A case‐study of model‐informed drug development of a novel PCSK9 antisense oligonucleotide
Dr Dinko Rekic, PhD and Dr Jane Knöchel, PhD
Global Product Leader – ticagrelor; Clinical Pharmacometrician, AstraZeneca, Gothenburg, Sweden
Knöchel CPT:PSP. 2022 Dec;11(12):1569-1577.
Wojciechowski 2015 Interactive pharmacometrics applications using R and the Shiny package. CPT:PSP.
This talk focuses on model‐informed drug development (MIDD) of a novel antisense oligonucleotide, targeting PCSK9 for treatment of hypocholesteremia. The case study exemplifies use of MIDD to analyze emerging data from an ongoing first‐in‐human study, utility of the US Food and Drug Administration MIDD pilot program to accelerate timelines, innovative use of competitor data to set biomarker targets, and use of MIDD to optimize sample size and dose selection, as well as to accelerate and de‐risk a phase IIb study. The focus of the case‐study is on the cross‐functional collaboration and other key MIDD enablers that are critical to maximize the value of MIDD, rather than the technical application of MIDD.