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.

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

Rebecca Taub, MD, CMO & Executive Vice 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

Dean Hum, PhD, CSO and COO, 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

Karin 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 Combination Therapy for NASH

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

11:25 Combining CVC and Tropifexor to Treat NASH

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

11:55 Sponsored Presentation (Opportunity Available)

12:25 pm 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

Barrett 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 Presentation to be Announced

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

4:05 Drug Development for NASH Cirrhosis

Peter 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

Brad 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

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. Visit the conference website for discussion topics and moderators.

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

Eric 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

Kendra 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.

9:05 Sponsored Presentation (Opportunity Available)

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

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

Claus 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

Erin Morgan, Project Manager, 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

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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