Prevention & lipidsRandomised controlled trial
An Oral PCSK9 Inhibitor for Treatment of Hypercholesterolemia
- Randomised controlled trial
- phase 2
- double-blind
- placebo-controlled
- multicentre
Authors' conclusion
AZD0780 demonstrated robust, dose-dependent reductions in LDL-C with a favorable safety and tolerability profile supporting further development of this once daily, oral treatment.
Study context
- Population
- Patients with hypercholesterolemia on background moderate-to-high-intensity statin (low-density lipoprotein cholesterol (LDL-C) ≥70 and <190 mg/dL, TG <400 mg/dL), with or without ezetimibe426 started treatment
- Participants
- 428 patients
- Comparison
- AZD0780 vs placebo
- Primary endpoint
- Percent change of LDL-C from baseline
- Follow-up
- Week 12
Primary result
As reported in the abstract
Placebo-corrected difference in LS mean % change of LDL-C
Each group vs placeboPlacebo-corrected difference, % (95% CI)
AZD0780 1 mg
−35.3(−43.6% to −26.9%)
AZD0780 3 mg
−37.9(−46.3% to −29.5%)
AZD0780 10 mg
−45.2(−53.5% to −36.9%)
AZD0780 30 mg
−50.7(−59.0% to −42.4%)
Further findings
LDL-C efficacy by baseline statin intensity (subgroup)
- Baseline statin use, moderate vs high intensity, did not alter AZD0780 efficacy
Reaching ACC/AHA LDL-C goal for high-risk patients
- Proportion increased in a dose-proportional manner
Safety
Adverse events
Adverse events
- Adverse events compared similarly
Original publication
An Oral PCSK9 Inhibitor for Treatment of Hypercholesterolemia. J Am Coll Cardiol. Published 31 March 2025. doi:10.1016/j.jacc.2025.03.499
Summarised from the published abstract; every number is checked against it