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April 22-25, 2019 | Boston, MA

Day One
Tuesday April 23

Day Two
Wednesday April 24

08.20
Chair’s Opening Remarks

  • Laurent Fischer Senior Vice President, Head Liver Therapeutic Area Allergan

Critically Assessing Patient Recruitment Challenges Now & in the Future

08.30
NASH/NAFLD: Does Genotype Connect to Phenotype?

  • Linda Morrow Vice President & Chief Medical Officer , ProSciento

Synopsis

  • The slow rise of individualized medicine
  • Genetic markers of risk for NASH/NAFLD
  • Is NASH the poster child for individualized medicine?

09.00
NAFLD & NASH from the Patient Perspective – What Are Our Next Steps?

Synopsis

  • Presenting the work of the European Liver Patients Association (ELPA)
  • Outlining what can be learnt from HCV patient advocacy and policy successes
  • Transferring good practice from HCV, HCC to NASH/NAFLD
  • Overcoming basic problems for patients in NAFLD/NASH
  • Where and how we move the field forward?

Achieving Metabolic & Hepatic Effects for the Treatment of NASH

09.30
The Therapeutic Potential of Inhibitors of Steatosis for the Treatment of NASH

Synopsis

  • Demonstrating the work of the Pfizer Internal Medicine Research Unit to treat NASH by targeting the root cause of the disease, the accumulation of hepatic lipids
  • Presenting the scientific rationale for targeting Diacylglycerol Transferase 2 (DGAT2) to ameliorate steatosis will be discussed
  • Sharing results of DGAT2 inhibition in preclinical models of NASH will presented

10.00
Morning Break & Networking

Analyzing, Quantifying & Targeting Multiple NASH Pathways

11.00 An Analysis of NASH Pathways by Single Cell Sequencing

• NASH develops through an interplay of several cell types in the liver which exist in varying abundances, although the precise nature of these
interactions remains unknown
• Single Cell Sequencing (SCS) advances the sequencing capabilities of RNAseq by providing quantifiable RNA transcript reads on a single-cell basis
• By applying SCS technology to NASH and healthy liver samples, an understanding of cell-specific biology can be achieved as it relates to the pathological features of NASH

James Conway
Senior Scientist- Translational
Bioinformatics
MedImmune

11.30 Preclinical to Clinical Translation: Critically Review Quantification Methods of Liver Fat Oxidation as well as Protein Flux Biomarkers

• Exploring proteomic methods for a dynamic measurement of physiologic function and confidently quantify liver fat oxidation
• Reviewing recent progress in protein flux biomarkers as a noninvasive diagnostic
• Implementing quantification methods of liver fat oxidation and protein flux biomarkers to improve development from preclinical to clinical translation

Stephen Previs
Director
Merck

12.00 Targeting Multiple Drivers of NASH by GSNOR Inhibition: Inflammation, Oxidative Stress, Steatosis, Glucose Dysregulation, & Fibrosis

• Regulating cellular nitrosylation through GSNOR inhibition and therefore inhibiting many drivers of
pathology
• Demonstrating efficacy of SAJE’s small molecule GSNOR inhibitor
• Evaluating the potential to prevent progression of NASH and, perhaps, to reverse it, and obviate the need for multiple drugs to regulate the
disease

Matthews Bradley
Founder, Chairman, President &
Chief Technical Officer
SAJE Pharma

The Relationship of NASH in the Context of Wider Liver & Cardiovascular Disease

11.00 Combating NASH as a Metabolic, Non-Communicable Disease with Significant Global Burden

  • Investigating strategies for target discovery
  • Evaluating options for precision medicine

Maria-Chiara Magnone
Vice President, Metabolic Complications
Janssen Research & Development

11.30 Understanding Hepatocellular Carcinoma as an Extension of NASH: The Cirrhosis-HCC Connection, Practical Implications for Preclinical & Clinical Drug Testing

  • Investigating cellular and molecular mechanism of fibrosis-HCC crosstalk
  • Analyzing caveats of current practices of preclinical anti-fibrotic drug testing – short duration, mild disease, reliance on surrogate endpoints instead of clinically-relevant long-term outcomes
  • Strategizing overcoming these caveats, and how to move towards using clinically-relevant end-points in preclinical drug evaluation in practice

Yury Popov
Assistant Professor of Medicine
Harvard Medical School

12.00 The Endocrinologist Perspective: Profiling NASH in the Context of Metabolic Syndrome

  • The systemic and heterogenous nature of NAFLD and its relationship to type 2 diabetes and cardiovascular disease
  • Common mechanistic underpinnings

