Chief Scientific Officer EpiVax, Inc. Providence, Rhode Island
Defining a fit-for-purpose, minimal IND data package remains a critical challenge, particularly for complex, personalized, and emerging therapeutic modalities. While regulatory frameworks provide general expectations, there is increasing tension between generating extensive datasets and focusing on clinically and translationally meaningful information that truly informs early development decisions.
This presentation will challenge conventional assumptions in immunogenicity and broader bioanalytical strategies, emphasizing a shift from data volume to data relevance. Specifically, it will highlight the need to move beyond traditional paradigms—such as reliance on ADA titers and rigid cut point approaches—which may fail to capture clinically meaningful immune responses and can obscure important biological context.
Through case studies and cross-modality examples (including PK/PD, immunogenicity, cfRNA, and multi-omics), the session will examine: -Instances where intensive sampling and analysis yielded limited actionable insight -Opportunities to leverage alternative and surrogate biomarkers to better inform IND decisions -The feasibility of generating robust data from limited patient samples, particularly in rare disease and advanced therapy settings
A key focus will be the transition toward a risk-based, context-of-use (COU)-driven bioanalytical strategy, where preclinical and clinical risk assessments directly inform assay selection, data collection, and interpretation. This includes rethinking immunogenicity assessment goals to prioritize clinically relevant responses over exhaustive detection, and integrating diverse datasets to improve translational understanding.
The session will also address global regulatory perspectives, including alignment challenges, assay comparability, and evolving expectations for non-traditional data packages.
Ultimately, this talk advocates for a more strategic, patient-centric approach to IND-enabling data—one that reduces unnecessary burden, enhances scientific rigor, and accelerates development timelines while maintaining patient safety. This is particularly relevant for orphan indications, gene and nucleic acid-based therapies, and settings where sample availability and assay robustness are limiting factors.
Attendees will gain practical insights into designing lean, scientifically justified IND packages that balance regulatory requirements with innovation, enabling more efficient and impactful drug development.
Learning Objectives:
Differentiate between the minimal data required for IND submission and additional exploratory data that may add value but are not essential.
Distinguish between detecting all ADA signals versus identifying and characterizing clinically meaningful immune responses
Identify gaps where over-collection of unnecessary samples or use of non-validated complex assays creates technical and patient feasibility challenges and leads to inconclusive data generation.
Formulate approaches that move beyond standardized methods toward clinically driven, risk-informed decision making
