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Review
. 2023 Aug 22;7(16):4358-4365.
doi: 10.1182/bloodadvances.2023009854.

Olutasidenib: from bench to bedside

Sangeetha Venugopal  1 , Justin Watts  1
Affiliations
Review

Olutasidenib: from bench to bedside

Sangeetha Venugopal et al. Blood Adv. .

Abstract

The discovery of isocitrate dehydrogenase 1 (IDH1) mutations in acute myeloid leukemia (AML) and the resounding success of molecularly targeted therapies in related myeloid malignancies swiftly prompted the development of IDH1mut inhibitors. Olutasidenib (formerly known as FT-2102) is an orally administered novel IDH1mut inhibitor that entered clinical development in 2016, proceeded briskly through the developmental process, and was granted regular approval to treat patients with R/R IDH1mut AML on 1 December 2022. Single agent olutasidenib, a potent and selective IDH1mut inhibitor, demonstrated highly durable remission rates along with meaningful outcomes, such as transfusion independence, in patients with R/R IDH1mut AML. This review will examine the preclinical and clinical development and the positioning of olutasidenib in the IDH1mut AML treatment landscape.

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Conflict of interest statement

Conflict-of-interest disclosure: J.W. is an advisory board member for Takeda, Bristol Myers Squibb, Servier, Rigel, and Daiichi Sankyo; serves on the DMC for Rafael Pharma and Reven Pharma; and has received research funding from Takeda and Immune Systems Key, Ltd. outside the submitted work. S.V. declares no competing financial interests.

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