Scientific Presentations

Catabasis Publications

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, 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. 2017 Jan 12;60(1):458-473. doi: 10.1021/acs.jmedchem.6b01539. Epub 2016 Dec 23.

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

Zimmer M, Bista P, Benson EL, Lee DY, Liu F, Picarella D, Vega RB, Vu CB, Yeager M, Ding M, Liang G, Horton JD, Kleemann R, Kooistra T, Morrison MC, Wielinga PY, Milne JC, Jirousek MR, Nichols AJ. CAT-2003: A Novel Sterol Regulatory Element-Binding Protein Inhibitor That Reduces Steatohepatitis, Plasma Lipids, and Atherosclerosis in Apolipoprotein E*3-Leiden Mice.
Hepatology Communications. Epub 2017 May 12. doi:10.1002/hep4.1042

Scientific Presentations

April 2018 (American Academy of Neurology Conference): MoveDMD®: Positive Effects of Edasalonexent, an NF-κB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy in Phase 2 Study with an Open-Label Extension
Richard Finkel1, Krista H. E. Vandenborne2, H. Lee Sweeney2, Erika Finanger3, Gihan Tennekoon4, Perry Shieh5, Rebecca J. Willcocks2, Sean C. Forbes2, William Triplett2, Sabrina Yum4, Maria Mancini6, Angelika Fretzen6, Joanne Donovan6
1Nemours Children's Hospital, 2University of Florida, 3Oregon Health Science University, 4The Children's Hospital of Philadelphia, 5UCLA, 6Catabasis Pharmaceuticals

March 2018 (Muscular Dystrophy Association Clinical Conference): Edasalonexent, an NF- ĸB Inhibitor, Slows Disease Progression in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy
Richard Finkel, MD1, Krista Vandenborne, PT, PhD for the ImagingDMD Consortium, H Lee Sweeney, PhD2, Erika Finanger, MD3, Gihan Tennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Rebecca J Willcocks, PhD2, Sean C Forbes, PhD2, Glenn Walters, PhD2, William Rooney, PhD3, William T Triplett, BSc2, Sabrina S Yum, MD4, Maria Mancini, MHP6, Pradeep Bista, PhD6, Angelika Fretzen, PhD6, Joanne Donovan, MD, PhD6
1Nemours Children’s Health System, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon health Sciences University, Portland, OR; 4The Children’s Hospital of Philadelphia, Philadelphia, PA; 5University of California, Los Angeles, Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA

February 2018 (International Conference on Duchenne and Becker Muscular Dystrophy): Edasalonexent (CAT-1004): An NF-ĸB Inhibitor in Development for Patients with Duchenne Muscular Dystrophy
Richard Finkel, MD1, Krista Vandenborne, PT, PhD2, H Lee Sweeney, PhD2, Glenn Walters, PhD2, Erika Finanger, MD3, Bill Rooney, PhD3, Gihan Tennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Rebecca Willcocks, PhD2, Sean C. Forbes, PhD2, William T. Triplett, BSc2, Sabrina Yum, MD4, Joanne Donovan,
MD, PhD6
1
Nemours Children’s Health System, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon Health Sciences University, Portland, OR; 4The Children’s Hospital of Philadelphia, Philadelphia, PA; 5University of California, Los Angeles, Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA

November 2017 (Action Duchenne International Conference): MoveDMD®: Positive Effects of Edasalonexent, an NF-ĸB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy in Phase 2 Study with an Open-Label Extension
Richard Finkel, MD1, Krista Vandenborne, PT, PhD2, H Lee Sweeney, PhD2, Erika Finanger, MD3, Gihan Tennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Rebecca Willcocks, PhD2, Sean C. Forbes, PhD2, William T. Triplett, BSc2, Sabrina Yum, MD4, Maria Mancini, MHP6, Angelika Fretzen, PhD6, Pradeep Bista, PhD6, Andrew Nichols, PhD6, Joanne Donovan, MD, PhD6
1Nemours Children’s Health System, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon Health Sciences University, Portland, OR; 4The Children’s Hospital of Philadelphia, Philadelphia, PA; 5University of California, Los Angeles, Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA

November 2017 (Action Duchenne International Conference): Edasalonexent Dosing Regimen: Preclinical Modeling and Clinical Translation
John F. Reilly, Hanlan Liu, Pradeep Bista, Derek Wachtel, Sachin Chandran, Rafif Dagher, Dominic Picarella, Maria Mancini, Angelika Fretzen, Joanne Donovan and Andrew Nichols
Catabasis Pharmaceuticals, Cambridge, MA, US

October 2017 (Congress of the World Muscle Society): Edasalonexent (CAT-1004), an NF-ĸB Inhibitor, Enhances Myotube Formation In Vitro, and Increases Exon-skipped Sarcolemmal Dystrophin in Muscle of Mdx Mice
Nichols, A1, Reilly, J1, Liu, F1, Bista, P1, Lee, D1, Webb, S1, Picarella, D1, Wood, J2, Yao, M2, Passini, M2, Estrella, N2
1Catabasis Pharmaceuticals, Inc. Cambridge USA, 2Sarepta Therapeutics, Inc. Cambridge USA

