8:30 am Online Registration & Virtual Coffee Networking

9:00 am Chair’s Opening Remarks

Driving Innovation in Fluid Alzheimer Biomarkers

9:10 am Addressing the Primary Issue – What Function are we Developing Biomarkers For?

  • Steven Arnold Professor of Neurology, Harvard Medical School, Translational Neurology Head , MGH Interdisciplinary Brain Center

Synopsis

  • Contextualizing the importance of biomarkers in terms of diagnosis, prognosis, appreciated heterogeneity and stratification
  • What do we require from the performance of the biomarker for it to be useful in a clinical trial?
  • What is the role of biomarkers for answering the questions that a patient has when they come to a neurologist?
  • What have we unravelled over the last few years that will drive more effective biomarker discovery and development?
  • Where does the field need to put more efforts to develop biomarkers that will be able to help us test new hypothesis in the Alzheimer’s field?

9:30 am SavaDx, an Investigational Blood-Based AD Diagnostic/Biomarker

Synopsis

  • SavaDx provides a quantitative interpretation of Alzheimer’s disease
  • SavaDx achieves its intended purpose through biochemical assessments in a small quantity of blood
  • In a small clinical study, SavaDx strongly correlated with patient improvements in validated CSF biomarkers of AD

9:50 am Neuron Derived Exosomes – A Novel & Flexible Platform for Disease & Therapy Biomarkers

Synopsis

  • The specificity of ExoSORT™ for neuron derived exosomes using spike-in recovery, protein and RNA analysis.
  • Utilizing neuron-derived exosomes for proteomic, lipidomic and transcriptomic analysis
  • The potential of neuron-derived exosomes for early detection of Alzheimer’s patients, additional neurological indications, and as a therapy response monitoring biomarkers
  • The ExoSORT™ potential – beyond neurons, GLIA cells and non-CNS indications

10:20 am Live Q&A

  • Steven Arnold Professor of Neurology, Harvard Medical School, Translational Neurology Head , MGH Interdisciplinary Brain Center
  • Lindsay Burns SVP of Neuroscience, Cassava Sciences, Inc
  • Erez Eitan Chief Scientific Officer, NeuroDex
  • Dimitrios Kapogiannis Clinical Investigator, Laboratory of Clinical Investigation, National Institute on Aging, NIH , Adjunct Associate Professor, Dept. of Neurology, National Institute of Health

Synopsis

Your chance to ask your expert speakers those burning questions

10:50 am Virtual Speed Networking

Synopsis

Grab a quick cup of coffee from the comfort of your own kitchen and jump straight into this exclusive virtual speed networking! This session is the ideal opportunity to meet face-to-face (albeit 2D faces) with many of the brightest minds working in the biomarkers for Alzheimer’s field and establish meaningful business relationships.

Exploring Biomarker Implementation in Clinical Trials to Drive Therapeutic Success

11:40 am Leveraging Biomarker Data & Preclinical Models to Guide the Design of Clinical Studies

  • Christopher Winrow Senior Director, Clinical Development - Neuroscience Program Lead, Cyclerion

Synopsis

  • How can we select and capitalize on the most appropriate preclinical studies to guide clinical strategies?
  • Evaluating the different modalities including CSF, plasma markers and imaging approaches to define the right clinical end points
  • How we can design early clinical translational pharmacology studies to rapidly evaluate biomarkers

12:00 pm Employing Biomarkers to De-risk Therapeutic Development in AD

Synopsis

  • Aiding the developing of novel therapeutics in clinical trials by inclusion of fluid and imaging biomarkers
  • Exploring AD case study evidence to evaluate the importance of patient selection tools, target engagement/ pharmacodynamic measures and biomarkers of disease progression

12:20 pm Therapeutic & Diagnostic Biomarkers that Target Synaptic Loss in AD

Synopsis

  • Peripheral molecular biomarkers – autopsy validated diagnostics for AD
  • Clinical evidence that PKC epsilon – BDNF therapeutics improve cognitive function
  • Pre-clinical evidence that PKC epsilon-BDNF therapeutics enhances synaptic restoration, new synapse formation, prevention of neuronal death, reduces A Beta oligomers and plaques, and prevents tau hyper-phosphorylation
  • Convergence of diagnostics and therapeutics suggests targeting of early causal factors in AD including brain deficits of the synaptic growth factor, BDNF, and the synaptic growth coordinator PKC epsilon
  • Autopsy validation of AD suggests a Gold Standard of AD pathology that includes dementia in life, amyloid plaques and neurofibrillary tangles, and synaptic loss at autopsy

12:40 pm Live Q&A – Fire Away & Ask Those Questions!

  • Daniel Alkon Scientific Director, Neuro Diagnostics LLC
  • Christopher Winrow Senior Director, Clinical Development - Neuroscience Program Lead, Cyclerion
  • Danielle Graham Senior Director, Discovery & Clinical Biomarkers, Biogen

1:00 pm Lunch Break & Panel Discussion

1:10 pm Panel Discussion: Advancing Digital Biomarkers for Neurodegenerative Diseases

  • Mark Frasier Senior Vice President, Research Programs Michael J Fox Foundation
  • Rhoda Au Professor of Anatomy, Neurobiology, Framingham Heart Study, Boston University Schools of Medicine

Synopsis

This panel discussion aims to discuss the strengths and limitations of digital health technologies, the opportunities and risks associated with this emerging business ecosystem, and how these technological advances in combination may lead to reframing the types of questions to pose.

