Explore the Agenda
Smarter Model Selection: In Vivo, In Vitro & Computational Integration for Toxicology
8:55 am Chair’s Opening Remarks
9:00 am Evolving Safety Models: Integrating In Silico Insights & Tailored In Vitro Approaches for Smarter Early Target & Candidate Prioritization
- Target safety and in silico alerts: leverage data and mechanistic insights to identify hazards linked to target biology or chemical structure before synthesis
- Tailored in vitro models: beyond standard screens to understand toxicity drivers and prioritize candidates with the lowest risk
- De-risk earlier with confidence: integrate early data into a decision strategy to reduce attrition and accelerate decision
9:30 am Advancing Computational Toxicology to Better Predict Safety for Complex Modalities Including Larger Molecules
- Benchmark evolving in silico approaches that bridge data gaps for large molecules and gene therapy where limited clinical and preclinical datasets hinder model accuracy and validation
- Learn strategies to integrate computational insights with in vitro and in vivo evidence to strengthen species relevance decisions mitigate sampling bias and improve translatability across platforms
- Explore how to evaluate combination therapy safety by modelling target pathway interactions dose considerations and additive or offsetting toxicity risks to support more confident regulatory acceptance
10:00 am Morning Break & Speed Networking
11:00 am Impact of Structural Alerts on Drug Discovery: A Comprehensive Analysis of Marketed Drugs
- Re-examining the role of structural alerts by analyzing over 1,000 orally administered marketed drugs to understand how frequently alerts occur in clinically successful compounds
- Disentangling correlation from causation by evaluating relationships between structural alerts, reactive metabolite formation, DILI potential, daily dose, and therapeutic area
- Informing smarter de-risking strategies by showing where structural alerts meaningfully contribute to risk assessment and where over-reliance may unnecessarily limit innovation
11:30 am Case Study of Microphysiological System Models for ILD: Developing In Vitro Assays for Evaluating ADC Lung Safety
- Leveraging complex in vitro models to evaluate ADC-induced lung toxicity
- Building in vitro tools for high-throughput compound screening
- Pending relevance to pathogenesis of ILD, high-throughput imaging-based assays may potentially be incorporated into a holistic screening approach to differentiate ADCs and guide selection of safer compounds
12:00 pm Lunch Break
Regulatory Strategy & Translational Model Selection in Toxicology
1:00 pm IQ MPS Affiliate & Industry Collaboration: Accelerating Adoption of In Vitro NAMs
- Cross-pharma collaboration for in vitro NAM implementation in drug development
- Strategic global regulatory engagement and thought leadership
- Driving industry benchmarking, model qualification, and education
1:30 pm Applying Liver MPS Technologies to Improve Preclinical DILI Prediction
- Demonstrate how liver microphysiological systems improve mechanistic understanding and human relevance in assessing drug‑induced liver injury
- Present case studies where liver MPS provide earlier or more predictive detection of hepatotoxic liabilities compared with traditional in vitro assays
- Highlight translational biomarkers and functional readouts from MPS that strengthen decision‑making in preclinical safety assessment
2:00 pm Afternoon Break & Poster Session
3:00 pm Panel Discussion: Regulatory & Pharma Expectations in the Adoption of NAMs: IQ MPS Affiliate Perspective
- How pharma teams are collaborating within IQ MPS Affiliate to progress simple and complex in vitro NAM platforms toward regulatory acceptance
- Review insights from recent FDA publication on submitted NAM case examples and reviewer feedback – what works and what can be improved (need for well-articulated context-of-use)
- Where AI-enabled validation initiatives like VICT3R can strengthen confidence by benchmarking prediction accuracy against real clinical outcomes
- Gain practical guidance for integrating in vitro and in silico NAMs into toxicology packages without over-claiming translatability
- What industry needs from consortia, regulators, and data-sharing frameworks to accelerate adoption of novel platforms covering diverse mechanisms of injury
4:00 pm Translating Preclinical Toxicology & Efficacy Insights into a Successful IND
- Evaluate in vitro discovery and investigative toxicology in cell therapy platforms to define target biology, evaluate therapeutic liabilities, and establish early human relevance
- Review fit-for-purpose in vivo models in cell therapies to integrate safety with efficacy, PK, and biodistribution, while acknowledging species differences and model limitations
- Discover a strategy integrating in vitro and in vivo data to address safety liabilities while leveraging efficacy strengths for a regulatory-aligned IND in cell therapy
Managing Toxicity Alongside Efficacy to Optimize Study Designs
4:30 pm Balancing Toxicology & Efficacy to Design Studies That Predict Real Clinical Value
- Avoid costly pipeline pivots by distinguishing regulatory-driven toxicology studies from internal proof of-efficacy experiments that determine whether a program is worth advancing
- Improve Phase 1 success rates by integrating mechanistic efficacy insights upfront, not after GLP, ensuring safety signals are interpreted in the context of therapeutic promise
- Strengthen decision-making by aligning toxicology, pharmacology, and regulatory expectations early so resource-limited programs don’t over-optimize for approval while under-investing in efficacy