7:30 am Registration & Morning Coffee
8:20 am Chair’s Opening Remarks
Exploring the Clinical Landscape to Gain Insights Into Game-Changing Therapies on the Horizon (Continued)
8:30 am APOL1 Kidney Disease: A Precision Medicine Approach
Synopsis
- Leveraging genetic insight to identify subpopulations at risk
- Overview of Phase 1 data for APOL1-directed therapy
- Translating genetic insights into precision clinical trial design
9:00 am APOL1-Mediated Kidney Disease (AMKD): Pathophysiology & Promise of Antisense Oligonucleotide (ASO) Therapy
Synopsis
- Discussing multiple proposed mechanisms of disease for AMKD
- Sharing Phase 1 data for APOL1 ASO Therapy
- Outlining AMKD Phase 2 study design challenges for APOL1 ASO Therapy
9:30 am Bringing Genetic Therapy to Rare Kidney Diseases
Synopsis
- Developing ELX-02: New small molecule genetic therapy to treat Alport patients with nonsense mutations developed for superior PTC readthrough compared to Gentamicin. Focusing on rare kidney diseases
- Compelling proof of concept in Phase 2 study showing that in only 8 weeks ELX-02 treatment improved morphology by reducing podocyte foot process effacement in Alport Syndrome patients with nonsense mutations
- Planning a larger, longer trial to validate study results
10:00 am Morning Refreshments, Networking Break & Poster Session
10:30 am Intersection of Emerging Genetic Evidence for Accelerated Disease Progression & a Late-Stage Clinical Candidate for a Novel Preclinical Candidate for Rare & Genetic Kidney Disease
Synopsis
- Discovering the genetic background and new alleles influencing ADPKD
- Demonstrating aberrant purine metabolism and exploring preclinical model data
- Discussing the path forward
11:00 am Review of Recent Data Evaluating the Impact of Atacicept on Long-Term Disease Modification in IgAN
Synopsis
- Investigating the role of cytokines BAFF and APRIL and B cells in the pathogenesis of IgAN
- Reviewing the goals of disease modification in IgAN with regards to Gd-IgA1, hematuria, proteinuria, and eGFR stabilization
- Examining the results of the ORIGIN2b 96-week open label extension study
Clarifying Regulatory Paths Towards Accelerated Approval & Understanding Paediatric Investigations in the Rare Renal Disease Setting
11:30 am Q&A Session: Discussing How to Prepare for & Pursue the Accelerated Approval Pathway for Rare & Genetic Kidney Diseases
Synopsis
- This is your opportunity to prepare your burning questions for this dynamic Q&A session with the FDA on accelerated approval pathways for drug candidates in the rare renal space!
12:00 pm Overcoming Specific Challenges in the Development of Drugs for Pediatric Renal Diseases
Synopsis
- Reviewing the unique ethical obligations in conducting pediatric clinical trials
- Discussing specific challenges related to the design and conduct of pediatric clinical trials, including dose finding, endpoint selection and use of pediatric extrapolation
- Providing examples of innovative trial design approaches and new therapies made available to pediatric populations
12:30 pm Lunch & Networking Break
Enhancing Awareness, Patient Participation & Real-World Evidence Integration in Ultrarare Kidney Diseases to Streamline Trials & Reduce Patient Burden
1:30 pm Rare Disease, Rare Evidence? How to Fill the Gap
Synopsis
• Talk details to be announced
2:00 pm Reducing Patient Burden & Diversifying Rare Disease Trials: What is the Best-Case Scenario?
Synopsis
- Investigating limited representation in rare diseases and how we can ensure heterogeneous populations and diseases presentations are represented in trials
- Explore innovative recruitment approaches to enhance diversity of patient enrolment
- Assessing ongoing efforts at the sites and global levels to minimize patient burden
2:30 pm Lessons Learned from Incorporating Patient-Centric Outcomes in Clinical Trials of ADPKD
Synopsis
- Discussing the complexity of choosing and/or developing patient-centered outcomes for studies in ADPKD
- Exploring unique considerations of using patient-centric outcomes in ADPKD
- Describing the lessons learned from using a patient-based registry to evaluate patient-centric outcomes in patients with ADPKD
3:00 pm Afternoon Networking Break
3:30 pm PARASOL: A Cross-Stakeholder Problem-Solving Model
Synopsis
- Sharing the methodology used for FSGS endpoint progress
- Demonstrating the value and structure of collaboration among academia, industry, regulators, and nonprofits
- Discussing lessons learned and future paths
Understanding the Offering of Associations & Foundations to Gain Practical Insights on Getting Patient Input
4:00 pm Panel Discussion: What Do Patients & Advocates Wish Drug Developers Understood to Build Trust & Improve Insight Exchange
Synopsis
- Evolving the patient engagement paradigm in rare diseases to ensure patient voice is never lost through the development process
- Connecting undiagnosed individuals with testing and resources to build community
- Stories of successful patient engagement and areas for industry to improve
Discussing Market Access Strategies to Bring Improved Treatment Options to Patients
4:30 pm The Criticality of Good Regulatory Strategy in Path to Market Planning – A Small Company, Rare Renal Disease Perspective
Synopsis
- Maximising chances of success by correctly sequencing regulatory activities with the Division review team and OOPD
- Taking the time to find alignment and involve independent experts in every stage of the regulatory pathway – genuine, authentically independent experts can be very influential
- Making it paramount to establish a collaborative relationship because everyone has the same goal: to bring improved treatment options to patients