Conference Day One
7:00am Check-In & Coffee + Light Breakfast
7:50 am Chair’s Opening Remarks
Where is the Landscape Heading in Terms of Acceptable Surrogate Endpoints in CKD?
8:00 am FDA Perspective on the Evolution of Acceptable Endpoints for Both Broad & Specialized CKD
Synopsis
- Looking beyond IgA Nephropathy for considerations of other accelerated approval paths that might be open to investigation using surrogate endpoints
- Navigating the differential relationship between proteinuria and eGFR across different indications, which poses a challenge when establishing proteinuria as a surrogate in other diseases
- Determining prerequisites for utilizing GFR decline for CKD studies in general as an endpoint
8:30 am Clarifying the Genomic Landscape of Cystic Disease
Synopsis
- Leveraged 61K+ clinico-genomic dataset of chronic kidney disease pateints for novel insights into and molecular stratification of cystic disease
- Apply variant classification framework to identify a hidden population of genetic cystic disease patients
9:00 am Panel Discussion | Translating Learnings from IgA Nephropathy to Strive for Acceptable Surrogate Endpoints in Broader CKD
Synopsis
- Debating whether hard outcome studies are needed to demonstrate meaningful changes in renal function
- Extending the emerging learnings from IgA Nephropathy to other orphan kidney diseases
- Debating the validity of surrogate endpoints for demonstrating renal function
9:30am Speed Networking
Synopsis
A prime chance to make the most of in-person networking within the continuously growing CKD community. Designed to maximize your introduction to numerous new individuals and serve as a catalyst for ongoing discussions during the summit.
10:00am Morning Break & Refreshments
Navigating Varied Heterogeneity Across CKD Populations to Innovate More Precise & Subgroup-Specific Treatments
11:00 am Discovering Distinct Genetic Signatures within Diverse CKD Populations for the Development of Precise Therapeutic Targets Tailored to Subtypes
Synopsis
- Comprehending the genetic and phenotypic variability among various kidney diseases
- Uncovering underlying disease pathologies preceding the common pathways shared by all CKD subtypes
- Reconceptualizing distinct disease states through insights into genetic drivers specific to CKD subtypes
11:30 am Navigating Heterogeneity & Underlying Pathophysiology in Primary FSGS to Innovate Specific Targets of Immune Dysregulation & Podocyte Injury
Synopsis
- Deepening understanding of specific immune factors driving kidney scarring in FSGS
- Determining particular immune pathways triggered in FSGS to enable a targeted approach rather than broad suppression of the entire immune system
- Identifying distinct histological morphologies to be detected in a biopsy to allow specific and accelerated diagnosis of primary FSGS
12:00 pm Fibrosis: Is It a Realistic Therapeutic Target for CKD?
Synopsis
- Navigating translational challenges for targeting fibrosis in CKD
- What are the challenges for moving fibrosis from bench to clinic?
- Is there a way forward?
