Nonclinical safety pharmacology studies for IND and regulatory filings
This workshop will provide an integrated overview of how safety pharmacology studies support IND‑enabling development, with a focus on regulatory expectations, evolving scientific practices, and translational decision‑making. Topics will begin with an overview of current regulatory guidance and agency expectations for safety pharmacology programs, setting the framework for study scope, timing, and interpretation.
Core systems safety pharmacology will be addressed in depth, including nonclinical cardiovascular (CV) safety assessment, with discussion of study design, endpoints, and integration with clinical risk assessment. This will be complemented by a clinical pharmacology perspective on CV safety, including TQT studies, criteria for TQT waivers, and strategies for leveraging nonclinical and early clinical data to support regulatory decisions.
Central nervous system (CNS) safety will be covered through focused discussions on seizure liability, the use of EEGs, and interpretation of neurophysiologic signals in both discovery and IND‑enabling contexts. Respiratory safety pharmacology will be reviewed, highlighting standard approaches, key endpoints, and relevance to human risk.
Broader safety pharmacology considerations beyond the core battery will be discussed, including evaluation of CNS, brain, gastrointestinal, renal, and related organ system effects, with emphasis on fit‑for‑purpose study design and data integration. The workshop will conclude with emerging approaches, featuring novel safety pharmacology studies that have elucidated Nav1.7‑associated dysautonomia, illustrating how targeted mechanistic assessments can inform clinical safety and enhance translational understanding.