ALXN Alexion Pharmaceuticals Inc.

182.5
0  0%
Previous Close 182.5
Open
52 Week Low 99.91
52 Week High 187.45
Market Cap $40,336,009,475
Shares 221,019,230
Float 220,513,908
Enterprise Value $39,569,509,475
Volume 0
Av. Daily Volume 4,202,617
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Upcoming Catalysts

Drug Stage Catalyst Date
ALXN1840 (WTX101)
Wilson disease
Phase 3
Phase 3
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ALXN1720
Healthy volunteers
Phase 1
Phase 1
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ULTOMIRIS (ravulizumab-cwvz) - CHAMPION-ALS
Amyotrophic lateral sclerosis (ALS)
Phase 3
Phase 3
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ALXN2050
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2
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CAEL‐101
AL amyloidosis
Phase 3
Phase 3
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ULTOMIRIS (ravulizumab-cwvz)
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Phase 3
Phase 3
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ALXN2040 add-on therapy
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ULTOMIRIS (ravulizumab-cwvz)
Generalized myasthenia gravis (gMG)
Phase 3
Phase 3
Phase 3 top-line data met primary endpoint - July 15, 2021.
ULTOMIRIS (ravulizumab-cwvz)
Paroxysmal Nocturnal Hemoglobinuria (PNH) - children
Approved
Approved
FDA approval announced June 7, 2021.
Acoramidis (AG10) / BBP-265
ATTR-PN
Phase 2
Phase 2
Phase 3 trial has been initiated.
SOLIRIS (Eculizumab)
Refractory generalized myasthenia gravis (gMG)
Approved
Approved
Approval announced October 23, 2017.
SOLIRIS (eculizumab)
Neuromyelitis Optica Spectrum Disorder (NMOSD) - children
Phase 2/3
Phase 2/3
Phase 2/3 trial underway.
SOLIRIS (Eculizumab)
Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD)
Approved
Approved
FDA Approval announced June 27, 2019.
ANDEXXA (Andexanet alfa)
Factor Xa inhibitor reversal agent
Approved
Approved
Prior Approval Supplement (PAS) FDA Approval announced December 31, 2018.
BEVYXXA (betrixaban)
Venous thromboembolism (VTE) Prevention
Approved
Approved
FDA Approval noted June 23, 2017.
ULTOMIRIS (ravulizumab-cwvz)
Dermatomyositis (DM)
Phase 2/3
Phase 2/3
Phase 2/3 trial to be initiated 2H 2021.
ULTOMIRIS (ravulizumab-cwvz)
Complement Mediated Thrombotic Microangiopathy (CM-TMA)
Phase 3
Phase 3
Phase 3 trial to be initiated 2Q 2021.
ULTOMIRIS (ravulizumab-cwvz)
Hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA)
Phase 3
Phase 3
Phase 3 trial initiated 4Q 2020.
ULTOMIRIS (ravulizumab-cwvz) subcutaneous
Paroxysmal nocturnal hemoglobinuria (PNH) and atypical Hemolytic Uremic Syndrome (aHUS)
Phase 3
Phase 3
Phase 3 trial met primary endpoint - June 24, 2020. Regulatory filing due 3Q 2021.
ALXN1830 (subcutaneous)
Warm autoimmune hemolytic anemia (WAIHA)
Phase 2
Phase 2
Phase 2 trial to be initiated 2H 2021.
ALXN2040
Geographic Atrophy (GA)
Phase 2
Phase 2
Phase 2 trial to be initiated 2H 2021.
ALXN1820
Healthy volunteers
Phase 1
Phase 1
Phase 1 trial has been initiated.
ALXN1850
Hypophosphatasia (HPP)
Phase 1
Phase 1
Phase 1 trial to be initiated 2Q 2021.
ALXN2040
COVID-19
Phase 2
Phase 2
Phase 2 trial has been initiated.
ULTOMIRIS (ravulizumab-cwvz)
COVID-19 Coronavirus
Phase 3
Phase 3
Independent data monitoring committee recommended pausing enrollment due to lack of efficacy in pre-specified interim analysis - January 13, 2021.
Elamipretide
Leber’s hereditary optic neuropathy (LHON)
Phase 2
Phase 2
Phase 2 trial did not meet primary endpoint.
ALXN2040
C3 Glomerulopathy (C3G)
Phase 2
Phase 2
Development discontinued - noted July 29, 2020.
ULTOMIRIS (ravulizumab-cwvz)
atypical Hemolytic Uremic Syndrome (aHUS)
Approved
Approved
FDA Approval announced October 18, 2019.
ULTOMIRIS (ravulizumab-cwvz)
Paroxysmal nocturnal hemoglobinuria (PNH)
Approved
Approved
FDA approval announced December 21, 2018.

Latest News

  1. NEW YORK, July 15, 2021 /PRNewswire/ -- Moderna Inc. (NASD:MRNA) will replace Alexion Pharmaceuticals Inc. (NASD:ALXN) in the S&P 500 effective prior to the opening of trading on Wednesday, July 21. AstraZeneca Plc (LSE:AZN; NASD:AZN) is acquiring Alexion Pharmaceuticals in a deal expected to be completed soon pending final closing conditions.

    Following is a summary of the changes that will take place prior to the open of trading on the effective date:

    Effective Date

    Index Name      

    Action

    Company Name

    Ticker

    GICS Sector

    July 21, 2021

    S&P 500

    Addition

    Moderna

    MRNA

    Health Care


    S&P 500

    Deletion

    Alexion Pharmaceuticals

    ALXN

    Health Care

    For more information about S&P Dow Jones Indices, please visit www.spdji.com

    ABOUT S&P DOW JONES INDICES

    S&P…

    NEW YORK, July 15, 2021 /PRNewswire/ -- Moderna Inc. (NASD:MRNA) will replace Alexion Pharmaceuticals Inc. (NASD:ALXN) in the S&P 500 effective prior to the opening of trading on Wednesday, July 21. AstraZeneca Plc (LSE:AZN; NASD:AZN) is acquiring Alexion Pharmaceuticals in a deal expected to be completed soon pending final closing conditions.

