7:00am Check-In, Coffee & Light Breakfast
8:05 am Chair’s Opening Remarks
BUILDING ON A PIVOTAL YEAR OF AD DRUG DEVELOPMENT: LESSONS LEARNED & NOVEL OPPORTUNITIES
8:15 am Panel Discussion: What Have we Learned from the Last 12 Months of Alzheimer’s Drug Development?
Synopsis
- What have been the main obstacles to integrating first class of anti-amyloids into the treatment paradigm?
- What is the impact of ARIA on penetration of these new anti-amyloid therapies into market? What is the plan to better manage the burden of ARIA going forward?
- Shifting focus to new emerging therapies: From anti-inflammatory and anti-tau treatments to combination therapies, what are the most promising novel approaches in the pipeline?
9:00 am Innovating Second Generation Antibodies Targeting Toxic Proteinopathies with Enhanced Blood-Brain Barrier Penetrance
Synopsis
- Reviewing the latest progress of shuttling technologies to enhance therapeutic targeting to the brain and reduce side effects like ARIA
- Investigating mechanisms of transport across the BBB to uncover opportunities to more effectively target neurodegenerative protein aggregates
- Widening the cargo space of antibody brain shuttles; what other modalities can be applied?
9:30 am NULISA CNS Disease Biomarker Platform – The Only High-Plex, High Sensitivity Platform for Investigation of Biomarkers of Neurodegenerative Diseases & Treatment Response
Synopsis
- NULISA™ combines ultra-sensitive femtogram level detection with barcode-based high-plex biomarker analysis.
- The NULISA CNS Disease Panel is unique in enabling investigation of hundreds of proteins involved in neuroinflammation, vascular, and synaptic dysfunction, and proteinopathies.
- NULISA enables the unique ability to translate discoveries based on broad profiling into single and low-plex biomarker assays on the same instrumentation
- NULISA single-, low-, and high-plex assays all run fully automated on the ARGO HT System.
- NULISA CNS Disease Panel 120 and Inflammation Panel 250 support exploratory clinical research into therapeutic effects on neurodegenerative disease processes.
- NULISA IVD and ARGO DX in development with funding from Gates Ventures and DxA.
10:00am Speed Networking
10:30am Morning Break & Refreshments
TRACK 1: TARGET DISCOVERY & IN VITRO VALIDATION
Chair:
ADVANCING MODELLING & SCREENING TECHNOLOGIES TO TRANSFORM DRUG DISCOVERY CAPABILITIES
11:00 am Building a Complex Cell Culture Comprising Neurons, Microglia, Astrocytes & More to Better Recapitulate In Vivo Alzheimer’s Phenotypes
Synopsis
- Increasing complexity of the system to bridge the gap between basic assays and animal models
- Linking complex cultures with patient data
- Ensuring the right mix of cells to represent the in vivo phenotype
- Studying cell-cell crosstalk in complex in vitro cultures to enhance understanding of disease progression
11:30 am From Cells to Insights: Advancing Neuroscience with Tailored In Vitro Platforms
Synopsis
- Showcasing a novel neuroscience platform with state-of-the art technologies for drug screening and MOA studies
- Using complex in vitro models in the context of neurodegenerative conditions
- Exploring the all-in-one approach for intricate phenotypes in AD and PD
11:40 am In Vivo Phenotypic Screens to Identify Novel Therapeutic Targets to Treat Conserved Pathologies in Neurodegenerative Disorders
Synopsis
- Unbiased in vivo phenotypic screens can discover novel therapeutic targets underlying conserved pathological problems in neurodegenerative disorders
- Identifying neuro-protective/-regenerative therapies to preserve spared neuronal populations affected by progressive, chronic neurodegenerative diseases.
