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Olutasidenib (FT-2102) in patients with relapsed or refractory IDH1-mutant glioma: A multicenter, open-label, phase
Macarena I de la Fuente1, Howard Colman2, Mark Rosenthal3
1Sylvester Comprehensive Cancer Center and Department of Neurology, University of Miami, Miami, Florida, USA.
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 IDH1R132X mutation.
Methods:
This was an open-label, multicenter, nonrandomized, phase Ib/II clinical trial. Eligible patients (≥18 years) had histologically confirmed IDH1R132X-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 IDH1R132X mutation and demonstrated preliminary evidence of clinical activity in this heavily pretreated population.
Insights
Olutasidenib showed good tolerability and preliminary clinical activity in patients with relapsed/refractory IDH1-mutated gliomas. This targeted therapy offers a new option for patients with this specific genetic mutation.
Area of Science:
- Neuro-oncology
- Molecular targeted therapy
- Clinical trial research
Background:
- Olutasidenib is a potent, orally bioavailable inhibitor of mutant isocitrate dehydrogenase 1 (IDH1).
- IDH1 mutations are implicated in the development of certain gliomas.
Purpose of the Study:
- To evaluate the safety and clinical activity of olutasidenib in patients with relapsed/refractory gliomas.
- To determine the recommended dose for further clinical trials.
Main Methods:
- An open-label, multicenter, phase Ib/II clinical trial was conducted.
- Patients received olutasidenib 150 mg orally twice daily.
- Safety and objective response rate were primary endpoints.
Main Results:
- Olutasidenib was well tolerated, with no dose-limiting toxicities observed.
- The disease control rate was 48%, with 8% partial responses and 32% stable disease for at least 4 months.
- Common Grade 3-4 adverse events included elevated liver enzymes.
Conclusions:
- Olutasidenib 150 mg BID is well tolerated in patients with relapsed/refractory IDH1-mutated gliomas.
- Preliminary clinical activity was observed in this heavily pretreated population.
