Explore the Agenda
7:30 am Check In, Coffee & Light Breakfast
8:25 am Chair’s Opening Remarks
Patient Perspectives in Alzheimer’s & Parkinson’s: Improving Trial Design & Treatment Development
8:30 am Panel Discussion: Patient Perspectives in Neurodegeneration: Shaping the Future of Alzheimer’s & Parkinson’s Drug Development
- Hear directly from individuals living with Alzheimer’s and Parkinson’s alongside leading foundations, offering a unified view of lived experience and scientific context
- Explore real-world treatment challenges, clinical trial barriers, and the outcomes that matter most to patients across both diseases
- Gain actionable, patient-driven insights to inform more inclusive, meaningful, and effective drug development and trial design
9:30 am NULISA Neurology Biomarker Platform – Brain-derived pTau217 & High-plex Neurodegenerative Disease & Neuroinflammatory Biomarker Analysis
- NULISA™: Ultra-sensitive femtogram level detection with barcode-based high-plex biomarker analysis
- First brain-derived (BD) pTau217 assay for highest accuracy blood-based Alzheimer's detection
- CNS Disease Panel: Unique in its coverage of hundreds of proteins involved in neurodegeneration, inflammation, vascular and synaptic dysfunction New BD-Tau181,217, 231, total assays
- Inflammation Panel: Comprehensive coverage of cytokines and key immune markers underlying neuroinflammatory disease
- NULISA platform: Unmatched ability to translate targeted neuroimmune proteomic discoveries into low-plex quantitative assays on the same ARGO HT System
- ARGO HT System: Fully automated instrument for running single-, low-, and high-plex assays
- NULISA IVD and ARGO DX in development with funding from Gates Ventures and DxA
10:00 am Precision Medicine in Alzheimer’s Disease: Leveraging Deep Disease Insights & Biomarker-Guided Therapeutics
- Characterizing Alzheimer’s Disease into biologically meaningful subtypes based on underlying pathophysiology (e.g., amyloid, tau, neuroinflammation, synaptic dysfunction)
- Integrating multi-modal biomarkers (fluid, imaging, genetics) to define patient subgroups and align therapeutic mechanisms with disease biology
- Using validated biomarkers for patient selection, enrichment, and response monitoring to accelerate development of disease-modifying therapies
- Matching therapeutic interventions to molecular and clinical subtypes to maximize efficacy and minimize unnecessary exposure
- Combining precision diagnostics with adaptive trial designs to transform Alzheimer’s treatment into a personalized care paradigm
10:30 am Morning Break & Refreshments
Track One: Discovery & Preclinical
Expanding Therapeutic Frontiers in AD & PD: Blocking Protein Spread & Unlocking Inflammasome Modulation Beyond Amyloid & α-Synuclein
Chair:
11:00 am Drugs in Preclinical Development: Targeting Pathological Spreading of Alpha-Synuclein, Beta-Amyloid & Tau
- Molecular mechanisms responsible for prion-like propagation and spreading of misfolded protein aggregates / amyloid proteopathic seeds
- Olfactory bulb injections of alpha-synuclein Preformed Fibrils (PFFs) reproduce slow brain wide spread of alphasynucleinopathy in rodents, mirroring PD
- Preclinical development of a novel Alpha-Synuclein/Beta- Amyloid/Tau/ /glycosaminoglycan inhibitor for PD and AD
11:30 am A Human Neuronal Based Alpha-Synuclein Assay Useful for Drug Discovery
- Human iPSC derived neurons
- Imaging and TR-FRET assay
- Small molecule and antibody testing
11:40 am CRO Services for CNS Oriented NHP Studies
- Modeling of PD and AD pathology in macaques for therapeutic efficacy testing
- MRI and PET imaging capabilities related to pathology and efficacy endpoints
- Biodistribution study designs with CNS dosing, biomarker quantification, safety/pathology and pharmacodynamic readouts
- Behavior and cognitive assessment capabilities
11:50 am Advancing Inflammasome-Targeted Therapeutics: A Novel Pathway Beyond Amyloid & Alpha-Synuclein
- Leveraging decades of safety data from nucleoside reverse transcriptase inhibitor analogues to accelerate development and reduce risk in Alzheimer’s, Parkinson’s, and MS
- Demonstrating compelling real-world and preclinical evidence showing up to 75% reduced incidence of neurodegenerative disease and robust efficacy in gold-standard models
- Positioning inflammasome modulation as a broad, diseaseagnostic strategy with potential to transform treatment timelines and bypass limitations of single-pathway targets
Track Two: Clinical & Regulatory
Advancing Precision Endpoints in Neurodegeneration: Co-Developing Imaging Companion Diagnostics & Harnessing Digital Wearables for More Inclusive AD/PD Trials
Chair:
11:00 am Companion Diagnostic Co-Development for New Imaging Brain Scans or as Outcome Measures
- How can sponsors and tracer makers team up (for example, the widely used but still investigational Lantheus tau tracer) to collect validation data side-by-side with regulatory approval in mind for patient pre-selection
- How co-developing can enable the label to specify a clear uptake cut-off instead of treating “everyone”
- Use of Hippocampal volume as eligibility criterion and as an outcome measure in APOE-4 carriers with AD
- Partnering early avoids the hold-up when the diagnostic lags behind, allowing doctors to use the scan on launch day to pick the right patients
