RATIONALE 304: A randomized phase 3 trial in nearly 332 patients with 1L, locally advanced or metastatic Non-squamous NSCLC
The safety and tolerability was consistent with the known profile of PD-1 inhibitors in combination with chemotherapy‡
| TEAEs, n (%) | etapidi + Chemoteraphy alone (n=222) | Chemotherapy alone (n=110) |
|---|---|---|
| Patients with ≥1 TEAE | 222 (100) | 109 (99.1) |
| Grade ≥3 TEAE | 150 (67.6) | 59 (53.6) |
| Serious TEAE | 74 (33.3) | 23 (20.9) |
| TEAE leading to death | 7 (3.2) | 2 (1.8) |
| TEAEs leading to discontinuation | 57 (25.7) | 10 (9.1) |
| TEAEs leading to dose modification or treatment delays | 149 (67.1) | 57 (51.8) |
|
Grade ≥3 TRAEs in which any grade of event occurred in ≥20% of patients, n (%) |
etapidi + Chemoteraphy (n=222) | Chemotherapy alone (n=110) |
| Anaemia¹ | 30 (13.5) | 11 (10.0) |
| Leukopenia¹ | 48 (21.6) | 16 (14.5) |
| Thrombocytopenia² | 43 (19.4) | 15 (13.6) |
| Nausea | 1 (0.5) | 1 (0.9) |
| Increased ALT | 8 (3.6) | 3 (2.7) |
| Increased AST | 4 (1.8) | 0 (0.0) |
| Neutropenia¹† | 99 (44.6) | 39 (35.5) |
| Fatigue†† | 3 (1.4) | 1 (0.9) |
| Decreased appetite | 3 (1.4) | 1 (0.9) |
| Vomiting | 1 (0.5) | 1 (0.9) |
Most reported AEs were mild or moderate in severity and were in line with the known toxicity profile
of chemotherapy in this patient population¶2
• Most immune-mediated AEs were grades 1 to 2 and did not lead to treatment discontinuation¶2
• No new safety signals were identified at the final analysis compared with the interim analysis§4
Reference: 1. etapidi. Prescribing Information. BeOne Medicines USA, Inc.; 2025.