Risk Assessment of Novel Neurotherapeutics: Mechanistic Translation of Preclinical Safety and PK/PD Data to a Clinical Setting

Risk assessment is a complex process intended to protect human health. In drug development, this process includes identifying drug candidate-related hazards in relationship to dose and exposure via traditional in vitro and in vivo frameworks. Nonclinical studies in pharmacology, pharmacokinetics and toxicology are designed and conducted under conditions that closely mimic the clinical setting to facilitate proper risk assessment. The introduction of novel model-informed approaches can further translate this information to predict clinical pharmacokinetic and pharmacodynamic (PK/PD) outcomes for supporting clinical development. A well-designed risk assessment strategy, plan and analysis ultimately ensures patient safety relative to potential therapeutic benefit.

In risk assessment for drugs designed to treat neurological diseases, there are additional challenges. These include overcoming the barriers of drug delivery to the central nervous system (CNS), verification of CNS drug exposure and mitigation of safety concerns unique to the CNS such as seizure risk or off-target adverse effects on neurotransmitter pathways. Therefore, novel and intelligent designs for a risk management strategy for CNS drug development are essential. This strategy may be informed by various types of data that include in vitro and in vivo findings, as well as data from multiple types of modeling: e.g., physiologically-based pharmacokinetic (PBPK) modeling, in vitro in vivo extrapolation (IVIVE) and more.

View this webinar to hear about:

  • Approaches to assess anatomical and functional neurotoxicity in animal species
  • Integrating various types of data to create a toxicity profile and manage risk
  • Case studies illustrating the intricacies of assessing and managing CNS risks

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About the Presenters

Marcus S. Delatte, PhD – Vice President, Regulatory Strategy

Dr. Delatte served as a Senior Pharmacology/Toxicology Reviewer at the US FDA for 12 years. He is an established expert in translating nonclinical data to inform clinical program design, including dose selection, safety monitoring, inclusion/exclusion criteria for clinical protocols, and informed consent forms. Dr. Delatte has extensive experience in creating and implementing strategies for regulatory pathways in the US, as well as preparing and creating strategies for formal FDA meetings.  His expertise and experience span multiple therapeutic areas including psychiatry, neurology, pain, rheumatology, infectious disease, immunology, cardiovascular, pulmonary, and oncology.

Devin Welty, PhD – Vice President, Clinical Pharmacology

Dr. Welty has over 30 years of extensive experience in preclinical and clinical pharmacology leading organizations and project teams from early discovery to approval and commercialization in multiple therapeutic areas and modalities. Dr. Welty’s primary therapeutic area of experience includes neurology, ophthalmology, and rare diseases. He has extensive experience in pharmacokinetics, pharmacodynamics, and systems pharmacology and adopting model-informed approaches for candidate nomination and clinical development. He has experience with small molecule, biologics, and cell and gene therapy products, and has supported > 50 IND/CTAs and >20 NDA/MAA/BLAs.

Alison Wakeford, PhD – Scientist

Dr. Wakeford has over a decade of experience in cannabinoid, psychostimulant, and entactogenic pharmacology, including thirteen manuscripts on nonclinical drug abuse liability across all three classes of drugs. Her nonclinical expertise includes both rodent and nonhuman primate experimental design and analysis of these compounds, with a specific focus on central nervous system (CNS) effects.

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