Exhibit 99.1
January 12, 2021 Chelsey living with NMOSD 39th AnnualJ.P. Morgan Healthcare Conference Aradhana Sarin, M.D.Chief Financial Officer
Forward Looking Statements This presentation contains forward-looking statements, including statements related to: the proposed acquisition by AstraZeneca and the anticipated timing of such acquisition; the benefits of the acquisition and the ability of the acquisition to deliver value to shareholders; the ability of AstraZeneca to successfully integrate Alexion's operations, and the ability of AstraZeneca to implement its plans, forecasts and other expectations with respect to Alexion's business after the completion of the proposed acquisition and realize expected synergies; Alexion’s anticipated financial results (including short-term guidance and long-range financial guidance), anticipated 2020 revenue, operating margin and non-GAAP EPS, revenue by 2025, our cumulative average growth rate through 2025, and peak revenue from our pipeline beyond 2025 (and all of the assumptions, judgments and estimates related to such anticipated future results); ambition to quadruple the number of neurology patients in the US by 2025; ambition for 10 product launches by 2023; anticipated future product launches (and the timing of those launches); plans to establish 7 blockbuster franchises and the targeted indications in each franchise; plans to make regulatory filings for approval of certain products and product candidates, the expected timing of such filings as well as the expected timing of the receipt of certain regulatory approvals to market a product; our ambition to treat 7,500 neurology patients by 2025; our strategy and ability to grow the ANDEXXA business both in indication and geography; ability to realize continued and sustainable growth in our aHUS franchise and metabolic business; the ability of our pipeline and existing products to provide long-term sustainable growth for shareholders; Company’s plans for future clinical trials and studies, the timing for the commencement and conclusion of future clinical trials and the expected timing of the receipt of results of clinical trials and studies; the anticipated number of patients that may be treated with the Company’s products both currently approved and in our pipeline; the Company’s goals for 2021 and near term events to support value creation for shareholders; the Company’s strategy for long-term value creation (including the following: establishing ULTOMIRIS as the new standard of care in PNH, aHUS and Neurology, plans to launch our next generation C5 formulations, plans to expand our presence in Neurology, focus expansion of ULTOMIRIS on direct-to-phase 3 rapid proof of concept trials, plans to further diversify our assets and establish novel platforms and the benefits of those plans); plans for additional formulations of ULTOMIRIS (high concentration and subcutaneous) and the timing for regulatory approval and potential benefits of such formulations; Alexion’s ambitions for its portfolio of assets; the anticipated pricing of ULTOMIRIS in PNH and aHUS; ambitions to increase aHUS program; the affected patient populations in the indications we are pursuing; plans to develop and launch ALXN1720; plans for our CSR program; the growth potential and plans for our FcRn program; and continued diversification of the pipeline. 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 risk that the proposed acquisition of Alexion by Astra Zeneca may not be completed and such failure could negatively affect our stock price and future business and financial results and if the Astra Zeneca merger agreement is terminated, we may be forced to pay a termination fee to Astra Zeneca; the severity of the impact of the COVID-19 pandemic on Alexion’s business, including on commercial and clinical development programs; our dependence on sales from our C5 products (SOLIRIS and ULTOMIRIS); delays (expected or unexpected) in the time it takes regulatory agencies to review and make determinations on applications for the marketing approval of our products; Alexion’s inability to timely submit (or failure to submit) future applications for regulatory approval for our products and product candidates; payer, physician and patient acceptance of ULTOMIRIS as an alternative to SOLIRIS; appropriate pricing for ULTOMIRIS; future competition from biosimilars and novel products; inability to timely initiate (or failure to initiate) and complete future clinical trials due to safety issues, IRB decisions, CMC-related issues, expense or unfavorable results from earlier trials (among other reasons); the number of patients that will use our products and product candidates in the future; decisions of regulatory authorities regarding the adequacy of our research, marketing approval or material limitations on the marketing of our products; delays or failure of product candidates to obtain regulatory approval; delays or the inability to launch product candidates due to regulatory restrictions, anticipated expense or other matters; interruptions or failures in the manufacture and supply of our products and our product candidates; failure to satisfactorily address matters raised by the FDA and other regulatory agencies; results in early stage clinical trials may not be indicative of full results or results from later stage or larger clinical trials (or broader patient populations) and do not ensure regulatory approval; the possibility that results of clinical trials are not predictive of safety and efficacy and potency of our products (or we fail to adequately operate or manage our clinical trials) which could cause us to halt trials, delay or prevent us from making regulatory approval filings or result in denial of regulatory approval of our product candidates; unexpected delays in clinical trials; unexpected concerns that may arise from additional data or analysis obtained during clinical trials; future product improvements may not be realized due to expense or feasibility or other factors; uncertainty of long-term success in developing, licensing or acquiring other product candidates or additional indications for existing products; inability to complete acquisitions due to failure of regulatory approval or material changes in target or otherwise; inability to complete acquisitions and investments due to increased competition for technology; the possibility that current rates of adoption of our products are not sustained (or anticipated adoption rates are not realized); internal development efforts do not result in commercialization of additional products; the adequacy of our pharmacovigilance and drug safety reporting processes; failure to protect and enforce our data, intellectual property and proprietary rights and the risks and uncertainties relating to intellectual property claims, lawsuits and challenges against us (including intellectual property lawsuits relating to products brought by third parties against Alexion); the risk that third party payors (including governmental agencies) will not reimburse or continue to reimburse for the use of our products at acceptable rates or at all; failure to realize the benefits and potential of investments, collaborations, licenses and acquisitions; failure by regulatory authorities to approve transactions; the possibility that expected tax benefits will not be realized or that tax liabilities exceed current expectations; assessment of impact of recent accounting pronouncements; potential declines in sovereign credit ratings or sovereign defaults in countries where we sell our products; delay of collection or reduction in reimbursement due to adverse economic conditions or changes in government and private insurer regulations and approaches to reimbursement; uncertainties surrounding legal proceedings, company investigations and government investigations; the risk that estimates regarding the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D and other future indications we are pursuing are inaccurate; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; risks related to the acquisition of companies and co-development and collaboration efforts; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended September 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.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 Financial Guidance for explanations of the amounts adjusted to arrive at non-GAAP net income, non-GAAP and non-GAAP earnings per share amounts for the three and nine month periods ended September 30, 2020 and 2019 and for the projected twelve months ending December 31, 2020.Amounts may not foot due to rounding.
