Targeting NASH


( 非酒精性脂肪性肝炎(NASH)标的化 )

Non-alcoholic steatohepatitis (NASH) is a disease whose incidence is rising and is related to an accumulation of fat in the liver that can lead to its disfunction due to excessive inflammation and fibrosis. No medical treatments yet exist for NASH but it’s a hopeful time for the field because several drug candidates are in Phase II and III clinical trials. New NASH drug targets are also being revealed due to progress in the fields of NASH contributors: metabolic disfunction, inflammation and fibrosis. Significant challenges remain, however, such as the need for non-invasive biomarkers and better models for the disease. At Cambridge Healthtech Institute's Targeting NASH conference, join academic and industry investigators to learn and discuss with one another drug development progress, challenges and solutions in the arena of treating fatty liver disease.

Choose 2 Short Courses and 2 Conferences/Training Seminars
September 16 Pre-Conference Short Course: SC1: Immunology Basics: Focusing on Autoimmunity and Cancer
September 17-18 Conference: Targeting NASH
September 18 Dinner Short Course: SC9: Targeted Protein Degradation Using PROTACs, Molecular Glues and More
September 18-19 Conference: Targeting Fibrosis

Final Agenda


1:00 pm Pre-Conference Short Course Registration
Click here for details on short courses offered.


7:00 am Registration Open and Morning Coffee

NASH Drug Candidates

8:00 Organizer's Welcome Remarks

8:05 Chairperson’s Opening Remarks

Claus Kremoser, PhD, CEO, Phenex

8:10 FEATURED PRESENTATION: Thyroid Hormone Receptor Agonists

Taub_RebeccaRebecca Taub, MD, CMO & President, R&D, Madrigal Pharmaceuticals

I will present data from clinical studies of resmetirom (MGL-3196). MGL-3196 is an orally administered, small-molecule, liver-directed compound that is currently in Phase III development for NASH. The data show highly significant reduction of liver fat and biomarkers of inflammation and fibrosis and resolution of NASH on liver biopsy in a 36-week serial liver biopsy study.

8:40 FEATURED PRESENTATION: Parallel Development of Elafibranor and an in vitro Diagnostic (IVD) to Identify Patients for Drug Therapy

Suneil Hosmane, PhD, Executive Vice President, Strategic Development, Genfit

Elafibranor is a first-in-class PPARα/δ agonist which has demonstrated in a Phase 2b study NASH resolution without the worsening of fibrosis while also improving cardio-metabolic risk. Furthermore, NASH resolution correlated with fibrosis improvement. Elafibranor is safe, tolerable and is now being investigated in Phase III. Additionally, GENFIT is developing a blood-based in vitro diagnostic to identify NASH patients who are at risk of disease progression and should be considered for therapeutic intervention – a key unmet clinical need.

9:10 Targeting GLP-1 for NASH

Conde-Knape_KarinKarin Conde-Knape, PhD, Corporate Vice President, Cardiovascular and Liver Disease Research, Novo Nordisk

GLP1 receptor agonists have been successfully positioned for the treatment of diabetes and obesity. It has been documented that weight loss either by dietary or surgical intervention leads to improvement in NASH and fibrosis. Initial clinical data suggests a beneficial effect of GLP1 receptor agonists in NASH clinical trials. An overview of GLP1 receptor agonism in the treatment of NASH and future directions will be provided.

9:40 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing

NASH Therapeutic Combinations

10:25 Combinations with ACC Inhibitor for Treating NASH

Archana Vijayakumar, PhD, Research Scientist, Fibrosis, Gilead

Firsocostat (FIR), a liver-targeted acetyl-CoA carboxylase inhibitor (ACCi), improves hepatic steatosis and liver biochemistry in NASH patients, but may increase plasma TGs in patients with pre-existing hypertriglyceridemia. This is likely mediated by repression of PPARα activity. In diet-induced obese mice, co-administration of fenofibrate, a PPARα agonist, with a liver-targeted FIR analogue ACCi completely normalized elevations in plasma TGs, and improved liver metabolism. The data suggest that ACCi/fenofibrate combination may improve NASH efficacy more than either monotherapy.

10:55 CC-Chemokine Receptor Antagonism as a Therapeutic Target in NASH

Star Seyedkazemi, PharmD, Associate Vice President, Clinical Development, Liver Therapeutic Area, Allergan


11:25 Combination Therapy for NASH

Pedrosa_MarcosMarcos Pedrosa, MD, MPH, Global Program Clinical Head, Therapeutic Area Hepatology and Transplantation, Novartis Pharma AG

Several drugs are currently in development for NASH. Beyond monotherapy, there is also an opportunity for combination therapy. Targeting different pathways in this complex disease with many different phenotypes may provide better clinical outcomes. Also increase the possibility for a more personalized treatment, i.e. precision medicine.

CrownBio 11:55 Accelerating NASH in an Improved Translational Model: MS-NASH Mouse on Western Diet and CCl4

Guodong Zhang, PhD, Director, Cardiovascular and Metabolic Disease, Crown Bioscience

Enhanced models are needed to improve NASH drug development, combining the severe pathology and dysmetabolism seen clinically. Discover the MS-NASH mouse on Western diet, fructose, and CCl4, a new preclinical NASH model combining both severe fibrosis and dysmetabolism in an accelerated timeframe. Explore model characterization and outcomes following OCA treatment.

