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MDX2001

Phase 1

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

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
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment285
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06239194Dose Escalation and Dose Expansion Study of MDX2001 in Patients With Advanced Solid TumorsPHASE1 RECRUITING 285 — — Jun 12, 2024Feb 1, 2029May 6, 20266 United States
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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 — 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)
LOWMay 24, 2026NCT06239194studyFirstPostDate: changed