ALEXION’S NEXT CHAPTER Our Next Chapter & Advances shared mission of following the science and using innovative approaches to develop life-changing medicines for patientsStrengthens AstraZeneca’s presence in immunology by adding Alexion’s strong pipeline and unique complement technology platformsCombined company to have broad global coverage across primary and specialty careAstraZeneca plans to create rare disease business unitCombined organization will be well positioned to accelerate innovation and deliver enhanced value for our shareholders, patients and rare disease communities we serve
Standalone ALXN Targeting $9-10B in Global Revenues in 2025 1Ambition Baseline - 12/31/19 1,885 patients 2 2020 based guidance issued 10/29/20 32025 $9-10B target is at constant currencies (9/30/20 levels) 4 Illustrative, non risk-adjusted revenues, peak sales year varies by program Key Building Blocks To Achieving 2025 Revenue Ambition Sustainable PNH and Growing aHUS & Metabolic Businesses Initial Revenue Contribution From 10 Launches By 2023 Expand Neurology U.S. Patient Volume 4x (to ~7,500 US Patients) 1 Grow ANDEXXA Utilization PATH FOR STRONG GROWTH ~$6BGlobal Revenues2 $9-10BGlobal Revenues3 > 10% CAGR PNH (~30%) PNH (~15%) 2025 2020 2023 >$10B+in pipeline peak sales potential4 Beyond 2025 1 2 3 4
MISSION FOCUSED EXECUTION Compelling Portfolio For Patients Today ULTOMIRIS®(ravulizumab-cwvz) SOLIRIS®(ECULIZUMAB) STRENSIQ®(ASFOTASE ALFA) KANUMA®(SEBELIPASE ALFA) ANDEXXA/ONDEXXYA®(ANDEXANET ALFA) 5 Transformative Products PNH(Paroxsysmal Nocturnal Hemoglobinuria) 7 Rare & Devastating Conditions aHUS(Atypical Hemolytic Uremic Syndrome) gMG(Generalized Myesthenia Gravis) NMOSD(Neuromyelitis Optica Spectrum Disorder) HPP(Hypophosphatasia) LAL-D(Lysosomal Acid Lipase Deficiency) FxA Reversal(For Major Life-Threatening Bleeds) Patient Centric Rare Disease Model Best In Class Data Analytics Rare Disease Focused Field Force Operational Excellence In Manufacturing & Supply Patient-centered Access Models Personalized High-Touch Patient Support Services* *OneSource is a U.S. Patient Service Model RARE DISEASE TAILORED CAPABILITIES, FOCUS & GLOBAL SCALE
Ph3 to Initiate 1H ‘21 Transformed Our Development Pipeline ULTOMIRIS ALXN1720 SOLIRIS LEAD and EXPAND IV (ALS) SC Weekly IV (gMG) IV (NMOSD) IV (HSCT-TMA) SC (DM)3 IV (CM-TMA) IV (Renal Basket) IV (COVID-19)2 IV (GBS) Japan Only SC (gMG)3 ALXN1840 ALXN1830 CAEL-101 AG10 ALXN2040 ALXN2050 ANDEXXA CERDULATINIB DIVERSIFY IV (AL Amyloidosis)5 Oral (Wilson) SC FcRn (WAIHA)4 SC FcRn (gMG)4 Oral (PNH with EVH) Oral (PNH Monotherapy) Oral (ATTR-CM) Japan Only6 Oral (Renal Basket) ANDEXXA-S (Urgent Surgery) Lymphoma (CTCL, PTCL, FL) Oral (Geographic Atrophy) Estimated TLR 2H ‘221 Estimated Filing 3Q ‘21 Estimated TLR 2H ‘211 Estimated TLR 2H ‘221 Ph3 Initiated 3Q ‘20 Estimated TLR 1H ‘211 Ph3 Initiated 4Q ‘20 Est. TLR 1H ‘211 Ph3 Initiated 4Q ‘20 Estimated TLR 2H ‘211 Ph3 Initiated 4Q ‘20 Ph2 to Initiate 1H ‘21 Ph3 to Initiate 1H ‘21 Ph2 Initiated 4Q ‘20 Estimated TLR 1H ‘211 Ph2 to Initiate 1H ‘21 Estimated TLR 1H ‘211 Ph2 to Initiate 2H ‘21 Est. TLR 1H ‘211 Hematology Metabolics Nephrology Ophthalmology Cardiology Neurology Acute Care PHASE 1 PHASE 2 PHASE 3 1TLR: Topline readout; 2Adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome (ARDS); 31720 currently in HV Ph1 with topline readout estimated 1H ‘21 and subsequent DM and gMG trials to begin after that; 41830 Ph1 HV program to reinitiate for SC formulation with WAIHA and gMG Ph2 programs to follow in 2021; 5Structured as option to acquire Caelum; 6Exclusive license to develop & commercialize in Japan MISSION FOCUSED EXECUTION ALXN1820 SC HV Study To Initiate 1Q ‘21 ALXN1850 SC HV Study To Initiate 2Q ‘21 NEW NEW Other/TBD Ph2 to Initiate 2H ‘21 Ph2 to Initiate 2H ‘21 INITIATED INITIATED INITIATED INITIATED IND Filed 4Q ‘20 IND Filed 4Q ’20
MISSION FOCUSED EXECUTION With Potential for 7 Blockbuster Franchises LEAD AND EXPAND Hematology Neurology Nephrology Metabolics Cardiology Ophthalmology Acute Care DIVERSIFY 1Japan Development Only Paroxysmal Nocturnal Hemoglobinuria (PNH) Warm Autoimmune Hemolytic Anemia(WAIHA) Hypophosphatasia (HPP) Lysosomal Acid Lipase Deficiency(LAL-D) Wilson Disease Geographic Atrophy(GA) ALAmyloidosis Transthyretin Amyloid Cardiomyopathy1(ATTR-CM) Factor Xa Major Bleeds Factor Xa Reversal for Urgent Surgery Generalized Myasthenia Gravis(gMG) Neuromyelitis Optica Spectrum Disorder(NMOSD) Amyotrophic Lateral Sclerosis(ALS) Dermatomyositis(DM) Guillain-Barre Syndrome1(GBS) Atypical Hemolytic Uremic Syndrome (aHUS) Renal Basket (LN, IgAN, PMN, C3G) Hematopoietic Stem Cell Transplantation2(HSCT-TMA) Complement Mediated TMA(CM-TMA)
