Prevention & lipidsRandomised controlled trial
Muvalaplin, an Oral Small Molecule Inhibitor of Lipoprotein(a) Formation
- Phase 1 randomised controlled trial
- double-blind
- parallel-design
- placebo-controlled
- single-ascending dose and multiple-ascending dose
- single-centre
Authors' conclusion
Muvalaplin, a selective small molecule inhibitor of Lp(a) formation, was not associated with tolerability concerns and lowered Lp(a) levels up to 65% following daily administration for 14 days.
Study context
- Population
- Healthy participants (any Lp(a) level) and patients with Lp(a) ≥30 mg/dL, 1 site in the Netherlands105 completed
- Participants
- 114 participants
- Comparison
- Muvalaplin vs placebo
- Single ascending dosen = 55
- Multiple ascending dosen = 59
- Primary endpoint
- Safety, tolerability, pharmacokinetics and exploratory pharmacodynamic biomarkers
Primary result
As reported in the abstract
Lp(a) lowering (multiple ascending dose) · 14 days
Maximum placebo-adjusted Lp(a) reduction
63% to 65%
Participants reaching Lp(a) <50 mg/dL
93%
Further findings
Pharmacokinetics (30 mg to 800 mg daily for 14 days)
- Increasing plasma concentrations; half-life
- 70 to 414 hours
Safety
Tolerability, adverse effects and plasminogen
- No tolerability concerns or clinically significant adverse effects
- No clinically significant changes in plasminogen levels or activity
Original publication
Muvalaplin, an Oral Small Molecule Inhibitor of Lipoprotein(a) Formation. JAMA. Published 19 September 2023. doi:10.1001/jama.2023.16503
Summarised from the published abstract; every number is checked against it