Explore the Agenda
7:30 am Check In, Coffee & Light Breakfast
8:25 am Chair’s Opening Remarks
Redefining Disease Modification: Multi Mechanism, Biomarker Driven & Ethically Accessible Therapies for Alzheimer’s & Parkinson’s
8:30 am Panel Discussion: Ushering in a New Era of Disease-Modifying Therapies in AD & PD – Beyond Amyloid & α-Synuclein
- Exploring emerging mechanisms such as mitochondrial stress, ferroptosis, and microglial TREM2 to diversify therapeutic strategies and expand opportunities
- Evaluating how mixed-modality combinations may outperform single antibodies, offering more robust and durable disease-modifying potential
- Implementing biomarker panels to de-risk first-in-human trials, enabling smarter patient selection and stronger regulatory confidence
9:15 am The Implications of a Biological Definition of Parkinson’s Disease on Preclinical Research
- Examine the impact of biological definitions of Parkinson’s disease on clinical trial design
- Investigate the role of emerging biomarkers in the early detection, monitoring of disease progression, and assessment of therapeutic efficacy in Parkinson’s disease
- Discuss how evolving clinical trial designs and emerging biomarkers will redefine evaluation of efficacy and target engagement in preclinical models
9:45 am Prasinezumab: Advancing Towards the First Disease Modifying Treatment for Parkinson’s Disease
- Demonstrating clinical potential of Prasinezumab to delay motor progression, highlighting meaningful functional benefit for patients
- Outlining α-synuclein targeting mechanism with supporting biomarker evidence to validate biological activity and translational relevance
- Exploring future Phase III directions and beyond to position Prasinezumab as a first-in-class disease-modifying therapy
VIRTUAL
10:15 am Speed Networking & Morning Break
A prime chance to make the most of in-person networking and forge new connections with an expanding landscape of companies entering, or expanding their presence within, the neurodegenerative drug development space. Designed to maximize your introduction to numerous new individuals and serve as a catalyst for ongoing discussions during the summit, connect with peers across discovery, preclinical, translational, clinical, regulatory and commercial Alzheimer’s and Parkinson’s research.
Track One: Discovery & Preclinical
Chair: Alessandra Welker, Principal Research Scientist I, AbbVie
Unlocking Alzheimer’s & Parkinson’s Targets: In Vitro Tau Models, Functional Genomics & iPSC Systems for Translational Success
11:00 am Innovating Tau-Targeted Preclinical Models to Strengthen Translation in Alzheimer’s Drug Discovery
- Developing advanced cell-based tau models to more accurately capture disease-relevant biology, enhancing translational reliability before clinical entry
- Screening novel therapeutic approaches in tau-focused systems to identify mechanisms that directly impact aggregation and pathology, reducing late-stage trial failure
- Aligning preclinical tau biomarkers with clinical endpoints to build stronger continuity across the R&D pipeline, de-risking candidate progression
11:30 am Human iPSC-derived Neurons & Glia to Model Alzheimer’s & Parkinson’s Disease
BrainXell is a leading provider of hiPSC-derived neurons and glia, working with over 90% of the world’s largest pharmaceutical companies.
- Comprehensive off-the-shelf portfolio featuring key Alzheimer’s-related mutations (APOE, TREM2, APP, PSEN1, etc.) with matched isogenic controls.