Strong Financial Execution 1A reconciliation of GAAP to non-GAAP financial results is provided in the appendix and is available at www.alexion.com *2020E based on midpoint of 2020 financial guidance issued on October 29, 2020. The 2020 estimates (and related assumptions) set forth on this slide reflect estimates as of October 29, 2020 and the information on this slide has not been updated to reflect any events subsequent to October 29, 2020.. +19% CAGR +26% CAGR +1000 bps Double Digit Revenue Growth ($M) Consistent Non-GAAP EPS Growth1 Best-In-Class Non-GAAP Operating Margins1 * MISSION FOCUSED EXECUTION +19% YoY Revenue Growth vs. 2019 highlights resilience of business despite Covid-19
Neurology is Key Growth Driver through 2025 PATH FOR STRONG GROWTH 1Ambition Baseline - 12/31/19 1,885 patients (4x growth ambition includes only gMG and NMOSD indications) AMBITION TO TREAT 4x U.S. NEUROLOGY PATIENTS US Neurology Patients (gMG & NMOSD) gMG Launch NMOSD Launch gMG Launch NMOSD Launch Estimated ULTOMIRIS launch in gMG 2H 2022 ALXN1720 ~5-8KPatients ~20KPatients Majority of gMG AChR+ Patients Addressable Addressing Incremental Share of gMG Population With ULTOMIRIS Key to Achieving Ambition 60-80K Total US gMG Patients 1 NEW ALXN1720 gMG Launch
PATH FOR STRONG GROWTH Maximizing ANDEXXA Potential Access Criteria FormularyBleeding ProtocolEMR System Availability1DUR Conducted2 Awareness / Advocacy Clinical ChampionsReimbursement Pathway Awareness (incl. NTAP)Clinical & Economic Value Education Demand Generation Network Center Adoption & UtilizationReferral Network Activation Underway Underway Underway Executing Against Re-Powered Launch Strategy Key Progress Acceleration of demand to pre-COVID levels in the US Progressing EU payer & access negotiations, launch planning Executing against clinical and economic value education plans Integration and Re-Allocation of Commercial Efforts Expand Geographic Reach and Label of ANDEXXA Shift field teams to focus towards access and champion mobilization Seek reimbursement in new markets and pursue development for broader label (edoxaban/enoxaparin & urgent surgery) Nearing Completion Underway Focus On Optimizing New and Existing Top Tier Accounts 1EMR: Electronic Medical Record; 2DUR: Drug Use Review Filed sBLA to expand US label to include enoxaparin and edoxaban
PATH FOR STRONG GROWTH Confidence in Sustainability of C5 Franchise ALXN1720 First Generation C5 Second Generation C5 Third Generation C5 Expanding to Rare >50K Potential Addressable Patients Ultra-Rare Focus<6K Patients Continued Expansion in Rare>100K Potential Addressable Patients IMPROVING PROFILE FOR PATIENTS THROUGH THREE GENERATIONS of C5 INHIBITION Compelling ULTOMIRIS Profile Majority of C5 market will convert to ULTOMIRIS vs. SOLIRISPoint estimates in favor of ULTOMIRIS on all 11 endpoints across two large Ph3 studiesProven long-term safety recordDosing convenience with only 6-7 (Q8W) 45-minute infusions per year Expected dosing optionality with once-weekly SC self-administration in PNH/aHUS; exploring SC optionality in neurology as clinical data would likely be requiredConvenient product profile offered at a discount annually relative to SOLIRISAnnual treatment cost per patient vs. SOLIRIS is 10% lower in PNH / ~30% lower in aHUS and future Neurology indications in maintenance phaseLayers of intellectual property protection across indications & geographies Bi-Specific Mini-Body Long-Acting, Small Volume Subcutaneous Dosing Potential for auto-injector or pre-filled syringe On Track for Ph1 Healthy Volunteer Data 1H 2021
PATH FOR STRONG GROWTH On Track For 10 Launches By 2023 1. Commercial estimate 2. Prevalence of ALS-United States, 2015 MMWR Morb Mortal Wkly Rep. 2018 Nov 23; 67(46): 1285-1289 3. Jodele S, Davies SM, Lane A, et al. Diagnostic and risk criteria for HSCT-associated thrombotic microangiopathy: a study in children and young adults. Blood. 2014;124(4):645–653. 4. Aligned with our Phase 3 PREVENT criteria 5.Alexion estimated market opportunity incremental to existing aHUS market 6. Saito T, Arimura K, No M. Result report of the National Epidemiology Survey secondary questionnaire survey on Guillain-Barré syndrome, Ministry of Health, Labour and Welfare specific disease, Immunologic neurological disease investigation sub-group Year 2000 Research Report, 2000;83-84. 7. Quock, T. P., et al. Epidemiology of AL amyloidosis: a real-world study using US claims data. Blood Adv. 2018; 2(10):1046-1053 8. Eidos Therapeutics 9. Poujois, A., et al. Characteristics and prevalence of Wilson's disease: A 2013 observational population-based study in France. Clin Res Hepatol Gastroenterol. 