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ALN-BCAT

Phase 1

Advanced Hepatocellular Carcinoma | Small molecule | Oncology |Alnylam Pharmaceuticals, Inc.|Last Updated: Jun 17, 2026

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment158

FDA Designations

No designations recorded

Clinical trial landscape

ALN-BCAT · 1 trial · 2 indications

Phase 1 1
NCT06600321A Study to Evaluate ALN-BCAT in Patients With Hepatocellular CarcinomaAdvanced Hepatocellular Carcinoma
RECRUITING158 Analytics
PHASE1RECRUITING
A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma
Advanced Hepatocellular CarcinomaUnlock trial analytics

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
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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
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Eligibility Criteria

Age Range18 Years to 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
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Recent Changes (Last 90 Days)

LOWJun 18, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 18, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 18, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 18, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 2, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 2, 2026NCT06600321lastUpdatePostDate: changed
LOWJun 2, 2026NCT06600321lastUpdatePostDate: changed

Frequently asked questions about ALN-BCAT

What is ALN-BCAT used for?

ALN-BCAT is an investigational drug being developed for the treatment of advanced hepatocellular carcinoma, a type of liver cancer. It is also being studied in patients with metastatic hepatocellular carcinoma. The drug is currently in Phase 1 clinical development and has not yet been approved by regulatory authorities.

Who makes ALN-BCAT?

ALN-BCAT is being developed by Alnylam Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol ALNY. The company is conducting a Phase 1 clinical trial to evaluate the safety and efficacy of ALN-BCAT in patients with advanced hepatocellular carcinoma.

What phase is ALN-BCAT in?

ALN-BCAT is currently in Phase 1 clinical development. It is an investigational drug, meaning it has not been approved by the FDA or any other regulatory agency. The ongoing Phase 1 trial is actively recruiting participants to evaluate the drug's safety, tolerability, and preliminary efficacy in patients with advanced hepatocellular carcinoma.

What clinical trials is ALN-BCAT in?

ALN-BCAT is being evaluated in a Phase 1 clinical trial with the identifier NCT06600321, titled 'A Study to Evaluate ALN-BCAT in Patients With Hepatocellular Carcinoma.' This trial is currently recruiting participants and aims to enroll approximately 158 patients with advanced or metastatic hepatocellular carcinoma across the United States, Italy, and South Korea.

Is ALN-BCAT the same as any other drug?

ALN-BCAT is the primary name for this investigational drug candidate. No alternative names have been reported for this compound. It is being developed by Alnylam Pharmaceuticals and is currently in Phase 1 clinical trials for the treatment of advanced hepatocellular carcinoma.