Day One

• Differentiating the immunologic mechanisms underlying AChR-positive versus MuSK-positive MG
• Understand subtype-specific pathophysiology to better align complement, FcRn and B-cell approaches with appropriate patient subtypes
• Connecting disease biology to therapeutic mechanism selection and responder prediction to strengthen subtype-driven clinical trial design and reduce misaligned mechanism deployment

• Comparing how FcRn inhibition, complement blockade, B-cell depletion and CAR-T differ mechanistically and where each may sit best based on disease biology
• Examining how mechanistic sequencing could address patients exposed to prior lines of therapy without clear biomarkerguidance
• Identifying emerging strategies for treating MG patients who fail FcRn inhibition, IgG degraders or complement therapies
• Gain insight into how to position assets in a saturated market while addressing inadequate responders and future curative ambition

A prime chance to make the most of in-person networking and forge new connections as new companies enter and existing ones broaden their presence within the autoimmune neuromuscular and nerve disorders space. This is designed to maximize your exposure to a wide range of new individuals and serve as a catalyst for ongoing discussions throughout the summit.

• Interrogating autoantibodies as central but incompletely defined disease drivers, differentiating therapeutic strategies that lower circulating IgG via FcRn blockade, modulate or deplete upstream B-cells, or remove antibodies through plasma exchange to clarify which modality best addresses underlying immune biology rather than simply reducing antibody burden
• Re-examining “seronegativity” as a potential misclassification challenge and whether so-called seronegative MG and CIDP reflect true absence of pathogenic antibodies or limitations in assay sensitivity and testing paradigms to refine patient stratification and avoid biologically inappropriate therapeutic deployment
• Expanding diagnostic and therapeutic focus toward low-frequency or emerging autoantibody targets to increase diagnostic coverage beyond the current majority subset and enabling precision-guided development across heterogeneous patient populations

• Investigate CIDP as a clinical syndrome diagnosed by appearance, not cause
• Examine how biological heterogeneity creates problems – variable placebo response, diluted effects, uninterpretable results – because functional endpoints don’t capture immunologic activity
• Propose the path forward in CIDP: immunophenotyping and biomarker-stratified trials to target defined mechanisms precisely

• Evaluating the limited spinal and brain penetration of biologics and questioning whether partial efficacy in CIDP reflects anatomical inaccessibility rather than target irrelevance
• Comparing small molecule approaches, acknowledging that small molecules are uniquely capable of accessing central nervous system or spinal tissues to assess whether modality selection could unlock improved disease modification in CIDP
• Aligning anatomical access considerations with translational modelling, ensuring that preclinical systems reflect the compartments drugs must reach to improve the predictive value of translational research in peripheral neuropathies

This informal session provides the perfect opportunity to build meaningful connections with pharma, biotech, and academic KOLs, exchange insights on translational strategies and trial design, and gain exclusive, first-hand perspectives on the latest breakthroughs in this niche subset of rare and autoimmune-driven diseases.

• Potential therapeutic strategies for treating IgG-mediated diseases
• Biology of FcRn
• Approved therapeutic approaches for FcRn targeting

• How can refining diagnosis, prevalence understanding and immune drivers (e.g. complement and autoantibodies) in MMN uncover a clearer therapeutic opportunity, enabling companies to confidently expand beyond MG into an underrecognised but potentially high-value market?
• What defines the true biological window of intervention in GBS, and how can aligning immune modulation strategies to disease phase (rather than late-stage symptoms) improve clinical outcomes and prevent irreversible damage?
• How should evolving insights into MMN and GBS pathophysiology reshape trial design, from patient identification to endpoint selection, to ensure therapies demonstrate meaningful efficacy within the right population and timing?