8:15 am Morning Refreshments
Reviewing Preclinical Studies & Clinical Progress to Bridge the Gap to Enhance the Translation of MASH Therapies
8:55 am Chair’s Opening Remarks
9:00 am Formulating Innovative & Patient Centred Phase 3 Trials to Improve Results for Translating More Drugs to Clinic
Synopsis
- Designing Phase III MASH trials to integrate robust methodologies and evaluating efficacy and safety across diverse patient populations, accelerating the path to regulatory approval to bring therapies to patients faster
- Incorporating non-invasive testing throughout Phase III trials to reduce patient burden and enhance data collection efficiency, leading to more comprehensive and patient-friendly assessments of treatment response
- Implementing patient retention strategies to ensure sustained participation in long-term Phase III studies, providing more complete and reliable data for demonstrating durable treatment benefits
9:30 am Leveraging Noninvasive Markers of Fibrosis Alongside Serial-Biopsy Clinical Studies of Efruxifermin to Elucidate Breadth of Response Across Advanced Fibrosis & Cirrhosis Due to MASH
Synopsis
- Characterization of disease progression in untreated patients through longitudinal assessments in long-term studies
- Rapid and sustained effects of efruxifermin on fibrotic pathways to arrest progression and reverse fibrosis in MASH patients with advanced fibrosis and compensated cirrhosis
- Corroboration of improved liver health and underlying disease drivers as assessed by multiple measures of liver injury, fibrosis, and metabolic parameters
10:00 am Navigating the Path to the First MASH Therapeutic Approval & Progressing Beyond to Advance Clinical Development Strategies to Expand the Horizon of Approved Drugs
Synopsis
- Delving into the strategic clinical development and trial design methodologies that led to the unprecedented first therapeutic approval in MASH, setting a crucial benchmark for future candidates
- Reviewing the comprehensive clinical data and real-world evidence to demonstrate the significant efficacy and safety profiles that underpin the treatment’s success and inform optimal patient management
- Exploring how lessons learned from Madrigal’s development journey, alongside ongoing research, are influencing the design of subsequent clinical trials and accelerating the pipeline for next-generation MASH therapies
10:30 am Morning Break & Networking
Employing Cell Targeting Approaches to Enhance Liver Cell-Specific MASH Treatment Precision & Minimize Off-Target Effects
11:15 am Reducing & Reversing Liver Fibrosis Using Targeted Elimination of Pathogenic Stellate Cells
Synopsis
- Identifying pathogenic stellate cells using advanced markers for targeting the primary drivers of liver fibrosis
- Developing antibody drug conjugate for the selective delivery of payloads directly to pathogenic stellate cells to minimize off-target effects
- Establishing the in vivo pharmacokinetic and pharmacodynamic relationship for optimizing dosing, predicting efficacy, and ensuring safety in reducing liver fibrosis
11:45 am Identification of Targeting Pathogenic Cells for Fibrotic Disease
Synopsis
- Utilization of ‘omics to identify pathogenic cell types, mechanisms, and targets
- Methods to validate transcriptionally identified targets for development of therapeutics
- Applying novel therapeutic approaches to target unique pathogenic cell populations
12:15 pm Lunch
1:15 pm Roundtable Discussion: Adapting Trial Methodologies to Reflect Real-World MASH Populations to Broaden Treatment Applicability
Synopsis
- Implementing more inclusive trial designs that incorporate diverse patient populations to ensure trial results are generalizable to a broader range of MASH patients to lead to more effective and equitable treatment strategies
- Utilizing real-world data (RWD) and patient registries to provide valuable insights into disease progression and treatment outcomes in routine clinical practice to complement traditional trial data and enhance the development of patient-centred treatment approaches
- Exploring novel endpoints beyond traditional histology to capture the full spectrum of MASH-related outcomes, including patient-reported quality of life and long-term complications to facilitate the development of therapies that address the holistic needs of MASH patients
Harnessing GLP-1s to Maximize Treatment Effectiveness to Expand the MASH Therapeutic Potential
2:00 pm Roundtable Discussion: Navigating the Future of MASH Drug Development in the GLP-1 Era From Biopsy to Biomarker
Synopsis
- A Paradigm Shift in Trial Design – The recent FDA acceptance of a non-invasive biomarker as a surrogate endpoint is a pivotal moment for drug development, which is set to transform clinical trials by reducing patient burden, improving recruitment and retention, and accelerating the path to regulatory approval for new therapies
- The New Therapeutic Foundation – The approval of GLP-1s for MASH by Novo Nordisk establishes this class of therapies as a core component of treatment, meaning future drug development strategies must now consider how to build upon the benefits of GLP-1s, focusing on combination or sequential therapies to achieve more complete disease resolution and patient outcomes
- Raising the Bar for Efficacy – The combination of GLP-1 success and the new FDA guidance signals a higher standard for what constitutes a successful MASH therapy so moving forward, developers will be challenged to design trials that not only address liver fibrosis but also demonstrate clear, quantifiable improvements using non-invasive methods, ultimately de-risking their pipelines