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Therapy Name Azacitidine + Revumenib + Venetoclax
Synonyms
Therapy Description

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Drug Name Trade Name Synonyms Drug Classes Drug Description
Azacitidine Vidaza azacytidine|CC-486|5-azacytidine|5-AC|U-18496|Onureg DNMT inhibitor (Pan) 5 Vidaza (azacitidine) is a cytidine analog that incorporates into DNA and RNA and binds to DNA methyltransferases (DNMTs), resulting in DNMT degradation and decreased DNA methylation, and leading to increased tumor cell death (PMID: 28159832, PMID: 28067760). Vidaza (azacitidine) is FDA-approved for use in patients with some subtypes of myelodysplastic syndrome and Onureg (azacitidine) is FDA approved for use in continued treatment of acute myeloid leukemia(FDA.gov).
Revumenib Revuforj SNDX5613|SNDX 5613|SNDX-5613 MEN1-KMT2A Inhibitor 8 Revuforj (revumenib) inhibits the interaction between Menin and KMT2A (MLL), potentially resulting in antitumor activity and decreased proliferation of tumor cells with KMT2A (MLL) rearrangements or NPM1 mutations (PMID: 36922593, PMID: 36922589). Revuforj (revumenib) is FDA-approved for use in adult and pediatric patients 1 year and older with relapsed or refractory acute leukemia harboring KMT2A translocation, and in adult and pediatric patients 1 year and older with relapsed or refractory acute myeloid leukemia harboring susceptible NPM1 mutations (FDA.gov).
Venetoclax Venclexta ABT-199|RG7601|GDC-0199|ABT119|Venclyxto BCL2 inhibitor 29 Venclexta (venetoclax) is a BH3-mimetic that binds to and inhibits BCL2, resulting in increased tumor cell apoptosis (PMID: 26589495, PMID: 25048785). Venclexta (venetoclax) is FDA approved for use in patients with chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), and in combination with chemotherapy in patients 75 years old or older with newly-diagnosed acute myeloid leukemia (FDA.gov).

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Molecular Profile Indication/Tumor Type Response Type Therapy Name Approval Status Evidence Type Efficacy Evidence References
KMT2A rearrange acute myeloid leukemia sensitive Azacitidine + Revumenib + Venetoclax Phase I Actionable In a Phase I trial, combination of Vidaza (azacitidine), Veclexta (venetoclax), and Revuforj (revumenib) led to an overall response rate (ORR) of 88.4% (38/43), complete response (CR) rate of 67.4%, composite CR (CRc) rate of 81.4%, and median overall survival of 15.5 mo in older adults with newly diagnosed acute myeloid leukemia harboring KMT2A rearrangements (KMT2Ar) or NPM1 mutations, ORR, CR, and CRc rates were 100%, 78%, and 89% in KMT2Ar (n=9) (PMID: 40504618; NCT03013998). 40504618

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Clinical Trial Phase Therapies Title Recruitment Status Covered Countries Other Countries
NCT06177067 Phase I Cytarabine + Hydrocortisone + Methotrexate Azacitidine + Revumenib + Venetoclax Study of Revumenib, Azacitidine, and Venetoclax in Pediatric and Young Adult Patients With Refractory or Relapsed Acute Myeloid Leukemia Recruiting USA 0


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