Biomarker Innovation to Facilitate Early Phase CKD Trials
11:00 am Leveraging Markers of Renal Function for Early Phase Decision Making in CKD Trials
Synopsis
- Utilizing clinical and biomarker data for dose-finding in early phase kidney diseases trials
- Developing and incorporating biomarkers relevant to renal pathophysiology
- Integrative data approaches to enable go/no-go decisionmaking
11:30 am When Imaging Adds Value in Early-Stage Kidney Disease Drug Development
Synopsis
- How imaging can minimize risk to your drug development program by providing early insights about efficacy or mode of action (MoA)
- Imaging can also help to predict progression and/or outcomes and thereby be used to enrich your trial population
- Treatment effects across organs that are metabolically linked to the kidney (i.e. liver, heart, etc.) can be investigated simultaneously using tailored imaging protocols
12:00 pm Leveraging Data Analytics to Uncover Prognostic Biomarkers for Patient Stratification in PKD
Synopsis
- Leveraging RWD (real-world data) and MIDD (model-informed drug development) approaches to build a clinical trial simulator tool for ADPKD
- Modelling kidney disease progression via longitudinal TKV and eGFR coupled with time-to-event kidney failure covariates can lead to insights into patient stratification, clinical trial size and duration, and drug response outcomes
- The CTS tool can potentially be applied to other CKD settings outside of ADPKD
Progress on Late Phase Endpoints & Regulatory Acceptance
11:00 am Statistical Considerations for the Development of a Novel Hierarchical Composite Endpoint for Broad CKD Trials
Synopsis
- Identifying a novel HCE to provide a reasonable clinical sample size
- Outlining design considerations to establish this HCE as a primary endpoint to be used broadly across CKD trials
- Analysis and reporting considerations for the novel HCE (win statistics and maraca plots)
11:30 am Bridging Key Learnings of Surrogate Endpoint Development Across the CKD & Transplantation Communities
Synopsis
- Developing a reasonably likely surrogate endpoint to allow for innovation in the kidney transplantation field
- Evaluating current endpoints used to measure success and outcomes of kidney transplantation
- Extracting applicable takeaways from advancements in phase 3 studies within the transplant field
12:00 pm Navigating Trade-Offs Between Surrogates & Clinical Endpoints in Relation to Generalizability
Synopsis
- Highlighting how treatment effects on a clinical endpoint may reflect a relatively small subgroup of the trial population
- Contrasting how surrogate endpoints such as GFR slope can be evaluated in the full trial population
- Addressing the tradeoffs between having of a more definitive of benefit vs. a broader evaluation across the trial population for clinical and surrogate endpoints
12:30pm Lunch & Networking Break
Developing Next-Generation Preclinical Models to Recapitulate Human Renal Function & Pathophysiology
1:30 pm Generating Robust & Predictive In Vivo Systems to Model CKD at Various Stages & Truly Capture Specific Pathophysiology
Synopsis
- Tweaking and refining singular genetic mutations to confidently recapitulate the human form
- Discussing developments that have been made to demonstrate the suitability of different models for testing particular targets
- Exploring how to induce CKD in animal systems in the most translatable way
2:00 pm Employing Ex Vivo Studies to Recapitulate Renal Architecture & Advancing Non-Invasive In Vivo Monitoring of Renal Function
Synopsis
- Enhancing translatability of animal studies
- Leveraging ex vivo kidney cultures as a more relevant cell system to prepare for animal studies
- Developing approaches to non-invasively monitor renal function in vivo
2:30 pm Preclinical RCTs to More Reliably Predict Human RCT Results: Data on Lupus Nephritis & CKD Combination Therapies
Synopsis
- Strategic “go-no-go” decisions involving more reliable ways of animal experimentation to minimize investment failures
- Insights from a multicentre pRCT on baricitinib in murine SLE/ LN correctly predicting the outcome of two human RCTs
- Insights from a single centre pRCT on triple RAS/SGLT2/MR blockade in mice with progressive CKD
3:00 pm A Human Primary in vitro Model of the Glomerular Filtration Barrier for Disease Modelling
Synopsis
- Primary podocyte model maintains high marker expression, including Nephrin, Podocin, WT1, and CD2AP
- TNF sensitivity leads to de-differentiation and decreased barrier integrity, with no effect on 70 kDa FITC-dextran permeability or cell viability
- Co-culture with Glomerular Microvascular Endothelial Cells presents an in vivo-like environment for disease modelling
Showcasing Novel Innovation in Early-Phase Clinical Evaluation of CKD Candidates
1:30 pm APOL1 Related Kidney Disease: Navigating the Heterogeneity of CKD Populations Through Genetic Testing to Develop Targeted Therapies
Synopsis
- The unmet need: genetics and epidemiology of Apol1 in CKD
- Identifying the target population: pathophysiology of Apol1 kidney disease
- Overview of therapies currently in development
2:00 pm Harnessing Basket Trials to Demonstrate Initial Safety & Effectiveness in Numerous Glomerular Diseases
Synopsis
- Providing proof of concept that a therapy targeted to a specific pathway in CKD can be effective across multiple diseases
- Proving safety across different CKD patient subsets
2:30pm Quick Break
2:40 pm Roundtable Discussion | Designing GFR Decline Studies & Considering the Potential of Home Measurements
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas:
- Challenges and mitigations of acute effects when designing GFR slope studies
- Suitable trial length and frequency of kidney function measurements
- Incorporating home based assessments of eGFR to enhance measurement frequency
Moderator Feedback & Audience Debate Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open wider audience debate.
