Prevention & lipids

Anti-ANGPTL3 Antibody SHR-1918 for Homozygous Familial Hypercholesterolemia: A Nonrandomized Clinical Trial

JAMA CardiolPublished Nonrandomised clinical trial

Authors' conclusion

Results show that SHR-1918 was associated with a substantial reduction in LDL-C level and favorable safety profile among patients with HoFH taking stable lipid-lowering therapy.

Study context

Population
Adults with homozygous familial hypercholesterolemia (HoFH) taking stable lipid-lowering therapy
Participants
26 adults
Outcome
Percent change in serum LDL-C from baseline
Follow-up
Week 12
Methodological detailsmulticentre, single-arm, phase 2, 8 sites in China, December 19, 2023, to April 2, 2024
  • Multicentre
  • Single-arm
  • Phase 2
  • 8 sites in China
  • December 19, 2023, to April 2, 2024

Main finding

As reported in the abstract

% change in LDL-C from baseline

Mean % change in low-density lipoprotein cholesterol (LDL-C)

−59.09%

SD, 11.71%; 95% CI, −63.81% to −54.36%

Further findings

Percent change in LDL-C by HoFH genotype (subgroup) · week 12

Homozygous
−61.32%
Compound heterozygous
−56.40%
Double heterozygous
−72.21%

LDL-C reduction during follow-up · 8-week follow-up

Reduction observed throughout the entire 8-week follow-up period

Safety

Treatment-emergent adverse events

≥1 TEAE, n (%)
16 (61.5%)
Proteinuria (most common), n (%)
4 (15.4%)
Injection site reaction (pain, rash or erythema; both grade 1), n (%)
1 (3.8%)

Original publication

Anti-ANGPTL3 Antibody SHR-1918 for Homozygous Familial Hypercholesterolemia: A Nonrandomized Clinical Trial. JAMA Cardiol. Published 1 February 2026. doi:10.1001/jamacardio.2025.4878

Summarised from the published abstract; every number is checked against it

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