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Session 4 Track 1 and 2: Applying Toxicology Testing to the Clinic
Session Chair(s)
Elena Braithwaite, PhD
Toxicologist
FDA, United States
Andrew Slugg, MBA, MS
Senior Vice President, Global Head of Regulatory Affairs
Alnylam Pharmaceuticals, United States
Due to the unique pharmacokinetic properties of some oligonucleotide-based therapeutics, it can be challenging to compare the exposure achieved in animal pharmacology or toxicology studies to humans. Furthermore, understanding the relevant concentration of drug at the site of action (on and off target pharmacology and DDI) is key to the prediction of clinical outcomes. With a lack of clear guidance, several strategies have been employed to assess the relevance of nonclinical findings and predict clinical efficacy or safety. This session will share case examples of how programs have navigated from preclinical to clinical development including strategies used to calculate safety margins and DDI Liability with the goal of better predicting clinical outcomes.
Learning Objective : At the conclusion of this session, participants should be able to:- Define different parameters including pharmacokinetic metrics that can be used to calculate safety margins
- Describe how in silico PK/PD models can be used in combination with cellular and subcellular kinetics to predict drug concentrations at the site of action
Speaker(s)
Refinement of PKPD Models and DDI Assessment
Representative Invited
GlaxoSmithKline, United Kingdom
Considerations for Determining Safety Margins for Oligonucleotides During Clinical Development
Meena Meena, PhD
Stoke Therapeutics, United States
SVP of Translational DMPK and Clinical Pharmacology
Speaker
Representative Invited
N-Lorem Foundation, United States
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