Alexion data presented at 2025 AANEM Annual Meeting and MGFA Scientific Session underscores pioneering C5 inhibition leadership in gMG

New data on gefurulimab, a novel dual-binding nanobody, from the PREVAIL Phase III trial will highlight its potential as an effective, self-administered treatment option

Robust real-world evidence will reinforce the clinical benefits of ULTOMIRIS^® and SOLIRIS^®, including reduced steroid burden

Alexion, AstraZeneca Rare Disease, will deliver 18 presentations, including four oral presentations, from its leading rare neurology portfolio at the American Association of Neuromuscular & Electrodiagnostic Medicine (AANEM) Annual Meeting and the Myasthenia Gravis Foundation of America (MGFA) Scientific Session in San Francisco, California on October 29 to November 1, 2025.

Presentations will feature topline data from the global PREVAIL Phase III trial evaluating gefurulimab in adults with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) generalized myasthenia gravis (gMG), as well as robust real-world data and clinical insights that further reinforce the safety profiles and efficacy of ULTOMIRIS^® (ravulizumab-cwvz) and SOLIRIS^® (eculizumab).  

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “At this year’s MGFA Scientific Session, we are proud to reinforce our pioneering leadership in C5 inhibition with topline data from the PREVAIL Phase III trial supporting the potential for gefurulimab to deliver early and sustained disease control to patients with gMG. Alongside this, we will share real-world and clinical evidence further establishing ULTOMIRIS as a differentiated therapeutic option and underscoring the impact of our portfolio in addressing critical unmet needs in gMG treatment, such as reducing steroid burden.”

New data on gefurulimab will highlight its potential to provide early and sustained disease control

An oral presentation at the MGFA Scientific Session will share topline results from the global PREVAIL Phase III trial evaluating gefurulimab, a dual-binding nanobody optimized for subcutaneous self-administration, in adult patients with AChR-Ab+ gMG. The trial met its primary and all secondary endpoints, with gefurulimab demonstrating a statistically significant and clinically meaningful improvement from baseline in Myasthenia Gravis Activities of Daily Living (MG-ADL) total score at week 26 compared to placebo.

Two additional posters at the MGFA Scientific Session will provide further insights into the delivery of gefurulimab via prefilled syringe (PFS) and auto injector (AI).  Results from a Phase I parallel-group study in healthy adults will show that pharmacokinetic exposure following a single subcutaneous dose of gefurulimab administered via autoinjector (AI) was comparable to administration via prefilled syringe (PFS). Separately, results from a human factors validation study will demonstrate that gefurulimab AI and PFS user interfaces can be used safely and effectively with positive user experiences.

Evidence further substantiates the steroid-sparing role of ULTOMIRIS and SOLIRIS

Several presentations at the MGFA Scientific Session will explore the real-world impact of corticosteroid use on patients with gMG. An encore oral presentation will highlight a retrospective study of patients with gMG showing that corticosteroid-related toxicity was higher in patients with ≥3 months’ continuous use of corticosteroids versus those with <3 months’ continuous use, or no use at all. Furthermore, a retrospective study of patients with gMG will demonstrate a significant increase in healthcare resource utilization in patients with high corticosteroid toxicity.

Data will also be presented at the AANEM Annual Meeting on the reduced corticosteroid burden following treatment with ULTOMIRIS or SOLIRIS. An encore poster presentation will highlight data from the global MG SPOTLIGHT Registry showing reduced concomitant immunosuppressive therapies and oral corticosteroids burden in patients with AChR-Ab+ gMG treated with ULTOMIRIS in routine clinical practice. Additionally, an encore poster presentation of a retrospective analysis of a United States (US) claims database will demonstrate greater reduction in oral corticosteroid use after 12 months in patients with gMG receiving treatment with ULTOMIRIS or SOLIRIS compared with a neonatal Fc receptor (FcRn) inhibitor.