2018 Feb;42(1):57-6 10. Risitano AM, et al. Blood.2009;113(17):4094-4100 LEAD AND EXPAND IN COMPLEMENT GBSJapan Only gMG ALS NMOSD HSCT-TMA CM-TMA AL Amyloidosis Wilson Disease ATTR-CMJapan Only PNH with EVH ALXN1840 ALXN2040 PNH / aHUS WEEKLY SC DIVERSIFY INTO NEW GROWTH AREAS Best in Class C5 InhibitorULTOMIRIS Ph3 Trial>80% Enrolled Best in ClassC5 InhibitorULTOMIRIS First in Class C5 InhibitorULTOMIRIS U.S. Diagnosed Population2 U.S. Target Population4 U.S. Diagnosed Population3 Ph3 TrialEnrollmentUnderway First and OnlyC5 InhibitorULTOMIRIS Ph3 TrialTo Initiate 1H ‘21 ~2K Addt U.S. Opportunity5 <2K JP Diagnosed Population6 First and Only C5 InhibitorSOLIRIS Best in Class C5 InhibitorULTOMIRIS Ph3 TrialTo Initiate 1H ‘21 ~10K U.S. Diagnosed Population7 <6K JP Diagnosed Population8 ~5K U.S. Diagnosed Population9 First and OnlyTargeted Therapy Ph3 TrialInitiated3Q ‘20 First and OnlySmall Molecule Ph3 TrialEnrollment Underway Potential Superiority vs Standard of Care Ph3 TrialOn-Going; Data 1H ‘21 Address PatientsSuffering from EVH Ph3 TrialEnrollmentUnderway Ph3 TrialTo File 3Q ‘21 First in Class SC InfusionC5 Inhibitor <6K EachU.S. Ultra-Rare Population U.S. Target Population1 <10% U.S. PNH Population10 ~20K ~15K ~5K ~4.5K Ph3 TrialEnrollment Complete NEW NEW Ph3 Trial>50% Enrolled NEW NEW NEW NEW
MOMENTUM CONTINUES IN 2021 Advancing Shared Mission to Deliver Life-Changing Medicines PROPOSED ASTRA ZENECA ACQUISITION OF ALEXION EXPECTED TO CLOSE IN 3Q 2021 LEAD IN COMPLEMENT EXPAND IN COMPLEMENT DIVERSIFY Into New Growth Areas 2020 2021 Establish ULTOMIRIS as standard of careContinue to innovate for patientsDevelop and launch next generation C5 >70% PNH ULTOMIRIS converted in US, DE, JPULTOMIRIS 100mg/mL approval (US & EU)ALXN1720 Ph1 continued to enroll >70% aHUS ULTOMIRIS converted in US (2H)ULTOMIRIS once-weekly SC filing (3Q)ALXN1720 Ph1 top line data (1H) Expand presence in Neurology Focus new ULTOMIRIS expansion on direct to Ph3 and rapid proof of concept studies Expand rare disease focus with novel assetsGrow acute care presence with ANDEXXA 4x US Neuro ambition set: >700 new patientsgMG Ph3 ULTOMIRIS enrollment completeNMOSD Ph3 ULTOMIRIS enrollment >80%ALS Ph3 ULTOMIRIS trial initiated; >50% enrolled gMG Ph3 ULTOMIRIS top line data (2H)gMG ULTOMIRIS filing (2H)NMOSD & ALS Ph3 ULTOMIRIS full enrollment (2H)ULTOMIRIS Nephrology1 enrollment progress (FY) Ph3 ALXN1840 fully enrolledPh3 CAEL-101 trial initiatedPTLA acquisition closed Ph3 ALXN1840 top line data (1H)ALXN1840 filing in Wilson Disease (2H)Ph2 ALXN2040 Geographic Atrophy initiation (2H)ANDEXXA growth (FY) 1 Refers to ULTOMIRIS HSCT-TMA and CM-TMA Ph3 and Renal Basket Ph2 Trials
Our Mission: Transform the lives of people affected by rare diseases and devastating conditions by continuously innovating and creating meaningful value in all we do Jesse living with gMG Thank You Committed to Corporate Social Responsibility Diversity, Inclusion, & Belonging At AlexionAt Alexion, Diversity is having a seat at the table. Inclusion is having a voice. Belonging is having that voice be heard. Our Commitment: The MassBio CEO Pledge for a More Equitable and Inclusive Life Sciences Industry Serve Communities And Sustain Our PlanetTransform Patient LivesAdvance Our People And Our CompanyRedefine What It Means To Live With A Rare Disease Ethics & Compliance: Our Foundation
Investor dayAPPENDIX 0 | Jesse living with gMG
Rare Disease By The Numbers RARE DISEASES AFFECT APPENDIX 30MILLION OF ALL RAREDISEASES 5% PEOPLE IN THE UNITED STATES ARE AFFECTED BY RARE DISEASETHAT IS APPROXIMATELY APPROVEDTREATMENTS ARE AVAILABLE FOR ONLY Source: GAO Orphan Drugs November 2016; Global Genes 1 IN 10 7,000RARE DISEASES ARE KNOWN TO EXIST TODAY 50% OF RARE DISEASE PATIENTS IN THE U.S. ARE CHILDREN BY DEFINITION A RARE DISEASE AFFECTS PATIENTS IN THE U.S. <200,000 Donnan living with aHUS
Albie living with LAL-D Sumaira living with NMOSD Our Mission: Transform the lives of people affected by rare diseases and devastating conditions by continuously innovating and creating meaningful value in all we do Jesse living with gMG Justice living with aHUS Bunny living with PNH Aira living with HPP
APPENDIX LEAD AND EXPAND IN COMPLEMENT DIVERSIFY INTO NEW GROWTH AREAS Secure and grow our base business Drive new growth opportunities outside C5 LEADEstablish ULTOMIRIS as the new standard of carePNHaHUSNeurology in 2022/2023Develop and launch next-generation innovative C5 formulations EXPANDExpand presence in Neurology Focus new ULTOMIRIS expansion opportunities on direct-to-Phase 3, rapid Proof of Concept DIVERSIFYExecute novel asset development to expand rare disease focusGrow acute care presence with ANDEXXA Our Value Creation Strategy
