Third Quarter 2019

Third Quarter 2019E A R N I N G S C A L L

O C T O B E R 2 3 , 2 0 1 9

2 | R A R E I N S P I R A T I O N . C H A N G I N G L I V E S .

T H I R D Q U A R T E R 2 0 1 9 E A R N I N G S C A L L

Introduction

CEO Opening Remarks

Financial Update

Available for Q&A

Susan Altschuller, Ph.D., Vice President, Investor Relations

Ludwig Hantson, Ph.D., Chief Executive Officer

Paul Clancy, CFO

John Orloff, M.D., Head of R&D

Aradhana Sarin, M.D., Incoming CFO

R&D Highlights

Brian Goff, Chief Commercial OfficerCommercial Highlights

CEO Closing Remarks Ludwig Hantson, Ph.D., Chief Executive Officer

Provided October 23, 2019, as part of an oral presentation and is qualified by such, contains forward-looking statements, actual results may vary materially; Alexion disclaims any duty to update.

A G E N D A


F O R W A R D L O O K I N G S T AT E M E N T S

This presentation contains forward-looking statements, including statements related to: guidance regarding anticipated financial results for 2019 (and all of the assumptions and estimates related to such guidance); Company plans to build out the product pipeline for long term value creation; 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 certain clinical trials andstudies the opportunity to treat atypical HUS patients with Ultomiris; the Company’s ambition and expectations regarding conversion of patients to Ultomiris in the future for PNH and aHUS (and other indications once approved) in the US and other countries; plans to make future regulatory filings for approval of certain products and product candidates, including Ultomiris (ALXN1210/ravulizumab-cvwz), ALXN1840, CAEL 101, AG10, elamipretide, anti-FcRN portfolio and Factor D programs, and the expected timing of such filings as well as the expected timing of the receipt of certain regulatory approvals to market a product; expectation that neurology will be the largest patient franchise in the US by year end; ALXN1840 can be a transformative therapy to patients; potential benefits (and transformative impact) of current products and products under development and in clinical trials (including further extended dosing intervals), including elamipretide has the potential to become a life-changing treatment for mitochondrial-driven disease; 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 certain clinical trials and studies; the goal of building out the clinical pipeline and advancing development programs; business development will be a focus in the future and expect to do additional transactions (and Company will look to build-out core therapeutic areas and expand into others); goal to grow the metabolic portfolio; the anticipated timing and successful completion of the Achillion acquisition; Company expects high single digit volume growth from the C5 franchise; the Company expects that the vast majority of patients will be treated with Ultomiris before the earliest potential biosimilar launch; timing for R&D expenses and increase in R&D spend; the Company is building durable, blockbuster franchises in PNH/aHUS, metabolics, neurology and FcRn (and plans to expand and strengthen these franchises and expand into other areas of rare disease); the timing and expected benefits of the Company’s key late-stage pipeline programs for 2019, 2020 and beyond; Alexion’s goal is to develop treatments for ALS, PPMS; the Company’s ambition to maintain a relatively narrow pricing band globally for Ultomiris; potential additional country launches for Ultomiris; Company’s aspiration is to facilitate a rapid, best in class conversion to Ultomiris in each launch geography; Company is optimizing the ALXN1830 profile to address evolving gMG competitive landscape; neurology represents a significant growth opportunity; goal is to expand treatment options for gMG patients with Ultomiris and ALXN1830 (and the portfolio approach will help to serve patients across the spectrum of the disease); the steps to be taken to leverage and strengthen neurology presence with NMOSD launch; Company continues to identify new patients for Kanuma and Strensiq (and plans to seek reimbursement agreements in additional geographies); Company’s opportunities for growth (including neurology and late-stage pipeline programs); the Company’s strategy to deliver long-term growth; potential transformative impact of certain products and pipeline products; plans to expand patient optionality with C5 portfolio; and anticipated developments and advancements in the products in the Company’s pipeline (including subcutaneous formulations of certain products) and for new products to enter the product 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: our dependence on sales from our principal product (SOLIRIS); our ability to facilitate the timely conversion of PNH and aHUS patients (and any future indications) from SOLIRIS to 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); decisions of regulatory authorities regarding the adequacy of ourresearch, 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 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 (or anticipated adoption rates are not realized); 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; failure by regulatory authorities to approve transactions; 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, including investigations of Alexion by the U.S. Securities and Exchange Commission (SEC) and U.S. Department of Justice; the risk that estimates regarding the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D and other future indications we are pursuing are inaccurate; the risks of changing foreign exchange rates; risks relating to the potential effects of the Company's restructuring; risks related to the acquisition of companies and co-development and collaboration efforts; and a variety of other risks set forth from time to time in Alexion's filings with the SEC, including but not limited to the risks discussed in Alexion's Quarterly Report on Form 10-Q for the period ended June 30, 2019 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 presentation 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. The non-GAAP results 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 collaborations, acquired in-process research and development assets, impairment of intangible assets, change in value of strategic equity investments, litigation charges, gain or loss on sale of a business or asset 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 2019 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, 2019 and 2018 and projected twelve months ending December 31, 2019.

