Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06852222 | A Study of Bleximenib, Venetoclax and Azacitidine For Treatment of Participants With Newly Diagnosed Acute Myeloid Leukemia (AML) | PHASE3 | RECRUITING | 600 | — | — | Jun 4, 2025 | Aug 15, 2029 | Jun 5, 2026 | 250 | United States, Australia +22 |
| NCT05453903 | A Study of Bleximenib in Combination With Acute Myeloid Leukemia (AML) Directed Therapies | PHASE1 | ACTIVE NOT_RECRUITING | 196 | — | — | Oct 4, 2022 | Mar 3, 2027 | Jun 5, 2026 | 32 | United States, Australia +6 |
CR is defined as Bone marrow blasts less than (\<) 5 percent (%); Absence of circulating blasts; Absence of extramedullary disease; Absolute neutrophil count (ANC) greater than or equal to (\>=) 1.0 \* 10\^9/Liter (1,000/microliter \[mcL\]); Platelet count \>= 100 \* 10\^9/L (100,000/mcL).
Overall survival time is defined as the time duration from the date of randomization to death due to any cause.
An AE is any untoward medical occurrence in a participant participating in a clinical study that does not necessarily have a causal relationship with the pharmaceutical/biological agent under study.
Number of Participants with AEs by severity will be reported. Severity will be graded according to the National Cancer Institute Common Terminology Criteria for Adverse Events (NCI-CTCAE) version 5.0. Severity scale ranges from Grade 1 (Mild) to Grade 5 (Death). Grade 1= Mild, Grade 2= Moderate, Grade 3= Severe, Grade 4= Life-threatening and Grade 5= Death related to adverse event.
Number of participants with DLT will be reported according to National Cancer Institute Common Terminology Criteria for Adverse Events (NCI CTCAE) version 5.0.
| Arm | Type | Description |
|---|---|---|
| Arm A: Bleximenib and Venetoclax (VEN) + Azacitidine (AZA) | EXPERIMENTAL | Participants with acute myeloid leukemia (AML) will receive bleximenib in combination with venetoclax (VEN) and azacitidine (AZA) for 28-days treatment cycles and treatment will continue until progression or unacceptable toxicity. |
| Arm B: Placebo and Venetoclax (VEN) + Azacitidine (AZA) | PLACEBO_COMPARATOR | Participants with AML will receive placebo in combination with VEN and AZA for 28-days treatment cycles, and treatment will continue until progression or unacceptable toxicity. |
| Arm A: Relapsed/Refractory Setting | EXPERIMENTAL | Participants with relapsed/refractory AML harboring NPM1, KMT2A, NUP98, or NUP214 alterations will receive bleximenib in combination with either venetoclax (VEN) (Cohort A1: bleximenib+VEN) or azacitidine (AZA) (Cohort A2: bleximenib +AZA) or VEN+AZA (Cohort A3: bleximenib+VEN+AZA) or VEN + AZA (Cohort A4: bleximenib + VEN + AZA) in adolescent participants aged greater than or equal to (\>=) 12 years and less than (\<) 18 years of age, to select the recommended phase 2 dose (RP2D) of bleximenib in combination with VEN, AZA or VEN+AZA (dose selection). In dose expansion portion of the study, participants will receive bleximenib in combination with AML directed therapies at the RP2D(s). |
| Arm B: Newly Diagnosed Chemotherapy Ineligible Setting | EXPERIMENTAL | Participants will receive bleximenib in combination with VEN+AZA as frontline chemo therapy for newly diagnosed AML participants harboring KMT2A, NPM1, NUP98, or NUP214 alterations who are \>=75 years of age or \>=18 years of age to \<75 years of age with comorbidities that preclude the use of intensive induction chemotherapy. |
| Arm C: Newly Diagnosed Chemotherapy Eligible Setting | EXPERIMENTAL | Participants will receive combination of bleximenib with cytarabine+daunorubicin or idarubicin chemotherapy as frontline treatment regimen for participants \>= 18 to \<75 years of age with AML harboring NPM1, KMT2A, NUP98, or NUP214 alterations and eligible for intensive chemotherapy. |
| Name | Type | Description |
|---|---|---|
| Bleximenib | DRUG | Bleximenib will be administered orally. |
| Venetoclax (VEN) | DRUG | VEN will be administered orally. |
| Azacitidine (AZA) | DRUG | AZA will be administered intravenously or subcutaneously. |
| Placebo | DRUG | Placebo will be administered orally. |
| Cytarabine | DRUG | Participants will receive cytarabine. |
| Daunorubicin or Idarubicin | DRUG | Participants will receive daunorubicin or idarubicin. |
Inclusion criteria: * Be 18 years of age or older at the time of informed consent * Previously untreated lysine N-methyltransferase 2A gene rearranged (KMT2Ar) or nucleophosmin 1 gene mutated (NPM1m) acute myeloid leukemia (AML) with greater than or equal to (\> or =) 10% bone marrow blasts per 202...