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ARQ 197

Phase 2

Unresectable Hepatocellular Carcinoma | Small molecule | Oncology |Merck & Company, Inc.|Last Updated: Feb 28, 2013

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
RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment107
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT00988741Trial of ARQ 197 in Patients With Unresectable Hepatocellular Carcinoma (HCC) Who Have Failed One Prior Systemic TherapyPHASE2 COMPLETED 107Sep 1, 2009Mar 1, 2012Feb 28, 201323 United States, Belgium +3
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Study Endpoints
Primary Endpoints
Evaluate time to progression among all patients treated with ARQ 197 compared to placebo
Patients will be evaluated every 6 weeks until unacceptable toxicity, disease progression or another discontinuation criterion is met
Secondary Endpoints
Evaluate progression-free survival, overall survival, objective response rate and disease control rate among all patients treated with ARQ 197 compared to placebo.
Patients will be evaluated for these endpoints every 6 weeks
Evaluate objective response rate in crossover population following radiographic disease progression on placebo.
Patients will be evaluated for these endpoints every 6 weeks
Further characterize the safety of ARQ 197 in patients with unresectable HCC
While on therapy, patients will be evaluated for safety every 4 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ARQ 197EXPERIMENTAL -
placeboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ARQ 197DRUGThe investigational drug ARQ 197 is supplied as capsules. A dose of 360 mg (3 capsules of 120 mg each) of ARQ 197 will be administered by mouth BID, once in the morning and once in the evening with meals, for a total daily dose of 720 mg. Under Amendment 2, a dose of 240 mg (2 capsules of 120 mg each) of ARQ 197/placebo will be administered by mouth BID, once in the morning and once in the evening with meals, for a total daily dose of 480 mg. A treatment cycle is defined as 4 weeks for both treatment arms. Cycles will be repeated every 4 weeks (28 days) based on toxicity and response.
PlaceboDRUGThe placebo is provided in a capsule form.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites23

Inclusion Criteria: * Written informed consent granted prior to initiation of any study-specific screening procedures * 18 year of age or older * Histologically or cytologically confirmed HCC * Archival, fresh core needle biopsy or fine needle aspiration (FNA) tumor samples * Received at least one ...

Countries:United StatesBelgiumCanadaGermanyItaly
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