Explore the Agenda
8:00 am Check In, Coffee & Light Breakfast
8:55 am Chair’s Opening Remarks
Translating Target & Biomarker Discovery into Validated, Scalable Precision Tools for Global Neuromuscular Trials
9:00 am Reinventing Target Discovery in Autoimmune Neuromuscular Disease: Overcoming Model Limitations, Data Gaps & Translational Risk
- Improve disease relevance of discovery models by addressing limitations of primary, iPSC‑derived, and immortalised cell systems to strengthen early target validation
- Leverage integrated multi‑omics and AI‑driven analytics to enable robust pathway analysis and informed hit prioritization prior to wet‑lab screening
- Align early assay readouts with clinical biomarkers to improve translational relevance and reduce late‑stage failure in neuromuscular programs
9:30 am Biomarkers in Heterogeneous Autoimmune Neuropathies: Challenges, Trade Offs, & Opportunities to Advance Disease Understanding
- Disease heterogeneity as the central challenge for biomarker development
- Interpreting biomarker signals in heterogeneous biology
- Using biomarkers to support disease understanding – exploratory biomarkers as learning tools
10:00 am Morning Break & Refreshments
Designing Feasible & Mechanism-Aligned Clinical Trials in Competitive Rare Neuromuscular Landscapes
11:00 am Session Reserved
11:30 am Roundtable Discussion: Navigating Patient Recruitment & Competitive Saturation in Rare Autoimmune Diseases
- Managing patient recruitment in oversaturated MG landscapes
- Discuss washout challenges and competitive trial overlap to proactively mitigate enrolment risk and ethical challenges
- Identifying strategies to differentiate in competitive FcRn and complement markets to protect trial feasibility in rare disease populations
12:30 pm Lunch & Networking Break
Shifting from Antibody Suppression to Immune Reset: Upstream B-Cell Targeting & Genetic-Environment Interactions
1:30 pm Upstream Targeting of B-Cell Dysregulation via BAFF/APRIL Inhibition in Generalized Myasthenia Gravis and Related Autoimmune Conditions
- Discuss upstream modulation of B‑cell survival and differentiation through BAFF/APRIL inhibition as a therapeutic strategy in gMG
- Position BAFF/APRIL inhibition within the evolving gMG treatment landscape, in comparison with downstream approaches such FcRn and complement pathway blockade, to clarify how upstream B‑cell targeting may offer differentiated clinical benefits by addressing fundamental disease‑sustaining mechanisms
- Evaluate the efficacy and durability of BAFF/APRIL inhibition in gMG versusestablished mechanisms, assessing effects on clinical outcomes, B-cell homeostasis, immunoglobulin dynamics, and infection risk
2:00 pm Roundtable Discussion: Redefining Clinical Success Metrics to Ensure Meaningful Functional Recovery in Peripheral Neuropathies Disease
- Evaluating score-based severity reduction, such as reducing a scale from 1 to 0.5, and questioning whether statistical improvement translates into tangible muscle function recovery like improved grip strength, brushing teeth, or walking stability
- Comparing existing neurological assessment tools (e.g., grip strength, MRC scores and other scales), analysing their variability and lack of a clear gold standard to determine which tools best capture meaningful neuromuscular improvement in Phase III trials
- Defining what “good enough” improvement means, acknowledging that partial improvement may not reflect meaningful daily function restoration to align trial endpoints with patient-relevant outcomes rather than abstract metrics
Defining Clinical & Commercial Value in Peripheral Neuropathies Drug Development: From Meaningful Functional Recovery to Market Differentiation
3:00 pm Panel Discussion: Big Pharma Perspective on Emerging Commercial Opportunity to Investigate Existing Autoimmune Therapeutics within the Myasthenia Gravis & Peripheral Neuropathies Space
- Evaluating MG as a high-growth yet increasingly oversaturated market, recognising that multiple FcRn and C5 inhibitors show comparable aggregate efficacy but compete for a finite rare-disease population
- Identifying CIDP and MMN as commercially compelling but biologically underdefined opportunities, due to uncertainty around immunologic origin, incomplete real-world efficacy and unclear prevalence
- Balancing chronic suppression models against emerging immune-reset approaches, comparing repeated FcRn/complement administration with upstream B-cell or CAR-T interventions to inform long-term investment decisions focused on durable remission and defensible market positioning