ALXN Alexion Pharmaceuticals Inc.

120
-0.76  -1%
Previous Close 120.76
Open 120.44
52 Week Low 72.67
52 Week High 128.57
Market Cap $26,300,757,960
Shares 219,172,983
Float 210,376,414
Enterprise Value $26,409,626,462
Volume 909,464
Av. Daily Volume 2,257,813
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Upcoming Catalysts

Drug Stage Catalyst Date
ALXN1840 (WTX101)
Wilson disease
Phase 3
Phase 3
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ALXN2050
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2
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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
ALXN1830
Warm autoimmune hemolytic anemia (WAIHA)
Phase 1/2
Phase 1/2
Phase 2/3 trial to be initiated in 2021.
Cerdulatinib (PRT2070)
Refractory non-Hodgkin lymphoma and chronic lymphocytic leukemia - cancer
Phase 2
Phase 2
Phase 2a data presented at ASH December 9, 2019. ORR 48%; 76% in combo cohort.
CAEL‐101
AL amyloidosis
Phase 3
Phase 3
Phase 3 initiation announced September 14, 2020.
AG10 / BBP-265
ATTR-PN
Phase 2
Phase 2
Phase 3 trial planned for 2H 2020.
Elamipretide
Leber’s hereditary optic neuropathy (LHON)
Phase 2
Phase 2
Phase 2 trial did not meet primary endpoint.
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.
ALXN2040
C3 Glomerulopathy (C3G)
Phase 2
Phase 2
Development discontinued - noted July 29, 2020.
ULTOMIRIS
Neuromyelitis Optica Spectrum Disorder (NMOSD)
Phase 3
Phase 3
Phase 3 trial underway.
ULTOMIRIS (ravulizumab-cwvz)
Generalized myasthenia gravis (gMG)
Phase 3
Phase 3
Phase 3 trial ongoing.
ULTOMIRIS
Hematopoietic stem cell transplant-related thrombotic microangiopathy (HSCT-TMA)
Phase 3
Phase 3
Limited dose-ranging studies to commence 2H 2020, followed by Phase 3 trials in 2021.
ULTOMIRIS (ravulizumab-cwvz)
COVID-19 Coronavirus
Phase 3
Phase 3
Phase 3 open-label, randomized, controlled trial is underway - noted July 30, 2020.
Eculizumab
Neuromyelitis Optica Spectrum Disorder (NMOSD) - children
Phase 3
Phase 3
Phase 3 trial to commence 2H 2020.
Bevyxxa (betrixaban)
Venous thromboembolism (VTE) Prevention
Approved
Approved
FDA Approval noted June 23, 2017.
Andexxa
Factor Xa inhibitor reversal agent
Approved
Approved
Prior Approval Supplement (PAS) FDA Approval announced December 31, 2018.
ULTOMIRIS (ravulizumab) - CHAMPION-ALS
Amyotrophic lateral sclerosis (ALS)
Phase 3
Phase 3
Phase 3 trial initiated March 2020.
ULTOMIRIS (ravulizumab-cwvz)
atypical Hemolytic Uremic Syndrome (aHUS)
Approved
Approved
FDA Approval announced October 18, 2019.
Elamipretide
Primary mitochondrial myopathy
Phase 3
Phase 3
Phase 3 data did not meet primary endpoint - December 20, 2019.
Eculizumab
Generalized myasthenia gravis (gMG) - children
Phase 3
Phase 3
Phase 3 trial has commenced - noted October 23, 2019.
Eculizumab
Relapsing Neuromyelitis Optica Spectrum Disorder (NMOSD)
Approved
Approved
FDA Approval announced June 27, 2019.
ULTOMIRIS (ravulizumab-cwvz)
Paroxysmal nocturnal hemoglobinuria (PNH)
Approved
Approved
FDA approval announced December 21, 2018.
Eculizumab
Refractory generalized myasthenia gravis (gMG)
Approved
Approved
Approval announced October 23, 2017.

Latest News

  1. Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Company will report its financial results for the third quarter ended September 30, 2020 before the US financial markets open on October 29, 2020. Following the release of the financial results, Alexion management will conduct a conference call and audio webcast from 8:00-9:00 a.m. Eastern Time (ET).

    To participate in this conference call, dial (866) 762-3111 (USA) or (210) 874-7712 (International), conference ID 6582445 shortly before 8:00 a.m. ET. The audio webcast can be accessed on the Investor page of http://ir.alexion.com and an archived version will be available for a limited time following the presentation.

    [ALXN-E]

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Company will report its financial results for the third quarter ended September 30, 2020 before the US financial markets open on October 29, 2020. Following the release of the financial results, Alexion management will conduct a conference call and audio webcast from 8:00-9:00 a.m. Eastern Time (ET).

    To participate in this conference call, dial (866) 762-3111 (USA) or (210) 874-7712 (International), conference ID 6582445 shortly before 8:00 a.m. ET. The audio webcast can be accessed on the Investor page of http://ir.alexion.com and an archived version will be available for a limited time following the presentation.

