Scientific Presentations

Catabasis Publications and Scientific Presentations

Donovan JM, Zimmer M, Offman E, Grant T, Jirousek M. A Novel NF-κB Inhibitor, Edasalonexent (CAT-1004), in Development as a Disease-Modifying Treatment for Patients With Duchenne Muscular Dystrophy: Phase 1 Safety, Pharmacokinetics, and Pharmacodynamics in Adult Subjects. J Clin Pharmacol. 2017 Jan. doi: 10.1002/jcph.842

Hammers DW, Sleeper MM, Forbes SC, Coker CC, Jirousek MR5, Zimmer M, Walter GA, Sweeney HL. Disease Modifying Effects of Orally Bioavailable NF-kB Inhibitors in Dystrophin-Deficient Muscle. JCI Insight. 2016 December; 1(21): e90341

Vu CB, Bemis JE, Benson E, Bista P, Carney D, Fahrner R, Lee D, Liu F, Lonkar P, Milne JC, Nichols AJ, Picarella D, Shoelson A, Smith J, Ting A, Wensley A, Yeager M, Zimmer M, Jirousek MR. Synthesis and Characterization of Fatty Acid Conjugates of Niacin and Salicylic Acid. J Med Chem. Epub 2016 Jan 19

Vu CB, Bridges RJ, Pena-Rasgado C, Lacerda AE, Bordwell C, Sewell A, Nichols AJ, Chandran S, Lonkar P, Picarella D, Ting A, Wensley A, Yeager M, Liu F.Fatty Acid Cysteamine Conjugates as Novel and Potent Autophagy Activators that Enhance the Correction of Misfolded F508del-CFTR. J Med Chem. 2016 December. doi: 10.1021/acs.jmedchem.6b01539

April 2017 (American Academy of Neurology Annual Meeting): MoveDMD Results: Effects of Edasalonexent, an NF-κB Inhibitor, in 4 to 7 Year Old Patients with Duchenne Muscular Dystrophy
JM Donovan, MD, PhD1, K Vandenborne, PT, PhD2, HL Sweeney, PhD2, G Tennekoon, MBBS, MRCS, LCRP3, E Finanger, MD4, P Shieh, MD PhD5, S Yum, MD3, M Mancini, MHP1, P Bista, PhD1, A Nichols, PhD1, R Finkel, MD6
1Catabasis Pharmaceuticals, Cambridge MA; 2University of Florida Health, Gainesville FL; 3The Children's Hospital of Philadelphia, Philadelphia PA; 4Oregon Health Sciences University, Portland OR; 5UCLA, Los Angeles CA; 6Nemours Children's Health System, Orlando FL

March 2017 (Muscular Dystrophy Association Scientific Conference): MoveDMD: Phase 1/2 Trial of Edasalonexent, an NF-κB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy (Oral)
Richard Finkel, MD1, Krista Vandenborne, PT, PhD.2, H Lee Sweeney, PhD.2, Erika Finanger, MD3, Gihan Tennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Sabrina Yum, MD4, Maria Mancini, MHP6, Pradeep Bista, PhD6, Andrew Nichols, PhD6, Joanne Donovan, MD, PhD6

March 2017 (Muscular Dystrophy Association Scientific Conference): MoveDMD: Phase 1/2 Trial of Edasalonexent, an NF-κB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy (Poster)
Richard Finkel, MD1, Krista Vandenborne, PT, PhD.2, H Lee Sweeney, PhD.2, Erika Finanger, MD3, Gihan Tennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Sabrina Yum, MD4, Maria Mancini, MHP6, Pradeep Bista, PhD6, Andrew Nichols, PhD6, Joanne Donovan, MD, PhD6

October 2016 (American Neurology Association's Annual Meeting): Edasalonexent (CAT-1004), an Oral Agent Targeting NF-κB: MoveDMD Part A Results in Duchenne Muscular Dystrophy (Oral)
E Finanger1; K Vandenborne2; R Finkel3; HL Sweeney3; G Tennekoon4; S Yum4; M Mancini5; J Danis5; P Bista5; A Nichols5; J Donovan5
1Oregon Health Sciences University Pediatrics, Portland USA; 2University of Florida Health Physical Therapy Gainesville USA; 3Nemours Children's Health Pediatric Neurology Orlando USA; 3University of Florida Health Myology Institute Gainesville USA; 4Children's Hospital of Philadelphia Pediatric Neurology Philadelphia USA; 5Catabasis Pharmaceuticals Cambridge USA

October 2016 (American Neurology Association's Annual Meeting): Edasalonexent (CAT-1004), an Oral Agent Targeting NF-κB: MoveDMD Part A Results in Duchenne Muscular Dystrophy (Poster)
E Finanger1; K Vandenborne2; R Finkel3; HL Sweeney3; G Tennekoon4; S Yum4; M Mancini5; J Danis5; P Bista5; A Nichols5; J Donovan5
1Oregon Health Sciences University Pediatrics, Portland USA; 2University of Florida Health Physical Therapy Gainesville USA; 3Nemours Children's Health Pediatric Neurology Orlando USA; 3University of Florida Health Myology Institute Gainesville USA; 4Children's Hospital of Philadelphia Pediatric Neurology Philadelphia USA; 5Catabasis Pharmaceuticals Cambridge USA

