This is multisite, randomized, double-blind, placebo-controlled, multidose regimen, phase 1, first-in-human study of VXX-401, a synthetic peptide-based active immunotherapy candidate for preventing and treating hypercholesterolemia. The study will include Screening, Treatment, and Follow-up Periods. This study will enroll participants who are naïve to statin use. Each cohort is planned to randomize approximately 12 participants to receive priming doses of VXX-401 or placebo in a 3:1 ratio. This study will have 4 dosing cohorts, A - D. It is planned to test up to 4 dose regimens of VXX-401 or placebo, administered by IM injection into the deltoid muscle. All eligible participants will receive a priming regimen at Week 0 (Baseline, Day 1), Week 4, and Week 12, in Cohorts A and C, and additionally at Week 8 in Cohorts B and D. The last dose administration will be at Week 12. There is one dose escalation from 100 μg to 300 μg between cohorts B and C. All participants will be followed through Week 30 (EOS).
This first-in-human (FIH) study of VXX-401, an anti-PCSK9 peptide-based immunotherapeutic candidate, is designed to assess the safety, tolerability, immunogenicity, and pharmacodynamics (PD) of VXX-401 and to determine an optimal dose regimen for LDL-C lowering in subsequent clinical trials.