Preclinical Development – Biomolecular

Keynote: Intra-Target Microdosing (ITM) - A Novel Drug Development Approach - Enabling Safe,  Human-Specific, and Rapid Preclinical Candidate Selection and Translation of Novel Drug Compounds

Keynote Speaker(s)

• 

  Tal Burt, MD

  President
  Phase-0/Microdosing Network

Moderator(s)

• 

  Naveen Thakral, PhD

  Director, Pharmaceutical Sciences
  Schrodinger, Inc. USA
  New York, New York

The presentation will describe a new drug development approach - Intra-Target Microdosing (ITM). The approach administers a microdose of the test article, i.e., 1/100th of the therapeutic-level dose, into an area of the body that is 1/100th of the total body mass or less. This way, the targeted area is exposed to therapeutic levels for a short period of time while the rest of the body receives microdose exposure with the implied safety advantages. Exposure to therapeutic levels at targets of interest may enable obtaining pharmacodynamic (PD) and mechanism-of-action (MOA) data not otherwise available with other microdosing studies. The safety advantages are associated with considerable simplification of the regulatory package required for first-in-human (FIH) studies thus allowing such studies to conducted earlier and cheaper. The implied safety also means that the approach can be used directly in patients in FIH studies, which, together with the PD and MOA data could lead to arrival at meaningful developmental decisions 2 or more years before traditional approaches. Considerable progress has been done recently in the application of ITM and the development of dedicated modeling approaches.