*All times Eastern Standard Time*

Exploring the Regulatory Landscape: Learning How to Tailor Your Drug Development for Swift Approvals

7:30 am Registration & Networking

8:00 am Drug Development for NASH: A Regulatory Perspective

  • George Makar Medical Officer, Food and Drug Administration


• Understanding the importance of early clinical development and proof-of-concept trials
• Describing the rationale for the choice of the surrogate endpoint reasonably likely to predict clinical benefit
• Identifying the endpoints used to characterize the confirmation of clinical benefit

8:30 am Exploring the Impact of Future Approvals on NASH Drug Development Research from a Regulatory Perspective: What do Biopharma Need to Know?

  • Joachim Musaeus EMA Product Lead/Deputy Head of Rheumatology, Gastroenterology and Immunology, European Medicines Agency


• Uncovering how approvals will alter the NASH regulatory landscape
• Learning how biopharma can adapt their drug development approach to optimize chances of approval
• What do current failures mean for future NASH drug development endeavors?

Exploring the Potential for Artificial Intelligence to Optimize Prediction of NASH Disease & Progression

9:00 am Audience Discussion: Exploring Artificial Intelligence to Read Histology Samples for NASH


For years pathologists have led the way when it comes to reading histology samples for NASH but the arrival of artificial intelligence is sparking up a debate amongst NASH drug developers across the globe. What exactly is AI going to do? How will that work? Where is biopharma included in its design? What’s the state of the art on AI assessment of pathology, both from a scientific perspective and regulatory perspective? Attend this interactive, discursive session to debate your ideas on this topical subject with your peers and leave with the knowledge you need to cultivate an informed opinion of one of the most important issues for NASH drug developers to date.

9:30 am Speed Networking

10:00 am Morning Break & Networking

Understanding How to Manipulate Diabetes to Fast Track Your NASH Drug Development

10:30 am Diabetes & NASH: Exploring its Clinical Implications & Impact for Drug Development

  • Kenneth Cusi Professor of Medicine & Chief, Division of Endocrinology, Diabetes and Metabolism, The University of Florida


• Investigating the magnitude of NASH within the context of the diabetes epidemic
• Outlining practical implications for the management and treatment of NASH
• Learning why diabetes is important to consider for pharmaceutical companies developing agents for NASH


Learning How to Utilize Emerging Therapeutic Modalities for Effective Target Discovery

11:00 am Discovering PNPLA3 Antisense Oligonucleotides: Novel Precision Medicine Approach to Treat NASH


• Exploring genetic targets in NASH and the concept of precision medicine for the treatment of the disease
• Analyzing the biology of PNPLA3 and mechanistic insights on how a point mutation in the gene associates with NASH
• Understanding silencing hepatic PNPLA3 by using a liver targeted GalNAc-conjugated antisense oligonucleotide in NASH

11:30 am Characterization of Thyroid Hormone Receptor (THR) Agonists for the Treatment of Non-Alcoholic Steatohepatitis (NASH) by Quantification of In Vitro & In Vivo Gene Transcription


• Hearing how Aligos has implemented a strategy to rank the efficacy of THR agonists by quantifying changes in the transcription of genes that lead to metabolic alterations, an effect that is directly downstream of THR binding and activation.
• Comparing the ability of THR-agonism-based NASH treatment candidates to modulate the expression of genes related to cholesterol and fatty acid biosynthesis and metabolism in vitro using Huh-7 cells and primary human hepatocytes.
• Understanding how our observations in vitro were confirmed in a HFD-fed rodent model, where treatment with THR agonists resulted in significant, dose-dependent increases in the liver gene expression that correlate well to a reduction in lipid levels.


Spotlight on NASH Model Design

11:00 am Analyzing NASH Model Designs & Their Translatability to the Clinic

  • Banu Cole Scientific Director, HemoShear Therapeutics, Inc.


• Learning how to model pathophysiological relevance
• Hearing how to effectively model clinical relevance.
• Understanding how to execute successful systemic / multiorgan modeling.

11:30 am Designing Robust Models Which Accurately Illustrate Cirrhosis Complications in Order to Accelerate Development of Targeted Therapeutics to Effectively Treat Late-Stage NASH


• Uncovering animal models which accurately replicate cirrhosis complications in NASH patients
• Deep diving into organoids for cirrhosis
• Modeling cirrhosis beyond models: what do biopharma need to know?


Hearing Tips & Tricks to Optimize the Design of Your Next NASH Trial

11:00 am Maximizing the Success of Your Next NASH Trial: Understanding What Physiological Factors You Must Monitor to Accurately Track Disease Progression & Predict Liver Function Restoration When Testing Novel Therapeutics


• Deep diving into metacrines FXR platform overview
• Reviewing the phase 2 program
• Analyzing considerations for translation to biopsybased clinical trials

11:30 am Looking into the Crystal Ball: What Methods Should You Use to Detect & Record the Efficacy of Your NASH Drug in the Patient Population Once Approved?