Studies spotlight the effectiveness of meningococcal infection risk mitigation strategies for treatment with Ultomiris and Soliris

Two oral presentations at the MGFA Scientific Session will reinforce the effectiveness of Alexion’s meningococcal infection risk mitigation strategies. An analysis of the Alexion safety database will suggest that US Neisseria meningitidis-related risk mitigation strategies are effective in patients receiving ULTOMIRIS or SOLIRIS. Separately, a study of antibiotic prophylaxis (AB-PPx) risk mitigation practices across pivotal trials of Alexion’s C5 inhibitors in adults will demonstrate AB-PPx durations varied at initiation of treatment with ULTOMIRIS or SOLIRIS, with the majority of patients receiving it for either 1-30 days (US) or >1y (ex-US). Penicillin-class AB-PPx was most utilized, regardless of geography.  

Real-world evidence underscores HCP confidence in ULTOMIRIS and its value across the gMG treatment paradigm

At the MGFA Scientific Session, a poster presentation of a retrospective subanalysis of US medical records will show that patients treated with ULTOMIRIS within 2 years of gMG diagnosis trended toward greater improvements in MG-ADL scores, minimal manifestation and MG exacerbation-associated hospitalizations than those receiving efgartigimod, a FcRn inhibitor. A separate poster presentation will highlight real-world evidence showing that patients with gMG who initiated treatment with ULTOMIRIS had a statistically significantly greater reduction in healthcare resource utilization than patients who started on efgartigimod.

Two additional poster presentations will provide real-world insights on the drivers of healthcare provider-initiated switches to ULTOMIRIS in gMG, revealing that transitions were driven by unmet needs and provider confidence in ULTOMIRIS, or limitations of intermittent therapy and inability to meet expectations for improved, sustained disease control.

Alexion presentations during the 2025 AANEM Annual Meeting and MGFA Scientific Session

INDICATION(S) & IMPORTANT SAFETY INFORMATION FOR ULTOMIRIS ^® (ravulizumab-cwvz)

INDICATION(S)

What is ULTOMIRIS?

ULTOMIRIS is a prescription medicine used to treat:

• adults and children 1 month of age and older with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH.
• adults and children 1 month of age and older with a disease called atypical Hemolytic Uremic Syndrome (aHUS). ULTOMIRIS is not used in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
• adults with a disease called generalized Myasthenia Gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.
• adults with a disease called Neuromyelitis Optica Spectrum Disorder (NMOSD) who are anti-aquaporin 4 (AQP4) antibody positive.

It is not known if ULTOMIRIS is safe and effective in children younger than 1 month of age.

It is not known if ULTOMIRIS is safe and effective for the treatment of gMG or NMOSD in children.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about ULTOMIRIS?

ULTOMIRIS is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.

• ULTOMIRIS increases your chance of getting serious meningococcal infections that may quickly become life-threatening or cause death if not recognized and treated early.

1. You must complete or update meningococcal vaccine(s) at least 2 weeks before your first dose of ULTOMRIS.
2. If you have not completed your meningococcal vaccines and ULTOMIRIS must be started right away, you should receive the required vaccine(s) as soon as possible.
3. If you have not been vaccinated and ULTOMIRIS must be started right away, you should also receive antibiotics for as long as your healthcare provider tells you.
4. If you had a meningococcal vaccine in the past, you might need additional vaccines before starting ULTOMIRIS. Your healthcare provider will decide if you need additional meningococcal vaccines.
5. Meningococcal vaccines do not prevent all meningococcal infections. Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: fever, fever with high heart rate, headache and fever, confusion, muscle aches with flu-like symptoms, fever and a rash, headache with nausea or vomiting, headache with a stiff neck or stiff back, or eyes sensitive to light.

Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. Your risk of meningococcal infection may continue for several months after your last dose of ULTOMIRIS. It is important to show this card to any healthcare provider who treats you. This will help them diagnose and treat you quickly.

ULTOMIRIS is only available through a program called the ULTOMIRIS and SOLIRIS Risk Evaluation and Mitigation Strategy (REMS). Before you can receive ULTOMIRIS, your healthcare provider must: enroll in the REMS program; counsel you about the risk of serious meningococcal infections; give you information about the signs and symptoms of serious meningococcal infection; make sure that you are vaccinated against serious infections caused by meningococcal bacteria, and that you receive antibiotics if you need to start ULTOMIRIS right away and are not up to date on your vaccines; give you a Patient Safety Card about your risk of meningococcal infection.