October 2017 (Congress of the World Muscle Society): Positive Effects of Edasalonexent, an NF-ĸB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy in Phase 2 Study with an Open-Label Extension
Richard Finkel, MD1, Krista Vandenborne, PT, PhD2, H Lee Sweeney, PhD2, Erika Finanger, MD3, Gihan Tennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Rebecca Willcocks, PhD2, Sean C. Forbes PhD2, William T. Triplett, BSc2, Sabrina Yum, MD4, Maria Mancini, MHP6, Angelika Fretzen PhD6, Joanne Donovan, MD, PhD6
1Nemours Children’s Health System, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon Health Sciences University, Portland, OR; 4The Children's Hospital of Philadelphia, Philadelphia, PA; 5University of California, Los Angeles, Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA

September 2017 (Ottawa Neuromuscular Disease Conference): Preclinical Development and Clinical Translation of Edasalonexent (CAT-1004), a Small Molecule Using SMART Linker℠ Technology as a Potential Disease Modifying Therapy for the Treatment of Duchenne Muscular Dystrophy 
Hanlan Liu, Joanne Donovan, Maria Mancini, Mike Zimmer, Rafif Dagher, Dominic Picarella, Amal Ting, Diana Lee, Derek Wachtel, Feng Liu, Pradeep Bista, Sachin Chandran, Ron Shmueli, Angelika Fretzen, and Andrew Nichols
Catabasis Pharmaceuticals, Inc., Cambridge, MA, USA

June 2017 (PPMD Connect Conference): Phase 2 Trial of Edasalonexent, an NF-ĸB Inhibitor, in 4 to 7-Year Old Patients with Duchenne Muscular Dystrophy
Richard Finkel, MD1, Krista Vandenborne, PT, PhD2, H Lee Sweeney, PhD2, Erika Finanger, MD3, GihanTennekoon, MBBS, MRCS, LCRP4, Perry Shieh, MD, PhD5, Rebecca Willcocks, PhD2, Sean C. Forbes PhD2, William T. Triplett, BSc2, Sabrina Yum, MD4, Maria Mancini, MHP6, Michael Friedman, PhD7, Angelika Fretzen PhD6, Joanne Donovan, MD, PhD6
1Nemours Children’s Health System, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon Health Sciences University, Portland, OR; 4The Children's Hospital of Philadelphia, Philadelphia, PA; 5University of California, Los Angeles, Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA; 7Statistical Consultant

May 2017 (NEDMDG Spring Meeting): In vitro and in vivo metabolite profiling and identification of edasalonexent (CAT-1004), a bioconjugate of salicylic acid (SA) and docosahexaenoic acid (DHA) using SMART Linker technology
Liu H1, Ting A1, Lee D1, Zimmer M1, Wachtel D1, Dagher R1, Mancini M1, Donovan J1, Liu F1, Greene R2, Keehn R2, Cardinal K2, Luo G2, Erratico C-A3, Fonsi M3, and Nichols A1
1Catabasis Pharmaceuticals, Inc., Cambridge, MA, USA; 2Covance Laboratories Inc., Madison, WI, USA; 3CiTox Lab, Evreux Cedex, France

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, PhD
1Nemours Children’s Hospital, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon Health Sciences University, Portland, OR; 4The Children’s Hospital of Philadelphia, PA; 5University of Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA

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, PhD
1Nemours Children’s Hospital, Orlando, FL; 2University of Florida Health, Gainesville, FL; 3Oregon Health Sciences University, Portland, OR; 4The Children’s Hospital of Philadelphia, PA; 5University of Los Angeles, CA; 6Catabasis Pharmaceuticals, Cambridge, MA

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

June 2018 (European Cystic Fibrosis Conference): CAT-5571 Restores Autophagy, a Fundamental Defect in Cystic Fibrosis, and is a Potential New Treatment for People with Cystic Fibrosis 
Feng Liu1, Kathrin Krause2, Amal Amer2, Luanne Hall-Stoodley2, John F. Reilly1 and Andrew J Nichols1
1Catabasis Pharmaceuticals, Inc. Cambridge, MA 02139, USA; 2Department of Microbial Infection and Immunity, The Ohio State University, Columbus, OH 43210, USA

November 2017 (North American Cystic Fibrosis Conference): CAT-5571 Improves the Clearance of Intracellular Burkholderia Cenocepacia from Primary Cystic FibrosisF508del /F508del­ Macrophages
Kathrin Krause1, Andrew J Nichols2, Feng Liu2, Amal O Amer1
1Department of Microbial Infection and Immunity, Center for Microbial Interface Biology, The Ohio State University, Columbus OH; 2Catabasis Pharmaceuticals, Inc. Cambridge USA

June 2017 (European Cystic Fibrosis Conference): CAT-5571 As an Autophagy Activator that Enhances the Clearance of Intracellular Bacteria
Feng Liu1, Amal Amer2, Kathrin Krause2, Tracey L. Bonfield3, David Fletcher3, Claire Richards4, John F. Reilly1, Andrew J. Nichols1, Chi B. Vu1
1Catabasis Pharmaceuticals, Cambridge, MA, USA; 2 Department of Microbial Infection and Immunity, The Ohio State University, Columbus, OH, USA; 3Pediatrics, Case Western Reserve University, Cleveland, OH, USA; 4KWS BioTest, Bristol, UK

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