Conference Day Two
Thursday March 20, 2025

8:00am Check-In, Coffee & Light Breakfast

8:50 am Chair’s Opening Remarks

FULFILLING GROWING UNMET PATIENT NEEDS AMONG ALZHEIMER’S & PARKINSON’S POPULATIONS

9:00 am Panel Discussion: Perspectives from Patients & Prescribing Neurologists on the Clinical Adoption of Disease-Modifying Anti-Amyloid Therapeutics

Synopsis

  • What is the patient burden associated with anti-amyloid therapies and how might these treatments be improved?
  • What are the key factors driving Lecanemab and Donanemab uptake among patients?
  • What are the hesitancies from prescribing neurologists to adapt to these new therapies?

9:30 am The Journey Toward a Disease-Transformative Therapy for Parkinson’s Disease: What are Patients Seeking from the Next Wave of Therapeutics?

  • Peter DiBiaso Patient, The Michael J. Fox Foundation for Parkinson's Research

Synopsis

  • What are the greatest challenges and burdens of currently available treatments for Parkinson’s disease?
  • What do those with early onset Parkinson’s disease hope to see in the evolving therapeutic landscape?
  • What are the key factors that impact the clinical trial decision-making process for Parkinson’s patients?
  • What do the advancements in the Parkinson's Disease staging system mean for patients?

10:00am Morning Break & Refreshments

TRACK 1: TARGET DISCOVERY & IN VITRO VALIDATION

Chair:

EMPLOYING THE LATEST COMPUTATIONAL TOOLS TO DIRECT FUTURE TARGET DISCOVERY

11:30 am Developing a Computational Method for Targeting Only the Toxic Misfolded Proteins in Alzheimer’s & Parkinson’s Disease

  • Neil Cashman Chief Scientific Officer & Co-Founder, ProMIS Neurosciences

Synopsis

  • Rationale for reducing toxicity by only targeting oligomeric forms
  • Demonstrating promising applications of the idea for targeting toxic species of amyloid, tau and alpha-synuclein
  • Leveraging the model to identify more efficacious, less toxic targets

12:00 pm Showcasing Experiences with Artificial Intelligence as a New Avenue for Neurodegenerative Target Discovery

Synopsis

  • Demonstrating AI facilitation in the accelerated development of Vincere's pipeline of small molecules
  • Analyzing clinical data to guide the optimization of drug development
  • Forward-looking insights on the potential of AI technology platforms to optimize clinical trial strategies, particularly in patient stratification

12:30 pm Roundtable Discussion: How Can Artificial Intelligence be Utilized & Implemented into Discovery Workflows to Identify a Biologically Driven Target?

Synopsis

More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas.

Are there real-world examples of how AI can be utilized to facilitate the following aspects of drug discovery?

Predicting how druggable a target is Incorporating AI with biology & bet

Predicting brain penetrance of potential therapeutics

Incorporating AI with biology & better understanding the pathophysiology of AD and 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

1:00 pm Lunch & Networking Break

DECIPHERING UNKNOWN GENETIC & MECHANISTIC DRIVERS OF NEURODEGENERATIVE DISEASE

2:00 pm Leveraging Population-Scale Genetics to Identify Heterogeneous Pathways in Alzheimer’s Risk & Progression

Synopsis

  • Integration of genome-wide association study summary statistics at scale
  • Methods for deconvolving distinct pathways from genomic data for target discovery
  • Exploring the pleiotropic heterogeneity of AD GWAS – lessons for patient stratification

2:30 pm Deciphering Genetic Risk Factors of Alzheimer’s Disease to Inform on Novel Therapeutic Approaches

  • Ralf Schmid Associate Director, Pre-Clinical Research, Novartis AG

Synopsis

  • Studying genetic markers in Alzheimer’s patients
  • Identifying the definitive genes or gene mutations that increase AD risk and may have a causal relationship
  • Identifying the definitive genes or gene mutations that increase AD risk and may have a causal relationship

TRACK 2: IN VIVO TRANSLATION & EARLY CLINICAL

REVIEWING EARLY CLINICAL PROGRESS OF PRECISION GENE THERAPIES

REVIEWING EARLY CLINICAL PROGRESS OF PRECISION GENE THERAPIES

11:30 am Demonstrating a Precision Medicine Approach to Targeting APOE4 Homozygotes to Treat the Multivariate Pathologies of Alzheimer’s Disease

Synopsis

  • Outlining the rationale for using APOE2 to develop a more specific genetic medicine
  • Showcasing the ability of LX1001 to supplement levels of APOE2
  • Reviewing the clinical data across all four dose cohorts

ADVANCING ANTI-INFLAMMATORY AGENTS TARGETING NEURODEGENERATION

12:00 pm Panel Discussion: What is Still Not Understood About the Role of Inflammation in Alzheimer’s & Parkinson’s?

  • Malú Tansey Director of the Center for Translational Research in Neurodegenerative Disease, University of Florida
  • Anick Auger Head of Pharmacology, Ventus Therapeutics
  • Manuela Polydoro Vice President, Head of Translational Medicine, Muna Therapeutics

Synopsis

  • How can we overcome cross-model differences in neuroinflammation to develop more translatable targets?
  • What are the latest understandings of the roles of microglia and astrocytes in AD and PD pathology? What is still unknown?
  • What is currently understood from preclinical studies about the possible peripheral immune component?

