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EPHARNA is a therapeutic agent consisting of small interfering RNA (siRNA) targeting the EphA2 receptor, delivered via 1,2-Dioleoyl-sn-Glycero-3-Phosphatidylcholine (DOPC) nanoliposomes. EphA2 is a receptor tyrosine kinase overexpressed in many human cancers such as lung, breast, prostate, colorectal, pancreatic, melanoma, esophageal and endometrial cancers. Overexpression of EphA2 promotes tumor growth and metastasis through kinase-dependent and independent mechanisms. EPHARNA works by RNA interference to reduce EphA2 protein levels selectively in cancer cells. Preclinical studies demonstrated significant tumor growth reduction and synergy with chemotherapy in ovarian cancer models. Safety studies in mice and rhesus macaques showed good tolerability with mild inflammatory responses at higher doses. A first-in-human phase I clinical trial (NCT01591356) for advanced recurrent solid tumors overexpressing EphA2 is ongoing.
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