Day One

Wednesday, May 17th

7:30 am Morning Refreshments & Registration

8:25 am Chair’s Opening Remarks

SHOWCASING THE CUTTING-EDGE OF ALS GENOMICS & BIOMARKERS TO INVESTIGATE PATIENT SUBPOPULATIONS & MEASURE DISEASE PROGRESSION

8:30 am Evaluating Neurofilament Light Chain as a Biomarker to Measure Therapeutic Effect on ALS Disease Progression

Synopsis

• Exploring the capabilities of neurofilament light chain, poly(GP), poly(GA) and poly(GA) as biomarkers for ALS diagnosis

• Highlighting discrepancies in neurofilament light changes in recent ALS clinical trials

• Discussing serum vs CSF measurements to measure impact on disease

9:00 am Inside the Genomics of ALS – Implications for Drug Discovery & Development

  • Mark Kiel Chief Science Officer & Founder, Genomenon Inc.

Synopsis

  • Discover how the genetic heterogeneity of ALS has ramifications for clinical practice
  • Understand the elements of comprehensive genomic landscapes and why they are needed to fully understand the genetic basis of ALS
  • Review the ALS Genomic Landscape, and learn how to identify key findings and their implications for ALS drug discovery development

9:10 am An On-Going ALS Natural History Study to Distinguish Patient Subpopulations & Identify Novel Biomarkers

  • Fernando Vieira Chief Executive Officer & Chief Scientific Officer, ALS Therapy Development Institute

Synopsis

• Identifying biomarkers to distinguish ALS patient subpopulations for optimized clinical trial recruitment

• Connecting biomarkers and target discovery to reveal ALS biology engaged in different subpopulations

• Building data to apply approaches for predicting ALS susceptibility and early disease diagnosis

9:40 am Utilizing Biomarkers to Measure Changes in Neuroinflammation & Neurodegeneration to Validate Safety & Efficacy in ALS Clinical Trials

Synopsis

• Rationale for targeting the LINE-1 reverse transcriptase against ALS and FTD

• Exploring biomarkers to measure changes in neuroinflammation and neurodegeneration

• Contextualizing biomarker usage for safety and efficacy measurements in phase 2a clinical trials

10:10 am Morning Break & Networking

EXPLORING ALS THERAPIES OF DIFFERERNT MODALITIES TO DEVELOP EFFECTIVE NOVEL TREATMENTS FOR ALS PATIENTS

11:00 am Exploring a Splice Modulation Antisense Oligonucleotide to Restore STATHMIN-2 Levels in ALS Patients

  • Dan Elbaum Chief Scientific Officer, QurAlis Corp.

Synopsis

• A splice modulation ASO strategy with the aim to treat 90% of the total ALS population (TDP-43 positive patients)

• Optimizing dosage to ensure efficacious and safe therapeutic output

• Biomarker strategies to evaluate target engagement and efficacy for STMN2 ASO trials

11:30 am – Evaluating a Next Generation Anti-CD40L Antibody as a Therapy for Patients with ALS

Synopsis

  • The rationale for targeting CD40L in patients with ALS
  • Results of a pilot study targeting CD40L in patients with ALS
  • Looking to the future with targeting CD40L in patients with ALS

12:00 pm Modality Roundtable Discussion

Synopsis

Join conversations with your fellow peers to discuss the benefits and challenges of different ALS therapeutic modalities. Join tables discussing therapeutic index, CNS delivery methods, target engagement and participant considerations

  • ASO's
  • Gene Therapy
  • Biologics
  • Small Molecules

12:45 pm Lunch & Networking

LEARNING THE REGULATORY PERSPECTIVE TO UNDERSTAND CURRENT ACCEPTED REQUIREMENTS FOR ALS THERAPY ENDPOINTS & APPROVALS

1:45 pm Reviewing ALSFRS-R as the Established Endpoint for ALS Clinical Trials

  • Stacy Lindborg Co-Chief Executive Officer, BrainStorm Cell Therapeutics

Synopsis

  • Demonstrating and quantifying challenges with the ALSFRS-R endpoint in its current state
  • Showcasing evidence from alternative measurements to demonstrate impact on disease progression
  • Outlining ongoing strategy to approach regulatory review through leveraging regulatory flexibility

2:15 pm Showcasing Clinical Findings for Toferson: Disease-Modifying Capabilities Targeting SOD1 Genetic Mutation

Synopsis

  • Explaining ongoing clinical strategy in extended phase 3 clinical trials
  • Outlining lessons learnt regarding clinical endpoints from interactions with regulatory authorities
  • What is the future for therapies targeting familial ALS seeking approval?

2:45 pm Panel Discussion: Creating an Open Dialogue Between People Living with ALS & Drug Developers to Co-ordinate Therapeutic Development Priorities

Synopsis

  • Hearing directly from people living with ALS – what are the most important priorities in ALS drug development?
  • Discussing participant engagement and experiences from most recent clinical trials – what improvements can be made?
  • Identifying the immediate impacts people living with ALS are looking for from novel drug development

3:15 pm Chair’s Closing Remarks

3:30 pm End of Conference

DAY ONE
PRE CONFERENCE DAY