11:30 am Multi-Protein Panels for Co-Pathology Detection & Biological Patient Stratification; Towards Personalised Medicine in Neuro Trials. Learnings from HER-096 development for Parkinson’s Disease
- Herantis approach to developing personalised medicine: How can oncology-style biological characterisation help define the right target patient population for disease modifying therapies
- Deep biological characterisation of patients and HER-096 responses across multiple analysis platforms together with digital biomarker readouts
- Including baseline multi‑protein panels to capture copathologies, such as AD pathology
- Potential for novel biomarkers, e.g. skin‑biopsy + seeding assay for less‑invasive stratification tests to boost recruitment
- Learnings for Phase 2 clinical development
12:00 pm Extended Q&A
12:20 pm Lunch & Networking Break
Track One: Discovery & Preclinical
Harnessing AI & Cellular Pathways: Advancing Novel Target Discovery & Restoring Autophagy-Lysosomal Function in Neurodegeneration
Chair:
1:00 pm Lysosomal Ion Channels as a New Therapeutic Class in Neurodegeneration: Advancing TRPML1 and TMEM175 Small-Molecule Agonists
- Scientific and genetic rationale for targeting lysosomal ion channels in age-related neurodegenerative diseases (AD and PD)
- Development considerations, challenges, and translational promises in advancing a highly brain-penetrant, Phase 1–ready TRPML1 small-molecule agonist
- Emerging preclinical data of a development candidate TMEM175 agonist for Parkinson’s disease
1:30 pm AI / Advanced Computing for New Target Discovery for AD/PD or Protein Aggregation Disorders in CNS
- Exploring the development of novel CRISPR-based screening approaches to identify new targets
- Highlighting the need to improve the disease modelling systems to better reflect the progression of the disease, and improve upon the current models which are limited in their ability to capture the full complexity of the pathological processes
Track Two: Clinical & Regulatory
Advancing Precision Endpoints in Neurodegeneration: Co-Developing Imaging Companion Diagnostics & Harnessing Digital Wearables for More Inclusive AD/PD Trials
Chair:
1:00 pm Translatable Synaptic & Lipid Biomarkers for Early Parkinson’s: Insights from Longitudinal Multi-Modal Signatures
- Identifying novel plasma lipid and synaptic protein biomarkers that change longitudinally in early Parkinson’s, enabling earlier detection and patient stratification
- Correlating these biomarkers with clinical progression and functional decline to enhance predictive power in trial endpoints
- Enabling tighter selection and more efficient monitoring in clinical trials, reducing sample sizes and improving sensitivity to detect disease-modifying effects
1:30 pm Roundtable Discussion: Digital Wearable Sensors to Provide Continuous, Objective Motor Data & Unlock Faster, Leaner Parkinson’s Trials as Primary Endpoints
- Capturing wrist worn and smartphone sensor streams that continuously log tremor, bradykinesia, and gait, supplying objective high frequency data that can shrink sample sizes and sharpen signal detection
- Highlighting the first regulated Parkinson’s study to use a digital movement biomarker as its primary outcome, setting a clear regulatory precedent that boosts sponsor confidence to adopt wearables in future trials
2:00 pm Afternoon Break & Refreshments
Beyond the Science: Translating Novel Mechanisms Into Real-World Value & Investment Confidence
2:30 pm Beyond Amyloid: Small Molecule Inflammasome Modulators
- Showing that inflammasome modulating small molecule reverses memory loss even with amyloid still present, opening a non amyloid treatment path and diversifying therapeutic risk after 30 years of single target focus
- Leveraging blood biomarkers like p Tau217 to provide objective read outs of anti inflammatory efficacy, enabling faster, smaller proof of concept studies
- Delivering orally bioavailable small molecules that bypass blood-brain barrier hurdles faced by antibodies, simplifying dosing and improving long term patient adherence
3:00 pm Market Access Reality Check for Disease Modifying AD Drugs
- CMS coverage gap for pre clinical anti amyloids and spill over to private payers
- Cost drivers: MRI monitoring, infusion logistics and diagnostic bundling
- Generating evidence packages that HTA bodies prioritise (QoL, caregiver, budget impact) with responsible data storytelling to maintain policymaker trust
3:30 pm Panel Discussion: Bridging Innovation & Investment: De-Risking Disease-Modifying Neurodegenerative Therapies to Secure Investor Backing
- What does “de-risked” biology actually mean in AD/PD right now, and how does that differ for first-in-class versus best-in-class programs?
- In today’s market, what milestones genuinely unlock the next cheque: target validation, Phase 1 biomarkers, early clinical signal, or differentiated strategy vs the competitive landscape?
- How are investors underwriting differentiation in crowded spaces (e.g., brain-shuttle Aβ), and what must a Phase 1 package prove to stand out?
- Beyond science, what determines investability: team quality, trial execution, commercial fit, and the ability to pivot, and why do these factors now carry more weight than ever?