Expert: Oral small molecule PCSK9 inhibitor

Everyday: Lowers cholesterol with a pill instead of injection

Targets: ["PCSK9"]

Primary EP: [{"id":"ldlc-change","name":"Percent change from baseline in LDL-C at Week 12","type":"PRIMARY","unit":"%","results":[{"label":"Placebo +4.1%","value":4.08,"arm_id":"placebo"},{"label":"1mg: -31.2% (p

Safety: AEs comparable: 38.2% AZD0780 vs 32.6% placebo. No new safety signals.

Oral small-molecule PCSK9 inhibitor (cholesterol synthesis inhibitor). AZD0780 blocks PCSK9 production at the mRNA level in hepatocytes, unlike mAbs (Repatha, Praluent) or siRNA (Leqvio) which target circulating PCSK9 protein. Once-daily oral pill vs injections. Phase 2b PURSUIT: 60-65% LDL-C reduction at 30mg dose (comparable to injectable PCSK9 mAbs). Phase 3 AZURE program initiated 2025. Competitive landscape: MRK oral PCSK9 (enlicitide/MK-0616) is the main head-to-head competitor, also in Phase 3. Both aim to replace injectable PCSK9 therapies. Peak sales estimate: USD5B (oral convenience drives much larger addressable market than injectable PCSK9s).