DAY ONE AGENDA
7:00 am
Registration & Morning Coffee
7:50 am
Chair’s Opening Remarks
Providing Critical Updates on the Current Framework for CDx Development to Facilitate Approvals & Applications
8:00 am Exploring the Current Regulatory Framework for Approvals of CDx by the FDA
Synopsis
- Providing an overview of successful submissions to the FDA, the typical challenges encountered, how to overcome and how to increase success
- Discussing novel approvals, new guidelines and devices that were recently approved that have set a new regulatory precedent
- Looking at what's on the horizon, the emerging trends and cutting-edge technologies and biomarkers
8:20 am How to Accelerate Your Global Trials and CDx Development with Approved and Novel Biomarkers
Synopsis
- Best practices for CDx development with approved tissue and liquid biopsy tests in US & Japan
- Our experience facilitating global trials, from Europe under IVDR to profiling in China
- How to use novel biomarkers – like our tumor fraction, HRD signature – to identify more patients for trials and gain new molecular insights to inform clinical development
8:50 am An Overarching History of CDx Development: How to Break Ground in Future CDx Development & Applications
Synopsis
- Highlighting successes and failures of CDx development from the perspective of big pharma with 50 CDx approved
- Understanding the process and considerations of getting approval for tissue-based diagnostics
- Exploring next generation of diagnostic biomarkers to influence the next wave of CDx applications
9:10 am Leveraging MRD to Provide High-Definition Insights for Clinical Trials
Synopsis
Through the recent acquisition of Haystack Oncology, Quest Diagnostics aims to combine its expertise in oncology, genomics, and pathology with Haystack's next generation liquid biopsy technology. This collaboration will drive the development of reliable MRD tests and expand their accessibility for a range of cancer types, including colorectal, breast, and lung cancers. ctDNA analysis has the potential to provide high-definition insights in drug development. Learn how you can:
- Stratify patients for trials
- Identify therapy response and resistance
- Quantifiably track variants
- Assess treatment outcome early
9:40 am Building a Foundation for Successful Companion Diagnostics in an Evolving International Regulatory Landscape
Synopsis
- Flexible strategies for global CDx launches to adapt to regulatory, clinical and reimbursement changes
- Developing a quality, regulatory and clinical framework that anticipates diverse and evolving compliance requirements
- Preparing for regulatory and quality compliance around novel and emerging technologies
Reimagining Equity, Diversity & Inclusion (ED&I) in Clinical Trials to Improve Accessibility
There is a pressing need to pay closer attention to ED&I as biopharma seeks to encourage more people to take part in clinical trials. The next session aims to discuss the innovation in clinical research and how we can hold ourselves accountable in ensuring precision medicine is a fit-for-all.
10:10 am Panel Discussion: Driving Actionable Steps Towards Clinical Trial Diversity Through Harnessing Equity, Diversity & Inclusion (ED&I) to Ensure Precision Medicine is a Fit-for-All
Synopsis
- Addressing the under-representation of the world’s population in genomic datasets available to pharmaceutical companies and clinicians
- Illustrating the importance of implementing ED&I in CDx design to facilitate more accurate clinical decisions
- Establishing regulatory frameworks and guidelines to advocate for the wide-spread ED&I in biomarker discovery, clinical biomarker development and CDx development and ensure precision medicine is a fit-for-all
10:30 am
Morning Break & Speed Networking
Synopsis
As the CDx community is reunited, this valuable session will ensure you can reconnect with your peers in the room to make new and lasting connections. All attendees will have the opportunity to meet and network with their academic and industry colleagues!