    Following is a summary of the changes that will take place prior to the open of trading on the effective date:

    Effective Date

    Index Name      

    Action

    Company Name

    Ticker

    GICS Sector

    July 21, 2021

    S&P 500

    Addition

    Moderna

    MRNA

    Health Care



    S&P 500

    Deletion

    Alexion Pharmaceuticals

    ALXN

    Health Care

    For more information about S&P Dow Jones Indices, please visit www.spdji.com

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    S&P Dow Jones Indices is the largest global resource for essential index-based concepts, data and research, and home to iconic financial market indicators, such as the S&P 500® and the Dow Jones Industrial Average®. More assets are invested in products based on our indices than products based on indices from any other provider in the world. Since Charles Dow invented the first index in 1884, S&P DJI has been innovating and developing indices across the spectrum of asset classes helping to define the way investors measure and trade the markets.

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  2. Study met primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26 –

    ULTOMIRIS demonstrated compelling efficacy as early as Week 1, sustained for 52 weeks –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced positive topline results from a Phase 3 study evaluating the safety and efficacy of ULTOMIRIS® (ravulizumab-cwvz) in adults with generalized myasthenia gravis (gMG). The study met, with high statistical significance, its primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score, a patient-reported assessment, at Week 26, and for the subset of patients who have completed 26 weeks in the…

    Study met primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26 –

    ULTOMIRIS demonstrated compelling efficacy as early as Week 1, sustained for 52 weeks –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced positive topline results from a Phase 3 study evaluating the safety and efficacy of ULTOMIRIS® (ravulizumab-cwvz) in adults with generalized myasthenia gravis (gMG). The study met, with high statistical significance, its primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score, a patient-reported assessment, at Week 26, and for the subset of patients who have completed 26 weeks in the extension study to date, the positive treatment effect was maintained through a total of 52 weeks. ULTOMIRIS was well tolerated with a safety profile consistent with that observed in Phase 3 studies in paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). Based on these results, Alexion plans to make regulatory filings in the U.S., European Union and Japan in late 2021/early 2022.

    "The treatment landscape for people living with gMG has advanced and expanded rapidly in recent years, empowering both patients and caregivers. However, as a clinician and scientist, I know the work is not done," said Professor James F. Howard, M.D., Department of Neurology at The University of North Carolina, Chapel Hill, USA, and lead primary investigator in the Phase 3 study. "These Phase 3 ULTOMIRIS results reinforce the critical role complement inhibition plays in treating gMG. I am encouraged by the opportunity this could provide for more patients to be treated early with a mechanism of action designed to preserve neuromuscular function."

    "The approval of SOLIRIS was a critically important first step in addressing the urgent need for a treatment for people with severe symptoms and complications of MG, and was the first new treatment for this devastating disease in more than 60 years. Today's results demonstrate that ULTOMIRIS may help a broader range of patients than was studied in the SOLIRIS Phase 3 trial, including those with milder symptoms or earlier in their treatment journey, while still offering clinically meaningful benefits that were seen as early as Week 1 and maintained up to 52 weeks," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "These data provide confidence that ULTOMIRIS has the potential to become the new standard of care for gMG and may reduce patient burden with its less frequent dosing schedule, leading to better treatment adherence and patient satisfaction. We are working to prepare regulatory submissions in the U.S., EU and Japan as quickly as possible."

    About the Phase 3 Study

    This global Phase 3 randomized, double-blind, placebo-controlled, multicenter 26-week study evaluated the safety and efficacy of ULTOMIRIS in adults with gMG who were not previously treated with a complement inhibitor medicine. The study enrolled 175 patients across North America, Europe, Asia-Pacific and Japan. To enter the study, participants were required to have a confirmed MG diagnosis at least 6 months prior to the screening visit with a positive serologic test for anti-AChR antibodies, MG-ADL total score of at least 6 at study entry and Myasthenia Gravis Foundation of America (MGFA) Clinical Classification Class II to IV at screening. There was no requirement for prior treatment failure, and patients could stay on stable standard of care medicines, with a few exceptions, for the duration of the study.

    Patients were randomized 1:1 to receive ULTOMIRIS or placebo for a total of 26 weeks. Patients received a single weight-based loading dose on Day 1, followed by regular weight-based maintenance dosing beginning on Day 15, every 8 weeks. The primary endpoint of change from baseline in the Myasthenia Gravis-Activities of Daily Living Profile (MG-ADL) total score at Week 26 was assessed along with multiple secondary endpoints evaluating improvement in disease-related and quality of life measures.

    The study met its primary endpoint, with a statistically significant change in MG-ADL score from baseline through Week 26 for patients receiving ULTOMIRIS compared to those receiving placebo (ULTOMIRIS: -3.1, placebo: -1.4, treatment difference: -1.6, p<0.001).

    In the prospectively-defined secondary endpoints of change from baseline through Week 26 in Quantitative Myasthenia Gravis (QMG) total score – a physician-administered assessment of MG clinical severity – as well as the proportion of patients who achieved an improvement of at least 5 points in QMG, ULTOMIRIS also demonstrated clinically meaningful and statistically significant improvements (p<0.001 and p=0.005, respectively). Nearly three times as many patients receiving ULTOMIRIS experienced an improvement of at least 5 points in their QMG score compared to patients receiving placebo (30.0% vs 11.3%). These improvements in MG-ADL and QMG scores were observed as early as Week 1 and were sustained through Week 26.