ULTOMIRIS may also increase the risk of other types of serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria gonorrhoeae.  If your child is treated with ULTOMIRIS, your child should receive vaccines against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). • Certain people may be at risk of serious infections with gonorrhea. Talk to your healthcare provider about whether you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing. 

Who should not receive ULTOMIRIS?

Do not receive ULTOMIRIS if you have a serious meningococcal infection when you are starting ULTOMIRIS treatment.

Before you receive ULTOMIRIS, tell your healthcare provider about all of your medical conditions, including if you:

• have an infection or fever
• are pregnant or plan to become pregnant.  It is not known if ULTOMIRIS will harm your unborn baby.
  • Pregnancy Registry: There is a registry for pregnant women who take ULTOMIRIS. The purpose of this registry is to check the health of the pregnant mother and her baby. If you are pregnant or become pregnant while taking ULTOMIRIS, talk to your healthcare provider about how you can join this pregnancy registry or you may contact the registry at 1-833-793-0563 or www.UltomirisPregnancyStudy.com to enroll.
• are breastfeeding or plan to breastfeed.  It is not known if ULTOMIRIS passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ULTOMIRIS and other medicines can affect each other causing side effects. Know the medicines you take and the vaccines you receive. Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

If you have PNH and you stop receiving ULTOMIRIS, your healthcare provider will need to monitor you closely for at least 16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in your red blood cell count, tiredness, blood in your urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males.

If you have aHUS, your healthcare provider will need to monitor you closely for at least 12 months after stopping treatment for signs of worsening aHUS or problems related to a type of abnormal clotting and breakdown of your red blood cells called thrombotic microangiopathy (TMA). Symptoms or problems that can happen with TMA may include: confusion or loss of consciousness, seizures, chest pain (angina), difficulty breathing and blood clots or stroke.

What are the possible side effects of ULTOMIRIS?

ULTOMIRIS can cause serious side effects including infusion-related reactions. Infusion-related reactions may happen during your ULTOMIRIS treatment. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, stomach (abdominal) pain, muscle spasms, changes in blood pressure, tiredness, feeling faint, shaking chills (rigors), discomfort in your arms or legs, or bad taste. Stop treatment of ULTOMIRIS and tell your healthcare provider right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion-related reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, or feel faint or pass out

The most common side effects of ULTOMIRIS in people treated for PNH are upper respiratory tract infection and headache.

The most common side effects of ULTOMIRIS in people treated for aHUS are upper respiratory tract infection, diarrhea, nausea, vomiting, headache, high blood pressure and fever.

The most common side effects of ULTOMIRIS in people with gMG are diarrhea and upper respiratory tract infections.

The most common side effects of ULTOMIRIS in people with NMOSD are COVID-19 infection, headache, back pain, urinary tract infection, and joint pain (arthralgia).

Tell your healthcare provider about any side effect that bothers you or that does not go away.  These are not all of the possible side effects of ULTOMIRIS.  Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please see the accompanying full Prescribing Information and Medication Guide for ULTOMIRIS, including Boxed WARNING regarding serious meningococcal infections.  

INDICATION(S) & IMPORTANT SAFETY INFORMATION FOR SOLIRIS^® eculizumab)

INDICATION(S) 

What is SOLIRIS?

SOLIRIS is a prescription medicine used to treat:

• people with paroxysmal nocturnal hemoglobinuria (PNH).
• people with atypical hemolytic uremic syndrome (aHUS). SOLIRIS is not for use in treating people with Shiga toxin E. coli related hemolytic uremic syndrome (STEC-HUS).
• people 6 years of age and older with generalized myasthenia gravis (gMG) who are anti-acetylcholine receptor (AChR) antibody positive.
• adults with a disease called neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive.

It is not known if SOLIRIS is safe and effective in children with PNH or NMOSD.

It is not known if SOLIRIS is safe and effective in children with gMG below 6 years of age.

IMPORTANT SAFETY INFORMATION 

What is the most important information I should know about SOLIRIS?

SOLIRIS is a medicine that affects your immune system and may lower the ability of your immune system to fight infections.  

