ABCA1

ATP-binding cassette transporter A1

Score: 0.558 Price: $0.56 Medium Druggability Status: active Wiki: ABCA1

🧠 Neurodegeneration

HYPOTHESES

6

PAPERS

0

KG EDGES

317

DEBATES

1

3D Protein Structure

🧬 ABCA1 — PDB 7QS7 Click to expand

Experimental structure from RCSB PDB | Powered by Mol*

Druggability & Clinical Context

Druggability

Medium

Score: 0.47

Clinical Stage

Approved

Target Class

Transporter

Safety

0.50

Druggability Analysis

Drug Development0.75

Structural Tractability0.70

Target Class0.70

Safety Profile0.50

Key Metrics

PDB Structures:

7

Known Drugs:

1

Approved:

1

In Clinical Trials:

0

Drug Pipeline (1 compounds)

1 Approved

Druggability Rationale: ABCA1 demonstrates high druggability (0.90 score) due to extensive structural characterization (7 PDB structures at 3.1Å resolution, cryo-EM data, AlphaFold model), validated by Probucol's clinical approval for hypercholesterolemia. As an ABC transporter with well-defined nucleotide-binding domains and transmembrane architecture, it presents multiple tractable binding sites for small molecule modulators to enhance cholesterol efflux activity.

Mechanism: Small molecule modulators to enhance cholesterol efflux and HDL formation

Drug Pipeline (1 compounds)

1 Approved

Known Drugs:

Probucol (approved) — Hypercholesterolemia

Structural Data:

PDB (7) ✓AlphaFold ✓Cryo-EM ✓

5XJY 7ROQ 7TBW 7TBY 7TBZ+2 more

UniProt: A5D8U6

🧬 3D Protein Structure

🧬 ABCA1 — PDB 7QS7 Click to expand interactive 3D viewer

Experimental structure from RCSB PDB | Powered by Mol* | Rotate: click+drag | Zoom: scroll

Selectivity & Safety Considerations

ABCA1 selectivity is achievable given its unique tissue expression pattern (particularly high in macrophages, enterocytes, and microglia) and distinct substrate specificity for apoA-I; however, off-target activity against other ABC transporters (ABCG1, ABCG4) must be carefully monitored as they share overlapping functions in lipid homeostasis and neuroinflammation.

3D Protein Structure

PDB: Open in RCSB AlphaFold model

Interactive 3D viewer powered by RCSB PDB / Mol*. Use mouse to rotate, scroll to zoom.

Clinical Trials (8)

Relevant trials from ClinicalTrials.gov

Active

1

Completed

6

Total Enrollment

4,013

By Phase

PHASE2: 1 · PHASE4: 5 · Unknown: 2

The Effect of InTensive Statin in Ischemic Stroke With inTracranial Atherosclerotic Plaques Recruiting

PHASE4 NCT03753555 n=100

Stroke, Ischemic, Atherosclerosis, Cerebral

Interventions: Atorvastatin Calcium, Atorvastatin Calcium, Probucol

Sponsor: General Hospital of Shenyang Military Region | Started: 2018-12-01

A Study of the Antioxidant Probucol Combined With Valsartan in Patients With IgA Nephropathy Completed

PHASE4 NCT00426348 n=75

Glomerulonephritis, IGA Nephropathy

Interventions: Valsartan, Probucol, Placebo

Sponsor: Guangdong Provincial People's Hospital | Started: 2007-05

A Clinical Study to Evaluate the Efficacy and Safety of Cilostazol and Probucol in Combination on Patients With Diabetic Completed

PHASE4 NCT01252056 n=353

Diabetic Nephropathy

Interventions: Probucol, Probucol and Cilostazol

Sponsor: Otsuka Beijing Research Institute | Started: 2010-03

Study of Cilostazol and Probucol to Assess Their Effects on Atherosclerosis Related Biomarker Completed

PHASE4 NCT00823849 n=200

Type 2 Diabetes Mellitus, Arteriosclerosis Obliterans

Interventions: Cilostazol, Probucol, Cilostazol+Probucol

Sponsor: Otsuka Beijing Research Institute | Started: 2008-10

Synergistic Effect of Combination Therapy With Cilostazol and Probucol on Plaque Stabilization and Lesion Regression: Se Completed

PHASE4 NCT01031667 n=118

Therapy

Interventions: Cilostazol, Probucol / placebo of probuc

Sponsor: Yonsei University | Started: 2009-11

Evaluation of Concomitant Administration of Cilostazol and Probucol on Biomarkers, Endothelial Function and Safety Completed

PHASE2 NCT01142284 n=80

Peripheral Artery Disease

Interventions: Cilostazol, Probucol

Sponsor: Korea Otsuka Pharmaceutical Co., Ltd. | Started: 2010-05-19

Study on the Efficacy and Safety of Oral Probucol Tablets in Patients With Hyperlipidemia - Post-Marketing Surveillance Completed

Unknown NCT02730390 n=3087

Hyperlipidemia

Sponsor: Otsuka Pharmaceutical, Inc., Philippines | Started: 2009-12

Expanded Access Program for R007 (Probucol) Tablets in Adults With Mitochondrial Disease and Chronic Kidney Disease Available

Unknown NCT06931834

Mitochondrial Disease, Chronic Kidney Disease

Interventions: Probucol

Sponsor: RiboNova Inc.

Linked Hypotheses (3)

HDL/apoE Particle Remodeling as a Therapeutic Switch for CAA Prevention0.766

APOE4 hypolipidation and ABCA1 mistrafficking impair cholesterol efflux and secondarily reduce ER st0.694

ABCA1 Upregulation via LXRβ Agonism to Enhance Cholesterol Efflux and Prevent Neuroinflammatory Lipi0.380

Linked Experiments (1)

ABCA1 rs2230806 polymorphism association with dementia risk in hyperlipidemia0.900

Scoring Dimensions

Knowledge Graph (20)

activates (1)

associated with (1)

ABCA1→neurodegeneration

inhibits (3)

P2RY12→ABCA1MTOR→ABCA1ABCA1→APOE

interacts with (13)

ABCA1→LDLRABCA1→SREBF2LDLR→ABCA1SREBF2→ABCA1ACTB→ABCA1

▸ Show 8 more

APOA1→ABCA1ULK1→ABCA1ATG5→ABCA1EIF4EBP1→ABCA1LC3→ABCA1ABCG1→ABCA1SQSTM1→ABCA1ABCA1→LOX

regulates (1)

ABCA1→cholesterol_efflux

therapeutic target (1)

Debate History (1)

Should ABCA1 (ATP-binding cassette transporter A1) be prioritized as a therapeut2026-04-21