    Additional secondary endpoints assessing qualify of life measures, such as Revised 15-Component Myasthenia Gravis Quality of Life (MG-QOL15r) score (p=0.064) and Neuro-QOL Fatigue score (p=0.373), did not meet statistical significance at Week 26. The proportion of patients who achieved an improvement of at least 3 points in MG-ADL score (ULTOMIRIS: 56.7%, placebo: 34.1%, nominal p=0.005), was not considered statistically significant based on hierarchical testing.

    During the randomized controlled period, adverse events were comparable between the ULTOMIRIS and placebo groups. The most frequently observed adverse events were headache (ULTOMIRIS: 18.6%; placebo: 25.8%), diarrhea (ULTOMIRIS: 15.1%; placebo: 12.4%) and nausea (ULTOMIRIS: 10.5%; placebo: 10.1%). The most frequently observed serious adverse events were MG crisis (ULTOMIRIS: 1.2%) and MG worsening (placebo: 3.4%).

    Through 52 weeks (26 weeks randomized controlled period + 26 weeks of open-label extension), there were four patient deaths in the ULTOMIRIS group – three of them were due to COVID-19 and none were considered related to treatment with ULTOMIRIS. No cases of meningococcal infection were observed through 52 weeks.

    Patients who completed the randomized controlled period were eligible to continue into an open-label extension period evaluating the safety and efficacy of ULTOMIRIS for up to two years, which is ongoing. At the time of this preliminary analysis of the open-label extension period, 75 patients had completed 26 weeks of treatment, for a total of 52 weeks of treatment. Among the patients who received ULTOMIRIS in the randomized controlled period, the treatment effects were maintained through an additional 26 weeks of treatment, demonstrating sustained efficacy for a total of 52 weeks. In addition, patients who received placebo in the randomized controlled period and switched to ULTOMIRIS at the beginning of the open-label extension showed immediate and sustained improvement in MG-ADL and QMG scores in a similar magnitude and time course to that observed in the ULTOMIRIS group during the randomized controlled period.

    About Generalized Myasthenia Gravis

    Generalized myasthenia gravis (gMG) is a rare autoimmune disorder characterized by severe muscle weakness. In gMG, inflammation causes damage at the connection point between nerve cells and the muscles they control (known as the neuromuscular junction or NMJ). This damage leads to a breakdown of communication between the brain and muscles, causing loss of muscle function and severe weakness.

    About 85 percent of people with gMG produce specific antibodies that bind to the surface of the cells at the NMJ. This binding activates the complement cascade and causes the immune system to attack the NMJ. People with gMG can suffer from initial symptoms, such as slurred speech, droopy eyelids, double vision, and lack of balance, which can often lead to more severe symptoms like choking, impaired swallowing, extreme fatigue and even episodes of respiratory failure.

    gMG can occur at any age, but it most commonly begins for women before the age of 40 and for men after the age of 60. The prevalence of gMG is estimated at 107 to 278 per million people.

    About ULTOMIRIS®

    ULTOMIRIS® (ravulizumab) is the first and only long-acting 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 complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States for the treatment of adults and children (one month of age and older) with paroxysmal nocturnal hemoglobinuria (PNH), as well as in the European Union (EU) and Japan as a treatment for adults with PNH. It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit www.alexion.com.

    INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab-cwvz)

    INDICATIONS

    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).

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

    IMPORTANT SAFETY INFORMATION

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

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

    • ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
    1. You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you are not vaccinated.
    2. If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
    3. If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
    4. If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
    5. Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms and eyes sensitive to light.

    Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.

    ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must: enroll in the ULTOMIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.

    ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilis influenzae type b (Hib) if treated with ULTOMIRIS. Call your doctor right away if you have any new signs or symptoms of infection.

    Who should not receive ULTOMIRIS?

    Do not
    receive ULTOMIRIS if you have a meningococcal infection or have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed.

    Before you receive ULTOMIRIS, tell your doctor 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 ULTOMIRIS will harm your unborn baby or if it passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

    Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

    If you have PNH and you stop receiving ULTOMIRIS, your doctor 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 doctor 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. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and 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 with aHUS are upper respiratory tract infection, diarrhea, nausea, vomiting, headache, high blood pressure and fever.

    Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your doctor or pharmacist. Call your doctor right away if you miss an ULTOMIRIS infusion or 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 and life-threatening meningococcal infections/sepsis.

    About Alexion

    Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com

    [ALXN-P]

    Forward-Looking Statements

    This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion and its products, including statements related to: the anticipated benefits of ULTOMIRIS for gMG patients; Alexion plans to make regulatory filings in the U.S., European Union and Japan in late 2021/early 2022; ULTOMIRIS has the opportunity to provide more patients that are treated early with a mechanism of action designed to preserve neuromuscular function; ULTOMIRIS may help a broader range of patients than was studied in the SOLIRIS Phase 3 trial; ULTOMIRIS has the potential to become the new standard of care for gMG and may reduce patient burden with its less frequent dosing schedule, leading to better treatment adherence and patient satisfaction; and ULTOMIRIS' continued safety and efficacy profile.

    Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those expected by these forward looking statements, including for example: ULTOMIRIS may not be approved for use by regulatory agencies (or such approval may be delayed); the filing of requests for regulatory approval may not ultimately be submitted (or may be delayed); ULTOMIRIS may not generate the expected benefits to patients (or the healthcare system) that are anticipated; anticipated regulatory approvals may be delayed or refused; the Company may experience delays (or be prevented) for obtaining approval or commencing or continuing sales of ULTOMIRIS for gMG, due to manufacturing or other reasons; results of clinical trials may not be sufficient to satisfy regulatory authorities to approve ULTOMIRIS as a treatment for gMG and/or other indication (or they may request additional trials or additional information); results in clinical trials may not be indicative of results from later stage or larger clinical trials (or the use in broader patient populations once the product is approved for use by regulatory agencies); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products including ULTOMIRIS (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexion's business, including on commercial and clinical trial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); our dependence on sales from our complement inhibitors; future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense, manufacturing issues, or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the possibility that current rates of adoption of our products are not sustained; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us (including intellectual property lawsuits relating to ULTOMIRIS brought by third parties); the risk that third party payors (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all; failure to realize the benefits and potential of investments, collaborations, licenses and acquisitions; the possibility that expected tax benefits will not be realized; potential declines in sovereign credit ratings or sovereign defaults in countries where we sell our products; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on our supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including the coronavirus; uncertainties surrounding legal proceedings, company investigations and government investigations; the risk that estimates regarding the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D and other indications we are pursuing are inaccurate; the impact of the proposed transaction between Alexion and AstraZeneca plc; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructurings; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021 and in our other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

