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Silence Therapeutics reveals positive data from SLN124 study
19th May 2021 10:20
(Sharecast News) - Silence Therapeutics announced positive topline data from the 'GEMINI' phase 1 study of its wholly-owned product candidate 'SLN124' in healthy volunteers on Wednesday.
The AIM-traded firm said SLN124, a short interfering ribonucleic acid 'siRNA' which targets TMPRSS6, was in development for the treatment of iron-loading anemia conditions, thalassemia and myelodysplastic syndrome (MDS).
It said the GEMINI phase 1, randomised, double-blind, placebo-controlled, single-ascending dose study evaluated the safety and tolerability of SLN124 in 24 healthy volunteers, with 18 taking an active dose and six receiving placebo.
Pharmacokinetic parameters and pharmacodynamic biomarkers of iron metabolism were also measured to assess 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, or adverse events leading to withdrawal.
The company said treatment emergent adverse events did not appear to be dose dependent, adding that the majority were mild, including transient injection site reactions which resolved without intervention.
Notably, up to an approximate four-fold increase in average hepcidin and a 50% reduction in plasma iron levels were also observed after a single dose of SLN124.
Effects on hepcidin and iron appeared to be dose dependent, and were still observed at the end of the eight-week study at all dose levels, indicating a sustained and long duration of action.
Silence said the clinical data supported preclinical findings which demonstrated SLN124 effectively improved red blood cell production and reduced anemia by increasing levels of hepcidin, which is a key natural regulator of iron balance and distribution in the body.
The company said it expected to measure red blood cell production and effects on anemia in the ongoing 'GEMINI II' phase 1 study of SLN124 in people with thalassemia and MDS, who, unlike healthy volunteers, had significantly elevated iron levels.
"These data represent the first clinical data from our 'mRNAi GOLD' platform and underscore the promising potential for our technology to deliver precision medicines," said president and chief executive officer Mark Rothera.
"We look forward to further data in patients anticipated from both of our wholly owned clinical programmes later this year - the GEMINI II study of SLN124 for iron-loading anemia conditions, and the APOLLO study of SLN360 for cardiovascular disease due to high lipoprotein(a)."
Silence said it expected to present full data from the GEMINI phase 1 study at an "appropriate scientific meeting" later in the year.
In addition, it said it was planning to report data from the single-ascending dose portion of the ongoing GEMINI II phase 1 study in the second half of the year.
SLN124 has orphan drug designation for both conditions, and rare pediatric disease designation for beta thalassemia.
At 0920 BST, shares in Silence Therapeutics were up 1.45% at 630p.
The AIM-traded firm said SLN124, a short interfering ribonucleic acid 'siRNA' which targets TMPRSS6, was in development for the treatment of iron-loading anemia conditions, thalassemia and myelodysplastic syndrome (MDS).
It said the GEMINI phase 1, randomised, double-blind, placebo-controlled, single-ascending dose study evaluated the safety and tolerability of SLN124 in 24 healthy volunteers, with 18 taking an active dose and six receiving placebo.
Pharmacokinetic parameters and pharmacodynamic biomarkers of iron metabolism were also measured to assess 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, or adverse events leading to withdrawal.
The company said treatment emergent adverse events did not appear to be dose dependent, adding that the majority were mild, including transient injection site reactions which resolved without intervention.
Notably, up to an approximate four-fold increase in average hepcidin and a 50% reduction in plasma iron levels were also observed after a single dose of SLN124.
Effects on hepcidin and iron appeared to be dose dependent, and were still observed at the end of the eight-week study at all dose levels, indicating a sustained and long duration of action.
Silence said the clinical data supported preclinical findings which demonstrated SLN124 effectively improved red blood cell production and reduced anemia by increasing levels of hepcidin, which is a key natural regulator of iron balance and distribution in the body.
The company said it expected to measure red blood cell production and effects on anemia in the ongoing 'GEMINI II' phase 1 study of SLN124 in people with thalassemia and MDS, who, unlike healthy volunteers, had significantly elevated iron levels.
"These data represent the first clinical data from our 'mRNAi GOLD' platform and underscore the promising potential for our technology to deliver precision medicines," said president and chief executive officer Mark Rothera.
"We look forward to further data in patients anticipated from both of our wholly owned clinical programmes later this year - the GEMINI II study of SLN124 for iron-loading anemia conditions, and the APOLLO study of SLN360 for cardiovascular disease due to high lipoprotein(a)."
Silence said it expected to present full data from the GEMINI phase 1 study at an "appropriate scientific meeting" later in the year.
In addition, it said it was planning to report data from the single-ascending dose portion of the ongoing GEMINI II phase 1 study in the second half of the year.
SLN124 has orphan drug designation for both conditions, and rare pediatric disease designation for beta thalassemia.
At 0920 BST, shares in Silence Therapeutics were up 1.45% at 630p.