ALXN Alexion Pharmaceuticals Inc.

102.67
+1.43  (+1%)
Previous Close 101.24
Open 100.79
52 Week Low 72.67
52 Week High 125.52
Market Cap $22,502,490,165
Shares 219,172,983
Float 210,376,414
Enterprise Value $22,085,343,508
Volume 817,008
Av. Daily Volume 1,823,881
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Upcoming Catalysts

Drug Stage Catalyst Date
ALXN1840 (WTX101)
Wilson disease
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
CAEL‐101
AL amyloidosis
Phase 2/3
Phase 2/3
Phase 3 trial dosing to commence 3Q 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.
ALXN1830
Warm autoimmune hemolytic anemia (WAIHA)
Phase 1/2
Phase 1/2
Phase 2/3 trial to be initiated in 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.
ALXN2040 (Danicopan/ACH-4471)
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 3
Phase 3
Phase 3 trial to be initiated by the end of 2020.
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.
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.
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.
ALXN2050 (ACH-5228)
Paroxysmal nocturnal hemoglobinuria (PNH)
Phase 2
Phase 2
Phase 2 trial is underway - noted May 5, 2020.
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 the appointment of Uzair Qadeer as the company's first Chief Diversity Officer. In this new role, Mr. Qadeer will be responsible for shaping the company's diversity, inclusion and belonging (DI&B) strategy, building relationships with key leaders, communities, and organizations to create awareness and advocacy for diversity and inclusion efforts, and advancing a unique culture of belonging for Alexion's patients and employees. The appointment of Mr. Qadeer will also accelerate Alexion's efforts to embed DI&B across the company, spanning employee programs, corporate social responsibility initiatives, culture efforts, and patient programs. Mr. Qadeer will serve on Alexion's Executive…

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the appointment of Uzair Qadeer as the company's first Chief Diversity Officer. In this new role, Mr. Qadeer will be responsible for shaping the company's diversity, inclusion and belonging (DI&B) strategy, building relationships with key leaders, communities, and organizations to create awareness and advocacy for diversity and inclusion efforts, and advancing a unique culture of belonging for Alexion's patients and employees. The appointment of Mr. Qadeer will also accelerate Alexion's efforts to embed DI&B across the company, spanning employee programs, corporate social responsibility initiatives, culture efforts, and patient programs. Mr. Qadeer will serve on Alexion's Executive Committee, reporting to Ludwig Hantson, Ph.D., Chief Executive Officer at Alexion.

    This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20200806005090/en/

    Uzair Qadeer (Photo: Business Wire)

    Uzair Qadeer (Photo: Business Wire)

    "Establishing the Chief Diversity Officer role at Alexion is an important next step in our continued efforts to cultivate diversity, inclusion and a unique sense of belonging at the company, all of which enhances our ability to deliver on our mission of transforming the lives of patients with rare diseases and devastating conditions," said Dr. Hantson. "Uzair has been instrumental in helping to shape Alexion's approach to diversity and inclusion to date, and I am confident that, in this new role, he will help us build a stronger, even more inclusive organization, while ensuring every voice within the company is heard."

    Prior to assuming the role of Chief Diversity Officer, Mr. Qadeer served as Vice President, Head of Enterprise Partnership at Alexion, helping the company build leading-edge vision and capabilities in executive coaching, HR business partnership, and strategic workforce planning, while shaping the company's initial DI&B strategy. Before joining Alexion in February 2019, Mr. Qadeer worked in roles of increasing responsibility in Deloitte's Human Capital consulting practice where he advised clients across industries and geographies on forward-looking human capital topics spanning talent management, organizational design and development, human resources management, and diversity, equity and inclusion. Prior to that, Mr. Qadeer worked at Bristol Myers Squibb Company, expatriating to Rome, Italy and gaining deep insights in global and international work. Additionally, Mr. Qadeer also taught at Rutgers, The State University of New Jersey. Mr. Qadeer received his master's degree as well as his B.A. and B.S. degrees from Pennsylvania State University.

    "At Alexion, we believe that diversity is having a seat at the table, inclusion is having a voice, and belonging is having that voice be heard. Our work isn't done until our employees and patients can feel a true sense of belonging," said Mr. Qadeer. "Magnetizing and incubating diverse talent will allow us to harness diverse insights that fuel innovation and create value for the patients we serve. I am committed to activating this purposeful vision."

    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: the Company's planned implementation of the diversity, inclusion and belonging (DI&B) strategy; the expected benefits of the DI&B strategy and its implementation; the acceleration of Alexion's efforts to embed DI&B across the organizational ecosystem, spanning employee programs, corporate social responsibility initiatives, culture efforts, and patient programs; the Company will continue efforts to cultivate diversity, inclusion and a unique sense of belonging at the Company, all of which enhances our ability to deliver on our mission of transforming the lives of patients with rare diseases and devastating conditions; and the Chief Diversity Officer will help build a stronger, even more inclusive organization. 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: the Company may not be able to implement its planned DI&B initiatives, the Company may not realize the benefits from the DI&B initiatives contemplated by the Company; the Company may not be able to implement some or all of its DI&B plans; our dependence on sales from our principal product (SOLIRIS); our ability to facilitate the timely conversion from SOLIRIS to ULTOMIRIS; payer, physician and patient acceptance of ULTOMIRIS as an alternative to SOLIRIS; the impact of the COVID-19 pandemic on Alexion's business, including its sales, clinical trials, operations, DI&B and HR initiatives, and supply chain; 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 reasons; results in early stage clinical trials may not be indicative of full results or results from later stage or larger clinical trials (or in broader patient populations) and do not ensure regulatory approval; 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 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; 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 indications we are pursuing are inaccurate; 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.

    View Full Article Hide Full Article
    • 2Q20 total revenues of $1,444.6 million, a 20% increase over 2Q19
    • 2Q20 GAAP diluted EPS of $(4.84); non-GAAP diluted EPS of $3.11
    • Achieved goal of establishing ULTOMIRIS® (ravulizumab-cwvz) as new standard of care in PNH with more than 70% patient conversion from SOLIRIS® (eculizumab) in U.S.
    • Diversified commercial-stage portfolio with acquisition of Portola and addition of ANDEXXA® [coagulation factor Xa (recombinant), inactivated-zhzo]
    • Received EU approval for ULTOMIRIS in atypical hemolytic uremic syndrome (aHUS) & announced positive Phase 3 data for weekly subcutaneous ULTOMIRIS formulation
    • Updated capital allocation strategy with commitment to return $500-$550 million in 2020 & at least 1/3 average annual free cash flow to shareholders…
    • 2Q20 total revenues of $1,444.6 million, a 20% increase over 2Q19
    • 2Q20 GAAP diluted EPS of $(4.84); non-GAAP diluted EPS of $3.11
    • Achieved goal of establishing ULTOMIRIS® (ravulizumab-cwvz) as new standard of care in PNH with more than 70% patient conversion from SOLIRIS® (eculizumab) in U.S.
    • Diversified commercial-stage portfolio with acquisition of Portola and addition of ANDEXXA® [coagulation factor Xa (recombinant), inactivated-zhzo]
    • Received EU approval for ULTOMIRIS in atypical hemolytic uremic syndrome (aHUS) & announced positive Phase 3 data for weekly subcutaneous ULTOMIRIS formulation
    • Updated capital allocation strategy with commitment to return $500-$550 million in 2020 & at least 1/3 average annual free cash flow to shareholders from 2021-2023
    • Increased revenue and non-GAAP EPS guidance to reflect momentum of the business; GAAP EPS guidance negatively impacted by impairment charges

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced financial results for the second quarter of 2020. Total revenues in the second quarter were $1,444.6 million, a 20 percent increase compared to the same period in 2019. The negative impact of foreign currency on total revenues year-over-year was 1 percent, or $15.9 million, inclusive of hedging activities. On a GAAP basis, diluted EPS in the quarter was $(4.84), compared to $2.04 in the prior year. The second quarter of 2020 includes impairment charges of $2,053.3 million primarily relating to the KANUMA intangible asset as a result of the Company's revised strategic view of KANUMA. Non-GAAP diluted EPS for the second quarter of 2020 was $3.11, an 18 percent increase versus the second quarter of 2019.

