Non-alcoholic Steatohepatitis (NASH) is the result of a fatty liver that starts to undergo fibrosis whose scarring can eventually lead to a liver that is non-functional. The increasing global incidence of NASH may be linked to the concomitant rise in
diabetes, obesity and other metabolic disorders all over the world. No pharmaceutical treatment yet exists for NASH, but it is an area of active research and industry focus, with several treatments in late-stage clinical trials. However, many translational
challenges remain, such as non-invasive ways to measure disease progression and response to treatment and best ways to model the disease outside of the patient. Join fellow drug discovery researchers working in the area of liver disease and/or fibrosis
to stay abreast of clinical and preclinical advances in the field and share insights on translational tools and approaches for spurring drug development progress.
Final Agenda
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Recommended All Access Package:
27 November: Organ-on-a-Chip and MicroPhysiological Systems
27 November Dinner Course: SC1: Immunology Basics for Drug Discovery, Part 1: Immune System Overview
28-29 November: NASH and Fibrosis: Translational Research and Strategies
29-30 November: Induced Pluripotent Stem Cells
29 November Dinner Course: SC4: Immunology Basics for Drug Discovery, Part 2: Immune-Oncology and Autoimmunity
Wednesday 28 November
7:00 Registration and Morning Coffee
8:50 Welcome Remarks
Anjani Shah, PhD, Conference Director, Cambridge Healthtech Institute
8:55 Chairperson’s Remarks
Rebecca Taub, MD, CMO & Executive Vice President, R&D, Madrigal Pharmaceuticals
9:00 Development of Elafibranor - a Dual PPARα/δ Agonist for the Treatment of NASH
Robert Walczak, PhD, EVP, Head of Research, Genfit
Elafibranor is a first-in-class PPARα/δ agonist which has demonstrated in a Phase IIb 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. NASH is a multifactorial disease and it is increasingly acknowledged that combination therapies with drugs with complementary mechanisms of action will be required
to increase the proportion of patients that reach all treatment goals. Recent data from studies in disease models indicate that Elafibranor is a universal backbone for combination drug therapies in NASH.
9:30 Metabolomic Profiling to Identify Molecular
Determinants of Liver Disease
Nicolas Schauer, PhD, Managing Director Metabolon, Inc.
A diverse set of disease drivers including genetics, environmental cues, microbiota metabolism and lifestyle influences come together resulting in an uncertain natural history of NAFLD/NASH. Metabolomics provides a comprehensive picture of the metabolome
and maps metabolites to their key biochemical pathways providing a holistic assessment of complex diseases.
10:00 Grand Opening Coffee Break in the Exhibit Hall with Poster Viewing
10:45 Thyroid Hormone Receptor Agonists
Rebecca Taub, MD, CMO & Executive Vice President, R&D, Madrigal Pharmaceuticals
I will present topline public data from our latest clinical study on MGL-3196, a β-selective thyroid hormone receptor (THR) agonist. MGL-3196 is an orally administered, small-molecule, liver-directed compound that is currently in Phase II development
for NASH. The data show highly significant reduction of liver fat and biomarkers of inflammation and fibrosis at 12 weeks in a 36 week serial liver biopsy study.
11:15 KEYNOTE PRESENTATION: 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.
11:45 Enjoy Lunch on Your Own
13:45 Chairperson’s Remarks
Diane Shevell, PhD, Director, Clinical Biomarkers and Innovative Medicines Development, Bristol-Myers Squibb
13:50 Animal Models of NASH in Preclinical Drug Development
Iwona Ksiazek, PhD, Senior Investigator I, Chemical Biology & Therapeutics, Novartis Institutes for Biomedical Research
Despite the tremendous progress in development and characterization of animal models of NAFLD/NASH made over the last years, we still lack robust and appropriately validated preclinical models with proven clinical translatability. Advantages and limitations
of selected mouse NASH models used to test pharmacological agents will be discussed and a newly developed diet-induced obesity model of NASH with fibrosis will be presented, covering the diet, systemic metabolic and inflammatory milieu, as well as
the histological spectrum of human NASH disease and its application in drug discovery, including validation with drugs currently in clinical development for NASH.
14:20 LXR Inverse Agonists for the Treatment of NASH
Claus Kremoser, PhD, CEO, Phenex
Nuclear Receptor targeted drugs such as FXR, TRbeta or PPAR agonists have emerged as effective approaches to combat NASH but they all come with limitations. LXR is known as a functional counterplayer of FXR and as such, inhibiting LXR function by inverse
agonist ligands should yield similar effects than activating FXR. Animal data show that beyond strong anti-steatotic properties, LXR inverse agonists demonstrate novel, unprecendeted antidiabetic effects.
