NCT07489209

A Dose-exploration Study of EDP167 in HoFH

A Multicenter, Dose-exploration, Open-label Phase II Study to Evaluate the Efficacy and Safety of EDP167 in Adult Patients With Homozygous Familial Hypercholesterolaemia

• Status: Recruiting
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (estimated)
• Sponsor: Eddingpharm (Zhuhai) Co., Ltd. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

EDP167 is a double-stranded small interfering RNA (siRNA) drug targeting angiopoietin-like 3 protein (ANGPTL3), which may bring benefits for patients with dyslipidemia conditions. This is a dose exploration study in Homozygous Familial Hypercholesterolaemia (HoFH) patients to evaluate the efficacy and safety and pharmacokinetics (PK)/pharmacodynamics (PD) profiles of multiple EDP167 injections.

Detailed description

Angiopoietin-like 3 protein (ANGPTL3) is a key regulator of lipid metabolism. Clinical studies have shown that inhibition of ANGPTL3 could exert lipid-lowering effects in patients with dyslipidemia. EDP167 is a novel N-Acetylgalactosamine (GalNAc)-conjugated siRNA therapeutic that selectively silences hepatic ANGPTL3 mRNA expression, offering a promising strategy for lipid lowering. This trial includes two phase, main study phase (including screening period and 24 weeks treatment period) and extension phase (including 6 months treatment period). In main study phase, patients with HoFH will be randomized into two dose cohorts (200mg and 300mg, 10 subjects each), receiving EDP167 injections on Day 1 and at Week 12. At Week 24 of the main study phase, subjects will be evaluated and enter the extension phase, receiving EDP167 injections at Month 0 and at Month 3. The follow-up will last till Month 6 in extension phase to evaluate the efficacy, safety, PK profile and PD effects (ANGPTL3, low-density lipoprotein cholesterol \[LDL-C\], triglyceride \[TG\], and other lipid parameters) in HoFH patients after multiple EDP167 injections.

Conditions

• Homozygous Familial Hypercholesterolemia (HoFH)

Interventions

Type	Name	Description
DRUG	EDP167	EDP167 sc injection

Timeline

• Start date: 2026-02-06
• Primary completion: 2026-10-04
• Completion: 2027-03-31
• First posted: 2026-03-24
• Last updated: 2026-03-27

Locations

1 site across 1 country: China