8:25 am Chair’s Opening Remarks
Validating Biomarkers of Target Engagement & Disease Progression to Transform Early & Sensitive Indicators of Therapeutic Efficacy
8:30 am Interrogating Neurofilament as a Marker of Disease Activity & Treatment Response
Synopsis
- Assessing data from tofersen studies to explore the potential utility of neurofilament to enrich or stratify study populations
- Evaluating the relationship between treatment-induced reductions in neurofilament and clinical outcome measures
- Exploring neurofilament as an early marker of disease activity in presymptomatic mutation carriers
9:00 am Implementing Remote Monitoring Technology for Tracking ALS Outside the Clinic Using Wearable Sensors & Smartphone Apps
Synopsis
- Leveraging technology for early diagnosis and remote monitoring of ALS patients
- Aggregating digital data coming from activity monitors, speech recording apps, and smartphone-powered platforms for motor function tests at home.
- Roadmap for integrating digital technology into ALS trials: from feasibility and usabillity testings to clinical validation of new digital endpoints
9:30 am Advancing Novel CSF Biomarkers to Evaluate ALS Target Engagement & Improve Therapeutic Outcomes
Synopsis
- CSF biomarkers interrogate multiple neuroinflammatory, neurodegenerative and neuroprotection pathways to confirm target engagement
- Employing biomarkers at baseline and on-treatment in combination with clinical variables to predict clinical responders
- Supporting biobanks and continuing dialogue with regulators to advance adoption of novel biomarkers
10:00 am In-Person Structured Networking
Synopsis
- This session is the ideal opportunity to take advantage of face-to-face networking time, and understand who is also prioritising and overcoming challenges within ALS drug development.
10:30 am Morning Break & Networking
11:00 am Exploring Electrophysiological Biomarkers of Diagnosis & Outcome in ALS
Synopsis
- Examining applications of electrophysiology as a biomarker of disease progression
- Using electrophysiology to track clinical disease progression and efficacy
- Enhancing repeatability in patients and investigating potential as a primary outcome measure
11:30 am Validating Biomarkers & Use in Clinical Trials to Demonstrate Target Engagement & Eligibility Criteria for Enrollment
Synopsis
- Investigating neurodegenerative and inflammatory biomarkers to evaluate treatment effects in ALS
- Correlating research markers with clinical outcome and interpreting conflicting results
- Utilizing a biomarker panel as enrollment criteria for a clinical trial
Delineating Accurate Primary Outcome Measures to Correlate with Clinical Outcome
12:00 pm Innovation in ALS Trial Design & Clinical Outcome Measures
Synopsis
- Inclusion criteria can be used to ensure a homogenious trial population with regard to disease progression rate and other attributes
- Well-designed open label follow on periods can support findings from placebocontrolled treatment
- New outcome measures can enhance trial sensitivity and reduce participant burden
12:30 pm Lunch & Networking
1:30 pm Moving Beyond ALSFRS-R & Survival Endpoints: Exploring Patient Reported & Non-Traditional Outcome Measures
Synopsis
- Evaluating patient reported outcome measures remotely
- Measuring movement, accuracy of typing, and speech: developing and steps to validating alternative readouts
- Correlating biomarkers and clinical outcomes to accurately reflect disease progression and ultimately demonstrate treatment response in trials
- Integrating novel digital outcome measures into clinical trials
2:00 pm Panel Discussion: Refining Sensitive Primary Outcome Measures & Navigating Innovative Trial Design in ALS
Synopsis
- Pursuing sensitive markers of disease activity and delineating accurate primary outcome measures beyond ALSFRS-R
- Driving early diagnosis, patient diversity, and decentralized trial design to establish effective patient recruitment
- Can we capture statistically meaningful changes in an acute timeframe? Balancing length of trial to demonstrate safety and clinical benefit
Trial Design, Recruitment & Patient-Centric Approaches: Innovating Clinical Trials to Advance Effective Treatments
2:30 pm Driving Experimental Trial Design in ALS to Evaluate Multiple Interventions
Synopsis
- Breaking ground on platform trial design: accelerating towards effective ALS treatments and enabling greater access for patients
- Establishing a common protocol across multiple drug candidates
- Managing patient recruitment across different sites and creatively navigating patients on placebo
3:00 pm Afternoon Break & Networking
3:30 pm Rethinking Inclusion & Exclusion Criteria: Should we be Dissecting Patient Heterogeneity?
Synopsis
- Interrogating Phase II clinical trial data to inform Phase III inclusion and exclusion criteria
- How predictive algorithms may be used as part of inclusion and exclusion criteria
- What types of therapy could be relevant to a wider patient population?
4:00 pm Considerations for Novel ALS Clinical Trials
Synopsis
- Incorporation of clinical trial innovations into ALS studies
- The role of consortia in ALS trial design and execution
- The inclusion of the voice of the patient in ALS clinical trials
4:30 pm TRICALS & the ENCALS Prediction Model: Innovating Clinical Trials in ALS
Synopsis
- TRICALS: the highway towards a cure
- Including more patients in trials by using the ENCALS prediction model
- Trial duration for ALS clinical trials
5:00 pm Chair’s Closing Remarks
5:10 pm Scientific Poster Session
Synopsis
After the formal presentations have finished, the learning and networking carries on. The Poster Session is allows you to connect with your peers in a relaxed atmosphere and continue to forge new and existing relationships. During this session scientific posters will be presented on the very latest advancements in ALS therapeutics with a focus on better understanding and overcoming translational challenges to inform the direction of future drug development efforts.