8:00 am
Morning Refreshments

8:30 am Chair’s Opening Remarks

TACKLING THE MULTIPLE APPROACHES TO DEGENERATIVE DISEASE & ENRICHING MODERN DEVELOPMENT TACTICS

8:45 am BIIB122: a LRRK2 Small Molecule Inhibitor for Parkinson’s Disease

  • Tien Dam Executive Medical Director - Neurodegeneration Development Unit, Head of Movement Disorders, Biogen

Synopsis

  • LRRK2 as a target against Parkinson’s Disease
  • Reviewing phase I data safety and target engagement
  • Evaluating study design approaches for ongoing clinical trials

9:15 am Update on Animal Models of Alpha-Synucleinopathy for Use in Therapeutic Drug Development

Synopsis

*Speaker to be Confirmed, Atuka

  • Investigating neuroinflammation with a focus on alpha-synuclein specific T cell responses
  • Exploring preformed fibrils such as motor and non-motor models
  • Deep Divining into AAV-alpha synuclein in rodent and non-human primate models and exploring motor and non motor endpoints

9:45 am Relating Pathway Biology & Human Genetics to Validate Therapeutic Targets for Parkinson’s Disease

Synopsis

  • Discussing preclinical validation of Parkinson’s targets derived from human genetics, focusing on lysosomal pathways
  • Discussing how lysosomal function can be quantified in the context of target validation
  • Comparison to validation of Parkinson’s targets not strongly linked to human genetics

9:45 am Lessons Learned from Clinical Trials in Parkinson’s Disease: The Prasinezumab Experience

  • Gennaro Pagano Expert Medical Director, Group Leader in Early Development, Roche

Synopsis

  • Recap of Prasinezumab reaching phase II trials targeting aggregated α-synuclein
  • Analysis of primary and secondary endpoint results
  • Looking ahead: applicable lessons learnt for future Parkinson’s trials

10:15 am
Morning Break & Networking

9:45 am Relating Pathway Biology & Human Genetics to Validate Therapeutic Targets for Parkinson’s Disease

Synopsis

  • Discussing preclinical validation of Parkinson’s targets derived from human genetics, focusing on lysosomal pathways
  • Discussing how lysosomal function can be quantified in the context of target validation
  • Comparison to validation of Parkinson’s targets not strongly linked to human genetics

DISCOVERY & PRE-CLINICAL TRACK Multifaceted Target Decisions for Parkinson’s Disease

11:15 am Developing Novel Plant-Inspired Treatments for Parkinson’s Disease

Synopsis

  • Identifying proprietary Minimum essential mixtures (MEM) from Cannabis extracts using a serially-reductionist approach, screening in silico, cell, and animal models of Parkinson’s disease
  • Cell-based studies identified novel complex mixtures of 8-12 Cannabis ingredients with both significant neuroprotective potential and the ability to increase dopamine secretion
  • Animal-based studies demonstrated that our simplified MEM containing three cannabinoids each significantly reduced Parkinsonian movement symptoms elicited using 6-OHDA
  • Our MEM for the treatment of Parkinson’s motor symptoms are being developed for their first-in-human trials by incorporation into Oral Dissolving Tablets which enhance drug delivery and patient compliance

11:45 am HER-096: Novel Brain-Penetrating Peptidomimetic that Promotes Proteostasis & Reduces Neuroinflammation in Aged Mouse Model of Synucleinopathy

Synopsis

  • Discovery and lead optimization of HER-096
  • Blood-brain barrier penetration in small and large animals
  • Target engagement, neuroprotection and modulation of neuroinflammation in an alpha-synuclein mouse model

TRANSLATIONAL & CLINICAL TRACK Reviewing the Clinical Developments in Parkinson’s Disease Research

11:15 am A Small Molecule c-Abl Kinase Inhibitor, IkT-148009, as a Potential Disease Modifying Therapy in Parkinson’s Disease