    [ALXN-E]

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  2. – New 100 mg/mL formulation will reduce infusion time by approximately 60 percent, lessening the burden on patients –

    – ULTOMIRIS is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL formulation for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) and for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy for adult and pediatric (one month of age and older) patients. ULTOMIRIS 100 mg/mL is an advancement in the treatment experience for patients with…

    – New 100 mg/mL formulation will reduce infusion time by approximately 60 percent, lessening the burden on patients –

    – ULTOMIRIS is approved for the treatment of paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS) –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the U.S. Food and Drug Administration (FDA) has approved ULTOMIRIS® (ravulizumab-cwvz) 100 mg/mL formulation for the treatment of adults with paroxysmal nocturnal hemoglobinuria (PNH) and for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy for adult and pediatric (one month of age and older) patients. ULTOMIRIS 100 mg/mL is an advancement in the treatment experience for patients with aHUS and PNH, as it reduces average annual infusion times by approximately 60 percent compared to ULTOMIRIS 10 mg/mL while delivering comparable safety and efficacy. With ULTOMIRIS 100 mg/mL, most patients will spend six hours or less a year receiving treatment.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20201012005090/en/

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

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

    "We have seen the significant clinical benefits ULTOMIRIS has demonstrated for patients, through its complete C5 inhibition and sustained efficacy," said Jamile Shammo, M.D., FASCP, FACP, Professor of Medicine and Pathology, Department of Internal Medicine, Rush University Medical Center. "Based on the scientific evidence presented, the advanced formulation demonstrated comparable safety and efficacy to the original formulation, with the additional benefit of significantly shorter infusion times. This reduced treatment burden is important to consider for patients, as it has the potential to make a meaningful difference in their lives."

    PNH is a blood disorder characterized by complement-mediated destruction of the red blood cells that can cause a wide range of debilitating symptoms and complications, including thrombosis, which can occur throughout the body, and result in organ damage and premature death. Atypical HUS can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Affecting both adults and children, atypical HUS patients can present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of both aHUS and PNH can be poor in many cases, so a timely and accurate diagnosis—in addition to appropriate treatment—is critical to improving patient outcomes.

    "ULTOMIRIS is the market leader for the treatment of PNH and is emerging as the standard of care for the treatment of aHUS," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "The approval of ULTOMIRIS 100 mg/mL represents the next step in advancing patient care for the PNH and aHUS communities, as it reduces infusion times for patients and decreases the number of vials that need to be stored and prepared for the majority of patients' infusions, allowing healthcare providers more time to focus on the patient. With ULTOMIRIS 100 mg/mL, most patients will spend six hours or less each year receiving treatment, giving them more time to do what they enjoy, which we believe will provide a meaningful benefit to their quality of life."

    Alexion plans to make ULTOMIRIS 100 mg/mL available within a few days and has comprehensive training plans to educate all stakeholders about the new formulation. ULTOMIRIS 10 mg/mL will continue to be available until mid-2021, at which time Alexion will remove ULTOMIRIS 10 mg/mL from the market, as communicated to the FDA. The transition period is intended to provide a seamless conversion to the new formulation without interruption to patients' infusion schedules.

    Regulatory filings for marketing authorizations of the 100 mg/mL formulation of ULTOMIRIS are under review with regulators in the European Union (EU) and Japan. The Committee for Medicinal Products for Human Use (CHMP) has recently adopted a positive opinion, recommending marketing authorization of ULTOMIRIS 100 mg/mL in the European Union, and we anticipate receiving a decision from the European Commission in November.

    Alexion continues to innovate with ULTOMIRIS, with the goal of improving the patient experience. We plan to submit regulatory filings in the U.S. and EU in the third quarter of 2021 for an ULTOMIRIS subcutaneous formulation and device combination for PNH and aHUS that can be self-administered at home, pending completion of the ongoing Phase 3 study and collection of 12-month safety data. In addition, the collective ULTOMIRIS clinical development programs present an opportunity to expand the treated patient populations across hematology, nephrology, neurology and for the treatment of severe COVID-19, with seven Phase 3 programs that are ongoing or have planned clinical trial initiations in 2020.

    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. PNH can strike men and women of all races, backgrounds and ages without warning, with an average age of onset in the early 30s.

    About Atypical Hemolytic Uremic Syndrome (aHUS)

    aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. aHUS occurs when the complement system—a part of the body's immune system—over-responds, leading the body to attack its own healthy cells. aHUS can cause sudden organ failure or a slow loss of function over time—potentially resulting in the need for a transplant, and in some cases, death. aHUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosis—in addition to treatment—is critical to improving patient outcomes. Available tests can help distinguish aHUS from other hemolytic diseases with similar symptoms.

    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.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (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 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 with PNH.

    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, pain with the infusion, 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 infection and headache.

    The most common side effects of ULTOMIRIS in people with aHUS are upper respiratory 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 the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. 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]

    For patient or advocacy inquiries please contact .

