WCLC 2025 - Posters & ePosters-EP.18.03 Neoadjuvant Toripalimab for Stage Ii-Iiib Resectable NSCLC With EGFR Mutation and PD-L1 Positive Expression (TOPLINE)

EP.18.03 Neoadjuvant Toripalimab for Stage Ii-Iiib Resectable NSCLC With EGFR Mutation and PD-L1 Positive Expression (TOPLINE)

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Pdf Summary

The TOPLINE study is an open-label, multicenter, single-arm phase trial evaluating the efficacy and safety of neoadjuvant toripalimab, an anti–PD-1 immune checkpoint inhibitor, in patients with resectable stage II–IIIB non-squamous non-small cell lung cancer (NSCLC) harboring EGFR mutations and positive PD-L1 expression. The trial addresses the unmet need for optimal neoadjuvant treatment in this population, given that standard neoadjuvant chemotherapy or EGFR-TKIs have shown limited efficacy with approximately 10% major pathological response (MPR) rates, while chemoimmunotherapy combinations have demonstrated preliminary improved MPR (~40%) in EGFR-mutated, PD-L1 positive NSCLC.<br /><br />Eligible patients were adults with histologically confirmed primary NSCLC, clinical stages II to IIIB (except N3), EGFR mutation positive (including common, uncommon, or complex mutations), PD-L1 tumor proportion score (TPS) ≥1%, stratified into 1–49% and ≥50% groups. Patients had ECOG performance status 0–1 with no prior systemic therapy.<br /><br />The primary endpoint was MPR rate, with secondary endpoints including pathological complete response (pCR), objective response rate (ORR), two-year event-free survival (EFS), overall survival (OS), safety, and feasibility. The design employed a Simon’s two-stage approach.<br /><br />To date, two patients have completed neoadjuvant toripalimab followed by surgery—one with PD-L1 TPS 1% and one with 50%. Both experienced manageable treatment-related adverse events (hypothyroidism, diarrhea) without grade 3 or higher toxicities. However, neither patient achieved MPR. Further enrollment and data are needed to determine the efficacy and safety profile of toripalimab in this specific NSCLC subgroup.

Asset Subtitle

Yichao Han

Meta Tag

Speaker Yichao Han

Topic Clinical Trials in Progress

Keywords

TOPLINE study

neoadjuvant toripalimab

anti-PD-1 immune checkpoint inhibitor

resectable stage II–IIIB non-squamous NSCLC

EGFR mutations

PD-L1 positive expression

major pathological response (MPR)

pathological complete response (pCR)

Simon’s two-stage design

treatment-related adverse events