Abstract: 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.

Bio: Dr Jane Knöchel is a skilled professional with a strong background in applied mathematics. Dr Knöchel received her Diploma in Mathematics from Humboldt University zu Berlin and completed a PhD in applied mathematics at Potsdam University. For her PhD she received a 3.5 year research followship as part of the PharMetrX program. In 2020, Dr Knöchel was honored with the Hans Günter Schäfer award for her outstanding contributions to the field of pharmacometrics. Currently, Dr Knöchel is a clinical pharmacometrician at AstraZeneca, where she applies her mathematical skills to inform the clinical development of compounds for cardiovascular, renal or metabolic diseases with special focus on NASH and represents AstraZeneca on the industry board of PharMetrX (a german PhD program).