    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, including statements related to: the safety, efficacy and benefits of the 100 mg/mL ULTOMIRIS formulation as a treatment for PNH and aHUS; the ability of the ULTOMIRIS 100 mg/mL formulation to reduce infusion time as compared to the 10mg/mL formulation of ULTOMIRIS by approximately 60% with comparable safety and efficacy; the potential for shorter infusion times will make a meaningful difference in patient lives; ULTOMIRIS is becoming the standard of care for patients with aHUS; the shorter infusion times for the 100mg/mL ULTOMIRIS formulation will result in patients spending six hours or less per year receiving treatment; the approval of ULTOMIRIS 100 mg/mL represents the next step in advancing patient care for the PNH and aHUS communities; ULTOMIRIS 100 mg/mL reduces infusion times for patients and decreases the number of vials that need to be stored and prepared for the majority of patients' infusions, allowing healthcare providers more time to focus on the patient; Alexion's plans to make ULTOMIRIS 100 mg/mL available to patients and its training plans to educate stakeholders about the new formulation; . Alexion's plans with respect to the availability of ULTOMIRIS 10 mg/mL formulation; plans for transitioning patients to the 100 mg/mL formulation; Alexion expects a marketing approval decision from the European Commission on the ULTOMIRIS 100 mg/mL formulation in November; our plans to submit regulatory filings in the U.S. and EU in the third quarter of 2021 for an ULTOMIRIS subcutaneous formulation and device combination for PNH and aHUS that can be self-administered at home; completion of ongoing Phase 3 study and collection of 12-month safety data; that the collective ULTOMIRIS clinical development programs present an opportunity to expand the treated patient populations across hematology, nephrology, neurology and for the treatment of severe COVID-19; and planned clinical trial initiations in 2020. 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: the anticipated safety profile and the benefits of the ULTOMIRIS 100 mg/mL formulation may not be realized (and the results of the clinical trials may not be indicative of future results); results of clinical trials may not be sufficient to satisfy regulatory authorities; results in clinical trials may not be indicative of results from later stage or larger clinical trials (or in broader patient populations); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (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 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 principal product (SOLIRIS); 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 failure of product candidates to obtain regulatory approval; 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 our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; inability to complete acquisitions or grow the product pipeline through acquisitions (including due to failure to obtain antitrust approvals); 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 COVID-19; 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 (as well as patients requiring a Factor Xa inhibitor reversal agent) are inaccurate; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; risks related to the acquisitions of Portola Pharmaceuticals, Achillion and other companies and co-development efforts; 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 period ended June 30, 2020 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. - Four abstracts accepted for presentation, including new clinical trial analyses that provide further support for the safety and efficacy of ULTOMIRIS® (ravulizumab-cwvz) for the treatment of pediatric patients with atypical hemolytic uremic syndrome (aHUS) -

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that four abstracts have been accepted for presentation at the American Society of Nephrology's Kidney Week 2020, taking place virtually from October 22 to 25, 2020. New data will be presented from Alexion's pivotal Phase 3, single-arm trial evaluating the efficacy and safety of ULTOMIRIS® (ravulizumab-cwvz) to resolve thrombotic microangiopathy (TMA) in pediatric patients with atypical hemolytic uremic syndrome (aHUS). The…

    - Four abstracts accepted for presentation, including new clinical trial analyses that provide further support for the safety and efficacy of ULTOMIRIS® (ravulizumab-cwvz) for the treatment of pediatric patients with atypical hemolytic uremic syndrome (aHUS) -

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that four abstracts have been accepted for presentation at the American Society of Nephrology's Kidney Week 2020, taking place virtually from October 22 to 25, 2020. New data will be presented from Alexion's pivotal Phase 3, single-arm trial evaluating the efficacy and safety of ULTOMIRIS® (ravulizumab-cwvz) to resolve thrombotic microangiopathy (TMA) in pediatric patients with atypical hemolytic uremic syndrome (aHUS). The data demonstrate that ULTOMIRIS improved hematologic and renal outcomes by 50 weeks in 94 percent of pediatric patients who had not previously received treatment with a complement inhibitor, without any unexpected safety concerns, when administered every four or eight weeks (depending on body weight). Additionally, new data will be presented that shows results from a switch study involving pediatric patients with aHUS from stable treatment with SOLIRIS® (eculizumab) to treatment either every four or eight weeks (depending on body weight) with ULTOMIRIS. The study demonstrated continued efficacy, presented no additional safety concerns and the benefit of reduced dosing frequency.

    Alexion also plans to present on the comparative efficacy of ULTOMIRIS and SOLIRIS in the treatment of adults with aHUS. Data from Phase 3 clinical trials show there are no significant differences in outcomes between patients treated with SOLIRIS verses those treated with ULTOMIRIS at 26 weeks, after balancing patient characteristics between the study groups.

    "ULTOMIRIS is quickly emerging as the new standard of care for patients living with atypical hemolytic uremic syndrome," said John Orloff, M.D., Executive Vice President and Head of Research and Development at Alexion. "The data we are presenting during the American Society of Nephrology's virtual meeting reinforce our commitment to advancing the understanding of the safety and efficacy of ULTOMIRIS among different patient populations—including pediatric patients—regardless of their previous treatment experience. We look forward to sharing these data at the upcoming Congress, which continues to provide the scientific community with valuable insights into ongoing research in nephrology, despite the meeting being held virtually this year."

