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AX102 is a DNA oligonucleotide aptamer that selectively inhibits platelet-derived growth factor B (PDGF-B), disrupting PDGF-B signaling in the tumor microenvironment. Its primary mechanism is the depletion of PDGF-B-dependent pericytes from the vasculature of tumors, resulting in regression and normalization of tumor vessels. This leads to improved delivery and efficacy of cytotoxic chemotherapy agents, notably cyclophosphamide, in preclinical models. AX102 does not itself reduce tumor growth unless combined with cytotoxic agents. The drug was developed to function as a targeted anti-angiogenic agent and demonstrates a dose-dependent effect on pericyte loss and vessel regression.
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