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Pelacarsen

Phase 1

Healthy Participants | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Apr 20, 2022

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_CONTROLLEDBiomarker
Total Trials1
Total Enrollment29
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT05337878A Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pelacarsen (ISIS 681257) in Healthy Japanese ParticipantsPHASE1 COMPLETED 29 — — Oct 15, 2018Dec 18, 2019Apr 20, 20221 United States
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Study Endpoints
Primary Endpoints
SAD: Percentage of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Evaluation Parameters
Up to Day 90
SAD: Percentage of Participants With Adverse Events
Up to Day 90
SAD: Percentage of Participants With Serious Adverse Events
Up to Day 90
MAD: Percentage of Participants With Clinically Significant Changes From Baseline in Clinical Laboratory Evaluation Parameters
Up to Day 204
MAD: Percentage of Participants With Adverse Events
Up to Day 204
MAD: Percentage of Participants With Serious Adverse Events
Up to Day 204
Secondary Endpoints
Maximum Observed Drug Concentration (Cmax) in Plasma After Single Ascending Dose of Pelacarsen
Day 1: Pre-dose; 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 up to 168 hours post-dose
Area Under Curve (AUC) From Time Zero to the Last Quantifiable Concentration (AUClast) in Plasma After Single Ascending Dose of Pelacarsen
Day 1: Pre-dose; 0.5, 1, 1.5, 2, 3, 4, 6, 8, 12 up to 168 hours post-dose
Maximum Observed Drug Concentration (Cmax) in Plasma After Multiple Doses of Pelacarsen
Days 1 and 85: Pre-dose; 1, 2, 4, 8, 24 up to 168 hours post-dose
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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelSEQUENTIAL
PurposeOTHER
Treatment Arms
ArmTypeDescription
SAD: PlaceboPLACEBO_COMPARATORSingle dose of Pelacarsen-matching placebo administered by SC injection on Day 1 of single-dose treatment period.
SAD: Pelacarsen 20 milligrams (mg)EXPERIMENTALSingle dose of Pelacarsen, 20 mg, administered by SC injection on Day 1 of single-dose treatment period.
SAD: Pelacarsen 40 mgEXPERIMENTALSingle dose of Pelacarsen, 40 mg, administered by SC injection on Day 1 of single-dose treatment period.
SAD: Pelacarsen 80 mgEXPERIMENTALSingle dose of Pelacarsen, 80 mg, administered by SC injection on Day 1 of single-dose treatment period.
MD: PlaceboPLACEBO_COMPARATORMultiple doses of Pelacarsen-matching placebo administered by SC injection every 4 weeks, on Days 1, 29, 57 and 85 of multiple-dose treatment period.
MD: Pelacarsen 80 mgEXPERIMENTALMultiple doses of Pelacarsen, 80 mg, administered by SC injection every 4 weeks, on Days 1, 29, 57 and 85 of multiple-dose treatment period.
Interventions
NameTypeDescription
PlaceboDRUGPelacarsen-matching placebo administered by SC injection.
PelacarsenDRUGPelacarsen administered by SC injection.
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: 1. Provided written informed consent (signed and dated) and any authorizations required by local law and was able to comply with all study requirements. 2. Male and female of first-, second- or third-generation Japanese participants. 3. Japanese healthy or obese male and female ...

Countries:United States
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