ASTX-029 is primarily being investigated for its potential in cancer therapy. It functions as a dual inhibitor of the kinases ERK1/2, which are critical components of the RAS/MAPK signaling pathway. This pathway is often dysregulated in various cancers, leading to uncontrolled cell proliferation and survival. By inhibiting ERK1/2, ASTX-029 aims to disrupt this pathway, thereby slowing or stopping the growth of cancer cells.

Currently, ASTX-029 is in clinical trials to evaluate its efficacy and safety in treating solid tumors, particularly those with mutations in the RAS or RAF genes, which are common in cancers like melanoma, colorectal cancer, and non-small cell lung cancer. Early-phase trials focus on determining the optimal dosage, assessing side effects, and understanding its impact on tumor progression. Researchers are also exploring its potential in combination with other therapies to enhance its anticancer effects. If successful, ASTX-029 could offer a targeted treatment option for patients with cancers driven by aberrant RAS/MAPK signaling.