Amounts may not foot due to rounding.


D I S C L O S U R E S

R A R E I N S P I R A T I O N . C H A N G I N G L I V E S .

CEO Opening RemarksLudwig Hantson, Ph.D.Chief Executive Officer


R E C E N T K E Y A C C O M P L I S H M E N T S


O P E N I N G R E M A R K S

NMOSD EU Approval

Atypical HUSU.S. Approval

ULTOMIRIS ®

PNH patient conversion:

U.S. 51% Germany >45%

Japan >15%

Over 1,500 SOLIRIS® gMGand NMOSD

patients in the U.S.

Note: Entered into definitive agreement to acquire Achillion Pharmaceuticals in October 2019. Closing subject to approval of Achillion shareholders, customary closing conditions and relevant regulatory approvals. Stealth Biotherapeutics transaction is an option to co-develop and commercialize elamipretide for mitochondrial diseases. Eidos transaction is an exclusive license to develop and commercialize AG10 in Japan.


G R E AT P R O G R E S S I N T H E T H I R D Q U A R T E R


ULTOMIRIS® Conversion in PNH & atypical HUS US Launch1 • 51% PNH patients converted in the U.S. ; >45% in Germany, >15% in Japan

• Approved for aHUS in U.S. October 18, launch underway; under review in EU & JP

Accelerate Neurology Portfolio2• SOLIRIS® approved in U.S. & E.U. for NMOSD; Launches on track• SOLIRIS for NMOSD under priority review in Japan• Best quarter yet for SOLIRIS in gMG

Grow our Metabolics Portfolio3 • Continued growth for STRENSIQ® & KANUMA®

• ALXN1840 Phase 3 enrollment on track for completion early 2020

Execute & Expand the Pipeline4• ULTOMIRIS Phase 3 programs underway (gMG, SC for PNH/aHUS)• Preparing for multiple additional late-stage clinical trial initiations in 2019 & 2020 • Executed 3 business development deals (Eidos, Stealth, Achillion)

Deliver on Financial Ambitions5 • 3Q19 Revenue Growth of +23% y/y• 3Q19 Non-GAAP1 EPS up +38% y/y


1 A reconciliation of GAAP to non-GAAP financial results is provided in the appendix and is available at www.alexion.com.

Increasing FY Revenue and EPS Outlook

http://www.alexion.com/


F O C U S O N C O N V E R S I O N , E X P A N S I O N & D I V E R S I F I C AT I O N T O D R I V E P AT I E N T A N D S H A R E H O L D E R V A L U E



Conversion Expansion Diversification

ULTOMIRIS®

Beyond PNH and aHUS;

SC optionality

C5 Optionality

o Weekly SCo gMGo NMOSDo ALSo HSCT-TMAo PPMSo Others to be

announced

o ALXN 1720o ALXN 1810

FcRnALXN1830, ABY-039

CAEL-1011

Eidos: AG10

Stealth2: Elamipretide

Further Disciplined BD

o WAIHA, gMGo Rare autoimmune

o AL Amyloidosis

o ATTR

o PMMo Barth Syndromeo LHONo GA in AMD

o C3Go EVH in PNHo Other complement

Achillion3: Factor D

C5 Franchise

ALXN 1840 o Wilson Disease

KANUMA® o LAL-D

STRENSIQ® o HPP

1. Structured as an option to acquire2. Option to co-develop and commercialize3. Subject to transaction closure


E M B A R K I N G O N A L E X I O N ’ S N E X T C H A P T E R

Over Ten Potential Launches by 2023

2019

2021

2020

2022

2023

SOLIRIS®

NMOSDULTOMIRIS®

PNHULTOMIRIS

aHUS

ULTOMIRIS QW SCPNH, aHUS

ULTOMIRIS IV/SC gMG

ALXN1840Wilson’s Disease

CAEL1012

AL AmyloidosisULTOMIRIS

IV/SC NMOSD

ULTOMIRIS HSCT-TMA

ULTOMIRIS ALS

EIDOS AG10 ATTR-CM(Japan Only)

