Blood-Based Biomarkers Focus Day
Pre-Conference Workshop Day
7:30 am Registration & Coffee
7:55 am Chair’s Opening Remarks
REFINING BIOMARKER DEVELOPMENT STRATEGY FOR EARLIER DETECTION & INTERVENTION OF AD & PD
8:00 am Integrating Non-Invasive Biomarkers to Allow for Earlier Intervention in Treating Neurodegenerative Disease
Synopsis
- The hallmarks of Alzheimer’s Disease require a multimodal approach of biomarkers to evaluate heterogeneity of disease
- Non-invasive biomarker modalities for diagnosis, staging and treatment response
- Enabling a personalized approach using global proteomics and molecular subtyping to reflect disease heterogeneity
HARNESSING BLOOD-BASED BIOMARKER DISCOVERY & VALIDATION FOR IMPROVED SPECIFITY & MORE SENSITIVE QUANTIFICATION OF NEURODEGENERATION
8:30 am Identifying Pathological Tau Aggregates as a Biomarker in CSF of Alzheimer’s Disease Patients
Synopsis
- Differentiating isolation approaches applied to identify seeding competent tau aggregates from AD brain homogenates
- Generating high-affinity antibodies towards specific conformations and phospho sites of pathological tau
- Harnessing them to identify tau aggregates in CSF of Alzheimer’s disease
9:30 am Speed Networking
Synopsis
Network and form lasting connections with your peers working in blood-based biomarker R&D for neurodegenerative disease in a small relaxed environment
10:00 am Diagnostics and Therapeutics for Selective Targeting of Early Toxic Oligomers
Synopsis
- Highly specific and sensitive plasma Soluble Oligomer Binding Assay (SOBA) with FDA breakthrough designation for AD
- Soluble Oligomer Binding Inhibitor (SOBIN) for neutralization of toxic oligomers and their effects in vivo
- SOBA test applicable to other amyloid diseases, including PD
10:30 am Proof of Mechanism that Investigational Therapeutic CT1812 Impacts Key Alzheimer’s Disease Biologies
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 favourable clinical impact of CT1812
- Comparative analysis shows replication across independent clinical cohorts
11:00 am Roundtable Discussion: Identifying Patient-Derived Biomarkers through Lipid Signatures from Non-Invasive Patient Samples for Earlier Detection of Neurodegeneration
Synopsis
- Assessing early PK/PD relationship to inform early trial dynamics of markers
- Integrating blood based biomarkers, clinical presentation, patient history and imaging for more streamlined patient diagnosis
- Harnessing biofluids for less invasive diagnosis, staging and prediction of treatment response in patients
12:00 pm Lunch
1:00 pm Unbiased Multiomic Analysis to Identify Blood Based Biomarkers in Neurodegeneration
Synopsis
- Integrating proteomics, metabolomics and methylation into a molecular map
- Causal inference to identify novel genes driving associations to act as a druggable target
- Integrating findings into refined prediction models
1:30 pm Distinguishing Pathological & Healthy Neuronal Signatures on Brain-Derived Analytes in the Blood for Neurodegenerative Disease
Synopsis
- Concordance between CSF and blood-derived biomarkers
- Predictive power of fluid biomarkers
- Utility in patient selection and efficacy
- Exploring the latest insights from Phase 1 and ongoing Phase 2 data from a disease-modifying Parkinson’s Disease trial
ENABLING NEURODEGENERATIVE BLOOD-BASED BIOMARKERS AS CLINICAL & SURROGATE ENDPOINTS
2:00 pm Afternoon Break & Refreshments
2:30 pm What can Parkinson’s Disease Learn from the Recent Progress in Alzheimer’s Disease? Lessons for HER-096
Synopsis
- Learning from the recent Alzheimer’s Disease trial successes and how this can help inform PD treatment development, highlighting the importance of biomarkers
- Understanding the importance of surrogate markers and development of clinical endpoints for a faster read on clinical trials
- Pushing early biology and biomarker discovery to support clinical development, examples from HER-096
3:00 pm Panel Discussion: Overcoming Barriers to Clinical Utility of Emerging Non-Invasive Blood & Imaging Tests
Synopsis
- Navigating accessibility challenges internationally to blood based biomarkers for early diagnosis
- Employing consortia to strategically make biomarkers more reliable, generalizable and clinically relevant to enhance the quality and credibility of biomarker validation studies
- Exploring future directions in biofluid sampling at-home for improved patient experience and retention in clinical trial
7:30 am Registration & Coffee
8:00 am Workshop A: Next-Generation Blood-Brain Barrier Penetrant Therapeutic Antibodies
Synopsis
The recent approval of anti-Aβ antibodies has ushered in new possibilities and challenges in the realm of neurodegenerative diseases. A significant hurdle lies in the blood-brain barrier, permitting only 0.01-0.1% of antibodies to traverse into the brain, primarily through cerebrospinal fluid. The pivotal and complex challenge of achieving selective delivery of therapeutic antibodies with high bioavailability to the brain is at the forefront of drug development.
