Table 1.
Patient demographics and baseline characteristics (Phase 1b and 2).
| Phase 1b | Phase 2 | |||
|---|---|---|---|---|
| 300 mg/day | 500 mg/day | Total | 500 mg/day | |
| (n = 4) | (n = 13) | (N = 17) | (N = 49) | |
| Age, years, median (range) | 65.5 (58−73) | 69.0 (51−79) | 69.0 (51−79) | 66.0 (19−82) |
| <65 years, n (%) | 1 (25.0) | 3 (23.1) | 4 (23.5) | 21 (42.9) |
| ≥65 years, n (%) | 3 (75.0) | 10 (76.9) | 13 (76.4) | 28 (57.1) |
| Sex, n (%) | ||||
| Male | 2 (50.0) | 11 (84.6) | 13 (76.5) | 41 (83.7) |
| Female | 2 (50.0) | 2 (15.4) | 4 (23.5) | 8 (16.3) |
| Race, n (%) | ||||
| White | 1 (25.0) | 8 (61.5) | 9 (52.9) | 26 (53.1) |
| Black | 0 (0.0) | 0 (0.0) | 0 (0.0) | 1 (2.0) |
| Asian | 0 (0.0) | 0 (0.0) | 0 (0.0) | 2 (4.1) |
| Other | 0 (0.0) | 1 (7.7) | 1 (5.9) | 0 (0.0) |
| Missing | 3 (75.0) | 4 (30.8) | 7 (41.2) | 20 (40.8) |
| ECOG PS at baseline, n (%) | ||||
| 0 | 3 (75.0) | 10 (76.9) | 13 (76.5) | 25 (51.0) |
| 1 | 1 (25.0) | 3 (23.1) | 4 (23.5) | 24 (49.0) |
| Cause of HCC (investigator-assessed), n (%) | ||||
| HBV | 4 (8.2) | |||
| HCV | 13 (26.5) | |||
| Alcohol-induced cirrhosis | 11 (22.4) | |||
| Non-alcoholic steatohepatitis | 2 (4.1) | |||
| Steatosis | 2 (4.1) | |||
| Othera | 16 (32.7) | |||
| Missing | 1 (2.0) | |||
| Vascular invasion, n (%) | ||||
| Yes | 1 (25.0) | 5 (38.5) | 6 (35.3) | 12 (24.5) |
| No | 0 (0.0) | 5 (38.5) | 5 (29.4) | 24 (49.0) |
| Missing | 3 (75.0) | 3 (23.1) | 6 (35.3) | 13 (26.5) |
| MET IHC, n (%)b | ||||
| IHC 0 | 0 (0.0) | 1 (7.7) | 1 (5.9) | |
| IHC 1+ | 1 (25.0) | 4 (30.8) | 5 (29.4) | |
| IHC 2+ | 3 (75.0) | 4 (30.8) | 7 (41.2) | 41 (83.7) |
| IHC 3+ | 0 (0.0) | 2 (15.4) | 2 (11.8) | 8 (16.3) |
| Missing | 0 (0.0) | 2 (15.4) | 2 (11.8) | 0 (0.0) |
| MET amplification, n (%)c | ||||
| Present | 0 (0.0) | 0 (0.0) | 0 (0.0) | 6 (12.2) |
| Absent | 4 (100.0) | 10 (76.9) | 14 (82.4) | 43 (87.8) |
| Missing | 0 (0.0) | 3 (23.1) | 3 (17.6) | 0 (0.0) |
| AFP elevation, n (%) | ||||
| <200 µg/L | 1 (25.0) | 9 (69.2) | 10 (58.8) | 20 (40.8) |
| ≥200 µg/L | 3 (75.0) | 3 (23.1) | 6 (35.3) | 27 (55.1) |
| Missing | 0 (0.0) | 1 (7.7) | 1 (5.9) | 2 (4.1) |
Please note, as numbers have been rounded to one decimal place, some columns may not total 100%.
AFP alpha-fetoprotein, ECOG PS Eastern Cooperative Oncology Group performance status, HBV hepatitis B virus, HCC hepatocellular carcinoma, HCV Hepatitis C virus, IHC immunohistochemistry.
aOther investigator-assessed root causes of HCC were: metabolic cirrhosis (n = 1), adenomatous hyperplasia (n = 1), hemochromatosis (n = 1), regenerative nodular hyperplasia (n = 1), previous drug abuse (n = 1); cirrhosis (n = 1); both alcohol-induced cirrhosis and HCV (n = 1) and idiopathic/unknown/none/not assessable (n = 9).
bIn Phase 1b, patients were not required to have MET overexpression.
cMET amplification is defined as MET:CEP7 ratio ≥2 or mean gene copy number ≥5.