2 research outputs found
A caseâstudy of modelâinformed drug development of a novel PCSK9 anti sense oligonucleotide. Part 1: First time in man to phase II
Abstract Here, we show modelâinformed 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 firstâinâhuman 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 deârisk a phase IIb study. The focus of the caseâstudy is on the crossâfunctional collaboration and other key MIDD enablers that are critical to maximize the value of MIDD, rather than the technical application of MIDD