    "Our teams have demonstrated remarkable resilience and agility in their successful navigation of the uncertain COVID-19 pandemic environment. Despite these challenges, we have delivered another strong quarter and continue to advance our LEAD-EXPAND-DIVERSIFY strategy for long-term value creation," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "As a result of execution and delivery against our objectives, we have entered a new phase of company growth and diversification, which enables us to adjust our capital allocation priorities and return value to shareholders through an expanded stock buyback program. I am incredibly proud of what we have accomplished so far and am confident that we are well positioned to build on this momentum in the second half of the year."

    Second Quarter 2020 Financial Highlights

    • Net product sales were $1,444.5 million in the second quarter of 2020, compared to $1,202.5 million in the second quarter of 2019.
    • SOLIRIS net product sales were $975.5 million, compared to $980.8 million in the second quarter of 2019.
    • ULTOMIRIS net product sales were $251.1 million, compared to $54.2 million in the second quarter of 2019, representing a 363 percent increase.
    • STRENSIQ net product sales were $184.3 million, compared to $141.3 million in the second quarter of 2019, representing a 30 percent increase.
    • KANUMA net product sales were $33.6 million, compared to $26.2 million in the second quarter of 2019, representing a 28 percent increase.
    • GAAP cost of sales was $144.9 million, compared to $99.2 million in the second quarter of 2019. Non-GAAP cost of sales was $141.8 million, compared to $95.7 million in the second quarter of 2019.
    • GAAP R&D expense was $221.1 million, compared to $187.6 million in the second quarter of 2019. Non-GAAP R&D expense was $204.6 million, compared to $148.7 million in the second quarter of 2019.
    • GAAP SG&A expense was $301.4 million, compared to $299.3 million in the second quarter of 2019. Non-GAAP SG&A expense was $253.6 million, compared to $255.8 million in the second quarter of 2019.
    • GAAP impairment of intangible assets was $2,053.3 million primarily related to an impairment charge recorded during the second quarter 2020 related to the KANUMA intangible asset.
    • GAAP income tax benefit was $284.0 million, compared to income tax expense of $39.7 million in the second quarter of 2019. GAAP income tax benefit for the second quarter 2020 includes a deferred tax benefit of $377.3 million associated with the impairment charge related to the KANUMA intangible asset. Non-GAAP income tax expense was $125.5 million, compared to $90.2 million in the second quarter of 2019.
    • GAAP diluted EPS was $(4.84), inclusive of impairment charges of $2,053.3 million primarily relating to the KANUMA intangible asset, compared to $2.04 in the second quarter of 2019. Non-GAAP diluted EPS was $3.11, compared to $2.64 in the second quarter of 2019.

    COVID-19

    We continue to take steps to proactively respond to the evolving COVID-19 pandemic and to plan for related uncertainties. We remain focused on continuing to serve patients, protecting the health and safety of our employees and the communities in which we live and work, and supporting patients in clinical trials. We are also focused on minimizing potential interactions that could contribute to the spread of the virus and put additional strain on healthcare systems through the use of innovative virtual means where possible.

    • Clinical Trials: We have implemented a pandemic response business continuity plan designed to protect patients and site staff safety while continuing our clinical trials with limited interruption to the extent we are able. While the COVID-19 impact varies by study and program, so far, we have seen little timing impact on fully-enrolled trials and a timing shift of at least three months on trials that are enrolling patients and activating sites, or have not yet started to do so. We are working to re-initiate healthy volunteer studies that had been temporarily paused, pending local dynamics where these studies are being conducted.
    • Business Impact: We continue to take proactive measures designed to mitigate the risk of potential interruptions in supply and/or access to patients' customary site-of-care locations. As anticipated, we have seen accelerated conversion from SOLIRIS to ULTOMIRIS. Treatment compliance rates across all our medicines have remained strong and continue to be consistent with pre-pandemic compliance rates. We have also seen the predicted slowing of new patient initiations and delays in treatment starts, and we are continuing to closely monitor this environment as the pandemic continues.

    Research and Development

    PHASE 3/4

    • SOLIRIS - Neuromyelitis Optica Spectrum Disorder (NMOSD): Alexion plans to initiate a Phase 2/3 study in children and adolescents with NMOSD in the second half of 2020.
    • SOLIRIS - Generalized Myasthenia Gravis (gMG): A Phase 3 study of SOLIRIS in children and adolescents with gMG is underway.
    • SOLIRIS - Guillain-Barre syndrome (GBS): In June 2020, Japan's Ministry of Health, Labour and Welfare granted SAKIGAKE designation for SOLIRIS in GBS. Alexion plans to initiate a Phase 3 study of SOLIRIS in GBS in Japan in 2021, pending regulatory feedback.
    • ULTOMIRIS - Severe COVID-19: A Phase 3 randomized controlled trial of ULTOMIRIS in adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome is underway.
    • ULTOMIRIS - Paroxysmal Nocturnal Hemoglobinuria (PNH): A Phase 3 study of ULTOMIRIS in children and adolescents with PNH is underway.
    • ULTOMIRIS - Atypical Hemolytic Uremic Syndrome (aHUS): In June 2020, the European Commission approved ULTOMIRIS for adults and children with aHUS. An application for approval of ULTOMIRIS for aHUS is also under review in Japan. A Phase 3 study of ULTOMIRIS in children and adolescents with aHUS is underway.
    • ULTOMIRIS - 100mg/mL: Applications for approval of ULTOMIRIS 100mg/mL formulation are under review in the EU and U.S. The FDA has set a Prescription Drug User Fee Act target action date of October 11, 2020. This higher concentration formulation is designed to reduce infusion time by more than 50 percent to approximately 45 minutes. Alexion plans to file for regulatory approval of this formulation in Japan in the third quarter of 2020.
    • ULTOMIRIS - Subcutaneous: In June 2020, Alexion announced that the Phase 3 study of weekly subcutaneous (SC) ULTOMIRIS demonstrated PK-based non-inferiority versus intravenous ULTOMIRIS. Pending collection of 12-month safety and drug-device combination data, Alexion plans to file for approval in the U.S. and EU for the ULTOMIRIS SC formulation and device combination in PNH and aHUS in the third quarter of 2021.
    • ULTOMIRIS - gMG: A Phase 3 study of ULTOMIRIS in adults with gMG is underway.
    • ULTOMIRIS - NMOSD: A Phase 3 study of ULTOMIRIS in NMOSD is underway.
    • ULTOMIRIS - Amyotrophic Lateral Sclerosis (ALS): A Phase 3 study of ULTOMIRIS in ALS is underway.
    • ULTOMIRIS - Hematopoietic Stem Cell Transplant-Associated Thrombotic Microangiopathy (HSCT-TMA): Alexion plans to initiate limited dose-ranging studies of ULTOMIRIS in HSCT-TMA in the second half of 2020, followed by Phase 3 trials in 2021, pending regulatory feedback.
    • ULTOMIRIS - Complement Mediated Thrombotic Microangiopathy (CM-TMA): Alexion plans to initiate a Phase 3 study of ULTOMIRIS in CM-TMA in the first half of 2021, pending regulatory feedback.
    • ALXN1840 (WTX101) - Wilson Disease: Enrollment is complete in a Phase 3 study of ALXN1840 in Wilson disease. Study results are expected in the first half of 2021.
    • CAEL-101 - Caelum Biosciences: Alexion is collaborating with Caelum Biosciences to develop CAEL-101, which has the potential to be a first-in-class therapy targeting amyloid deposits in patients with light chain (AL) amyloidosis. A pivotal Phase 2/3 program is underway to investigate CAEL-101 as an add-on to current standard-of-care therapy. Dosing is complete in the Phase 2 dose selection portion of the program; the Phase 3 portion of the program is planned to begin in the third quarter of 2020, pending dose selection.
    • AG10 - Eidos: Alexion holds an exclusive license to develop and commercialize AG10 in Japan. Eidos is currently evaluating AG10 in a Phase 3 study in the U.S. and Europe for ATTR cardiomyopathy (ATTR-CM) and plans to begin a Phase 3 study in ATTR polyneuropathy (ATTR-PN) in the second half of 2020. Alexion plans to expand the AG10 program into Japan in 2020, pending regulatory feedback.
    • ALXN2040 (Danicopan/ACH-4471) - PNH with Extravascular Hemolysis (EVH): Alexion plans to initiate a Phase 3 study of ALXN2040 as an add-on therapy for PNH patients with EVH by the end of 2020.
    • ANDEXXA - Acute Intracranial Hemorrhage (ICH): In July 2020, Alexion announced the completion of its acquisition of Portola. The acquisition added ANDEXXA to the company's commercial and development portfolios. ANDEXXA has conditional approval in the U.S. and EU (marketed as ONDEXXYA in the EU) for the reversal of anticoagulation in patients experiencing life-threatening or uncontrolled bleeding who are treated with rivaroxaban or apixaban. The Phase 4 ANNEXA-I study - designed to provide clinical data supporting full approval - is underway to assess ANDEXXA compared to usual standard of care in patients presenting with acute intracranial hemorrhage while taking an oral Factor Xa inhibitor.