1:00 pm Lunch & Networking Break

PRECLINICAL PROGRESS OF NOVEL TARGETS BEYOND Aβ, TAU & α-SYNUCLEIN

2:00 pm Tackling the Root of Neuroinflammation & Mitochondrial Dysfunction: Leveraging a Translational Transgenic Model to Investigate the Reversal of Parkinson’s Pathophysiology

  • Dean Petkanas Co-Founder, Chairman & Chief Executive Officer, KannaLife Sciences Inc

Synopsis

  • Developing and testing a dual-acting compound to downregulate neuroinflammation in PD
  • Utilizing a transgenic model predictive of mitochondrial dysfunction and neuroinflammation
  • Showcasing results from first phase of the study and future directions

2:30 pm Showcasing Preclinical Promise of Targeting TDP-43 in Alzheimer’s Disease

  • Katie Planey Chief Executive Officer & Board Member, Acelot, Inc.

Synopsis

  • Reviewing why targeting TDP-43 has historically been difficult
  • Outlining the mechanisms of the small molecule to directly target TDP-43
  • Characterizing the animal efficacy data
  • Discussing TDP-43 prevalence in AD and future directions for clinical translation of this target

TRACK 3: LATE CLINICAL, REGULATORY & COMMERCIAL

Chair:

NAVIGATING THE EVOLVING REGULATORY LANDSCAPE FOR NEURODEGENERATIVE THERAPIES

11:30 am Securing Alignment with Regulators Around the Use of Optimal Endpoints & Evidence to Demonstrate Efficacy of Disease Modifying Therapies in Neurodegeneration

Synopsis

  • Reviewing the evolving regulatory landscape for AD and PD treatments
  • What can we learn from approval success stories in AD?
  • Navigating regulatory interactions to align on approvable endpoints that can demonstrate treatment efficacy over stand of care

12:00 pm Unpacking FDA’s Response to the Alpha-Synuclein Seed Amplification Assay: What Does This Mean for the Future of Parkinson’s Clinical Trials?

  • Fraser Bocell Senior Clinical Outcomes Assessment Scientist, Critical Path Institute (C-Path)

Synopsis

  • Showcasing the latest advancements of αSyn-SAA in monitoring drug-induced improvement in PD progression
  • Highlighting opportunities unlocked by αSyn-SAA to establish objective endpoints and monitor efficacy of PD drugs

12:30 pm Advancing the Reality of Using Biomarker-Driven Endpoints to Demonstrate Improvements in Cognitive Function & Behaviour

  • Manal Morsy Chief Regulatory Officer, Head of Global Regulatory Affairs, Vaxxinity

Synopsis

  • Exploring progress in biomarker assays and remote data collection
  • Outlining the key characteristics for selecting the most suitable assay, and at what cut off, to be used as evidence
  • Reviewing the evolving regulatory guidance on the use of these assays

1:00 pm Lunch & Networking Break

ADDRESSING MARKET ACCESS CHALLENGES TO DRIVE MORE COMMERCIALLY VIABLE NEURODEGENERATIVE DRUG PRODUCTS

2:00 pm Transitioning from Phase III Success to Commercial Activities: Ensuring Pre-Launch Readiness in the Alzheimer’s Treatment Landscape

  • Daniel Klamer Vice President, Business Development & Scientific Strategy, Anavex

Synopsis

  • Showcasing the positive clinical results of blarcamesine as a novel oral therapy for AD
  • Outlining key components of market research to support pre-launch readiness activities
  • Highlighting key commercial bottlenecks to consider and anticipate in preparations

2:30 pm Roundtable Discussion: Factoring Commercial Strategy into Clinical Trial Design & Decision Making

  • Elena Dale Executive Director, Neuroscience, Bristol Myers Squibb

Synopsis

More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas.

When is the right time to start thinking about commercialization and building up the infrastructure?

How might the approach and strategy need to be adjusted for launching the drug in US markets vs international markets?

How can we improve access to anti-amyloid therapies? Do the risks of ARIA outweigh the rewards?

3:00pm Afternoon Break & Refreshments

FORWARD-LOOKING INSIGHTS & FUTURE DIRECTIONS FOR NEURODEGENERATIVE DRUG DEVELOPMENT

3:30 pm 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?

4:00 pm Unveiling the Potential of a Novel Liposomal Therapeutic Vaccine Targeting Tau in Alzheimer’s Disease

  • Tricia Thornton-Wells Senior Director & Compound Development Team Leader, Johnson & Johnson Services, Inc

Synopsis

  • Showcasing the phase 2b study testing anti-tau active immunotherapy in preclinical AD
  • Utilizing plasma pT217 for prescreening directly from the community (leading to unprecedented diversity in recruitment)
  • Implementing a novel imaging analysis methodology looking at tau spread on Tau PET

4:30 pm Highlighting the Mechanism of GLP-1 Analogues & Demonstrating Their Promise to Reduce Neurodegenerative Diseases

Synopsis

  • Overview of the different mechanism by which GLP-1 analogues work
  • Reduction of neuroinflammation, Tau, Influence on insulin resistance, astrocyte etc.
  • Clinical studies evaluating GLP-1 analogues in Alzheimer’s disease and Parkinson’s disease
  • Potential mechanism on other neurodegenerative processes

5:00 pm Chair’s Closing Remarks & End of Conference