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Pelacarsen s.c.

Phase 3

Hyperlipoproteinemia(a) | Small molecule | Other |Novartis AG|Last Updated: Feb 14, 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
RandomizedDouble-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment51
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05305664A Multicenter Trial Assessing the Impact of Lipoprotein(a) Lowering With Pelacarsen (TQJ230) on the Rate of Weekly Lipoprotein Apheresis Sessions in Patients With Hyperlipoproteinemia(a) and Established Cardiovascular Disease in GermanyPHASE3 COMPLETED 51 — — Aug 19, 2022Jan 28, 2025Feb 14, 202513 Germany
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Study Endpoints
Primary Endpoints
Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule
Over 52 Weeks

Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the rate of lipoprotein apheresis sessions in patients with hyperlipoproteinemia(a) and established CVD during 52 weeks of treatment

Secondary Endpoints
Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study)
At least 24 weeks up to Week 52
Total avoidance of lipoprotein apheresis from week 12 to week 52
Week 12 to Week 52
Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis)
52 weeks
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Pelacarsen (TQJ230)EXPERIMENTAL -
PlaceboPLACEBO_COMPARATOR -
Interventions
NameTypeDescription
Pelacarsen (TQJ230) 80 mg s.c.DRUGPelacarsen (TQJ230) 80 mg s.c. Q4W
Corresponding PlaceboDRUGPlacebo to Pelacarsen
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Eligibility Criteria
Age Range18 Years — 80 Years
SexALL
Healthy VolunteersNo
Study Sites13

Inclusion Criteria: * Patients currently undergoing lipoprotein apheresis for isolated Lp(a) on a weekly schedule in Germany for ≥ 12 months prior to screening with at least 40 sessions within the past 52 weeks prior to randomization * Lipoprotein(a) (Lp(a))\> 60 mg/dL at screening * Spontaneous pr...

Countries:Germany
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