Silence Therapeutics' Thalassemia Hopeful Shows Favorable Safety Profile In Early Stage Study

Silence Therapeutics plc SLN 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.
Price Action: SLN shares are trading at $10.00 on the last check Thursday.