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Profile Name | MDM2 amp TP53 wild-type |
Gene Variant Detail | |
Relevant Treatment Approaches |
Molecular Profile | Indication/Tumor Type | Response Type | Relevant Treatment Approaches | Therapy Name | Approval Status | Evidence Type | Efficacy Evidence | References |
---|---|---|---|---|---|---|---|---|
MDM2 amp TP53 wild-type | osteosarcoma | sensitive | KRT-232 | Preclinical | Actionable | In a preclinical study, KRT-232 (AMG 232) inhibited tumor growth in osteosarcoma cell line xenograft models with wild-type TP53 and MDM2 amplification (PMID: 25567130, PMID: 24967612). | 24967612 25567130 | |
MDM2 amp TP53 wild-type | osteosarcoma | sensitive | SAR405838 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SAR405838 inhibited growth of TP53 wild-type, MDM2 amplified osteosarcoma in culture and resulted in tumor regression in cell line xenograft models (PMID: 25145672). | 25145672 | |
MDM2 amp TP53 wild-type | osteosarcoma | sensitive | Doxorubicin + KRT-232 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, the combination of KRT-232 (AMG 232) and Adriamycin (doxorubicin) inhibited tumor growth and induced tumor regression in an MDM2-amplifed TP53 wild-type osteosarcoma cell line xenograft model, with increased efficacy over either agent alone (PMID: 25567130). | 25567130 | |
MDM2 amp TP53 wild-type | liposarcoma | sensitive | SAR405838 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, SAR405838 decreased viability and induced cell cycle arrest in MDM2-amplified dedifferentiated liposarcoma cell lines with wild-type TP53 in culture, and induced TP53 pathway activation and tumor regression in xenograft models (PMID: 26475335). | 26475335 | |
MDM2 amp TP53 wild-type | glioblastoma | sensitive | RG7112 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, glioblastoma multiforme (GBM) cell lines with amplification of MDM2 and wild-type TP53 demonstrated sensitivity to RG7112 in culture, resulting in decreased viability and restoration of Tp53 signaling, and treatment with RG7112 resulted in growth inhibition in MDM2-amplified TP53 wild-type GBM cell line xenograft models (PMID: 26482041). | 26482041 | |
MDM2 amp TP53 wild-type | glioblastoma | predicted - sensitive | KRT-232 | Phase I | Actionable | In a Phase I trial, KRT-232 (AMG 232) demonstrated acceptable safety, and resulted in stable disease in 60% (6/10) of patients with TP53 wild-type, MDM2 amplified glioblastoma, with a median duration of 1.8 months (PMID: 31359240; NCT01723020). | 31359240 | |
MDM2 amp TP53 wild-type | Advanced Solid Tumor | predicted - sensitive | KRT-232 | Phase I | Actionable | In a Phase I trial, KRT-232 (AMG 232) demonstrated acceptable safety, and resulted in stable disease in 62.5% (10/16) of patients with TP53 wild-type, MDM2 amplified solid tumors other than liposarcoma, glioblastoma, and breast cancer, with a median duration of 3.3 months, and a patient with squamous cell carcinoma achieved unconfirmed partial response per central evaluation (PMID: 31359240; NCT01723020). | 31359240 | |
MDM2 amp TP53 wild-type | Advanced Solid Tumor | predicted - sensitive | ALRN-6924 | Phase I | Actionable | In a Phase I trial, treatment with ALRN-6924 resulted in a disease control rate of 71% (5/7) in advanced solid tumors patients with wild-type TP53 and MDM2 amplification (PMID: 34301750; NCT02264613). | 34301750 | |
MDM2 amp TP53 wild-type | dedifferentiated liposarcoma | predicted - sensitive | BI 907828 | Phase I | Actionable | In a Phase I trial, BI 907828 treatment demonstrated activity in patients with TP53 wild-type, MDM2-amplified dedifferentiated liposarcoma, resulting in a disease control rate (DCR) of 75% (9/12, all stable disease) and progression-free survival (PFS) ranging from 1.5 to 22 months, with PFS greater than 10.5 months in 41.6% (5/12) of patients (PMID: 37269344; NCT03449381). | 37269344 | |
MDM2 amp TP53 wild-type | well-differentiated liposarcoma | predicted - sensitive | BI 907828 | Phase I | Actionable | In a Phase I trial, BI 907828 treatment demonstrated activity in patients with TP53 wild-type, MDM2-amplified well-differentiated liposarcoma, resulting in a disease control rate of 100%, (7/7, 4 partial responses of at least 12 months, 3 stable disease), and a progression-free survival (PFS) of greater than 7.5 months, with a PFS of at least 14 months or more in 5 patients (PMID: 37269344; NCT03449381). | 37269344 | |
