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ACH-0145548

Development stage: Unknown
Lead developer: Alexion Pharmaceuticals
Modality: Small Molecules
Administration: Oral

Overview

ACH-0145548 is an investigational oral small molecule inhibitor of the alternative pathway of the complement system, specifically targeting Factor D. Developed by Achillion Pharmaceuticals (now part of Alexion, the rare disease unit of AstraZeneca), it was designed to treat complement-mediated diseases such as paroxysmal nocturnal hemoglobinuria (PNH) and C3 glomerulopathy (C3G). The compound was evaluated in a Phase 1 first-in-human, randomized, double-blind, placebo-controlled, single ascending dose (SAD) study to assess its safety, tolerability, pharmacokinetics, and pharmacodynamics in healthy volunteers.

Targets

CFD (Complement Factor D)

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