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
Integrating Patient‑Centred Outcomes & Model‑Informed Design to Shape the Next Generation of CKD & ADPKD Trials
9:00 am Chair’s Opening Remarks & Audience Welcome
9:20 am Shaping the Future of CKD & ADPKD Trials with Meaningful Patient-Reported Outcomes (PROs)
- Ensuring PROs capture what matters to the ADPKD patient population (symptoms, QoL, treatment burden) especially in early or mild CKD, not just lab or imaging changes
- Integrating PROs early in development (Phase II/POC) to inform Phase III design and labelling claims
- Harmonizing clinician-designed PRO measures with what regulators (e.g., FDA/EMA) expect to ensure trial endpoints are accepted
- Leveraging real-world evidence and external controls to validate PRO metrics, reduce placebo arms, and speed regulatory approval
9:50 am PKD Trial Simulator: Insights from PKD Consortium as Tools for CKD Drug Development
- 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.
10:20 am Morning Break & Networking
Bridging Mechanistic Dosage Biology & Cutting‑Edge PKD Modeling Tools for More Translationally Relevant Discovery
10:50 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
11:30 am Defining the PKD Dosage Threshold: Mechanistic Drivers & Therapeutic Strategies to Elevate PC1 in ADPKD
- Dissecting the two‑hit model and how sub‑threshold PC1 levels initiate cystogenesis
- Characterizing PKD1/PKD2 dosage requirements that modulate disease penetrance and progression
- Reviewing emerging therapeutic approaches designed to increase functional PC1 and restore cellular homeostasis
- Integrating mechanistic insights with translational efforts to accelerate dose‑targeted ADPKD therapies
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: Advancing CKD & Rare Glomerular Disease Approvals Through Surrogate Endpoints, Patient- Centered Strategies & Real world Evidence
As therapeutic innovation expands across both rare glomerular diseases and more common causes of CKD, developers continue to face complex questions around endpoint selection, evidence expectations, and patient relevance. This workshop brings together regulatory, clinical, and industry perspectives to explore how surrogate endpoints, patient‑reported outcomes, and real‑world evidence can be thoughtfully applied to strengthen trial design.
Across two expert presentations and an interactive fireside chat, faculty will discuss learnings from C3G and other rare disease programs, considerations for trial design in more common CKD conditions, and evolving expectations around background therapies and standard of care. Participants will then work through facilitated discussion groups on post‑marketing evidence, treatment guidelines, patient identification, and payer‑relevant business cases to translate these insights into practical development strategies.
This session is designed to help teams advance the science of CKD drug development by aligning earlier with regulators, refining endpoint selection, and incorporating patient‑centered approaches that meaningfully strengthen the evidentiary package.
Key Objectives:
- Evolving use and interpretation of surrogate and composite in rare and common kidney diseases
- Approaches to trial design in therapeutic landscapes with multiple background therapies and established guidelines
- Integrating patient‑reported outcomes, disease‑specific function measures, and real‑world evidence to enhance trial relevance and reduce patient burden
- Lessons from recent development programs in C3G and other rare kidney conditions, focused on regulatory science, collaboration, and endpoint development
- Practical considerations for post‑marketing evidence, payer expectations, treatment guideline positioning, and improving diagnosis and access for diverse CKD populations
12:00 pm Lunch & Networking
Unlocking Innovative PKD Therapies Through Harmonised Data, Robust Standards & Breakthrough Delivery Approaches
1:00 pm Roundtable Discussion: Data, Standards & Infrastructure to Accelerate PKD Research: What Does the Field Actually Need Next?
- Barriers to data sharing, standardization, and interoperability across PKD studies
- What “good” looks like for harmonised imaging, biomarker, and clinical datasets
- Opportunities for pre‑competitive consortia to build shared resources (models, registries, repositories)
- What infrastructure gaps most slow down target validation, trial design, and regulatory engagement
1:50 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
2:20 pm Afternoon Break & Networking
Understanding Standard of Care in PKD: Navigating Endpoints & Regulatory Pathways
2:55 pm Balancing eGFR Decline & TKV Growth for Regulatory Endpoints in PKD
- Explore the manner of disease progression of ADPKD Evaluating whether TKV growth meets regulatory standards for “reasonably likely surrogate” endpoints, especially underaccelerated 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
3:25 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
3:55 pm Chair’s Closing Remarks
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