8:55 am Chair’s Opening Remarks
Unravelling the Latest Industry Progress Accelerating Towards Continued Approval
9:00 am Detailing the First Non-Immunosuppressive Therapy Approved for IgAN
Synopsis
- Uncovering its unique mechanism of action of blocking both endothelin and angiotensin receptors
- Highlighting the study design and targeted population
- Discussing the potential for its use in combination therapy
9:30 am Highlighting a Two-Step Clinical Development Program Using Total Kidney Volume as a Surrogate in ADPKD
Synopsis
- Reformulating a xanthine oxidase inhibitor for use in PKD treatment
- Moving towards accelerated approval: smaller registration trial using total kidney volume as a surrogate
- Putting in place a more extensive study for full approval
10:00 am Application of Real-World Evidence in Rare Kidney Disease Clinical Development
Synopsis
- Use cases of natural history data in clinical trials for rare diseases
- Incorporating external controls in clinical development
- Optimizing the use of EMR and registry data
10:30 am Structured Networking
Synopsis
This session is a great opportunity to introduce yourself to the attendees that you would like to have more in-depth conversations with. This session is the ideal opportunity to get face-to-face time with many of the brightest minds working in the rare and genetic kidney disease field and establish meaningful business relationships.
10:45 am Morning Break
11:00 am Morning Break & Networking
Delineating the Current & Future Therapeutic Modalities to Illuminate the Windows of Opportunity for Renal Precision Medicine
11:00 am Highlighting the Current Landscape for ARPKD Therapies When One Size Doesn’t Fit All Cystic Kidney Diseases
Synopsis
- Illustrating the differences between ARPKD and ADPKD
- Interrogating translational models for ARPKD
- What are the current therapies and ongoing clinical trials for ARPKD?
11:30 am Panel Discussion: Unlocking the Potential of RNABased Therapeutics for the Treatment of Rare Kidney Diseases
Synopsis
- Discovering the renewed hope in siRNA, ASO and other oligonucleotides-based therapeutics for renal disease caused by genetic mutations
- Understanding the reduction in the risk for off-target insertional mutagenesis
- Deliberating the delivery approaches at present and ensuring organ specificity
12:00 pm Remodel: Understanding the Kidney-Specific Mode of Action Using Novel Technologies
Synopsis
Introducing the patient population
• Highlighting the use of mechanistic trials in drug development
• Biomarker development using kidney biopsies and MRI scans
Highlighting the Opportunities for the Development of Clinically Effective Biomarkers for Early Clinical Development
11:00 am Unravelling the Potential of Atrasentan Beyond IgAN: Integrating Biomarker, Genetic & Clinical Data to Identify Future Indications
Synopsis
- Preliminary data from AFFINITY in FSGS, Alport’s and DKD cohorts
- Genetic, transcriptomic, and proteomic profiling to understand failed tubular repair
- Integrating these diverse types of data to identify promising additional indications
11:30 am Demonstrating the Use of Anti-PLA2R Biomarker in PMN to Test the Efficacy of ACTH Against Rituximab
Synopsis
- Introducing the use of anti-PLA2R as a biomarker to monitor disease progression in Phase III trials
- Improved efficacy and safety of ACTH vs Rituximab in PMN
- Faster response of ACTH
12:00 pm Development of Disease Progression Modelling & a Clinical Trials Simulator Tool for PKD
Synopsis
Generating a Clinical Trial Simulator tool to improve PKD clinical development programs
– Developing total kidney volume as a prognostic biomarker for ADPKD clinical trials
– Future efforts of the PKD outcomes consortium in the renal field
12:30 pm Lunch Break & Networking
1:30 pm Pioneering Gene Therapy Approaches for Kidney Disease
Synopsis
- Bringing a novel gene therapy approach to treat glomerular diseases, an area of huge unmet medical need
- Revealing early results with effective gene therapy in novel animal models representative of human disease
- Opening the way for gene therapy to treat kidney disease
2:00 pm Preclinical Characterization of ADX-097, a Tissue Targeted Complement Therapeutic for Kidney Diseases
Synopsis
- Characterizing the role of local and systemic inhibition in treating kidney tissue injury
- Translational considerations around moving a drug from preclinical to clinical
- Thoughts around biomarkers and their use within precision medicine
1:30 pm Panel Discussion: A Global Initiative: Increasing Collaborative Work to Unite Towards a Streamlined Trials Process
Synopsis
- Agreeing on samples and linked clinical data to decipher the relationship between the biomarker and clinical outcomes
- Collecting patient databases by improving the global repository
- Including study coordinators to aid in prioritising measurements
2:00 pm Protein Restoration Therapy: Circumventing the Genetic Defect in Alport Syndrome & PKD
Synopsis
- Delving into the challenges and opportunities of carrying out a protein restoration therapy for rare kidney diseases
- Is a kidney biopsy sufficient proof of efficacy?
- Using registry studies to achieve rapid approval to be converted into full-approval
2:30 pm Afternoon Break & Networking
3:00 pm Conducting Large & Detailed Profiling Studies in Human Patients: A Guide for Precision Medicine
Synopsis
- Profiling rare kidney disease patients to identify pathway activation
- Encouraging partnerships to get the right drug to the right patient at the right time
- Increasing data availability and open science
3:30 pm Shaping the Trajectory of Trials by Understanding the Benefits of Multicentre, Placebo-Controlled Studies for IgAN
Synopsis
- Understanding the pathogenesis of IgAN and why proliferation-inducing ligands could be an ideal target
- Outlining the advantages of a phase 3 randomized controlled trial to assess the efficacy and safety for IgAN drug development
- Using eGFR and uPCR as surrogate endpoints and their capabilities to shorten clinical trials
4:00 pm Chair’s Closing Remarks
4:00 pm IgAN and Beyond: Navigating the Crowded Landscape in Rare Kidney Diseases & Extrapolating Data from Adults to Children
Synopsis
- Assessing the potential of precision medicine and life cycle management across the spectrum of rare protein-spilling kidney diseases
- ApproZ`aching paediatric patients and extrapolating SGLT2 data from adults
- Reimagining endpoints for FSGS to advance potential treatments