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ML Risk
Also known as AROANG3, Zodasiran
VSA003 · 4 trials · 5 indications
safety and tolerability
| Arm | Type | Description |
|---|---|---|
| Zodasiran | EXPERIMENTAL | - |
| Placebo | PLACEBO_COMPARATOR | Calculated volume to match active treatment by sc injection (randomized period). 4 or 5 doses of zodasiran by sc injection (optional open-label period) |
| VSA003 | EXPERIMENTAL | subcutaneous injections |
| Name | Type | Description |
|---|---|---|
| Zodasiran | DRUG | By sc injection |
| zodasiran Injection | DRUG | ARO-ANG3 Injection |
| Placebo | DRUG | sterile normal saline (0.9% NaCl) |
| VSA003 | DRUG | subcutaneous injections |
| 0.9% NaCl | DRUG | placebo |
Inclusion Criteria: * Adolescents 12 to \<18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study * Body weight ≥35 kilograms (kg) at screening * HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening gen...
VSA003 is an investigational drug being developed for dyslipidemias and homozygous familial hypercholesterolemia (HoFH), a rare genetic condition causing extremely high cholesterol. It is currently in clinical development and has not been approved by regulatory authorities.
VSA003 targets ANGPTL3, a protein that regulates lipid metabolism. By inhibiting ANGPTL3, the drug aims to reduce levels of cholesterol and triglycerides in the blood, which is relevant for treating dyslipidemias and HoFH.
VSA003 is being developed by Arrowhead Pharmaceuticals, Inc., a biopharmaceutical company traded on the NASDAQ under the ticker ARWR. The company is conducting clinical trials for this drug in patients with lipid disorders.
VSA003 is in Phase 1 clinical development. A Phase 1 study in healthy volunteers has been completed, and a Phase 3 trial in HoFH patients is active but not yet recruiting. The drug is investigational and not yet approved.
VSA003 has two clinical trials. NCT05851066 is a completed Phase 1 study in healthy volunteers in China. NCT06712771 is an active Phase 3 trial in Chinese HoFH patients, with an enrollment target of 46 participants.
No alternative names for VSA003 have been reported. The drug is identified solely by its code name VSA003 in clinical trial registrations and company communications.