7:45 am Chair’s Opening Remarks
CAPITALIZING THE GROUND-BREAKING MOMENTUM: PAVING THE ROAD FOR A NEW PARADIGM OF APPROVED ALS THERAPEUTICS
8:00 am Lessons Learnt from the AMX0035 Approval to Inform Strategies for Future Candidate Approvals
Synopsis
• Outlining Amylyx’s clinical strategy to achieve candidate market approval
• Lessons learnt from approval – what were the key takeaways from interactions with regulatory bodies?
8:30 am 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?
9:00 am Panel Discussion: A Year in Review: Showcasing the Breakthrough Developments in ALS Drug Development
Synopsis
• Reviewing the ground-breaking developments in ALS drug development in the past year
• Understanding the impact on people living with ALS, clinicians, drug developers and regulatory bodies
• Looking to the future: what are the steps towards the next generation of ALS therapeutics?
9:30 am Structured Networking
Synopsis
With more companies emerging into the ALS space, this session is a great opportunity to introduce yourself to ALS
experts across leading drug developer biopharma. This session is the ideal opportunity to get face-to-face time with
many of the brightest minds capturing the main challenges and changes in the ALS field to establish meaningful business
relationships
10:15 am Morning Break & Networking
DEBATING IDENTIFICATION & PRECLINICAL RECAPITULATION OF GENETIC ALS TARGETS TO OPTIMIZE DEMONSTRATIONS OF PRECLINICAL SAFETY & EFFICACY
11:15 am Panel Discussion: Evaluating Current Strategies for ALS Target Identification: What are the Avenues to Still Prioritise?
Synopsis
• Outlining current avenues of disease pathology to identify and validate targets for familial and sporadic ALS
• Processing disease pathology information to validate genetic ALS targets
• Where are the current innovations for novel target identification?
11:45 am Leveraging Accurate Human Disease Models for Target Validation & Drug Optimization to Rapidly Advance Novel & Effective Treatments for ALS
Synopsis
• Discovering novel and broadly effective therapeutic targets, such as PIKFYVE and SYF, through unbiased screening in sporadic ALS patient models
• Optimizing ASO drug candidates’ potency to reverse aberrant UNC13A splicing in ALS patient models
• Investigating safety, potency and biodistribution of lead ASO candidate AS-202, a near-to-clinic PIKFYVE suppressing ASO
12:15 pm Assessing Neuronal Degeneration Mechanisms in In Vivo & In Vitro C9orf72 Models
Synopsis
• Application of genome-wide chromatin accessibility profiling and transcriptional signatures in neurons
• Utilizing genome-wide screens to define mechanisms of neurodegenerative disease
• Defining the diverse cellular and molecular mechanisms driving neuronal and axonal degeneration
12:45 pm Lunch & Networking
2:15 pm – Preclinical Validation of RACK1 as a Novel Target for Sporadic ALS
Synopsis
- Explaining RACK1 co-aggregation with TDP-43 in postmortem spinal cord tissues of sporadic and C9orf72 ALS patients and cultured cells
- Outlining preclinical results from RACK1 knock-down in in vitro cultured cells
- Showcasing results from RACK1 knock-down preclinical testing in in vivo transgenic D. melanogaster ALS models
2:45 pm Blending In Vitro & In Vivo Models to Overcome Species Sequence Variation for Preclinical Testing of Human Genetic Targets
Synopsis
• Differentiating motor neurons from human stem cells derived from ALS patients
• Creating a model system: implanting differentiated motor neurones into mouse brains
• Utilizing model system to investigate preclinical engagement of candidates against validated human genetic targets
3:15 pm Preclinical Roundtable Discussion:
Synopsis
Join conversations with your fellow peers to discuss the benefits and challenges of ALS disease models. Join tables led by preclinical experts to discuss sourcing, reproducibility, novel innovations, and translational strategy
3:45 pm Afternoon Break & Networking
PARTICIPANT REALITY CHECK: ARE PEOPLE LIVING WITH ALS BOUGHT INTO CLINICAL RESEARCH?
4:15 pm Learning the Perspective from People Living with ALS to Understand Priorities to Apply to Novel ALS Drug Development
Synopsis
• Gathering insights from people with lived experience to apply in trial design and endpoint selection
• ALS community-specific considerations for communicating early access programs
4:45 pm Highlighting the Benefits of Decentralized Clinical Trials & Remote Monitoring Technology for People Living with ALS
Synopsis
• Decentralized clinical trials: lowering barriers to broaden enrolment of diverse participant populations
• Integrating patient-friendly technology for conducting clinical assessments for tracking ALS progression at home
• Highlighting strategies to improve participant engagement in ALS clinical trials involving remote monitoring technology
5:15 pm Panel Discussion: Building on Experience Interacting with People Living with ALS to Outline Areas to Improve Future ALS Drug Development
Synopsis
• Outlining insights from interacting with people with ALS to identify the most important priorities to incorporate into ALS drug development
• What can be done to improve participant experience in ALS clinical research?
• Identifying the immediate impacts people living with ALS are looking for from novel drug development
5:45 pm Chair’s Closing Remarks
6:00 pm Scientific Poster Session:
Synopsis
This is an informal session to help you connect with your peers in a relaxed atmosphere and continue forging new relationships. With an audience of ALS enthusiasts eager to hear the latest cutting-edge advancements, you will have the opportunity to display a poster presenting your own work and review other’s posters displaying preclinical and clinical development of ALS candidates, novel target discovery, biomarker innovations and more.