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AKCEA-ANGPTL3-LRx

Phase 2

Familial Chylomicronemia Syndrome | Small molecule | Rare Disease |Ionis Pharmaceuticals, Inc.|Last Updated: Feb 16, 2021

Success Probability

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Trial Design

UNCONTROLLED
Total Trials1
Total Enrollment3

FDA Designations

No designations recorded

Clinical trial landscape

AKCEA-ANGPTL3-LRx · 2 trials · 4 indications

Phase 2 2
NCT03514420Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)Familial Partial Lipodystrophy
COMPLETED4 Analytics
NCT03360747Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)Familial Chylomicronemia Syndrome
COMPLETED3 Analytics
PHASE2COMPLETED
Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Partial Lipodystrophy (FPL)
Familial Partial Lipodystrophy
NCT035144204
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PHASE2COMPLETED
Phase 2 Study of AKCEA-ANGPTL3-LRx (ISIS 703802) in Participants With Familial Chylomicronemia Syndrome (FCS)
Familial Chylomicronemia Syndrome
NCT033607473
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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.

Absolute Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline to Month 3

Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

Percent Change From Baseline to Month 3 in Fasting Triglycerides (TG)
Baseline to Month 3

Baseline was defined as the average of Day 1 predose fasting assessment and the last fasting measurement prior to Day 1 pre-dose fasting assessment. Month 3 was defined as the average of Week 13 and Week 14 fasting assessments.

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 to 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 StatesCanada
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Frequently asked questions about AKCEA-ANGPTL3-LRx

What is AKCEA-ANGPTL3-LRx used for?

AKCEA-ANGPTL3-LRx is an investigational drug being studied for the treatment of Familial Partial Lipodystrophy and Familial Chylomicronemia Syndrome, both rare diseases. It is currently in Phase 2 clinical development and is not yet approved by regulatory authorities.

What does AKCEA-ANGPTL3-LRx target?

AKCEA-ANGPTL3-LRx targets ANGPTL3, a protein involved in lipid metabolism. By targeting this protein, the drug aims to reduce lipid levels in patients with rare lipid disorders. The drug is designed to inhibit the production of ANGPTL3, which plays a role in regulating triglycerides and cholesterol.

Who is developing AKCEA-ANGPTL3-LRx?

AKCEA-ANGPTL3-LRx is being developed by Ionis Pharmaceuticals, Inc., a biopharmaceutical company focused on RNA-targeted therapies. The company's stock ticker is IONS. Ionis is conducting clinical trials to evaluate the safety and efficacy of this drug in patients with rare lipid disorders.

What phase is AKCEA-ANGPTL3-LRx in?

AKCEA-ANGPTL3-LRx is in Phase 2 clinical development. It is an investigational drug, meaning it has not been approved by regulatory agencies. The drug is being studied in patients with Familial Partial Lipodystrophy and Familial Chylomicronemia Syndrome to assess its safety and potential therapeutic effects.

What clinical trials is AKCEA-ANGPTL3-LRx in?

AKCEA-ANGPTL3-LRx has been studied in two completed Phase 2 clinical trials. One trial, NCT03360747, enrolled 3 participants with Familial Chylomicronemia Syndrome in Canada. Another trial, NCT03514420, enrolled 4 participants with Familial Partial Lipodystrophy in the United States. Both trials are completed.

Is AKCEA-ANGPTL3-LRx the same as ISIS 703802?

Yes, AKCEA-ANGPTL3-LRx is also known as ISIS 703802. The drug is referred to by both names in clinical trial records. It is being developed by Ionis Pharmaceuticals for the treatment of rare lipid disorders, including Familial Partial Lipodystrophy and Familial Chylomicronemia Syndrome.