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KTX-1001

Phase 1

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

Target and mechanism

ModalitySmall molecule

Also known as Cohort A1 & A2: KTX-1001

Success Probability

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Market & Valuation

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Trial Design

CONTROLLEDBiomarker
Total Trials1
Total Enrollment165

FDA Designations

No designations recorded

Clinical trial landscape

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 221 trials

Top 20 of 25 competitors

CompanyTickerTrialsLead PhaseDrugs
AbbVie, Inc.ABBV16PHASE3Pomalidomide, Dexamethasone, Venetoclax
Bristol-Myers Squibb CompanyBMY18PHASE3Iberdomide, Lenalidomide
Takeda Pharmaceutical Co. Ltd. Sponsored ADRTAK5PHASE3IGI, 10%
GSK plc Sponsored ADRGSK17PHASE3Belantamab mafodotin, Pomalidomide, Dexamethasone, Bortezomib
Johnson & JohnsonJNJ28PHASE3Talquetamab, Pomalidomide, Teclistamab, Elotuzumab, Dexamethasone
Regeneron Pharmaceuticals, Inc.REGN11PHASE3Linvoseltamab, Carfilzomib, Daratumumab, Dexamethasone, Pomalidomide
Pfizer Inc.PFE11PHASE3Elranatamab, Lenalidomide
Sanofi SA Sponsored ADRSNY17PHASE3Isatuximab, Dexamethasone, Pomalidomide, Montelukast, Paracetamol/ Acetaminophen
AstraZeneca PLCAZN5PHASE3AZD0120, Daratumumab, Carfilzomib, Dexamethasone, Bortezomib
Gilead Sciences, Inc.GILD3PHASE3Anitocabtagene Autoleucel, Cyclophosphamide, Fludarabine, Pomalidomide, Bortezomib
Karyopharm Therapeutics, Inc.KPTI6PHASE3Selinexor, Elotuzumab, Pomalidomide, Dexamethasone
Grifols, S.A. Sponsored ADR Class BGRFS1PHASE3Xembify
BioLineRX Ltd. Sponsored ADRBLRX1PHASE3BL-8040/kg, G-CSF
C4 Therapeutics, Inc.CCCC3PHASE2Cemsidomide, Dexamethasone
Cellectar Biosciences, Inc.CLRB1PHASE2Iopofosine I 131 single dose, Iopofosine I 131 fractionated dose
GeoVax Labs, Inc.GOVX1PHASE2COVID-19 Vaccine, Synthetic MVA-based SARS-CoV-2 Vaccine GEO-CM04S1
Autolus Therapeutics Plc Sponsored ADRAUTL1PHASE2AUTO CAR T cell therapy
Incyte CorporationINCY2PHASE1Ruxolitinib, Lenalidomide, Methylprednisolone
Moderna, Inc.MRNA2PHASE1mRNA-2808
BeOne Medicines Ltd. Sponsored ADRONC1PHASE1Sonrotoclax, Dexamethasone, Carfilzomib, Daratumumab, Pomalidomide
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