    The accepted abstracts are listed below and are now available on the ASN website:

    Oral Presentations

    Characteristics and Outcomes of Pregnancy-Triggered Atypical Hemolytic-Uremic Syndrome: Global aHUS Registry Analysis. Abstract ID #SU-OR40 – oral presentation, October 25, 2020, 5:00 p.m. ET.

    ePoster Presentations

    Efficacy and Safety of Ravulizumab in Pediatric Patients with Atypical Hemolytic Uremic Syndrome Naïve to Complement Inhibitor Treatment: 26-week and 1-year Data. Abstract ID #PO2358 – poster presentation, October 22, 2020, 10:00 a.m. ET.

    Efficacy and Safety of Ravulizumab in Pediatric Patients with Atypical Hemolytic Uremic Syndrome Previously Treated with Eculizumab: 26–week and 1–year Data. Abstract ID #PO2331 – poster presentation, October 22, 2020, 10:00 a.m. ET.

    Comparative Efficacy of Ravulizumab and Eculizumab in the Treatment of Atypical Hemolytic Uremic Syndrome: An Indirect Comparison Using Clinical Trial Data. Abstract ID #PO1851 – poster presentation, October 22, 2020, 10:00 a.m. ET.

    About Atypical Hemolytic Uremic Syndrome (aHUS)

    Atypical HUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. Atypical HUS occurs when the complement system—a part of the body's immune system—over-responds, leading the body to attack its own healthy cells. Atypical HUS can cause sudden organ failure or a slow loss of function over time—potentially resulting in the need for a transplant, and in some cases, death. Atypical HUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosis—in addition to treatment—is critical to improving patient outcomes. Available tests can help distinguish aHUS from other hemolytic diseases with similar symptoms.

    About ULTOMIRIS® (ravulizumab‑cwvz)

    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 every four weeks for pediatric patients less than 20 kg, following a loading dose. ULTOMIRIS is approved in the United States (U.S.), European Union (EU) and Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (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.

    About 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. In many countries around the world, SOLIRIS is approved to treat paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), adults with generalized myasthenia gravis (gMG) who are acetylcholine receptor (AchR) antibody positive and/or adults with neuromyelitis optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated for the treatment of patients with Shiga-toxin E. coli-related hemolytic uremic syndrome (STEC-HUS). To learn more about the regulatory status of SOLIRIS 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 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 with PNH.

    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.

    ULTOMIRIS may also increase the risk of other types of serious infections. 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 reactions. Symptoms of an infusion reaction with ULTOMIRIS may include lower back pain, pain with the infusion, 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 infection and headache.

    The most common side effects of ULTOMIRIS in people with aHUS are upper respiratory 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.

    INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS® (eculizumab)

    INDICATIONS

    What is SOLIRIS?

    SOLIRIS is a prescription medicine used to treat:

    • patients with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH).
    • adults and children with a disease called atypical Hemolytic Uremic Syndrome (aHUS). SOLIRIS is not for use 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 SOLIRIS is safe and effective in children with PNH, gMG, or NMOSD.

    IMPORTANT SAFETY INFORMATION

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

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

    • SOLIRIS 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 SOLIRIS if you are not vaccinated.
    2. If your doctor decided that urgent treatment with SOLIRIS is needed, you should receive meningococcal vaccination as soon as possible.
    3. If you have not been vaccinated and SOLIRIS therapy must be initiated immediately, you should also receive two 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 3 months after your last SOLIRIS dose. It is important to show this card to any doctor or nurse to help them diagnose and treat you quickly.

    SOLIRIS is only available through a program called the SOLIRIS REMS. Before you can receive SOLIRIS, your doctor must enroll in the SOLIRIS 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 the 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.

    SOLIRIS may also increase the risk of other types of serious infections. Make sure your child receives vaccinations against Streptococcus pneumoniae and Haemophilus influenzae type b (Hib) if treated with SOLIRIS. Certain people may be at risk of serious infections with gonorrhea. Certain fungal infections (Aspergillus) may occur 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 meningococcal infection or have not been vaccinated against meningitis infection unless your doctor decides that urgent treatment with SOLIRIS is needed.

    Before you receive SOLIRIS, 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 SOLIRIS will harm your unborn baby or if it passes into your breast milk.

    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. It is important that you have all recommended vaccinations before you start SOLIRIS, receive 2 weeks of antibiotics if you immediately start SOLIRIS, and stay up-to-date with all recommended vaccinations during treatment with SOLIRIS.

    If you have PNH, your doctor 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 doctor will need to monitor you closely during and for at least 12 weeks after stopping treatment 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 allergic reactions. Tell your doctor 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 allergic reaction to SOLIRIS, your doctor may need to infuse SOLIRIS more slowly, or stop SOLIRIS.

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

    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 SOLIRIS. For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit MedWatch, or call 1-800-FDA-1088.