Hematology

Metabolics

Neurology

Cardiology



Beyond ABY-039Undisclosed

ULTOMIRISPPMS

Elamipretide1

GA in AMD

Elamipretide1

PMMElamipretide1,4

Barth’s

ACH-47713

C3G

ACH-44713

PNH+Clinical EVHNephrology

Elamipretide1

LHONOther/TBD

Pending Closure of Achillion Acquisition

1. Option to co-develop and commercialize2. Structured as an option to acquire3. Subject to transaction closure4. Pending acceptability of existing data package

ACH 52283

PNH, Undisclosed

+ Plans to further expand and diversify pipeline

Significantly Expanded Pipeline Since 2017

Only 3 late-stage development programs in 2017 Led to 3 launches in 2019

ALXN1830WAIHA & gMG


Financial UpdatePaul ClancyChief Financial Officer


T H I R D Q U A R T E R 2 0 1 9 K E Y P E R F O R M A N C E M E T R I C S

Total Revenues

GAAP1

Operating Margin

GAAP1 EPS

$1.263B

42%

$2.08

+23%

+703bps

+41%

3Q19

SOLIRIS® sales grew 12% driven by 11% increase in volume ULTOMIRIS® contributed $90M, primarily driven by patient

conversion from SOLIRIS Metabolic sales grew 32% driven by 32% increase in volume

Driven by topline growth offset by $30M in 3Q19 related to the collaboration agreement with Eidos and the impact of more significant tax benefits recorded during 3Q18 compared to 3Q19

Non-GAAP1 EPS $2.79 +38%

Non-GAAP1

Operating Margin 57% +357bps Primarily driven by top-line growth

Primarily driven by topline growth

Driven by topline growth offset by $30M in 3Q19 related to the collaboration agreement with Eidos

F I N A N C I A L H I G H L I G H T S

1 A reconciliation of GAAP to non-GAAP financial results is provided in the appendix and is available at www.alexion.com.


vs 3Q18



N E T P R O D U C T S A L E S


Net Product Sales by Geography 3Q19 Net Product Sales Analysis

505 563 602 672 696

283290 288

307 302106114 112

124 137132

161 139100 128

3Q18 4Q18 1Q19 2Q19 3Q19

Mill

ions

($)

US Europe APAC ROW

$1,027

$1,027 $1,263

3Q18 Price Volume FX 3Q19

Mill

ions

($) +1%

+23%--

1

3Q18 vs 3Q19

+23% YoY$1,129 $1,140


$1,203 $1,263

1Net of hedging activities


S O L I R I S ® A N D U L T O M I R I S ® N E T P R O D U C T S A L E S


3Q19 Highlights

SOLIRIS Net Product Sales

405 452 464 496 497

262 270 265 280 25698 105 101 110 118123

150 133 94 120

3Q18 4Q18 1Q19 2Q19 3Q19

Mill

ions

($)

US Europe APAC ROW

$888$977 $962 $981 $991

SOLIRIS: +12% YoY revenue growth; +11% YoY volume growth

US, Europe and Japan partially impacted by ULTOMIRIS patient conversion

ULTOMIRIS: Promising facilitated conversion from SOLIRIS since FDA (Dec ‘18), PMDA (Jun ‘19), and EMA (Jul ‘19) approvals

Total C5: +22% YoY revenue growth across PNH, aHUS, gMG and NMOSD


ULTOMIRIS Net Product Sales

2554 65

214

1Q19 2Q19 3Q19

Mill

ions

($)

US Europe APAC ROW

$25

$54

$90

C5 Franchise Net Product Sales

405 452 489 550 562

262 270 265 280 27798 105 101110 122123 150 133 94 120

3Q18 4Q18 1Q19 2Q19 3Q19

Mill

ions

($)

US Europe APAC ROW

$888$977 $988 $1,034 $1,080


M E T A B O L I C S N E T P R O D U C T S A L E S


STRENSIQ®

+36% YoY revenue growth +36% YoY volume growth

KANUMA®

+12% YoY revenue growth +16% YoY volume growth

STRENSIQ® Net Product Sales

87 99 100 106 11817

15 18 2019

79 10

1214

3Q18 4Q18 1Q19 2Q19 3Q19

Mill

ions

($)

US Europe APAC ROW

$113

14 13 14 15 16

5 5 6 7 61 1 11 16 7 3 3 5

3Q18 4Q18 1Q19 2Q19 3Q19

Mill

ions

($)

US Europe APAC ROW

$25 $26

3Q19 Highlights

$126

$26

KANUMA® Net Product Sales

$130

$24


$141$154

$28


3 Q 2 0 1 9 F I N A N C I A L P E R F O R M A N C E

(1) A reconciliation of GAAP to non-GAAP financial results is provided in the appendix and is available at www.alexion.com.


