Discovery and Development of the Oral Complement Factor D Inhibitor ACH-4471.

CONCLUSION: ACH-4471 is well tolerated and demonstrates meaningful improvement in clinical efficacy endpoints for the treatment of PNH. Because CFD is not required for activation of either the CP or LP, selective CFD inhibition by ACH-4471 presents a favorable therapeutic approach to modulating complement activity that leaves intact the effector functions following CP and LP activation and thus poses a lower risk of bacterial infection than other complement-directed approaches. PMID: 31573880 [PubMed - as supplied by publisher]
Source: Current Medicinal Chemistry - Category: Chemistry Authors: Tags: Curr Med Chem Source Type: research