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MDX2001

Phase 1

Biliary Tract Cancer | Small molecule | Oncology |Opko Health Inc.|Last Updated: May 6, 2026

Target and mechanism

Molecular targetTrop2
Target classProtein
ModalitySmall molecule

Success Probability

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Market & Valuation

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Trial Design

RandomizedCONTROLLEDDMC
Total Trials1
Total Enrollment285

FDA Designations

No designations recorded

Clinical trial landscape

MDX2001 · 1 trial · 16 indications

Phase 1 1
NCT06239194Dose Escalation and Dose Expansion Study of MDX2001 in Patients With Advanced Solid TumorsBiliary Tract Cancer
RECRUITING285 Analytics
PHASE1RECRUITING
Dose Escalation and Dose Expansion Study of MDX2001 in Patients With Advanced Solid Tumors
Biliary Tract CancerUnlock trial analytics

Study Endpoints

Primary Endpoints

All Phases: Adverse events (AEs)
Baseline until end of study, up to approximately 9 months

Incidence and severity of AEs and serious AEs (SAEs) graded according to the National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5.0 including changes in clinical laboratory parameters

Phase 1b and Phase 2a: Objective response rate of MDX2001
From date of enrollment until the end of treatment, up to approximately 6 months

Objective response rate is defined as the proportion of patients who achieve a complete response (CR) or partial response (PR) per Response Evaluation Criteria in Solid Tumors (RECIST) v1.1.

Phase 1: Recommended Phase 2 dose (RP2D)
Baseline until end of study, up to approximately 9 months

Recommended Phase 2 dose is determined following the evaluation of MDX2001 safety including the incidences of dose limiting toxicities (DLTs), MDX2001 anti-tumor activity, and MDX2001 pharmacokinetics

Secondary Endpoints

Phase 1a: Objective response rate of MDX2001
From date of enrollment until the end of treatment, up to approximately 6 months
All Phases: Duration of response (DOR)
From date of enrollment until the end of treatment, up to approximately 6 months
All Phases: Time to response (TTR)
From date of enrollment until the first documentation of response (CR or PR), approximately 4 months
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Study Design & Arms

AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Phase 1a - MDX2001 Dose EscalationEXPERIMENTALPatients with metastatic solid tumors will receive MDX2001 as intravenous (IV) infusion.
Phase 1b - Indication OptimizationEXPERIMENTALPatients with a single tumor indication receive MDX2001 as intravenous (IV) infusion.
Phase 1b - Dose OptimizationEXPERIMENTALPatients with a single tumor indication will receive MDX2001 as intravenous (IV) infusion.
Phase 2a - Cohort ExpansionEXPERIMENTALPatients with a single tumor indication will receive MDX2001 as intravenous (IV) infusion at the recommended Phase 2 dose.

Interventions

NameTypeDescription
MDX2001DRUGMDX2001 intravenous infusion
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites6

Inclusion Criteria: * Patients must be ≥ 18 years of age * Histologically or cytologically confirmed diagnosis of metastatic solid tumors * Eastern Cooperative Oncology Group (ECOG) performance status 0-1 * All patients should have at least 1 measurable disease per RECIST v1.1. An irradiated lesion...

Countries:United States
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Recent Changes (Last 90 Days)

LOWAug 1, 2026NCT06239194primaryCompletionDate: changed
LOWAug 1, 2026NCT06239194primaryCompletionDate: changed
LOWAug 1, 2026NCT06239194primaryCompletionDate: changed

Frequently asked questions about MDX2001

What is MDX2001 used for in Biliary Tract Cancer?

MDX2001 is an investigational small molecule being studied for the treatment of Biliary Tract Cancer. It is currently in Phase 1 clinical development, with an active trial enrolling patients with advanced solid tumors, including Biliary Tract Cancer, to evaluate its safety and efficacy.

What does MDX2001 target?

MDX2001 targets Trop2, a protein involved in tumor growth. By targeting Trop2, MDX2001 aims to interfere with cancer cell proliferation. This mechanism is being evaluated in a Phase 1 clinical trial for advanced solid tumors, including Biliary Tract Cancer.

Who makes MDX2001?

MDX2001 is being developed by Opko Health Inc., a biopharmaceutical company. The company is conducting a Phase 1 clinical trial to evaluate the drug in patients with advanced solid tumors, including Biliary Tract Cancer.

What phase is MDX2001 in?

MDX2001 is in Phase 1 clinical development. It is an investigational drug, not yet approved by regulatory authorities. The ongoing Phase 1 trial is a dose escalation and dose expansion study in patients with advanced solid tumors, including Biliary Tract Cancer.

What clinical trials is MDX2001 in?

MDX2001 is being studied in a Phase 1 clinical trial with the identifier NCT06239194. This trial is a dose escalation and dose expansion study in patients with advanced solid tumors, including Biliary Tract Cancer. The trial is currently recruiting participants in the United States.

Is MDX2001 the same as any other drug?

MDX2001 is a distinct investigational drug being developed by Opko Health Inc. It is not known to be the same as any other marketed or investigational drug. Its unique mechanism targets Trop2, and it is being evaluated for the treatment of advanced solid tumors.