Efficacy and Safety of Subcutaneous Efgartigimod PH20 Administered by Prefilled Syringe in Adults With Ocular Myasthenia Gravis: Interim Results of ADAPT OCULUS Part A
Vern Juel1, Carolina Barnett-Tapia2, Andreas Meisel3, James Howard4, Chongbo Zhao5, Jianying Xi5, Akiyuki Uzawa6, Stephen Reddel7, Kristl Claeys8, Carlo Antozzi9, Łukasz Rzepiński10, Rodrigo Álvarez-Velasco11, Oleg Lukash12, Sirine Bougamra12, Fien Gistelinck12, Fien Verhamme12, Sui Wong13
1Duke University School of Medicine, 2University of Toronto, 3Charité – Universitätsmedizin Berlin, 4The University of North Carolina, 5Huashan Hospital, Fudan University, 6Chiba University, 7Concord Hospital, 8University Hospitals Leuven; and KU Leuven, 9Fondazione IRCCS Istituto Neurologico Carlo Besta, 1010th Military Research Hospital and Polyclinic; and University of Science and Technology, 11Hospital Universitario Ramón y Cajal, 12argenx, 13Moorfields Eye Hospital
Objective:

To present interim results of the Phase 3 ADAPT OCULUS trial (NCT06558279) evaluating the efficacy and safety of subcutaneous (SC) efgartigimod PH20 in adults with ocular myasthenia gravis (oMG).

Background:

There is an unmet need for approved, effective treatments for patients with oMG. Efgartigimod PH20 is a human immunoglobulin G1 (IgG1) antibody Fc fragment coformulated with recombinant human hyaluronidase PH20 that selectively reduces IgG levels by blocking neonatal Fc receptor–mediated IgG recycling. Retrospective analysis of data supporting the approval of efgartigimod for treatment of generalized MG indicated an improvement in ocular symptoms.

Design/Methods:

In Part A, adults with confirmed oMG and a Myasthenia Gravis Impairment Index (MGII) patient-reported outcome (PRO) subcomponent ocular score ≥6 are randomized 1:1 to receive 4 once-weekly efgartigimod PH20 SC 1000 mg or placebo injections administered via prefilled syringe, followed by 4 weeks of follow-up. Participants may continue the study in Part B, a ≤2-year open-label extension.

Results:

The primary endpoint is change in MGII PRO ocular score from baseline to Week 4. Key secondary endpoints include, among others, changes from baseline to Week 4 in MGII ocular score (PRO plus physical examination) and MGII total score. Safety assessments include adverse event incidence and severity. Topline results from an interim analysis of Part A will be presented at the conference.

Conclusions:

ADAPT OCULUS is the first Phase 3 clinical trial to address the unmet need for treatment in oMG by evaluating the safety and efficacy of efgartigimod PH20 SC in participants with oMG.

10.1212/WNL.0000000000215629
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