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inclisiran

Phase 3

Hypercholesterolemia | Small molecule | Metabolic |Novartis AG|Last Updated: Dec 30, 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_CONTROLLEDDMCBiomarker
Total Trials3
Total Enrollment1,007
FDA Designations
No designations recorded
Clinical Trials (3)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05763875Efficacy and Safety of Inclisiran as Monotherapy in Patients With Primary Hypercholesterolemia Not Receiving Lipid-lowering Therapy.PHASE3 COMPLETED 350Mar 15, 2023Jun 20, 2024Oct 16, 202542 United States, Colombia +3
NCT04765657Study of Efficacy and Safety of Inclisiran in Asian Participants With Atherosclerotic Cardiovascular Disease (ASCVD) or ASCVD High Risk and Elevated Low Density Lipoprotein Cholesterol (LDL-C)PHASE3 ACTIVE NOT_RECRUITING 345Mar 1, 2021Dec 28, 2026Dec 30, 202545 China, Singapore +2
NCT04666298Study of Efficacy and Safety of Inclisiran in Japanese Participants With High Cardiovascular Risk and Elevated LDL-CPHASE2 COMPLETED 312Jan 29, 2021Oct 19, 2022Jun 20, 202442 Japan
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Study Endpoints
Primary Endpoints
Percentage Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150
Baseline, Day 150

Percentage change in LDL-C from Baseline (day 1) to Day 150, Inclisiran arm versus Ezetimibe and placebo. There were two estimands of interest in comparing efficacy of inclisiran as monotherapy against that of placebo or ezetimibe that differ on the treatment of interest used for each and the management of intercurrent events as follows: * Monotherapy Estimand: Inclisiran as monotherapy compared to the use of comparator. This estimand uses a hypothetical strategy where participants who permanently discontinued treatment, died or used other LLTs were handled in a hypothetical scenario of what would have happened if the intercurrent event did not happen. * Treatment-policy Estimand: Inclisiran as monotherapy compared to the use of comparator with or without other lipid lowering therapies (LLTs) added. This estimand ignored the use of other LLTs and treatment discontinuation. Deaths (if any) were handled as an unfavorable outcome using a composite variable strategy.

Core: Percentage change in low- density lipoprotein cholesterol (LDL-C)
Baseline, Day 330

Superiority of inclisiran compared to placebo in reducing LDL-C from baseline to Day 330

Extension: Number of participants with Adverse Events
Day 360 until study completion, an average of 3 years

Evaluation of the safety and tolerability of inclisiran, treatment emergent Adverse events and Serious Adverse Events

Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) to Day 180
Baseline, Day 180

Percent change from baseline in LDL-C was calculated to evaluate the effect of inclisiran at Day 180. Difference between different inclisiran dose groups and the placebo group in percentage change in LDL-C levels from baseline to Day 180 were calculated to capture both, the effect of the study drug and the effect of additional medications, mirroring the conditions in clinical practice. An MMRM (Mixed-effect Model with Repeated Measurement) was used as the primary analysis model, with treatment group, visits, interaction between visits and treatment groups, current use of statins or other lipid-modifying therapies as fixed effects, and baseline LDL-C as a continuous covariate.

Secondary Endpoints
Absolute Change in LDL-C From Baseline to Day 150
Baseline, Day 150
Percentage Change in Proprotein Convertase Subtilisin/Kexin Type 9 (PCSK9) From Baseline to Day 150
Baseline, Day 150
Percentage Change in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) From Baseline to Day 150
Baseline, Day 150
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Study Design & Arms
AllocationRANDOMIZED
MaskingTRIPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
InclisiranEXPERIMENTALInclisiran s.c and Placebo p.o
EzetimibeACTIVE_COMPARATORPlacebo s.c. and Ezetimibe p.o.
PlaceboPLACEBO_COMPARATORPlacebo s.c. and Placebo p.o.
inclisiran sodium 300 mgEXPERIMENTALSubcutaneous injection
300 mg inclisiran sodiumEXPERIMENTALSubcutaneous injection
200 mg inclisiran sodiumEXPERIMENTALSubcutaneous injection
100 mg inclisiran sodiumEXPERIMENTALSubcutaneous injection
Interventions
NameTypeDescription
InclisiranDRUG284 mg (equivalent to 300 mg inclisiran sodium) subcutaneous injection given on Day 1 and Day 90
EzetimibeDRUG10 mg over-encapsulated tablet taken once a day from Day 1 through Day 149
Matching Placebo for InclisiranDRUG0mg placebo injection solution for subcutaneous injection on Day 1 and Day 90
Matching Placebo for EzetimibeDRUG0mg over-encapsulated placebo tablet taken once a day from Day 1 through Day 149
inclisiran sodiumDRUGSubcutaneously injected on Day 1, 90 and 270 (Core Part). Subcutaneously injected on Day 360 and every 6 months thereafter until EOS visit (Extension Part)
PlaceboDRUGSubcutaneously injected on Day 1, 90, and 270.
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Eligibility Criteria
Age Range18 Years — 75 Years
SexALL
Healthy VolunteersNo
Study Sites42

Inclusion Criteria at screening: * informed consent signed prior to participation in study * fasting LDL-C of \>= 100 mg/dL but \< 190 mg/dL * fasting triglycerides \<= 400 mg/dL * 10-year ASCVD risk score \< 7.5% * not on any lipid-lowering therapy within 90 days of screening Key Exclusion Criter...

Countries:United StatesColombiaGermanyHungaryMexicoChinaSingaporeSouth KoreaTaiwanJapan
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Recent Changes (Last 90 Days)
LOWMay 26, 2026NCT04765657primaryCompletionDate: changed
LOWMay 24, 2026NCT04765657studyFirstPostDate: changed