Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 With Evolocumab in HoFH

Phase 3 Completed 65 enrolled LIB Therapeutics LLC

RandomizedCrossoverOpen-labelTreatment

Summary

To compare the safety, tolerability and LDL-C response after 24 Weeks of monthly (every 4 weeks \[Q4W\]) subcutaneous (SC) dosing of LIB003 300 mg with monthly (Q4W) SC dosing of 420 mg evolocumab (Repatha®) in patients with HoFH on stable diet and oral LDL-C-lowering drug therapy

Timeline

Start: 2019-12-07
Primary completion: 2022-09-12
Completion: 2023-01-30

Outcome

Missed primary endpoint

paper the mean difference between treatments was 5·4% (95% CI -0·2 to 11·1), which did not show non-inferiority at the prespecified 6% margin. PMID 39870096 ↗

Publications

Raal FJ, Mehta V, Kayikcioglu M, Blom D, Gupta P, Elis A, Turner T, Daniels C, Vest J, Mitchell T, Caldwell K, Bahassi EM, Kallend D, Stein EA. Lerodalcibep and evolocumab for the treatment of homozygous familial hypercholesterolaemia with PCSK9 inhibition (LIBerate-HoFH): a phase 3, randomised, open-label, crossover, non-inferiority trial. Lancet Diabetes Endocrinol. 2025 Mar;13(3):178-187. doi: 10.1016/S2213-8587(24)00313-9. Epub 2025 Jan 24.

Drugs

Evaluation	Drug	Modality	Dose	Route
Comparator	Evolocumab	Monoclonal antibody	420 mg	Subcutaneous
Subject	lerodalcibep	Protein / enzyme biologic	300 mg	Subcutaneous