Alexion strengthens research leadership in gMG care with new data at 2026 AANEM Annual Meeting and MGFA Scientific Session

Fresh long-term findings add further weight to gefurulimab’s case as a once-weekly, patient-administered subcutaneous treatment for adults with generalized myasthenia gravis

Additional research on display underscores the company’s continued investment in gMG science and clinical practice

Alexion, AstraZeneca’s rare disease division, is taking part in the American Association of Neuromuscular & Electrodiagnostic Medicine Annual Meeting and the Myasthenia Gravis Foundation of America Scientific Session in Orlando, Florida, running September 29 through October 2, 2026, where the company will share 15 abstracts as part of its broader push to improve outcomes for people with generalized myasthenia gravis.

Among the standout presentations is fresh 52-week data drawn from the Phase III PREVAIL trial, which looks at how well gefurulimab, a novel dual-binding nanobody delivered as a self-injected subcutaneous C5 inhibitor, performs over time in adults who test positive for anti-acetylcholine receptor antibodies and have generalized myasthenia gravis. A separate set of analyses from the same trial, covering the first 26 weeks, will dig into gefurulimab’s clinical impact and its effect on patients’ quality of life. A related oral session will unveil new preclinical work examining a functional biomarker that could help track how the disease progresses and how well patients respond to treatment.

Christophe Hotermans, senior vice president and head of global medical affairs at Alexion, said the volume of data on display at this year’s AANEM and MGFA meetings speaks to Alexion’s ongoing push to open up fresh possibilities in gMG treatment and honor its commitment to patients. He pointed to the open-label extension results from PREVAIL as evidence of how gefurulimab continues to support functional gains through a full year of treatment, extending findings already reported at the six-month mark. Paired with new analyses showing gefurulimab lowers the likelihood of gMG-related hospital stays and rescue therapy, he said the body of evidence strengthens the argument for gefurulimab as a practical, self-administered option that can bring quick and lasting control to a disease known for its unpredictability.

Extended PREVAIL results and follow-up analyses bolster the case for gefurulimab

Data from PREVAIL’s open-label extension phase point to disease control holding steady through the one-year mark, tracked via changes from baseline on three separate scales: Myasthenia Gravis Activities of Daily Living, Quantitative Myasthenia Gravis, and Myasthenia Gravis Composite. Patients who stayed on gefurulimab kept their clinical gains throughout the extension, posting least squares mean shifts (with 95% confidence intervals) of -5.3 (-5.8 to -4.8) on MG-ADL, -5.3 (-6.0 to -4.6) on QMG, and -9.4 (-10.3 to -8.5) on MGC. For the group that crossed over from placebo to gefurulimab at the six-month point, benefits showed up at their very first post-switch evaluation and held through week 52, with mean shifts of -4.9 (-5.4 to -4.4) on MG-ADL, -4.6 (-5.4 to -3.9) on QMG, and -8.3 (-9.2 to -7.3) on MGC. The drug was well tolerated overall, and no instances of meningococcal infection turned up.

A separate look back at the long-term PREVAIL data, conducted after the fact, found that patients on gefurulimab responded quickly and reached minimal symptom levels early on, a benefit that held through the full year, with some patients showing measurable improvement in as little as a week.

A further oral session will detail how gefurulimab, measured at the six-month point against placebo, was tied to fewer gMG-related hospital admissions and less reliance on rescue therapy within the PREVAIL trial.

Animal research points toward a new way to monitor disease and treatment response

A separate oral talk will introduce a preclinical biomarker system that pairs live measurements of nerve-triggered muscle function with detailed analysis of neuromuscular junction structure, tested in a rodent model of the disease. The early findings hint that this combined approach could help scientists characterize damage at the neuromuscular junction, chart how the disease advances, and gauge how well a given treatment is working. Within this animal model, blocking complement C5 was linked to partial recovery in both the structure of the neuromuscular junction and muscle performance, a result that supports pushing the research further, both to refine the platform itself and to explore whether its findings might eventually translate to human patients.

Real-world data support a digital tool for tracking gMG symptoms

A joint presentation with Ad Scientiam will detail results from the ME&MG™ open study, showing that digital measurements gathered through the ME&MG app line up with how severe a patient’s gMG is, based on standard MGFA classification, lending support to the app’s potential role in everyday clinical use.

Alexion’s presentations at the 2026 AANEM Annual Meeting and MGFA Scientific Session

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2026 AANEM Annual Meeting, Alexion, gMG care, MGFA Scientific Session