12:10 pm Sponsored Presentation (Opportunity Available)

12:25 Session Break

12:35 Luncheon Presentation (Sponsorship Opportunity Available) or Enjoy Lunch on Your Own

1:15 Refreshment Break in the Exhibit Hall with Poster Viewing

NASH Drug Development Challenges

1:50 Chairperson’s Remarks

Kendra K. Bence, PhD, Senior Director, Metabolism, Internal Medicine Research Unit (IMRU), Pfizer, Inc.

1:55 Are Circulating Fibrosis Biomarkers Useful in NASH Drug Development?

Saurabh Gupta, PhD, Director, Translational Medicine and Early Clinical, Takeda Pharmaceuticals International Co.

Clinical Diagnosis of NASH and evaluation of anti-fibrotic activity in clinical trials heavily relies on the histological readouts based on liver biopsy, a highly invasive, variable and a non-representative technique. We will summarize soluble biomarkers which have shown most promising results in terms of fibrosis and NASH staging, and measuring the anti-fibrotic activity in clinical trials.

2:25 Federal Landscape for NASH Patients and Products

Thornhill_BarrettBarrett Thornhill, JD, Executive Director, NASH Alliance

Washington, DC has become the confluence of products, policy, pricing and access, but NAFLD-NASH is a virtual unknown among federal policymakers. The now-silent public health crisis will grow dramatically in coming years, and the NASH community of clinicians, innovators and patients is developing the public health infrastructure to better understand NASH implications and support product commercialization. This session will provide an overview of these efforts and explore how federal programs can be a ‘pull incentive’ that bolsters product development.

2:55 Precision Metabolomics: Understanding the Complexity of NAFLD/NASH

Kari Wong, Senior Study Director, Metabolon, Inc.

Disease drivers including genetics, environmental cues and microbiota drive the development of nonalcoholic-steatohepatitis(NASH).  The constellation of factors inciting NASH has spawned a remarkable array of targets.  A tool for augmenting the challenges of R&D programs is Metabolon’s Metabolomics platform. By identifying 1000+ metabolites in a single sample across pathways that underly NASH pathogenesis, key insights of target/molecule combinations can be illuminated with this platform.  This approach can be applied at any stage of drug development.

3:25 Refreshment Break in the Exhibit Hall with Poster Viewing and Poster Competition Winner Announced

4:05 Drug Development for NASH Cirrhosis

Traber_PeterPeter Traber, MD, Partner, Alacrita Consulting; Adjunct Professor of Medicine, University of Pennsylvania School of Medicine

NASH is a chronic, slowly progressive inflammatory and fibrotic disease of the liver which progresses in some individuals to cirrhosis with its attendant complications, including death and liver transplant. In this presentation, the differences in the pathophysiology and impact on patients between precirrhotic and cirrhotic NASH will be reviewed. Additionally, acceptable and potential regulatory endpoints for clinical trials will be reviewed and put in the context of current ongoing development programs. The publicly disclosed information on established clinical trial programs in cirrhotic NASH will be reviewed and compared, and thoughts about future clinical development for NASH cirrhosis will be discussed.

4:35 Translational Challenges in NASH

Geddes_BradBrad Geddes, PhD, Senior Director, Innate Immunity Research Unit, GSK

This presentation will focus on recent FDA guidance regarding fibrosis-centric primary endpoints for NASH clinical trials and its impact on preclinical development of potential NASH therapeutics.


5:05 Interactive Breakout Discussion Groups - View All Breakouts

Join a breakout discussion group. These are informal, moderated discussions with brainstorming and interactive problem solving, allowing participants from diverse backgrounds to exchange ideas and experiences and develop future collaborations around a focused topic.

Animal Models for NASH

Moderator: Bryan C. Fuchs, PhD, Assistant Professor of Surgery, Harvard Medical School

  • Re-evaluating NASH animal models in the context of the new FDA guidelines for NASH cirrhosis
  • Which noncirrhotic NASH animal models have the most representative histology?
  • Are there NASH cirrhosis animal models that develop complications?
  • Which biomarkers will be reflective of improvements in histology or predictive of complications?

Efficacy of MOA in the Treatment of NASH

Moderator: Karin Conde-Knape, PhD, Corporate Vice President, Cardiovascular and Liver Disease Research, Novo Nordisk

  • What is the decrease in liver fat telling us?
  • What will be the needed efficacy to make a difference to patients?
  • Are we any closer to having surrogate markers of efficacy?