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  3. NEW YORK, July 14, 2021 (GLOBE NEWSWIRE) -- Nasdaq (NASDAQ:NDAQ) today announced that Honeywell International Inc. (NASDAQ:HON), will become a component of the NASDAQ-100 Index® (NASDAQ:NDX), the NASDAQ-100 Equal Weighted Index (NASDAQ:NDXE) and the NASDAQ-100 Ex-Technology Index (NASDAQ:NDXX) prior to market open on Wednesday, July 21, 2021. Honeywell International Inc. will replace Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) in the NASDAQ-100 Index®, the NASDAQ-100 Equal Weighted Index and the NASDAQ-100 Ex-Technology Index.

    For more information about the company, go to https://www.honeywell.com/ .

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    Nasdaq (NASDAQ:NDAQ) is a global technology company serving the capital markets and other industries. Our diverse offering of…

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  4. - Exploratory biomarker analyses suggest possible cardiac disease improvements and renal response -

    Caelum Biosciences and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced new Phase 2 safety and tolerability data for CAEL-101, a potentially first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in patients with AL amyloidosis. The data, presented in two e-posters at the European Hematology Association (EHA) Congress 2021, strengthen the safety and tolerability profile of CAEL-101, further support the dose selection for the ongoing Phase 3 study, and suggest possible cardiac and renal response. An e-poster featuring the first data from a new arm of the study demonstrated that CAEL-101 administered…

    - Exploratory biomarker analyses suggest possible cardiac disease improvements and renal response -

    Caelum Biosciences and Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced new Phase 2 safety and tolerability data for CAEL-101, a potentially first-in-class amyloid fibril targeted therapy, in combination with standard-of-care (SoC) therapy in patients with AL amyloidosis. The data, presented in two e-posters at the European Hematology Association (EHA) Congress 2021, strengthen the safety and tolerability profile of CAEL-101, further support the dose selection for the ongoing Phase 3 study, and suggest possible cardiac and renal response. An e-poster featuring the first data from a new arm of the study demonstrated that CAEL-101 administered in combination with cyclophosphamide-bortezomib-dexamethasone (CyBorD) plus daratumumab was generally safe and well-tolerated in the first four weeks of treatment. Data presented in a second e-poster showed longer-term evidence that CAEL-101 in combination with CyBorD was generally well-tolerated for a median treatment duration of 49 weeks, and exploratory clinical biomarker data suggesting possible cardiac disease improvements and renal response among patients with cardiac or renal impairment at baseline, respectively.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210611005037/en/

    "AL amyloidosis is a relentless disease that is particularly devastating when it impacts the heart, with some of these patients facing a median survival of less than one year following diagnosis. Current treatments for AL amyloidosis are designed to prevent or suppress the formation of new amyloids, but they do not address the existing amyloid buildup in the involved organs like the heart and kidneys, which can result in continued organ damage and can ultimately be fatal," said Michael Spector, President and Chief Executive Officer of Caelum. "Understanding that CAEL-101 has the potential to be the first therapy to address the devastating organ damage caused by AL amyloidosis, we are urgently working to advance the ongoing CARES Phase 3 program in collaboration with Alexion."

    Safety and Tolerability of CAEL-101 in Combination with Cyclophosphamide-Bortezomib-Dexamethasone and Daratumumab in Patients with AL amyloidosis (#EP1017)

    As was previously announced, the Phase 2 study of CAEL‑101 in combination with CyBorD met its primary objectives, supporting the safety and tolerability of CAEL-101 and the selection of the 1000 mg/m2 dose for the ongoing Phase 3 study. Results presented from an additional study arm that included 11 patients receiving CAEL-101 (1000 mg/m2 dose) in combination with CyBorD plus daratumumab suggested that treatment with this combination was generally well-tolerated in the first four weeks of treatment. Specifically, adding daratumumab to the CAEL-101 and CyBorD regimen did not result in any new safety signals, nor did it alter the pharmacokinetic (PK) exposure to CAEL-101. The most common adverse events (AEs) reported in the first four weeks in the additional arm were nausea, constipation, and insomnia.

    Safety and Tolerability of CAEL-101 in Patients with AL Amyloidosis in a Phase 2 Study for a Median of 49 Weeks (#EP1018)

    Additional longer-term data presented from the Phase 2 study demonstrated that CAEL-101 in combination with CyBorD in patients with AL amyloidosis (N=13) was generally well tolerated up to a median treatment duration of 49 weeks (range 12-57 weeks), with most patients having received more than 20 infusions of CAEL-101. The most common AEs reported were diarrhea, nausea, fatigue, rash, and anemia. In addition, exploratory clinical biomarker evaluations showed early signals suggesting possible cardiac and renal response. Specifically, median percent changes for biomarkers of cardiac disease (cTnT and NT-proBNP) were lower at each subsequent time point measured, suggesting improvement in cardiac function among eight patients with active cardiac disease at baseline. Additionally, seven patients with active renal impairment at baseline demonstrated renal response, as defined by a decrease of at least 30 percent in proteinuria (an excess of protein in the urine) following treatment.

