Table 6. Summary of observed responses (CR + PR)a.
| Agent | Tumor type | Mutation | Prior lines of therapy, n | Best response | Treatment duration, weeks | Response confirmed (Yes/No)b | Age, years |
|---|---|---|---|---|---|---|---|
| BGJ398 | Ovarian | FGF23 and FGF6 ligand amplifications | 8 | PRc | 9.9 | No | 63 |
| BGJ398 | HNSCC | FGF19, FGF4, FGF23, FGF3, and FGF6 ligand amplifications | 3 | PR | 63 | Yes | 49 |
| BGJ398 | HNSCC | FGFR3 mutation; FGF3, FGF4, and FGF19 ligand amplifications | Unknown | PR | 16 | Yes | 59 |
| BGJ398 | Tumor-induced osteomalaciad | FGFR1 translocation | 0e | PR | 77.9 | Yes | 62 |
| BGJ398 | HNSCC | FGF3, FGF4, and FGF19 ligand amplifications | 4 | PR | 21.6 | No | 71 |
| BGJ398 | CNS | FGFR3 amplification; FGFR3 fusion | 1 | PR | 71.9 | Yes | 45 |
| BGJ398 | Ovarian | FGFR1 amplification | 4 | PRc | 6.3 | No | 73 |
| BGJ398 | NSCLC (squamous) | FGFR1 mutation | 4 | PR | 30.4 | Yes | 72 |
| BGJ398 | Bladder | FGFR3 mutation | 3 | PRc | 12.9 | No | 80 |
| BGJ398 | HNSCC | FGF3, FGF4, and FGF19 ligand amplifications | 1 | PRc | 15 | No | 56 |
| BGJ398 | NSCLC (squamous) | FGFR2 mutation | 2 | PR | 96.6f | Yes | 74 |
| LDK378 | Lung, non-small cell adenocarcinoma | ROS1 rearrangement | 4 | PR | 29.3 | Yes | 81 |
| LDK378 | Lymphoma | ALK mutation | 3 | PR | 102.7f | Yes | 22 |
| LDK378 | Lung, non-small cell adenocarcinoma | ROS1 rearrangement | 3 | PR | 23.9 | Yes | 58 |
| BKM120 | Cervical | PTEN loss (by IHC) | 4 | PR | 24 | No | 57 |
| BKM120 | Vaginal | PIK3CA | 1 | CR | 7.6 | Yes | 68 |
| BKM120 | HNSCC | PIK3CA and PIK3CA amplification | 1 | PR | 32.3 | Yes | 65 |
| TKI258 | Ovarian | FGFR2 | 5 | PR | 32.7 | No | 74 |
| TKI258 | GIST | cKit | 3 | PR | 33.1 | Yes | 49 |
| MEK162 | AML | NRAS | 1 | CR | 20 | NA | 68 |
| MEK162 | Ovarian | KRAS (KRAS amplification and KRAS G12D mutation) | 5 | PR | 44.3 | Yes | 78 |
| MEK162 | Ovarian | KRAS | 3 | PR | 15.9 | No | 45 |
| MEK162 | Thyroid | NRAS | 1 | PR | 36 | Yes | 75 |
| MEK162 | Uterine | KRAS (G12V) | 7 | PR | 21.6 | Yes | 59 |
| MEK162 | Myeloma | KRAS | 2 | Very good PR | 16.6 | NA | 59 |
| LEE011 | Bladder | CCND1 amplification | 2 | PR | 43 | Yes | 53 |
| LEE011 | Ovarian | CDK6 mutation | 2 | PR | 38.1 | No | 66 |
| LEE011 | Sarcoma | CDK4 amplification | 1 | PR | 53.7 | Yes | 25 |
| LEE011 | Unknown primary | CDK6 amplification | 1 | PR | 153.1f | Yes | 59 |
Abbreviations: ALK, anaplastic lymphoma kinase; AML, acute myeloid leukemia; CCND, cyclin D; CDK, cyclic-dependent kinase; CNS, central nervous system; CR, complete response; FGF, fibroblast growth factor; FGFR, fibroblast growth factor receptor; GIST, gastrointestinal stromal tumor; HNSCC, head and neck squamous cell cancer; IHC, immunohistochemistry; KRAS, Kirsten rat sarcoma viral oncogene homolog; NA, not applicable; NRAS, neuroblastoma RAS viral oncogene homolog; NSCLC, non-small cell lung cancer; PIK3CA, phosphatidylinositol 4,5-bisphosphate 3-kinase catalytic subunit α; PR, partial response; PTEN, phosphate and tensin homolog.
aAll patients experiencing a CR or PR at any time are included. Note that not all responses were confirmed responses by Response Evaluation Criteria In Solid Tumors or other related criteria.
bUnconfirmed response; patients may not have had a confirmatory scan due to protocol deviation or withdrawal from the trial before the confirmatory scan.
cResponse not > 16 weeks.
dAs defined by protocol.
ePatient had not been treated with prior chemotherapy/medication, but had 1 prior radiotherapy and surgery.
fPatient was still on study at time of data cutoff.