APPENDIX Q3 2020 YTD Revenue Composition * Through 9/30/2020, as reported 10/29/2020 Geography Product US, DE, and JP >70% of Global Revenues C5 Franchise ~85% of Global Revenues
APPENDIX ULTOMIRIS Conversion Progress Dec 2018U.S. PNH ULTOMIRIS Approval Oct 2019U.S. aHUS ULTOMIRISApproval Jun 2020EU aHUS ULTOMIRISApproval 3Q 2020 Sep 2020JP aHUS ULTOMIRISApproval Jun 2019JP PNH ULTOMIRIS Approval Jul 2019EU PNH ULTOMIRIS Approval aHUS ULTOMIRIS Conversion On Track T0 70% Ambition1 PNH ULTOMIRIS 70% Conversion In Top Three Markets Achieved 1aHUS ambition of 70% of total patients on ULTOMIRIS within 2 years of launch; 2Pending regulatory approval following completion of Phase 3 studies 2H 2022Estimated gMG ULTOMIRISApproval2 1H 2023Estimated NMOSD ULTOMIRISApproval2 Ambition for best-in-class conversion across all indications
APPENDIX ULTOMIRIS Conversion Dynamic: Two Key Considerations Conversion Loading Dose Dynamic Quarter-on-quarter (QoQ) Variability ULTOMIRIS vs. SOLIRIS U.S. Annual Cost Per Patient Maintenance Dosing Year 1: Loading dose + Maintenance Dosing SOLIRIS indication-specific dosing: aHUS, gMG, NMOSD labeled dose higher than PNHDrives indication-specific pricing differences when comparing SOLIRIS vs. ULTOMIRIS pricingULTOMIRIS weight-based dosing ULTOMIRIS every 8 week infusion schedule drives variability in quarterly patient treatment costs Expect quarterly variability to be negligible on year-over-year (YoY) revenue comparisons Note: pricing discounts are approximations, not exact Infusion Timing Drives QoQ Variability Patient Sample 1: Loading dose + 2 Maintenance Infusions Patient Sample 2: Loading dose + 1 Maintenance Infusion Loading dose Maintenance Infusion
Ample Opportunity to Expand C5 Platform Reach APPENDIX 2023 gMG ALS NMOSD HSCT-TMA CM-TMA Best in Class C5 InhibitorULTOMIRIS Ph3 Trial>50% Enrolled Best in ClassC5 InhibitorULTOMIRIS Ph3 TrialEnrollment On-Going First in Class C5 InhibitorULTOMIRIS U.S. Diagnosed Population2 U.S. Target Population4 U.S. Diagnosed Population3 Ph3 TrialInitiated4Q ‘20 First and OnlyC5 InhibitorULTOMIRIS Ph3 TrialTo Initiate 1H ‘21 ~2K Addt U.S. Opportunity5 Best in Class C5 InhibitorULTOMIRIS U.S. Target Population1 ~20K ~15K ~5K ~4.5K Ph3 Trial>90% Enrolled ULTOMIRIS expansion a key component of ambition for 10 launches by 2023 Pursing role of terminal complement inhibition in IgAN and LNUS diagnosed prevalence of ~150K patientsPh2 proof of concept trial initiated 4Q 2020 Renal Basket Evolving gMG portfolio strategy to target the majority of AChR+ gMG patientsUS diagnosed prevalence of 60-80K patientsTo pursue following Ph1 data in 1H 2021 gMG Expanding footprint in neuromuscular and rare autoimmune diseases US diagnosed prevalence of classic DM ~40KTo pursue following Ph1 data in 1H 2021 DM With even broader rare diseases populations in scope for development beyond ALXN1720 ULTOMIRIS
Diversifying Beyond C5 APPENDIX 2023 Opportunities to diversify broaden ambition for 10 launches by 2023 Once-weekly SC FcRn supports gMG portfolio strategyPh2 trial to initiate 2H 2021 gMG Systemic, oral approach to slow disease progressionPh2 trial to initiate 2H 2021 with ALXN2040 GA With innovative platforms and novel assets continuing to diversify portfolio long-term Factor D ALXN1830 AL Amyloidosis Wilson Disease ATTR-CMJapan Only PNH with EVH ~10K U.S. Diagnosed Population7 <6K JP Diagnosed Population8 ~5K U.S. Diagnosed Population9 First and OnlyTargeted Therapy Ph3 TrialInitiated3Q ‘20 Best in ClassSmall Molecule Ph3 TrialTo Initiate 4Q ‘20 Potential Superiority vs Standard of Care Ph3 TrialOn-Going; Data 1H ‘21 Address PatientsSuffering from EVH Ph3 TrialTo Initiate 4Q ‘20 <10% U.S. PNH Population10 Once-weekly SC FcRn expands hematology presencePh2 trial to initiate 2H 2021 WAIHA Exploring fD in LN, IgAN, PMN, and C3GPh2 trial to initiate 1H 2021 with ALXN2050 Renal Basket Launch “reboot” and label expansion efforts underwayPh2 Urgent Surgery trial to begin 2H2021 ANDEXXA Novel anti-properdin mini-bodyFirst-in-human studies to begin 1H 2021 ALXN1820 Next-gen asfotase alfa (STRENSIQ)First-in-human studies to begin 1H2021 ALXN1850