- New dopaminergic neuron differentiation protocol generates high-purity cells capable of dopamine secretion by DIV 14
- Robust, scalable manufacturing processes generate billions of cells, and flexible custom workflows deliver the same high-quality cell types from any customer-supplied iPSC line
11:40 am Leveraging Functional Genomics for Discovery of Novel Parkinson’s Disease Targets: From Genetics to Mechanism
- Integrating human genetics with iPSC-based disease models to recapitulate causal biology and developing isogenic systems that faithfully model PD-relevant risk variants, to accelerate discovery of differentiated, genetically validated targets with higher translational value
- Using functional genomics and human iPSC-models to uncover new target opportunities for Parkinson’s disease
- Developing predictive models in iPSC-derived neurons, to provide tractable, disease-relevant efficacy readouts
12:10 pm Parkinson’s Disease Model Using Human iPSC-derived Neuron-astrocyte-microglia tri-cultures
- Fujifilm has previously developed human iPSC-derived aged astrocytes exhibiting senescent features, which can be induced into neurotoxic astrocytes by cytokine stimulation
- By combining these aged astrocytes with dopaminergic neurons and microglia, we established a tri-culture system that demonstrates α-synuclein-induced cytokine release and neuronal damage, highlighting its potential utility for Parkinson’s disease drug development
- Fujifilm offers advanced compound evaluation using this novel model, along with opportunities for collaboration in disease modeling and CNS drug development
12:20 pm The Need for High-Purity A9 Dopaminergic Neurons Within Parkinson’s Disease Models
- Understand the biological significance of A9 vs. A10 dopaminergic neuron subtypes in Parkinson’s disease
- Learn how HD-DoE technology enables targeted development of A9 neurons with desired phenotypic profiles
- Review of key phenotypic and functional QC benchmarks
- Discussion of recommended use cases and applications in discovery and disease modeling
Track Two: Clinical & Regulatory
Chair: Bruce Morimoto, Head, Drug Development, Fuku Biotech
Advancing Disease-Modifying Alzheimer’s & Parkinson’s Therapies: Biomarker-Driven Antibody Innovation and Overcoming Clinical Trial Endpoint Barriers
11:00 am Sabirnetug (ACU193) Case Study: A Next-Generation Anti-Aβ Oligomer Antibody Advancing Disease-Modifying Claims with Translational Biomarker Evidence in Alzheimer’s Therapeutics
- ACU193 selectively targets soluble amyloid-β oligomers (AβOs), sparing monomers and plaques, minimizing the risk of ARIA while preserving synaptic function
- Enhance safety profiles and therapeutic potential for early Alzheimer’s intervention
- Demonstrated CSF biomarker effects in INTERCEPTAD Phase 1 trial (β p tau181, β Aβ42/40 ratio), validating downstream impact on tau pathology and synaptic markers
- Exploring the ongoing ALTITUDE-AD Phase 2 trial with robust biomarker and cognitive endpoints, designed to confirm mechanistic action and clinical efficacy in early AD, and the implications of this for regulatory success and meaningful patient outcomes
11:30 am Overcoming Barriers in Parkinson’s Disease Trial & Endpoint Development Through Statistics
- Designing smarter clinical trials for slowly progressing Parkinson’s populations, reducing operational hurdles while improving feasibility and signal detection
- Optimizing endpoint selection with robust statistical frameworks that reflect disease biology and patient reality, supporting regulatory and clinical confidence
- Enabling sponsors to run faster, leaner, and more conclusive studies that unlock disease-modifying therapies for patients sooner
VIRTUAL
12:00 pm Extended Q&A
12:30 pm Lunch & Networking Break
Track One: Discovery & Preclinical
Chair: Alessandra Welker, Principal Research Scientist I, AbbVie
Molecular Staging & Genetic Modifiers: Refining Alzheimer’s Risk, Progression & Trial Design
1:30 pm Genetic Analysis of Alzheimer’s Disease Progression in Clinical Trials
- Applying AI powered genome scans to uncover genetic modifiers of disease tempo and enable tighter trial designs and quicker read outs
- Mining longevity and centenarian genomes for protective variants to reveal resilience pathways, offering novel, lower risk targets to slow progression
- Incorporating C9orf72 and other cross disease variants into baseline panels to delineate molecular subtypes, reducing heterogeneity and boosting drug effect signals
2:00 pm Session Reserved for Ricoh Biosciences
2:30 pm Roundtable Discussion: Is Biomarker‑Only “Preclinical Alzheimer’s” a Disease Stage or a Risk Flag
- Comparing FDA/Alzheimer’s Association staging versus the International Working Group’s “risk‑only” view, unifying language for target‑discovery teams so translational models match the biology regulators will ultimately judge
