9:00 am Morning Refreshments
9.25 am Chair’s Opening Remarks
IMPROVING BIOMARKER SENSITIVITY FOR MORE QUANTITATIVE MONITORING OF DISEASE PROGRESSION & DRUG EFFECT
9:30 am Generating & Validating an Alzheimer’s Disease Blood-Based Biosignature for Predicting Clinical Progression
Synopsis
- Developing a prognostic biosignature of clinical progression in preclinical and mild cognitive impairment (MCI) due to AD individuals
- Harmonizing fluid biomarker measures across assay platforms and cohorts
- Investigating combinations of blood-blood markers (BBMs) and health status indicators that best forecast a change in cognitive/ functional scores
10:00 am Mapping Tau PET Tracers to Correlate with Cognitive Decline in Different Patient Cohorts for a More Biologically Driven Staging of Alzheimer’s Disease & Other Neurological Diseases
Synopsis
- Exploring plasma p217+tau to show promise for separating either intermediate/advanced stage AD from lower stage AD to provide prognostic information and inform better selection for trials and disease modifying therapies
- Employing biological PET stages recommended by NIA-AA revised criteria to show disease progression with plasma p217+tau as an incremental stepwise trend capable of determining population-specific patient improvement on drug
- Navigating utility and reproducibility challenges in implementing plasma p217+tau for group level discrimination between disease stages across the globe
- Looking ahead to developing risk prediction models to combine several factors to accurately predict individual-level disease stages
10.30am Morning Break & Networking
11:00 am Advancements & Applications of Imaging Flow Technologies in the CNS
Synopsis
- Exploring novel flow cytometry assays and combining them with imaging applications in neuroscience
- Validating proof of concept and mechanism of action studies
11:30 am Evaluating the Diagnostic Activity of Alpha Synuclein PET Tracer in PD, DLB & MSA for Earlier Diagnosis & Treatment
Synopsis
- Delving into preclinical characterization with ACI-12589:
- in vivo correlations between a-syn PET and dopaminergic loss
- Mapping of ACI-12589 to a-syn fibrils
- Clinical data with ACI-12589: robust quantification from dynamic scanning with arterial blood sampling and kinetic modelling to simplified methods
- Combination of PET imaging with SAA improves diagnostic accuracy of MSA and support clinical trial design
- Next generation tracer, ACI-15916 with significantly improved target occupancy has the potential to detect synucleinopathies including Parkinson’s disease
12:00 pm Lunch & Networking
HARNESSING BIOMARKERS AS SURROGATE ENDPOINTS IN NEUROSCIENCE TRIALS FOR STREAMLINED REGULATORY APPROVALS
1:00 pm Roundtable Discussion: Lessons Learnt from Navigating Assay Regulatory Acceptance Criteria in the CNS
Synopsis
Splitting into diverse, cross-disciplinary groups, to crowdsource and troubleshoot queries on the regulatory requirements
for harnessing biomarkers in neuroscience
Evaluating the Acceptance Criteria of Fluid &
Imaging Biomarkers as Surrogate Endpoints in
Neuroscience
Evaluating Regulatory Considerations in IUO vs
RUO Assay in Neuroscience Research
- Outlining the ongoing efforts for obtaining more validated
biomarkers for neurological disorders - Exploring when and under which circumstances biomarker
data can be used as a surrogate endpoint in
neurological trials - Exploring lessons learnt from NFL in SOD-1 ALS and potential
expansion to broad ALS - Considering the requirements for drug approval using
amyloid, NFL or other novel biomarkers as surrogate
endpoints
- Differentiating between investigational use only (IUO) and
research use only (RUO) assays, when to use them within
clinical development and outlining their role in early-phase
investigational studies and regulatory scrutiny compared
with RUO assays - Transitioning to in vitro diagnostic use to highlight
implications for assay validation, regulatory approval
processes and importance of early planning to ensure
compliance with evolving regulatory standards in the CNS - Navigating different approaches globally to understand
regulatory requirements needed for global approval
2:00 pm Can We Slow Neurodegeneration in Multiple Indications without Reducing Neurofilament Light Chain?
Synopsis
- Reviewing data on NFL in different neurodegenerative diseases
- Exploring examples of different therapeutic trials where NFL was reduced with treatment response
- Determining if there are neurodegenerative patient populations for which NFL is not a relevant marker of disease
- Evaluating feasibility for controlling baseline heterogeneity with NFL for more confident confirmation of efficacy
- If NFL is not a ubiquitous marker of disease progression, what other biomarkers in other pathways should be considered?
2:30 pm Monitoring Changes of RNA & Protein in the Blood as a Surrogate Measure for Changes in Monogenetic Disorders in the CNS
Synopsis
- Case studies
- Biological confirmation in animal models
- Considerations in developing clinical grade assays to demonstrate target engagement
3:00 pm Panel Discussion: Looking Beyond Amyloid, Tau, TDP43 & Alpha Synuclein, How Can we Validate Biomarkers for Drugs with Alternative Mechanisms?
Synopsis
- Evaluating how these markers are for TDP43 & Alpha Synuclein across disease?
- Exploring use cases of biomarker usage for neuroinflammatory pathology, microglia and oxidative stress where there is little relevance to the disease indication
- Navigating future directions to improve ease of biomarker utility in drug development
3.30 pm Chair’s Closing Remarks
3.35 pm End of Conference
