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SP-420

Phase 2

Beta Thalassemia Major Anemia | Small molecule | Hematology |Icon Plc|Last Updated: Sep 24, 2025

Success Probability
Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
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Market & Valuation
rNPV $3.2B
Market Size $9.4B
Revenue Basis $1.6B
Competitors 6
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Trial Design
RandomizedCONTROLLEDDMCBiomarker
Total Trials1
Total Enrollment90
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05693909A Trial Testing SP-420 in Subjects With Transfusion-dependent β-thalassemia or Low-risk Myelodysplastic SyndromesPHASE2 RECRUITING 90 — — Sep 4, 2023May 1, 2028Sep 24, 20251 Denmark
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Study Endpoints
Primary Endpoints
To establish dose-response relationship of SP-420 for 24 weeks in the treatment of subjects with transfusion-dependent β-thalassemia
24 weeks
To assess the safety and tolerability of ascending doses of SP-420 after 12 weeks treatment of subjects with transfusion-dependent low-risk myelodysplastic syndrome
12 weeks
Secondary Endpoints
To assess the efficacy of SP-420 in clearing iron from the liver after 24 weeks treatment of subjects with transfusion-dependent β-thalassemia
24 weeks
To assess the efficacy of SP-420 in clearing iron from the liver after 12 and 48 weeks treatment of subjects with transfusion-dependent β-thalassemia
12 and 48 weeks
To assess the efficacy of SP-420 on serum (s-) ferritin
up to 48 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelSEQUENTIAL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Cohort 1a-1bEXPERIMENTALSP-420 for 48 weeks
Cohort 2a-2bEXPERIMENTALSP-420 for 48 weeks
Cohort 3a-3bEXPERIMENTALSP-420 for 48 weeks
1c-1dEXPERIMENTALSP-420 for 48 weeks
2c-2dEXPERIMENTALSP-420 for 48 weeks
3c-3dEXPERIMENTALSP-420 for 48 weeks
Interventions
NameTypeDescription
SP-420DRUGCapsules for oral intake
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Thalassemia cohorts: Inclusion criteria: * Women and men aged 18 years or older * Transfusion-dependent β-thalassemia including HbE/β-thalassemia requiring iron chelation therapy (β-thalassemia with mutation and/or multiplication of α-globin is allowed) * On a stable dose of iron chelation for at ...

Countries:Denmark
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT05693909primaryCompletionDate: changed
LOWMay 24, 2026NCT05693909studyFirstPostDate: changed