    PHASE 1/2

    • ULTOMIRIS - Renal Diseases: Alexion plans to initiate a proof-of-concept trial of ULTOMIRIS in patients with various renal diseases in 2020.
    • ALXN1830 (SYNT001): Due to COVID-19, Alexion discontinued the Phase 2 study of ALXN1830, administered intravenously, in warm autoimmune hemolytic anemia (WAIHA) and paused the Phase 1 study of a subcutaneous formulation of ALXN1830 in healthy volunteers. The paused Phase 1 study and new Phase 2 studies of subcutaneous ALXN1830 in gMG and WAIHA are planned to begin in 2021.
    • ALXN2040 - C3 Glomerulopathy (C3G): Interim data from two Phase 2 studies of ALXN2040 in C3G suggest that inhibition of Factor D is a promising potential target for treating the cause of C3G. However, the study showed that the clinical response with ALXN2040 was suboptimal, due to insufficient PK/PD response and incomplete inhibition of the alternative pathway. As a result, development of ALXN2040 in C3G will be discontinued, but potential future development options with ALXN2050, a more potent Factor D inhibitor, are being assessed.
    • ALXN2050 (ACH-5228) - PNH: A Phase 2 study of ALXN2050 monotherapy in PNH is underway.
    • ALXN2050 - Renal Diseases: Alexion plans to initiate a proof-of-concept trial of ALXN2050 in patients with various renal diseases in 2021.
    • ALXN1720: Seven of nine cohorts are complete in a Phase 1 healthy volunteer study of ALXN1720, a novel anti-C5 albumin-binding bi-specific mini-body that binds and prevents activation of human C5. Due to COVID-19, the study was temporarily paused but is planned to restart in the third quarter of this year.
    • ANDEXXA - Urgent Surgery: ANDEXXA is currently being evaluated in a single-arm, open-label study in patients taking apixaban, rivaroxaban, edoxaban, or enoxaparin who require urgent surgery. The results of this study will inform the design of a randomized controlled clinical trial to expand the label in this population.
    • Cerdulatinib: Acquired as part of the Portola acquisition, cedulatinib is a dual spleen tyrosine kinase and janus kinase (SYK/JAK) inhibitor being evaluated in a Phase 1/2a study in patients with relapsed/refractory chronic lymphocytic leukemia or B-cell or T-cell non-Hodgkin lymphoma.

    2020 Financial Guidance

    Alexion is increasing total revenues and non-GAAP EPS guidance and decreasing operating margin guidance. GAAP EPS guidance is negatively impacted by the impairment charges recorded during the second quarter 2020. Full guidance updates are outlined below.

     

    Previous

    Updated

    Total revenues

    $5,230 to $5,330 million

    $5,550 to $5,600 million

    SOLIRIS/ULTOMIRIS revenues

    $4,495 to $4,570 million

    $4,725 to $4,755 million

    Metabolic revenues

    $735 to $760 million

    $785 to $800 million

    ANDEXXA/ONDEXXYA revenues

    $40 to $45 million

    R&D (% total revenues)

     

     

    GAAP

    17.5% to 18.6%

    18.1% to 19.2%

    Non-GAAP

    16.0% to 17.0%

    16.5% to 17.5%

    SG&A (% total revenues)

     

     

    GAAP

    22.2% to 23.5%

    24.5% to 25.7%

    Non-GAAP

    18.5% to 19.5%

    21.0% to 22.0%

    Operating margin

     

     

    GAAP

    42.4% to 43.8%

    3.8% to 5.4%

    Non-GAAP

    55.0% to 56.0%

    53.0% to 54.0%

    Earnings per share

     

     

    GAAP

    $8.14 to $8.47

    $0.96 to $1.30

    Non-GAAP

    $10.45 to $10.75

    $10.65 to $10.95

    Updated 2020 financial guidance assumes a GAAP effective tax rate of (27.0) to (26.0) percent and a non-GAAP effective tax rate of 15.5 to 16.5 percent. The 2020 GAAP and non-GAAP tax rates do not benefit from one-time events that benefited the tax rates in 2019.

    Updated 2020 financial guidance includes the impact of the July 2, 2020 acquisition of Portola but excludes the impact of certain GAAP-only purchase accounting items related to the Portola acquisition, including amortization of purchased intangible assets, fair value adjustment of inventory acquired and the related tax effects.

    Alexion's financial guidance is based on current foreign exchange rates net of hedging activities and does not include the effect of acquisitions, license and other strategic agreements, intangible asset impairments, litigation charges, changes in fair value of contingent consideration, gains or losses related to strategic equity investments or restructuring and related activity outside of the previously announced activities that may occur after the issuance of this press release.

    Conference Call/Webcast Information:

    Alexion will host a conference call/audio webcast to discuss the second quarter 2020 results today at 8:00 a.m. Eastern Time. To participate in the call, dial 866-762-3111 (USA) or 210-874-7712 (International), conference ID 6053185 shortly before 8:00 a.m. Eastern Time. A replay of the call will be available for a limited period following the call. The audio webcast can be accessed on the Investor page of Alexion's website at: http://ir.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-E]

    Forward-Looking Statement

    This press release contains forward-looking statements, including statements related to: guidance regarding anticipated financial results for 2020 (and the assumptions related to such guidance); our expectations regarding the affects COVID-19 will have on our business and operations, including clinical trials and product supply; the strength of our business and continued growth; the Company's capital allocation strategy; plans to expand the Company's pipeline; future plans for, and the timing for, the commencement of future clinical trials and the expected timing of the receipt of results of certain clinical trials and studies, including clinical programs for ULTOMIRIS, a higher concentration formulation of ULTOMIRIS, a subcutaneous administration of ULTOMIRIS, SOLIRIS, ALXN1840, CAEL-101, AG10, ALXN2040, ALXN2050, ALXN1720, ALXN1830, ANDEXXA and CERDULATINIB; potential benefits of current products and products under development and in clinical trials; plans for development programs with third parties; and Alexion's future clinical, regulatory, and commercial plans for ULTOMIRIS and other products and product candidates. 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 principal product (SOLIRIS); our ability to facilitate the timely conversion from SOLIRIS to ULTOMIRIS; payer, physician and patient acceptance of ULTOMIRIS as an alternative to SOLIRIS; the impact of the COVID-19 pandemic on Alexion's business, including its sales, clinical trials, operations and supply chain; appropriate pricing for ULTOMIRIS; 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 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 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 planned 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; 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 against Alexion and inter partes review petitions submitted 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, including the acquisition of Portola Pharmaceuticals, Inc.; the possibility that expected tax benefits will not be realized; 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 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 March 31, 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.

    In addition to financial information prepared in accordance with GAAP, this press release also contains non-GAAP financial measures that Alexion believes, when considered together with the GAAP information, provide investors and management with supplemental information relating to performance, trends and prospects that promote a more complete understanding of our operating results and financial position during different periods. Alexion also uses these non-GAAP financial measures to establish budgets, set operational goals and to evaluate the performance of the business. The non-GAAP results, determined in accordance with our internal policies, exclude the impact of the following GAAP items (see reconciliation tables below for additional information): share-based compensation expense, fair value adjustment of inventory acquired, amortization of purchased intangible assets, changes in fair value of contingent consideration, restructuring and related expenses, upfront payments related to licenses and other strategic agreements, acquired in-process research and development, impairment of purchased intangible assets, gains and losses related to strategic equity investments, litigation charges, gain or loss on sale of a business or asset, gain or loss related to purchase options, contingent milestone payments associated with acquisitions of legal entities accounted for as asset acquisitions, acquisition-related costs and certain adjustments to income tax expense. These non-GAAP financial measures are not intended to be considered in isolation or as a substitute for, or superior to, the financial measures prepared and presented in accordance with GAAP, and should be reviewed in conjunction with the relevant GAAP financial measures. Please refer to the attached Reconciliations of GAAP to non-GAAP Financial Results and GAAP to non-GAAP 2020 Financial Guidance for explanations of the amounts adjusted to arrive at non-GAAP net income and non-GAAP earnings per share amounts for the three and six month periods ended June 30, 2020 and 2019 and projected twelve months ending December 31, 2020.