• SOLIRIS increases your chance of getting serious meningococcal infections that may quickly become life-threatening or cause death if not recognized and treated early.

1. You must complete or update your meningococcal vaccine(s)at least 2 weeks before your first dose of SOLIRIS.
2. If you have not been vaccinated and SOLIRIS must be started right away, you should receive the required vaccine(s) as soon as possible.
3. If you have not been vaccinated and SOLIRIS must be started right away, you should also receive antibiotics for as long as your healthcare provider tells you.
4. If you had a meningococcal vaccine in the past, you might need additional vaccines before starting SOLIRIS. Your healthcare provider will decide if you need additional meningococcal vaccines.
5. Meningococcal vaccines do not prevent all meningococcal infections.  Call your healthcare provider or get emergency medical care right away if you get any of these signs and symptoms of a serious meningococcal infection: fever, fever with high heart rate, headache and fever, confusion, muscle aches with flu-like symptoms, fever and rash, headache with nausea or vomiting, headache with a stiff neck or stiff back, or eyes sensitive to light.

Your healthcare provider will give you a Patient Safety Card about the risk of serious meningococcal infection.  Carry it with you at all times during treatment and for 3 months after your last dose of SOLIRIS. Your risk of meningococcal infection may continue for several weeks after your last dose of SOLIRIS.  It is important to show this card to any healthcare provider who treats you.  This will help them diagnose and treat you quickly.   

SOLIRIS is only available through a program called the ULTOMIRIS and SOLIRIS Risk Evaluation and Mitigation Strategy (REMS).  Before you can receive SOLIRIS, your healthcare provider must: enroll in the REMS program; counsel you about the risk of serious meningococcal infections; give you information about the signs and symptoms of serious meningococcal infection; make sure that you are vaccinated against serious infections caused by meningococcal bacteria, and that you receive antibiotics if you need to start SOLIRIS right away and you are not up to date on your vaccines;  give you a Patient Safety Card about your risk of meningococcal infection. 

SOLIRIS may also increase the risk of other types of serious infections caused by encapsulated bacteria, including Streptococcus pneumoniae, Haemophilus influenzae, and Neisseria gonorrhoeae.   If your child is treated with SOLIRIS, your child should receive vaccines against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib). Certain people may be at risk of serious infections with gonorrhea. Talk to your healthcare provider about whether you are at risk for gonorrhea infection, about gonorrhea prevention, and regular testing.  Certain fungal infections (aspergillus) may also happen if you take SOLIRIS and have a weak immune system or a low white blood cell count. 

Who should not receive SOLIRIS? 

Do not receive SOLIRIS if you have a serious meningococcal infection when you are starting SOLIRIS treatment.

Before you receive SOLIRIS, tell your healthcare provider about all of your medical conditions, including if you: have an infection or fever, are pregnant or plan to become pregnant, and are breastfeeding or plan to breastfeed.  It is not known if SOLIRIS will harm your unborn baby or if it passes into your breast milk. 

Tell your healthcare provider about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. SOLIRIS and other medicines can affect each other causing side effects. Know the medications you take and the vaccines you receive.  Keep a list of them to show your healthcare provider and pharmacist when you get a new medicine.

 If you have PNH, your healthcare provider will need to monitor you closely for at least 8 weeks after stopping SOLIRIS. Stopping treatment with SOLIRIS may cause breakdown of your red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the number of your red blood cell count, drop in your platelet count, confusion, kidney problems, blood clots, difficulty breathing, and chest pain. 

If you have aHUS, your healthcare provider will need to monitor you closely during and for at least 12 weeks after stopping SOLIRIS for signs of worsening aHUS symptoms or problems related to abnormal clotting (thrombotic microangiopathy). Symptoms or problems that can happen with abnormal clotting may include: stroke, confusion, seizure, chest pain (angina), difficulty breathing, kidney problems, swelling in arms or legs, and a drop in your platelet count.  

What are the possible side effects of SOLIRIS? 

