Efficacy and Safety of Epicutaneous Immunotherapy in Peanut-Allergic Toddlers: Open-Label Extension to EPITOPE.

Abstract

Background: The pivotal phase 3 EPITOPE trial, a 12-month, double-blind, placebo-controlled study of epicutaneous immunotherapy with the VIASKIN® patch containing 250 μg peanut protein (VP250) previously reported significant treatment response vs placebo in peanut-allergic toddlers aged 1-through-3 years.

Objective: To assess interim efficacy and safety of VP250 from the first year of the EPITOPE open-label extension (OLE) study.

Methods: Eligible participants enrolled in the OLE study for up to 3 years of total treatment with annual double-blind, placebo-controlled food challenges (DBPCFC) and safety assessments; here we report the first year OLE (Year 2) results.

Results: 266 EPITOPE participants enrolled in the OLE study; 244 underwent Month 24 DBPCFC (n=166 VP250; n=78 placebo). After 24 months of VP250, 81.3% reached an eliciting dose (ED) ≥1000 mg, 63.8% reached an ED ≥2000 mg, and 55.9% completed the DBPCFC (cumulative dose: 3444 mg) without meeting stopping criteria. No treatment-related anaphylaxis or serious treatment-related adverse events occurred during Year 2 in this treatment arm. Local application-site reactions occurred less frequently in Year 2 vs Year 1. In placebo-treated EPITOPE participants, outcomes after 1 year of open-label VP250 were consistent with EPITOPE treatment results: 62.7% reached an ED ≥1000 mg, 36.5% reached an ED ≥2000 mg, and 28.4% completed the DBPCFC without meeting stopping criteria; and there was 1 treatment-related anaphylaxis event.

Conclusions: Two years of VP250 in young peanut-allergic children demonstrated continued treatment effect increases without new safety signals. This supports the potential of VP250 as a safe and effective treatment for peanut allergy in young children.