-
Study met primary endpoint (p-value = 0.012)
-
First registrational study to specifically evaluate a targeted treatment for patients living with ocular MG
-
Results support planned Supplemental Biologics License Application (sBLA) submission to U.S. Food and Drug Administration (FDA) to expand label into oMG
February 26, 2026, 6:30 AM CET--Amsterdam, the Netherlands – argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced positive topline results from the Phase 3 ADAPT OCULUS study evaluating VYVGART® (efgartigimod alfa and hyaluronidase-qvfc) in adults with ocular myasthenia gravis (oMG).
ADAPT OCULUS met its primary endpoint (p-value=0.012), showing that patients living with oMG and treated with VYVGART demonstrated statistically significant improvement from baseline in Myasthenia Impairment Index (MGII) Patient Reported Outcome (PRO) ocular scores at Week 4 compared to placebo. In the overall population, mean change from baseline in patients treated with VYVGART was a 4.04 point improvement in MGII PRO versus a mean change of 1.99 MGII PRO score in patients treated with placebo. Patients treated with VYVGART experienced a marked reduction of key ocular symptoms: diplopia (double vision) and ptosis (drooping of the upper eyelids).
“Ocular myasthenia gravis significantly impacts patients’ daily lives, affecting vision, independence and the ability to do routine tasks, such as work or drive a car. Yet today, there are no approved targeted medicines for this disease,” said Carolina Barnett-Tapia, M.D., Ph.D., Associate Professor of Medicine (Neurology) at the University of Toronto. “The improvements observed with VYVGART in the OCULUS trial offer hope to the thousands of myasthenia gravis patients with ocular involvement.”
VYVGART was well tolerated and had a favorable safety profile in patients with oMG, consistent with prior studies. No new safety concerns were identified.
“ADAPT OCULUS is the first registrational study specifically designed to evaluate a targeted therapy for ocular myasthenia gravis,” said Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “Ocular MG has been historically under-studied and represents a significant unmet need in the MG community. These positive results deliver on our patient-centered approach to drug development and bring us one step closer to our vision of delivering a targeted, transformative treatment option to as many MG patients as possible and ensuring no patient is left behind.”
Data from the ADAPT OCULUS study will be presented at an upcoming medical meeting.
About the ADAPT OCULUS Study Design
ADAPT OCULUS is a Phase 3, randomized, double-blind, placebo-controlled, parallel-group design study evaluating the efficacy and safety of VYVGART SC administered by prefilled syringe in adult patients with ocular MG (MGFA Class I) (n=141) across North America, Europe and Asia-Pacific. In Part A, randomized participants (1:1) received four once-weekly injections of efgartigimod PH20 SC or placebo PH20 SC followed by a 4-week follow-up. In Part B, open-label extension, participants received 2 cycles of four once-weekly efgartigimod injections with a 4-week interval between cycles. Additional cycles from Cycle 3 onward could start ≥1 week after the last administration of the previous cycle, based on clinical status.
The primary endpoint was the change from baseline in Myasthenia Gravis Impairment Index (MGII) (patient-reported outcome [PRO] subcomponent) ocular score at week 4 (day 29) compared to placebo in Part A. Enrolled participants were either seropositive or seronegative for AChR-Ab, and MGFA Class I with only ocular muscle weakness as determined by an MGII (PRO) ocular score of ≥6 with at least 2 ocular items with a score of ≥2. Participants were on a stable dose of gMG treatment prior to randomization, including acetylcholinesterase inhibitors, corticosteroids or nonsteroidal immunosuppressive drugs.
MGII is a validated measure of disease severity based on the signs and symptoms of myasthenia gravis and includes an ocular-specific subdomain that evaluates the two key clinical symptoms of oMG: diplopia and ptosis.
About VYVGART and VYVGART Hytrulo
VYVGART® (efgartigimod alfa fcab) is a first-in-class human IgG1 antibody fragment that binds to the neonatal Fc receptor (FcRn), resulting in the reduction of circulating IgG autoantibodies. VYVGART® Hytrulo is a subcutaneous combination of efgartigimod alfa (VYVGART) and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. VYVGART is approved for generalized myasthenia gravis (gMG) and immune thrombocytopenia (Japan only). VYVGART Hytrulo is approved for gMG and chronic inflammatory demyelinating polyneuropathy (CIDP). VYVGART Hytrulo may be marketed under different proprietary names in other regions.
About Ocular Myasthenia Gravis (oMG)
Ocular myasthenia gravis (oMG) is a rare and chronic autoimmune disease characterized by muscle weakness limited to the muscles controlling the eyes and eyelids. Symptoms commonly include ptosis (drooping eyelids), diplopia (double vision), and fluctuating visual disturbance that can impair daily activities. Approximately 80% of myasthenia gravis (MG) patients initially present with ocular symptoms, and up to 92% experience ocular involvement at some point during the course of disease. While many progress to generalized myasthenia gravis (gMG), in 15–25% of patients, weakness remains restricted to the ocular muscles. oMG is driven by pathogenic IgG autoantibodies that disrupt communication at the neuromuscular junction. Despite the functional and quality-of-life burden associated with persistent ocular symptoms, there are currently no approved targeted therapies specifically for oMG. Treatment approaches often rely on symptomatic therapies and generalized immunosuppression, underscoring the need for additional therapeutic options for this distinct MG population.
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker and is evaluating its broad potential in multiple serious autoimmune diseases while advancing several earlier stage experimental medicines within its therapeutic franchises.