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MIRX002 is a microRNA-based anticancer therapeutic developed by PURMX Therapeutics, a Hiroshima University spin-off. The drug is a synthetic mimic of miR-3140-3p, a naturally occurring microRNA that targets cancer stem cells to inhibit tumor growth, progression, and recurrence. To overcome the challenges of nucleic acid stability and delivery, MIRX002 utilizes the surfactant peptide A6K as a delivery carrier, which protects the miRNA from enzymatic degradation until it reaches the target cells. It is currently being evaluated in Phase 1/2 clinical trials for malignant pleural mesothelioma (MPM) and head and neck squamous cell carcinoma (HNSCC). The U.S. FDA has granted Orphan Drug Designation to miR-3140-3p for the treatment of mesothelioma.
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