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ALN-PCS02

Phase 1

Elevated LDL-Cholesterol (LDL-C) | Small molecule | Metabolic |Alnylam Pharmaceuticals, Inc.|Last Updated: Oct 12, 2012

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
RandomizedPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment32
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01437059Trial to Evaluate Safety and Tolerability of ALN-PCS02 in Subjects With Elevated LDL-Cholesterol (LDL-C)PHASE1 COMPLETED 32Sep 1, 2011Sep 1, 2012Oct 12, 20122 United Kingdom
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Study Endpoints
Primary Endpoints
The proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs) and study drug discontinuation.
Up to 28 days
Secondary Endpoints
Pharmacokinetics (PK) of ALN-PCS02 (Cmax, tmax, t1/2, AUC0-last, CL).
Up to 180 days
Effect of ALN-PCS02 on Circulating PCSK9 Levels (Determination of % Lowering of PCSK9 to pretreatment/Baseline PCSK9 Level).
Up to 28 days
Effect of ALN-PCS02 on Circulating LDL-c Levels (Determination of % Lowering of LDL-c to pretreatment/Baseline LDL-c Level).
Up to 28 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-PCS02ACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-PCS02DRUGDose levels between 15 and 400 μg/kg by intravenous (IV) infusion
Sterile Normal Saline (0.9% NaCl)DRUGCalculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites2

Inclusion Criteria: * Elevated LDL-C of \>3.0 mmol/L and \<5.7 mmol/L * Fasting triglyceride concentration ≤2.8 mmol/L * Body weight \>60.0 kg; body mass index (BMI) between 19.00 kg/m2 and \<35.00 kg/m2 * Adequate blood counts, liver and renal function * May not received any lipid lowering drug/ag...

Countries:United Kingdom
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