Baseline characteristics
| Dose escalation (n = 26) | GIST expansion (n = 9) | |
|---|---|---|
| Age | 53.3 years (range: 23–71) | 61.6 years (range 45–75) |
| Sex | ||
| Female | 10 (38.5%) | 2 (22.2%) |
| Male | 16 (61.5%) | 7 (77.8%) |
| ECOG PS | ||
| 0 | 4 | 0 |
| 1 | 19 | 9 |
| 2 | 3 | 0 |
| Malignancy | ||
| Melanoma | 8 | 0 |
| RCC (clear cell) | 5 | 0 |
| NSCLC | 3 | 0 |
| GIST | 3 | 9 |
| Other | 7a | 0 |
| Prior treatment regimens (avg, range) | 4 (0–11) | 4 (1–6) |
| Prior anti-CTLA4 | 1 | 0 |
| Prior imatinib | 3 | 9 |
| Prior regorafenibb | -- | 8 |
| Prior sunitinibb | -- | 7 |
| Prior nilotinibb | -- | 3 |
| Prior sorafenibb | -- | 1 |
| Molecular profile | ||
| KIT | 8 | 9 |
| Exon 5 | 1 | 0 |
| Exon 9 | 1c | 0 |
| Exon 10 | 4 | 0 |
| Exon 11 | 2 | 8c |
| Exon 13 | 0 | 2c |
| Exon 17 | 1c | 2c |
| NRAS | 3 | 1 |
| TP53 | 3 | 0 |
| KDR | 2 | 0 |
| MET | 2 | 0 |
| PIK3CA | 2 | 0 |
| KRAS | 1 | 0 |
ECOG PS Eastern cooperative group performance status, GIST gastrointestinal stromal tumor, RCC renal cell carcinoma, NSCLC non-small cell lung cancer
aProstate, Mesothelioma, Osteosarcoma, Germ-cell tumor, Salivary duct, Renal medullary, Anal
bPrior TKI exposure reported for GIST expansion only
cRepresent patients with co-mutations