Phase 3 Results Show Therapy Effective for Ocular MG

The combination of efgartigimod and hyaluronidase (Vyvgart; argenx) improved the Myasthenia Impairment Index (MGII) Patient Report Outcome (PRO) ocular scores of patients living with ocular myasthenia gravis (oMG), according to results from a phase 3 trial.¹ The study marks the first study dedicated to evaluating treatment efficacy in oMG.

Efgartigimod has previously been approved for use in patients with generalized MG who are positive for acetylcholine receptor (AChR) antibodies.² The FDA approved the medication as a subcutaneous therapy in 2023 based on data from the phase 3 ADAPT-SC study (NCT04735432). This ADAPT OCULUS trial aimed to assess if the treatment could also be used to specifically treat those with oMG.¹

“I think a major advancement for the field is that this is really the first time that a new therapy has been studied in a rigorous fashion for patients with oMG,” said Jeff Guptill, MD, disease area development lead for neuromuscular programs at argenx. “Thus far, the clinical trial world has really focused on the patients with generalized MG, and unfortunately the oMG patient population has been kind of left out of the treatment advances that are kind of working through the field.”

The phase 3 ADAPT OCULUS (NCT06298552) is a randomized, double-blind, placebo-controlled study that evaluated the efficacy of subcutaneous efgartigimod and hyaluronidase administered in a syringe to adult patients with oMG in North America, Asia-Pacific, and Europe. Participants of the study were randomized 1:1 to receive either the treatment or placebo once per week for 4 weeks for Part A of the trial. For Part B, there was an open-label extension, and participants received 2 cycles of weekly injections of the treatment within a 4-week interval between the cycles.

Change in MGII PRO ocular score from baseline to week 4 compared with placebo was the primary end point of Part A. Participants could be seropositive or seronegative for AChR antibodies and had ocular muscle weakness determined by an MGII PRO ocular score of 6 or higher and 2 ocular items having a score of 2 or higher. All participants were on treatment for generalized MG prior to their randomization.

ADAPT OCULUS was able to meet its primary end point by showing that there was a statistically significant improvement in MGII PRO from baseline to week 4 compared with the placebo. The mean change in MGII PRO was a 4.04-point improvement in those taking efgartigimod and hyaluronidase compared with a 1.99-point improvement in those taking the placebo. Key ocular symptoms of diplopia and ptosis, which are double vision and drooping of the upper eyelids, respectively, were reduced in those taking the combination therapy.

“The improvements observed with VYVGART in the OCULUS trial offer hope to the thousands of myasthenia gravis patients with ocular involvement,” said Carolina Barnett-Tapia, MD, PhD, associate professor of medicine at the University of Toronto, in a statement.¹

Overall, these results bring new options to the table for patients diagnosed with oMG to help them address their specific needs in treatment. These results will form the basis of a planned supplemental biologics license application that will attempt to extend the use of efgartigimod and hyaluronidase beyond generalized MG and into oMG.

References

1. Argenx announces positive topline results from phase 3 ADAPT OCULUS trial of VYVGART in ocular myasthenia gravis. News release. Argenx. February 26, 2026. Accessed March 30, 2026. https://argenx.com/news/2026/press-release-3245183.html
2. Meglio M. Efgartigimod meets primary end point in phase 3 ADAPT OCULUS study of ocular myasthenia gravis. NeurologyLive^®. February 26, 2026. Accessed March 30, 2026. https://www.neurologylive.com/view/efgartigimod-meets-primary-end-point-phase-3-adapt-oculus-study-ocular-myasthenia-gravis