TABLE 1.
Small molecules targeting Wnt signaling and their clinical trials.
| Compound | Tumor type | Phase (Clinicaltrials.gov identifier) | Efficacy outcomes | Status | References |
|---|---|---|---|---|---|
| PRI‐724 with gemcitabine | Advanced metastatic pancreatic cancer | Phase Ib (NCT01764477) | 8 patients had SD (40.0%) | Clinical studies ongoing | 359 |
| DKN‐01 | Advanced‐stage DKK1‐positive esophageal cancer or gastroesophageal junction tumors | Phase Ib study (NCT02013154) | Encouraging early efficacy signals (no further details reported) | Clinical studies ongoing | 360 |
| Ipafricept | Advanced‐stage solid tumors | Phase I (NCT01608867) | 3 patients had SD for more than 6 months (1 with germ cell cancer and 2 with desmoid tumor) | Clinical studies ongoing | 361 |
| Vantictumab | Advanced solid tumors | Phase I | SD in 3 patients with NET | Clinical studies ongoing | 362 |
| Vantictumab | Stage IV pancreatic ductal adenocarcinoma | Phase I (NCT02005315) | 53% evaluable patients (8 out of 15) had a PR and 27% had SD (4); Median PFS: 7.2 months | Clinical studies ongoing | 363 |
| Vantictumab | Metastatic HER2‐negative breast cancer | Phase Ib (NCT01973309) | 33% evaluable patients (7 of 21) had a PR and 29% (6) had SD | Updated results pending | 364 |
| Cirmtuzumab | Chronic lymphocytic leukemia | Phase I (NCT02222688) | 17 of 22 evaluable patients had SD | Clinical studies ongoing | 365 |
| OTSA101 | Synovial sarcoma | Phase I (NCT01469975) | 77% evaluable patients (3 of 8) had SD | Recruiting | 343 |
| CWP232291 alone or with lenalidomide and dexamethasone | Myeloma (relapsed or refractory) | Phase Ia/b (NCT02426723) | 27% patients (3 of 11) had SD | Clinical studies ongoing | 366 |
| CWP232291 (CWP291) | Relapsed and/or refractory AML or myelodysplastic syndrome | Phase I (NCT01398462) | 1 CR in a patient with AML | Clinical studies ongoing | 353 |
Abbreviations: AML, acute myeloid leukemia; CR, complete response; DKK1, Dickkopf‐related protein 1; Fzd, Frizzled; n, number of patients; NET, neuroendocrine tumor; PFS, progression‐free survival; PR, partial response; SD, stable disease.