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ISIS apoC-III Rx

Phase 2

Hypertriglyceridemia | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Jan 27, 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-BlindCONTROLLEDBiomarker
Total Trials1
Total Enrollment89
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT01529424Safety, Tolerability, and Pharmacokinetic Study of ISIS ApoC-III Rx in HypertriglyceridemiaPHASE2 COMPLETED 89Feb 1, 2012Jan 1, 2014Jan 27, 20227 United States, Canada
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Study Endpoints
Primary Endpoints
VLDL apoC-III
92 Days

The effect of treatment with ISIS ApoC-III Rx or Placebo on fasting total apoC-III levels.

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Study Design & Arms
AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
Group 1EXPERIMENTALNon-extensive PK/non post-prandial
Group 2aEXPERIMENTALExtensive PK
Group 2bEXPERIMENTALPost-prandial assessment
Group 3EXPERIMENTALStable dose of fibrate
Group 4EXPERIMENTALFredrickson Type 1 dyslipidemia
Interventions
NameTypeDescription
ISIS apoC-III RxDRUGDose 1
PlaceboDRUGDose 1
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites7

Inclusion Criteria: * Severe hypertriglyceridemia Exclusion Criteria: * HbA1c \>/=9.0%, type 1 diabetes, or history of outpatient insulin use for more than 2 weeks in the last year * Body mass index (BMI) \>40 kg/m2 * History of bariatric surgery or currently on weight loss drugs * Use of oral co...

Countries:United StatesCanada
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