- Exploring how the ongoing pre‑symptomatic Donanemab and Lecanemab studies will read out time‑to‑symptoms data, supplying hard evidence on early intervention that can recalibrate animal‑model endpoints and biomarker cut‑offs before the next IND
- Weighing the risk‑benefit of treating asymptomatic, amyloid‑positive individuals given ARIA and payer concerns, clarifying safety and economics thresholds that determine which pre‑clinical candidates advance and at what dose
Track Two: Clinical & Regulatory
Chair: Bruce Morimoto, Head, Drug Development, Fuku Biotech
Personalising Neurodegenerative Trials & Therapies: Biomarker-Guided Patient Stratification & Multi-Target Strategies to Achieve True Disease Modification
1:30 pm Combining Therapeutic Strategies in Parkinson’s Disease: Enhancing Efficacy through Multi-Target Approaches
- Targeting multiple pathogenic mechanisms simultaneously, including mitochondrial dysfunction, oxidative stress, and protein aggregation to slow neurodegeneration more effectively than single-agent approaches
- Combining neuroprotective agents in rational, mechanistically complementary regimens, creating synergistic effects against converging pathways of PD pathology to increase the likelihood of achieving true disease modification
- Leveraging preclinical and translational models to evaluate multi-target strategies, optimizing combinations before clinical trials to accelerate the development of effective therapies
2:00 pm Roundtable Discussion: Leveraging Real-World Add-On Therapy Templates to Advance Combination Strategies in Neurodegenerative Diseases
- Criteria for selecting patient subgroups most likely to benefit from multi-mechanism approaches (e.g., stage of disease, biomarker profiles)
- Prioritizing clinically meaningful primary endpoints and complementary secondary measures to capture additive or synergistic effects
- Using established PD add-on protocols and emerging AD combination trials as models for endpoint selection and regulatory alignment
3:00 pm Afternoon Break & Refreshments
Expanding Precision & Immune-Modulating Pathways for Disease Modification in Alzheimer’s & Parkinson’s
3:30 pm Optimizing Clinical Trials with Cutaneous Biomarkers in Neurodegenerative Diseases
- Use of Syn-One Biomarker Technology to support study subject homogeneity, identify target engagement, and measure P-SYN deposition changes over time
- Detection, visualization, and quantification of cutaneous misfolded alpha-synuclein
- Highlight preliminary data and topline results in current trials
4:00 pm Oral Amyloid Oligomer Inhibition with ALZ-801: A Precision Medicine Approach for Early Alzheimer’s Disease
- Targeting amyloid oligomer formation through an oral small molecule mechanism, avoiding ARIA-related safety concerns observed with antibody approaches, delivering a safer, scalable treatment option for early AD patients, including highrisk APOE4 carriers
- Presenting clinical findings from Phase 2 and 3 studies, demonstrating beneficial cognitive and functional outcomes in early disease, supporting ALZ-801 as a potential first-in-class oral disease-modifying therapy
- Positioning ALZ-801 in the evolving AD treatment landscape, offering a differentiated therapeutic pathway for APOE4 carriers, expanding precision medicine opportunities while complementing or replacing antibody-based strategies
4:30 pm Harnessing Neuroimmune Interactions for Disease Modification in Alzheimer’s & Parkinson’s
- Modulating TNF mediated neuroinflammation with dominant negative inhibitors, targeting chronic microglial activation across Alzheimer’s and Parkinson’s models, slowing neurodegeneration by restoring immune homeostasis
- Investigating gut–brain axis dynamics and microbiome-derived metabolites such as butyrate, unveiling epigenetic and immune pathways that differ in PD and Alzheimer’s, enabling novel therapeutic strategies that address both central and peripheral drivers of pathogenesis
- Exploring the roles of microglia, RGS10 signalling, and gene–environment interactions, revealing how immune regulation, stress responses, and genetic context influence neuronal resilience, informing personalized interventions to prevent or slow progression in neurodegenerative diseases
5:00 pm Chair’s Closing Remarks
5:05 pm Scientific Poster Session
This is an informal session to help you connect with your peers in a relaxed atmosphere to continue forging new and beneficial relationships. With an audience of CNS experts eager to hear the latest discoveries in Alzheimer’s and Parkinson’s therapeutic research, you will have the opportunity to display a poster presenting your own work and innovations. Don’t miss out on the chance to connect, learn, and present.