Enhancing Trial Design in Response to the Expanding CKD Market
1:30 pm Implications for Clinical Trial Design Following Introduction of New Endpoints That are Accepted for Registration
Synopsis
- Exploring how the introduction of new endpoints will affect patient selection and trial execution
- Outlining what introducing new endpoints means for broad CKD vs specific indications and factors to consider, such as clinical relevance, feasibility and trial logistics
- Ensuring clear explanations are laid out for the clinical implementation of novel endpoints in CKD trials
2:00 pm How to Design Feasible Clinical Trials in an Era of Growing Availability for Broad CKD Treatments
Synopsis
- Considering the impact of existing CKD drugs on the market on patient recruitment
- Searching for new ways of identifying patients still at risk of CKD progression to be enrolled in future trials in light of the emerging standard of care
- Optimizing inclusion/exclusion criteria and study design to reflect evolving standard of care
2:30pm Quick Break
2:40 pm Roundtable Discussion | Designing GFR Decline Studies & Considering the Potential of Home Measurements
Synopsis
More practical and highly interactive breakout roundtables where attendees can crowd-source solutions and share opinions around pre-assigned topic areas:
- Challenges and mitigations of acute effects when designing GFR slope studies
- Suitable trial length and frequency of kidney function measurements
- Incorporating home based assessments of eGFR to enhance measurement frequency
Moderator Feedback & Audience Debate Moderators will be assigned to each roundtable to facilitate discussion and collate the findings. Following the roundtable discussions, they will present back to the entire delegation and open wider audience debate.
3:10pm Afternoon Break & Refreshments
The Journey Towards More Personalized Therapies for Rare & Common Kidney Disease Patients
4:00 pm Panel Discussion | Replicating Precision-Based Approaches in Large-Scale Clinical Practice: What Will it Take to Get There?
Synopsis
- What must be done to bridge the gap between individual genetic tests or biomarker panels, to large scale clinical trials with thousands of CKD patients?
- How can we more efficiently identify which targets are more relevant for various populations of CKD?
- What will it take to bring forward a companion diagnostic biomarker into the clinic to classify a subgroup of best responders?
4:30 pm Harnessing the Message of the Extracellular Matrix to Find CKD Disease Endotypes & Inform on Treatment Efficacy
Synopsis
- Imbalance in the extracellular matrix (ECM) turnover causes kidney fibrosis, which is one of the best prognosticators of kidney failure
- Non-invasive biomarkers of ECM turnover are associated with an increased risk of outcome in CKD and can identify an endotype of patients with high disease activity that have the potential to respond to treatment
- Treatments that can alter the dysregulated ECM turnover have the potential to halt disease progression
5:00 pm Unveiling the Prospects of Precision Medicine in Future Kidney Disease Trials
Synopsis
- Reviewing progress in the development of predictive biomarkers of response to SGLT2s and ERAs to guide personalized treatment approaches for individual patients
- Highlighting case studies of recent success to match specific CKD therapies to individual patients
- Translating into clinical utility: how to deploy the tools developed primarily from cohort studies in a broader clinical trial setting
5:30 pm Chair’s Closing Remarks
5:45pm Scientific Poster Session & Drinks Reception Hosted by Natera
Synopsis
Join Natera and 200 industry leaders for an evening of networking drinks and scientific posters to collaboratively drive the new frontier of CKD therapies and network with the entire community