Catabasis Pharmaceuticals to Present Edasalonexent (CAT-1004), an Oral Agent Targeting NF-kB: MoveDMDSM Trial in Duchenne Muscular Dystrophy (DMD) at the 2016 PPMD Annual Connect Conference

Catabasis Pharmaceuticals to Present Edasalonexent (CAT-1004), an Oral Agent Targeting NF-kB: MoveDMD^SM Trial in Duchenne Muscular Dystrophy (DMD) at the 2016 PPMD Annual Connect Conference

Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, today announced that edasalonexent (CAT-1004) will be featured in an oral presentation and a poster presentation at the upcoming 2016 Parent Project Muscular Dystrophy (PPMD) Annual Connect Conference being held on Sunday, June 26 – Wednesday, June 29, 2016 at the Renaissance Orlando at Sea World Hotel in Orlando, FL.

• Joanne Donovan, M.D., Ph.D., Chief Medical Officer of Catabasis, will give a presentation titled “Edasalonexent (CAT-1004), an Oral Agent Targeting NF-kB: MoveDMD^SM Trial in Duchenne Muscular Dystrophy (DMD)”
  • The oral presentation will take place on Monday, June 27, 2016, from 3:20pm – 3:40pm ET in Crystal Ballrooms C, D & E.
  • The poster presentation will take place during the Poster Fair on Monday, June 27, 2016, from 12:30pm – 2:15pm ET in Crystal Ballrooms A & B.

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 the 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 inhibited NF-kB, reduced muscle degeneration and improved muscle regeneration and function, and beneficial effects were observed in skeletal, diaphragm and cardiac muscle. 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^SM 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 successful 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.

About MoveDMD^SM
MoveDMD is a Phase 1 / 2 clinical trial of edasalonexent (CAT-1004) in boys ages 4-7 affected with DMD (any confirmed mutation). The MoveDMD trial is a two-part clinical trial investigating the safety and efficacy of edasalonexent in DMD. Part A of the MoveDMD trial evaluated the safety, tolerability and pharmacokinetics of, and NF-kB target engagement with, edasalonexent and showed positive results. The boys in Part A of the trial are asked to participate, if eligible, in Part B of the trial. Part B of the trial is a Phase 2 trial to evaluate the safety and efficacy of edasalonexent in DMD over a 12-week treatment period and will enroll approximately 30 boys. The primary end point is changes in MRI of the 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 tool (PODCI).

About Catabasis
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.

Catabasis Pharmaceuticals, Inc.
Andrea Matthews, 617-349-1971
amatthews@catabasis.com

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