Safety and lipoprotein(a)-lowering effects of Kylo-11, a non-canonical, long-duration small interfering RNA targeting lipoprotein(a): a first-in-human, randomised, double-blind, placebo-controlled, phase 1 trial.
Ashish Sarraju, Xiaolin Du, Luping Zhou et al.
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In 30 seconds
This phase 1, randomized, double-blind, placebo-controlled trial evaluated the safety and lipoprotein(a)-lowering effects of Kylo-11 in 71 healthy adults aged 18-55 with elevated lipoprotein(a). The study found that a single dose of Kylo-11 significantly reduced serum lipoprotein(a) levels, with reductions of up to 97% at the highest dose of 600 mg over 48 weeks.
Key findings
- 53% of participants experienced adverse events, mostly grade 1-2 and unrelated to the drug.
- At 600 mg, median lipoprotein(a) reduction was 97% and -129 nmol/L.
- In cohort 7, median reductions were 96% and -208 nmol/L for baseline lipoprotein(a) >200 nmol/L.
Why it matters
Elevated lipoprotein(a) is a known risk factor for atherosclerotic cardiovascular disease. Effective lowering of lipoprotein(a) could potentially reduce cardiovascular risk in affected patients.