Conference Day Two
7:30am Check-In & Coffee + Light Breakfast
8:25 am Chair’s Opening Remarks
Combining Patient Insight & Fostering Diversity in Renal Trials
8:30 am Minimizing Patient Burden & Expanding Recruitment Efforts to Promote Diversity in CKD Trials
Synopsis
- Gaining a deeper comprehension of the factors contributing to the underrepresentation of minorities in CKD studies
- Exploring what can be done, from leveraging social media to collaborating with advocacy groups, to revolutionize recruitment strategies
- Evaluating what is being done on a site, study and global level to reduce patient burden and encourage participation
9:00 am Panel Discussion | Enhancing Global Inclusion of Underserved Communities in CKD Trials for Greater Representation in Clinical Samples
Synopsis
Opening up trials to more countries to address underserved populations
Exploring case studies of conducting CKD trials in different countries
Highlighting the benefits of expanding the reach into more countries
Emerging Therapies in Acute Kidney Injury (AKI)
9:30 am The Selective Cytopheretic Device: A Novel Cell-directed Extracorporeal Therapy Targeting the Host-Response
Synopsis
- Despite improvements in critical care medicine, acute kidney injury continues to cause high mortality rates in critically ill patients.
- AKI promotes a systemic inflammatory response resulting in microvascular damage and can lead to multi-organ dysfunction.
- This session will describe the processes of a dysregulated immune response, the mechanism of action of the novel selective cytopheretic device, FDA approval in pediatric AKI due to sepsis, and the study design of the pivotal NEUTRALIZE-AKI trial in adult AKI
10:00am Morning Break & Refreshments
Leveraging the Availability of Advancing Technologies to Identify New Targets
10:30 am REMODEL: Understanding the Kidney-Specific Mode of Action Using Novel Technologies
Synopsis
- Mechanistic, exploratory trials in clinical drug development
- Combining novel methodologies for a holistic understanding of the MoA
- The concept of kidney biopsies for research purpose • Applying Single-Cell RNA-Seq to Correlate Particular Genes to Certain Mechanistic Pathways
11:00 am Utilizing GWAS as a Tool to Define Pathogenic Signalling Pathways Associated with Glomerular Sclerosis & Prioritize New Drug Targets
Synopsis
- Performing large-scale and accurate genome-wide association studies to dissect the etiology of FSGS and its subphenotypes
- Applying this approach to identify novel and specific pathways that are amenable to drug targeting and intervention
11:30 am Harnessing Whole Exome Sequencing for Discovery of Chronic Kidney Disease Therapeutic Targets
Synopsis
- Use of whole exome sequencing at scale for discovering potential targets in chronic kidney disease
- Analytical challenges in genome-wide genetic analyses of CKD
Clinical Progress in Regenerative Medicine: Striving Towards a One-Dose Cure for CKD
10:30 am Systemic Cell Therapy in Early Phase Trials for Diabetic Kidney Disease
Synopsis
- Showcasing successful demonstration of feasibility, safety and preliminary efficacy of allogeneic cell therapy for Progressive CKD in a phase 1/2 trial setting
- Setting the scene for Phase 3 trials in US, Japan & EU
- Evaluating how close this is to widespread commercial/ clinical reality
11:00 am Harnessing Podocyte Gene Therapy to Provide LongTerm Remedies for Kidney Disease
Synopsis
- Reviewing gene therapy opportunities in both genetic and nongenetic glomerular disease
- Feasibility of AAV-based gene therapy for kidney disease
- Future perspectives on podocyte gene therapy
11:30 am Developing antifibrotic and anti-inflammatory therapeutics in clinical stage for common and rare renal diseases including CKD and Alport syndrome
Synopsis
- Developing Vonafexor in the Proof-Of-Concept Phase 2 study “ALPESTRIA-1” in Alport syndrome
- Validating efficacy of Vonafexor on kidney function and kidney remodeling in a severe preclinical mouse model for CKD as much as for Alport syndrome
- Expanding Vonafexor analog development to larger kidney indications
Translating New CKD Therapies into Clinical Practice Guidelines in a Rapidly Changing Landscape
10:30 am Evolving Clinical Practice Guidelines for CKD Management in Response to the Growing Availability of New Treatment Options
Synopsis
- Reviewing where all the current therapies fit into the current standard of care management
- Outlining how clinical practice guidelines are developed and evolved over time and delineating the criteria for assessing the inclusion of new drugs
- Investigating how the diverse mechanisms of action among the numerous drugs in development could lead to complementary treatments in practical scenarios
11:00 am Panel Discussion | Bringing Drugs Evaluated in Trial Cohorts Selected Based on Proteinuria Enrichment into Practical Application in Real-World Scenarios
