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Eprenetapopt

Eprenetapopt (APR-246) was a drug meant to refold mutant p53, the most common broken protein in cancer. Its phase 3 in blood cancer failed in 2020.

Eprenetapopt (APR-246) is a prodrug of methylene quinuclidinone, proposed to covalently modify mutant p53 and restore wild-type conformation. Phase 2 with azacitidine in TP53-mutant MDS reported ~50% complete remission. The phase 3 (n=154) missed its primary endpoint of complete remission rate in 2020 (33% vs 22%, not significant). Aprea pivoted away. Subsequent studies suggest much of the activity reflected glutathione depletion and oxidative stress rather than p53 refolding.

Lesson: TP53 remains undrugged by direct reactivation; the field moved to mutation-specific correctors (rezatapopt for Y220C) and to exploiting p53-loss dependencies (WEE1, ATR).

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Eprenetapopt (APR-246)
PubChem
Modality
Small molecule (p53 reactivator)
Mechanism
Converted to MQ, which alkylates cysteines in mutant p53 (claimed) and depletes glutathione.
Brand / code
APR-246

Trials

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ClinicalTrials.gov · phase 2/3
refreshed 2026-09-09
11 studies0 recruiting10 Phase 21 Phase 3
Search “Eprenetapopt” on ClinicalTrials.gov →
Counts are from a name search and may include unrelated studies; up to 100 studies are summarised.

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Eprenetapopt
intervention: Eprenetapopt
Open on ClinicalTrials.gov →

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Latest papers

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Literature trend25 papers in the last 12 months+25% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this drug: (TITLE:"Eprenetapopt" OR ABSTRACT:"Eprenetapopt" OR TITLE:"APR-246" OR ABSTRACT:"APR-246") AND (cancer OR tumor OR tumour OR oncology OR carcinoma OR lymphoma OR leukemia OR leukaemia OR myeloma OR sarcoma OR melanoma OR glioma). Results are unfiltered search hits about Eprenetapopt, not a curated reading list.

Connected

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