Achillion Pharmaceuticals (ACHN +24.4%) is up on a 6x surge in volume. Shares have rallied 102% this week after it announced encouraging Phase 1 data on complement factor D inhibitor candidate ACH-5228.
Results showed that the higher doses (120 mg – 200 mg twice-daily) achieved near-complete and sustained Alternative Pathway (AP) inhibition.
Factor D, originally identified as adipsin, is an essential enzyme (serine protease) in the AP of the complement system (part of the innate immune system). Inhibiting AP may have potential in treating complement system-related disorders like paroxysmal nocturnal hemoglobinuria (PNH), C3 glomerulopathy (kidneys malfunction leading to excess levels of protein in urine) and immune complex-mediated membranoproliferative glomerulonephritis (kidney malfunction due to inflammation of tiny filters called glomeruli).
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