Tuesday, February 25, 2025
8:00 am
MORNING REFRESHMENTS & CHECK IN
WORKSHOP STREAM A: TRANSLATIONAL BIOMARKERS & CLINICAL UTILITY
8:30 am WORKSHOP A
Optimizing Clinical Endpoints in Neuroimmunology Drug Development: Bridging the Gap from Preclinical Promise to Clinical Success
Synopsis
In the evolving field of neuroimmunology, defining and validating clinical endpoints is crucial for translating preclinical findings into successful therapies. This workshop will focus on the strategic selection and application of clinical endpoints to accurately assess efficacy and safety in neuroimmunology drug development. Participants will explore how to align endpoints with the unique challenges of neuroinflammatory diseases, ensuring they reflect meaningful clinical outcomes and patient benefits. The workshop will also address how innovative biomarkers and multi-modal data integration can enhance endpoint selection and interpretation.
Key Takeaways:
- End Point Selection Strategies: Learn best practices for choosing and validating clinical endpoints that align with disease mechanisms and therapeutic goals in neuroimmunology.
- Innovative Biomarkers Integration: Understand how emerging biomarkers, including imaging, proteomics, and genomics, can inform and refine clinical endpoints to provide more precise assessments of drug efficacy and safety.
- Data Interpretation and Translation: Gain insights into interpreting complex multi-modal data and translating preclinical results into relevant clinical outcomes, improving the likelihood of regulatory approval and clinical success
WORKSHOP STREAM B: OMICS DISCOVERY & PRECLINICAL MODELS
8:30 am WORKSHOP B
How Can We Best Utilize In Vivo & In Vitro Models for Clearer Insights on Efficacy & Safety of Neuroinflammatory-Targeted Agents for More Confident Translation to the Clinic?
Synopsis
This session explores how advanced in vivo and in vitro models, including microfluidic-based coculture systems and multiomic approaches, are revolutionizing the preclinical evaluation of neuroinflammatory-targeted agents. By simulating human brain conditions and leveraging AI-driven data analysis, these models offer clearer insights into efficacy
and safety, providing more confidence for clinical translation.
Key Takeaways:
- Examine the progress of microfluidic-based coculture systems that simulate human brain conditions and their potential to complement mouse models.
- Discuss how these sophisticated models enhance the accuracy of preclinical evaluations and build confidence for subsequent clinical trials
- Explore the use of multiomic approaches—incorporating metabolomics, proteomics, transcriptomics, and spatial dynamics—to provide comprehensive insights into neuroinflammatory processes.
- Assess how these approaches contribute to a deeper understanding of target agents and their effects at a molecular level
- Investigate how integrating machine learning and AI can address the challenges posed by complex and large-scale data sets.
- Learn about strategies for utilizing these technologies to uncover nuances and enhance data interpretation in neuroinflammatory research
11:00 am
SHORT COMFORT BREAK
11:15 am WORKSHOP C
Patient Stratification for Precision Medicine In Neuroscience: How Can We Ensure The Right Patients Are Enrolled On The Right Drugs To Treat Their Specific Disease Pathology?
Synopsis
In a setting where our understanding of disease heterogeneity and subtyping in neuroscience is getting more clearly defined, the role of stratification biomarkers becomes more clearly crucial to clinical trial outcomes.
Key Takeaways:
- Developing biomarkers has to go hand in hand with drug development to develop more sensitive tools to monitor function, cognition etc in a human.
- Using earlier screening process to stratify patients with neuroinflammatory phenotype earlier in disease to ensure greater success in trials
- Harnessing a matrix of endpoints to identify neuroinflammation including cytotoxins, GFAP, soluble TREM2 as more specific markers, NFL, synaptic markers (neurogranulin, Snap 25 and amyloid tau), as well as functional outcomes, cognition and daily living
- Evaluating biomarker utility across different diseases and types of neuroinflammation
*Lunch will be served during this session at 12:30
11:15 am WORKSHOP D
Leveraging Multiomics for Target Discovery and Drug Development in Neuroinflammation: From Bench to Bedside
Synopsis
This session highlights the transformative role of multiomics, particularly spatial transcriptomics, in neuroinflammation drug discovery. We will explore how these cutting-edge technologies enhance target identification, validation, and translation into clinical applications. Additionally, the session addresses the challenges of current preclinical models and the integration of spatial transcriptomics with other omics and AI-based approaches to improve drug development processes.
Key Takeaways:
- Understand how spatial transcriptomics can identify novel targets in human brain samples by mapping gene expression and protein stress in relation to neurodegeneration and cognitive decline
- Learn about the limitations of traditional preclinical models and how spatial transcriptomics can provide a more accurate representation of human disease states
- Discuss the challenges associated with preserving spatial information and avoiding technical artifacts.
- Explore solutions for maintaining sample integrity and ensuring reliable data generation
- Evaluate the potential of integrating spatial transcriptomics with other omics technologies, including proteomics and AI-based image analysis, to enhance target nomination and validation processes
- Examine how spatial transcriptomics can bridge the gap between early-stage research and industry applications, focusing on high-throughput screening and validation methods
- Address the limitations of current models, such as 3D brain organoids and xenograft systems, and discuss emerging strategies for improving reproducibility and scalability in drug discovery
2:15 pm SHORT COMFORT BREAK
2:30 pm WORKSHOP E
Optimizing Drug Development Workflows: Leveraging Human Data, Experimental Evidence & Known Biology for Improved Target Identification
Synopsis
This session focuses on refining neuroimmunology drug discovery workflows by integrating multiomics, genetics, and clinical data with novel experimental models. Attendees will explore strategies for improving CNS target identification, screening, and prioritization, with the goal of increasing the probability of success in drug development.
Key Takeaways:
- Learn how to effectively use multiomics studies, genetics, and clinical data for CNS target identification.
- Explore novel CNS experimental models to enhance target screening and validation.
- Understand methods to prioritize and rank targets based on known CNS disease biology, leveraging existing literature and databases.
*Afternoon break & refreshments will be served during this session at 3:30