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ALN-PCSSC

Phase 1

Hypercholesterolemia | Small molecule | Metabolic |Alnylam Pharmaceuticals, Inc.|Last Updated: Dec 17, 2015

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_CONTROLLED
Total Trials1
Total Enrollment70
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02314442A Phase 1 Study of an Investigational Drug, ALN-PCSSC, in Subjects With Elevated Low Density Lipoprotein Cholesterol (LDL-C)PHASE1 COMPLETED 70Dec 1, 2014Nov 1, 2015Dec 17, 20152 United Kingdom
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Study Endpoints
Primary Endpoints
The safety of ALN-PCSSC evaluated by the proportion of subjects experiencing adverse events (AEs), serious adverse events (SAEs), and AEs leading to study drug discontinuation
Part 1 (SAD phase): up to 180 days post dose; Part 2 (MD) phase: up to 180 days post final dose
Secondary Endpoints
The pharmacokinetics (PK) of ALN-PCSSC (Cmax, tmax, AUC, t1/2)
Part 1 (SAD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 42, 56 days post-dose; Part 2 (MD) phase: predose, 0.5,1,2,4,6,8,12,24,48,72,96 hours; 7, 14, 21, 28, 29, 35, 42, 56, 84 days post-dose
The effect of ALN-PCSSC on serum levels of LDL-C
Through the sooner of final follow up visit or 180 days post final dose
The effect of ALN-PCSSC on plasma levels of PCSK9
Through the sooner of final follow up visit or 180 days post final dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingSINGLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
ALN-PCSSCACTIVE_COMPARATOR -
Sterile Normal Saline (0.9% NaCl)PLACEBO_COMPARATOR -
Interventions
NameTypeDescription
ALN-PCSSCDRUGSingle or multiple doses of ALN-PCSSC by subcutaneous (sc) injection
Sterile Normal Saline (0.9% NaCl)DRUGcalculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 60 Years
SexALL
Healthy VolunteersYes
Study Sites2

Inclusion Criteria: * Adequate complete blood counts, liver and renal function * Female subjects must be of non-childbearing potential; e.g. post-menopausal or pre-menopausal with surgical sterilization * Male subjects agree to use appropriate contraception * Willing to provide written informed con...

Countries:United Kingdom
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