- Developing KCC2 neuromodulation therapies for neurodegenerative disorders involving excitation/inhibition imbalance and neuronal circuit disinhibition within the CNS
12:10 pm iPSC-Derived Neuron Panels for Biomarker Discovery & Patient Stratification
Synopsis
- Developing iPSC-derived neuronal and glial models to enhance biomarker discovery and patient stratification in Alzheimer’s disease drug development
- Improving translational relevance by integrating patient-derived iPSC neuron and glial panels
- Validation of morphological and functional capabilities from the iPSC-derived neurons, astrocytes, and microglia across mono-, co-, and triculture models
- Expansion of platform into Parkinson’s Disease using Dopaminergic Neurons
12:40 pm Lunch & Networking Break
INNOVATING BIOLOGICAL & SYMPTOM-BASED APPROACHES TO PARKINSON’S DRUG DEVELOPMENT
1:30 pm Discovering a Novel Mechanism Underlying LRRK2 Kinase Activity in Genetic & Sporadic Parkinson’s Disease Populations
Synopsis
- Leveraging patient-derived cell and animal model systems to investigate how 15-Lipoxygenase regulates LRRK2 kinase hyperactivity in PD
- Highlighting the potential of 15-Lipoxygenase inhibitors as a novel therapeutic approach to safely modify LRRK2 kinase activity and treat PD
2:00 pm ariadne.ai SPATIAL – Unlocking Neuroscience Applications in Spatial Omics
Synopsis
- Browser-based spatial (multi-)omics analysis for any modality.
- Specific neuroscience package for precise morphometric analysis of neurons and glia cells, including pathological features like amyloid-beta plaques
- One software solution for all analysis steps: Elastic registration, precise cell segmentation, intensity mapping, advanced gating and statistical analysis
2:10 pm Roundtable Discussion: Investigating the ‘Non-Motor’ Symptoms of Parkinson’s Disease
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas.
How can we better understand and target the non-motor symptoms of PD?
How could non-motor symptoms be used as biomarkers to detect those at high risk for early PD?
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open wider audience debate
TRACK 2: IN VIVO TRANSLATION & EARLY CLINICAL
Chair:
ADVANCING IN VIVO AD & PD MODELS TO DE-RISK THE TRANSLATIONAL GAP
11:00 am Overcoming the Lack of Functional Homology Conservation in Inflammatory Genes Across Mice, Monkeys & Humans to Enhance Model Recapitulations
Synopsis
- Addressing cross-species differences in inflammatory gene function, microglial response to stimuli, and how vasculature regulates neuroinflammation
- Exploring strategies to overcome these variances to enhance the translational value of in vivo studies of neuroinflammation in AD and PD
- Reviewing streamlined differentiation approaches to generate iPSC-derived microglia from patient-derived cells
11:30 am Developing a Unique In Vivo Model of Tau Propagation to Understand Tau Pathology Progression in the Alzheimer’s Brain
Synopsis
- Showcasing a novel mouse model of tau pathology propagation
- Utilizing the model to understand the ‘spreading’ of tau pathology between cells
- Testing antibody-mediated prevention of tau pathology in the model
12:00 pm Translation Based Multidimensional Platform Approach to Pre-clinical Models: Behaviour, Biomarkers, Histology & EEG
Synopsis
- Integration Across Modalities: Highlight the benefits of a multidimensional platform combining behavioral studies, biomarker profiling, histological analysis, and EEG to generate a comprehensive understanding of preclinical models.
- Symptom Spectrum Analysis: Demonstrate the platform’s ability to assess both core disease symptoms and comorbid features, such as anxiety, sleep disturbances, motor impairments and cognitive deficits, to provide a holistic understanding of disease phenotypes.
- Cross-Species Applicability: Explore the platform’s capability to characterize preclinical models across species, including rodents and canines, ensuring broader translational insights and validation.
12:30 pm Lunch & Networking Break
EVALUATING ANTI-TAU STRATEGIES AS A NOVEL APPROACH FOR COMBATTING COGNITIVE DECLINE
1:30 pm Discovering an Anti-Tau Antibody to Target Toxic Extracellular Tau Species in the Clinic
Synopsis
- Demonstrating ability to prevent tau propagation in vivo
- Rationale for targeting extracellular vs intracellular species of tau
- Future directions for moving this compound into the clinic
2:00 pm Panel Discussion: Evaluating Anti-Tau Programs Emerging in the Clinic & Debating the Most Promising Approaches
Synopsis
- Do longitudinal changes in tau correlate to longitudinal changes in cognition and function?
- How well does CSF tau correlate with tau PET?
- What are the advantages of targeting intracellular vs extracellular tau species? What are the side effects associated with this?
- Does targeting a different part of the tau protein make a difference?
