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AMY-101 is a peptidic inhibitor of the central complement component C3, based on the compstatin analog Cp40. It acts by selectively binding to and inhibiting C3, thereby blocking activation of the complement cascade and preventing complement-mediated inflammation and cell lysis. This mechanism targets diseases where excessive complement activation plays a pathogenic role, such as paroxysmal nocturnal hemoglobinuria (PNH), complement 3 glomerulopathy (C3G), gingivitis, periodontitis, and severe COVID‑19 pneumonia. AMY‑101 has shown superior pharmacologic properties compared to earlier compstatin analogs, including higher binding affinity for C3 and an extended half-life. The drug is under clinical investigation for several indications[1][2][3][4][5][6].
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