7:30 am Check In, Coffee & Light Breakfast
WORKSHOP A 8:30-10:30
Exploring a New Window of Opportunity: TDP-43-Related Biomarkers & Their Development into Clinical Use
Synopsis
With a pressing need for more disease-specific markers of ALS pathology, a promising avenue has emerged to explore the downstream effects of cryptic exon inclusion due to TDP-43 loss of function. For example, the generation of a novel peptide, leading to the development of methods to specifically detect it in individuals with ALS. Join this session for a comprehensive breakdown of recent and ongoing research to identify clinically useful TDP-43 related biomarkers for ALS:
- Deepen understanding of TDP-43 function to create new biomarker opportunities
- Investigate the correlation between new TDP-43-based biomarker candidates and the progression of ALS
- Evaluate their clinical feasibility to be detected in patient samples and utilized to demonstrate therapeutic impact on TDP-43 pathology
10:30 am Networking Break
WORKSHOP B 11:00-1:00
Navigating the Hurdles of a Combination Therapy Approach & the Need to Demonstrate Efficacy on Top of Existing ALS Treatments
Synopsis
In a world where numerous ALS drugs begin to exist, new challenges of trial enrolment, patient availability and an evolving standard of care emerge. Engage in forward-thinking discussions around the future of ALS drug development as increasing numbers of therapies reach the clinic:
- How can we overcome anticipated trial complications due to varying standards of care across geographical regions?
- What are considered optimal patient profiles for a combination therapy approach? How to select the right combination?
- What is required to demonstrate the benefit of the combination over a single dose?
- How must the treatment arms versus the placebo control group be handled when administering combination therapies?
1:00 pm Lunch & Networking Break
WORKSHOP C 2:00-4:00
Reinventing the Surrogate Landscape: Innovating Regulatory Acceptable Endpoints Most Meaningful to People Living with ALS
Synopsis
The recent accelerated approval brings new waves of promise for transformative ALS drugs to be approved based on surrogate markers that correlate with ALSFRS-R. With increasing demand to define more acceptable endpoints for ALS, collaborate with industry sponsors and regulatory experts to:
- Evaluate the potential of novel surrogate biomarkers that extend beyond ALSFRS-R to monitor patients, enhancing dialogue with regulatory bodies about what these could be
- From mobility to respiratory function, explore the most meaningful endpoints to people living with ALS to help inform clinical trial design
- Debate the best primary, secondary and exploratory endpoints, and how to balance these based on different regulatory requirements