Catabasis And Parent Project Muscular Dystrophy To Host A Webinar On MoveDMD: A Clinical Trial Of Edasalonexent (CAT-1004) In Boys With Duchenne Muscular Dystrophy

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Catabasis Pharmaceuticals, Inc. (NASDAQ:CATB), a clinical-stage biopharmaceutical company, and Parent Project Muscular Dystrophy (PPMD) will host a webinar “MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy” this afternoon on Wednesday, June 8, 2016 at 1:00 pm ET.

“MoveDMD: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy”

Speakers include:

• Dr. Joanne Donovan, Chief Medical Officer, Catabasis
• Pat Furlong, Parent Project Muscular Dystrophy

Dr. Donovan will discuss the positive results from Part A of the MoveDMD trial and review the design and inclusion criteria of Part B (Phase 2) that is currently running. The MoveDMD trial is a Phase 1/2 clinical trial of edasalonexent in boys with Duchenne between the ages of 4 and 7. The goal of Part A included evaluation of the safety and tolerability of edasalonexent. Part B will evaluate the safety and efficacy of edasalonexent in boys with Duchenne.

The webinar can be accessed by visiting www.readytalk.com and providing access code 9449985. The audio dial-in can be accessed by dialing 1.866.740.1260 and providing the access code 9449985.

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 MRI
Magnetic resonance imaging (MRI) is a non-invasive imaging technique that can visualize 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.

About Catabasis
At Catabasis Pharmaceuticals, our mission is to bring hope and life-changing therapies to patients and their families. We have product candidates in both rare diseases and serious lipid disorders. 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.