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KPT-8602

Phase 1

Relapsed/Refractory Multiple Myeloma (RRMM) | Small molecule | Oncology |Karyopharm Therapeutics Inc.|Last Updated: Mar 19, 2025

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
CONTROLLEDBiomarker
Total Trials1
Total Enrollment277
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02649790Study of the Safety, Tolerability and Efficacy of KPT-8602 in Participants With Relapsed/Refractory Cancer IndicationsPHASE1 COMPLETED 277Jan 1, 2016Dec 23, 2024Mar 19, 202546 United States, Canada +3
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Study Endpoints
Primary Endpoints
Part A1, A2, B, C, D, E, F: Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)
Approximately 4 weeks
Part A1, A2, B, C, D, E, F: Overall Response Rate (ORR)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Duration of Response (DOR)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Progression-free Survival (PFS)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Overall Survival (OS)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Clinical Benefit Rate (CBR)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Duration of Clinical Benefit Rate (CBR)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Disease Control Rate (DCR)
Approximately 8 years
Part A1, A2, B, C, D, E, F: Duration of DCR
Approximately 8 years
Part F Phase 2: ORR
Approximately 8 years
Part G: Maximum Tolerated Dose (MTD) and Recommended Phase 2 Dose (RP2D)
Approximately 8 years
Part H: 2- Year Progression-free Survival (PFS)
Approximately Up to 2 years
Secondary Endpoints
Part A1, A2, B, C, D, E, F, H: Area Under the Plasma Concentration Time Curve from Time Zero to the Time of the Last Quantifiable Concentration (AUC0-t) of Eltanexor
Pre-dose 2 hours post-dose Cycle 1 Day 1 and 15; Pre-dose 0.5, 1, 2, 3, 4, 6 and 8 hours post-dose on Cycle 1 Day 21 and 26; 24 hours post-dose Cycle 1 Day 2, 22, and 27; 48 hours post-dose Cycle 1 Day 23 and 28 and up to approximately 8 years
Part A1, A2, B, C, D, E, F, H: Maximum Observed Plasma Concentration (Cmax) of Eltanexor
Pre-dose 2 hours post-dose Cycle 1 Day 1 and 15; Pre-dose 0.5, 1, 2, 3, 4, 6 and 8 hours post-dose on Cycle 1 Day 21 and 26; 24 hours post-dose Cycle 1 Day 2, 22, and 27; 48 hours post-dose Cycle 1 Day 23 and 28 and up to approximately 8 years
Part A1, A2, B, C, D, E, F, H: Time to Reach Maximum Observed Plasma Concentration (Tmax) of Eltanexor
Pre-dose 2 hours post-dose Cycle 1 Day 1 and 15; Pre-dose 0.5, 1, 2, 3, 4, 6 and 8 hours post-dose on Cycle 1 Day 21 and 26; 24 hours post-dose Cycle 1 Day 2, 22, and 27; 48 hours post-dose Cycle 1 Day 23 and 28 and up to approximately 8 years
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Part A1: RRMM- KPT-8602 single agent; QoDx5/weekEXPERIMENTALParticipants received KPT-8602 once daily for 5 days per week (QDx5/week) at escalated doses (completed).
Part A2: RRMM- KPT-8602 single agent; QoDx3/weekEXPERIMENTALParticipants received KPT-8602 once daily for 3 days per week (QoDx3/week). The starting dose for Part A2 will be informed by Part A1 (completed).
Part B: RRMM- KPT-8602 with low-dose dexamethasone; QDx5/weekEXPERIMENTALParticipants received KPT-8602 for 5 consecutive days (QDx5/week) in combination with low dose dexamethasone (20 milligram \[mg\] on Days 1, 3, 8, 10, 15, 17, 22, and 24 of each 28-day cycle) (completed).
Part C: CRC- KPT-8602 single agentEXPERIMENTALParticipants were treated with KPT-8602 at a dose and schedule that has been cleared in Part A (completed).
Part D: mCRPC- KPT-8602 single agentEXPERIMENTALParticipants were treated with KPT-8602 at a dose and schedule that has been cleared in Part A (completed).
Part E: mCRPC- KPT-8602 with abiraterone and corticosteroidsEXPERIMENTALParticipants were treated with KPT-8602 at a dose and schedule that had been cleared in Part A in combination with abiraterone and corticosteroids. Participants continued to receive the dose and schedule of abiraterone and corticosteroids that they were receiving at the time of enrollment (completed).
Part F: High-risk Myelodysplastic Syndrome (MDS)- KPT-8602 single agentEXPERIMENTALParticipants were treated with KPT-8602 at a dose and schedule that had been cleared in Part A. In select cases (for example, participants achieving stable disease \[SD\], hematological improvement \[HI\], partial response \[PR\] and tolerating treatment, etc.), the dose may be escalated 1 level based on safety and efficacy considerations (completed).
Part F Phase 2: RR High-risk MDS- KPT-8602 single agentEXPERIMENTALParticipants will be enrolled at recommended Phase 2 doses (RP2D) of 10 mg daily on Days 1 to 5 of each week, in a dose expansion, based upon the results from the Phase 1 portion of Part F.
Part G: Newly Diagnosed Intermediate/High-Risk MDS -KPT-8602 with ASTX727EXPERIMENTALParticipants will receive KPT-8602 once daily at escalated doses. The starting dose for KPT-8602 is 5 mg orally once daily from Day 8 to Day 28 (Weeks 2 to 4) on a 28-day cycle in combination with ASTX727.
Part H: AML Maintenance Therapy- KPT-8602 single agentEXPERIMENTALParticipants with high-risk acute myeloid leukemia (AML) prior to transplant will be enrolled to receive maintenance therapy with KPT-8602 post-allogeneic stem cell transplantation. The dose for KPT-8602 will be 10 mg (RP2D from Part F) oral, to be administered once daily from Day 1 to Day 21 (Weeks 1 to 3) on a 28-day cycle.
Interventions
NameTypeDescription
KPT-8602DRUGParticipants will receive KPT-8602 oral tablets.
ASTX727DRUGASTX727 is a combination drug of 35 mg decitabine and 100 mg cedazuridine.
DexamethasoneDRUGParticipants will receive dexamethasone oral tablets.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites46

INCLUSION CRITERIA 1. Written informed consent signed prior to any screening procedures and in accordance with federal, local, and institutional guidelines. 2. Age ≥ 18 years. 3. Adequate hepatic function: 1. total bilirubin ≤ 2 times the upper limit of normal (ULN) (except participants with Gi...

Countries:United StatesCanadaChinaFranceSpain
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