    "We are grateful to clinical trial participants who are essential to advancing our work towards new treatment options for AL amyloidosis," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "We remain committed to working together with the AL amyloidosis community and Caelum to evaluate the potential of CAEL-101 as a potentially first-in-class treatment option for patients who are living with this devastating disease."

    As was previously announced, the Cardiac Amyloid Reaching for Extended Survival (CARES) Phase 3 clinical program to evaluate CAEL-101 in combination with SoC therapy in AL amyloidosis has begun. Enrollment is underway in two parallel Phase 3 studies – one in patients with Mayo stage IIIa disease (ClinicalTrials.gov Identifier: NCT04512235) and one in patients with Mayo stage IIIb disease (ClinicalTrials.gov Identifier: NCT04504825) – and will collectively enroll approximately 370 patients globally.

    About the CAEL-101 Phase 2 Study

    The Phase 2 multicenter, open-label, dose-selection study (ClinicalTrials.gov Identifier: NCT04304144) is designed to evaluate the safety and tolerability of CAEL-101 in combination with standard of care (SoC) therapy for patients with AL amyloidosis and determine the recommended dose for Phase 3 studies. The study is divided into two parts: Part A examined CAEL-101 in combination with cyclophosphamide-bortezomib-dexamethasone (CyBorD) and employed a 3+3 dose escalation design (cohort 1 – 500 mg/m2; cohort 2 – 750 mg/m2 ; cohort 3 1000 mg/m2); Part A patients were subsequently up titrated to 1000mg/m2, once this was identified as the Phase 3 dose. Part B is examining CAEL-101 at the 1000 mg/m2 dose in combination with CyBorD plus daratumumab. Patients from Parts A and B receive CAEL-101 therapy weekly for the four-week observation period followed by CAEL-101 doses every other week thereafter, all while continuing to receive SoC therapy. Patients continue to receive CAEL-101 per protocol until the end of the study or discontinuation.

    About CAEL-101

    CAEL-101 is a first-in-class monoclonal antibody (mAb) designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis. The antibody is designed to bind to misfolded light chain proteins and amyloid and shows binding to both kappa and lambda subtypes. In a Phase 1a/1b study, CAEL-101 demonstrated improved organ function, including cardiac and renal function, in 27 patients with relapsed and refractory AL amyloidosis who had previously not had an organ response to standard of care therapy. CAEL-101 has received Orphan Drug Designation from both the U.S. Food and Drug Administration and European Medicine Agency as a potential therapy for patients with AL amyloidosis.

    About AL Amyloidosis

    AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded immunoglobulin light chains produced by plasma cells aggregate and form fibrils that deposit in tissues and organs. This deposition can cause widespread and progressive organ damage and high mortality rates, with death most frequently occurring as a result of cardiac failure. Current standard of care includes plasma cell directed chemotherapy and autologous stem cell transplant, but these therapies do not address the organ dysfunction caused by amyloid deposition, and up to 80 percent of patients are ineligible for transplant.

    AL amyloidosis is a rare disease but is the most common form of systemic amyloidosis. There are approximately 22,000 patients across the United States, France, Germany, Italy, Spain and the United Kingdom. AL amyloidosis has a one-year mortality rate of 47 percent, 76 percent of which is caused by cardiac amyloidosis.

    About Caelum Biosciences

    Caelum Biosciences, Inc. ("Caelum") is a clinical-stage biotechnology company developing treatments for rare and life-threatening diseases. Caelum's lead asset, CAEL-101, is a novel antibody for the treatment of patients with amyloid light chain ("AL") amyloidosis. In 2019, Caelum entered a collaboration agreement with Alexion under which Alexion acquired a minority equity interest in Caelum and an exclusive option to acquire the remaining equity in the company. Caelum was founded by Fortress Biotech, Inc. (NASDAQ:FBIO). For more information, visit www.caelumbio.com.

    About Alexion

    Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

    [ALXN-P]

    Forward-Looking Statement

    This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion (and Caelum) and their products, including statements related to: CAEL-101 is a potential first-in-class amyloid fibril targeted therapy; CAEL-101 clinical studies suggest possible positive cardiac and renal response; the anticipated or possible benefits of CAEL-101 for patients (including exploratory clinical biomarker data suggesting possible cardiac disease improvements and renal response among patients with cardiac or renal impairment at baseline); CAEL-101 has the potential to be the first therapy to address the devastating organ damage caused by AL amyloidosis; exploratory clinical biomarker evaluations showed early signals suggesting possible cardiac and renal response; Alexion remains committed to working together with the AL amyloidosis community and Caelum to evaluate the potential of CAEL-101 as a potentially first-in-class treatment option for patients who are living with this devastating disease; CAEL-101 is designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients with AL amyloidosis; CAEL-101 is designed to bind to misfolded light chain protein and amyloid and shows binding to both kappa and lambda subtypes; and characteristics of clinical trials for CAEL-101 including the number and type of patients expected to be enrolled in clinical trials. Forward-looking statements are subject to factors that may cause Alexion's or Caelum's results and plans to differ materially from those expected by these forward looking statements, including for example: CAEL-101 may not generate the expected benefits to patients that are anticipated (including safety and efficacy benefits that were reported in earlier clinical trials); anticipated regulatory approvals may be delayed or declined; results of clinical trials may not be sufficient to satisfy regulatory authorities to approve CAEL-101 as a treatment for AL amyloidosis or other indication (or they may request additional trials or additional information); results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations once the product is approved for use by regulatory agencies); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of CAEL-101 (or failure to adequately operate or manage clinical trials) which could cause us or Caelum to discontinue sales of the product (or halt trials, delay or prevent submission of regulatory approval filings or result in denial of approval of product candidates); the severity of the impact of the COVID-19 pandemic on the businesses, including on commercial and clinical trial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of products; inability to timely submit (or failure to submit) future applications for regulatory approval for products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); Alexion dependence on sales from complement inhibitors; future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of the research, marketing approval or material limitations on the marketing of products; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of products and product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the adequacy of pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us (including intellectual property lawsuits relating to ULTOMIRIS brought by third parties); the risk that third party payors (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all; failure to realize the benefits and potential of investments, collaborations, licenses and acquisitions; the possibility that expected tax benefits will not be realized; potential declines in sovereign credit ratings or sovereign defaults in countries where products are sold; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on our supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including the coronavirus; uncertainties surrounding legal proceedings, company investigations and government investigations; the risk that estimates regarding the number of patients with AL amyloidosis and other indications we are pursuing are inaccurate; the impact of the proposed transaction between Alexion and AstraZeneca plc; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructurings; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021 and in our other filings with the SEC. Alexion and Caelum disclaim any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

