Wednesday, February 26, 2025
7:00 am CHECK-IN & COFFEE
7:55 am Chair’s Opening Remarks
INNOVATIVE APPROACHES IN MULTIPLE SCLEROSIS: ADVANCES IN BTK INHIBITION, COMPARATIVE EFFICACY OF DISEASE MODIFYING THERAPIES, AND BIOMARKER INSIGHTS
8:00 am Highlighting Tolebrutinib Shows Groundbreaking First-in-Class Reduction in Disability Accumulation for nrSPMS
Synopsis
- History and biology of neuroinflammation in multiple sclerosis
- Exploring the hypothesis of neuroinflammation as the driver of disease progression
- Peripherally restricted molecule not effective in progression, BTK was successful in slowing disease progression in the clinic
- PIRA and Tolebrutinib results in relapsing and progressive disease
- Applicability to neuroinflammation beyond MS
8:30 am Advancing Multiple Sclerosis Treatment with BTK Inhibitor Remibrutinib
Synopsis
- Preclinical neuroinflammation models, single cell RNAseq data, soluble biomarker histology to explore mechanism of action in the brain and navigate brain disruption histology in white and grey matter
- Characterization in preclinical potency and selectivity of BTKi’s specifically with regard to Remibrutinib selectivity
- Giving an overview on the current clinical safety profile of Remibrutinib across all indications
9:00 am Evaluating the Efficacy and Safety of BIIB091: Interim Results from the Phase 2 FUSION Trial of a Peripheral BTK Inhibitor for Targeting B Cell and Myeloid Pathways in Multiple Sclerosis
Synopsis
- Phase 2 BIIB091 selectively inhibits BTK, regulating B cell signalling and myeloid cells hypothesized to contribute to MS pathogenesis
- Evaluating interim safety and efficacy data for the FUSION trial focusing on BIIB091 mitigation of disease progression in MS patients, highlighting trial key endpoints such as reduction in relapse rates, MRI lesions and inflammatory biomarkers as well as any safety concerns or adverse events observed during the trial
- Reviewing the potential role of peripheral BTK inhibitors like BIIB091 in the broader landscape of MS treatment to complement or provide an alternative to current therapies, particularly in targeting both B cell driven and innate immune processes without direct CNS penetration
9:30 am SPEED NETWORKING:
Synopsis
This session is a great opportunity to introduce yourself to neuroimmunology and neuroinflammation from leading drug developers. This session is the ideal opportunity to get face-to-face time with the brightest minds in the neuroimmunology field to establish meaningful relationships.
10:00 am MORNING BREAK & NETWORKING
INTEGRATING MULTIOMICS & GWAS TO IDENTIFY & VALIDATE NOVEL BIOMARKERS & DRUGGABLE PATHWAYS IN NEUROINFLAMMATION
10:30 am Unbiased Multiomic Analysis to Identify Blood Based Biomarkers & Novel Targets in Neuroinflammation
Synopsis
- Leveraging biobanks and population-scale genetics to identify novel genes driving associations to act as a druggable target ID
- Combining genetics, proteomics and cell type information into a molecular map
- Integrating findings into refined prediction models in AD, PD and ALS
11:00 am Spatial Transcriptomics for Deep Biology Mechanisms of Action & Biomarker Discovery to Predict Disease Progression in Neurodegeneration
Synopsis
- Example use case for utilizing spatial transcriptomics as a use case of unbiased bulk RNAseq to understand mechanism of action-related questions
- Use case for microglial biology in Alzheimer’s Disease
- Finding when to deploy technologies can be challenging; strategic investment in discovery tools; learning from combined approaches from oncology, with fewer use cases in neurology
- Exploring a case study navigating spatial transcriptomics strategy
- Multiplexed IF following up on spatial transcriptomics and tech dev efforts on combined workflows
11:30 am Single Cell Approaches to Understand Drug Mechanism of Action & Target ID in Neuroinflammation
