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ALN-PCSSC

Phase 2

Atherosclerotic Cardiovascular Disease | Small molecule | Metabolic |Novartis AG|Last Updated: May 17, 2019

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 Trials1
Total Enrollment501
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02597127Trial to Evaluate the Effect of ALN-PCSSC Treatment on Low Density Lipoprotein Cholesterol (LDL-C)PHASE2 COMPLETED 501Jan 1, 2016Jun 7, 2017May 17, 201954 United States, Canada +3
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Study Endpoints
Primary Endpoints
Percentage Change in LDL-C From Baseline to Day 180
Baseline to 180 days

Percent Change in LDL-C (beta-quantification) from Baseline to Day 180 in MITT Population

Secondary Endpoints
Percentage Change in LDL-C From Baseline to Day 90
Baseline to 90 days
Percent Change From Baseline In LDL-C Levels At Day 60, Day 120, and Day 210
Baseline, Day 60, Day 120, and Day 210
Number of Participants With an LDL-C Greater Than 80% of the Baseline Value at Day 180 and Day 210
Baseline, Day 180, Day 210
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-PCSSC 200 mg (bi-annual dosing)EXPERIMENTALALN-PCSSC 200 milligram (mg) SC administration once at Day 1
ALN-PCSSC 300 mg (bi-annual dosing)EXPERIMENTALALN-PCSSC 300 mg SC administration once at Day 1
ALN-PCSSC 500 mg (bi-annual dosing)EXPERIMENTALALN-PCSSC 500 mg SC administration once at Day 1
Normal Saline (bi-annual dosing)PLACEBO_COMPARATORSaline SC administration once at Day 1
ALN-PCSSC 100 mg (quarterly dosing)EXPERIMENTALALN-PCSSC 100 mg SC administration twice at Day 1 and Day 90
ALN-PCSSC 200 mg (quarterly dosing)EXPERIMENTALALN-PCSSC 200 mg SC administration twice at Day 1 and Day 90
ALN-PCSSC 300 mg (quarterly dosing)EXPERIMENTALALN-PCSSC 300 mg SC administration twice at Day 1 and Day 90
Normal Saline (quarterly dosing)PLACEBO_COMPARATORSaline SC administration twice at Day 1 and Day 90
Interventions
NameTypeDescription
ALN-PCSSCDRUGALN-PCSSC is a small interfering ribonucleic acid (RNA) that inhibits PCSK9 synthesis and is given as SC injections
Normal SalineDRUGSaline (sterile, normal, 0.9%) solution given as SC injections
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Eligibility Criteria
Age Range18 Years — 99 Years
SexALL
Healthy VolunteersNo
Study Sites54

Inclusion Criteria: 1. Male or female participants ≥18 years of age. 2. History of ASCVD or ASCVD-risk equivalents (symptomatic atherosclerosis, Type 2 diabetes, familial hypercholesterolemia, including participants whose 10-year risk of a CV event assessed by Framingham Risk Score (Framingham Risk...

Countries:United StatesCanadaGermanyNetherlandsUnited Kingdom
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