7:55 am
Networking & Registration

8:55 am Chair’s Opening Remarks

Dissecting the 2022 Neurodegenerative Drug Development Landscape

9:00 am Exploring Big Pharma’s Direction in Neurodegenerative Disease

Synopsis

  • Analyzing current neurodegenerative drug development bottlenecks and strategizing how to progress beyond them: Spotlight on modelling, biomarkers and investment approaches
  • Exploring transformative new platforms on the horizon for Alzheimer’s, ALS, Parkinson’s, Huntington’s and more
  • Learning how to effectively incorporate novel research into your drug development approach to maximize success commercially and in the clinic

9:30 am How Regulatory-Ready Biosimulation is Revolutionizing Neurodegenerative Drug Development

Synopsis

  • Neurodegenerative diseases are complex and usually involve dysregulation in multiple biochemical pathways. Biosimulation is a proven technology to analyze these individual, integrated and combination challenges in silico.
  • Biosimulation can be used to simulate the interaction of different misfolded protein pathways with neuroinflammatory processes which is becoming increasingly important, not in the least by the identification of many microglia genes in large genetic studies.
  • Specifically, we discuss examples of how biosimulation couples the complex biological and pathological processes with the pharmacology of drugs in Alzheimer’s and Parkinson’s disease to evaluate the impact of mono and combination therapie

10:00 am
Structured Networking

Synopsis

This session is a great opportunity to introduce yourself to the attendees that you would like to have more in depth conversations with. Get face-to-face, peer-to-peer time with pioneering minds dedicated to neurodegenerative drug development research to establish meaningful business relationships

10:45 am
Morning Break & Networking

Discovery & Translational Track

Closing the Translational Gap: Update on Neurodegenerative Disease Models & Imaging

  • Phllip Kong Associate Director for Translational Medicine, Alector

11:20 am Discovery of Small Molecule Dual Inhibitors of Abeta and tau Aggregation using CCM technology

Synopsis

  • Development of an in-silico model (CCM) to identify small molecule inhibitors of protein misfolding
  • Description of the assay platform developed by Treventis to detect the earliest phases of intrinsically disordered protein aggregation
  • Presentation of in vivo target engagement of amyloid beta and tau oligomers

11:50 am In Vitro Transcytosis Models for BBB targeting CNS Drug Screening

Synopsis

  • Outlining challenges with in vitro models for neurodegenerative drug development and learning the key attributes for a good model
  • Developing in vitro models for different species
  • Utilizing in vitro models for BBB targeting neurodegenerative drug screening

12:20 pm High Throughput Platform for Rapid Screening of Compounds on Over 3 000 Patients’ Derived Neurons in 30 Minutes

Synopsis

  • Uncovering robust compound screening on patients’ derived neurons
  • Revealing rapid evaluation of compounds’ toxicity and efficacy in Alzheimer’s Disease models
  • Investigating phenotypic markers of degeneration and regeneration

12:30 pm Advancing Neurodegenerative Drug Discovery with a New Class of Consistent, Scalable Human iPSCDerived cells

Synopsis

  • Discovering how next generation precision reprogramming technology, opti-ox(TM), is overcoming the inefficiencies
    and inconsistencies of current cell generation methods
  •  Understanding how precision reprogramming is powering a new class of rapidly maturing, consistent and scalable
    human iPSC-derived cells (glutamatergic neurons, GABAergic neurons and microglia) and associated disease models for neurodegenerative disorders such as Gaucher’s, Huntington’s and Parkinson’s disease
  • Exploring insights into CNS drug discovery case studies, Compatibility of ioGlutamatergic Neurons for high throughout screening and the application of optiox powered GBA and Parkinson’s disease models in functional phenotypic screens

Late Translational & Early Clinical Track

Analyzing the Latest Biomarkers Research to Determine Which One is the Most Useful for Neurodegenerative Disease Drug Development

  • Eric Nelson Senior Business Development Consultant, US HealthVest

11:20 am How to Define Patient Selection, Selection of Optimal Intervention Points & Therapeutic Outcomes Using Biomarkers & “Prodromal AD”

Synopsis

  • Learning tips and tricks to streamline the use of “biomarkers” for patient selection
  • Using longitudinal biomarker measures of high scientific interest in the field to augment the use of ‘clinical/cognitive efficacy” outcomes
  • Uncovering data driven methods to optimize the selection of patient and protocol duration and endpoints

11:50 am Novel GEM Models For Exploring Neurodegenerative Disease Therapeutics

  • Steve Smith Director of Business Development, GemPharmatech

Synopsis

  • Challenges with in vivo models for neurodegenerative disease drug development
  • Key attributes of successful CNS disease in vivo models
  • Developing in vivo models for neurodegenerative drug
    screening

12:00 pm Audience Discussion: Understanding What Features the FDA/EMA Assess When Approving Biomarkers in the Context of Neurodegenerative Disease Drug Development

Synopsis

The difference between a successful and unsuccessful clinical trial can come down to what biomarker you choose to measure the efficacy of your therapeutic. It is critical to play close attention to just what features your selected biomarker must possess to optimize the success of your novel therapeutic in a clinical setting. Join this structured audience discussion to debate how to strategically select your next neurodegenerative biomarker in line with FDA/EMA guidelines. Learn how to optimize your approach to tackling common challenges associated with utilizing biomarkers in neurodegenerative drug development.

