Conference Day Two
8:00 Check-In & Coffee
8:45 Chair’s Opening Remarks
Refining Biomarker Clinical Readiness to Enable Better-Defined Trials & Accelerate Clinical Validation
9:00 Defining Context of Use to Anchor Biomarker Relevance & Streamline the R&D Pipeline
Synopsis
- Determining Context of Use: Diagnostic, prognostic, predictive and pharmacodynamic
- Assay validation across research, development, and clinical stages
- When is a biomarker meaningful enough to replace or complement clinical outcomes?
- From correlation to mechanistic specificity: Tracking vs. proxy biomarkers
9:30 Decoding Co-Pathologies to Refine Disease Definitions, Enhance Target Engagement, & Optimize Patient Stratification in Neurodegeneration
Synopsis
- Navigating overlapping pathologies in AD, PD, ALS, and MS: tau, amyloid, αsynuclein, TDP-43, and others
- Quantifying and differentiating overlapping pathologies and their impact on diagnosis and treatment
- What deciphering co-pathologies means for patient stratification and therapeutic targeting
10:00 Morning Break & Networking
10:30 Smarter Stratification With the Right Patients for the Right Trials & the Right Trials for the Right Patients
Synopsis
- Biomarkers for stratifying patient subgroups in heterogeneous diseases (e.g., MS, AD, PD, ALS)
- Distinguishing predictive, diagnostic, and pharmacodynamic biomarkers
- Leveraging biomarkers for adaptive trial designs
- Incorporating polygenic scores into stratification models (with genomic context) • Personalized medicine in neuroscience: myth or future?
11:00 How Imaging Can Be Used in a Clinical Setting to Improve the Credibility of Assessments – A Case Study
Synopsis
- Combining biomarker modalities to support development of therapies in neurodegenerative diseases
- Application of neurophysiological biomarkers in Parkinson’s and Alzheimer’s diseases
- Lost in translation: Selecting efficacious drug exposures based on experimental findings
11:30 Lunch Break & Networking
Regulatory & Clinical Validation Pathways: Clinical-Grade Biomarkers, Regulatory Guidelines, Surrogate Endpoints, & Biomarker Qualification
1:00 Outlining the Regulatory Roadmap for Efficient Official Validation of Biomarkers
Synopsis
- Presenting the necessary steps for different pathways of biomarker approval, minimal requirements, metrics and best practice
- Appropriately validating assays from an analytical, clinical and regulatory standpoint for with the correct context of use
- Outlining a strategic approach to IND filings by aligning timing, data readiness, and regulatory requirements
- Knowing when and how to push for approval based on the strength of supporting biomarker data
1:30 Harmonizing Different Official Consent Frameworks for CSF & Plasma Collection to Accelerate Ethical & Compliant Clinical Trials
Synopsis
- IRB and EC quantitative, qualitative and ethical requirements to ensure patient safety and well-being
- A walk-through best practice for getting informed patients consent for clinical trials considering populations with cognitive impairment
- Overview of FDA, EMA, and MHRA roles in shaping consent standards for CSF and plasma collection
2:00 Panel Discussion: Optimizing Imaging Endpoints, Mapping the Current Imaging Landscape & Emerging Tools in Neuroscience
Synopsis
- Review of Current Imaging Modalities: Overview of PET, MRI, fMRI, and other techniques used in neurodegenerative disease trials (e.g., AD, PD, MS, ALS)
- Limitations of Imaging Biomarkers in Clinical Trials: Challenges including accessibility, cost, patient burden, and limitations in sensitivity or specificity
- Innovations on the Horizon: Discussion of emerging imaging techniques and tools, including novel radiotracers, high-resolution modalities, and multimodal approaches
- Integrating Imaging with Other Biomarker Modalities: How imaging can complement fluid biomarkers, digital measures, and omics data for a more holistic disease profile
- What regulatory criteria must be met for a surrogate endpoint to be accepted as a validated substitute for a clinical endpoint?
2:30 Afternoon Break & Networking
From Molecules to Machines: Harmonizing Technologies, Imaging & AI in Data Analysis
3:30 Roundtable Discussion: Piecing It All Together: How Multi-Omics Reveals Hidden Patterns to Unlock a Deeper Understanding of Disease
Synopsis
Exploring how integrative multi-omics approaches are transforming our ability to decode disease complexity, particularly in heterogeneous neurological disorders like Alzheimer’s, Parkinson’s, MS, and ALS. Gain insight into how combining omics layers can improve biomarker discovery, support precision medicine, and drive better patient stratification.
- The limitations of relying on single-omic data in complex, multi-pathway diseases
- Overview of multi-omics: Combining layers to contextualise molecular interactions
- How integrated data enhances the resolution and specificity of biomarker discovery
- The Omics Spectrum: Genomics, transcriptomics, proteomics, epigenomics, lipidomics & metabolomics
- Discussion of emerging omics layers: Glycomics, single-cell omics, spatial omics