7:00 am Morning Refreshments & Registration
7:55 am Chair’s Opening Remarks
- Johanne Kaplan Chief Development Officer, ProMIS Neurosciences
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
- Tammy Sarnelli Vice President, Global Head of Regulatory Affairs, Amylyx Pharmaceuticals Corp.
- 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 Liquid Biopsy with 3D Genomic Biomarkers as A Clinical Tool For Diagnosis, Prognosis & Disease Understanding In ALS
- Alexandre Akoulitchev Chief Scientific Officer, Oxford BioDynamics plc
- 3D Genomic EpiSwitch® blood-based biomarkers: development of blood based clinical tests for early diagnosis, prognosis and prediction of response to treatment
- ALS diagnostic and prognostic EpiSwitch® tests: from development to deployment in MTPA Radicava trial
- EpiVerseTM profiling of ALS regulatory 3D genomic footprint: from ALS subtypes to neuro-inflammation and strong predictors as therapeutic targets
9:00 am Panel Discussion: A Year in Review: Showcasing the Breakthrough Developments in ALS Drug Development
- Alain Moussy Co-Founder & Chief Executive Officer, AB Science
- Machelle Manuel Vice President, Head of Global Medical Affairs, Amylyx Pharmaceuticals Corp.
- Antonio Trejo Diaz Vice President of Regulatory Affairs, BrainStorm Cell Therapeutics
- 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
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
10:15 am Morning Break & Networking
DEBATING IDENTIFICATION & PRECLINICAL RECAPITULATION OF GENETIC ALS TARGETS TO OPTIMIZE DEMONSTRATIONS OF PRECLINICAL SAFETY & EFFICACY
10:30 am Panel Discussion: Evaluating Current Strategies for ALS Target Identification: What are the Avenues to Still Prioritise?
- Enchi Liu VP Translational Sciences, Tranquis Therapeutics Inc.
- Sophie Parmentier Batteur Vice President, Head of Neuromuscular & Muscle Research Unit, Biogen
- Dan Elbaum Chief Scientific Officer, QurAlis Corp.
- Joseph Geraci Chief Technology Officer & Chief Scientific Officer, NetraMark Corp
- Sami Barmada Associate Professor & Director, University of Michigan Brain Bank
- 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?
11:15 am Human iPSC 2D & 3D Neuromuscular Junction Modelling for Assessing C9orf72 ALS Hyperexcitability In Vitro
- Stuart Prime Team Lead, Research & Development Programs, Axol Bioscience
- Identification of ALS phenotype from human iPSC differentiated motor neurons
- Creating 2D and 3D model systems for the establishment and identification of the neuromuscular junction
- Utilizing 3D Neuromuscular junction models to investigate ALS hyperexcitability using multi-electrode array endpoint
11:45 am Leveraging Accurate Human Disease Models for Target Validation & Drug Optimization to Rapidly Advance Novel & Effective Treatments for ALS
- Sam Alworth Chief Executive Officer, AcuraStem Inc.
- 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 Functional Phenotypic Disease Models of ALS
- Olaf Schröder Chief Executive Officer, NeuroProof GmbH
- Human IPSC-derived motor neurons and astrocytes from familial ALS mutations
- Functional electrophysiological and imaging disease models
- Results and model validation
12:25 pm Lunch & Networking
1:30 pm Assessing Neuronal Degeneration Mechanisms in In Vivo & In Vitro C9orf72 Models
- Maya Maor-Nof Postdoctoral Fellow, Genetics, Stanford University
- 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
2:00 pm Characterization of TDP-43 Delta NLS Mice: Behavior & Biomarker Analyses
- Taleen Hanania Executive Vice President, PsychoGenics Inc
- Exploring TDP-43ΔNLS mouse model of amyotrophic lateral sclerosis (ALS) and frontotemporal lobar degeneration (FTLD-TDP)
- Demonstrating a motor phenotype and impaired muscle function in TDP- 43ΔNLS animals using EMG assessment
- Discussing neurofilament light chain and inflammatory markers with disease progression and TDP-43 pathology
2:10 pm – Preclinical Validation of RACK1 as a Novel Target for Sporadic ALS
- Beibei Zhao Director, Research Programs, ProMIS Neurosciences
- 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
2:40 pm Blending In Vitro & In Vivo Models to Overcome Species Sequence Variation for Preclinical Testing of Human Genetic Targets
- Sandy Hinckley Senior Director, Head of Biology & Discovery, QurAlis Corp.
- 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
3:10 pm Afternoon Break & Networking
3:40 pm Learning the Perspective from People Living with ALS to Understand Priorities to Apply to Novel ALS Drug Development
- Robert Goldstein Associate Director, Global Patient Advocacy, Neuromuscular, Biogen
- Gathering insights from people with lived experience to apply in trial design and endpoint selection
- ALS community-specific considerations for communicating early access program
PARTICIPANT REALITY CHECK: ARE PEOPLE LIVING WITH ALS BOUGHT INTO CLINICAL RESEARCH?
4:10 pm Highlighting the Benefits of Decentralized Clinical Trials & Remote Monitoring Technology for People Living with ALS
- Amir Lahav Head of Digital Research & Development, Digital Health Innovation, Mitsubishi Tanabe Pharma
- 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
4:40 pm Panel Discussion: Building on Experience Interacting with People Living with ALS to Outline Areas to Improve Future ALS Drug Development
- Richard Bedlack Physician, Duke University
- Paul Wicks Advocate, Independent
- Shaunak Dwivedi Physician, Independent
• 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
5:10 pm Chair’s Closing Remarks
- Johanne Kaplan Chief Development Officer, ProMIS Neurosciences
5:15 pm Scientific Poster Session & Drinks Reception
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.