A well-characterized safety profile, consistent with the PD-1–inhibitor class11
Adverse reactions (≥10%) in RATIONALE-306* | etapidi + chemotherapy (n=324) | Placebo + chemotherapy (n=321) | ||
| All grades % | Grade 3 or 4 % | All grades % | Grade 3 or 4 % | |
|---|---|---|---|---|
| Blood and lymphatic system disorders | ||||
| Anemia | 61 | 17 | 56 | 16 |
| Neutropenia | 16 | 7 | 15 | 10 |
| General disorders and administration site conditions | ||||
| Fatigue† | 45 | 9 | 45 | 4.7 |
| Metabolism and nutrition disorders | ||||
| Decreased appetite | 44 | 6 | 39 | 2.2 |
| Gastrointestinal disorders | ||||
| Diarrhea | 28 | 4.3 | 24 | 1.9 |
| Stomatitis† | 22 | 4 | 16 | 2.2 |
| Vomiting | 22 | 1.5 | 27 | 2.5 |
| Dysphagia | 14 | 6 | 11 | 4 |
| Skin and subcutaneous tissue disorders | ||||
| Rash | 19 | 4 | 9 | 0.3 |
| Pruritus | 13 | 0.3 | 7 | 0 |
| Endocrine disorders | ||||
| Hypothyroidism† | 11 | 0 | 6 | 0 |
Discontinuation1:
- Permanent discontinuation of ETAPIDI due to adverse reactions occurred in 13% of patients
- Adverse reaction that resulted in discontinuation in ≥2% of patients was pneumonitis
imAEs leading to discontinuation2:
- 4.9% of patients receiving ETAPIDI + chemotherapy vs 0.6% of patients receiving placebo + chemotherapy
- The most common (≥2 patients in either arm) imAEs leading to discontinuation of ETAPIDI or placebo included immune-mediated pneumonitis (8 patients [2.5%] in the ETAPIDI + chemotherapy arm vs 2 [0.6%] in the placebo + chemotherapy arm), immune-mediated skin adverse reaction (2 patients [0.6%] vs 0 patients [0.0%])
*With a difference between arms of ≥5% for all grades or ≥2% for grades 3 and 4.
†Represents a composite of multiple, related preferred terms.
1L, first line; 2L, second line; ESCC, esophageal squamous cell carcinoma; imAE, immune-mediated adverse event; PD-1, programmed death receptor 1; PD-L1, programmed death ligand 1; TEAE, treatment-emergent adverse event.
References: 1. etapidi. Prescribing Information. BeOne Medicines USA, Inc.; 2025. 2. Data on file. BeOne, Ltd.