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Fisogatinib

Phase 1

Hepatocellular Carcinoma (HCC) | Small molecule | Oncology |Blueprint Medicines Corporation|Last Updated: Apr 12, 2024

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
UNCONTROLLEDBiomarker
Total Trials1
Total Enrollment146
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02508467A Phase 1 Study of Fisogatinib (BLU-554) in Patients With Hepatocellular CarcinomaPHASE1 COMPLETED 146 — — Jul 31, 2015Feb 28, 2024Apr 12, 202441 United States, China +10
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Study Endpoints
Primary Endpoints
Maximum tolerated dose (MTD) on qd and bid schedules
During cycle 1 (28 days) of treatment and will be determined by approximately 24 months after start of the study or earlier
Recommended Phase 2 dose of fisogatinib (BLU-554) on qd and bid schedules
At the end of every cycle (28 days) of treatment and will be determined by approximately 24 months after start of the study or earlier
Number of patients with adverse events, serious adverse events and changes in physical findings, vital signs, clinical laboratory results and ECG findings
Every cycle (28 days) for approximately 24 months or earlier if patient terminates from the study
Secondary Endpoints
Maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules
Every cycle (28 days) up to cycle 4 and at end of treatment (approximately 24 months or earlier if patient terminates from the study)
Time to maximum plasma concentration of fisogatinib (BLU-554) on qd and bid schedules
Every cycle (28 days) up to cycle 4 and at end of treatment (approximately 24 months or earlier if patient terminates from the study)
Fibroblast growth factor 19 (FGF19) status in tumor tissue
Cycle 2 (Day 56)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Fisogatinib (BLU-554)EXPERIMENTALFisogatinib (BLU-554) capsules for oral administration.
Interventions
NameTypeDescription
Fisogatinib (BLU-554)DRUG -
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites41

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 patie...

Countries:United StatesChinaFranceGermanyHong KongItalySingaporeSouth KoreaSpainSwitzerlandTaiwanUnited Kingdom
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