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APR-246+ Pembrolizumab

Phase 1

Bladder Cancer | Small molecule | Oncology |Aprea Therapeutics, Inc.|Last Updated: May 13, 2025

Success Probability

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Trial Design

CONTROLLEDDMC
Total Trials1
Total Enrollment40

FDA Designations

No designations recorded

Clinical trial landscape

APR-246+ Pembrolizumab · 1 trial · 6 indications

Phase 1 1
NCT04383938Phase 1/2 Study of APR-246 in Combination With Pembrolizumab in Subjects With Solid Tumor MalignanciesBladder Cancer
COMPLETED40 Analytics
PHASE1COMPLETED
Phase 1/2 Study of APR-246 in Combination With Pembrolizumab in Subjects With Solid Tumor Malignancies
Bladder CancerUnlock trial analytics

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 to 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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Frequently asked questions about APR-246+ Pembrolizumab

What is APR-246 used for?

APR-246 is an investigational small molecule being studied in oncology. It is being evaluated for use in acute myeloid leukemia, myelodysplastic syndromes, platinum sensitive recurrent high-grade serous ovarian cancer with mutated p53, bladder cancer, and other hematologic neoplasms. It is not approved and remains in clinical development.

What does APR-246 target?

APR-246 targets the p53 tumor suppressor protein. It is designed to reactivate mutant p53, restoring its tumor-suppressing function. This mechanism is being studied in cancers that carry p53 mutations, including high-grade serous ovarian cancer and TP53 mutant myelodysplastic syndromes.

Who makes APR-246?

APR-246 is being developed by Aprea Therapeutics, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker symbol APRE. The company is conducting clinical trials of APR-246 across multiple oncology indications.

What phase is APR-246 in?

APR-246 has completed clinical trials ranging from Phase 1 to Phase 3. A Phase 2 study in platinum-resistant high-grade serous ovarian cancer has been completed, as has a Phase 3 study in TP53 mutant myelodysplastic syndromes. The drug remains investigational and is not FDA approved.

What clinical trials is APR-246 in?

APR-246 has been studied in several completed trials. NCT00900614 was a Phase 1 safety study in refractory hematologic or prostate cancer. NCT02098343 was a Phase 1b/II study in recurrent ovarian cancer. NCT03268382 was a Phase 2 study in platinum-resistant ovarian cancer. NCT03745716 was a Phase 3 study in TP53 mutant MDS.

Is APR-246 the same as eprenetapopt?

Yes, APR-246 is also known as eprenetapopt. This alternative name is used in some clinical and scientific literature. Both names refer to the same investigational small molecule being developed by Aprea Therapeutics for p53-mutant cancers.