14:50 Interactive Breakout Discussion Groups
This session features various discussion groups that are led by a moderator/s who ensures focused conversations around the key issues listed. Attendees choose to join a specific group and the small, informal setting facilitates sharing of ideas and active
networking. Details on the topics and moderators are available on the conference website.
Animal Models for Fibrosis
Moderator: Iwona Ksiazek, PhD, Senior Investigator I, Chemical Biology & Therapeutics, Novartis Institutes for Biomedical Research
- Who has used what?
- Pros and cons of different models
- What looks promising
NASH Drug Development Challenges
Moderator: Rebecca Taub, MD, CMO & Executive Vice President, R&D, Madrigal Pharmaceuticals
- Role of biomarkers
- European v. FDA guidance
- Defining target population
16:00 Refreshment Break in the Exhibit Hall with Poster Viewing
16:45 NEW: Report-Back from Breakout Discussions
17:15 Navigating Liver-Derived Omics Data for Translational Research
Florian Nigsch, PhD, Senior Investigator I, Chemical Biology and Therapeutics, Data Science,Novartis Institutes for BioMedical Resesarch, Basel
Translational research is of paramount importance for drug discovery, and computational techniques can play a major role. This talk will focus on common challenges of computational translational liver research, and then provide some insights with specific
examples based on omics datasets of disease models, in vitro and in vivo, including single cell analyses of human liver tissue.
17:45 MTBL0036, a Promising, New Anti-NASH and Antifibrotic Candidate
Gabriel Baverel, PhD, President, Founder, CSO, Metabolys, Inc.
MTBL0036 is a small molecule, orally active with favorable PK characteristics, and safe. In the STAM mouse model of NASH, it greatly diminished the NAFLD Activity Score by drastically reducing inflammation and ballooning, the major drivers of fibrosis.
In mice fed a choline deficient amino acid defined high fat diet, it greatly ameliorated liver fibrosis. Unlike that of most anti-NASH candidates in development, its molecular target is not nuclear.
18:15 Welcome Reception in the Exhibit Hall with Poster Viewing
19:15 Close of Day
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Thursday 29 November
8:00 Registration and Morning Coffee
8:55 Chairperson’s Remarks
Iwona Ksiazek, PhD, Senior Investigator I, Chemical Biology & Therapeutics, Novartis Institutes for Biomedical Research
9:00 KEYNOTE PRESENTATION: Biomarkers to Assess the Impact of Therapeutics on Patients with Liver Fibrosis
Diane Shevell, PhD, Director, Clinical Biomarkers and Innovative Medicines Development,
Bristol-Myers Squibb
Non-alcoholic steatohepatitis (NASH), the most advanced form of non-alcoholic fatty liver disease (NAFLD), is characterized by steatosis with inflammation and liver cell injury, which can lead to liver fibrosis. Non-invasive, accurate biomarkers
are needed to identify patients at high risk for NASH and to monitor disease progression. The potential use of non-invasive biomarkers to characterize patients with NASH and their response to an investigational therapy will be presented.
9:30 Identification of NAFLD in Primary Care: Can Algorithms and Transient Elastography (TE) Predict Fibrosis?
Helena Cortez-Pinto, MD, PhD, Professor, Department of Gastroenterology, Faculty of Medicine of Lisbon
NAFLD is extremely frequent in primary care. However it is generally unrecognized or devalued by general practitioners. Although screening in the general population is not recommended, the rules of referral to specialists and the population to be
screened needs better definition. Definition of these groups, what are the best algorithms and role of TE to predict fibrosis severity will be presented.
10:00 Coffee Break in the Exhibit Hall. Last chance for poster viewing.
10:45 IL-1 beta Small Molecule Inhibitors for the Treatment of NASH
Juan J. Perez, PhD, Professor, Department of Chemical Engineering, Universitat Politecnica de Catalunya-BarcelonaTech
11:15 Modelling of NAFLD/NASH with Patient-Derived iPS Cells
James Adjaye, PhD, Professor,Director, Institute for Stem Cell Research and Regenerative
Medicine, Heidrich Heine University, Dusseldorf, Germany
Steatosis leading to NAFLD and NASH should be considered as a multifactorial metabolic disease. Studies based on rodents, patient liver-biopsies and serum have provided useful insights into the etiology of steatosis. Though useful, a better understanding
of disease mechanisms, biomarker discovery and drug development necessitates the use of hepatocyte-like cells differentiated from patient derived induced pluripotent stem cells. My talk will focus on genes and associated pathways implicated in
disease progression.
11:45 NEW: Close of Conference
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