In this workshop, we delve into innovative approaches to surmounting the challenges associated with the blood-brain barrier, including:
- Reviewing the challenges and improvements necessary for brain shuttles to effectively cross the blood-brain barrier.
- Investigating the unique clustering pattern of transferrin receptors expressed on brain vessels, which are more densely distributed than in other tissues.
- What is the consensus on transferrin receptor targeted therapeutic design needs: monovalent versus multivalent, as well as low affinity versus high affinity.
- Discussing BBB targets beyond transferrin receptors including CD98hc and IGF1R.
- Applying the principle of super-selectivity to engineer therapeutic antibodies, enhancing brain bioavailability without compromising systemic pharmacokinetics.
Join this session to gain insights into the latest research, contribute to the discussion, and explore innovative solutions in the delivery of therapeutic antibodies across the blood-brain barrier.
*There will be a short morning break and refreshements available at 9:30 during this session
10:30 am Workshop B: Advancing Gene Therapy to Treat Alzheimer’s & Parkinson’s Disorders
Synopsis
As the last couple of years have welcomed approved neurological gene therapies from Bluebird Bio, PTC Therapeutics, Novartis, Sarepta Therapeutics, what is the route forward for gene therapy development for Alzheimer’s & Parkinson’s disease? This workshop explores the most recent developments in gene therapy, including brain delivery, preclinical models, translation of gene therapy research into clinical trials, including viral, non-viral vectors.
- Evaluating models of gene therapy treatment in in vivo and in vitro and exploring new innovations to recapitulate gene therapy efficacy preclinically
- Reviewing technologies gaining approval for direct brain delivery in gene therapy
- Translating gene therapy biomarkers for CNS indications including the validation
- Comparing the safety and efficacy of viral and non-viral vectors for gene therapy including AAV, lentiviral, non-viral
*There will be a break for lunch at 12:00 during this session
1:30 pm Workshop C: Harnessing Digital Biomarkers for Internal Decision-Making in Alzheimer’s & Parkinson’s Disease
Synopsis
As the FDA moves towards defining digital biomarkers as digital health assessments, wearable technology becomes more refined and digital markers can provide early signs of drug efficacy. Digital biomarkers can support with earlier detection to provide more longitudinal and frequent assessment, but also support internal decision making for biopharma by better predicting drug efficacy.
Look forward to:
- Exploring stride velocity as a newly approved digital marker by the EMA and the direction that regulation is moving in
- Utilizing digital insoles to monitor stride velocity as a marker of motor symptoms for Parkinson’s Disease
- Utilizing passive collections monitoring activity over time from walking, shopping, social engagement and interactions
- Navigating the regulatory route to home testing to improve patient compliance
- Harnessing home EEG devices for measuring sleep and sleep disturbance as a marker of Alzheimer’s Disease
- Using artificial intelligence and machine learning to predict deviation from baseline to establish using digital biomarker