Conference Day Two
Thursday, September 26, 2024

7:30 am Check-In

8:25 am Chair’s Opening Remarks

8:30 am Utilizing RWD/RWE to Improve MASH Drug Development Efficiency

Synopsis

  • Detailing the RWD/RWE regulatory landscape including FDA, EMA, PMDA and CDE
  • Navigating global regulatory harmonization and convergence
  • Utilizing case studies to predict future trends

9:00 am Session Reserved for Perspectum

9:30 am Exploring Biomarker Developments for Assessing Liver Fibrosis

Synopsis

  • Overcoming inherent limitations of liver biopsy and histological analysis through non-invasive tool
  • Assessing potential of protein localization
  • Exploring Visium Spatial and Xenium

10:00 am Morning Break & Networking

Track 1: Preclinical & Translational

Navigating the Heterogeneity of MASH to Optimize Preclinical Success

10:45 am Elucidating the Potential of siRNA Approaches for MASH

Synopsis

  • siRNA therapies can modulate targets that were previously considered un-druggable by conventional small molecule or large molecule approaches
  • A GalNAc-SiRNA therapeutic approach is a novel modality to specifically target a single gene in the liver without impact on non-hepatic tissues, leading to a good safety profile

Track 2: Clinical & Regulatory

Utilizing Machine Learning & PPARs to Accelerate MASH Development

10:45 am Leveraging Quantitative Tools to Integrate Preclinical and Clinical Data to Advance MASH Drug Development

Synopsis

  • Integrating preclinical and clinical data via mathematical modeling predicts pharmcoldynamic effects on MASH biomarkers
  • Mathematical models also characterize the relationship between MASH biomarkers and histological endpoints
  • AI/ML techniques aiding participant selection and test model predictions to reduce screen failures

11:15 am The Potential of Pan-PPAR Agonist Therapy to Address Inflammation and Fibrosis in Patients with MASH – a Path to a Holistic Approach

Synopsis

  • Delving into the anti-fibrotic and anti-inflammatory impacts
  • Assessing developments from Phase 3 trials

11:45 am
Lunch

12:45 pm Delving into Portal Fibroblast Ablation to Treat Cholestatic Fibrosis

Synopsis

  • Activated Portal Fibroblasts (aPFs) and Hepatic Stellate Cells (aHSCs) contribute to populations of myofibroblasts in response to cholestatic liver fibrosis. Ablation of myofibroblasts with cellspecific immunotoxins or CART cells has been suggested as a potential immunotherapy of liver fibrosis
  • Ablation of aPFs suppresses cholestatic liver fibrosis without causing mortality or distress in mice and can be used for treatment of cholestatic liver fibrosis
  • Ablation of HSCs suppresses liver fibrosis but affects hepatocyte proliferation causing a defect in liver regeneration. Total ablation of HSC population cannot be used for anti-fibrotic therapy

1:15 pm Roundtable | Navigating MASH Model Development

Synopsis

A practical and highly interactive breakout roundtable session where attendees can crowd-source and share opinions around assigned topic areas:

  • How can model translatability be maximized?
  • How to achieve fibrotic models in conjunction with established metabolic models

12:45 pm Exploring the Need for Regulatory Guidance for Decompensated MASH

Synopsis

  • Analysing the need for guidelines for this critical population
  • Considering necessary updates to account for differences in endpoints
  • Guidance on patient selection and risk stratification

1:15 pm Roundtable | Optimizing MASH Drug Development

Synopsis

A practical and highly interactive breakout roundtable session where attendees can crowd-source and share opinions around assigned topic areas:

  • Exploring key learnings and takeaways from regulatory bodies to inform future direction
  • Exporting learnings from past clinical trials to optimize trial design and patient engagement

1:45 pm
Afternoon Break

Showcasing Antifibrotic Progress, Past Learnings & Innovations to Supercharge MASH Drug Development

2:15 pm Spotlighting Clinical Data Supporting Anti-Fibrotic Mechanisms of EFX

Synopsis

  • Detailing direct and indirect antifibrotic activity of EFX shown clinically
  • Improving insulin sensitivity and restoring lipoprotein profile to restore metabolic health and achieve sustained responses
  • Dissecting the implications of these findings on long term clinical outcomes

2:45 pm Harmonizing Patient Engagement & Patient Centered Clinical Trials

Synopsis

  • Evaluating efforts to drive patient focused drug development
  • Promoting diversity in MASH trials to increase access and representation
  • Exploring the impact of recent approvals on the patient journey

3:15 pm Detailing the Best Practices for Patient Engagement

Synopsis

  • Utilizing case studies from a MASH power council
  • Exemplifying best code of practise
  • Implementing strategies to enhance patient-focus drug development

3:45 pm Panel Discussion: What’s Next for MASH Drug Development: Deep Diving into Innovations in the Field

Synopsis

  • What are the future directions of MASH drug development?
  • From GLP agonists, RNA therapies, AAV and SNPs which of these therapies hold the most promise
  • Which pipelines should be prioritized?

4:15 pm Chair’s Closing Remarks