A Phase 1 Study of BLU-554 in Patients With Hepatocellular Carcinoma

Key Inclusion Criteria:

- Confirmed diagnosis of HCC by histological examination or by non-invasive criteria
according to European Association for the Study of the Liver (EASL) or American
Association for the Study of Liver Disease (AASLD) guidelines (Part 1, 2 and 3).

- For Part 1 and 2, the patient has unresectable disease and has been previously treated
with sorafenib, has declined treatment with sorafenib, or does not have access to
sorafenib.

- For Part 3, the patient has not received prior treatment with a TKI.

- Child-Pugh class A with no clinically apparent ascites

- Eastern Cooperative Oncology Group (ECOG) performance status of 0-1

- For Part 1, willing to provide archived tumor tissue (if available) and willing to
undergo pre- and on-treatment tumor biopsy (if considered safe and medically feasible
by the treating investigator)

- For Part 2 and 3, all patients must have an FGF19 IHC result available. Only FGF19
IHC+ HCC patients will be eligible for Part 3.

Key Exclusion Criteria:

- Central nervous system metastases

- Platelet count <75,000/mL

- Absolute neutrophil count <1000/mL

- Hemoglobin <8 g/dL

- Aspartate aminotransferase (AST) or alanine aminotransferase (ALT) >5x the upper limit
of normal (ULN)

- Total bilirubin >2.5 mg/dL

- International normalized ratio (INR) >2.3 or prothrombin time (PT) >6 seconds above
control

- Estimated (Cockroft-Gault formula) or measured creatinine clearance <40 mL/min