$ Millions, Except EPS GAAP (1) Non-GAAP (1) GAAP (1) Non-GAAP (1) ∆ Non-GAAP (1)

Total Revenue $1,263 $1,263 $1,027 $1,027 +23%

SOLIRIS® Revenue $991 $991 $888 $888 +12%

ULTOMIRIS® Revenue $90 $90 - - -

STRENSIQ® Revenue $154 $154 $113 $113 +36%

KANUMA® Revenue $28 $28 $25 $25 +12%

COGS% of Total Revenue

$958%

$927%

$919%

$879% -123 bps

R&D% of Total Revenue

$23318%

$18615%

$17517%

$16216% -108 bps

SG&A% of Total Revenue

$29924%

$26021%

$25925%

$22522% -125 bps

Operating Income $530 $725 $359 $552 +31%

Operating Margin 42% 57% 35% 54% +357 bps

Effective Tax Rate 13% 11% 3% 14% -265 bps

Earnings Per Share $2.08 $2.79 $1.47 $2.02 +38%

3Q ‘19 3Q ‘18




U P D AT E D F Y 1 9 O U T L O O K

SOLIRIS/ULTOMIRIS: Continued strength in gMG, ULTOMIRIS conversion, and modest contribution from SOLIRIS launch in NMOSD

Metabolics: Strong STRENSIQ® growth

Pricing: Headwind of ~2%

FX: Headwind of ~$40 million

Operating Expenses:

Late-stage clinical development spend expected to accelerate in 2020

Key Assumptions $ Millions, Except EPS Previous Guidance(1)(2)

Updated Guidance(1)(2)

YoY Growth (1)(2)

Total Revenue $4,750 to $4,800 $4,860 to $4,890 +18%

SOLIRIS®/ULTOMIRIS® $4,095 to $4,130 $4,180 to $4,200 +18%

Metabolic $655 to $670 $680 to $690 +21%

R&D (% of Total Revenue)GAAPNon-GAAP

17% to 19%14% to 16%

17% to 18%14% to 15%

-18 bps-114 bps

SG&A (% of Total Revenue)GAAPNon-GAAP

23% to 24%20% to 21%

24% to 25%21% to 22%

-241 bps-158 bps

Operating MarginGAAPNon-GAAP

42% to 43%55% to 56%

41% to 42%55% to 56%

+3,497 bps+275 bps

Earnings Per Share(3)

GAAPNon-GAAP

$8.13 to $8.41$9.65 to $9.85

$8.58 to $8.78$10.25 to $10.40

+2,380%+30%

Mid-point of Guidance: Revenue +18%, Non-GAAP Operating Profit +24%, Non-GAAP EPS +30%

Increasing FY Revenue and EPS Outlook

(1) 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 collaboration agreements, intangible asset impairments, litigation charges, changes in fair value of contingent consideration or restructuring and related activity outside the previously announced activities that may occur after the issuance of this presentation. Updated guidance excludes the financial impact of the recently announced agreement to acquire Achillion as it is anticipated to close in the first half of 2020.(2) A reconciliation of GAAP to non-GAAP financial guidance is provided in the appendix and is available at www.alexion.com. YoY growth uses the mid-point of the guidance range.In October 2019, the Board of Directors approved a new share repurchase authorization of $1 Billion. The repurchase program does not have an expiration date and we are not obligated to acquire a particular number of shares.





