Silence Therapeutics' siRNA Candidate Reduces Plasma Iron Levels, Early-Stage Study Shows

Silence Therapeutics plc SLN has announced topline data from GEMINI Phase 1 study evaluating SLN124 for iron-loading anemia conditions, thalassemia, and myelodysplastic syndrome (MDS).
SLN124 is a short interfering ribonucleic acid (siRNA) that targets TMPRSS6.
The single-ascending dose study evaluated the safety and tolerability of SLN124 in 24 healthy volunteers.
Pharmacokinetic parameters and pharmacodynamic biomarkers of iron metabolism were also measured to assess the reduction in iron.
Initial data from the study showed all doses of SLN124 were generally well-tolerated with no serious or severe treatment-emergent adverse events.
The adverse events did not appear to be dose-dependent, and the majority were mild, including transient injection site reactions, which resolved without intervention.
Up to a four-fold increase in average hepcidin and a 50% reduction in plasma iron levels were also observed after a single dose of SLN124.
Hepcidin is a protein that acts as a key regulator of the entry of iron into the circulation in mammals.
Effects on hepcidin and iron appeared to be dose-dependent and were still observed at the end of the 8-week study at all dose levels.
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