    (Tables Follow)

    ALEXION PHARMACEUTICALS, INC.

    TABLE 1: CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS

    (in millions, except per share amounts)

    (unaudited)

     

     

    Three months ended

     

    Six months ended

     

    June 30,

     

    June 30,

     

    2020

     

     

    2019

     

     

    2020

     

     

    2019

     

    Net product sales

    $

    1,444.5

     

     

     

    $

    1,202.5

     

     

     

    $

    2,889.1

     

     

     

    $

    2,342.7

     

     

    Other revenue

    0.1

     

     

     

    0.8

     

     

     

    0.3

     

     

     

    1.0

     

     

    Total revenues

    1,444.6

     

     

     

    1,203.3

     

     

     

    2,889.4

     

     

     

    2,343.7

     

     

    Costs and expenses:

     

     

     

     

     

     

     

    Cost of sales (exclusive of amortization of purchased intangible assets)

    144.9

     

     

     

    99.2

     

     

     

    256.6

     

     

     

    185.0

     

     

    Research and development

    221.1

     

     

     

    187.6

     

     

     

    422.0

     

     

     

    383.5

     

     

    Selling, general and administrative

    301.4

     

     

     

    299.3

     

     

     

    621.3

     

     

     

    580.8

     

     

    Acquired in-process research and development

     

     

     

    (4.1

    )

     

     

     

     

     

    (4.1

    )

     

    Amortization of purchased intangible assets

    73.7

     

     

     

    80.1

     

     

     

    147.4

     

     

     

    160.1

     

     

    Change in fair value of contingent consideration

    15.8

     

     

     

    6.1

     

     

     

    21.6

     

     

     

    (22.6

    )

     

    Acquisition-related costs

    4.6

     

     

     

     

     

     

    42.7

     

     

     

     

     

    Restructuring expenses

     

     

     

    2.5

     

     

     

    (0.8

    )

     

     

    11.6

     

     

    Impairment of intangible assets

    2,053.3

     

     

     

     

     

     

    2,053.3

     

     

     

     

     

    Total costs and expenses

    2,814.8

     

     

     

    670.7

     

     

     

    3,564.1

     

     

     

    1,294.3

     

     

    Operating (loss) income

    (1,370.2

    )

     

     

    532.6

     

     

     

    (674.7

    )

     

     

    1,049.4

     

     

    Other income and expense:

     

     

     

     

     

     

     

    Investment income (expense)

    41.5

     

     

     

    (14.9

    )

     

     

    36.3

     

     

     

    27.6

     

     

    Interest expense

    (23.6

    )

     

     

    (18.3

    )

     

     

    (49.4

    )

     

     

    (38.2

    )

     

    Other income and (expense)

    0.2

     

     

     

    0.1

     

     

     

    (0.7

    )

     

     

    2.5

     

     

    (Loss) income before income taxes

    (1,352.1

    )

     

     

    499.5

     

     

     

    (688.5

    )

     

     

    1,041.3

     

     

    Income tax (benefit) expense

    (284.0

    )

     

     

    39.7

     

     

     

    (178.0

    )

     

     

    (6.4

    )

     

    Net (loss) income

    $

    (1,068.1

    )

     

     

    $

    459.8

     

     

     

    $

    (510.5

    )

     

     

    $

    1,047.7

     

     

    Earnings (loss) per common share

     

     

     

     

     

     

     

    Basic

    $

    (4.84

    )

     

     

    $

    2.05

     

     

     

    $

    (2.31

    )

     

     

    $

    4.68

     

     

    Diluted

    $

    (4.84

    )

     

     

    $

    2.04

     

     

     

    $

    (2.31

    )

     

     

    $

    4.64

     

     

    Shares used in computing earnings (loss) per common share

     

     

     

     

     

     

     

    Basic

    220.6

     

     

     

    224.2

     

     

     

    221.1

     

     

     

    224.0

     

     

    Diluted

    220.6

     

     

     

    225.6

     

     

     

    221.1

     

     

     

    225.7

     

     

     

     

     

     

     

     

     

     

    ALEXION PHARMACEUTICALS, INC.

    TABLE 2: RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL RESULTS

    (in millions, except per share amounts)

    (unaudited)

     

    Three months ended

     

    Six months ended

     

    June 30,

     

    June 30,

     

    2020

     

     

    2019

     

     

    2020

     

     

    2019

     

    GAAP net (loss) income

    $

    (1,068.1

    )

     

     

    $

    459.8

     

     

     

    $

    (510.5

    )

     

     

    $

    1,047.7

     

     

    Before tax adjustments:

     

     

     

     

     

     

     

    Cost of sales:

     

     

     

     

     

     

     

    Share-based compensation

    3.1

     

     

     

    3.5

     

     

     

    6.2

     

     

     

    7.3

     

     

    Research and development expense:

     

     

     

     

     

     

     

    Share-based compensation

    16.5

     

     

     

    13.9

     

     

     

    31.7

     

     

     

    29.2

     

     

    Upfront payments related to licenses and other strategic agreements (1)

     

     

     

    25.0

     

     

     

     

     

     

    46.2

     

     

    Selling, general and administrative expense:

     

     

     

     

     

     

     

    Share-based compensation

    47.8

     

     

     

    43.5

     

     

     

    87.1

     

     

     

    81.2

     

     

    Litigation charges (2)

     

     

     

     

     

     

    21.5

     

     

     

    0.1

     

     

    Acquired in-process research and development

     

     

     

    (4.1

    )

     

     

     

     

     

    (4.1

    )

     

    Amortization of purchased intangible assets

    73.7

     

     

     

    80.1

     

     

     

    147.4

     

     

     

    160.1

     

     

    Change in fair value of contingent consideration (3)

    15.8

     

     

     

    6.1

     

     

     

    21.6

     

     

     

    (22.6

    )

     

    Acquisition-related costs (4)

    4.6

     

     

     

     

     

     

    42.7

     

     

     

     

     

    Restructuring expenses

     

     

     

    2.5

     

     

     

    (0.8

    )

     

     

    11.6

     

     

    Impairment of intangible assets(5)

    2,053.3

     

     

     

     

     

     

    2,053.3

     

     

     

     

     

    Investment income (expense):

     

     

     

     

     

     

     

    (Gains) and losses related to strategic equity investments

    (35.0

    )

     

     

    25.2

     

     

     

    (25.8

    )

     

     

    (8.6

    )

     

    Other income and (expense):

     

     

     

     

     

     

     

    Adjustments to income tax expense (6)

    (409.5

    )

     

     

    (50.5

    )

     

     

    (444.7

    )

     

     

    (197.5

    )

     

    Non-GAAP net income

    $

    702.2

     

     

     

    $

    605.0

     

     

     

    $

    1,429.7

     

     

     

    $

    1,150.6

     

     

     

     

     

     

     

     

     

     

    GAAP earnings (loss) per common share - diluted

    $

    (4.84

    )

     

     

    $

    2.04

     

     

     

    $

    (2.31

    )

     

     

    $

    4.64

     

     

    Non-GAAP earnings per common share - diluted

    $

    3.11

     

     

     

    $

    2.64

     

     

     

    $

    6.33

     

     

     

    $

    5.04

     

     

     

     

     

     

     

     

     

     

    Shares used in computing diluted earnings (loss) per common share (GAAP)

    220.6

     

     

     

    225.6

     

     

     

    221.1

     

     

     

    225.7

     

     

    Shares used in computing diluted earnings per common share (non-GAAP)

    225.7

     

     

     

    228.9

     

     

     

    225.9

     

     

     

    228.5

     

     

    (1) During the three months ended June 30, 2019, we recorded expense of $25.0 million in connection with an upfront payment on a strategic agreement that we entered into with Affibody AB (Affibody). During the six months ended June 30, 2019, we recorded expense of $46.2 million in connection with upfront payments on strategic agreements that we entered into with Affibody and Zealand Pharma A/S.