12:30 pm Fluid & Imaging Biomarkers in ALZ-801 Development: A First-in-Class Oral Disease Modifying Agent for Alzheimer’s Disease

  • John Hey Chief Scientific Officer, Alzheon

Synopsis

  • Harnessing the power of fluid biomarkers and imaging modalities as diagnostic and theragnostic biomarkers in drug development for Alzheimer’s disease
  • Revealing novel Alzheimer’s biomarkers used in modification trials and understanding their impact on disease modification trials
  • Comparing and contrasting CSF versus plasma biomarkers on disease progression and severity

1:00 pm An AI-Driven Locus-Specific Database (LSDB) for 35 ALS-Associated Genes

Synopsis

  • Understanding the challenges of diagnosing and treating ALS
  • Exploring how a combination of AI (artificial intelligence) and expert review can accelerate variant interpretation
  • Reviewing key findings, and highlighting their implications for drug development

1:10 pm
Lunch & Networking

Discovery & Translational Track

Streamlining Target Discovery

  • Eric Nelson Senior Business Development Consultant, US HealthVest

2:00 pm Learning How to Integrate Information on Cellular Communication & Pathways for a Multi-Omic Approach to Target Identification

Synopsis

  • Hearing how to use fluid biomarkers for patient stratification demonstrates the current limitations of clinical disease classifications.
  • Understanding the urgent need to better understand causative mechanisms of CNS pathology, novel insights from bulk and single cell transcriptomic
  • Exploring reverse translation of clinical trial data; learning more from man than mouse

2:30 pm The New Target Journey: How to Progress from Target Identification & Illustrate Meaningful Effect in Pre-Clinical Models

  • Je Min Yoo Chief Technology Officer, BioGraphene Inc

Synopsis

  • Exploring the serendipitous discovery of the therapeutic candidate
  • Outlining the journey of advancement from discovery to in vitro & in vivo studies
  • Investigating expansion to further indications

3:00 pm Abeta oligomers in Alzheimer Disease: Target Engagement and Target Distraction

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

Synopsis

  • Exploring aggregated Abeta (fibrils and oligomers, not monomers) is the target of many emerging antibodies and other therapeutics
  • Investigating oligomeric Abeta as the appropriate molecular species for therapeutic activity without adverse effects (e.g., ARIA)
  • Understanding why PMN310 from ProMIS is specific for Abeta oligomers, without target distraction from monomers or fibrils

Late Translational & Early Clinical Track

Analyzing Key Clinical Trial Updates

2:00 pm Uncovering Bryostatin Targets Restoration of Synaptic Wiring Lost in Alzheimer’s Degeneration

Synopsis

  • Investigating actual cognitive improvement over baselines measured in initial placebo-controlled clinical trials
  • Hearing evidence for sustained benefit for weeks after drug regimens have been in trialled
  • Showcasing targets including synaptic growth factors, the apoptosis pathway, as well as amyloid and neurofibrillary tangles

2:30 pm From Concept to Clinic: Targeting the Nitric Oxide-Soluble Guanylate Cyclase-cGMP Pathway for Neurodegenerative Diseases

  • Chris Winrow Vice President & Head of Translational Medicine, Cyclerion

Synopsis

  • Unlocking the full therapeutic potential of the nitric oxide-soluble guanylate cyclase-cGMP signaling pathway in the brain
  • Demonstrating successful clinical translation of sGC stimulation for CNS drug development
  • Exploring the benefits of incorporating objective biomarkers in early clinical development

3:00 pm End of Track

3:30 pm
Afternoon Break & Networking

The Beta Amyloid Revival & Beyond: A Deep Dive into Clinical Trials for Alzheimer’s Disease

4:00 pm ALZ-801 Phase 3 Program: Lessons & The Path Forward in Developing Targeted Therapies for Alzheimer’s Disease

  • Martin Tolar Founder, President & Chief Executive Officer, Alzheon

Synopsis

  • Highlighting the benefits of applying genomic stratification of patients for validation of biomarkers from target discovery to therapeutic development
  • Describing discovery of a novel molecular mechanism of action of ALZ- 801, which blocks formation of toxic amyloid oligomers associated with development and progression of Alzheimer’s disease
  • Reviewing development of ALZ-801, a Phase 3, first-in-class, small molecule oral inhibitor of amyloid aggregation and neurotoxicity
  • Emphasizing need for application of Precision Medicine approach in neurodegeneration, based on individual genetic and biological information to advance therapies with the greatest impact for patients

4:30 pm Deep Diving into the Development of a PPAR Delta/Gamma Agonist T3D-959 for Alzheimer’s Disease

Synopsis

  • Understanding the PPAR rational in Alzheimer’s Disease
  • Revealing the latest pre-Clinical and early clinical data for T3D-959
  • Showcasing ongoing PIONEER Phase 2 Study with T3D-959

5:00 pm End of Day One