8:30 am Morning Networking

9:00 am Chair’s Opening Remarks

9:10 am Kick-off Panel Discussion

Synopsis

  • Overview of recent encouraging clinical case studies across the industry – what are the treatments that are around the corner promising results?
  • What has Biogen’s aducanumab story taught us?
  • What new research has been revealed to shine light on the mechanisms that are driving neurodegenerative pathology?
  • Is there space for all of these different modalities out there? Where should we concentrate our efforts?

9:50 am Applying Multi-Scale, Predictive Modeling to Optimize Neurodegenerative Disease Development using the Virtual Twin Neuro Platform

Synopsis

  • Translate preclinical and clinical knowledge to achieve patient outcomes
  • Individualized dosing regimen based on patient characteristics
  • Development of novel biologicals and modalities, including gene therapy
  • Selection and integration of pertinent biomarkers
  • Leverage big data to understand disease pathology and optimize combination therapy

10:20 am Speed Networking

Discovery Stream
Evaluating Monogenic Diseases, Protein Misfolding & Toxic Tau

11:20 am New Targets & Approaches to Targeting Monogenic Neurodegenerative Diseases

  • Michelle Hastings Director, Center for Genetic Diseases, Professor, Chicago Medical School, RFUMS

Synopsis

• What insights are we gaining from studying monogenic diseases to understand the complexity of much broader diseases?
• How can we leverage antisense oligonucleotide technology to find new targets for monogenetic diseases?
• Exploring the therapeutic efficacy of antisense oligonucleotides in mouse models of CLN3 Batten disease
• What is the clinical progress in rare monogenic neurodegenerative conditions?
• Broadening the therapeutic landscape for ASOs in the treatment of neurodegenerative diseases

11:50 am Precision Immunotherapies for Protein Misfolding Diseases to Advance ALS & FTD Therapeutics

  • Neil Cashman CSO of ProMIS Neurosciences, Professor, Neurology, University of British Columbia

Synopsis

  • Exploring how the ProMIS Platform is being leveraged to develop protein misfolding-specific immunotherapies for neurodegeneration
  • Case study: selective targeting of toxic forms of TDP-43 in ALS and FTD, while sparing normally folded functional TDP-43
  • Dissecting the role of prion-like propagation of misfolded proteins in neurodegeneration

12:20 pm Intraneuronal Transmission of Toxic Tau Species

Synopsis

  • There are multiple species of tau but specific toxic species are taken up by neurons
  • Others species of tau have different properties but also bind potential therapeutic antibodies
  • Antibodies that selectively bind to the toxic tau species have the potential to be the next generation of therapeutics for the treatment of Alzheimer’s Disease

12:50 pm Live Q&A With Your Expert Speakers

  • Mark Treherne Chairman, Gen2 Neuroscience
  • Michelle Hastings Director, Center for Genetic Diseases, Professor, Chicago Medical School, RFUMS
  • Neil Cashman CSO of ProMIS Neurosciences, Professor, Neurology, University of British Columbia
  • Selina Wray Alzheimer’s Research UK Senior Research Fellow, Professor of Molecular Neuroscience, UCL

Translational Stream
Driving Innovation in Clinical Trial Design

11:20 am Lessons Learned From Clinical Trials Targeting Alpha-Synuclein to Design Next-Generation Clinical Trials

  • Gennaro Pagano Expert Medical Director, Group Leader in Early Development, and Chair of Medical Directors Fellowship, Roche Pharma and Early Development (pRED)

Synopsis

  • How the results of the Phase 2 PASADENA study of Prasinezumab, a humanized monoclonal antibody for Parkinson’s disease, can inform future clinical trials
  • How to apply a precision medicine approach in Parkinson’s disease clinical trials?
  • Can we predict cognitive decline in Parkinson’s Disease to design studies evaluating this endpoint (lesson learned from PRECODE studies)?

11:50 am Pioneering Innovative Trial Design to Develop New Treatments for ALS

Synopsis

  • How to design more efficient and more effective clinical trials and broaden access for patients
  • Key considerations when choosing novel endpoints and biomarkers into innovative trial designs
  • Identifying and working with multiple stakeholders
  • Diving into the HEALEY ALS Platform Trial

12:20 pm Reverse Translation in Alzheimer’s Disease

Synopsis

  • Reverse translation allows hypothesizing on additional pathological pathways, on their “timing” along the continuum and gives an idea of the potential effect size
    of an intervention
  • APOE4 not only increases the risk of amyloid deposition but also generates structural vulnerability
  • New CSF biomarkers are showing early physiopathological changes in multiple biological cascades
  • It is key to define WHEN to intervene as a function of the compound MoA for a successful proof of concept
  • Fluid and imaging biomarkers allow us understanding the role of genetic and environmental risk factor