Track A: Biomarker Discovery & Translational Development
Leveraging Digital Pathology for Biomarker Identification to Revolutionize Patient Stratification
11:30 am Next-Generation Multi-Omics: in Minutes, Without Wet-Lab Assays
Synopsis
- Panakeia’s software offers multi-omics biomarker profiles in minutes, without need for wet-lab assays
- This is achieved using an AI-driven software that analyses H&E stained images to determine molecular biomarkers directly from the tissue image
- The company has offerings for both clinical and research
11:40 am Harnessing Next Generation Digital Pathology to Advance Biomarker Discovery Using New Technologies & Tools
Synopsis
- Immune oncology therapies work through complex mechanisms requiring coordinated responses involving multiple classes of cells and activation states over time. The identification of biomarkers to inform drug development, clinical trials, and selection of patients in the clinic cannot be done as it has been in the past
- Emerging tools and techniques have dramatically altered the scale and complexity of spatial biology data, requiring new approaches to image and data analysis and opening new avenues of possibility in disease diagnosis and classification of patients. Furthermore, increasing plex of markers (100s-1000s), improving resolution (cellular and subcellular), and genomic, transcriptional, and protein level data are on millions of cells per study require a systematic, scalable, and explainable approach to panel development, pressure testing, validation, longitudinal stability testing, and image and data analysis
- Recent advancements in digital pathology and address the advantages and liabilities of a bottom-up vs a top-down approach in the integration of complexity and scale of data
12:00 pm Leveraging AI Pathology In Immune Oncology Research & Implementation Into Clinical Trails
Synopsis
- Tumor evolution is shaped by the tumor microenvironment (TME) which regulates tumor survival and promotion and is an area of research for the development of novel medicines to treat cancer based on the immunological cellular landscape.
- However, considering there are roughly 1 billion cells per gram of tumor, counting and classifying each of them and their biomarkers, is scalably prohibitive.
- Here we show the power of AI in digital image analysis by using different machine learning (ML) approaches and leveraging multi-modal data input sources in translational research projects and clinical trials showcasing; precision, scalability and contextualization.
12:30 pm Implementing Digital Pathology to Characterize Immune Exclusion for Patient Stratification
Synopsis
- Utilizing digital pathology to identify and characterize immune exclusion for patient stratification
- Generating data on tumor heterogeneity to inform interpretation of digital-pathology derived assessment of immune phenotypes
- Translating digital pathology from a research tool to a robust and informative diagnostic tool for biomarker discovery
12:50 pm Is it time for Point of Care Companion Diagnostics?
Synopsis
Diagnostic testing beyond the central laboratory has been available for decades but was not widely used or accepted by clinical providers until the COVID pandemic when simple molecular and antigen tests became standard of care. Given the availability, accuracy, and acceptance of these tests, new multiplex Point of Care (POC) devices are under development beginning with respiratory pathogens such as influenza, RSV and adenovirus. We will explore the feasibility and clinical value of POC testing as a companion Diagnostic (CDx) strategy. Our presentation will focus on the following:
- Technical feasibility
- Possible formats
- Example applications
- Future Home Use/Over the Counter applications
- Regulatory strategies
- Community Applications
Track B: Clinical Biomarker Development
Ensuring Robust Clinical Outcomes Using Endpoints to Define Clinical Benefits
11:30 am Tumoral DKK1 Expression as a Predictor of Clinical Benefit to DKN-01
Synopsis
- DKK1 is overexpressed in many cancers, often leading to worse outcomes by promoting tumor growth, metastasis and contributing to an immunosuppressive tumor microenvironment
- DKN-01 in combination with standard of care therapies has demonstrated compelling clinical data with enhanced activity seen in DKK1-high patients
- DKK1 RNAscope CDx development to support the clinical development of DKN-01 is underway
11:50 am Precision Data: Matching the Right Multimodal Data to the Right Research Question to De-risk Therapy Development
Synopsis
- Overview of multi-modal data and its impact on therapy/CDx development, especially for trial designs (eg. eligibility, enrichment, etc), regulatory submissions, and pipeline/portfolio strategy development
- How to leverage precision multi-modal data to answer research questions and increase PTRS of therapy/CDx development programs
- Dissecting common data challenges, including incomplete patient journey (missing data points or inconsistent longitudinal data which limits understanding for disease progression and treatment response); lacks multi-modal data with depth and breadth (DNA-only or isolated panels of genes), biased data (does not represent real world population); or limitation of scale with small cohort sizes (unable to apply advanced analytical techniques)
- Collaboration and creative approaches to overcome challenges with real-world data and maximize impact to the next wave of therapeutics
12:20 pm Potential Utility of Surrogate Endpoints to Inform Clinical Outcomes
Synopsis
- Identifying new surrogate endpoints using novel monitoring tools to inform clinical outcomes
- Demonstrating correlations between biomarkers and clinical outcomes
- Understanding regulatory guidelines to satisfy correlations between and address unmet clinical needs
12:40 pm Utilities of ctDNA for Identifying and Tracking Molecular Residual Disease in Solid Tumors
Synopsis
- Circulating tumor DNA is an important biomarker in MRD determination, the detection technology is more sophisticated than therapy selection ctDNA assay.
