Explore the Agenda
8:00 am Morning Check-In: Served with coffee + light breakfast
Polycystic Kidney Disease (PKD) Focus Day
Designed for CSOs, preclinical researchers, discovery scientists, directors of biology and research, novel modality scientists, in vivo/in vitro modelling experts and many more focusing on the promise of PKD therapeutics; whether you're new to PKD or a veteran of PKD R&D, connect and learn more in an aerial view of PKD research
Unveiling Hot Clinical Programs Trailblazing in PKD
9:00 am PKD Trial Simulator: Insights from PKD Consortium as Tools for CKD Drug Developme
- Leveraging the PKD Outcomes Consortium’s joint‐model linking longitudinal total kidney volume (TKV) growth to eGFR decline or ESRD to enrich trial populations
- Using a Clinical Trial Simulator tool to predict outcomes, design shorter or smaller trials, and optimize inclusion criteria
- Qualifying TKV as a prognostic biomarker with FDA and EMA to enable regulatory efficiency and accelerated pathways
- Integrating patient registry, natural history, and trial data into unified models for trial design and drug response prediction.
10:00 am Morning Break & Networking
Harnessing Novel Modalities & Cutting-Edge Models as Tools for Transformative Drug Research in ADPKD
11:00 am Roundtable Discussion: Models & Assays for PKD: Organoids, Rodents, EV-Based Diagnostics, and AI Tools
- Using human kidney organoids and zebrafish or C. elegans models for early screening of therapeutic targets and cystogenesis assays
- Developing EV-based urine assays to detect polycystin-1/2 levels and prognosticate disease before significant cyst development
- Applying AI-enabled imaging tools and organoid video analysis platforms (e.g. “Organoid Tracker”) to quantify cyst formation rate, growth velocity, morphology
- Standardizing rodent PKD model cores (e.g. varying progression rates) to allow cross-study comparability and better translational predictivity
Regulation, Access & Reimbursement Workshop Day
Tailor-made for medical directors, CMOs, clinical program leads, regulatory affairs professionals, access, reimbursement & HEOR colleagues, BD experts, clinicians and more; share expertise from diverse clinical challenges and innovation in CKD
9:00 am Workshop A: Accelerating CKD & Rare Glomerular Disease Approvals Through Surrogate Endpoints & Patient- Centered Strategies
As novel therapies emerge across the spectrum of CKD and rare glomerular diseases, developers face complex questions about regulatory expectations outside of IgA nephropathy. This interactive workshop will explore how agencies such as the FDA and EMA are evaluating surrogate endpoints, comparator requirements, and patient-centered outcomes in under-studied disease areas. With input from regulators, pharma, and trialists, the session will examine the evolving role of biomarkers, patient-reported outcomes, and real-world evidence in shaping accelerated approval pathways. Participants will gain a clear understanding of how to strategically design trials, minimize placebo burden, and align early with regulators to bring transformative therapies to patients faster.
Key Objectives:
- Evaluating acceptance of surrogate and composite endpoints (e.g., proteinuria reduction, MEG90, imaging biomarkers) in rare kidney diseases
- Understanding regulatory views on trial design in landscapes with multiple therapeutic classes and existing standards of care
- Incorporating patient-reported outcomes and real-world evidence into approval pathways to reduce placebo burden and enhance relevance
- Learning from recent case studies (e.g., Apellis in C3G, Filspari) to navigate accelerated approvals and companion diagnostic considerations
12:00 pm Lunch & Networking
Assessing Pathological Fibrosis & Autophagy Modulation in PKD in Treating Complex PKD Pathology
1:00 pm Kidney-Targeted LNP Delivery as a Next-Generation Therapeutic for ADPKD: Challenges, Design & Translational Path
- Strategies for achieving kidney tropism: tuning size, charge, PEGylation, and reabsorption pathways
- Balancing efficacy, immunogenicity, and safety in nonviral LNP delivery for renal cells
- Integrating EPIC-Cure design with LNP payloads to minimize off-target effects in ADPKD
- Path to translation: scale-up, regulatory considerations, and metrics for success in ADPKD clinical trials
2:00 pm Panel Discussion: Novel Delivery Modalities & Gene Therapy Approaches in PKD
- Exploring gene therapy, antisense oligonucleotides, and CRISPR-based approaches
- Overcoming delivery barriers to the kidney
- Designing preclinical studies with translational relevance
- Regulatory considerations for first-in-human studies
2:30 pm Afternoon Break & Networking
Understanding Standard of Care in PKD: Navigating Endpoints & Regulatory Pathways
3:00 pm Balancing eGFR Decline & TKV Growth for Regulatory Endpoints in PKD
- Evaluating whether TKV growth meets regulatory standards for “reasonably likely surrogate” endpoints, especially under accelerated approval pathways
- Investigating how well eGFR decline tracks patient-centered outcomes and long-term kidney failure risk compared to TKV alone
- Discussing whether combined endpoints (e.g. TKV + eGFR) offer stronger case for full approval versus accelerated or conditional approvals
- Understanding global regulatory differences (FDA, EMA, PMDA etc.) in accepting imaging- and biomarker-based endpoints, and how trials can harmonize design across jurisdictions
3:30 pm Empowering the PKD Community: Advancing Research, Awareness, and Clinical Progress Through Patient Partnership
- Strengthening patient engagement across all stages of drug development by connecting individuals and families with clinical trials, and ensuring the patient voice shapes research priorities and study design.
- Building partnerships with industry, academia, and advocacy organizations to drive innovation, expand funding, and accelerate progress in PKD research and treatment.
- Leveraging the PKD Foundation Registry and Centers of Excellence network to enhance clinician understanding, improve recruitment, and advance data-driven insights into PKD progression
1:00 pm Workshop B: Enhancing Market & Patient Access to Novel CKD Therapies Amongst a Growing Treatment Landscape
Demonstrating value to payors over existing treatments; overcoming systemic barriers to adoption, from shifting nephrology’s focus beyond dialysis to embedding education at centers of excellence, to navigating payer expectations and addressing inequities in patient access.
Key Objectives:
- Identifying barriers to adoption of novel CKD therapies across payer systems and clinical practice
- Leveraging centers of excellence and global nephrology networks (e.g., GlomCon, ISG, ISN) to accelerate HCP education and patient access
- Exploring payor and HTA perspectives on value-based pricing, reimbursement restrictions, and trial comparators
- Addressing disparities in access, mistrust, and underrepresentation of minorities, women, and children in CKD care
- Defining how early detection, tolerability, and patient education can shape real-world uptake of CKD treatments