12:50 pm Live Q&A With Your Expert Speakers

  • Sabrina Paganoni Assistant Professor, Harvard Medical School
  • José Luis Molinuevo VP Clinical Development, Neurodegeneration, Lundbeck
  • Gennaro Pagano Expert Medical Director, Group Leader in Early Development, and Chair of Medical Directors Fellowship, Roche Pharma and Early Development (pRED)
  • Christian Schubert Global Head of External Innovation, Research & Development, Servier

1:15 pm Lunch Break

2:00 pm Targeting Myeloid Immune Cell Dysfunction to Revolutionize Neurodegenerative Treatment – From Discovery to the Clinic

2:30 pm Coding Cells for Health – Consistent & Scalable Human iPSCDerived Cells for in vitro CNS Disease Modelling & Drug Discovery

2:40 pm Targeting Amyloid Beta for Alzheimer’s Disease

  • Michele Vendruscolo Professor & CSO, Cambridge University Centre for Misfolding Diseases & Wren Therapeutics

3:10 pm ALZ-801 Phase 3 Program: A Targeted Oral Disease Modifying Therapy for Alzheimer’s Disease

3:40 pm The Time for Effective Precision Therapeutics to Treat Sub- Forms of ALS is Now

3:50 pm Clinical Case Study: Exploring AL001 for the Treatment of FTD

4:20 pm Live Q&A With Your Expert Speakers

4:40 pm Afternoon Networking

Discovery Stream
Advancing New Approaches to Drug Discovery

5:00 pm Exploring Cellular Approaches to Neurodegenerative Drug Discovery

  • Larry Brown Executive Vice President & Chief Scientific Officer, Noveome Biotherapeutics

Synopsis

  • Origin of Amnion-derived Multipotent Progenitor (AMP) Cells
  • Stem-cell like properties of AMP cells
  • Neuroprotective and immunomodulatory activities of AMP cells
  • AMP cell secretome

5:30 pm Investigating Post Translational Modifications as a New Therapeutic Approach for CNS Disorders

Synopsis

  • Unravelling a new area of biology to help elucidate the functional relationship between certain PTMs and protein localization
  • Mapping the palmitoylome and palmitoylation gene interactome yields putative disease targets
  • Hypotheses of how dysregulation of palmitoylation and protein mislocalization drives disease
  • Developing a novel class of molecules to target this area, focusing on Batten Disease and leveraging these insights to drive further understanding of AD

6:00 pm Discovery of Small Molecules for the Treatment of Neurodegeneration Caused by Protein Misfolding: Huntington’s Disease as a Case Study

  • Beth Hoffman Founder, President & CEO, Origami Therapeutics

Synopsis

  • Phenotypic screening to identify protein conformation modulators: discovery of protein degraders and conformation correctors
  • Characterization of small molecules using patientderived disease models and machine learning to prioritize small molecules based on efficacy and safety
  • Why we chose to tackle Huntington’s disease and how we can leverage our learnings to approach other neurodegenerative diseases

6:30 pm Live Q&A With Your Expert Speakers

  • Larry Brown Executive Vice President & Chief Scientific Officer, Noveome Biotherapeutics
  • Andrew Lim Chief Executive Officer, Circumvent
  • Beth Hoffman Founder, President & CEO, Origami Therapeutics
  • Rouba Kozak Senior Director, Neuroscience & Translational Medicine, Takeda

Translational Stream
Exploring Regenerative Therapy & Translational Tools

5:00 pm Pioneering Neuron Regeneration Therapeutics to Fill the Void in Treatment Options for AD

Synopsis

  • Exploring the unique mechanism of action of NNI-362, selectively stimulating the conversion of human brain neural progenitor cells to mature functioning neurons
  • Allowing the function to guide the target, assessing the journey from phenotypic screening of regenerative targets to the clinic
  • Safety profile in aged humans (SAD)
  • Plasma biomarkers can be used post-Phase 1a to guide the future POC trial
  • Evaluating brain adult-born neurons numbers in future trials based on preclinical data in aged and Down syndrome-modeled animals

5:30 pm Drug Repositioning for Parkinson’s Disease

Synopsis

  • Session details to be confirmed!

6:00 pm An Integrated Platform for Identification & Validation of Targets for Mitigation of Neuroinflammation in Neurodegenerative Diseases

Synopsis

  • Exploring how our systems biology platform is used to reveal novel targets for dementias
  • Linking human disease datasets with ex vivo / in vivo models of neuroinflammation
  • Identifying disease biology across 5 neurodegenerative diseases
  • How can we best validate neuroinflammatory targets identified in patients?
  • Discovering the best translational tools and models for studying neuroinflammation

6:30 pm Live Q&A With Your Expert Speakers

6:45 pm Chair’s Closing Remarks