Pre-Conference Focus Day
Pre-Conference Glomerular Focus Day
Monday March 17, 2025
Advancing Therapeutics Options for Glomerular Disease
This year’s Glomerular Disease Focus Day aims to bring the spotlight onto the evolving landscape of glomerular disorders, which share complex pathophysiology and present significant challenges in both diagnosis and treatment. Building on the progress made in treating IgA nephropathy, this day will explore how recent advancements can be applied to other glomerular diseases, including focal segmental glomerulosclerosis, membranous nephropathy, and lupus nephritis. With breakthroughs in disease understanding and therapeutic development, there’s an opportunity to cross-leverage insights, accelerate drug discovery, and ultimately improve outcomes for all glomerular disease patients.
From IgA Nephropathy to Broader Glomerular Diseases: Learning from Success to Shape the Future
8:30 am Panel Discussion: IgA Nephropathy: Current Achievements, Unmet Needs, & Research Priorities
Synopsis
- Explore a comprehensive overview of breakthroughs in IgAN therapies that improve renal outcomes. How far have we come in slowing disease progression and reducing proteinuria?
- Despite advancements, where do gaps still persist? Distil the limitations of existing therapies, variability in patient responses, and the challenges of predicting long-term outcomes. What hurdles remain for patients and clinicians?
- What complexities do standard-of-care treatments (e.g., corticosteroids, calcineurin inhibitors) introduce to clinical trials of innovative therapies for glomerular diseases? Discuss ethical and regulatory considerations in designing trials that balance patient welfare and the need for novel treatment exploration.
- Where should the research community focus next? From addressing personalized medicine approaches to exploring new biomarkers, what areas hold the greatest potential for advancing care and transforming the treatment landscape for IgAN and other glomerular diseases?
9:00 am Leveraging the Heterogeneity of IgA Nephropathy as a Roadmap for Advancing Other Glomerular Diseases
Synopsis
- Explore the wide variation in IgA nephropathy’s pathogenesis, disease progression, and patient outcomes
- Investigating how the diverse presentations and disease progression in IgA Nephropathy offer key lessons in understanding and managing other glomerular diseases
- Explore how learnings from the heterogeneity of global IgA trials variability in patient populations, disease stages, and responses, can inform trial designs for global trials in similarly complex glomerular diseases.
9:30 am From IgAN to FSGS: Translating Recent Successes & Lessons Learned to Beyond IgAN
Synopsis
- Discuss how the FDA approval of sparsentan (Filspari) for IgA Nephropathy serves as a model for accelerating treatment development in other glomerular diseases, particularly FSGS
- Explore the ongoing FSGS trials and how lessons from the IgAN journey are being integrated to optimize clinical trial design and endpoint selection for rare glomerular diseases
- Highlight the role of regulatory pathways and approval frameworks in facilitating faster access to treatments in both IgAN and FSGS, including navigating accelerated approval requirements
10:00 am Morning Break
Examining the Intricacies of Glomerular Disease Drug Development to Uncover Innovative Approaches to Treatment
10:30 am Anti-Nephrin Autoantibodies: A Breakthrough in Kidney Disease Research & Future Impacts on Drug Discovery & Development
Synopsis
- Discuss the discovery of anti-nephrin autoantibodies as a major contributor to certain rare forms of nephrotic syndrome, offering new insights into disease etiology and progression
- Explore how this finding could revolutionize diagnostic frameworks and therapeutic strategies, improving development of targeted treatments for previously misunderstood or misdiagnosed conditions
- Evaluate the implications of anti-nephrin autoantibodies on future drug discovery, including development of specific immunomodulatory therapies that address autoimmune mechanisms in kidney disease
11:00 am An Introduction into the PARASOL Project
11:30 am Revolutionizing FSGS: Addressing Proteinuria & GFR as Clinical Endpoints in FSGS Through the PARASOL Initiative
Synopsis
- Explore the current challenges in identifying reliable, meaningful endpoints in FSGS clinical trials, and how the PARASOL initiative seeks to establish the relationship between short-term biomarker changes (proteinuria and GFR) and long-term outcomes
- Exploring how global patient registries and real-world data from diverse cohorts are informing endpoint identification, particularly in rare diseases like FSGS where patient numbers are limited
- Examine the implications of newly identified endpoints on clinical trial design, regulatory approval, and future therapeutic development for FSGS, with lessons for broader glomerular diseases.
12:00 pm Leveraging Patient Organizations & Advocacy Groups to Power Clinical Trials in Rare Glomerular Diseases
Synopsis
- Explore how collaboration with patient advocacy group can significantly enhance recruitment efforts for clinical trials through networks and platforms to reach patients directly
- Explore the unique insights these organizations provide regarding patient needs, concerns, and experiences, ensuring that trial designs incorporate patient perspectives and preferences to develop more patient-centered trials that address real-world concerns and improve retention rates
12:30 pm Lunch Break
Innovating Beyond the Generics: Innovative Approaches to Treating Glomerular Diseases & New Pathways for Intervention
1:30 pm Exploring the Recent Developments in Gene Therapy for Glomerular Diseases
Synopsis
- Examine recent advancements in gene therapy for glomerular diseases, focusing on novel vector technologies and targeted delivery systems designed to restore kidney function by addressing the underlying genetic drivers of disease
- Explore PureSpring Therapeutics’ innovative approach using adeno-associated virus (AAV) vectors to enable precision targeting of podocyte-specific pathways, highlighting its potential to revolutionize treatment for glomerular disorders
- Review preclinical and translational data supporting the feasibility and safety of gene therapies in glomerular diseases, emphasizing their promise in transforming care paradigms for high-burden, unmet medical needs
2:00 pm Leveraging the Power of the Complement System to Target Glomerular Disease Pathophysiology
Synopsis
- Understand how dysregulation of the complement system contributes to the pathogenesis of C3 Glomerulopathy (C3G) and Immune Complex Membranoproliferative Glomerulonephritis (IC-MPGN), highlighting the role of C3 inhibition in addressing these complement-driven kidney diseases.
- Explore the latest developments in targeting complement activity, focusing on emerging therapies that aim to reduce immune-mediated kidney damage and improve outcomes for patients with C3G and IC-MPGN
- Review clinical evidence and ongoing trials that position complement inhibitors as a cornerstone for managing C3G and IC-MPGN, emphasizing personalized approaches to address high unmet medical needs.
Future Directions for Glomerular Diseases
2:30 pm Panel Discussion & Audience Roundtable: Advancing Therapeutics in Glomerular Diseases: What’s Next?
Synopsis
- Discuss the need to identify reliable surrogate markers for other glomerular diseases and potential avenues for development and standardization
- Discuss what can be learned from the established biomarkers in IgA nephropathy to improve patient outcomes in other glomerular diseases
- Examine the variability in pathology and clinical presentation among glomerular disease patients, and what strategies can be employed to ensure that new treatments can effectively address this heterogeneity
- Discuss the ethical and regulatory hurdles in designing trials for innovative therapies, explore potential trial designs that balance patient safety with the need to test new treatments, and how regulatory frameworks can support innovation