Synopsis

  • Understanding disease pathology to validate the c-Abl kinase as a drug target for modification of Parkinson’s Disease
  • Therapeutic evaluation of efficacy in animal models of inherited and idiopathic Parkinson’s Disease
  • Strategy and preliminary results in Phase 1 and Phase 2 clinical development

11:45 am Translational Inhibition of Multiple Neurotoxic Proteins Leads to Improved Motor Function in Parkinson’s Disease

  • Cheng Fang Senior Vice President, Research & Development, Annovis Bio

Synopsis

  • Parkinson’s disease has mixed pathology and proteinopathy
  • Inhibiting multiple neurotoxic aggregating proteins together shows beneficial effects
  • Buntanetap improved PD patient’s motor functions

1:45 pm Small Molecule OGA Inhibitors as a Multi-Modal Therapy to Combat Parkinson’s Disease

  • Ryan Schubert Senior Vice President Translational Medicine & Clinical Strategy, ASCENEURON

Synopsis

  • Assessing clinical stage OGA inhibitors
  • Beyond tau – exploring OGA inhibitors for a-synuclein pathology
  • Evaluating the potential of OGA inhibitors as a therapy for Parkinson’s Disease

12:15 pm
Lunch & Networking

ADDRESSING THE SPECTRUM OF TECHNOLOGIES EMPLOYED TO BOOST NEURO-DISCOVERY

1:45 pm Small Molecule OGA Inhibitors as a Multi-Modal Therapy to Combat Parkinson’s Disease

  • Ryan Schubert Senior Vice President Translational Medicine & Clinical Strategy, ASCENEURON

Synopsis

  • Assessing clinical stage OGA inhibitors
  • Beyond tau – exploring OGA inhibitors for a-synuclein pathology
  • Evaluating the potential of OGA inhibitors as a therapy for Parkinson’s Disease

2:15 pm Panel Discussion: Prevailing Therapeutic Strategy in the Face of Heterogeneity in Neurodegeneration

Synopsis

  • How can disease endotypes be identified and drug treatments tailored in the face of developing markers and mechanistic understanding?
  • Is a single target approach likely to be effective in most patients where the disease course is highly varied?
  • What are the challenges and drawbacks of the polypharmacy multi-target methods in an aged population?

2:45 pm Employing AI to Identify Potential Repurposing of Drugs to Produce Valid Candidates & Reduced Timelines in Neurology

  • Ana Puhl Associate Director, Collaborations Pharmaceuticals Inc.

Synopsis

  • Exploring a machine learning approach to repurpose existing candidates against neurodegenerative diseases
  • Investigating Alzheimer’s Disease pathways and building models, and predicting molecules for disease target pathways
  • Assessing ongoing in vitro investigations and future plans for testing in cellular models.

3:15 pm
Afternoon Break & Networking

3:45 pm Audience Discussion: Resetting the Regulatory Standard in Matching Measures to Patient Benefit

Synopsis

  • Evaluating recent approvals in neuroscience and their impact on the field
  • Understanding how biological markers can be implemented in approval processes
  • What are the optimal pathways for reaching marketable drugs as research moves forward?

4:15 pm Leveraging Proteomics Studies & Mass Spectrometry to Map Out the Landscape for Biomarker Discovery

Synopsis

  • Depth provided by available proteomic techniques
  • Interpretation of data discordance between approaches
  • Deconvolution of disease markers in aging population with multiple diseases

4:45 pm Audience Discussion: Venturing into Concerted Efforts to Reinforce Financial Stability & Encourage Innovation

  • Meriel Owen Associate Director, Search and Evaluation, Alzheimer's Drug Discovery

Synopsis

  • Exploring the shift in research funding and practice between academia, biotech and pharma for neuroscience
  • Outlining how to bridge the gap between lengthy high cost trials and lessened fiscal interest
  • Capitalizing on exciting advances in early research to fuel forward planning and address these unmet areas of need

5:15 pm Chair’s Closing Remarks

5:30 pm End of Conference