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KTX-1001

Phase 4

Multiple Myeloma | Small molecule | Oncology |Bristol-Myers Squibb Company|Trials Updated: Jun 8, 2026

KTX-1001 development status

Highest phase Phase 4 run by Dr Sonia ben khalifa (PhD) (NCT03902249)
Phase scored for BMYPhase 1
Registered trials 330+ across 110 sponsors since Dec 2002

KTX-1001 target and mechanism

ModalitySmall molecule

Also known as Dexamethasone

Success Probability

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Market & Valuation

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Trial Design

CONTROLLED
Total Trials1
Total Enrollment165

FDA Designations

No designations recorded

KTX-1001 clinical trials

KTX-1001 · 1 trial · 3 indications

Phase 1 1
NCT05651932A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple MyelomaMultiple Myeloma
RECRUITING165 Analytics
PHASE1RECRUITING
A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma
Multiple MyelomaUnlock trial analytics

Study Endpoints

Primary Endpoints

Dose Escalation: Determination of Recommended Phase 2 Dose (RP2D) and/or Maximum Tolerated Dose (MTD) Dose Expansion: Provide preliminary efficacy data on the antitumor effects of KTX-1001 in combination with other anti-myeloma therapy
Cycle 1 (28 days)

Incidence of dose-limiting toxicity (DLTs), treatment-emergent adverse events (TEAEs), treatment-related AEs, and clinically significant changes in laboratory test results

Secondary Endpoints

Disease Specific Response to KTX-1001± Combination Therapy
Duration of Study
Pharmacokinetics & Pharmacodynamics KTX-1001± Combination Therapy
Duration of Study
Safety profile of KTX-1001± Combination Therapy
Duration of Study
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort A (Single agent): KTX-1001 + dexamethasoneEXPERIMENTALCohort A1 (single agent): KTX-1001 at RP2D1 + dex Cohort A2 (single agent): KTX-1001 at RP2D2 + dex
Cohort B (Mezigdomide): KTX-1001 + Mezigdomide + dexEXPERIMENTALCohort B1 (Mezigdomide): KTX-1001 at RP2D1 + Mezigdomide + dex Cohort B2 (Mezigdomide):: KTX-1001 at RP2D2 + Mezigdomide + dex
Cohort C (carfilzomib/KYPROLIS®): KTX-1001 + carfilzomib + dexEXPERIMENTALCohort C1 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D1 + carfilzomib + dex Cohort C2 (carfilzomib/KYPROLIS®): KTX-1001 at RP2D2 + carfilzomib + dex
Cohort D (pomalidomide): KTX-1001 + pomalidomide + dexEXPERIMENTALCohort D (pomalidomide): KTX-1001 at RP2D2 + pomalidomide + dex

Interventions

NameTypeDescription
Cohort A1 & A2: KTX-1001DRUGKTX-1001: Orally for 28 days each cycle until progression. Dexamethasone: Orally once weekly
Cohort B1 & B2: KTX-1001+MezigdomideDRUGDrug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once weekly Drug: Mezigdomide Dexamethasone: Orally once weekly
Cohort C1 & C2: KTX-1001 + Carfilzomib (KYPROLIS®)DRUGDrug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once weekly Drug: Carfilzomib (KYPROLIS®): IV, once weekly for 3 weeks in each 28-day cycle
Cohort D: KTX-1001+ pomalidomide (Pomalyst, Imnovid)DRUGDrug: KTX-1001: Orally for 28 days each cycle until progression Drug: Dexamethasone: Orally once a week Drug: Pomalidomide (Pomalyst, Imnovid): Orally, for 21 days in each 28-day cycle
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites22

Key Inclusion Criteria for Dose-Expansion: * ≥ 18 years of age * ECOG score ≤ 1 * Multiple myeloma (as per IMWG) * Prior therapy for MM: Participants must have received at least 1 and up to 3 prior lines of therapy as defined by IMWG, and the following drug classes: PI, IMiD, and anti-CD38 antib...

Countries:United StatesCanadaFranceSpain
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Competitive Landscape -Multiple Myeloma 340 trials

Frequently asked questions about KTX-1001

What is KTX-1001 used for?

KTX-1001 is an investigational small molecule being studied for the treatment of multiple myeloma, including relapsed and refractory disease. It is being evaluated in a Phase 1 clinical trial in adults. It is not approved for any indication and remains in clinical development.

Who is developing KTX-1001?

KTX-1001 is being developed by Bristol-Myers Squibb Company, which trades under the ticker BMY. The company is the sponsor of the Phase 1 study evaluating KTX-1001 in multiple myeloma.

What phase is KTX-1001 in?

KTX-1001 is in Phase 1 clinical development. It is investigational and has not been approved by the FDA or any other regulatory agency. The single active trial is currently recruiting participants.

What clinical trials is KTX-1001 in?

KTX-1001 is being studied in one active clinical trial, NCT05651932, titled A Study of an MMSET Inhibitor in Patients With Relapsed and Refractory Multiple Myeloma. The trial is recruiting and plans to enroll 165 participants across the United States, Canada, France, and Spain.

How does KTX-1001 work?

KTX-1001 is described as an MMSET inhibitor. MMSET is a molecular target in multiple myeloma, and the Phase 1 trial NCT05651932 is titled as a study of an MMSET inhibitor in patients with relapsed and refractory multiple myeloma.