 SOLIRIS can cause serious side effects including serious infusion-related reactions. Tell your healthcare provider or nurse right away if you get any of these symptoms during your SOLIRIS infusion: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and feel faint or pass out.  If you have an infusion-related reaction to SOLIRIS, your healthcare provider may need to infuse SOLIRIS more slowly, or stop SOLIRIS.   

The most common side effects in people with PNH treated with SOLIRIS include: headache, back pain, pain or swelling of your nose or throat (nasopharyngitis), and nausea.

The most common side effects in people with aHUS treated with SOLIRIS include:  headache, diarrhea, high blood pressure (hypertension), common cold (upper respiratory infection), stomach-area (abdominal) pain, vomiting, pain or swelling of your nose or throat (nasopharyngitis), low red blood cell count (anemia), cough, swelling of legs or feet (peripheral edema), nausea, urinary tract infections, and fever. 

The most common side effects in people with gMG treated with SOLIRIS include: muscle and joint (musculoskeletal) pain.  

The most common side effects in people with NMOSD treated with SOLIRIS include: common cold (upper respiratory infection), pain or swelling of your nose or throat (nasopharyngitis), diarrhea, back pain, dizziness, flu like symptoms (influenza) including fever, headache, tiredness, cough, sore throat, and body aches, joint pain (arthralgia), throat irritation (pharyngitis), and bruising (contusion).  

Tell your healthcare provider about any side effect that bothers you or that does not go away.  These are not all the possible side effects of SOLIRIS.  For more information, ask your healthcare provider or pharmacist. Call your healthcare provider for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088. 

Please see the accompanying full Prescribing Information and Medication Guide for SOLIRIS, including Boxed WARNING regarding serious meningococcal infections. 

Notes

ULTOMIRIS^® (ravulizumab-cwvz)

ULTOMIRIS^® (ravulizumab), the longest-acting C5 complement inhibitor, provides immediate, complete and sustained complement inhibition. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. Following a loading dose, ULTOMIRIS is administered intravenously every eight weeks in adults, or every four or eight weeks in pediatric patients (based on body weight).

ULTOMIRIS is approved in the US, EU, Japan and other countries for the treatment of certain adults with paroxysmal nocturnal hemoglobinuria (PNH) and is also approved for certain children with PNH in the US, EU and other countries.

ULTOMIRIS is also approved in the US, EU, Japan and other countries for the treatment of certain adults and children with atypical hemolytic uraemic syndrome (aHUS).

Additionally, ULTOMIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG).

Further, ULTOMIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD).

ULTOMIRIS is being assessed as a treatment for additional indications as part of a broad development program.

SOLIRIS^® (eculizumab)

SOLIRIS^® (eculizumab) is a first-in-class C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body’s immune system. When activated in an uncontrolled manner, the terminal complement cascade over-responds, leading the body to attack its own healthy cells. SOLIRIS is administered intravenously every two weeks, following an introductory dosing period.

SOLIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain children and adults with paroxysmal nocturnal hemoglobinuria (PNH). 

SOLIRIS is also approved in the US, EU, Japan, China and other countries for the treatment of certain children and adults with atypical hemolytic uremic syndrome (aHUS).

Additionally, SOLIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with generalized myasthenia gravis (gMG), and for certain pediatric patients with gMG in the US, EU, Japan and other countries.

Further, SOLIRIS is approved in the US, EU, Japan, China and other countries for the treatment of certain adults with neuromyelitis optica spectrum disorder (NMOSD). 

SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome.

Alexion
Alexion, AstraZeneca Rare Disease, is focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and delivery of life-changing medicines. A pioneering leader in rare disease for more than three decades, Alexion was the first to translate the complex biology of the complement system into transformative medicines, and today it continues to build a diversified pipeline across disease areas with significant unmet need, using an array of innovative modalities. As part of AstraZeneca, Alexion is continually expanding its global geographic footprint to serve more rare disease patients around the world. It is headquartered in Boston, US. For more information, please visit www.alexion.us.

AstraZeneca
AstraZeneca is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialization of prescription medicines in Oncology, Rare Diseases and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca’s innovative medicines are sold in more than 125 countries and used by millions of patients worldwide. Please visit www.astrazeneca-us.com and follow the Company on social media @AstraZeneca.

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