Background: Molecular characterization is important to inform treatment decisions for endometrial cancer (EC). Wild type TP53 (TP53wt) is found in ≥50% of advanced or recurrent EC; of those cases, 60%–78% are also categorized as proficient mismatch repair (pMMR). Evidence of benefit for these molecular subgroups is limited for patients with TP53wt tumors.
Methods: This was a long-term follow-up analysis of the prespecified TP53wt exploratory subgroup of ENGOT-EN5/GOG-3055/SIENDO (NCT03555422), a randomized, double-blind, phase 3 trial evaluating selinexor versus placebo as maintenance treatment for advanced or recurrent EC following response to prior systemic therapy.
Results: A total of 113 patients with TP53wt EC were randomized to selinexor (n=77) or placebo (n=36) as maintenance therapy. Median follow-up was 36.8 months; 15 patients remained on treatment as of April 1, 2024. Median progression-free survival (mPFS) was 28.4 months with selinexor versus 5.2 months with placebo (HR: 0.44; 95% CI [0.27–0.73], nominal one-sided p=0.0005). PFS improvement was observed regardless of mismatch repair status; respective mPFS was 39.5 months versus 4.9 months in the TP53wt/proficient mismatch repair (pMMR) subgroup and 13.1 months versus 3.7 months in the TP53wt/deficient mismatch repair (dMMR) subgroup. Any-grade treatment-emergent adverse events (TEAEs) occurred in 99% and 94% of patients in the selinexor and placebo arms, respectively. The most common TEAEs were nausea (selinexor/placebo: 89%/40%), vomiting (61%/14%), and diarrhea (45%/37%); 17% of patients discontinued selinexor due to TEAEs. One death occurred in the placebo group.
Conclusions: TP53wt status may represent a robust predictive biomarker for selinexor efficacy in EC. A strong PFS signal was observed regardless of mismatch repair status, particularly in the TP53wt/pMMR subgroup, a patient population with high unmet need.