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APR-246 + Pembrolizumab

Phase 1

Bladder Cancer | Small molecule | Oncology |Aprea Therapeutics, Inc.|Last Updated: May 13, 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
NCT04383938Phase 1/2 Study of APR-246 in Combination With Pembrolizumab in Subjects With Solid Tumor MalignanciesPHASE1 COMPLETED 40Jun 25, 2020Apr 30, 2022May 13, 20258 United States
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Study Endpoints
Primary Endpoints
To Evaluate the Safety of APR-246 in Combination With Pembrolizumab in Subjects With Solid Tumors.
Through study completion, approximately 1 year

To determine the Frequency of treatment-emergent adverse events (TEAEs), and serious adverse events (SAEs) related to APR-246 in combination with pembrolizumab.

To Confirm the Recommended Phase 2 Dose (RP2D) for APR-246 in Combination With Pembrolizumab
Through safety lead in period, during cycle 1 (approximately 21 days)

To determine the dose of APR-246 to be selected for the expansion phase based on the occurence of dose limiting toxicities (DLTs) experienced during the safety assessment period

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Study Design & Arms
AllocationNON_RANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Safety Lead In-Phase 1 Dose Level 1EXPERIMENTALAPR-246 4.5g/d with pembrolizumab 200 mg IV (day 3) every 21 days in patients with advanced non-CNS primary tumors
Expansion 1- Gastric CancerEXPERIMENTALAPR-246 4.5 g/d (days 1-4) with pembrolizumab 200 mg IV (day 3) every 21 days in patients with advanced gastric or GEJ tumors
Expansion 2- Bladder CancerEXPERIMENTALAPR-246 4.5 g/d (days 1-4) with pembrolizumab 200 mg IV (day 3) every 21 days in patients with advanced bladder or urothelial tumors
Expansion 3 -NSCLCEXPERIMENTALAPR-246 4.5 g/d (days 1-4) with pembrolizumab 200 mg IV (day 3) every 21 days in patients with advanced NSCLC.
Interventions
NameTypeDescription
APR-246 (eprenetapopt) + PembrolizumabDRUGAPR-246 D1-4 + Pembrolizumab D3
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites8

Inclusion Criteria: 1. Signed informed consent form (ICF) and ability to comply with protocol requirements. 2. Known tumor TP53 mutation status from recent or archival sample. 3. Histologically and/or cytologically confirmed solid tumor malignancy 1. Safety lead in- Advanced non-central nervous...

Countries:United States
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Competitive Landscape -Bladder Cancer 147 trials
CompanyTickerTrialsLead PhaseDrugs
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Johnson & JohnsonJNJ13PHASE3TAR-200, Mitomycin C, Gemcitabine, Cetrelimab, BCG Vesiculture
Merck & Co., Inc.MRK13PHASE3Pembrolizumab, BCG, Cisplatin, Fluorouracil, Mitomycin C
Pfizer Inc.PFE10PHASE3PF-06801591, Bacillus Calmette-Guerin, Avelumab, Lorlatanib, Talazoparib
Eli Lilly and CompanyLLY4PHASE3Vepugratinib, EV, Pembrolizumab, LY4101174, LY4052031
UroGen Pharma Ltd.URGN4PHASE3UGN-103, UGN-102, UGN-104, Jelmyto for pyelocalyceal
Natera, Inc.NTRA2PHASE3Atezolizumab
CG Oncology, Inc.CGON2PHASE3Cretostimogene Grenadenorepvec
Protara Therapeutics, Inc.TARA2PHASE3TARA-002
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ImmunityBio IncIBRX6PHASE2N-803 and BCG, N-803 + Pembrolizumab, BCG + N-803, Intravesical Recombinant Mycobacterium, N803 plus Bacillus Calmette-Guerin
BeOne Medicines Ltd. Sponsored ADRONC3PHASE3Tislelizumab, Cisplatin, Gemcitabine, Carboplatin, BGB-A445
Exelixis, Inc.EXEL4PHASE2Cabozantinib, Atezolizumab, Zanzalintinib, cabozantinib, atezolizumab
Gilead Sciences, Inc.GILD2PHASE2Sacituzumab Govitecan-hziy, Pembrolizumab, Cisplatin, Avelumab, Zimberelimab
Tyra Bioscience, IncTYRA3PHASE2Dabogratinib, Dabogratinib TBD, TYRA-300, TYRA-300 Dose TBD
BioNTech SE Sponsored ADRBNTX1PHASE2Autogene Cevumeran, Nivolumab, Saline
ICON PlcICLR1PHASE3padeliporfin VTP
AbbVie, Inc.ABBV1PHASE2Livmoniplimab, Budigalimab, Docetaxel, Paclitaxel, Gemcitabine
Jazz Pharmaceuticals Public Limited CompanyJAZZ1PHASE2Zanidatamab
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