    (2) During the six months ended June 30, 2020, we recorded $21.5 million in litigation charges in connection with legal proceedings.

    (3) Changes in the fair value of contingent consideration expense for the three and six months ended June 30, 2020 as well as the six months ended June 30, 2019 include the impact of changes in the expected timing of achieving contingent milestones, in addition to the interest component related to the passage of time. For the three months ended June 30, 2019, changes in fair value of contingent consideration expense reflected only the interest component of contingent consideration related to the passage of time.

    (4) For the three and six months ended June 30, 2020, we recorded $4.6 million and $42.7 million, respectively, of acquisition-related costs in connection with the Achillion Pharmaceuticals, Inc. and Portola Pharmaceuticals, Inc. acquisitions. Acquisition-related costs primarily consist of Achillion and Portola transaction costs, costs associated with the accelerated vesting of stock options previously granted to Achillion employees and Achillion restructuring-related costs.

    (5) In the second quarter 2020, we recognized impairment charges of $2,053.3 million, primarily related to our KANUMA intangible asset.

    (6) Alexion's non-GAAP income tax expense for the three and six months ended June 30, 2020 and 2019 excludes the tax effect of pre-tax adjustments to GAAP profit. Non-GAAP income tax expense for the six months ended June 30, 2019 also excludes certain one-time tax benefits of $95.7 million and $30.3 million associated with a tax election made with respect to intellectual property of Wilson and a release of an existing valuation allowance, respectively.

    ALEXION PHARMACEUTICALS, INC.

    TABLE 3: RECONCILIATION OF GAAP TO NON-GAAP FINANCIAL GUIDANCE

    (in millions, except per share amounts and percentages)

    (unaudited)

     

     

     

    Twelve months ending

     

     

    December 31, 2020

     

     

    Low

     

    High

     

     

     

     

     

    GAAP net income

     

    $

    214

     

     

     

    $

    290

     

     

     

     

     

     

     

    Before tax adjustments:

     

     

     

     

    Share-based compensation

     

    295

     

     

     

    282

     

     

    Impairment of intangible assets

     

    2,053

     

     

     

    2,053

     

     

    Amortization of purchased intangible assets

     

    202

     

     

     

    202

     

     

    Acquisition-related costs

     

    131

     

     

     

    131

     

     

    Change in fair value of contingent consideration

     

    31

     

     

     

    31

     

     

    Restructuring expenses

     

    (1

    )

     

     

    (1

    )

     

    (Gains) and losses related to strategic equity investments

     

    (26

    )

     

     

    (26

    )

     

    Litigation charges

     

    22

     

     

     

    22

     

     

    Adjustments to income tax expense

     

    (519

    )

     

     

    (515

    )

     

    Non-GAAP net income

     

    $

    2,402

     

     

     

    $

    2,469

     

     

     

     

     

     

     

    Diluted GAAP earnings per common share

     

    $

    0.96

     

     

     

    $

    1.30

     

     

    Diluted non-GAAP earnings per common share

     

    $

    10.65

     

     

     

    $

    10.95

     

     

     

    Costs and expenses and margin (% total revenues)

     

     

     

     

     

     

     

     

     

    GAAP research and development expense

     

    19.2

     

    %

     

    18.1

     

    %

    Share-based compensation

     

    1.7

     

    %

     

    1.6

     

    %

    Restructuring related expenses

     

    0.0

     

    %

     

    0.0

     

    %

    Non-GAAP research and development expense

     

    17.5

     

    %

     

    16.5

     

    %

     

     

     

     

     

    GAAP selling, general and administrative expense

     

    25.7

     

    %

     

    24.5

     

    %

    Share-based compensation

     

    3.3

     

    %

     

    3.1

     

    %

    Restructuring related expenses

     

    0.0

     

    %

     

    0.0

     

    %

    Litigation charges

     

    0.4

     

    %

     

    0.4

     

    %

    Non-GAAP selling, general and administrative expense

     

    22.0

     

    %

     

    21.0

     

    %

     

     

     

     

     

    GAAP operating margin

     

    3.8

     

    %

     

    5.4

     

    %

    Share-based compensation

     

    5.3

     

    %

     

    5.0

     

    %

    Litigation charges

     

    0.4

     

    %

     

    0.4

     

    %

    Impairment of intangible assets

     

    37.0

     

    %

     

    36.7

     

    %

    Amortization of purchased intangible assets

     

    3.6

     

    %

     

    3.6

     

    %

    Acquisition-related costs

     

    2.4

     

    %

     

    2.3

     

    %

    Change in fair value of contingent consideration

     

    0.6

     

    %

     

    0.6

     

    %

    Restructuring expenses

     

    0.0

     

    %

     

    0.0

     

    %

    Non-GAAP operating margin

     

    53.0

     

    %

     

    54.0

     

    %

     

     

     

     

     

    Income tax expense (% of income before income taxes)

     

     

     

     

     

     

     

     

     

    GAAP income tax expense (benefit)

     

    (26.0

    )

    %

     

    (27.0

    )

    %

    Tax effect of pre-tax adjustments to GAAP net income

     

    42.5

     

    %

     

    42.5

     

    %

    Non-GAAP income tax expense

     

    16.5

     

    %

     

    15.5

     

    %

    Amounts may not foot due to rounding.

    ALEXION PHARMACEUTICALS, INC.

    TABLE 4: NET PRODUCT SALES BY GEOGRAPHY

    (in millions)

    (unaudited)

     

     

     

    Three months ended

     

    Six months ended

     

     

    June 30,

     

    June 30,

     

     

    2020

     

    2019

     

    2020

     

    2019

    SOLIRIS

     

     

     

     

     

     

     

     

    United States

     

    $

    553.3

     

     

    $

    496.3

     

     

    $

    1,109.5

     

     

    $

    960.0

     

    Europe

     

    247.9

     

     

    280.2

     

     

    511.4

     

     

    544.7

     

    Asia Pacific

     

    82.4

     

     

    110.3

     

     

    169.5

     

     

    211.2

     

    Rest of World

     

    91.9

     

     

    94.0

     

     

    208.0

     

     

    226.9

     

    Total SOLIRIS

     

    $

    975.5

     

     

    $

    980.8

     

     

    $

    1,998.4

     

     

    $

    1,942.8

     

    ULTOMIRIS

     

     

     

     

     

     

     

     

    United States

     

    $

    158.1

     

     

    $

    54.2

     

     

    $

    289.6

     

     

    $

    78.8

     

    Europe

     

    32.0

     

     

     

     

    65.8

     

     

     

    Asia Pacific

     

    59.6

     

     

     

     

    116.7

     

     

     

    Rest of World

     

    1.4

     

     

     

     

    1.8

     

     

     

    Total ULTOMIRIS

     

    $

    251.1

     

     

    $

    54.2

     

     

    $

    473.9

     

     

    $

    78.8

     

    STRENSIQ

     

     

     

     

     

     

     

     

    United States

     

    $

    140.7

     

     

    $

    106.2

     

     

    $

    268.8

     

     

    $

    205.7

     

    Europe

     

    18.3

     

     

    19.5

     

     

    42.3

     

     

    37.0

     

    Asia Pacific

     

    15.0

     

     

    12.1

     

     

    28.6

     

     

    22.0

     

    Rest of World

     

    10.3

     

     

    3.5

     

     

    16.8

     

     

    6.7

     

    Total STRENSIQ

     

    $

    184.3

     

     

    $

    141.3

     

     

    $

    356.5

     

     

    $

    271.4

     

    KANUMA

     

     

     

     

     

     

     

     

    United States

     

    $

    15.4

     

     

    $

    15.3

     

     

    $

    31.8

     

     

    $

    29.1

     

    Europe

     

    8.4

     

     

    6.8

     

     

    15.9

     

     

    13.1

     

    Asia Pacific

     

    0.9

     

     

    1.3

     

     

    1.8

     

     

    2.1

     

    Rest of World

     

    8.9

     

     

    2.8

     

     

    10.8

     

     

    5.4

     

    Total KANUMA

     

    $

    33.6

     

     

    $

    26.2

     

     

    $

    60.3

     

     

    $

    49.7

     

     

     

     

     

     

     

     

     

     

    Net Product Sales

     

     

     

     

     

     

     

     

    United States

     

    $

    867.5

     

     

    $

    672.0

     

     

    $

    1,699.7

     

     

    $

    1,273.6

     

    Europe

     

    306.6

     

     

    306.5

     

     

    635.4

     

     

    594.8

     

    Asia Pacific

     

    157.9

     

     

    123.7

     

     

    316.6

     

     

    235.3

     

    Rest of World

     

    112.5

     

     

    100.3

     

     

    237.4

     

     

    239.0

     

    Total Net Product Sales

     

    $

    1,444.5

     

     

    $

    1,202.5

     

     

    $

    2,889.1

     

     

    $

    2,342.7

     

    ALEXION PHARMACEUTICALS, INC.

