Recent Updates
Recently added Catalysts

ALN-BCAT

Phase 1

Advanced Hepatocellular Carcinoma | Small molecule | Oncology |Alnylam Pharmaceuticals, Inc.|Last Updated: Jun 1, 2026

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
Premium
Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
Premium
Trial Design
CONTROLLEDBiomarker
Total Trials1
Total Enrollment158
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06600321A Study to Evaluate ALN-BCAT in Patients With Hepatocellular CarcinomaPHASE1 RECRUITING 158 — — Dec 30, 2024Oct 31, 2027Jun 1, 202623 United States, Italy +1
Unlock Drug Trial Details
Study Endpoints
Primary Endpoints
Frequency of Adverse Events (AEs)
From the time of first dose of study drug administration to 30-37 days after the last dose
Severity of AEs
From the time of first dose of study drug administration to 30-37 days after the last dose
Dose Escalation: Occurrence of Dose-limiting Toxicities (DLTs)
From the time of first dose of study drug administration up to 21 days
Dose Expansion: Antitumor Activity as assessed by Response Evaluation Criteria in Solid Tumors Version 1.1 (RECIST v1.1)
Up to 30-37 Days after the last dose
Secondary Endpoints
Concentrations of ALN-BCAT in Plasma
Up to the end of the last study drug administration
Percent Change in Gene that Encodes ß-catenin Protein (CTNNB1) Messenger Ribonucleic Acid (mRNA) Expression Comparing Pre- treatment with On-treatment Tumor Samples
Up to 30 days
Dose Escalation: Antitumor Activity as assessed by RECIST v1.1
Up to 30-37 Days after the last dose
Unlock Study Endpoints
Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Monotherapy: Dose EscalationEXPERIMENTALPatients will be administered multiple doses of ALN-BCAT.
Monotherapy: Dose ExpansionEXPERIMENTALPatients will be administered multiple doses of ALN-BCAT.
Combination Therapy: Dose EscalationEXPERIMENTALPatients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.
Combination Therapy: Dose ExpansionEXPERIMENTALPatients will be administered multiple doses of ALN-BCAT in combination with pembrolizumab.
Interventions
NameTypeDescription
ALN-BCATDRUGAdministered by intravenous (IV) infusion
PembrolizumabDRUGAdministered by intravenous (IV) infusion
Unlock Study Design Details
Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites23

Inclusion Criteria: * Has HCC confirmed histologically or cytologically, or, for patients with liver cirrhosis, clinically by the American Association for the Study of Liver Diseases (AASLD) criteria * Has had at least one line of systemic therapy for unresectable advanced or metastatic disease * H...

Countries:United StatesItalySouth Korea
Unlock Eligibility Criteria
Recent Changes (Last 90 Days)
LOWJun 2, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 2, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 2, 2026NCT06600321lastUpdatePostDate: changed
LOWMay 26, 2026NCT06600321primaryCompletionDate: changed
LOWMay 24, 2026NCT06600321studyFirstPostDate: changed