TEAE occurring in two or more patients at any grade and SAE occurring in one or more patients (safety analysis set)
| AE, n (%) | Total (N=15) | 100 mg two times per day (n=3) | 200 mg two times per day (n=3) | 400 mg two times per day (n=3) | 600 mg two times per day (n=3) | 800 mg two times per day (n=3) |
| Any AE | 15 (100) | 3 (100) | 3 (100) | 3 (100) | 3 (100) | 3 (100) |
| Any SAE | 3 (20.0) | 0 | 0 | 1 (33.3) | 1 (33.3) | 1 (33.3) |
| Fatigue | 5 (33.3) | 2 (66.7) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 1 (33.3) |
| Nausea | 4 (26.7) | 1 (33.3) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 1 (33.3) |
| Vomiting | 4 (26.7) | 2 (66.7) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) |
| Blood creatine phosphokinase increased | 3 (20.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) | 1 (33.3) |
| Blood creatinine increased | 3 (20.0) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 1 (33.3) |
| Cough | 3 (20.0) | 1 (33.3) | 1 (33.3) | 1 (33.3) | 0 (0.0) | 0 (0.0) |
| Decreased appetite | 3 (20.0) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 1 (33.3) |
| Diarrhea | 3 (20.0) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 2 (66.7) | 0 (0.0) |
| Lipase increased | 3 (20.0) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 2 (66.7) | 0 (0.0) |
| Abdominal distension | 2 (13.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) |
| Abdominal pain | 2 (13.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) |
| Alanine aminotransferase increased | 2 (13.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 1 (33.3) |
| Amylase increased | 2 (13.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) |
| Anemia of malignant disease | 2 (13.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) |
| Aspartate aminotransferase increased | 2 (13.3) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 2 (66.7) |
| Blood alkaline phosphatase increased | 2 (13.3) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) |
| Dehydration | 2 (13.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) |
| Dyspepsia | 2 (13.3) | 2 (66.7) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 0 (0.0) |
| Dysphagia | 2 (13.3) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 0 (0.0) |
| Gamma-glutamyl transferase increased | 2 (13.3) | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) |
| Hypercalcemia | 2 (13.3) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) | 0 (0.0) |
| Lymphocyte count decreased | 2 (13.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) |
| Palmar-plantar erythrodysesthesia syndrome | 2 (13.3) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 0 (0.0) |
| Platelet count decreased | 2 (13.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) |
| Fever | 2 (13.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) |
| Maculopapular rash* | 2 (13.3) | 1 (33.3) | 0 (0.0) | 1 (33.3) | 0 (0.0) | 0 (0.0) |
| Tumor pain | 2 (13.3) | 0 (0.0) | 0 (0.0) | 1 (33.3) | 1 (33.3) | 0 (0.0) |
All TEAEs were grade 3 or lower. Grade 3 TEAEs were experienced by a total of 6 patients within the 400, 600, and 800 mg M4112 two times per day cohorts.
*MedDRA preferred term: rash maculopapular.
AE, adverse event; MedDRA, Medical Dictionary for Regulatory Activities; SAE, serious adverse event; TEAE, treatment-emergent adverse event.