Cirrhosis and NASH

Moderator: Peter Traber, MD, Partner, Alacrita Consulting; Adjunct Professor of Medicine, University of Pennsylvania School of Medicine

  • Targets in common between chirrhosis and NASH
  • Challenges in treating cirrhotic NASH
  • Regulatory guidance for cirrhosis NASH trials

6:05 Welcome Reception in the Exhibit Hall with Poster Viewing (Sponsorship Opportunity Available)

7:10 Close of Day


7:30 am Registration Open and Morning Coffee

New Drug Targets Or Earlier Stage Compounds For Fatty Liver Disease

8:00 Chairperson’s Remarks

Weilin Xie, PhD, Senior Principal Scientist, Biotherapeutics, Celgene

8:05 Targeting Integrin αVβ1 for the Treatment of Liver Fibrosis Associated with NASH

Lefebvre_EricEric Lefebvre, PhD, CMO, Pliant Therapeutics

Fibrosis is a common pathway for progression of many debilitating diseases associated with loss of organ function. Integrins play a key role in regulating TGF-β activation and cell-matrix interactions, and thus represent attractive antifibrotic targets. We evaluated small molecule integrin inhibitors with different selectivity profiles in lung, liver and kidney models of injury and fibrosis, in tissue slices from patients with lung and liver fibrosis, as well as assessed non-invasive in vivo biomarkers of target engagement.

8:35 Targeting Fructose Metabolism: Update on KHK Inhibitor for NASH

Bence_KendraKendra K. Bence, PhD, Senior Director, Metabolism, Internal Medicine Research Unit (IMRU), Pfizer, Inc.

Excessive fructose intake leads to increased energy storage in the form of fat as well as accumulation of pro-inflammatory metabolites, both important components in the development of NAFLD and NASH. To understand the specific role of fructose in T2DM and NAFLD/NASH, we designed and tested a potent, novel, orally-bioavailable inhibitor of ketohexokinase (KHK), the primary enzyme which catalyzes the conversion of fructose to fructose-1-phosphate (F1P) in the first step of fructose metabolism.

WuXi9:05 Population-scale Human Genetics and Genomics for NASH Drug Target & Biomarker Discovery

Williams_RIchardRichard Williams, MBBS, PhD, Global Head, Medicine, WuXi NextCODE

New drug targets & biomarkers are desperately needed for effective NASH therapy, monitoring & prevention.

In our global NASH discovery programs, we are (1) using whole genome sequencing (WGS) to compare thousands of patients with high fibrosis NASH, Low or No fibrosis NASH & NAFL, and population controls, and (2) interrogating hundreds of fresh liver biopsies from NASH & NAFL patients and control individuals with ‘bulk’ multi-omic genomic analysis (WGS, RNA-Seq, DNA methylation), single cell RNA-seq and fully-integrated AI to reveal novel disease biology, drug targets and biomarkers.

9:35 Coffee Break in the Exhibit Hall with Poster Viewing

10:20 Liver X Receptor (LXR) Agonists PX665 for NASH

Kremoser_ClausClaus Kremoser, PhD, CEO, Phenex

The liver X receptor is a nuclear hormone receptor and is therefore similar to other promising NASH targets such as PPARs and FXR. LXRs regulate cholesterol, fatty acid and glucose levels in the cell. We present our results on an LXR inverse agonist, PX665, which combines antisteatotic, weight lowering, antifibrotic and insulin sensitizing properties in one approach to combat NASH.

10:50 An Antisense DGAT Inhibitor for NASH

Morgan_ErinErin Morgan, Executive Director, Clinical Development, Ionis Pharmaceuticals

DGAT2 catalyzes the terminal step in the synthesis of triacylglycerols from de novo synthesized fatty acids and newly formed diglycerides. In pre-clinical models of NAFLD, we previously reported that specific inhibition of DGAT2 caused a marked improvement in hepatic steatosis. The talk will discuss results with a Novel antisense inhibitor of diacylglycerol acyltransferase 2 (IONIS-DGAT2RX) administered in NAFLD patients with type 2 diabetes. Results from the international randomized placebo-controlled Phase 2 trial demonstrated that IONIS DGAT2RX significantly improved hepatic steatosis without causing hypertriglyceridemia or affecting hepatic function in type-2 diabetes mellitus patients with steatosis, supporting further development of IONIS- DGAT2RX in NASH patients.

11:20 Enjoy Lunch on Your Own

11:20 Conference Registration for Programs 1B-7B

Click here for full abstracts.

12:20 pm Event Chairperson’s Opening Remarks

An-Dinh Nguyen, Team Lead, Discovery on Target 2019, Cambridge Healthtech Institute


12:30 Plenary Keynote Introduction

Anjan Chakrabarti, Vice President, Discovery Chemistry, Syngene International Ltd

12:40 Base Editing: Chemistry on a Target Nucleotide in the Genome of Living Cells

David R. Liu, PhD, Howard Hughes Medical Institute Investigator, Professor of Chemistry & Chemical Biology, Harvard University



1:20 PROTACs: Past, Present, and Future

Craig M. Crews, PhD, Professor, Chemistry; Pharmacology; Molecular, Cellular & Developmental Biology; Yale University



2:00 Close of Plenary Keynote Program

2:00 Dessert Break in the Exhibit Hall with Poster Viewing

2:45 Close of Targeting NASH Conference
Please click here to continue to the agenda for Targeting Fibrosis

* 活动内容有可能不事先告知作更动及调整。

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