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

Autor: Jane Knöchel, Catarina Nilsson, Björn Carlsson, Linda Wernevik, Alexis Hofherr, Peter Gennemark, Rasmus Jansson‐Löfmark, Rikard Isaksson, Tina Rydén‐Bergsten, Bengt Hamrén, Dinko Rekić
Jazyk: angličtina
Rok vydání: 2022
Předmět:
Zdroj: CPT: Pharmacometrics & Systems Pharmacology, Vol 11, Iss 12, Pp 1569-1577 (2022)
Druh dokumentu: article
ISSN: 2163-8306
DOI: 10.1002/psp4.12866
Popis: Abstract 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.
Databáze: Directory of Open Access Journals
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