Carolina Barnett-Tapia, MD, Neuromuscular Neurologist and the University of Toronto, discusses the safety and efficacy of subcutaneous efgartigimod PH20 in ocular myasthenia gravis (oMG).
oMG is a neuromuscular disease characterized by autoantibody production against post-synaptic proteins in the neuromuscular junction. oMG typically presents as almost exclusively ocular symptoms and can evolve into generalized myasthenia gravis (gMG) in about 20% to 60% of cases. Common ocular manifestations include fluctuating ptosis, diplopia, and orbicularis weakness.
The Phase 3 ADAPT OCULUS trial (NCT06558279) is a randomized, double-blind, placebo-controlled, parallel-group design study evaluating the efficacy and safety of efgartigimod PH20 subcutaneous administered by a prefilled syringe in adults 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.
In Part A of this trial, adults with confirmed oMG and a Myasthenia Gravis Impairment Index (MGII) patient-reported outcome subcomponent ocular score of 6 or greater are randomized to receive 4 once-weekly efgartigimod PH20 1000 mg or placebo, followed by 4 weeks of follow-up.
The primary endpoint is change in MGII patient-reported outcome ocular score from baseline to week 4. Key secondary endpoints include changes from baseline to week 4 in MGII ocular score (patient-reported outcome plus physical examination) and MGII total score. Safety assessments include adverse event incidence and severity. Topline results highlighted that the primary endpoint was met.
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To learn more about oMG and other rare eye conditions, visit https://checkrare.com/diseases/ophthalmology-eye-diseases/
