8:15 am Chair’s Opening Remarks

SHOWCASING THE CUTTING-EDGE OF ALS BIOMARKERS TO DRIVE EARLY PARTICIPANT RECRUITMENT & MEASURE DISEASE PROGRESSION

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

  • Li Chin Wong Executive Director, Translational Biomarkers and Bioanalytics, Prevail

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

  • Fernando Vieira CEO and Chief Science 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

9:30 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

10:00 am Morning Break & Networking

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

11:00 am Session Reserved for Axol Biosciences

11:30 am Exploring a Splice Modulation Antisense Oligonucleotide to Restore STATHMIN-2 Levels in ALS Patients

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

12:00 pm – 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

12:30 pm Lunch & Networking

2:00 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

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

2:30 pm Reviewing ALSFRS-R as the Established Endpoint for ALS Clinical Trials

  • Stacy Lindborg Chief Development 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

3:00 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

3:45 pm Chair’s Closing Remarks

4:00 pm End of Conference