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Olutasidenib (FT-2102) in patients with relapsed or refractory IDH1-mutant glioma: A multicenter, open-label, phase Ib/II trial
Journal article   Open access   Peer reviewed

Olutasidenib (FT-2102) in patients with relapsed or refractory IDH1-mutant glioma: A multicenter, open-label, phase Ib/II trial

Macarena de la Fuente, Howard Colman, Mark Rosenthal, Brian A. Van Tine, Danijela Levacic, Tobias Walbert, Hui K. Gan, Maria Vieito, Mohammed M. Milhem, Kathryn Lipford, …
Neuro-oncology (Charlottesville, Va.), Vol.25(1), pp.146-156
05/25/2022
DOI: 10.1093/neuonc/noac139
PMCID: PMC9825299
PMID: 35639513
url
https://doi.org/10.1093/neuonc/noac139View
Published (Version of record) Open Access

Abstract

Background Olutasidenib (FT-2102) is a highly potent, orally bioavailable, brain-penetrant and selective inhibitor of mutant isocitrate dehydrogenase 1 (IDH1). The aim of the study was to determine the safety and clinical activity of olutasidenib in patients with relapsed/refractory gliomas harboring an IDH1(R132X) mutation. Methods This was an open-label, multicenter, nonrandomized, phase Ib/II clinical trial. Eligible patients (>= 18 years) had histologically confirmed IDH1(R132X)-mutated glioma that relapsed or progressed on or following standard therapy and had measurable disease. Patients received olutasidenib, 150 mg orally twice daily (BID) in continuous 28-day cycles. The primary endpoints were dose-limiting toxicities (DLTs) (cycle 1) and safety in phase I and objective response rate using the Modified Response Assessment in Neuro-Oncology criteria in phase II. Results Twenty-six patients were enrolled and followed for a median 15.1 months (7.3-19.4). No DLTs were observed in the single-agent glioma cohort and the pharmacokinetic relationship supported olutasidenib 150 mg BID as the recommended phase II dose. In the response-evaluable population, disease control rate (objective response plus stable disease) was 48%. Two (8%) patients demonstrated a best response of partial response and eight (32%) had stable disease for at least 4 months. Grade 3-4 adverse events (>= 10%) included alanine aminotransferase increased and aspartate aminotransferase increased (three [12%], each). Conclusions Olutasidenib 150 mg BID was well tolerated in patients with relapsed/refractory gliomas harboring an IDH1(R132X) mutation and demonstrated preliminary evidence of clinical activity in this heavily pretreated population.
Clinical Neurology Life Sciences & Biomedicine Neurosciences & Neurology Oncology Science & Technology

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