Synopsis
- Recognizing that the enrichment criteria employed in a trial do not necessarily limit a drug’s suitability exclusively to that specific population in real-world scenarios
- Translating the evidence generated in trials into suitable recommendations for treating patients with varying UACRs
- Discussing how clinical practice guidelines anticipate keeping pace with the rapidly evolving landscape of new CKD therapies
12:00pm Lunch & Networking Break
Innovating Translatable Model Systems to Assess Novel Drug Modalities & Specific CKD Targets
1:00 pm Nanoparticle Carriers: Translating into Clinical Practice to Minimize Systemic Side Effects & Enhance Therapeutic Accumulation in the Kidney
Synopsis
- Reviewing the emerging nanoparticle design strategies available to potentially clinically deliver CKD therapeutics
- Optimizing targeting ligands to actively direct nanoparticles to specific kidney components
- Reviewing challenges for the successful clinical translation of nanoparticle-based therapies
1:30 pm Panel Discussion | Sharing Learnings from Successful Model Systems Used to Demonstrate Efficacy of Treatment in CKD & Accelerate Progression to the Clinic
Synopsis
- Ensuring confidence of in vivo or in vitro kidney tissue to behave like in the human condition
- Assessing the suitability of different models for testing specific targets
- Despite successes, outlining key aspects of preclinical model development in CKD that require further refinement to enhance translational capabilities
Exploring Current & Potential Immune Targets in Clinical Development for Kidney Disease
1:00 pm Translating Advancing Knowledge of Complement’s Role in Glomerular Disease Pathophysiology to Drive Clinical Development of Novel Targets
Synopsis
- Deepening understanding of the precise mechanisms driving complement activation in different glomerular diseases
- Exploring the interactions between complement and other inflammatory pathways to develop targeted agents that disrupt underlying pathophysiological processes
- Analyzing opportunities for new trials to be instigated with therapeutic complement inhibitors
1:30 pm Exploring the Potentials of Complement Inhibition as a Therapeutic Strategy to Improve Kidney Transplant Outcomes
Synopsis
- Role of complement dysregulation in kidney transplant recipients
- Potential role for complement inhibition in improving kidney transplant outcomes
Optimizing Real-World Evidence to Supplement Clinical Studies & Maximizing Payer Support
1:00 pm Panel Discussion | Navigating Payers Perspectives & Moving Towards Surrogate Endpoints to Demonstrate Reimbursement for CKD Patient Populations
Synopsis
- How important is having hard outcomes data vs a surrogate measure of CKD progression or improvement?
- What will it take to pivot away from hard endpoints to demonstrate convincing evidence of improvements in renal function?
- What are the clinical evidence requirements from payers and insurers to demonstrate reimbursement and offer effective therapies to CKD patients sooner?
1:30 pm Securing Payer Buy-In to Enhance Patient Access to Your CKD Treatment
Synopsis
- Strategically analyzing optimal avenues for reimbursement generation post-approval
- Emphasizing the significance of educating payers on the burden of CKD and the unmet need of existing therapies
- Establishing robust, evidence-based treatment guidelines, specific to CKD stage or patient subtype, to maximize support from payers and insurers
2:00 pm Benchmarking Real-World Evidence Data to Transform Efforts of Earlier Stage Diagnosis & Improve Patient Outcomes
Synopsis
- Outlining insights on widespread underdiagnosis and importance of detecting CKD earlier obtained from RWE studies
- Transforming patient care using RWE data to optimize CKD treatment guidelines
2:30pm Afternoon Break & Refreshments
What’s Next for CKD Drug Development?
3:00 pm Optimizing the Potential of the Kidney Cell Atlas: A New Era in Target & Pathway Discovery
Synopsis
- Translating the data collected from the kidney cell atlas to identify new pathways and targets associated with CKD
- Mapping out the target landscape in each kidney disease
- Charting molecular signatures to ultimately predict which patients are at risk of progression to kidney failure
3:30 pm Panel Discussion | From Slowing Progression to Halting Entirely: Debating the “What’s Next” for Diabetic, Hypertensive & Rare Kidney Diseases
Synopsis
- From SGLT2s and ERAs, to GLP1s and anti-APRIL: where is the field heading next?
- Evaluating the most exciting opportunities for new treatment options
- Shifting the focus towards more pivotal therapies specific to underlying kidney pathophysiology, with an end goal of stopping, rather than slowing, CKD progression