Lead: A preclinical study by Yang et al. described KC1036, a novel small molecule candidate that demonstrated anti‑tumor activity in Ewing sarcoma models and revealed mechanistic insights into its mode of action. The work included in vitro cytotoxicity assays and in vivo efficacy in xenograft models. The authors reported tumor growth inhibition, changes in signaling pathways linked to Ewing biology, and an initial safety profile supporting further optimization. Translational experiments identified biomarkers correlated with sensitivity, offering potential selection strategies for future trials. Investigators called for IND‑enabling toxicology and formulation work, framing KC1036 as a candidate for early‑phase clinical testing given the limited targeted options in this aggressive pediatric and young‑adult malignancy.
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