- Considering the prognosis value of using ctDNA to determine MRD status in solid tumor, a number of MRD solutions had been invented and implemented in many clinical studies.
- Burning Rock a tumor-informed personalized MRD assay, brPROPHET, which tracks 50 personalized cancer-specific variants with LoD of 40ppm, and the updated clinical data are presented.
Track C: CDx Development & Commercialization
Exploring Strategic Guidelines to Implement CDx Assays in Clinical Trials for Successful Applications
11:30 am Considerations for Strategy, Regulatory Requirements, Implementation & Commercialization of a NASH CDx Assay
Synopsis
- The global prevalence of Nonalcoholic steatohepatitis, or NASH, is increasing with over 5% of the general population affected. If untreated, NASH may progress to liver cirrhosis, liver carcinoma, and death
- Numerous risk factors influence the development of NASH, including genetic predisposition. We initiated an investigational trial for a hepatocyte-targeted siRNA approach in NASH
- A clinical trial assay is used to screen patients for a precision-medicine-based approach. This presentation will cover challenges related to implementing a screening test for patient enrollment in our trial
- We will review the CDx strategy, regulatory requirements, diagnostic development process (including commercial considerations), and the implementation of the clinical trial assay
11:50 am Project Managing Biomarker / CDx Programmes with Pharma – A Diagnostic Company’s Perspective
Synopsis
Laura Nelson has been a Senior Project Manager at Almac for many years, with responsibility for multiple biomarker and CDx programmes. In this talk she will discuss Almac’s model for working with Pharma and Biotech companies and highlight some of the realities of what it takes to successfully deliver on a precision medicine programme:
- Aligning the ‘value proposition’
- Defining a clear biomarker strategy from the outset
- Agreement on timelines and project delivery
- The importance of selecting the appropriate technology
- How to successfully run clinical trials
- Regulatory requirements for global territories
- Areas for future improvement and better collaboration
12:20 pm Global Companion Diagnostic Development to Identify NSCLC Patients with EGFR Exon 20 Insertions for Exkivity
12:40 pm A Perspective on IVDR Regulations in Europe: A Focus on Article 5(5)
Synopsis
Speaker to be confirmed: Unilabs
- IVDR Article 5(5) Explained: Understand the specific provisions outlined in Article 595) of the In Vitro Diagnostic Regulation (IVDR) and its impact on Laboratory Developed Tests (LDTs) in Europe. Gain insights into the requirements for LDTs and the obligations for manufacturers, including performance evaluation and conformity assessment procedures
- Impact on Companion Diagnostic (CDx) – Gain insights into strategies to navigate these challenges and the potential of CDx commercialization under the new regulations
12:50 pm
Networking Lunch Break
Ensuring Robust Pre-Clinical Analysis to Facilitate Translational Development
1:50 pm The Use of Biomarkers to Advance Clinical Trials in Rare Genetic Disorders
Synopsis
- How biomarkers are used for clinical trials of rare genetic disorders as compared to oncology
- Example of biomarker usage to understand the PD/safety of gene editing based therapy, EDIT-301, in the treatment of sickle cell disease.
