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APOC-III-L-Rx

Phase 1

Elevated Triglycerides (TG) | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: May 22, 2018

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 Enrollment56
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT02900027Safety, Tolerability, PK, and Pharmacodynamics(PD) of IONIS APOCIII-LRx in Healthy Volunteers With Elevated TriglyceridesPHASE1 COMPLETED 56Sep 1, 2016Apr 30, 2018May 22, 20181 Canada
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Study Endpoints
Primary Endpoints
To evaluate the safety and tolerability of single and multiple doses of IONIS- APOC-III-LRx - (incidence, severity, and dose-relationship of adverse effects and changes in the laboratory parameters)
Up to 183 days

The safety and tolerability of IONIS- APOC-III-LRx will be assessed by determining the incidence, severity, and dose relationship of adverse effects and changes in the laboratory parameters by dose. Safety results in subjects dosed with IONIS APOC-III-LRx will be compared with those from subjects dosed with placebo.

Secondary Endpoints
To evaluate the plasma pharmacokinetics of single and multiple doses of IONIS- APOC-III-LRx
Up to 183 days
To evaluate the urine pharmacokinetics of single and multiple doses of IONIS¬-APOC-III-LRx
Up to 183 days
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Study Design & Arms
AllocationRANDOMIZED
MaskingDOUBLE
ModelPARALLEL
PurposeOTHER
Treatment Arms
ArmTypeDescription
IONIS-APOC-III-LRxEXPERIMENTALAscending single and multiple doses of IONIS-APOC-III-LRx by subcutaneous (SC) injection
Placebo (Normal Saline)PLACEBO_COMPARATORSterile Normal Saline (0.9% NaCl) calculated volume to match active comparator
Interventions
NameTypeDescription
APOC-III-L-RxDRUGAscending single and multiple doses of IONIS-APOC-III-LRx by subcutaneous (SC) injection
Placebo ComparatorDRUGSterile Normal Saline (0.9% NaCl) calculated volume to match active comparator
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Eligibility Criteria
Age Range18 Years — 65 Years
SexALL
Healthy VolunteersYes
Study Sites1

Inclusion Criteria: * Must have given written informed consent and be able to comply with all study requirements * Healthy males or females aged 18-65 inclusive * Females must be non-pregnant and non-lactating, and either surgically sterile or post- menopausal * Males must be surgically sterile, ab...

Countries:Canada
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