Toxicology & Safety Assessment

Key Considerations:

  • In vitro cytotoxicity and genotoxicity screening
  • In vivo safety pharmacology and acute/chronic toxicity
  • No Observed Adverse Effect Level (NOAEL) determination and risk-benefit analysis
  • Good Laboratory Practice (GLP) compliance
  • Safety pharmacology
  • Acute and repeat-dose toxicity
  • Genotoxicity, reproductive toxicity


Preclinical safety evaluation forms the critical foundation for advancing antimicrobial candidates into human trials, requiring integration of early safety thinking into compound design from discovery onwards. This section addresses the essential toxicology frameworks encompassing in vitro cytotoxicity and genotoxicity screening; GLP-compliant in vivo safety pharmacology and repeat-dose toxicity studies; and systematic determination of No Observed Adverse Effect Levels (NOAEL) to establish risk-benefit profiles. Comprehensive species selection, formulation considerations, and target-mediated off-target safety risks are integrated throughout preclinical planning. Practical strategies for de-risking unexpected toxicity and designing early complex cell-based models are examined to expedite human safety translation.


Resources

8 resources

Webinar / Video
ApconiX

Making Safety Part of Drug Design Preclinical Safety for Anti-infectives

Considerations for making safety part of drug design.


Webinar / Video
GARDP

Exploring safety issues in antimicrobial drug development – REVIVE

Highlights common toxicology risks in early antibiotic development and explores strategies to anticipate and mitigate safety-related programme failures.


Webinar / Video
CARB-X

Bootcamp: Preclinical Toxicology (ESCMID/ASM 2018)

Highlights regulatory pitfalls in preclinical development, emphasising the importance of integrating safety considerations early in drug design. A real-world case of unexpected toxicity is used to illustrate key lessons for developers.


Webinar / Video
CARB-X

Coping with Preclinical Toxicology Challenges

Explores how preclinical toxicology can be used effectively to advance antimicrobial drug development. Drawing on regulatory expertise, it covers key toxicology considerations, common development challenges and the timing of nonclinical studies to help developers plan for progression towards clinical testing.


Webinar / Video
Boyds

How to de-risk your IND application

Provides a plan of action that can help to de-risk your Investigational New Drug (IND) application and expedite the path to gaining study clearance.


Webinar / Video
Medicines Discovery Catapult

Is my compound safe (Webinar 8)

Discusses safety in early drug discovery, providing a practical guide to designing safety studies and using complex cell models in early de-risking strategies and toxicity testing.


Guidance
FDA

FDA Guidance for Industry: Safety Testing of Drug Metabolites

Offers recommendations on when and how to identify and evaluate the safety of drug metabolites.


Guidance
EMA

ICH guideline M3(R2) on non-clinical safety studies for the conduct of human clinical trials and marketing authorisation for pharmaceuticals

Promotes international harmonisation of nonclinical safety study requirements to support clinical trials and marketing authorisation, aiming to improve efficiency, reduce animal use, and facilitate the ethical development of new pharmaceuticals.






PACE Delivery Partners

Delivery Partner Network members with expertise relevant to this theme