Manu Chakravarthy
Senior Vice President & Chief Medical Officer
Axcella Health

Safety & Efficacy Within the Clinic

11.00 IDL-2965: A Clinical Stage, Oral, RGD-binding Integrin Antagonist for the Treatment of NASH & IPF

  • Indalo’s small molecule therapeutics selectively inhibit key RGD-binding integrins involved in multiple processes of pathologic fibrosis including TGFB activation, fibroblast durotaxis, and myofibroblast survival
  • IDL-2965 displays potent antifibrotic activity at low oncedaily oral doses in multiple animal models of liver and lung fibrosis
  • IDL-2965 is currently being investigated in an adaptive, biomarker driven Phase 1/2a clinical program, allowing rapid progression from healthy volunteers to NASH and IPF patients

Scott Seiwert
Chief Scientific Officer
Indalo Therapeutics

11.30 Adhering to Regulatory Guidelines & Overcoming Clinical Challenges Related to NASH Proof of Concept Studies

  • Filling the gap between non-invasive assessment of NASH PoC endpoints and the clinically relevant endpoints
  • Robustly applying non-invasive MRI imaging in addition to liver fat quantification
  • Revewing in silico trial simulations using 10,000 virtual NASH patients to explore and support diverse study designs
  • Are there more relevant and helpful approaches in the European vs. North American current thinking in the moving target NASH development field?

Pietro Scalfaro
Chief Medical Officer
Enyo Pharma

12.00 Adhering to Regulatory Guidelines & Overcoming Clinical Challenges Related to NASH Proof of Concept Studies

  • Filling the gap between noninvasive assessment of NASH PoC endpoints and the clinically relevant endpoints
  • Robustly applying non-invasive MRI imaging in addition to liver fat quantification
  • Revewing in silico trial simulations using 10,000 virtual NASH patients to explore and support diverse study designs
  • Are there more relevant and helpful approaches in the European vs. North American current thinking in the moving target NASH development field?

Pietro Scalfaro
Chief Medical Officer
Enyo Pharma

Patient Recruitment From the Perspective of the Patient

12.00 Finding NASH Patients: Uncovering Insights into the Lives of Those Who Likely Have it — But Don’t Know it

  • Clinical trial enrollment barriers and opportunities identified by our research study
  • The patient journey — their thoughts, feelings, and emotions — leading to a better understanding for both healthcare professionals and their patients as the industry continues investigating treatment for this life-threatening condition
  • How sponsor of clinical trials can more successfully engage this patient population

Cathleen Dohrn
Senior Scientific Director
Continuum Clinical

Advancing Clinical Development with AI

12.15 AI powered NASH registry to enable efficient clinical development and evidence generation

  • Non-invasive, real world data based self learning AI model can ease the burden of patient while reducing screening failure for clinical trials
  • The use of AI algorithm to establish prospective longitudinal registry as core strategy to support long term evidence needs beyond RCT

Qin Ye
Global RWE Lead
ZS Associates

Lisa Bance
Lifecycle Evidence Strategy Lead
ZS Associates

12.45
Lunch & Networking

Reviewing Clinical Advances of NASH Therapeutics in Phase 2 & Phase 3 Clinical Trials

13.45
Regulatory Guidance on NASH Drug Development

  • Frank Anania Division of Gastroenterology & Inborn Errors Products , FDA

14.15
Showcasing the Clinical Development of MGL3196

  • Rebecca Taub Chief Medical Officer, Executive Vice President Research & Development , Madrigal Pharmaceuticals

Synopsis

  • Reviewing Madrigal’s clinical development of MGL3196
  • Advancing understanding surrounding thyroid hormone receptor beta agonist reducing lipotoxicity in the NASH liver
  • Critically analysing MGL-3196 as the first truly beta selective THR-beta agonist

14.45
Afternoon Refreshments & Networking

15.15
Session Reserved for Intercept

15.45
Aramchol Phase 2b Results & Phase 3 Outlook

Synopsis

  • NASH develops through an interplay of several cell types in the liver which exist in varying abundances, although the precise nature of these interactions remains unknown
  • Single Cell Sequencing (SCS) advances the sequencing capabilities of RNAseq by providing quantifiable RNA transcript reads on a single-cell basis
  • By applying SCS technology to NASH and healthy liver samples, an understanding of cellspecific biology can be achieved as it relates to the pathological features of NASH

16.15
An Update of Elafibranor & Use as the Backbone for Combinations

  • Dean Hum Senior Executive Vice President & Chief Scientific Officer , Genfit

Synopsis

  • Overview of Elafibranor including disease model data
  • Presenting data from combination program to identify synergistic mechanisms of action with Elafibranor

16.45
Chairs’ Closing Remarks