APPENDIX Vast Opportunity In FcRn Landscape ALXN1830 Value Proposition SC single doses suggest meaningful IgG-lowering potential prior to study pause due to COVID-19 Preliminary PK/PD modeling suggests 1500mg weekly SC may have the potential to provide >70% IgG loweringDosing would be compatible with convenient SC delivery via on-body device Rapid onset of action and sustained IgG lowering after a single doseExcellent PK/PD profile for indications of interest with >70% IgG lowering expected and high specificity to IgGReduces IgG immunocomplexes levelsSuperior dosing profile with once weekly subcutaneous administrationFavorable safety profile to date: No effect on albumin, eliminating concerns of hypoalbuminemiaNo headache seen thus far in SC HVPotential for combination therapy with Alexion’s complement mini-bodies including ALXN1720 and ALXN1820 Positive Early Signal from SC Phase 1 Study HV Ph1 WAIHA 1H 2021 ALXN1830 SC ALXN1830 SC 2H 2021 Ph2 SAD/MAD gMG ALXN1830 SC Ph2 FcRn Has Potential to Treat Hundreds of Thousands of Patients with IgG Mediated Diseases Including gMG, WAIHA, CIDP etc
APPENDIX ULTOMIRISNew IndicationsALS (2023)HSCT-TMA (2023)CM-TMA (2023) $500M to $1B $1.0B+Combined $500M to $2.0B+ ALXN1840Wilson Disease (2022) CAEL-1011AL Amyloidosis (2023)AG10ATTR-CM (2023) Japan Only ALXN1830WAIHA (2023+)gMG (2023+) Factor D2PNH (2023+)GA (2023+)Renal (2023+) LEAD and EXPAND in complement DIVERSIFY into new growth areas (sourced through BD) $1.0B+ ANDEXXAFactor Xa (2020)Urgent Surgery (2023+) SOLIRISGBS (2023) Japan Only ALXN1720DM (2023+)gMG (2023+) $1.0B+ <$100M $2 to $3B $1 to $4B Illustrative only; timing shown represents launch year; based on non-adjusted peak revenue estimates for incremental market opportunity; 1Structured as an option to acquire Caelum; 2Factor D represents both ALXN2040 and ALXN2050 Development-Stage Pipeline with >$10B+ in Potential Peak Sales 7 BlockbusterFranchises
APPENDIX Near-Term Events Support Alexion’s Value Creation Strategy LEAD EXPAND DIVERSIFY US IPR Settlement (SOLIRIS Patents) ULTOMIRIS PNH Subcutaneous Ph3 Top Line Results (PK) 2Q 2020 2Q 2020 ULTOMIRIS aHUS EMA Approval by EC Mid 2020 ULTOMIRIS 100mg/ml Formulation FDA & EMA Approval 2H 2020 4Q 2020 ULTOMIRIS Ph2 Renal Basket Trial Initiation ALXN2040 C3G Ph2 Top Line Results Mid 2020 Portola Acquisition Close 3Q 2020 CAEL-101 Ph3 Trial Initiation 2H 2020 ALXN2060 (AG10) Japan Ph3 Initiation 4Q 2020 ALXN1840 Wilson Ph3 Top Line Results 1H 2021 4Q 2020 ULTOMIRIS HSCT-TMA Ph3 Trial Initiation 1H 2021 ULTOMIRIS COVID-19 Ph3 Interim Results ULTOMIRIS Subcutaneous PNH/aHUS Launch Mid 2022 2H 2022 ULTOMIRIS ALS Ph3 Top Line Results 2H 2021 ULTOMIRIS gMG Ph3 Top Line Results 2H 2022 ULTOMIRIS NMOSD Ph3 Top Line Results ALXN2050 PNH Ph2 Top Line Results 2H 2021 ALXN1840 Wilson Launch 2H 2022 ALXN2040 GA Ph2 Initiation 2H 2021 ALXN2060 (AG10) Japan Ph3 Top Line Results 2H 2022 ALXN1850 IND Filing 2H 2020 ALXN1820 IND Filing 2H 2020 2H 2022 ULTOMIRIS gMG FDA Approval
APPENDIX CSR and ESG at Alexion COMMITTED TO CONTINUING ELEVATION OF CSR REPORTING IN 2021 (Alexion’s Inaugural CSR Report Published in 2020) Download Alexion’s Inaugural 2019 CSR Report at csr.alexion.com “At Alexion, we work to change lives for the better – ours, people living with rare diseases and the communities we serve – and our commitment to being a responsible corporate citizen helps make it possible.”CEO LUDWIG HANTSON Recognition 1st Decile Rank(Pharmaceuticals & Biotech) Double Digit Growth #1 ESG Risk Rating(Biotech) Ranked161 out of 400 (Top 40%)
APPENDIX Commercial Portfolio Patent & Orphan Exclusivity Product Region Patent Exclusivity Orphan Exclusivity Data Exclusivity ULTOMIRIS US 2035 PNH 2025aHUS (SC only; filing 3Q 2021) 2030 EU 2035 N/A 2029 Japan 2035 PNH 2029 2029 SOLIRIS US 20271 gMG 2024NMO 2026 2019 EU 20202 aHUS 2023gMG 2027NMO 2029 2018 Japan 2027 gMG 2027NMO 2029 2020 STRENSIQ US 2029 2022 2027 EU 2030 2027 2025 Japan 2028 2025 2025 KANUMA US 2031 2022 2027 EU 2031 2027 2025 Japan 2031 2026 2026 ANDEXXA/ONDEXXYA US 2030 2025 2030 EU 2033 N/A 2029 Japan 2028 N/A 1 Alexion licensed Amgen to commercialize biosimilar eculizumab effective March 1, 2025 (or earlier in certain circumstances). See IPR settlement agreement dated May 28, 20202 The following patents are under appeal which would extend patent to 2027: '834 Method of Use Patent was approved, then subsequently revoked in January 2019. Patent is in effect as Alexion appeals. ‘888 and ‘029 patent applications were rejected, and Alexion has begun the process to appeal these decisions. These patents are not in effect during appeal
