AstraZeneca's Oral PCSK9 Inhibitor AZD0780 Cleared for Phase III Trial by CDSCO Panel

AZD0780 is a novel oral PCSK9 inhibitor—a small molecule drug designed to reduce low-density lipoprotein cholesterol (LDL-C) levels. Unlike existing PCSK9 inhibitors such as alirocumab and evolocumab, which are injectable biologics (monoclonal antibodies), AZD0780 is administered orally, offering a potentially more convenient treatment option for patients with cardiovascular risk.

The drug works by binding to a specific pocket in the C-terminal domain of the PCSK9 (Proprotein Convertase Subtilisin/Kexin Type 9) protein. This binding inhibits the interaction between PCSK9 and the LDL receptor (LDLR), thereby promoting the recycling of LDLRs to the cell surface. The result is enhanced clearance of LDL-C from the bloodstream, leading to lower cholesterol levels.

Following a detailed review, the expert panel found the trial design acceptable and recommended the grant of permission to proceed with the Phase III study as proposed by the company.

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