• Critically analyzing why NASH trials fail
• Discussing “lessons learned” from current and ongoing NASH clinical trials
• Investigating the role and impact of non-invasive tests to demonstrate efficacy

12:00 pm Lunch & Networking

1:00 pm C-Suite Discussion: Investigating Non-Conventional Approaches & Mechanisms of Actions to Address NASH: Spotlight on Gene Therapy, Mitochondria Based Therapeutics (MBTs), Regenerative Medicine, & RNA Therapeutics


• How to apply lessons learned from the application of gene therapy tools for rare diseases to strategize a novel approach to NASH treatment
• Investigating how mitochondria based therapeutics (MBTs) provide a differentiated mechanism of action to treat NASH patients
• Understanding how to utilize regenerative medicine to systematically address critical challenges in NASH drug development
• Exploring the latest research on mRNA therapies for NASH

1:30 pm What the Latest Advances in Our Understanding of NASH Pathogenesis & Pathophysiology Can Tell Us About Past & Future Therapeutic Approaches


• Outlining our understanding of the pathogenesis and pathophysiology of NAFLD and NASH, from the molecular to the whole-body level
• Hearing what clinical trials in NASH have taught us about different therapeutic targets and pathways, and why “what you measure” matters
• Integrating the latest basic, translational, and clinical research to improve NASH therapeutic target selection

1:00 pm Striking the Balance Between Quality & Efficiency: How to Devise a Fool Proof Plan to Maximize Convenience & Minimize Errors When Creating NASH Models for Biopharma


• Describing two novel pathways/first-in-class molecules with potential in NASH
• Exploring alternative approaches to increasing the probability of success of translation to humans and leveraging available clinical data from a related molecule
• Utilizing the 505(b)(2) regulatory pathway

1:45 pm Audience Discussion: Maximizing the Partnership Between Industry & Academia in the Context of NASH Drug Development to Upgrade Existing Models


The significance and importance of academic research for NASH model development cannot be stressed enough and every year, exciting progress is made by academics across the globe to help improve models for drug developers. Join a small group of your peers at this focused audience discussion to discuss your experience with partnering with academia and lave with the knowledge you need to develop meaningful relationships with academics to help optimize your NASH model development in 2021 and beyond.

1:00 pm TVB-2640, a Novel First In Class Fatty Acid Synthase (FASN) Inhibitor: Metabolomics Can Predict Clinical, Robust MRI-PDFF Response


• FASN inhibition decreases liver fat and markers of inflammation and fibrosis in Phase 2 NASH
• Correlation of non-invasive tests with liver fat clinical response
• Investigation of serum metabolomic markers to predict liver fat clinical response

1:30 pm Exploring Patient Perspectives on NASH Drug Development

  • Jeff McIntyre Advocate for Public Health, Public Policy & The Public Interest , Global Liver


• Understanding the importance of patient involvement in clinical trial design and protocol review
• Learning why it’s important to prioritize consistent messaging and planning via GLI’s Language of NASH and US NASH Action Plan
• Uncovering insights from GLI’s recent FDA NASH Patient-Focused Drug Development Symposium and NASH patient survey

2:00 pm Afternoon Break & Networking

Uncovering the Emerging Consensus on Single, Dual & Triple NASH Therapy Strategies

2:30 pm Panel Discussion: Comparing & Contrasting Single, Dual & Triple NASH Therapy Treatment Options for NASH


• Outlining the relative benefits & drawbacks of combination therapies in comparison to monotherapies – are these the future of NASH treatment?
• Examining the latest research on combination therapies to understand how to identify which factors you must consider when deciding to combine separate drugs when treating NASH patients.
• Designing a combination therapy from scratch: How should you approach the development of a combination therapy from the discovery stage as oppose to further on in the drug development process.

3:00 pm Discovering the Future State of Combination Treatment for NASH


• Deep diving into the limitations of single agent treatments
• Understanding the rationale for combination treatment
• Investigating current and future combination approaches

3:30 pm Combination efficacy of a GLP-1R agonist, FXR agonist & ACC inhibitor in preclinical models of NASH & Fibrosis


• Exploring combination approach for NASH therapy
• Analyzing preclinical models of NASH in the context of single, dual and triple treatment approaches
• Addressing underlying metabolic disease as a therapy for NASH

Discovering How Liver Function Impacts NASH Drug Development

4:00 pm Normalization of Liver Fat Following Treatment with Efruxifermin & Relationship with Histologic & Metabolic Improvements


• Learning about the six-fold higher probability of NASH resolution with liver fat normalization
• Investigating the correlation between change in liver fat and decrease in markers of liver stress, injury, and restoration of metabolic health
• Analyzing the consistent improvements in fibrosis as assessed by serum markers, imaging, and histology in NASH patients with F1-F4 fibrosis

4:30 pm Drug Induced Liver Injury (DILI): Impacts on Liver Functionality & Influence on NASH Drug Development

  • Ramy Younes Hepatologist & Clinical Program Leader NASH/Metabolism, Boehringer Ingelheim


• Uncovering why DILI is a common liver issue in clinical trials
• Revealing the reasons why NASH patients have different level of alteration of liver enzymes
• Appreciating the fact that the assessment of DILI in NASH trials requires careful planning and evaluation

5:00 pm End of Day Two