APPENDIX: LATE STAGE PIPELINE TRACKER Identifier (Other) Name (INN) MOA ROA Indication Phase Study Start Anticipated Study End SOLIRIS (eculizumab) Anti-C5 Q2W IV Guillain Barre Syndrome Ph3 Initiating 1H ‘21 Not yet disclosed ALXN1210 ULTOMIRIS (ravulizumab) Anti-C5 Q1W SC Paroxsymal Nocturnal Hemoglobinuria (PNH)Atypical Hemolytic Uremic Syndrome (aHUS) Ph3 Initiated 1Q ‘19 TLR 2Q ‘20Filing 3Q ’21 Q8W IV Generalized Myasthenia Gravis (gMG) Ph3 Initiated 1Q ‘19 TLR 2H ‘21 Neuromyelitis Optica Spectrum Disorder (NMOSD) Ph3 Initiated 4Q ‘19 TLR 2H ‘22 Amyotrophic Lateral Sclerosis (ALS) Ph3 Initiated 1Q ‘20 TLR 2H ‘22 Hematopoetic Stem Cell Transplant Thrombotic Microangiopathy (HSCT-TMA) Ph3 Initiated 4Q ’20 Not yet disclosed Complement Mediated Thrombotic Microangiopathy (CM-TMA) Ph3 Initiating 1H ’21 Not yet disclosed Adults with COVID-19 who are hospitalized with severe pneumonia or ARDS Ph3 Initiated 2Q ‘20 TLR 1H ‘21 Renal Basket Study Ph2 Initiated 4Q ’20 Not yet disclosed ALXN1720 N/A Anti-C5 Bi-Specific SC Generalized Myasthenia Gravis (gMG)1 Ph1 HV Reinitiated 3Q ’20 TLR 1H ‘21 Dermatomyositis (DM)1 ALXN1840(WTX-101) (Bis-choline tetrathiomolybdate) Copper chelator Oral Wilson Disease Ph3 Initiated 1Q ’18 TLR 1H ‘21 ALXN1830(SYNT-001) N/A Anti-FcRn SC Warm Autoimmune Hemolytic Anemia (WAIHA)2 Ph1 HV Reinitiating 1H ‘21 TLR 1H ‘21 Generalized Myasthenia Gravis (gMG)2 CAEL-101 N/A AL/AL fibril reactive antibody IV Amyloid Light-Chain (AL) Amyloidosis Ph3 Initiated 3Q ’20 TLR 2H ‘22 ALXN2060 (AG10) (acoramidis) TTR tetramers stabilizer (small molecule) Oral Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Ph3 Initiated 4Q ’20 TLR 2H ‘22 ALXN2040(ACH-4471) (danicopan) Factor D inhibitor (small molecule) TID Oral PNH with Extravascular Hemolysis (PNH w/ EVH) Ph3 Initiated 4Q ’20 TLR 2H ‘22 TBD Geographic Atrophy Ph2 Initiating 2H ’21 Not yet disclosed ALXN2050(ACH-5228) (vermicopan) Factor D inhibitor (small molecule) BID Oral Paroxsymal Nocturnal Hemoglobinuria (PNH) Ph2 Initiated 4Q ’19 TLR 2H ‘21 Renal Basket Study Ph2 Initiating 1H ’21 Not yet disclosed ALXN2070 ANDEXXA (andexanet alfa) Factor Xa Reversal IV Urgent Surgery Ph2 Initiating 1H ’21 Not yet disclosed ALXN2075 (cerdulatinib) SYK/JAK kinase inhibitor Oral Lymphoma (CTCL, PTCL, FL) Ph2 PTLA Acquisition TLR 1H ‘21 ALXN1820 N/A Anti-Properdin Mini-Body SC Not yet disclosed Ph1 Initiating 1Q ‘21 Not yet disclosed ALXN1850 N/A Next generation alfotase alfa SC Not yet disclosed Ph1 Initiating 2Q ‘21 Not yet disclosed 11720 currently in HV Ph1 with topline readout estimated 1H ‘21 and subsequent DM and gMG trials to begin after that; 21830 Ph1 HV program to reinitiate for SC formulation with WAIHA and gMG Ph2 programs to follow in 2021
APPENDIX Alexion Current Indications Indication Description Links PNH Paroxysmal Nocturnal Hemoglobinuria Chronic, debilitating, and potentially life-threatening ultra-rare blood disorder, with an average age of onset in the early 30s more info aHUS atypical Hemolytic Uremic Syndrome Ultra-rare, genetic, chronic, potentially life-threatening disease. Chronic uncontrolled complement activation results in thrombotic microangiopathy (TMA) more info gMG Generalized Myasthenia Gravis Debilitating, chronic, and progressive autoimmune neuromuscular disease. more info NMOSD Neuromyelitis Optica Spectrum Disorder Rare, devastating, complement-mediated disorder of the central nervous system characterized by relapses where each individual attack results in cumulative disability including blindness and paralysis, and sometimes premature death (primarily affects women) more info HPP Hypophosphatemia Inherited, progressive, ultra-rare metabolic disease in which patients experience devastating effects on multiple systems of the body, and face debilitating or life-threatening complications more info LAL-D Liposomal Acid Lipase Deficiency Genetic, chronic, and progressive ultra-rare metabolic disease in which infants, children, and adults experience continuous, uncontrolled accumulation of cholesteryl esters (CEs) and triglycerides (TGs) that may lead to multi-organ damage and premature death more info ANDEXXA Coagulation factor Xa reversal (recombinant) Reversal agent for life-threatening bleeds induced by factor Xa inhibitors more info
