Olezarsen

Summary

Olezarsen (CHEMBL4650438) is a phase-3 clinical-stage oligonucleotide; indicated across 2 conditions including hypertriglyceridemia and cardiovascular disorder.

At a glance

• Status: Max clinical phase 3 (not approved)
• Modality: Oligonucleotide
• Indications: 2 conditions
• Clinical trials: 11

Identifiers

Drug identity and classification

Field	Value
ChEMBL ID	CHEMBL4650438
Name	Olezarsen
Type	Oligonucleotide
Max phase	3

Also known as: Akcea-apociii-lrx, AKCEA-APOCIII-LRx, IONIS- APOCIII-LRx, IONIS-APOCIII-LRX, ISIS 678354, ISIS-678354, ISIS-APOCIII-LRX, Olezarsen, OLEZARSEN

Targets

Targets

No target linkage available.

Bioactivity

No ChEMBL bioactivity rows at pChembl ≥ 5 (expected for biologics / antibodies).

Target pathways

No target-pathway data for this drug (no mapped target genes).

Indications & clinical

Indications

2 indications (0 at ChEMBL trial phase 4). Phase below is the highest clinical-trial phase recorded for this drug against each disease — not the molecule’s overall approval status (that is in the Summary).

Indication	Trial phase	MONDO	EFO
hypertriglyceridemia	3	MONDO:0005347	EFO:0004211
cardiovascular disorder	1	MONDO:0004995	EFO:0000319

Clinical trials

Total trials: 11.

Phase distribution

Phase	Trials
PHASE3	7
PHASE2	2
PHASE1	1
Not specified	1

Top trials by phase / activity

NCT	Phase	Status	Title
NCT05130450	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Participants With Familial Chylomicronemia Syndrome (FCS)
NCT05185843	PHASE3	ACTIVE_NOT_RECRUITING	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRX) Administered to Adults With Familial Chylomicronemia Syndrome (FCS) Previously Treated With Volanesorsen
NCT05681351	PHASE3	ACTIVE_NOT_RECRUITING	CORE-OLE: A Study of Olezarsen (ISIS 678354) Administered Subcutaneously to Participants With Severe Hypertriglyceridemia (SHTG)
NCT04568434	PHASE3	COMPLETED	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) Administered to Patients With Familial Chylomicronemia Syndrome (FCS)
NCT05079919	PHASE3	COMPLETED	A Study of Olezarsen (ISIS 678354) Administered to Participants With Severe Hypertriglyceridemia
NCT05552326	PHASE3	COMPLETED	A Study of Olezarsen Administered Subcutaneously to Participants With Severe Hypertriglyceridemia
NCT05610280	PHASE3	COMPLETED	A Study of Olezarsen (ISIS 678354) in Participants With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease, or With Severe Hypertriglyceridemia
NCT03385239	PHASE2	COMPLETED	Study of ISIS 678354 (AKCEA-APOCIII-LRx) in Participants With Hypertriglyceridemia and Established Cardiovascular Disease (CVD)
NCT05355402	PHASE2	COMPLETED	A Study of Olezarsen (Formerly Known as AKCEA-APOCIII-LRx) in Adults With Hypertriglyceridemia and Atherosclerotic Cardiovascular Disease (Established or at Increased Risk for), and/or With Severe Hypertriglyceridemia
NCT05579860	PHASE1	COMPLETED	A Study Comparing Two Subcutaneous Formulations: Vial and Autoinjector (AI) With Olezarsen, at Two Dose Levels, in Healthy Adult Participants
NCT06360237	Not specified	APPROVED_FOR_MARKETING	Olezarsen Early Access Program for Patients With Familial Chylomicronemia Syndrome (FCS)

Clinical evidence (CIViC)

No CIViC predictive evidence (expected for non-precision-medicine drugs).

Pharmacology

Pharmacogenomics

No PharmGKB pharmacogenomic data curated for this drug.

Related molecules

Related molecules

No competitor molecules sharing a primary target (ChEMBL phase ≥2 or PubChem drug-class).