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

    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 the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. 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, including statements related to: the safety and efficacy of ULTOMIRIS for the treatment of pediatric patients with aHUS; the safety and efficacy of ULTOMIRIS to resolve TMA in pediatric patients with aHUS; ULTOMIRIS is quickly emerging as the new standard of care for patients living with atypical hemolytic uremic syndrome; and a timely and accurate diagnosis of aHUS — in addition to treatment — is critical to improving patient outcomes. 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: anticipated safety profile and the benefits of ULTOMIRIS to resolve TMA in pediatric patients with aHUS may not be realized; results of clinical trials may not be sufficient to satisfy regulatory authorities (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 after the product is approved for use by regulatory agencies); the severity of the impact of the COVID-19 pandemic on Alexion's business, including on commercial 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 principal product (SOLIRIS); 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 failure of product candidates to obtain regulatory approval; 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 our products and our product candidates; failure to satisfactorily address matters raised by regulatory agencies regarding our products and product candidates; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; inability to complete acquisitions or grow the product pipeline through acquisitions (including due to failure to obtain antitrust approvals); 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 or that tax liabilities exceed current expectations; 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 COVID-19; 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 risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; 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 period ended June 30, 2020 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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  4. - Robust pipeline of 20+ development programs across 7 rare disease franchises with future plans to continue growing pipeline with >5 novel INDs by 2025 -

    - Increasing depth and breadth of growth opportunities expected to generate 2025 global revenues of $9-10 billion & >10% revenue CAGR through 2025 and beyond -

    - Expect to raise 2020 full-year revenue guidance by >$200 million -

    - Robust share repurchase commitment expected to return ~$3 billion cash to shareholders through 2023 -

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced continued progression of the company's LEAD-EXPAND-DIVERSIFY value-creation strategy and will highlight strategic advancements at today's Virtual Investor Day. The day will be led by Alexion's Chief…

    - Robust pipeline of 20+ development programs across 7 rare disease franchises with future plans to continue growing pipeline with >5 novel INDs by 2025 -

    - Increasing depth and breadth of growth opportunities expected to generate 2025 global revenues of $9-10 billion & >10% revenue CAGR through 2025 and beyond -

    - Expect to raise 2020 full-year revenue guidance by >$200 million -

    - Robust share repurchase commitment expected to return ~$3 billion cash to shareholders through 2023 -

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced continued progression of the company's LEAD-EXPAND-DIVERSIFY value-creation strategy and will highlight strategic advancements at today's Virtual Investor Day. The day will be led by Alexion's Chief Executive Officer Ludwig Hantson, Ph.D., who will be joined by Board Chairman David Brennan and other members of the leadership team, in a series of presentation and Q&A sessions to provide further insight into the company's robust pipeline, future growth potential and continued progress advancing its mission of improving the lives of people with rare diseases and devastating conditions.

    The day will highlight select strategic programs to illustrate the significant promise of Alexion's portfolio and its value-creating potential, which includes:

    • Robust pipeline of more than 20 development programs across seven rare disease franchises, with expected continued growth from more than five novel investigational new drug applications (INDs) by 2025
    • Anticipated 2025 global revenue target of $9 to $10 billion, and at least 10 percent revenue compound annual growth rate (CAGR) through 2025 and beyond
    • Plan to raise 2020 full-year revenue guidance by more than $200 million when reporting third quarter results
    • Expect to return at least $3 billion to shareholders through multi-year stock buyback program

    "Approximately three years ago, we laid out an ambitious, multi-year strategy to dramatically transform Alexion, position us for the future and drive continued value creation. I am so proud of our tremendous progress advancing and successfully executing on that strategy across the entire organization," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "As a result, today, Alexion is a very different company than it was in 2017. Our base business is stronger than ever before. Even more importantly, we are in a new stage of company expansion and diversification that provides a path to long-term sustainable growth and allows us to reinvest in innovation for the future and return value to shareholders."

    At this year's Virtual Investor Day, Alexion will highlight key portfolio opportunities and drivers of future growth, including:

    Robust R&D Portfolio

    Alexion's pipeline now includes more than 20 development programs – up from four at the end of 2017 – with the potential for 10 promising launches by 2023. The company continues to expand into additional therapeutic areas as it builds seven rare disease franchises across hematology, nephrology, metabolics, neurology, cardiology, ophthalmology and acute care, which have the potential to deliver more than $10 billion in future peak sales. Despite the challenges posed by COVID-19, Alexion has made significant progress in advancing these programs in 2020.

    Sustainability in C5: Leading & Expanding

    LEAD: Alexion has already established ULTOMIRIS® (ravulizumab) as the market leader in paroxysmal nocturnal hemoglobinuria (PNH) and is working to make it the new standard of care across the C5 franchise with recent global atypical hemolytic uremic syndrome (aHUS) launches. The company is continuing to innovate to improve the patient experience with both high concentration ULTOMIRIS (100 mg/mL) and once weekly subcutaneous ULTOMIRIS.

    EXPAND: Alexion is continuing efforts to expand its C5 franchise into new therapeutic areas. This expansion began with a commitment to neurology, which, in just two years, grew into the company's largest franchise in the U.S., and is on track to quadruple the number of U.S. neurology patients treated by 2025. The company is also working to broaden the reach of ULTOMIRIS with new indications across a variety of therapeutic areas – including through ongoing Phase 3 studies in generalized myasthenia gravis (gMG), neuromyelitis optica spectrum disorder (NMOSD) and amyotrophic lateral sclerosis (ALS) – and is applying its expertise in complement biology with internal patient-driven innovation to develop a third-generation C5 inhibitor, ALXN1720, which was developed with the goal of enabling expansion into new larger rare diseases because of its low-volume subcutaneous administration.