2:10 pm AI-Powered Analysis of Histopathology Drives Identification and Use of Predictive Biomarkers
Synopsis
- AI-driven PD-L1 analysis in routine clinical use: Lunit SCOPE PD-L1 used for TPS/CPS reading as part of Guardant Health’s tissue testing
- TIL analysis from H&E holds predictive potential for Immuno-Oncology therapeutics via LUNIT SCOPE IO
- AI-based analysis of IHC provides quantification and localization of cell surface proteins enabling predictive biomarkers
2:40 pm Panel Discussion: Utilizing PK/PD Analysis of Circulating Biomarkers to Identify Predictive Biomarkers of Response to Inform Translational Development Decisions
Synopsis
- Addressing how PK/PD can be used to identify early predictive and response biomarkers
- Leveraging the PK/PD analysis to inform dose and patient selection strategies in clinical development and practice
- Integrating a quantitative framework with biomarker research to enable personalized medicine for cancer treatment and beyond
3:00 pm Panel Discussion: Oncological Biomarker Testing Paradigm: Determining the right Molecular Test at the Right Time for the Patient: With Biocartis
Synopsis
- Who makes the decisions at which point—pathology, oncology, diagnostic testing, follow up?
- Patient care: The Pharma perspective
- Where are we failing and where can we do better?
Assessing Safety & Efficacy of ctDNA for Improved Immunotherapies
1:50 pm Joint Modeling of Safety & ctDNA to Support Recommended Phase 2 Dose Identification in Phase 1 Study
Synopsis
- Circulating tumor DNA (ctDNA)-based molecular response has been shown to predict clinical response earlier than RECIST response and is associated with long-term survival with different therapies across solid tumor types
- Beyond dose-limiting toxicity (DLT), pharmacodynamic markers or pharmacokinetics can be used to help identify optimal dosing of biologics
- We used an innovative integrative approach that factors in peripheral key mode of action biomarkers, ctDNA, and dose-limiting toxicity (DLT) rate from the Phase 1 HAMMER study (NCT04009681) to help identify the recommended Phase 2 dose (RP2D) for SAR444245
2:10 pm Pinpoint Disease-Driving Clones to Deliver a More Targeted Therapy
Synopsis
• A novel approach to stratification and monitoring of patients with heme malignancies
• How to leverage single-cell DNA multi-omics for accurate determination of mutations, understand resistance mechanisms, and develop more targeted therapies.
• A case study on how clonal surveillance can be used to inform clinical and therapeutic decisions.
2:40 pm Redefining Surrogate Endpoints for Enhanced Efficacy in Novel Immunotherapies
Synopsis
- Building cell-free DNA (cfDNA) for tumor informed and tumor naive panel to monitor tumor mutational burden (TMB)
- Identifying biochemical molecular responses and translating them using biopsy sequencing and ex vivo immunology assays
- Identifying mechanisms of action to drive individual translational assays built for personalized medicine
3:00 pm The Evolving Regulatory Landscape for AI-Powered Drug and Diagnostic Development: An Expert Panel
Synopsis
- What can we expect to change based on this guidance, and what does this mean for pharma clinical development?
- What impact should we expect to see in the near-term vs. long-term? What additional evidence needs to be collected to drive that next wave of change?
- How can clinical development teams get ahead of these anticipated changes to maximize the impact and benefit to patients and R&D pipeline?
