)
Edasalonexent (CAT-1004) is an oral small molecule that has the potential to be a 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 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. We have previously reported safety, tolerability and reduction in NF-kB activity in Phase 1 trials in adults. We are currently conducting the MoveDMD® trial of edasalonexent in 4-7 year-old boys affected by Duchenne. From Part A of the MoveDMD trial, we have reported that edasalonexent was generally well tolerated with no safety signals observed and we observed NF-kB target engagement. Pharmacokinetic results demonstrated edasalonexent plasma exposure levels consistent with those previously observed in adults, at which inhibition of NF-kB was observed.
The MoveDMD trial is a three-part clinical trial investigating the safety and efficacy of edasalonexent in boys ages 4 – 7 affected with DMD (any confirmed mutation). Part A of the MoveDMD trial evaluated the safety, tolerability and pharmacokinetics of, and NF-kB target engagement with, edasalonexent and showed positive results. Sixteen of the 17 boys enrolled in Part A continued to Part B of the trial, which is a Phase 2 trial to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week period in approximately 30 boys. The primary end point is change in MRI of the lower leg muscles, and the secondary end points are age-appropriate timed function tests: 10-meter walk/run, 4-stair climb and time to stand. Additional assessments include muscle strength, the North Star Ambulatory Assessment and the pediatric outcomes data collection instrument (PODCI). Part C is an open-label extension that includes dosing with edasalonexent for 36 weeks beyond the 12-week placebo-controlled portion of the trial (Part B) and will evaluate longer term safety and efficacy with the same clinical end points as Part B.
Magnetic resonance imaging (MRI) is a non-invasive imaging technique that can assess muscle structure and composition and measure disease status in children with DMD. Two MRI measures used in Duchenne to indicate muscle degeneration are T2 and fat fraction. MRI is sensitive to changes in muscle structure and composition induced by disease processes such as the inflammation, edema, muscle damage and fat infiltration that occur in Duchenne. Changes in T2 may be seen in less than 12 weeks while changes in fat fraction may take longer. Changes in these MRI measures have been correlated with longer-term changes in clinically meaningful measures of functional activity. Changes in MRI can show the effects of an investigational therapy on disease progression in Duchenne in an objective and quantifiable manner.
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 linker 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.