Silence Therapeutics: Positive Topline Data in Phase 1 in High Lipoprotein(a)

 Silence Therapeutics plc, Nasdaq: SLN (“Silence” or “the Company”), a leader in the discovery, development and delivery of novel short interfering ribonucleic acid (“siRNA”) therapeutics for the treatment of diseases with significant unmet medical need, today announced positive topline results in its phase 1 single-ascending dose study of SLN360, an siRNA targeting lipoprotein(a) (“Lp(a)”), in healthy adults with high Lp(a).

High Lp(a), defined as ≥ 50 mg/dL (c.125nmol/L), affects approximately 20% of the world’s population and is a genetic risk factor for cardiovascular disease. There are no approved medicines that selectively lower Lp(a). SLN360 is a siRNA that is designed to lower Lp(a) production by targeting messenger RNA transcribed from the LPA gene.

“These first-in-human data for SLN360, which align with our pre-clinical findings, reinforce our confidence in its potential to substantially lower Lp(a) levels with long-lasting action and address a major unmet need in cardiovascular disease,” said Giles Campion, M.D., EVP, Head of R&D and Chief Medical Officer at Silence. “More broadly, siRNA is proving to be a powerful modality for treating genetic conditions, both common and rare, by precisely engaging targets that have previously been considered ‘undruggable’.”

This phase 1 study evaluated the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics of SLN360 at escalating doses in 32 adults with plasma concentrations at screening of Lp(a) ≥150 nmol/L (approximately ≥ 60 mg/dL) with no known cardiovascular disease. Individuals were randomly assigned to receive a single subcutaneous dose of SLN360 (30 mg, 100 mg, ≤ 300 mg or ≤ 600 mg) or placebo and were observed for up to 150 days.

https://www.bakersfield.com/ap/news/silence-therapeutics-announces-positive-topline-data-in-sln360-phase-1-single-ascending-dose-study-in/article_c2a1b40f-7eba-5297-a24a-bad712f185b3.html