    TABLE 5: CONDENSED CONSOLIDATED BALANCE SHEETS

    (in millions)

    (unaudited)

     

     

    June 30,

     

    December 31,

     

    2020

     

    2019

    Cash and cash equivalents

    $

    2,825.0

     

     

    $

    2,685.5

     

    Marketable securities

    26.8

     

     

    64.0

     

    Trade accounts receivable, net

    1,372.2

     

     

    1,243.2

     

    Inventories

    577.7

     

     

    627.6

     

    Prepaid expenses and other current assets

    566.2

     

     

    456.1

     

    Property, plant and equipment, net

    1,196.4

     

     

    1,163.3

     

    Intangible assets, net

    2,059.7

     

     

    3,344.3

     

    Goodwill

    5,075.2

     

     

    5,037.4

     

    Right of use operating assets

    209.9

     

     

    204.0

     

    Deferred tax assets

    2,332.4

     

     

    2,290.2

     

    Other assets

    461.7

     

     

    429.0

     

    Total assets

    $

    16,703.2

     

     

    $

    17,544.6

     

     

     

     

     

    Accounts payable and accrued expenses

    $

    861.6

     

     

    $

    966.7

     

    Current portion of long-term debt

    126.8

     

     

    126.7

     

    Other current liabilities

    131.7

     

     

    100.9

     

    Long-term debt, less current portion

    2,311.6

     

     

    2,375.0

     

    Contingent consideration

    374.7

     

     

    192.4

     

    Deferred tax liabilities

    1,946.8

     

     

    2,081.4

     

    Noncurrent operating lease liabilities

    169.4

     

     

    164.1

     

    Other liabilities

    289.8

     

     

    265.6

     

    Total liabilities

    6,212.4

     

     

    6,272.8

     

    Total stockholders' equity

    10,490.8

     

     

    11,271.8

     

    Total liabilities and stockholders' equity

    $

    16,703.2

     

     

    $

    17,544.6

     

    ALEXION PHARMACEUTICALS, INC.

    TABLE 6: CONDENSED CONSOLIDATED STATEMENT OF CASH FLOWS

    (in millions)(unaudited)

     

     

    Six months ended June 30,

     

    2020

     

     

    2019

     

    Cash flows from operating activities:

     

     

     

    Net (loss) income

    $

    (510.5

    )

     

     

    $

    1,047.7

     

     

    Adjustments to reconcile net (loss) income to net cash flows from operating activities:

     

     

     

    Depreciation and amortization

    179.1

     

     

     

    193.7

     

     

    Change in fair value of contingent consideration

    21.6

     

     

     

    (22.6

    )

     

    Share-based compensation expense

    125.0

     

     

     

    117.6

     

     

    Deferred taxes (benefit)

    (226.6

    )

     

     

    (40.8

    )

     

    Unrealized foreign currency loss (gain)

    3.3

     

     

     

    (4.1

    )

     

    Unrealized (gain) loss on forward contracts

    (11.5

    )

     

     

    11.3

     

     

    Unrealized gain on strategic equity investments

    (25.8

    )

     

     

    (8.6

    )

     

    Inventory obsolescence charge

    17.2

     

     

     

     

     

    Impairment of intangible assets

    2,053.3

     

     

     

     

     

    Other

    10.5

     

     

     

    (2.3

    )

     

    Changes in operating assets and liabilities, excluding the effect of acquisitions:

     

     

     

    Accounts receivable

    (137.6

    )

     

     

    (196.4

    )

     

    Inventories

    (15.1

    )

     

     

    (24.0

    )

     

    Prepaid expenses, right of use operating assets and other assets

    (54.8

    )

     

     

    (126.8

    )

     

    Accounts payable, accrued expenses, lease liabilities and other liabilities

    (88.5

    )

     

     

    23.6

     

     

    Net cash provided by operating activities

    1,339.6

     

     

     

    968.3

     

     

    Cash flows from investing activities:

     

     

     

    Purchases of available-for-sale debt securities

    (19.4

    )

     

     

    (41.1

    )

     

    Proceeds from maturity or sale of available-for-sale debt securities

    166.3

     

     

     

    139.3

     

     

    Purchases of mutual funds related to nonqualified deferred compensation plan

    (9.5

    )

     

     

    (10.9

    )

     

    Proceeds from sale of mutual funds related to nonqualified deferred compensation plan

    5.3

     

     

     

    9.0

     

     

    Purchases of property, plant and equipment

    (18.4

    )

     

     

    (82.8

    )

     

    Payment for acquisition of business, net of cash acquired

    (837.7

    )

     

     

     

     

    Purchases of strategic equity investments and options

    (38.1

    )

     

     

    (43.8

    )

     

    Purchase of intangible assets

     

     

     

    (8.0

    )

     

    Other

     

     

     

    0.2

     

     

    Net cash used in investing activities

    (751.5

    )

     

     

    (38.1

    )

     

    Cash flows from financing activities:

     

     

     

    Payments on term loan

    (65.3

    )

     

     

    (32.7

    )

     

    Payments on revolving credit facility

     

     

     

    (250.0

    )

     

    Repurchases of common stock

    (360.8

    )

     

     

    (48.9

    )

     

    Net proceeds from issuance of common stock under share-based compensation arrangements

    12.9

     

     

     

    20.5

     

     

    Other

    (17.5

    )

     

     

    (2.4

    )

     

    Net cash used in financing activities

    (430.7

    )

     

     

    (313.5

    )

     

    Effect of exchange rate changes on cash and cash equivalents and restricted cash

    (8.1

    )

     

     

    0.7

     

     

    Net change in cash and cash equivalents and restricted cash

    149.3

     

     

     

    617.4

     

     

    Cash and cash equivalents and restricted cash at beginning of period

    2,723.6

     

     

     

    1,367.3

     

     

    Cash and cash equivalents and restricted cash at end of period

    $

    2,872.9

     

     

     

    $

    1,984.7

     

     

     

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  2. Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the Company will report its financial results for the second quarter ended June 30, 2020 before the US financial markets open on July 30, 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 6053185 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 second quarter ended June 30, 2020 before the US financial markets open on July 30, 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 6053185 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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  3. - Acquisition diversifies company's hematology, neurology and critical care commercial portfolio with addition of only approved Factor Xa inhibitor reversal agent -

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the successful completion of its acquisition of Portola Pharmaceuticals, Inc. (NASDAQ:PTLA). The acquisition adds Factor Xa inhibitor reversal agent Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo], marketed as Ondexxya® in Europe, to Alexion's commercial portfolio. Andexxa is the first and only approved Factor Xa inhibitor reversal agent and has demonstrated transformative clinical value by rapidly reversing the anticoagulant effects of Factor Xa inhibitors rivaroxaban and apixaban in severe and uncontrolled…

    - Acquisition diversifies company's hematology, neurology and critical care commercial portfolio with addition of only approved Factor Xa inhibitor reversal agent -

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the successful completion of its acquisition of Portola Pharmaceuticals, Inc. (NASDAQ:PTLA). The acquisition adds Factor Xa inhibitor reversal agent Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo], marketed as Ondexxya® in Europe, to Alexion's commercial portfolio. Andexxa is the first and only approved Factor Xa inhibitor reversal agent and has demonstrated transformative clinical value by rapidly reversing the anticoagulant effects of Factor Xa inhibitors rivaroxaban and apixaban in severe and uncontrolled bleeding.