    Forward-Looking Statement Regarding Acquisition of Alexion by AstraZeneca

    This communication contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Forward-looking statements are generally identified by the use of forward-looking terminology such as "anticipate," "believe," "continue," "could," "estimate," "expect," "explore," "evaluate," "intend," "may," "might," "plan," "potential," "predict," "project," "seek," "should," or "will," or the negative thereof or other variations thereon or comparable terminology. These forward-looking statements are only predictions and involve known and unknown risks and uncertainties, many of which are beyond Alexion's and AstraZeneca plc's "AstraZeneca") control. Statements in this communication regarding Alexion, AstraZeneca and the combined company that are forward-looking, including anticipated benefits of the proposed transaction, the impact of the proposed transaction on Alexion's and AstraZeneca's businesses and future financial and operating results, the amount and timing of synergies from the proposed transaction, the terms and scope of the expected financing for the proposed transaction, the aggregate amount of indebtedness of the combined company following the closing of the proposed transaction, are based on management's estimates, assumptions and projections, and are subject to significant uncertainties and other factors, many of which are beyond Alexion's and AstraZeneca's control. These factors include, among other things, market factors, competitive product development and approvals, pricing controls and pressures (including changes in rules and practices of managed care groups and institutional and governmental purchasers), economic conditions such as interest rate and currency exchange rate fluctuations, judicial decisions, claims and concerns that may arise regarding the safety and efficacy of in-line products and product candidates, changes to wholesaler inventory levels, variability in data provided by third parties, changes in, and interpretation of, governmental regulations and legislation affecting domestic or foreign operations, including tax obligations, changes to business or tax planning strategies, difficulties and delays in product development, manufacturing or sales including any potential future recalls, patent positions and the ultimate outcome of any litigation matter. Additional information concerning these risks, uncertainties and assumptions can be found in Alexion's and AstraZeneca's respective filings with the SEC, including the risk factors discussed in Alexion's most recent Annual Report on Form 10-K, as updated by its Quarterly Reports on Form 10-Q, in AstraZeneca's most recent Annual Report on Form 20-F and in each company's future filings with the SEC. Important risk factors could cause actual future results and other future events to differ materially from those currently estimated by management, including, but not limited to, the risks that: a condition to the closing the proposed acquisition may not be satisfied; a regulatory approval that may be required for the proposed acquisition is delayed, is not obtained or is obtained subject to conditions that are not anticipated; AstraZeneca is unable to achieve the synergies and value creation contemplated by the proposed acquisition; AstraZeneca is unable to promptly and effectively integrate Alexion's businesses; management's time and attention is diverted on transaction related issues; disruption from the transaction makes it more difficult to maintain business, contractual and operational relationships; the credit ratings of the combined company declines following the proposed acquisition; legal proceedings are instituted against Alexion, AstraZeneca or the combined company; Alexion, AstraZeneca or the combined company is unable to retain key personnel; and the announcement or the consummation of the proposed acquisition has a negative effect on the market price of the capital stock of Alexion or AstraZeneca or on Alexion's or AstraZeneca's operating results. No assurances can be given that any of the events anticipated by the forward-looking statements will transpire or occur, or if any of them do occur, what impact they will have on the results of operations, financial condition or cash flows of Alexion or AstraZeneca. Should any risks and uncertainties develop into actual events, these developments could have a material adverse effect on the proposed transaction and/or Alexion or AstraZeneca, AstraZeneca's ability to successfully complete the proposed transaction and/or realize the expected benefits from the proposed transaction. You are cautioned not to rely on Alexion's and AstraZeneca's forward-looking statements. These forward-looking statements are and will be based upon management's then-current views and assumptions regarding future events and operating performance, and are applicable only as of the dates of such statements. Neither Alexion nor AstraZeneca assumes any duty to update or revise forward-looking statements, whether as a result of new information, future events or otherwise, as of any future date.

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  5.  – With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –

    – Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5 inhibitor that offers immediate, complete and sustained complement inhibition, is now the first and only FDA-approved medicine for children and adolescents with PNH…

     – With this approval, ULTOMIRIS is the first and only medicine approved in the U.S. to treat children and adolescents with PNH –

    – Approval based on interim results from Phase 3 study showing ULTOMIRIS demonstrated complete terminal complement inhibition through 26 weeks –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved the expanded use of ULTOMIRIS® (ravulizumab-cwvz) to include children (one month of age and older) and adolescents with paroxysmal nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5 inhibitor that offers immediate, complete and sustained complement inhibition, is now the first and only FDA-approved medicine for children and adolescents with PNH.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210607005638/en/

    Image of ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL vials (3 mL and 11 mL). (Photo: Business Wire)

    Image of ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL vials (3 mL and 11 mL). (Photo: Business Wire)

    "It can take months, and sometimes years, to receive a correct diagnosis for PNH — a chronic, progressive and potentially life-threatening rare disease – which can be an overwhelming experience for children and their families," said Satheesh Chonat, M.D., principal investigator for the pediatric clinical trial and pediatric hematologist and oncologist at the Aflac Cancer & Blood Disorders Center at Children's Healthcare of Atlanta, as well as assistant professor of pediatrics at the Emory University School of Medicine. "Managing the disease can be extremely burdensome for these children and their families, who often miss school and work for infusions, blood transfusions, and medical appointments. It's exciting to finally have an approved medicine for these patients who are diagnosed as children."