    Select Diversification Opportunities in the Portfolio

    Alexion has made significant progress diversifying its portfolio beyond C5 over the last several years. Select promising programs from its pipeline – including individual programs and broader platform opportunities – will be highlighted at Investor Day, including:

    • ALXN1840 in Wilson disease: Alexion is developing a new biomarker to directly quantify the labile bound copper (LBC) in patients with Wilson disease. As the first direct measure of loosely bound, biologically active copper, this assay could provide a new tool for clinicians to assess copper in the body, helping them more readily diagnose and manage those with the disease. Enrollment is complete in a Phase 3 study of ALXN1840 in Wilson disease, and the data readout remains on track for the first half of 2021.
    • CAEL-101 in AL amyloidosis: CAEL-101 has the potential to be the first therapy to remove amyloid from tissues and improve organ function, with the aim of prolonging survival. In collaboration with Caelum Biosciences, the Phase 3 program is underway, with the goal of supporting a potential regulatory filing and launch by 2023. Alexion has the option to acquire Caelum based on the Phase 3 trial results.
    • Factor D platform (ALXN2040 & ALXN2050): Alexion's Factor D platform provides the opportunity to expand treatment for diseases that are currently in the company's portfolio, and to grow into new diseases and therapeutic areas.
      • ALXN2040: ALXN2040 has the potential to address clinically evident extravascular hemolysis (EVH) for the small portion of PNH patients on ULTOMIRIS or SOLIRIS® (eculizumab) (<10% of patients) that experience EVH. A Phase 3 study of ALXN2040 as an add-on therapy for these patients is on track to initiate in the fourth quarter of 2020, with topline results expected in the second half of 2022. In addition, Alexion is expanding the ALXN2040 development program with plans to begin a Phase 2 proof-of-concept study in geographic atrophy (GA) in 2021.
      • ALXN2050: ALXN2050 has the ability to further build the Factor D platform, first in PNH, where a Phase 2 monotherapy study is ongoing with topline results estimated in the second half of 2021, and secondly, in renal diseases, with a proof-of-concept trial in various renal diseases planned to begin in 2021.
    • Anti-FcRn platform (ALXN1830): Alexion's anti-FcRn platform has broad applicability across numerous rare IgG-mediated autoimmune diseases. Preliminary PK/PD modeling from the Phase 1 study suggests weekly subcutaneous (SC) injections of 1500mg may have the potential to provide greater than 70 percent IgG lowering. Development of ALXN1830 was paused due to COVID-19, but Alexion plans to reinitiate SC formulation development in early 2021 to complete the Phase 1 healthy volunteer study and to begin Phase 2 studies in warm autoimmune hemolytic anemia (WAIHA) and gMG.

    Internal Research & Discovery

    Supporting its near-term pipeline, Alexion is continuing to relentlessly pursue innovation and has an unwavering focus on research that will develop solutions to address patient needs. With the company's rare disease capabilities and expertise, in combination with its rebuilt pre-clinical pipeline and expanded areas of focus, Alexion believes it is on track to produce more than five novel INDs by 2025, including two this year.

    Capital Allocation Strategy

    Since 2017, Alexion has continued to consistently execute from a financial perspective across a number of key areas, driven by strong revenue growth, and expects to achieve a more than 16 percent revenue compound annual growth rate by the end of 2020. In addition, the company has continued to focus on financial discipline to achieve strong operating results and position it for the future. The company will provide updated 2020 financial guidance when it reports third quarter results and expects to raise full-year revenue guidance by more than $200 million. Because of the confidence in its commercial platform, multiple launches in the coming years and a renewed pipeline, Alexion recently announced a new capital allocation strategy that will return value to shareholders, including a commitment to dedicate at least one third of annual free cash flow to share repurchases from 2021 through 2023, for what is expected to be a total of at least $3 billion in stock buybacks covering the four years ending December 2023.

    Featured Speakers

    The virtual set up of Alexion's Investor Day provides the opportunity to hear from both Alexion executives and scientific leaders, including:

    • David Brennan, Chairman of the Board
    • Ludwig Hantson, Ph.D., CEO
    • Aradhana Sarin, M.D., CFO
    • John Orloff, M.D., Head of R&D
    • Brian Goff, Chief Commercial & Global Operations Officer
    • Cristina Quarta, M.D., Ph.D., CAEL-101 Clinical Development Lead
    • Gianluca Pirozzi, M.D., Ph.D., Head of Clinical Development & Translational Sciences
    • Anita Hill, M.D., Ph.D., Hematology Global Medical Affairs Lead
    • Darius Moshfeghi, M.D., Alexion consultant on Geographic Atrophy, Professor of Ophthalmology & Chief of the Retina Division, Stanford University School of Medicine
    • Sharon Barr, Ph.D., Head of Research, Bioinformatics & Diagnostics

    Investor Day Webcast Information

    Alexion will host an audio webcast today from 8:00 a.m. to 12:00 p.m. Eastern Time. The live audio webcast can be accessed here, or from the Investor page of Alexion's website at: http://ir.alexion.com. An archived version of the webcast will also be available through the company's website for a limited time following the event.