October 2016 (World Muscle Society Congress): Edasalonexent (CAT-1004), an Oral Agent Targeting NF-κB: MoveDMD Part A Results in Duchenne Muscular Dystrophy
E Finanger1; K Vandenborne2; R Finkel3; HL Sweeney3; G Tennekoon4; S Yum4; M Mancini5; J Danis5; P Bista5; A Nichols5; J Donovan5
1Oregon Health Sciences University Pediatrics, Portland USA; 2University of Florida Health Physical Therapy Gainesville USA; 3Nemours Children's Health Pediatric Neurology Orlando USA; 3University of Florida Health Myology Institute Gainesville USA; 4Children's Hospital of Philadelphia Pediatric Neurology Philadelphia USA; 5Catabasis Pharmaceuticals Cambridge USA

October 2016 (World Muscle Society Congress): Serum Pro-inflammatory Proteins Have Potenital Utility as Biomarkers for NF-κB Targeting Approaches in DMD
P Bista1; G Walter2; K Vandenborne3; B Lee2; A Nichols1; J Donovan1
1Catabasis Pharmaceuticals, Cambridge USA; 2University of Florida Physiology and Functional Genomics, Gainesville USA; 3University of Florida Health Physical Therapy, Gainesville USA

June 2016 (PPMD Connect Conference): MoveDMDSM: A Clinical Trial of Edasalonexent (CAT-1004) in Boys with Duchenne Muscular Dystrophy
JM Donovan1, K Vandenborne2, EL Finanger3, HL Sweeney2, G Tennekoon4, SW Yum4, MC Mancini1, RS Finkel5
1Catabasis Pharmaceuticals, Inc., 2University of Florida, 3Oregon Health & Science University, 4Children's Hospital of Philadelphia, 5Nemours Children's Hospital, Orlando

March 2016 (MDA Annual Conference): Poster: MoveDMD: A Phase 1 / 2 Clinical Trial with CAT-1004 in Boys with Duchenne Muscular Dystrophy
Presentation: MoveDMD: A Phase 1 / 2 Clinical Trial with CAT-1004 in Boys with Duchenne Muscular Dystrophy
EL Finanger1, JM Donovan2, K Vandenborne3, HL Sweeney3, G Tennekoon4, SW Yum4, MC Mancini2, JR Danis2, RS Finkel5
1Oregon Health & Science University 2Catabasis Pharmaceuticals*, 3University of Florida, 4Children’s Hospital of Philadelphia, 5Nemours Children’s Hospital, Orlando

November 2015 (Action Duchenne Conference): CAT-1004, an Oral Agent Targeting Activated NF-κB in Development as a Disease-Modifying Treatment for Treatment for Duchenne Muscular Dystrophy: Design of MoveDMD, a Phase 1 / 2 Trial
JM Donovan1, HL Sweeney2, K Vandenborne3, BS Russman4, M Jirousek1, RS Finkel5
1Catabasis Pharmaceuticals, 2U of Pennsylvania Perelman School of Medicine, 3U of Florida, 4Oregon Health & Science University, 5Nemours Children's Hospital

October 2016 (North American Cystic Fibrosis Conference): CAT-5571 As a Novel and Potent Autophagy Activator that Enhances the Trafficking of the F508DEL-CFTR (Oral)
F Liu1; D Carney1; S Chandran1; D Lee1; P Lonkar1; A Nichols1; D Picarella1; A Ting1; S Webb1; A Wensley1; M Yeager1; R J Bridges2; C PenaRasgado2; A Lacerda3; C Bordwell3; C B Vu1
1Catabasis Pharmaceuticals, Cambridge, MA, USA 2 Chicago Medical School, North Chicago, IL, USA 3Charles River, Cleveland, OH, USA

October 2016 (North American Cystic Fibrosis Conference): CAT-5571 As a Novel and Potent Autophagy Activator that Enhances the Trafficking of the F508DEL-CFTR (Poster)
F Liu1; D Carney1; S Chandran1; D Lee1; P Lonkar1; A Nichols1; D Picarella1; A Ting1; S Webb1; A Wensley1; M Yeager1; R J Bridges2; C PenaRasgado2; A Lacerda3; C Bordwell3; C B Vu1
1Catabasis Pharmaceuticals, Cambridge, MA, USA 2 Chicago Medical School, North Chicago, IL, USA 3Charles River, Cleveland, OH, USA

October 2016 (North American Cystic Fibrosis Conference): CAT-5571 As a Novel Autophagy Activator That Enhances the Clearance of Pseudomonas Aeruginosa
T Bonfied1; F Liu2; S Chandran2; R Fahrner2; D Lee2; A Nichols2; D Picarella2; A Ting2; S Webb2; M Yeager2; C B Vu2
1Case Western Reserve Univ., School of Medicine, Cleveland, OH, USA; 2Catabasis Pharmaceuticals, Cambridge, MA, USA

May 2016 (DDW): CAT-2003, an Analog of CAT-2054, a Novel Oral Sterol Regulatory Element Binding Protein Inhibitor, Inhibits Inflammation and Fibrosis in a Murine Model of Nonalcoholic Steatohepatitis (NASH)
Dominic Picarella1, Mike Zimmer1, Diana Lee1, Michael Briggs2, ArlinRogers3, Joanne Donovan1 and Andrew Nichols1
1Catabasis Pharmaceuticals, Inc., 2Woodland Biosciences, 3Cummings School of Veterinary Medicine Tufts University

May 2016 (NLA): Phase 1 Multiple Ascending Dose Study of CAT-2054, a Novel Oral Sterol Regulatory Element Binding Protein Inhibitor
Joanne Donovan MD PhD1, Maria Mancini MHP1, Andrew Nichols PhD1
1Catabasis Pharmaceuticals