    Since its initial approval in 2018, ULTOMIRIS has quickly become the standard of care in the U.S. for the treatment of adults with PNH. PNH is a complement-mediated disease, which means the symptoms and complications are caused by a lack of regulation, or control, of the complement system, an essential part of the immune system. ULTOMIRIS is designed to target the part of the complement system at the site of disease activity (terminal complement), while preserving function of other parts of the immune system to be able to fight common pathogens and infections. ULTOMIRIS reduces red blood cell destruction in the blood vessels, also known as intravascular hemolysis, and thrombosis (blood clot) risk by providing immediate, complete, and sustained terminal complement inhibition.

    "This expanded approval is a significant step forward for the PNH community as we work to elevate awareness of this rare disease in children and adolescents and ensure patients, both pediatric and adult, have meaningful treatment options available," said Janice Frey-Angel, Chief Executive Officer and Executive Director of the Aplastic Anemia and Myelodysplastic Syndrome International Foundation (AAMDSIF). "PNH can have significant physical, emotional and/or psychological impacts on families, and we are pleased there is now an approved medicine for the younger members of our community and the families who care for them."

    This approval is based on interim Phase 3 study results, which showed that ULTOMIRIS was effective in achieving complete C5 complement inhibition through 26 weeks in children and adolescents up to 18 years of age. Additionally, ULTOMIRIS had no reported treatment-related severe adverse events, and no patients discontinued treatment during the primary evaluation period or experienced breakthrough hemolysis, which can lead to disabling or potentially fatal blood clots. The efficacy and safety of ULTOMIRIS in children and adolescents is consistent with the established profile of ULTOMIRIS in clinical studies involving adults with PNH and is representative of the broad PNH patient population seen in the real-world clinical setting. The results of the pediatric study continue to demonstrate Alexion's commitment to treating complement-mediated diseases, which spans more than 17 clinical trials – including the largest pediatric and adult trials in PNH completed to date – and over 60,000 patient-years of trial and real-world data, across more than 50 countries. Data from the interim analysis will be presented as an e-poster during the European Hematology Association 2021 Virtual Congress and will be made available on the congress website on June 11, 2021 at 9:00 a.m. CEST (3:00 a.m. EDT).

    "PNH can have a profound impact on a child's development and quality of life. With its established safety and efficacy profile, ULTOMIRIS has the potential to transform the lives of children and adolescents suffering from this devastating rare disease," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "We are inspired by the bravery and resilience of the children and adolescents, as well as their families, who participated in the study, and we are grateful for their commitment — as well as that of the trial investigators — to advancing the understanding of PNH and disease management in younger people. We also appreciate the sense of urgency shown by regulators in prioritizing reviewing and approving the first treatment in the U.S. for children and adolescents with PNH."

    Alexion plans to make ULTOMIRIS available to pediatric patients in the U.S. immediately. A regulatory filing for ULTOMIRIS in pediatric patients with PNH is under review in the European Union (EU).

    About Paroxysmal Nocturnal Hemoglobinuria (PNH)

    PNH is a serious ultra-rare blood disorder with devastating consequences. It is characterized by the destruction of red blood cells, which is also referred to as hemolysis. PNH occurs when the complement system—a part of the body's immune system—over-responds, leading the body to attack its own red blood cells. PNH often goes unrecognized, with delays in diagnosis from one to more than five years. Patients with PNH may experience a range of symptoms, such as fatigue, difficulty swallowing, shortness of breath, abdominal pain, erectile dysfunction, dark-colored urine and anemia. The most devastating consequence of chronic hemolysis is the formation of blood clots, which can occur in blood vessels throughout the body, damage vital organs, and potentially lead to premature death. The prognosis of PNH can be poor in many cases, so a timely and accurate diagnosis — in addition to appropriate treatment — is critical to improving patient outcomes.

    About ULTOMIRIS®

    ULTOMIRIS® (ravulizumab-cwvz) is the first and only long-acting 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 complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or, for pediatric patients less than 20 kg, every four weeks, following a loading dose. ULTOMIRIS is approved in the United States (U.S.) for the treatment of adults and children (one month of age and older) with paroxysmal nocturnal hemoglobinuria (PNH), as well as in the European Union (EU) and Japan as a treatment for adults with PNH. It is also approved in the U.S. and Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients, as well as in the EU for the treatment of adults and children with a body weight of at least 10 kg with aHUS. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit www.alexion.com.

    INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS® (ravulizumab-cwvz)

    INDICATIONS

    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).

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

    IMPORTANT SAFETY INFORMATION

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

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

    • ULTOMIRIS increases your chance of getting serious and life-threatening meningococcal infections that may quickly become life-threatening and cause death if not recognized and treated early.
    1. You must receive meningococcal vaccines at least 2 weeks before your first dose of ULTOMIRIS if you are not vaccinated.
    2. If your doctor decided that urgent treatment with ULTOMIRIS is needed, you should receive meningococcal vaccination as soon as possible.
    3. If you have not been vaccinated and ULTOMIRIS therapy must be initiated immediately, you should also receive 2 weeks of antibiotics with your vaccinations.
    4. If you had a meningococcal vaccine in the past, you might need additional vaccination. Your doctor will decide if you need additional vaccination.
    5. Meningococcal vaccines reduce but do not prevent all meningococcal infections. Call your doctor or get emergency medical care right away if you get any of these signs and symptoms of a meningococcal infection: headache with nausea or vomiting, headache and fever, headache with a stiff neck or stiff back, fever, fever and a rash, confusion, muscle aches with flu-like symptoms and eyes sensitive to light.

