Comprehensive Report on ALXN2080 (Zoracopan): An Investigational Complement Factor D Inhibitor

1. Executive Summary

ALXN2080, also known by the International Nonproprietary Name (INN) Zoracopan, is an orally administered small molecule inhibitor of Complement Factor D (CFD) under development by Alexion, AstraZeneca Rare Disease. Originating from Alexion Pharmaceuticals, ALXN2080 targets the alternative pathway of the complement system, a critical component of innate immunity whose dysregulation is implicated in numerous diseases. As a New Molecular Entity, ALXN2080 represents a novel therapeutic candidate.

The drug has successfully completed a series of Phase 1 clinical trials in healthy volunteers. These include a first-in-human study (NCT05428696) evaluating single and multiple ascending doses, which established initial safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD), and two dedicated drug-drug interaction (DDI) studies (NCT06160414 and NCT06173596). These DDI studies assessed interactions with commonly prescribed medications (rosuvastatin, metformin) and potent CYP450 enzyme modulators (itraconazole, fluconazole, carbamazepine), providing crucial data for future development in patient populations. The rapid completion of these foundational studies between late 2022 and mid-2024 suggests an efficient early clinical program without major reported safety or PK impediments.