TRAEs occurring at any grade in ≥5% of patients or grade ≥3 in any patient and any AEs of special interest (AESIs) from the full analysis set
| Study 001 (n=43) | Study 012 (n=16) | Full analysis set (N=59) | ||||
| Any grade | Grade 3 | Any grade | Grade 3 | Any grade | Grade 3 | |
| Patients with any TRAE | 35 (81.4) | 11 (25.6) | 14 (87.5) | 5 (31.3) | 49 (83.1) | 16 (27.1) |
| Pruritus | 10 (23.3) | 0 | 5 (31.3) | 0 | 15 (25.4) | 0 |
| Dermatitis acneiform | 7 (16.3) | 0 | 5 (31.3) | 0 | 12 (20.3) | 0 |
| Keratoacanthoma | 9 (20.9) | 2 (4.7) | 0 | 0 | 9 (15.3) | 2 (3.4) |
| Hypothyroidism | 7 (16.3) | 1 (2.3) | 2 (12.5) | 0 | 9 (15.3) | 1 (1.7) |
| Rash maculopapular | 6 (14.0) | 0 | 3 (18.8) | 0 | 9 (15.3) | 0 |
| Anemia | 4 (9.3) | 1 (2.3) | 5 (31.3) | 3 (18.8) | 9 (15.3) | 4 (6.8) |
| Fatigue | 2 (4.7) | 0 | 5 (31.3) | 1 (6.3) | 7 (11.9) | 1 (1.7) |
| Stomatitis | 3 (7.0) | 0 | 2 (12.5) | 0 | 5 (8.5) | 0 |
| Rash macular | 3 (7.0) | 1 (2.3) | 0 | 0 | 3 (5.1) | 1 (1.7) |
| Alanine aminotransferase increased | 2 (4.7) | 0 | 1 (6.3) | 0 | 3 (5.1) | 0 |
| Aspartate aminotransferase increased | 2 (4.7) | 0 | 1 (6.3) | 0 | 3 (5.1) | 0 |
| Asthenia | 3 (7.0) | 0 | 0 | 0 | 3 (5.1) | 0 |
| Diarrhea | 2 (4.7) | 0 | 1 (6.3) | 0 | 3 (5.1) | 0 |
| Epistaxis | 2 (4.7) | 0 | 1 (6.3) | 0 | 3 (5.1) | 0 |
| Decreased appetite | 3 (7.0) | 0 | 0 | 0 | 3 (5.1) | 0 |
| Influenza-like illness | 1 (2.3) | 0 | 2 (12.5) | 0 | 3 (5.1) | 0 |
| Infusion-related reaction | 2 (4.7) | 0 | 1 (6.3) | 0 | 3 (5.1) | 0 |
| Mouth hemorrhage (mucosal bleeding) | 0 | 0 | 3 (18.8) | 0 | 3 (5.1) | 0 |
| Nausea | 3 (7.0) | 0 | 0 | 0 | 3 (5.1) | 0 |
| Colitis | 1 (2.3) | 1 (2.3) | 1 (6.3) | 0 | 2 (3.4) | 1 (1.7) |
| Pneumonitis | 2 (4.7) | 1 (2.3) | 0 | 0 | 2 (3.4) | 1 (1.7) |
| Hypokalemia | 1 (2.3) | 1 (2.3)* | 0 | 0 | 1 (1.7) | 1 (1.7)* |
| Squamous cell carcinoma of skin | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| γ-glutamyltransferase increased | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| Diabetic ketoacidosis | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| Neutrophil count decreased | 0 | 0 | 1 (6.3) | 1 (6.3) | 1 (1.7) | 1 (1.7) |
| Hyperglycemia | 0 | 0 | 1 (6.3) | 1 (6.3) | 1 (1.7) | 1 (1.7) |
| Cystitis non-infective | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| Impaired gastric emptying | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| Pleural effusion | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| Upper gastrointestinal hemorrhage | 0 | 0 | 1 (6.3) | 1 (6.3) | 1 (1.7) | 1 (1.7) |
| Hyperkeratosis follicularis et parafollicularis | 1 (2.3) | 1 (2.3) | 0 | 0 | 1 (1.7) | 1 (1.7) |
| Any AESIs | ||||||
| Skin lesions† | 12 (27.9) | 4 (9.3) | 0 | 0 | 12 (20.3) | 4 (6.8) |
Data are n (%) of the safety set.
*Grade 3 hypokalemia progressed to grade 4.
†Includes MedDRA V.2.0.0 and 21.1 preferred terms squamous cell carcinoma of skin, basal cell carcinoma, keratoacanthoma, hyperkeratosis, actinic keratosis, lip squamous cell carcinoma, and Bowen’s disease. Not included in the table were five patients (8%) in study 012 who were noted by the MedDRA System Organ Class of Neoplasms benign, malignant, and unspecified (including cysts and polyps), but the MedDRA preferred term was not captured (although it was deemed to be related to keratoacanthoma).
AESI, adverse event of special interest; MedDRA, Medical Dictionary for Regulatory Activities; SCC, squamous cell carcinoma; TRAE, treatment-related adverse event.