    Your doctor will give you a Patient Safety Card about the risk of meningococcal infection. Carry it with you at all times during treatment and for 8 months after your last ULTOMIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.

    ULTOMIRIS is only available through a program called the ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor must: enroll in the ULTOMIRIS REMS program; counsel you about the risk of meningococcal infection; give you information and a Patient Safety Card about the symptoms and your risk of meningococcal infection (as discussed above); and make sure that you are vaccinated with a meningococcal vaccine, and if needed, get revaccinated with the meningococcal vaccine. Ask your doctor if you are not sure if you need to be revaccinated.

    ULTOMIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilis influenzae type b (Hib) if treated with ULTOMIRIS. Call your doctor right away if you have any new signs or symptoms of infection.

    Who should not receive ULTOMIRIS?

    Do not receive ULTOMIRIS if you have a meningococcal infection or have not been vaccinated against meningococcal infection unless your doctor decides that urgent treatment with ULTOMIRIS is needed.

    Before you receive ULTOMIRIS, tell your doctor 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 ULTOMIRIS will harm your unborn baby or if it passes into your breast milk. You should not breastfeed during treatment and for 8 months after your final dose of ULTOMIRIS.

    Tell your doctor about all the vaccines you receive and medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements which could affect your treatment.

    If you have PNH and you stop receiving ULTOMIRIS, your doctor 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 doctor 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. Symptoms of an infusion-related reaction with ULTOMIRIS may include lower back pain, feeling faint or discomfort in your arms or legs. Tell your doctor or nurse right away if you develop these symptoms, or any other symptoms during your ULTOMIRIS infusion that may mean you are having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of your face, tongue, or throat, and 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 with aHUS are upper respiratory tract infection, diarrhea, nausea, vomiting, headache, high blood pressure and fever.

    Tell your doctor about any side effect that bothers you or that does not go away. These are not all the possible side effects of ULTOMIRIS. For more information, ask your doctor or pharmacist. Call your doctor right away if you miss an ULTOMIRIS infusion or 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 and life-threatening meningococcal infections/sepsis.

    About Alexion

    Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare diseases and devastating conditions through the discovery, development and commercialization of life-changing medicines. As a leader in rare diseases for more than 25 years, Alexion has developed and commercializes two approved complement inhibitors to treat patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS), as well as the first and only approved complement inhibitor to treat anti-acetylcholine receptor (AchR) antibody-positive generalized myasthenia gravis (gMG) and neuromyelitis optica spectrum disorder (NMOSD). Alexion also has two highly innovative enzyme replacement therapies for patients with life-threatening and ultra-rare metabolic disorders, hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D) as well as the first and only approved Factor Xa inhibitor reversal agent. In addition, the company is developing several mid-to-late-stage therapies, including a copper-binding agent for Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D inhibitor as well as several early-stage therapies, including one for light chain (AL) amyloidosis, a second oral Factor D inhibitor and a third complement inhibitor. Alexion focuses its research efforts on novel molecules and targets in the complement cascade and its development efforts on hematology, nephrology, neurology, metabolic disorders, cardiology, ophthalmology and acute care. Headquartered in Boston, Massachusetts, Alexion has offices around the globe and serves patients in more than 50 countries. This press release and further information about Alexion can be found at: www.alexion.com.

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    For patient or advocacy inquiries please contact .

    Forward-Looking Statements

    This press release contains forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Alexion and its products, including statements related to: the anticipated benefits of ULTOMIRIS for PNH patients (including children and adolescents); ULTOMIRIS' continued safety and efficacy profile; anticipated timing to receive a correct diagnosis for PNH; ULTOMIRIS' expanded approval is a significant step forward for the PNH community; PNH can have a profound impact on a child's development and quality of life; with its established safety and efficacy profile, ULTOMIRIS has the potential to transform the lives of children and adolescents suffering from this devastating rare disease; and Alexion plans to make ULTOMIRIS available to pediatric patients in the U.S. immediately. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those expected by these forward looking statements, including for example: ULTOMIRIS may not generate the expected benefits to patients or the healthcare system that are anticipated; anticipated regulatory approvals may be delayed or refused; the Company may experience delays (or be prevented), due to manufacturing or other reasons, from making available ULTOMIRIS to pediatric patients in the U.S. immediately; results of clinical trials may not be sufficient to satisfy regulatory authorities to approve ULTOMIRIS as a treatment for PNH and/or aHUS or other indication (or they may request additional trials or additional information); results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations (including children and adolescents) once the product is approved for use by regulatory agencies); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products including ULTOMIRIS (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexion's business, including on commercial and clinical trial and clinical development programs; unexpected delays in clinical trials; unexpected concerns regarding products and product candidates that may arise from additional data or analysis obtained during clinical trials or obtained once used by patients following product approval; future product improvements may not be realized due to expense or feasibility or other factors; delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); our dependence on sales from our complement inhibitors; future competition from biosimilars and novel products; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense, manufacturing issues, or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; the possibility that current rates of adoption of our products are not sustained; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us (including intellectual property lawsuits relating to ULTOMIRIS brought by third parties); the risk that third party payors (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all; failure to realize the benefits and potential of investments, collaborations, licenses and acquisitions; the possibility that expected tax benefits will not be realized; potential declines in sovereign credit ratings or sovereign defaults in countries where we sell our products; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; adverse impacts on our supply chain, clinical trials, manufacturing operations, financial results, liquidity, hospitals, pharmacies and health care systems from natural disasters and global pandemics, including the coronavirus; uncertainties surrounding legal proceedings, company investigations and government investigations; the risk that estimates regarding the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D and other indications we are pursuing are inaccurate; the impact of the proposed transaction between Alexion and AstraZeneca plc; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructurings; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the quarter ended March 31, 2021 and in our other filings with the SEC. Alexion disclaims any obligation to update any of these forward-looking statements to reflect events or circumstances after the date hereof, except when a duty arises under law.

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