    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 the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. 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-G]

    Forward-Looking Statement

    This press release contains forward-looking statements, including statements related to: anticipated financial results (including short-term guidance and long-range financial guidance), including expected increases to the revenue guidance for 2020, our revenue cumulative average growth rate of at least 10% through 2025 and beyond, and peak revenue from our current pipeline beyond 2025 (and all of the assumptions, judgments and estimates related to such anticipated future results); ambition to quadruple the number of neurology patients in the US by 2025; ambition for 10 product launches by 2023; future plans to continue growing the company's pipeline with more than 5 novel INDs by 2025; plans for additional formulations of ULTOMIRIS (high concentration and subcutaneous) and the timing for regulatory approval and potential benefits of such formulations; anticipated future product launches (and the timing of those launches); that we are in a new stage of company expansion and diversification that provides a path to long-term sustainable growth and allows us to reinvest in innovation for the future and return value to shareholders; the company's capital allocation strategy and plans concerning the repurchase of Alexion shares; the anticipated amount and timing of future share repurchases by the company; plans to make regulatory filings for approval of certain products and product candidates, the expected timing of such filings as well as the expected timing of the receipt of certain regulatory approvals to market a product; the ability of our pipeline and existing products to provide long-term sustainable growth for shareholders; company's plans for future clinical trials and studies, the timing for the commencement and conclusion of future clinical trials and the expected timing of the receipt of results of clinical trials and studies; the company's strategy for long-term value creation; plans to further diversify our assets and establish novel platforms and the benefits of those plans; plans to establish 7 franchises and the targeted indications in each franchise; potential peak sales of our pipeline assets; potential launches of ULTOMIRIS for additional indications and in additional countries, including for ALS; plans and anticipated timing for the development of ALXN1840 in Wilson disease; the development of a new biomarker for Wilson disease to detect labile bound copper and the potential benefits of such biomarker; plans for the development and launch of CAEL-101 as a treatment for AL-Amyloidosis; plans for development and potential indications for ALXN1720; development and commercialization plans for ALXN2040 and ALXN2050, including in PNH, geographic atrophy and renal diseases, and the potential benefits of those therapies; development plans and the potential of ALXN1830; and continued diversification of the pipeline and products. Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those forward-looking statements, including for example: our dependence on sales from our C5 products (SOLIRIS and ULTOMIRIS); 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; Alexion's inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; payer, physician and patient acceptance of ULTOMIRIS as an alternative to SOLIRIS; appropriate pricing for ULTOMIRIS; future competition from biosimilars and novel products; 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); the number of patients that will use our products and product candidates in the future; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; 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 our products and our product candidates; failure to satisfactorily address matters raised by the FDA and other regulatory agencies; results in early stage clinical trials may not be indicative of full results or results from later stage or larger clinical trials (or broader patient populations) and do not ensure regulatory approval; the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to halt trials, delay or prevent us from making regulatory approval filings or result in denial of regulatory approval of our product candidates; unexpected delays in clinical trials; unexpected concerns that may arise from additional data or analysis obtained during clinical trials; future product improvements may not be realized due to expense or feasibility or other factors; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; inability to complete acquisitions due to failure of regulatory approval or material changes in target or otherwise; inability to complete acquisitions and investments due to increased competition for technology; the possibility that current rates of adoption of our products are not sustained (or anticipated adoption rates are not realized); internal development efforts do not result in commercialization of additional products; 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 products brought by third parties against Alexion); 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; failure by regulatory authorities to approve transactions; the possibility that expected tax benefits will not be realized or that tax liabilities exceed current expectations; assessment of impact of recent accounting pronouncements; 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; 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 future indications we are pursuing are inaccurate; the risks of changing foreign exchange rates; risks relating to the potential effects of the company's restructuring; risks related to the acquisition of companies and co-development and collaboration efforts; 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 period ended June 30, 2020 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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  5. – ULTOMIRIS is the first and only long-acting C5 inhibitor for aHUS, reducing the treatment burden for adults and children with administration every other month –

    – ULTOMIRIS has the potential to become the new standard of care in Japan for the treatment of aHUS, an ultra-rare disease which may progressively damage the kidney and other organs –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) approved ULTOMIRIS® (ravulizumab) for adults and children living with atypical hemolytic uremic syndrome (aHUS). ULTOMIRIS is the first and only long-acting C5 inhibitor for aHUS and is administered every other month for adults and children (20 kg or more) and monthly for children…

    – ULTOMIRIS is the first and only long-acting C5 inhibitor for aHUS, reducing the treatment burden for adults and children with administration every other month –

    – ULTOMIRIS has the potential to become the new standard of care in Japan for the treatment of aHUS, an ultra-rare disease which may progressively damage the kidney and other organs –

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that Japan's Ministry of Health, Labour and Welfare (MHLW) approved ULTOMIRIS® (ravulizumab) for adults and children living with atypical hemolytic uremic syndrome (aHUS). ULTOMIRIS is the first and only long-acting C5 inhibitor for aHUS and is administered every other month for adults and children (20 kg or more) and monthly for children (<20 kg). Atypical HUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots.

    "The goal of aHUS treatment is to prevent the body from attacking its own immune system through the inhibition of uncontrolled C5 complement activation," said Prof. Shoichi Maruyama, Director, Department of Nephrology, Nagoya University Hospital. "Importantly, ULTOMIRIS demonstrated good control, while also offering more time between infusions, which provides a relevant difference to patients and providers."