TRACK 3: LATE CLINICAL, REGULATORY & COMMERCIAL
Chair:
EXPLORING CLINICAL INNOVATIONS THAT COULD UNLOCK NEW NEURODEGENERATIVE THERAPIES
11:00 am Advancing Clinical Trial Design to Accommodate Biological & Clinical Heterogeneity Underlying Alzheimer’s Pathology
Synopsis
- Overcoming challenges of Alzheimer’s heterogeneity in clinical trials
- Adapting trial designs to enable a more personalized approach
- Showcasing examples of advances in repeatable biomarkers and digital phenotyping
11:30 am Developing Novel Digital Measures for Neurodegenerative Diseases
Synopsis
- Incorporating functional and mobility performance assessments in Alzheimer’s and Parkinson’s populations
- Highlighting the potential of digital measures to capture patient experiences in real-world settings
- Outlining feedback from regulatory agencies about how these endpoints can be further developed
12:00 pm Optimizing Clinical Trials with Cutaneous Biomarkers in Neurodegenerative Diseases
Synopsis
- 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
12:30 pm Lunch & Networking Break
ENHANCING THE LANDSCAPE OF ANTI-AMYLOIDS & EVALUATING COMBINATION APPROACHES
1:30 pm Targeting Novel Forms of Amyloid to Improve Upon the Current Standard of Care & Reduce Implications of ARIA
Synopsis
- Outlining the promise of sabirnetug as a next generation treatment for AD
- Demonstrating significant reductions in ARIA-E and biomarker-driven signals of efficacy
- Implications for the future development of sabirnetug
2:00 pm Use of Digital Twins in a Phase 2a Trial to Predict Disease-Progression of Participants with Mild-to-Moderate AD Supports Early Signals of Efficacy
Synopsis
- Machine learning-generated digital twins of disease progression which can be seamlessly integrated across different stages of clinical development
- In this study, we demonstrate the utility of digital twins in a Phase 2a trial of REM0046127, a small molecule designed to restore the integrity of septin filaments in neurons affected by Alzheimer’s disease
- Digital twins validated the preliminary results on tau pathology biomarkers, providing valuable additional evidence to support advancement of the clinical program
2:10 pm Roundtable Discussion: Discussing the Promises & Challenges of Elevating Neurodegenerative Therapeutics with a Combination Approach
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas.
Are there opportunities to enhance therapies in phase I/II by combining with another treatment to improve them?
What regulatory hurdles might arise when demonstrating the additional risks or benefits of a combination therapy?
How can we avoid risks of drug-drug interactions and decipher the optimal timing of the two therapies?
Moderator Feedback & Audience Debate
Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open wider audience debate
2:45pm Afternoon Break & Refreshments
INNOVATING A NEW ERA OF DISEASE-MODIFYING THERAPIES TARGETING ALPHA-SYNUCLEIN & BEYOND TO TRANSFORM THE PD TREATMENT LANDSCAPE
3:30 pm Highlighting Phase III Buntanetap Data & Embracing a More Holistic Approach to Neurodegenerative Disease Targeting
Synopsis
- Showcasing phase III success in improving cognition in Alzheimer’s disease (as measured by ADAScog) as well as movement and function in Parkinson’s (as measured by MDS-UPDRS)
- Outlining the cascade of events that follow Buntanetap-induced mRNA inhibition
- Leveraging its impact on several brain targets to emphasize the need for novel, more holistic drug approaches
4:00 pm Trehalose: From Research Compound to Clinical Candidate for Parkinson’s Disease
Synopsis
- Overview of trehalose and its effects as a modulator of autophagy
- Pharmacokinetics and brain penetration of trehalose in rodents and non-human primates
- Lowering the therapeutic dose of trehalose and moving into clinical development
4:30 pm Panel Discussion: Exploring Opportunities in Alpha-Synuclein & Beyond for More Transformative & Efficacious Therapies: What’s Coming Next in Parkinson’s Drug Development?
Synopsis
- Overview of trehalose and its effects as a modulator of autophagy
- Pharmacokinetics and brain penetration of trehalose in rodents and non-human primates
- Lowering the therapeutic dose of trehalose and moving into clinical development
5:15 pm Chair’s Closing Remarks
5:20pm Scientific Poster Session & Drinks Reception
Synopsis
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.