  • Digital phenotyping as the first step to identifying and validating digital biomarkers
  • Discuss how to build the right infrastructure needed to drive innovation in digital biomarker discovery
  • Hardware, Software and Data challenges
  • Driving forward to a vision of AD precision medicine and AD prevention

Exploring Biomarker Implementation in Clinical Trials to Drive Therapeutic Success

2:10 pm ALZ-801 Phase 3-Ready Program: A Targeted Therapy for AD

Synopsis

  • How we approach AD biomarker development for ALZ-801 Phase 3 program and Alzheon portfolio, roadmap to value
  • Exploring key biomarker of AD progression: hippocampus volume loss correlating with cognitive impairment and clinical deterioration
  • Driving optimal phase 3 study design by defining primary, secondary and biomarker endpoints (hippocampus volume on MRI, CSF & plasma Ab42/40, pTau181, NfL, sTREM2, neurogranin)

Inflammatory Markers & the Relationship with Cognitive Performance, Cognitive Decline & Clinical Progression

2:30 pm One Size Does Not Fit All: Inflammatory Biomarkers Across the Disease & Drug Development Spectrum

Synopsis

  • Very few things have been as hotly debated as inflammation in Alzheimer’s disease. Main points of contention are chicken or the egg, good vs bad inflammation, and what’s the right inflammatory target?
  • Diving into how we have used inflammatory biomarkers to address these, and other, questions and how we have implemented biomarkers in our clinical development program
  • Evaluating the current limitations of inflammatory biomarkers, how can we overcome these

2:50 pm Immune Dysfunction in Parkinson’s Disease: Insights into Neuroinflammation

Synopsis

  • Investigating how specific aspects of the human immune system relate to neurodegeneration
  • Approaches to stratifying patients quantitatively with different forms of Parkinson’s disease based on immune function and cell phenotypes
  • How do we segment the various Parkinson patient populations for the best results?
  • Precision enrollment for Clinical Trials
  • Translatability from Parkinson’s clinical biomarker studies to Alzheimer’s biomarker development

3:10 pm Live Q&A – Fire Away & Ask Those Questions!

3:40 pm Virtual Speed Networking

Evaluating How Genetics Can Guide Biomarker Discovery & Development

4:10 pm Finding the Right Patients: Can Genetics Inform Selection of Fluid Biomarkers that Identify Subjects with a Specific Disease Biology?

Synopsis

  • Heterogeneity in patient populations is likely due, in part, to heterogeneity in disease biology
  • There is a need for biomarkers that can identify patients with a common disease biology
  • An approach that leverages genetics and bioinformatics can be used to find new biomarkers and new uses for existing biomarkers

4:30 pm Combining Genomics & Multiplex Proteomic Profiling for AD Drug Discovery & Development

Synopsis

  • Examining the role of multiplex proteomics for biomarker discovery and greater understanding of disease biology
  • Advances in genetics-guided drug discovery – combining genetic and proteomic data to discover and validate powerful new drug targets
  • Pressure-testing polygenic risk scores, plasma biomarkers, and rapid cognitive batteries as screening tools for Alzheimer’s disease
  •  Thoughts on how the field is moving towards personalized medicine

Learning from other Neurodegenerative Disorders: Major Developments in Biomarkers for Parkinson’s to Drive Your Own R&D

4:50 pm Accelerating Parkinson’s Disease Outcome Measures: Progress & Challenges

  • Mark Frasier Senior Vice President, Research Programs Michael J Fox Foundation

Synopsis

  • Brief overview of the Michael J Fox Foundation strategy to accelerate biomarker research
  • Highlight areas of scientific progress in Parkinson’s biomarkers across different modalities including biochemical, genomic, neuroimaging, digital sensor markers and clinical scales
  • Outline remaining challenges in outcome measure development in Parkinson’s disease

5:10 pm Translating Key Developments in Biomarker Studies in Parkinson’s to Help Advance Disease Modifying Therapeutics for AD

  • Richard Wyse Global Director of Research and Development, Cure Parkinson’s Trust UK

Synopsis

  • Overview of the global approaches to employing biomarkers in preclinical and clinical trials for Parkinson’s, lessons from the International PD Linked Clinical Trials Initiative, and PPMI
  • Establishing biomarker-defined cohorts and identifying clinical and genetic Parkinson’s disease
  • Approaches to validating emerging biomarkers for PD
  • Developing biomarkers to elucidate the role of mitochondrial function in PD pathogenesis
  • Leveraging neurogenetics to help drive new drug targets and biomarkers for PD

5:40 pm Live Q&A – Fire Away & Ask Those Questions!

  • Richard Wyse Global Director of Research and Development, Cure Parkinson’s Trust UK
  • Mark Frasier Senior Vice President, Research Programs Michael J Fox Foundation
  • Jonathan Levenson Vice President of Translational Biology, Tiaki Therapeutics

6:00 pm End of Summit & Virtual Happy Hour Begins

Synopsis

Grab a beverage of your choice from your home bar and use this final networking opportunity to follow up on connections made earlier in the day, ask that burning question to a speaker whose talk got you thinking and forge relations that could lead to your next collaboration

7:00 pm End of Virtual Happy Hour

Exosomes: Drivers in AD Pathogenesis & a Valid Source of Biomarkers

  • Dimitrios Kapogiannis Clinical Investigator, Laboratory of Clinical Investigation, National Institute on Aging, NIH , Adjunct Associate Professor, Dept. of Neurology, National Institute of Health

Synopsis

  • Delving into the technical aspects and strategies for exosome-based biomarker development and optimizing existing methodologies for isolating brain-origin exosomes from peripheral blood
  • Exploring data from diverse cohorts that tell us to what extent exosomes can be used as biomarkers for predicting Alzheimer’s and other neurological diseases
  • Revealing how exosome biomarkers correlate with brain levels in animals and pathological features on autopsy in humans
  • Investigating and validating new biomarkers to assess response to experimental treatments