Silence Thalassemia Hopeful Shows Favorable Safety Profile In Early Stage
- Silence Therapeutics plc announced preliminary results from the single dose component of the GEMINI II phase 1 study of SLN124 in 24 adults with non-transfusion-dependent thalassemia.
- The primary objective of the single dose arm was to evaluate the safety and tolerability of SLN124 subcutaneous dosing (1.0, 3.0, and 6.0 mg/kg) in alpha/beta-thalassemia patients.
- Following a single dose, there were no serious adverse events, severe treatment-emergent adverse events (TEAEs) that were SLN124 related, or TEAEs leading to withdrawal.
- No dose-limiting toxicities or drug-related liver injuries were observed.
- Pharmacokinetic (PK) parameters and pharmacodynamic (PD) biomarkers of iron metabolism are being evaluated in the study's ongoing multiple dose arm, which is anticipated to read out next year.
- SLN124 is a gene 'silencing' therapy – one that is designed to temporarily block a specific gene's message that would otherwise trigger an unwanted effect. In this case, SLN124 aims to temporarily 'silence' TMPRSS6, a gene that prevents the liver from producing a particular hormone that controls iron levels in the body – hepcidin.
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