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ABC294640

Phase 2

Cholangiocarcinoma | Small molecule | Oncology |Redhill Biopharma Ltd.|Last Updated: Jul 23, 2025

Success Probability

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment65

FDA Designations

No designations recorded

Clinical trial landscape

ABC294640 · 1 trial · 5 indications

Phase 2 1
NCT03377179A Study of ABC294640 Alone and in Combination With HCQ Sulfate in the Treatment of Advanced CholangiocarcinomaCholangiocarcinoma
COMPLETED65 Analytics
PHASE2COMPLETED
A Study of ABC294640 Alone and in Combination With HCQ Sulfate in the Treatment of Advanced Cholangiocarcinoma
CholangiocarcinomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Part1 - Determine Response Rate
At least 4 months

To determine the response rate (RR) of CCA defined as objective responses (OR), i.e. complete and partial responses (CR, PR) plus stable disease (SD) of at least 4 months to treatment with ABC294640.

Part 2 - Determine the Durable Disease Control Rate
At least 4 months

To determine the Durable Disease Control Rate (DDCR) of CCA defined as Disease Control Rate (DCR) of at least 4 months duration to treatment with ABC294640 and HCQ

Secondary Endpoints

Physical exam to include eye exams (the latter only for patients enrolled in Part 2) to measure safety and tolerability of ABC294640 alone and in combination with HCQ
From screening phase, during beginning of each cycle of treatment, till 30 days after the end of treatment (an estimated median of 5 months)
A general neurological exam to measure safety and tolerability of ABC294640 alone and in combination with HCQ
From screening phase, during beginning of each cycle of treatment, till 30 days after the end of treatment (an estimated median of 5 months)
HADS score for depression and anxiety to measure safety and tolerability of ABC294640 alone and in combination with HCQ
From screening, during each cycle of treatment, till the end of treatment (an estimated median of 4 months). Patient diaries will be filled on a daily basis during treatment.
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Study Design & Arms

AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
ABC294640 +/- HCQ treatmentEXPERIMENTALPart 1: All participants will be receiving ABC294640, 500 mg twice a day (BID), continuously in 28 day cycles Part 2: All participants will be receiving ABC294640, 500 mg twice a day (BID) and HCQ at a determined level, continuously in 28 day cycles

Interventions

NameTypeDescription
ABC294640DRUGTwo 250 mg capsules of ABC294640 will be taken twice daily
Hydroxychloroquine Sulfate 200 MGDRUGHCQ tablets will be taken at a dose that will be determined
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites5

Inclusion Criteria: 1. Patients with histologically confirmed intrahepatic, perihilar or extra-hepatic CCA. 2. Patients with no more than 2 prior treatments with systemic anti-neoplastic therapy for CCA. 3. The tumor is unresectable and not amenable to curative therapy. 4. One or more tumors measur...

Countries:United States
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Competitive Landscape -Cholangiocarcinoma 58 trials

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Frequently asked questions about ABC294640

What is ABC294640 used for in cholangiocarcinoma?

ABC294640 is an investigational small molecule being studied for the treatment of advanced cholangiocarcinoma, including non-resectable, perihilar, extrahepatic, and intrahepatic forms of the disease. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does ABC294640 target?

ABC294640 is a small molecule that targets sphingosine kinase 2, an enzyme involved in the production of sphingosine-1-phosphate, which plays a role in cell growth and survival. By inhibiting this enzyme, ABC294640 aims to reduce tumor growth in cholangiocarcinoma.

Who makes ABC294640?

ABC294640 is being developed by Redhill Biopharma Ltd., a biopharmaceutical company. The company is conducting clinical trials to evaluate the drug's safety and efficacy in patients with advanced cholangiocarcinoma.

What phase is ABC294640 in?

ABC294640 is in Phase 2 clinical development for the treatment of advanced cholangiocarcinoma. It is an investigational drug, meaning it has not yet been approved by the FDA or other regulatory agencies for commercial use.

What clinical trials is ABC294640 in?

ABC294640 has been studied in a Phase 2 clinical trial with the identifier NCT03377179, titled 'A Study of ABC294640 Alone and in Combination With HCQ Sulfate in the Treatment of Advanced Cholangiocarcinoma.' The trial enrolled 65 participants in the United States and has been completed.

Is ABC294640 the same as opaganib?

ABC294640 is also known as opaganib. It is the same drug, and the name opaganib is used interchangeably in some contexts. The drug is being developed by Redhill Biopharma for the treatment of cholangiocarcinoma.