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PTS-02 is a small molecule anti-cancer drug developed by Gongwin Biopharm specifically for the treatment of Adenoid Cystic Carcinoma (ACC). The drug's active ingredient is para-toluenesulfonamide (PTS), which operates via a unique mechanism known as 'Minimally Invasive Targeted Tumor Ablation' (MITTA). When administered through intratumoral injection, PTS selectively penetrates the membranes of cancer cells while sparing normal cells. Within the cancer cell, it targets the lysosomes, causing membrane rupture and the release of digestive enzymes, and simultaneously interferes with mitochondrial ATP synthase to disrupt energy production. This dual action leads to rapid tumor cell necrosis. PTS-02 received Orphan Drug Designation from the U.S. FDA in 2019 and is currently being evaluated in Phase II clinical trials for patients with ACC.
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