Data from the following presentations are discussed:
OPTIC primary analysis: A dose-optimization study of 3 starting doses of ponatinib (PON). (Cortes, ASCO 2021 Abstract 7000)
Efficacy: In this phase 2 dose optimization trial, treatment or exposure to ponatinib suggested a correlation between dose level and rates of adverse events (AEs) and measurable treatment responses. An optimal benefit/risk ratio was noted at a dose of 45 mg/d (milligrams per day), with a further dose reduction to 15 mg/d when a ≤1% BCR-ABL1IS was achieved. This benefit was noted to be most pronounced in patients with a T315I mutation.
Safety: AEs and serious AEs were noted across all treatment cohorts, with the most commonly seen grades ≥3 TEAEs leading to treatment discontinuation being thrombocytopenia (27%), neutropenia (17%) and anemia (7%).