MDM2 amp TP53 wild-type | heart sarcoma | predicted - sensitive | Milademetan | Phase Ib/II | Actionable | In a Phase Ib/II trial, Milademetan treatment resulted in an overall response rate of 20% (2/10, both partial responses), a disease control rate of 60%, a median progression-free survival of 4.7 months, and a median overall survival of 12.2 months in patients with MDM2-amplified, TP53 wild-type intimal sarcoma (PMID: 37369013). | 37369013 | |
MDM2 amp TP53 wild-type | Advanced Solid Tumor | predicted - sensitive | BI 907828 | Phase I | Actionable | In a Phase I trial, BI 907828 treatment demonstrated safety and efficacy in patients with advanced solid tumors, resulting in a median progression-free survival of 8.1 months and an overall response rate of 11.1% (6/54, all partial responses (PR), 4 well-differentiated liposarcoma, 1 intrahepatic cholangiocarcinoma, 1 pancreatic cancer), with all PRs occurring in patients harboring an MDM2 amplification (PMID: 37269344; NCT03449381). | 37269344 | |
MDM2 amp TP53 wild-type | glioblastoma | sensitive | BI 907828 | Preclinical - Cell line xenograft | Actionable | In a preclinical study, BI 907828 treatment inhibited viability of a cell line derived from a TP53 wild-type, MDM2-amplified glioblastoma patient-derived xenograft (PDX) model in culture and inhibited tumor cell proliferation, induced apoptosis, and resulted in improved median survival compared to vehicle treatment (10 mg/kg treatment: 218 days and 2 mg/kg treatment: 57 days vs vehicle: 28 days, p<0.0001) in the PDX model, despite limited blood-brain barrier penetration (PMID: 37828724). | 37828724 | |
MDM2 amp TP53 wild-type | malignant fibrous histiocytoma | sensitive | BI 907828 | Preclinical - Pdx | Actionable | In a preclinical study, BI 907828 induced tumor regression in a patient-derived xenograft (PDX) model of MDM2-amplified, TP53 wild-type undifferentiated pleomorphic sarcoma (PMID: 39259562). | 39259562 | |
MDM2 amp TP53 wild-type | lung squamous cell carcinoma | sensitive | BI 907828 | Preclinical - Pdx | Actionable | In a preclinical study, BI 907828 induced prolonged tumor stasis and tumor regression in patient-derived xenograft (PDX) models of MDM2-amplified, TP53 wild-type squamous cell lung carcinoma (PMID: 39259562). | 39259562 | |
MDM2 amp TP53 wild-type | intrahepatic cholangiocarcinoma | predicted - sensitive | BI 907828 | Case Reports/Case Series | Actionable | In a Phase Ia/Ib trial, BI 907828 treatment resulted in a partial response in a patient with MDM2-amplified (CN=9), TP53 wild-type metastatic intrahepatic cholangiocarcinoma, with treatment lasting 12 months (PMID: 38567193; NCT03449381). | 38567193 | |
MDM2 amp TP53 wild-type | ampulla of Vater adenocarcinoma | predicted - sensitive | BI 907828 | Case Reports/Case Series | Actionable | In a Phase Ia/Ib trial, BI 907828 treatment resulted in a partial response in a patient with MDM2-amplified (CN=15) metastatic ampullary adenocarcinoma, with treatment ongoing at 19.1 months (PMID: 38567193; NCT03449381). | 38567193 | |
MDM2 amp TP53 wild-type | intrahepatic cholangiocarcinoma | predicted - sensitive | BI 907828 + Ezabenlimab | Case Reports/Case Series | Actionable | In a Phase Ia/Ib trial, treatment with the combination of BI 907828 and Ezabenlimab (BI 754091) resulted in 3 partial responses and 1 stable disease in 4 patients with MDM2-amplified, TP53 wild-type metastatic intrahepatic cholangiocarcinoma (PMID: 38567193; NCT03964233). | 38567193 | |
MDM2 amp TP53 wild-type | gallbladder adenocarcinoma | predicted - sensitive | BI 907828 + Ezabenlimab | Case Reports/Case Series | Actionable | In a Phase Ia/Ib trial, treatment with the combination of BI 907828 and Ezabenlimab (BI 754091) resulted in a partial response with a progression-free survival of 7.9 months in a patient with MDM2-amplified (CN=14), TP53 wild-type metastatic gallbladder adenocarcinoma (PMID: 38567193; NCT03964233). | 38567193 | |
MDM2 amp TP53 wild-type | liposarcoma | predicted - sensitive | Alrizomadlin | Case Reports/Case Series | Actionable | In a Phase I trial, Alrizomadlin (APG-115) treatment demonstrated safety in patients with advanced solid tumors, with an overall response rate of 10% (2/20, partial responses) and stable disease in 50% (10/20) of evaluable patients and a median progression-free survival (PFS) of 6.1 months, with an overall response rate of 25% (2/8) and disease control rate of 100% (8/8) in liposarcoma patients with wild-type TP53 and MDM2 amplification (PMID: 39002360). | 39002360 |