Acute myeloid leukemia (AML) is a hematological cancer with limited treatment options and characterized by frequent relapse and poor prognosis. The only approved antibody-drug conjugate for AML is gemtuzumab ozogamicin, which targets CD33.
Eilean Therapeutics LLC has presented data for ZE74-0282, a novel small molecule that targets the JAK2 JH2 domain harboring the V617F mutation with no impact on wild-type JAK2.
Despite therapeutic advances, multiple myeloma (MM) remains incurable, with most patients relapsing and developing resistance, especially those refractory to proteasome inhibitors, immunomodulatory drugs and anti-CD38 antibodies. Limited options and poor prognosis highlight the need for new agents with distinct mechanisms and better safety.
Crossbow Therapeutics Inc. has provided details on the preclinical characterization for the telomerase reverse transcriptase (TERT)-targeting T-cell engager CBX-663.
Dexoligo Therapeutics has presented data for their liver-targeted siRNA DXO-1801 for the potential treatment of inflammation-driven anemia, a serious complication of chronic diseases such as chronic kidney disease, myelofibrosis or advanced solid tumors characterized by elevated pro-inflammatory cytokines where the availability for iron for erythropoiesis is limited due to increased hepcidin levels.
JAK2 inhibitors (JAK2i) are the standard treatment for myelofibrosis (MF), offering symptom relief and reducing spleen size. However, all FDA-approved JAK2i are type I inhibitors, which fail to eliminate the mutant MPN clone, leading many patients to treatment discontinuation.
Using C-X-C chemokine receptor type 4 (CXCR4) antagonists as cell mobilization agents has resulted in some FDA approved agents, such as Plerixafor, for hematopoietic stem cell transplantation and neutropenia. Oral cell mobilizers could result in using them in conditions such as sickle-cell disease (SCD) and chronic neutropenia. Emory University has developed and presented data for their CXCR4 antagonist EMU-116.
Researchers from Chimerix, now part of Jazz Pharmaceuticals, presented preclinical data on ONC-206, a compound that functions as both an agonist of the mitochondrial protease Caseinolytic peptidase P (CLPP) and an antagonist of the G protein-coupled receptor DRD2, in models of triple-negative breast cancer (TNBC).
Most patients with myelodysplastic syndrome (MDS) exhibit variable degrees of anemia due to impaired erythropoiesis. Ameliorating anemia and reducing the dependence on transfusion may enhance the quality of life of these patients and improve their survival rates.
Although hormone receptor-positive (HR+) breast cancer accounts for over 70% of cases, 20%-30% of patients still experience relapse despite endocrine therapies such as tamoxifen. Recurrence is also observed in HER2+ breast cancer, even with HER2-targeted antibodies and antibody-drug conjugates (ADCs), the use of which can be limited by adverse effects such as interstitial lung disease.
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