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MT-401-OTS

Phase 1

Acute Myeloid Leukemia, in Relapse | Small molecule | Oncology |Marker Therapeutics, Inc.|Last Updated: Dec 16, 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
CONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment40
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT06552416Safety of MT-401-OTS in Patients With Relapsed AML or MDSPHASE1 RECRUITING 40Jun 16, 2025Sep 1, 2029Dec 16, 20253 United States
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Study Endpoints
Primary Endpoints
Safety of MT-401-OTS
Through study completion. Approximately 5 years

Assess: * Incidence of DLTs to determine the RP2D * Safety (including but not limited to): TEAEs, SAEs, deaths, and clinical laboratory abnormalities per NCI CTCAE, Version 5.0 * Acute GVHD per 2016 MAGIC criteria * CRS and ICANS per ASTCT criteria

Tolerability of MT-401-OTS
Through study completion. Approximately 5 years

cGVHD per 2014 NIH consensus criteria for cGVHD

Secondary Endpoints
To assess the efficacy of MT-401-OTS in participants with high-risk or very-high-risk MDS per IPSS-M who have evidence of disease following at least 4 cycles of a HMA
Following conclusion of the disease assessment of last MDS subject treated. Approximately 2 years
To assess the efficacy of MT-401-OTS in participants with intermediate or high-risk AML per 2022 ELN criteria who have evidence of disease following induction therapy or at least 4 cycles of nonintensive treatment
Following conclusion of the disease assessment of last AML subject treated. Approximately 2 years
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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort -1EXPERIMENTAL50 × 106 cells flat dose, 1 dose infused on Day 0
Cohort 1EXPERIMENTAL100 × 106 cells flat dose, 1 dose infused on Day 0
Cohort 2EXPERIMENTAL200 × 106 cells flat dose, 1 dose infused on Day 0
Cohort 3EXPERIMENTAL400 × 106 cells flat dose, 1 dose infused on Day 0
Cohort 4 (optional)EXPERIMENTALup to 400 × 106 cells flat dose TBD based on data from Cohorts 1-3; may include split dosing, dosing with or without lymphodepleting conditioning regimen, or dosing with or without HMA
Interventions
NameTypeDescription
MT-401-OTSDRUGMT-401-OTS is an off the shelf cellular therapy product given by IV infusion through either a peripheral or central line.
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Eligibility Criteria
Age Range65 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites3

Inclusion Criteria: * General 1. Must be ≥ 65 years of age and capable of giving signed informed consent, which includes compliance with the requirements and restrictions listed in the ICF and in the protocol, at the time of signing the ICF 2. Must have a life expectancy ≥ 12 weeks 3. Must h...

Countries:United States
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT06552416primaryCompletionDate: changed
LOWMay 24, 2026NCT06552416studyFirstPostDate: changed