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SLN124

Phase 1

Non-transfusion-dependent Thalassemia | Small molecule | Other |Silence Therapeutics Plc American Depository Share|Trials Updated: Dec 19, 2025

SLN124 development status

Highest phase Phase 1
Registered trials 3 across 1 sponsor since Sep 2020

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDDMC
Total Trials2
Total Enrollment113

FDA Designations

No designations recorded

SLN124 clinical trials

SLN124 · 2 trials · 4 indications

Phase 1 2
NCT05499013Study to Assess SLN124 in Patients With Polycythemia VeraPolycythemia Vera
ACTIVE NOT_RECRUITING69 Analytics
NCT04718844A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic SyndromeNon-transfusion-dependent Thalassemia
COMPLETED44 Analytics
PHASE1ACTIVE NOT_RECRUITING
Study to Assess SLN124 in Patients With Polycythemia Vera
Polycythemia Vera
NCT0549901369
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PHASE1COMPLETED
A Study Investigate the Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Response of SLN124 in Adults With Alpha/Beta-thalassaemia and Very Low- and Low-risk Myelodysplastic Syndrome
Non-transfusion-dependent Thalassemia
NCT0471884444
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Study Endpoints

Primary Endpoints

Phase 1: Incidence of treatment-emergent adverse events (AEs)
Day 239

Safety and tolerability will be reported separately following open-label dose escalation phase and double-blind phase

Phase 1: Assessment of the number of phlebotomies at intervals
6 months prior to dosing to Day 239
Phase 2: Proportion of patients who achieve response between week 18 and week 36 (placebo controlled double blind phase)
18 to 36 weeks
Incidence of treatment-emergent adverse events
Day 84

safety and tolerability will be reported separately following single-dose administration.

Secondary Endpoints

Phase 1: Pharmacokinetic: area under the plasma concentration (AUC)
Day 127
Phase 1: Pharmacokinetic: peak plasma concentration (Cmax)
Day 127
Phase 1: Pharmacodynamic: change in haematocrit
Day 1 to Day 239
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Study Design & Arms

AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Phase 1 open-label SLN124EXPERIMENTALSLN124 for subcutaneous (s.c.) injection
Phase 2 Blinded SLN124EXPERIMENTALSLN124 for subcutaneous (s.c.) injection
Phase 2 Blinded PlaceboPLACEBO_COMPARATORSodium chloride for s.c. injection
1.0mg/kg - ThalassaemiaEXPERIMENTAL -
3.0mg/kg - ThalassaemiaEXPERIMENTAL -
6.0mg/kg - ThalassaemiaEXPERIMENTAL -
Placebo - ThalassaemiaPLACEBO_COMPARATOR -
Xmg/kg - ThalassaemiaEXPERIMENTAL -
1.0mg/kg - Myelodysplastic SyndromeEXPERIMENTAL -
3.0mg/kg - Myelodysplastic SyndromeEXPERIMENTAL -
10.0mg/kg - Myelodysplastic SyndromeEXPERIMENTAL -
Xmg/kg - Myelodysplastic SyndromeEXPERIMENTAL -
3.0mg/kg - Thalassaemia multi doseEXPERIMENTAL -
10.0mg/kg - Thalassaemia multi doseEXPERIMENTAL -
Xmg/kg - Thalassaemia multi doseEXPERIMENTAL -
3.0mg/kg - Myelodysplastic Syndrome multi doseEXPERIMENTAL -
10.0mg/kg - Myelodysplastic Syndrome multi doseEXPERIMENTAL -
Xmg/kg - Myelodysplastic Syndrome multi doseEXPERIMENTAL -
Placebo - Thalassaemia multi dosePLACEBO_COMPARATOR -
Placebo - Myelodysplastic SyndromePLACEBO_COMPARATOR -
Placebo - Myelodysplastic Syndrome multi dosePLACEBO_COMPARATOR -

Interventions

NameTypeDescription
SLN124DRUGSLN124 is a double-stranded small interfering ribonucleic acid (siRNA) targeting transmembrane protease, serine 6 (TMPRSS6) messenger ribonucleic acid (mRNA).
PlaceboDRUGsodium chloride, solution for injection
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Eligibility Criteria

Age Range18 Years to N/A
SexALL
Healthy VolunteersNo
Study Sites28

Phase 1 and Phase 2 Inclusion Criteria: * Male and female patients aged 18 years or older. * A confirmed diagnosis of PV according to the revised 2016 World Health Organization criteria: * Suitable phlebotomy history * Must agree to adhere to appropriate contraception requirements * Patients who a...

Countries:United StatesAustraliaBulgariaCanadaGermanyItalyMalaysiaPolandSpainIsraelJordanThailandUnited Kingdom
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