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124I-AT-01
124I-AT-01 is a small molecule targeting the lysosomal acid lipase (LAL) enzyme.
124I-AT-01 is a small molecule targeting the lysosomal acid lipase (LAL) enzyme. Used for Lysosomal acid lipase deficiency.
At a glance
| Generic name | 124I-AT-01 |
|---|---|
| Also known as | AT-01, I124-AT-01 |
| Sponsor | Attralus, Inc. |
| Drug class | LAL inhibitor |
| Target | Lysosomal acid lipase (LAL) |
| Modality | Small molecule |
| Therapeutic area | Genetics and Rare Diseases |
| Phase | Phase 2 |
Mechanism of action
By inhibiting LAL, 124I-AT-01 aims to reduce the accumulation of cholesterol and triglycerides in cells, potentially treating lysosomal acid lipase deficiency.
Approved indications
- Lysosomal acid lipase deficiency
Common side effects
- Hypotension
- Nausea
Key clinical trials
- Repeat PET/CT Imaging in People With CAPS and Anakinra-Induced Amyloidosis Using an Amyloid-Reactive Peptide to Measure Changes in Organ-Specific Amyloid Load (PHASE1)
- Characterizing Iodine-124 Evuzumitide (AT-01) in Systemic Amyloidosis (PHASE2)
- A Study to Evaluate Organ Level Uptake Repeatability of 124I AT-01 in Subjects With Systemic Amyloidosis (PHASE2)
- Evuzamitide in PET/CT to Measure Potential Therapeutic Response in ATTR (PHASE2)
- 124I-Evuzamitide PET/CT Imaging In Patients With Lumbar Spinal Stenosis and Carpal Tunnel Syndrome (PHASE2)
- Repeat PET/CT Imaging of Patients With Amyloid 124I-AT-01 to Measure Changes in Organ-specific Amyloid Load (PHASE2)
Primary sources
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| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |