Courier | It can reduce the important risk factor of cardiovascular disease by 98%, and the clinical results of siRNA therapy phase 1 are positive
Silence Therapeutics today announced that it has obtained positive results in a single-dose phase 1 clinical trial of small interfering RNA (siRNA) therapy SLN360. SLN360 is a siRNA therapy that targets lipoprotein (a)-Lp(a).
High Lp(a), defined as Lp(a) levels in plasma≥ 50 mg/dL, affects about 20% of the world's population and is a genetic risk factor for cardiovascular disease. There are currently no approved drugs that selectively reduce Lp(a). SLN360 is a siRNA designed to reduce the production of Lp(a) by targeting mRNA that degrades transcription of the LPA gene.
The safety, tolerability, pharmacokinetics (PK), and pharmacodynamic profile of the increasing dose of SLN360 were assessed in 32 adults with no known cardiovascular disease in plasma Lp(a) ≥ 150 nmol/L (≥ approximately 60 mg/dL) and no known cardiovascular disease.
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The primary safety endpoint was to assess adverse events that occurred after treatment. No clinically important safety issues were identified. Mild adverse events were observed at the injection site, with the highest dose group being the most significant.
The key efficacy assessment is the percentage change in Lp(a) from baseline. SLN360 dose-dependent reduction of Lp(a) levels up to 98% and sustainable reduction of Lp(a) levels up to 81% over 150 days. The study follow-up period was extended from 150 days to 365 days to further assess the duration of action.
"The first human trial data for SLN360 are consistent with our preclinical findings, reinforcing our confidence that it will significantly reduce Lp(a) levels and have a lasting effect." Silence Chief Medical Officer and Head of Research and Development Dr Giles Campion said, "SiRNA is proving to be a powerful therapeutic model for treating both common and rare genetic diseases by acting precisely on targets previously considered 'incurable'. ”
Resources:
[1] Silence Therapeutics Announces Positive Topline Data in SLN360 Phase 1 Single-Ascending Dose Study in Healthy Adults with High Lipoprotein(a). Retrieved February 9, 2022, from Silence Therapeutics - Silence Therapeutics Announces Positi...