    "This acquisition provides the opportunity to grow our commercial portfolio, which builds on the significant progress we've made diversifying our pipeline over the last few years," said Ludwig Hantson, Ph.D., Chief Executive Officer of Alexion. "We are excited to add a transformative, first-in-class medicine like Andexxa, which rapidly reverses life-threatening bleeds that result from Factor Xa inhibitors, to our growing critical care portfolio. This important medicine is also a clear strategic fit with our existing expertise in hematology and neurology, and we are confident we can apply our demonstrated global commercial excellence to enhance access and broaden the number of patients helped by Andexxa."

    Transaction Details

    Alexion completed the acquisition through a tender offer and subsequent merger of Portola with Odyssey Merger Sub Inc., a wholly owned subsidiary of Alexion ("Buyer"). Portola is now a wholly owned subsidiary of Alexion. The tender offer for all of the outstanding shares of common stock of Portola at a price of $18.00 per share expired as scheduled, one minute following 11:59 p.m., New York City time, on July 1, 2020. American Stock Transfer & Trust Company, LLC, the depositary and paying agent for the tender offer, has advised Alexion that 62,654,962 shares of Portola common stock were validly tendered and not validly withdrawn in the tender offer, representing approximately 79.7% of the shares outstanding. In addition, the depositary has advised Alexion that, as of the offer expiration time, Notices of Guaranteed Delivery had been delivered with respect to 2,701,052 additional shares, representing approximately 3.4% of the shares outstanding. All of the conditions to the tender offer having been satisfied, Buyer has accepted for payment and will promptly pay for all shares tendered. The transaction will be funded with cash on hand.

    On July 2, 2020, Alexion completed its acquisition of Portola through the merger of Buyer with and into Portola without a vote of Portola's shareholders pursuant to Section 251(h) of the Delaware General Corporation Law. As a result of the merger, Portola became a wholly owned subsidiary of Alexion. In connection with the merger, all shares of Portola common stock outstanding immediately prior to the effective time (other than shares owned by Alexion, Buyer, Portola, any other subsidiary of Alexion or any subsidiary of Portola, or shares that are held in Portola's treasury, or shares held by any Portola stockholder who has properly demanded and perfected appraisal rights under Delaware law) have been converted into the right to receive $18.00 per share in cash, without interest (less any required withholding taxes), the same amount paid for all shares validly tendered and not validly withdrawn in the tender offer. As a result of the merger, as of July 2, 2020, Portola common stock will cease to be traded on the NASDAQ Global Select Market.

    About Andexxa

    Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo] is a recombinant modified human factor Xa (FXa) protein indicated for patients treated with rivaroxaban or apixaban, when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.

    IMPORTANT SAFETY INFORMATION

    The most frequently reported adverse reactions in clinical trials in healthy subjects with Andexxa were mild or moderate infusion-related reactions comprising symptoms such as flushing and feeling hot (very common), and cough, dysgeusia, and dyspnea (common). Amongst bleeding patients, commonly reported side effects were ischemic stroke and pyrexia, with uncommon reported side effects of cerebral infarction, cerebrovascular accident, transient ischemic attack, acute myocardial infarction, cardiac arrest, myocardial infarction, deep vein thrombosis, iliac artery occlusion, pulmonary embolism.

    Please refer to full Prescribing Information for more information, including Boxed Warning, at www.Andexxa.com.

    About Alexion

    Alexion is a global biopharmaceutical company focused on serving patients and families affected by rare and devastating diseases through the discovery, development and commercialization of life-changing medicines. As the global leader in complement biology and inhibition for more than 20 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

    Certain statements made in this press release may constitute forward-looking statements. Forward-looking statements include, among other things, statements related to the acquisition of Portola by Alexion, including: Alexion's belief that the acquisition will build on the progress that Alexion has made diversifying its portfolio over the last few years; that Andexxa is a transformative, first-in-class medicine; that Andexxa will be a strategic fit within Alexion's existing hematology and neurology portfolio; and Alexion's belief that it can apply its global commercial excellence to enhance access and broaden the number of patients helped by Andexxa. Forward-looking statements are based on management's current expectations, beliefs, estimates, projections and assumptions. As such, forward-looking statements are not guarantees of future performance and involve inherent risks and uncertainties that are difficult to predict. As a result, a number of important factors could cause actual results to differ materially from those indicated by such forward-looking statements, including: the anticipated benefits of Andexxa and other Portola therapies not being realized, including the result of delays or failure to obtain regulatory approval and failure to attain sales targets; the phase 4 study regarding Andexxa does not meet its designated endpoints and/or is not deemed safe and effective by the Food and Drug Administration (the "FDA") or other regulatory agencies (and commercial sales are prohibited or limited); future clinical trials of Portola products not proving that the therapies are safe and effective to the level required by regulators; anticipated Andexxa sales targets are not satisfied; Andexxa does not gain acceptance among physicians, payers and patients; the commercial efforts of Alexion do not result in increased sales of Andexxa; potential future competition by other Factor Xa inhibitor reversal agents (or other competitive therapies); decisions of regulatory authorities regarding the adequacy of the research and clinical tests, marketing approval or material limitations on the marketing of Portola products; delays or failure of product candidates or label extension of existing products to obtain regulatory approval; delays or the inability to launch product candidates (including products with label extensions) due to regulatory restrictions; unanticipated expenses; interruptions or failures in the manufacture and supply of products and product candidates; failure to satisfactorily address matters raised by the FDA and other regulatory agencies; the possibility that results of clinical trials are not predictive of safety and efficacy results of products in broader patient populations; the possibility that clinical trials of product candidates could be delayed or terminated prior to completion for a number of reasons; the adequacy of pharmacovigilance and drug safety reporting processes; the impact of the COVID-19 pandemic on Alexion's business operations, including sales, clinical trials, operations and supply chain; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the quarter ended March 31, 2020 and in its 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. – ULTOMIRIS is the first and only long-acting C5 inhibitor for aHUS, administered every other month, reducing the treatment burden for adults and children –

    – ULTOMIRIS has the potential to become the new standard of care in Europe for the treatment of aHUS –

    – aHUS is an ultra-rare disease which may progressively damage the kidney and other organs1,2

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the European Commission has approved ULTOMIRIS® (ravulizumab)—the first and only long-acting C5 complement inhibitor administered every eight weeks*—for the treatment of adults and children with a body weight of 10 kg or above with atypical hemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have…

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

    – ULTOMIRIS has the potential to become the new standard of care in Europe for the treatment of aHUS –

    – aHUS is an ultra-rare disease which may progressively damage the kidney and other organs1,2

    Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that the European Commission has approved ULTOMIRIS® (ravulizumab)—the first and only long-acting C5 complement inhibitor administered every eight weeks*—for the treatment of adults and children with a body weight of 10 kg or above with atypical hemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received SOLIRIS® (eculizumab) for at least three months and have evidence of response to eculizumab.

    "The consequences of aHUS are severe and potentially life-threatening, creating significant challenges and uncertainty for patients and their families. The goal of aHUS treatment is to prevent the body from attacking itself through the inhibition of uncontrolled C5 complement activation – a part of the body's immune system," said Prof. Dr. Hermann Haller, Clinic for Nephrology, University Hannover, Germany. "Clinical study results showed that both adults and children with aHUS had immediate and complete C5 inhibition following the first dose of ULTOMIRIS sustained for up to eight weeks. In addition to having demonstrated clinically meaningful benefits in people with aHUS, ULTOMIRIS provides greater freedom with significantly fewer infusions per year."

    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 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,3 so a timely and accurate diagnosis – in addition to treatment – is critical to improving patient outcomes.

    "At Alexion, our goal is to continue to improve the lives of people and families affected by aHUS and other serious rare diseases," said John Orloff, M.D., Executive Vice President and Head of Research & Development at Alexion. "Treatment with ULTOMIRIS offers convenient eight-week dosing, which we believe is preferred by patients because it provides them with greater flexibility and improved quality of life, while also reducing the burden on healthcare systems that currently face significant strain in many countries. Today's marketing authorization marks an important step in our efforts to establish ULTOMIRIS as the new standard of care for the aHUS patient community."

