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sapablursen

Phase 2

Phlebotomy Dependent Polycythemia Vera | Small molecule | Hematology |Ionis Pharmaceuticals, Inc.|Last Updated: Jun 1, 2026

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
RandomizedCONTROLLEDBiomarker
Total Trials1
Total Enrollment50
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05143957A Study to Evaluate Sapablursen (Formerly ISIS 702843, IONIS-TMPRSS6-LRx) in Patients With Polycythemia VeraPHASE2 ACTIVE NOT_RECRUITING 50 — — Dec 30, 2021Jun 1, 2026Jun 1, 202613 United States, Australia +3
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Study Endpoints
Primary Endpoints
Change in the frequency of phlebotomy comparing Baseline with the last 20 weeks of the 37 week Treatment Period
Week 17 to Week 37
Secondary Endpoints
Proportion of patients achieving a reduction in the frequency of phlebotomy by ≥ 30%, ≥ 50%, ≥ 75% and ≥ 90% comparing Baseline with the last 20 weeks of the 37-week Treatment Period
Week 17 to Week 37
Change in the Myeloproliferative Neoplasm Symptom Assessment Form-Total Symptom Score (MPN-SAF-TSS) From Baseline to Week 37
Baseline up to Week 37
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Study Design & Arms
AllocationRANDOMIZED
MaskingNONE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Sapablursen Dose Level 1EXPERIMENTALSapablursen will be administered by SC injection every 4 weeks.
Sapablursen Dose Level 2EXPERIMENTALSapablursen will be administered by SC injection every 4 weeks
Interventions
NameTypeDescription
sapablursenDRUGSapablursen will be administered by SC injection.
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Eligibility Criteria
Age Range18 Years — 90 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria 1. Meet modified World Health Organization (WHO) 2016 diagnostic criteria for polycythemia vera (PV) at the time of clinical diagnosis 2. Participant must be phlebotomy dependent. 3. Participants do not need to be on cytoreductive therapy and do not need to have been previously t...

Countries:United StatesAustraliaCanadaPolandUnited Kingdom
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Recent Changes (Last 90 Days)
LOWJun 2, 2026NCT05143957lastUpdatePostDate: changed
LOWJun 2, 2026NCT05143957lastUpdatePostDate: changed
LOWJun 2, 2026NCT05143957lastUpdatePostDate: changed
LOWMay 26, 2026NCT05143957primaryCompletionDate: changed
LOWMay 24, 2026NCT05143957studyFirstPostDate: changed