AstraZeneca’s oral PCSK9 inhibitor halves cholesterol in phase 2 trial
AstraZeneca's oral PCSK9 inhibitor AZD0780 demonstrated significant LDL cholesterol (LDL-C) reduction in the PURSUIT Phase IIb trial27.
Key Findings
- LDL-C Reduction:
AZD0780 achieved a 50.7% reduction in LDL-C over 12 weeks when administered with standard-of-care statin therapy, compared to placebo7.
- Target Achievement:
84% of participants receiving AZD0780 30mg reached their recommended LDL-C target (<70 mg/dL), versus only 13% in the control group7.
- Dosing:
The highest efficacy was observed with the 30mg once-daily dose7.
- Consistency:
The effectiveness was consistent across moderate and high-intensity statin doses7.
Safety Profile
AZD0780 was generally well-tolerated:
- Adverse events were comparable between treatment (38.2%) and placebo (32.6%) groups7.
- Discontinuation rates were similar (1.5% for AZD0780 vs 2.3% for placebo)7.
Significance
This oral PCSK9 inhibitor represents a potential breakthrough in cardiovascular disease management:
- It offers efficacy comparable to injectable PCSK9 inhibitors currently on the market7.
- The oral administration route without fasting restrictions addresses a critical unmet need in this therapeutic area7.
- It could substantially expand the addressable market beyond what existing injectables have captured7.
Next Steps
While promising, these are mid-stage results. The path to approval requires successful completion of larger Phase III trials, likely including cardiovascular outcomes data7.
AstraZeneca's AZD0780 shows potential as a convenient once-daily oral option for patients unable to meet their LDL-C targets with statins alone, potentially redefining treatment standards in dyslipidemia management7.
Sources:
2. https://endpts.com/astrazenecas-cholesterol-pill-is-set-for-phase-3-but-lags-behind-merck-rival/
7. https://www.stocktitan.net/news/AZN/azd0780-a-novel-oral-pcsk9-inhibitor-demonstrated-significant-ldl-nryi2eb5jck4.html