8:55 am Chair’s Opening Remarks

  • Hetal Kocinsky Vice President, Translational Medicine, Chinook Therapeutics

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

  • Julie Lin Global Project Head, Rare Disease and Rare Blood Disorders, Sanofi

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

  • Pablo Cabral Senior Director - External Innovation, Eli Lilly & Co.
  • Manish Maski Head Of Global Medical & Rare Nephrology, Sanofi Genzyme

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

  • Thomas Idorn Vice President - International Medical, Novo Nordisk

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

  • Hetal Kocinsky Vice President, Translational Medicine, Chinook Therapeutics

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

  • Ronald Perrone Co-Director, Polycystic Kidney Disease Outcomes Consortium, Critical Path Institute

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

  • Jeffrey Hafkin Senior Director, Otsuka
  • Alex Dinh Clinical Leader, Translational & Experimental Medicine, Janssen Research & Development

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

  • Ali Hariri Chief Medical Officer, Eloxx Pharmaceuticals

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

  • Sean Eddy Assistant Scientist - Research, University of Michigan

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

  • Hetal Kocinsky Vice President, Translational Medicine, Chinook Therapeutics

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