Vyxeos (cytarabine/daunorubicin liposomal formulation)

Key advancements include the integration of FLT3 inhibitors into intensive chemotherapy and the emergence of venetoclax...Innovations such as the liposomal formulation CPX-351 and oral formulations of CC-486 and oral decitabine/cedazuridine have further optimized treatment delivery and improved patient quality of life...Current research is actively exploring next-generation inhibitors, antibody-drug conjugates, and cellular immunotherapies such as BiTEs and CAR-T cells. This review summarizes the recent pharmacological evolution in AML and discusses how these emerging therapies bring us closer to the ultimate goal of achieving a definitive cure.

P1/2, N=28, Recruiting, Ohio State University Comprehensive Cancer Center | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026

P2, N=248, Recruiting, Centre Hospitalier Universitaire de Nice | Trial completion date: Oct 2027 --> Feb 2030 | Trial primary completion date: Oct 2027 --> Aug 2028

For ALL, recommendations highlighted avoiding transplant in most Philadelphia chromosome-positive patients achieving minimal residual disease (MRD)-negativity by noting the high relapse risk with certain IKZF1, CDKN2A/B, and PAX5 aberrations, and administering blinatumomab regardless of MRD status in Philadelphia chromosome-positive and -negative ALL...Future directions include refining treatment sequencing, improving outcomes for high-risk subtypes, evaluating the role of HSCT in the era of novel therapies, and standardizing MRD assessment. This consensus report provides valuable expert insights to inform clinical practice and guide future research in leukemia.

P1/2, N=24, Recruiting, Georgetown University | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Dec 2025 --> Dec 2026

Recent advances have led to the development of novel therapeutic strategies, such as BCL-2 inhibitors (venetoclax), IDH1/2 inhibitors (ivosidenib, enasidenib), CD47 inhibitors (magrolimab), TIM-3 inhibitors (sabatolimab), XPO1 inhibitors (eltanexor), NEDD8-activating enzyme inhibitors (pevonedistat), TP53-targeted agents (eprenetapopt), liposomal chemotherapy (CPX-351), and oral HMA formulations. Combinations of hypomethylating agents with these new drugs, as first-line treatment, have to date not proven more efficacious than HMA monotherapy. This review summarizes the current therapeutic landscape on novel therapies for HR-MDS, highlighting their mechanism of action, efficacy, and demonstrates the unmet clinical need for more effective therapies.

P1, N=24, Recruiting, Thomas Jefferson University | Suspended --> Recruiting | Trial completion date: Jan 2026 --> Jan 2029 | Trial primary completion date: Jan 2026 --> Jan 2028

sAML/AML-MRC with an adverse karyotype had a dismal outcome. These findings provide a benchmark for risk stratification in the pre- CPX-351 era.

The OS benefit of CPX-351 observed in the trial was driven by AML-MR with no benefit of CPX-351 in TP53-AML, where the primary prognostic factor was allelic state. Clinical Trial Information: NCT01696084.

P1/2, N=52, Recruiting, M.D. Anderson Cancer Center | Trial completion date: Dec 2025 --> Dec 2027 | Trial primary completion date: Dec 2025 --> Dec 2027

P2, N=21, Active, not recruiting, Roswell Park Cancer Institute | Suspended --> Active, not recruiting | N=46 --> 21 | Trial primary completion date: Jan 2027 --> Jun 2025