7:00 am Morning Refreshments & Registration

7:55 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

  • Tammy Sarnelli Vice President, Global Head of Regulatory Affairs, Amylyx Pharmaceuticals Corp.

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 Liquid Biopsy with 3D Genomic Biomarkers as A Clinical Tool For Diagnosis, Prognosis & Disease Understanding In ALS

Synopsis

  • 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

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

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

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?

11:15 am Human iPSC 2D & 3D Neuromuscular Junction Modelling for Assessing C9orf72 ALS Hyperexcitability In Vitro

  • Stuart Prime Team Lead, Research & Development Programs, Axol Bioscience

Synopsis

  • 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

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 Functional Phenotypic Disease Models of ALS

Synopsis

  • 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

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

2:00 pm Characterization of TDP-43 Delta NLS Mice: Behavior & Biomarker Analyses

Synopsis

  • 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

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

2:40 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.

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

3:10 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

Synopsis

  • 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

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

4:40 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

5:10 pm Chair’s Closing Remarks

5:15 pm Scientific Poster Session & Drinks Reception

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.

6:15 pm End of Conference Day One