Addressing Global Regulatory & Commercial Considerations to Inform the Future of Diagnostics
1:50 pm Diagnostic Landscape Review: How Changes in Technology, Drug Development Portfolio & Global Regulatory Landscape are Altering the Diagnostic Testing Landscape
Synopsis
- As the promise of precision medicine has come to fruition, there is increased attention on diagnostic tests for use in drug development, registration/approvals and for routine patient and health management
- This talk will focus on the changing landscape for diagnostics providing awareness for emerging challenges globally (IVDR) as well as challenges in implementing companion diagnostics outside of oncology from a regulatory, laboratory, IVD sponsor as well as early commercial considerations
- The future of diagnostics for drug development and patient care are starting to emerge and as an industry we need to be looking forward on what we need to do today to be prepared for what the future state of diagnostics will be
2:10 pm Future Convergence of Innovations in Advanced Oncology Diagnostic Technology
Synopsis
- Outline how advances in ctdna, digital pathology, and data analysis technologies are fueling a new wave of insights in oncology care and enabling applications such as early cancer detection, comprehensive genomic profiling, and minimal residual disease detection
- Provide insight into how these offerings might be able to navigate numerous regulatory, commercialization, and reimbursement challenges and how technology convergence will accelerate adoption
- Identify how strategic partnerships can best position early adopters of these technologies to take full advantage of this future paradigm
2:40 pm Regulatory Considerations for Companion Diagnostics to Guarantee Commercialization
Synopsis
- Evolving regulatory climate and need to adapt with focus on EU IVD regulations
- Considerations for biomarker-driven clinical trials
- Considerations for global registration of companion diagnostics
- Regulatory perspective of strategies for a successful companion diagnostic program
3:00 pm Fireside Chat: Harnessing the Genomics of Cancer: What Can We Learn from Orphan Drug Developers?
Synopsis
Is it time to treat cancer as a rare disease? Meet us at the intersection of cancer and orphan drug development for a candid discussion on how evolving genomic approaches for rare diseases can help advance clinical biomarker discovery and companion diagnostics for cancer precision medicines.
3:30 pm
Afternoon Networking Break
Navigating the Ever-Changing Biomarker & CDx Landscapes to Ensure Stakeholder Alignment & Wide-Spread Accessibility
4:00 pm Leveraging Advances in Spatial Biology to Develop Next-Generation Companion Diagnostics
Synopsis
- Utilizing multiomic spatial data from tissues to inform companion diagnostics
- Using biomarker multiplexing to capture clinically-relevant information
- Applying digital pathology and artificial intelligence to interpret complex patterns
4:30 pm The Genome Era: Unlocking The Future of Precision Medicine
Synopsis
- Whole Genome Sequencing (WGS): Illumina's vision and broad applications
- A disruptive approach to MRD detection and testing using WGS
- Accelerating clinical adoption of WGS-based MRD testing through Pharma collaborations
5:00 pm Building a New CDx Ecosystem to Support Early Clinical Development Through Commercial Launch
Synopsis
- Tempus combines RWD offering with CDx development to optimize biomarker design
- Supporting trial enrollment using design controlled IUO tests
- Improve speed to market using Tempus FDA-approved xT CDx assay
- Immediate commercial access to 1000s of oncologists in the US and growing internationally
5:30 pm Adapting to the Reimbursement Policies of CDx to Improve Precision Medicine in Oncology: A Payer’s Tale
Synopsis
- Understanding what is needed to achieve a successful reimbursement outcome on national and international levels
- Addressing clinical utility data that would fast-track turnaround times of reimbursements
- Delving into payer perspectives regarding the decision process for covering costs of CDx
5:50 pm NeoGenomics: Navigating the Ever-Changing Biomarker & CDx Landscapes to Ensure Stakeholder Alignment & Wide-Spread Accessibility
Synopsis
• Tumor informed MRD can support clinical development in a number of ways (e.g. patient enrichment, monitoring, surveillance)
• While many MRD applications for solid tumors continue to emerge, CDx development with these technologies will also be required in the near future
• NeoGenomics is uniquely positioned with the RaDaR technology to support all MRD applications for drug development, including CDx development through commercialization
6:20 pm Working Collaboratively with Cross-Sector Stakeholders to Build Projects to Accelerate Development & Broaden Accessibility to Biomarker Testing
Synopsis
- Strengthening collaborations between industry, government, academia, patient advocacy, and other non-profit and private sector organizations
- Kickstarting and refining projects aimed at biomarker development
- Widening accessibility to biomarker testing related to cancer therapeutics
6:40 pm
Chair’s Closing Remarks & End of Day One
6:40 pm
Drinks Reception
7:30 pm Dinner with Genomenon
Synopsis
Please be advised that confirmation of your registration to attend the Dinner is subject to approval from the host. You will receive confirmation from Annie, the Senior Event Manager within 1 week of registering your interest.