A case‐study of model‐informed drug development of a novel PCSK9 anti sense oligonucleotide. Part 1: First time in man to phase II

Here, we show 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 pil...

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Published in:CPT: pharmacometrics and systems pharmacology 2022-12, Vol.11 (12), p.1569-1577
Main Authors: Knöchel, Jane, Nilsson, Catarina, Carlsson, Björn, Wernevik, Linda, Hofherr, Alexis, Gennemark, Peter, Jansson‐Löfmark, Rasmus, Isaksson, Rikard, Rydén‐Bergsten, Tina, Hamrén, Bengt, Rekić, Dinko
Format: Article
Language:English
Subjects:
Case Report
Case Reports
Digitization
Drug Development
Drug dosages
Humans
Oligonucleotides, Antisense - pharmacology
Oligonucleotides, Antisense - therapeutic use
Online tutorials
Pharmaceutical Preparations
Pharmacodynamics
Prescription drugs
Proprotein Convertase 9 - genetics
Simulation
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Summary:Here, we show 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.
ISSN:2163-8306
2163-8306
DOI:10.1002/psp4.12866