    Atypical HUS affects both adults and children and many patients present in critical condition in the hospital setting, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, with 56 percent of adults and 29 percent of children developing end-stage renal disease or dying within a year of diagnosis with supportive care alone, so a timely and accurate diagnosis in addition to treatment, is critical to improving patient outcomes.

    "Today's approval marks another important step in our efforts to continue innovating for patients and improving their treatment experience," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "ULTOMIRIS' extended dosing interval offers patients more flexibility and time to focus on living their lives beyond their disease while also reducing the burden on healthcare systems, hospitals and providers, which are all under a tremendous amount of stress in the current environment."

    The approval is based on data from two ongoing, global, single-arm open-label studies of ULTOMIRIS – one in adults and one in children, referred to as pediatrics in the study. A total of 18 out of 21 complement inhibitor treatment-naïve children and 56 out of 58 complement inhibitor treatment-naïve adults were enrolled and included in the interim analysis. Efficacy evaluation of Complete TMA Response was defined by normalization of hematologic parameters (platelet count and LDH) and improved kidney function (as measured by ≥25 percent improvement in serum creatinine from baseline). In the initial 26-week treatment periods, 54 percent of adults and 77.8 percent (interim data) of children demonstrated Complete TMA Response. Treatment with ULTOMIRIS resulted in normalization of platelet count in 84 percent of adults and 94 percent of children, normalization of LDH (marker of hemolysis) in 77 percent of adults and 90 percent of children, and improved kidney function in 59 percent of adults and 83 percent (interim data) of children (for patients on dialysis at enrollment, baseline was established after they had come off dialysis). In the 52-week follow-up period, 4 additional adult patients and 3 pediatric patients had a Complete TMA Response that was confirmed after the 26-week Initial Evaluation Period resulting in an overall Complete TMA Response of 61 percent in adults and 94 percent in children (interim data). A second cohort of 10 pediatric patients who were SOLIRIS-experienced were included in the pediatric study, demonstrating that switching to ULTOMIRIS maintained disease control as evidenced by stable hematologic and renal parameters, with no apparent impact on safety.

    The most frequently observed adverse reactions reported in these studies were upper respiratory tract infection, diarrhea, nausea, fatigue, headache, nasopharyngitis, and pyrexia. Serious meningococcal infections have occurred in patients treated with ULTOMIRIS, however in aHUS studies, no meningococcal infections occurred in the 89 patients receiving treatment with ULTOMIRIS. To minimize the risk for patients, specific risk-mitigation plans, including a Risk Management Plan, have been established for ULTOMIRIS.

    ULTOMIRIS will be available at approval for the treatment of aHUS.

    About Atypical Hemolytic Uremic Syndrome (aHUS)

    aHUS is an ultra-rare disease that can cause progressive injury to vital organs, primarily the kidneys, via damage to the walls of blood vessels and blood clots. aHUS occurs when the complement system—a part of the body's immune system—over-responds, leading the body to attack its own healthy cells. aHUS can cause sudden organ failure or a slow loss of function over time—potentially resulting in the need for a transplant, and in some cases, death. aHUS affects both adults and children, and many patients present in critical condition, often requiring supportive care, including dialysis, in an intensive care unit. The prognosis of aHUS can be poor in many cases, so a timely and accurate diagnosis—in addition to treatment—is critical to improving patient outcomes. Available tests can help distinguish aHUS from other hemolytic diseases with similar symptoms.

    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 every four weeks for pediatric patients less than 20 kg, following a loading dose. ULTOMIRIS is approved in Japan as a treatment for adults with paroxysmal nocturnal hemoglobinuria (PNH). To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit www.alexion.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 the core therapeutic areas of hematology, nephrology, neurology, metabolic disorders and cardiology. 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]

    For patient or advocacy inquiries please contact .

    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, including statements related to: the impact to Alexion of ULTOMIRIS approval in Japan for adults and children living with atypical hemolytic uremic syndrome (aHUS); that ULTOMIRIS provides effective disease control and together with its dosing, makes a difference to patients; that ULTOMIRIS has the potential to become the new standard of care in Japan for the treatment of aHUS; timely and accurate diagnosis of aHUS patients– in addition to treatment – is critical to improving patient outcomes; the approval of ULTOMIRIS in Japan for aHUS marks another important step in our efforts to continue innovating for patients and improving their treatment experience; and ULTOMIRIS' extended eight-week dosing interval offers patients more flexibility and time to focus on living their lives beyond their disease while also reducing the burden on healthcare systems, hospitals and providers. 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: the anticipated benefits of ULTOMIRIS for aHUS patients may not be realized (and the results of the clinical trials may not be indicative of the results in Japan); results of clinical trials may not be sufficient to satisfy any other regulatory authority in order to approve ULTOMIRIS as a treatment for aHUS (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 our products (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); 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 principal product (Soliris); 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 failure of product candidates to obtain regulatory approval; 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 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; inability to complete acquisitions or grow the product pipeline through acquisitions (including due to failure to obtain antitrust approvals); 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; 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 risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; risks related to the acquisition of Achillion, Portola and other companies and co-development efforts; 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 period ended June 30, 2020 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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