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AKCEA-ANGPTL3-LRx

Phase 2

Familial Partial Lipodystrophy | Small molecule | Other |Ionis Pharmaceuticals, Inc.|Last Updated: Feb 16, 2021

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
UNCONTROLLED
Total Trials1
Total Enrollment4
FDA Designations
No designations recorded
Clinical Trials (1)
NCT IDTitlePhaseStatusEnrollmentVelocityDesignStartCompletionLast UpdatedSitesCountries
NCT03514420Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)PHASE2 COMPLETED 4 — — Jun 15, 2018Aug 21, 2019Feb 16, 20211 United States
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Study Endpoints
Primary Endpoints
Percent Change From Baseline in Fasting Triglycerides Levels at End of the Treatment (Week 27)
Baseline and End of the Treatment (Week 27)

The baseline was defined as the average of Day 1 pre-dose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment.

Secondary Endpoints
Change From Baseline in Area Under the Curve (AUC) of Plasma Glucose as Assessed by Mixed Meal Test (MMT) at End of the Treatment
Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Serum Insulin as Assessed by MMT at End of the Treatment
Baseline and End of the Treatment (Week 27)
Change From Baseline in AUC of Serum C-peptide as Assessed by MMT at End of the Treatment
Baseline and End of the Treatment (Week 27)
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Study Design & Arms
AllocationNA
MaskingNONE
ModelSINGLE_GROUP
PurposeTREATMENT
Treatment Arms
ArmTypeDescription
AKCEA-ANGPTL3-LRx 20 mgEXPERIMENTALParticipants received AKCEA-ANGPTL3-LRx 20 milligrams (mg) administered every week for 26 weeks by subcutaneous (SC) injection.
Interventions
NameTypeDescription
AKCEA-ANGPTL3-LRxDRUGAKCEA-ANGPTL3-LRx solution for SC injection.
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Eligibility Criteria
Age Range18 Years — N/A
SexALL
Healthy VolunteersNo
Study Sites1

Key Inclusion Criteria: * Must give written informed consent to participate in the study. * Clinical diagnosis of familial partial lipodystrophy plus diagnosis of type 2 diabetes mellitus and hypertriglyceridemia. * Diagnosis of diabetes mellitus, made at least 6 months prior to the Screening with ...

Countries:United States
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