Synopsis
Impact on immune cells to look for specific approaches Single cell approaches to understand translational value of preclinical models
12:00 pm Exploring Proteomic Driven Biomarker Discovery for Improved Monitoring of Patient Benefit in CT1812 Alzheimer’s Disease Trial
Synopsis
- Pharmacodynamic biomarkers of CT1812 identified
- Unbiased pathway analysis shows support that CT1812 is impacting synaptic biology, amyloid biology and neuroinflammation
- Correlates to functional or clinical outcome measures identified to support a favorable clinical impact of CT1812
- Comparative analysis shows replication across independent clinical cohorts
12:30 pm LUNCH & NETWORKING
NAVIGATING BEST PRACTICE IMPLEMENTATION & UTILITY OF CURRENT NEUROINFLAMMATORY BIOMARKERS
1:30 pm Roundtable Discussion: Critically Assessing Neuroinflammatory Biomarkers & their Relevance to Current Clinical Development: Exploring Comorbidity & Drug Mechanism to Neuroinflammation
Synopsis
- Evaluating relevance of GFAP as an astrocytic marker to TREM2 programs – need better markers of microglia activated microglia
- Exploring the relevance of 5 different neuroinflammatory biomarkers: increased microglial activation, evidence of astrocytosis, elevated peripheral CRP (peripheral immune response), NFL and cytokine sand chemokines
- Accounting for 3 comorbidities of diabetes, obesity and hypertension that come with aging to give a more detailed picture for patients in trials
2:30 pm Scientific Poster Session:
Synopsis
This session is a great opportunity to introduce yourself to experts across leading neuroimmunology biopharma. Enjoy face-to-face time and share your work with many of the brightest minds, displaying scientific posters capturing the main advancements and challenges across discovery, preclinical, translational, and early clinical neuroinflammation to establish meaningful business relationships
3:00 pm AFTERNOON BREAK & NETWORKING
DOES THE TREM2 HYPOTHESIS RING TRUE? EVALUATING RECENT TREM2 DATA IN-CLINIC TO DETERMINE THE NEXT STEPS TO PAVE THE WAY TO TARGETING NEUROIMMUNOLOGY
3:30 pm Modulating TREM2 with Multiple Modalities to Treat Rare Genetically Defined Microgliopathies (VGL101) & Alzheimer’s Disease (VG-3927)
Synopsis
- Navigating VG-3927 small molecule approach to TREM2 agonism to address unmet needs in Alzheimer’s Disease
- Exploring phase 2 results of targeting rare genetically defined microgliopathies with Iluzanebart (VGL101) for ALSP2 by increasing signaling through DAP12/SYK to mitigate microglial dysfunction downstream
- Navigating genetic and biomarker strategies to aid early development and how a small molecule TREM2 agonist may address the unmet needs in Alzheimer’s and beyond
4:00 pm TREM2 Modulation as Anti-Inflammatory Approach for the Treatment of AD – Is There a Space Beside Anti-Amyloid Beta & Tau Agents?
Synopsis
- Development of anti-amyloid antibodies were highly dependent on biomarker development and the correlation with therapeutic efficacy has been crucial
- The link with neuroinflammation, AD pathology and therapeutic efficacy is yet to be established
- Clinical assessment of agents acting at TREM2 and linking their mechanistic efficacy with the impact on AD pathology will be key to successfully develop these agents
4:30 pm Rescuing Neurodegeneration with TREM2 Agonism: a Small Molecule Approach Targeting Neuroimmunology in Alzheimer’s Disease
Synopsis
- Exploring a small molecule approach to enhance microglial function with TREM2 modulation, to improve neuroprotection
- Navigating implications on back translation to the clinic
- Outlining early biomarker data (GFAP, NFL,…) and implementation approach to further determine patient benefit
- Exploring patient stratification rationale to identify patients most likely to benefit from treatment