    The European Commission approval is based on data from two global, single-arm open-label studies of ULTOMIRIS – one in adults and one in children, referred to as pediatrics in the study – with aHUS. Both studies are ongoing. 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). Treatment with ULTOMIRIS resulted in normalization of platelet count in 86 percent of adults and 94 percent of children, normalization of LDH (marker of hemolysis) in 84 percent of adults and 94 percent of children, and improved kidney function in 63 percent of adults and 94 percent (interim data) of children (for patients on dialysis at enrollment, baseline was established after they had come off dialysis). 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.

    The U.S. Food and Drug Administration (FDA) approved ULTOMIRIS for the treatment of aHUS to inhibit TMA for adult and pediatric (one month of age and older) patients in October 2019. A regulatory filing for marketing authorization of ULTOMIRIS for the treatment of aHUS in Japan is currently under review. ULTOMIRIS is also approved for the treatment of adult patients with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S and Japan, and in the EU as a treatment for adult patients with PNH with hemolysis with clinical symptoms indicative of high disease activity and also for adult patients who are clinically stable after having been treated with eculizumab for at least the past six months.

    * Starting two weeks after the loading dose, maintenance doses are administered once every four or eight weeks (depending on body weight)

    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®

    ULTOMIRIS® (ravulizumab) is the first and only long-acting C5 complement inhibitor. The medication works by inhibiting the C5 protein in the terminal complement cascade, a part of the body's immune system. When activated in an uncontrolled manner, the complement cascade over-responds, leading the body to attack its own healthy cells. ULTOMIRIS is administered intravenously every eight weeks or 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) and in the U.S. for atypical hemolytic uremic syndrome (aHUS) to inhibit complement-mediated thrombotic microangiopathy (TMA) in adult and pediatric (one month of age and older) patients. To learn more about the regulatory status of ULTOMIRIS in the countries that we serve, please visit www.alexion.com.

    U.S. INDICATIONS & IMPORTANT SAFETY INFORMATION FOR ULTOMIRIS (ravulizumab-cwvz) 300 mg / 30 mL injection for intravenous use

    U.S. INDICATIONS

    ULTOMIRIS is a prescription medicine called a monoclonal antibody. ULTOMIRIS is used to treat adults with a disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). ULTOMIRIS is used to treat 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.

    U.S. IMPORTANT SAFETY INFORMATION

    ULTOMIRIS is a medicine that affects the immune system. ULTOMIRIS can lower the ability of the immune system to fight infections. ULTOMIRIS increases the chance of getting serious and life-threatening meningococcal infections. Meningococcal infections may quickly become life-threatening and cause death if not recognized and treated early.

    Meningococcal vaccines must be received at least 2 weeks before the first dose of ULTOMIRIS if one has not already had this vaccine. If one's doctor decided that urgent treatment with ULTOMIRIS is needed, meningococcal vaccination should be administered as soon as possible. If one has not been vaccinated and ULTOMIRIS therapy must be initiated immediately, 2 weeks of antibiotics should also be administered with the vaccinations. If one had a meningococcal vaccine in the past, additional vaccination might be needed before starting ULTOMIRIS. One's doctor will decide if additional meningococcal vaccination is needed. Meningococcal vaccines reduce the risk of meningococcal infection but do not prevent all meningococcal infections. Call one's doctor or get emergency medical care right away if any of these signs and symptoms of a meningococcal infection occur: 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. One's doctor will give a Patient Safety Card about the risk of meningococcal infection. Carry the card at all times during treatment and for 8 months after your ULTOMIRIS dose.

    ULTOMIRIS is only available through a program called the ULTOMIRIS REMS.

    ULTOMIRIS may also increase the risk of other types of serious infections. People who take ULTOMIRIS may have an increased risk of getting infections caused by Streptococcus pneumoniae and Haemophilus influenzae. Certain people may also have an increased risk of gonorrhea infection. To find out if one is at risk for gonorrhea infection, about gonorrhea prevention, and regular testing, talk to the doctor. Call the doctor right away if one has any new signs or symptoms of infection.

    Do not receive ULTOMIRIS if one has a meningococcal infection, or has not been vaccinated against meningococcal infection unless the doctor decides that urgent treatment with ULTOMIRIS is needed.

    Before one receives ULTOMIRIS, tell the doctor about all of the medical conditions, including if one: has 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 an unborn baby. It is not known if ULTOMIRIS passes into the breast milk. One should not breastfeed during treatment and for 8 months after one's final dose of ULTOMIRIS.

    Tell the doctor about all the medicines one takes, including prescription and over-the-counter medicines, vitamins, and herbal supplements. ULTOMIRIS and other medicines can affect each other causing side effects. Know the medicines one takes and the vaccines one receives. Keep a list of them to show the doctor and pharmacist when one gets a new medicine.

    If one has PNH and stops receiving ULTOMIRIS, the doctor will need to monitor closely for at least 16 weeks after one stops ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of the red blood cells due to PNH. Symptoms or problems that can happen due to red blood cell breakdown include: drop in the red blood cell count, tiredness, blood in the urine, stomach-area (abdomen) pain, shortness of breath, blood clots, trouble swallowing, and erectile dysfunction (ED) in males. If one has aHUS, the doctor will need to monitor closely for at least 12 months after stopping treatment for signs of worsening aHUS symptoms or problems related to a type of abnormal clotting and breakdown of the 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. If one misses an ULTOMIRIS infusion, call the doctor right away.

    ULTOMIRIS can cause serious side effects including infusion reactions. Infusion reactions may happen during one's ULTOMIRIS infusion. Symptoms of an infusion reaction with ULTOMIRIS may include lower back pain, pain with the infusion, feeling faint or discomfort in the arms or legs. Tell the doctor or nurse right away if these symptoms develop, or any other symptoms during the ULTOMIRIS infusion that may mean one is having a serious infusion reaction, including: chest pain, trouble breathing or shortness of breath, swelling of the face, tongue, or throat, and feel faint or pass out. One's doctor will treat the symptoms as needed.

    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 infections, diarrhea, nausea, vomiting, headache, high blood pressure, and fever.

    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 and devastating diseases through the discovery, development and commercialization of life-changing medicines. As the global leader in complement biology and inhibition for more than 20 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). 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 that involve risks and uncertainties relating to future events and the future performance of Alexion, including statements related to: treatment with ULTOMIRIS will offer convenient eight-week dosing without compromising efficacy or safety; treatment with ULTOMIRIS is preferred by patients because it provides them with greater flexibility and improved quality of life, while also reducing the burden on healthcare systems; the consequences of aHUS create significant challenges and uncertainty for patients and their families; timely and accurate diagnosis of aHUS – in addition to treatment – is critical to improving patient outcomes; the Company's goal is to continue to improve the lives of people and families affected by aHUS and other serious rare diseases; the marketing authorization marks an important step in our efforts to establish ULTOMIRIS as the new standard of care for the aHUS patient community; and ULTOMIRIS has the potential to become the new standard of care in Europe for the treatment of aHUS; the anticipated benefits of ULTOMIRIS as a treatment for patients with aHUS. 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 realized by patients); use of ULTOMRIS does not ultimately provide comparable efficacy and safety to patients using the therapy; 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); ULTOMIRIS may not be adopted by physicians, patients and payers as a treatment for aHUS (and may not be accepted as the standard of care); the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products, including ULTOMIRIS (or we fail to adequately operate or manage our clinical trials) which could cause us to discontinue sales of the product (or halt trials, delay or prevent us from making regulatory approval filings or result in denial of approval of our product candidates); the severity of the impact of the COVID-19 pandemic on Alexion's business, including on commercial and clinical 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 the European Commission and other 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 (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, including investigations of Alexion by the U.S. Securities and Exchange Commission; 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 pending acquisition of Portola Pharmaceuticals, Inc., the acquisition of 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 March 31, 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.

    References:

    1. Laurence J. Atypical hemolytic uremic syndrome (aHUS): making the diagnosis. Clin Adv Hematol Oncol. 2012;10(suppl 17):1-12.
    2. Campistol JM, Arias M, Ariceta G, et al. An update for atypical haemolytic uraemic syndrome: diagnosis and treatment. Nefrologia. 2013 Jan 18;33(1): 27-45.
    3. Fremeaux -Bacchi, V, et al. Genetics and outcomes of atypical HUS in Children and Adults: A Nationwide French Series Comparing Children and Adults. Clin J Am Soc Nephrol. April 2013, 8 (4) 554-562; DOI: https://doi.org/10.2215/CJN.04760512

     

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