APPENDIX Alexion Pipeline Indications - I Indication Description Links WD Wilson Disease Rare, chronic, genetic, and potentially life-threatening liver disorder of impaired copper transport. The disorder is characterized by build-up of intra-cellular hepatic copper. Untreated, Wilson disease leads to various combinations and severity of hepatic, neurologic, and psychiatric symptoms, and can be fatal. ALA AL (Light-chain) Amyloidosis A protein misfolding disorder in which B-cells produce incomplete and light chain antibodies which clump in certain organs / tissues (including heart, lungs, kidneys, nervous system, and liver, eventually causing organ damage and death. more info PNH-EVH Paroxysmal Nocturnal Hemoglobinuria with Extravascular Hemolysis Chronic, debilitating, and potentially life-threatening ultra-rare blood disorder, with an average age of onset in the early 30s. EVH occurs when C3 opsonization of red blood cells causes macrophages to destroy those cells in tissue. DM Dermatomyositis Progressive autoimmune condition that causes skin changes and muscle weakness. Symptoms can include a red skin rash around the eyelids, red bumps around the joints, and muscle weakness in the arms and legs. Dermatomyositis is most common in adults between ages 40 and 60, or in children between ages 5 and 15. more info HSCT-TMA Hematopoetic Stem Cell Transplant Thrombotic Micro-Angiopathy A significant and often lethal complication of HSCT. The condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, graft versus host disease (GVHD), and other factors associated with HSCT. HSCT-TMA prognosis is poor, with overall mortality reported as high as ~80-90%.
APPENDIX Alexion Pipeline Indications - II Indication Description Links CM-TMA Complement-Mediated Thrombotic Micro-Angiopathy Caused by abnormalities of regulation of the alternative pathway of complement activation. The indication describes a group of severe and chronic ultra-rare diseases that can cause progressive injury to vital organs— via damage to the walls of blood vessels and blood clots—potentially leading to organ failure and premature death. CM-TMA affects both adults and children and represents the population of patients with aHUS with or without triggers. COVID-19 Severe Acute Respiratory Distress Syndrome in COVID-19 patients Patients with severe illness include those who are hospitalized with severe pneumonia or acute respiratory distress syndrome. Evidence suggests that acute lung injury associated with COVID-19 may be mediated in part by complement pathway whereby elevated C5 ultimately leads to severe pneumonia, blood clots and multi-organ dysfunction in many advanced COVID patients. WAIHA Warm Auto-Immune Hemolytic Anemia Rare autoimmune disorder caused by pathogenic Immunoglobulin G (IgG) antibodies that react with and cause the premature destruction of red blood cells at normal body temperature. The disease is often characterized by profound, and potentially life-threatening anemia and other acute complications. ATTR-CM Transthyretin Amyloidosis (ATTR) with Cardiomyopathy (ATTR-CM) A progressive, fatal disease caused by the accumulation of misfolded tetrameric transthyretin (TTR) amyloid in the heart. Caused by the destabilization of TTR due to inherited mutations or aging, symptoms usually manifest later in life (age 50+), with median survival of three to five years from diagnosis.
APPENDIX Alexion Pipeline Indications - III Indication Description Links LN Lupus Nephritis An inflammatory renal disease that is a severe complication of systemic lupus erythematosus (SLE), in which deposits of immune complexes (e.g., IgG and complement) accumulate in the kidney and lead to injury. Approximately 30% SLE patients develop LN, and up to 30% of patients are refractory to treatment and progress to end stage renal disease requiring dialysis/transplant within 15 years . There are no FDA approved therapies for LN. PMN Primary Membranous Nephropathy Rare autoimmune disease characterized by autoantibodies to the podocyte membrane antigens PLA2R (~85%) and THSD7A (~5%) that causes nephrotic syndrome and chronic kidney disease. Approximately 30% of patients will progress to end stage renal disease within 10 years of diagnosis. IgAN IgA Nephropathy (IgAN) A heterogenous disease in terms of clinical manifestations and progression and is the most common cause of primary glomerulonephritis. In IgAN, locally deposited immune complexes lead to activation of the complement cascade & downstream endothelial organ damage. The Lectin and Alternative Pathways are believed to be the main driver of disease progression, which includes end stage renal disease and need for dialysis or transplant. C3G Complement 3 Glomerulopathy Ultra-rare, heterogenous renal disease characterized by uncontrolled continued activation of fluid and/or solid phase alternative pathway causing C3 deposition and inflammation, leading to kidney damage . ALS Amyotrophic lateral sclerosis A rare neurological disorder of progressive deterioration of nerve cells (motor neurons) in the brain and the spinal cord that control muscles throughout the body. Loss of motor neurons and muscle strength leads to loss of independence, paralysis and death, typically due to respiratory insufficiency.
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