Alexion data presented at the 2025 AAN Annual Meeting highlight advancements in improving care for rare neurological diseases

Clinical and real-world evidence will reinforce the long-term safety profile and sustained efficacy of ULTOMIRIS^® and SOLIRIS^® in NMOSD and gMG

Alexion, AstraZeneca Rare Disease, will present robust clinical and real-world data from its rare neurology portfolio at the American Academy of Neurology (AAN) Annual Meeting in San Diego, CA, April 5 to 9, 2025. The company will present 21 abstracts, including three oral presentations, across neuromyelitis optica spectrum disorder (NMOSD) and generalized myasthenia gravis (gMG).

Presentations will offer analyses of the CHAMPION-NMOSD and PREVENT trials as well as real-world data evaluating the long-term safety profile and efficacy of ULTOMIRIS^®  (ravulizumab-cwvz) and SOLIRIS^® (eculizumab) in NMOSD and gMG.

Christophe Hotermans, Senior Vice President, Head of Global Medical Affairs, Alexion, said: “At this year’s AAN, we continue to build on our legacy of innovation for people living with rare diseases by presenting new real-world and clinical evidence that highlight the transformational impact of ULTOMIRIS and SOLIRIS in the long-term treatment of NMOSD and gMG. This latest data will reinforce the sustained efficacy and safety profile of ULTOMIRIS and SOLIRIS, as well as the benefits of long-term treatment in these patient populations, offering meaningful insights to help improve patient care and deepen disease understanding.”

Continued evidence reinforcing the safety profile and efficacy of ULTOMIRIS and SOLIRIS

Two oral presentations will provide clinical and real-world evidence supporting the sustained long-term safety profile and efficacy of ULTOMIRIS and SOLIRIS in adults with the most common forms of NMOSD and gMG, respectively.

An interim analysis of the ongoing Phase III CHAMPION-NMOSD clinical trial will reinforce the potential for ULTOMIRIS to prevent relapses in adults with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) NMOSD. Data will demonstrate that zero patients treated with ULTOMIRIS experienced an adjudicated relapse through a median follow-up of 170.3 weeks. In addition, most patients treated with ULTOMIRIS were clinically stable or improved on disability measures through the longer-term follow-up highlighting the importance of preventing relapses to reduce disability decline in patients with NMOSD.

Results from a presentation spotlighting the three-year analysis from postmarketing surveillance (PMS) in Japan will show that treatment with SOLIRIS demonstrated sustained effectiveness in patients with anti-acetylcholine receptor (AChR) antibody-positive (Ab+) gMG, regardless of Myasthenia Gravis Foundation of America (MGFA) classification, which is used to assess the severity of disease.  Additionally, in an encore poster presentation, results from the first interim analysis from the ULTOMIRIS gMG PMS in Japan will show that clinical effectiveness benefits were maintained in adult patients with AChR-Ab+ gMG who switched to ULTOMIRIS from prior SOLIRIS treatment.

Real-world data underscore benefit of C5 inhibitors in NMOSD and gMG clinical practice

A poster featuring real-world evidence from a global NMOSD registry will underscore the strong clinical benefit of ULTOMIRIS and SOLIRIS in relapse prevention in adults with AQP4-Ab+ NMOSD.

Global gMG registry findings will be shared in poster presentations, including real-world data demonstrating reduced oral corticosteroid burden in patients with AChR-Ab+ gMG receiving SOLIRIS or ULTOMIRIS and reduced hospitalizations, MG crises and exacerbations among patients with AChR-Ab+ gMG receiving ULTOMIRIS.

Advancing understanding and innovation for the NMOSD and gMG communities

An oral presentation will share results of a study evaluating glial fibrillary acidic protein (GFAP) and Neurofilament Light Chain (NfL), biomarkers of astrocyte and neuronal injury, respectively, as potential indicators of disease activity in patients with AQP4-Ab+ NMOSD. In this analysis, high baseline GFAP and NfL levels in PREVENT and CHAMPION-NMOSD serum samples were associated with clinical characteristics related to age and ambulation. Data will show that no associations were observed between levels of these biomarkers and relapse risk or time to first adjudicated on-trial relapse.

A poster presentation will showcase preliminary results from Ad Scientiam’s decentralized research study, funded by Alexion, highlighting the potential clinical value of using the app-based tool, ME& MGopen^TM, to collect digital data to track the daily impact of gMG symptoms in adults with AChR-Ab+ gMG, supporting its use in patient care.

Two poster presentations will feature ongoing clinical studies, including the study design for the Phase III, open-label, single-arm trial evaluating ULTOMIRIS in pediatric patients with AChR-Ab+ gMG. Additionally, an encore poster will provide an overview of the ongoing Phase III PREVAIL trial assessing the safety profile and efficacy of gefurulimab, a novel, dual-binding nanobody, optimized for weekly subcutaneous administration, in adult patients with AChR-Ab+ gMG.

Further, results from Project ASPIRE (eliminAte diSparities and Promote equIty in Rare diseasE) will highlight health disparities among persons of color (POC) who are living with gMG, underscoring the need for nationwide action plans to reduce barriers to healthcare and support for those who are disproportionally affected.

Alexion presentations during AAN 2025

*Ad Scientiam research study supported by Alexion

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-cwvz), 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 for certain children with PNH in the US and EU.

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

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

Further, ULTOMIRIS is approved in the US, EU, Japan 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 (LSE/STO/Nasdaq: AZN) 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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