R&D HighlightsJohn Orloff, M.D.Head of R&D


PreclinEotaxin-1 Inhibitor (Immune Pharma)4

A L E X I O N D E V E L O P M E N T P O R T F O L I O


R & D H I G H L I G H T S

Next Gen HPP Treatment

Initiate POC Study 2H19

PreclinZealand Pharma A/S

Initiate POC Study 2H19

Initiated Phase 3 2H18Initiating Phase 3 1Q19Initiate POC Study 2H19

Preclin dev’t

Ongoing Phase 3 Trial

Preclin

Ongoing Phase 1 Trial

Ongoing Phase 1b/2a in PV/PF

ULTOMIRIS IV (ALS)

ULTOMIRIS SC QWULTOMIRIS IV (gMG)ULTOMIRIS IV (NMOSD)

CAEL-1011 (AL Amyloidosis)

ALXN1840 (WTX101)

GalXC™ Collaboration

ABY-039 (Affibody - FcRn)

ALXN1830 (WAIHA - FcRn)ALXN1830 SC (gMG - FcRn)

CP010 (Complement Pharma)

ULTOMIRIS IV (PPMS) Initiate POC Study 2H19

Preclin dev’t

Ongoing Phase 1b/2a in PV/PF

Preclin dev’tALXN1720 (Anti-C5 Bi-specific)

ALXN1810 SC

ULTOMIRIS IV (HSCT-TMA)

Multiple Internal Programs

PRECLINICAL PHASE 1 PHASE 3

Elamipretide2 (PMM)Elamipretide2 (Barth Syndrome)

Preclin dev’tElamipretide2 (LHON)

Preclin dev’tDanicopan3 (PNH with EVH)Preclin dev’tDanicopan3 (C3G)Preclin dev’tACH-5228 (PNH)3

PreclinAchillion Next Gen Factor D3

Ongoing Phase 1 TrialAG10 (ATTR) Japan

Preclin dev’tElamipretide2 (GA in AMD)

Preclin dev’tACH-5228 (Additional Indications)3

ULTOMIRIS ®

ALXN1840

ALXN1830

ABY-039ALXN1810CAEL-101

AG10

Elamipretide

Danicopan

ACH-5228

ALXN1720

Preclinical

Pending Transaction Close

22 development programs planned across 11 assets

Hematology Metabolics NeurologyNephrology Other/TBD Cardiology

PHASE 2

1. Structured as an option to acquire2. Option to co-develop and commercialize3. Subject to transaction closure4.Subject to completion of bankruptcy proceedings

Pending Option Exercise

Planned 4Q19

Planned 1H20

Planned 2H20Planned 1H20

Planned 1Q20

Planned 2H20

Planned 1H20

Planned 1H20

Planned 1H20

Planned 1H20

Planned 2020

Planned 2020


AG10

K E Y P I P E L I N E P R O G R A M S P L A N N E D F O R 2 0 1 9 & 2 0 2 0



• AL Amyloidosis - Plan to initiate CAEL-101 Registrational Phase 2/3 1H2020

• SC (PNH, aHUS) - Initiated QW SC Phase 3 in 2019

• gMG - Initiated Q8W IV Phase 3 in 2019 • Planned SC bridging

• NMOSD - Plan to initiate Q8W IV Phase 3 in 4Q2019• Planned SC bridging

• ALS - Plan to initiate registrational Phase 2/3 trial 1H2020• Protocol being reviewed & finalized with regulators

• Wilson Disease - Ongoing Phase 3 superiority trial • Anticipate enrollment completion early 2020; TLR 1H2021

• WAIHA - Plan to initiate Phase 2 study in 1Q2020

• HSCT-TMA - Plan to initiate Phase 3 trial 1H2020• Severe patient population with limited treatment options

CAEL-101

ALXN1840

FcRn• gMG - Plan to initiate Phase 2 SC trial in 2H20 after SC

Healthy Volunteer study completes

Hematology Metabolics Neurology Cardiology

• ATTR - Plan to initiate Japan Phase 3 1H2020 pending regulatory discussion

Nephrology

• PMM – Phase 3 data expected in 1Q2020

• Barth Syndrome – FDA meeting in 4Q2019 to discuss NDA submission

• LHON – Phase 2 complete; Plan to initiate Phase 3 early 2020

Other/TBD

• GA in AMD – Completion of Phase 2 enrollment in 2019

ACH-4471• PNH with Clinical EVH – Phase 2 topline results in 4Q2019

• C3G – Phase 2 study completion in 2020

• PNH – Plan to initiate Phase 2 study in 2020 pending regulatory feedback

ACH-5228



Elamipretide

• Complement-Mediated – Plans for potential additional indications in 2020


• Autoimmune - SAD/MAD data expected in 1H20

ALXN1830

ABY-039


AG10Japan Addressable Patients

S I G N I F I C A N T O P P O R T U N I T Y F O R D I V E R S I F I C AT I O N F R O M L AT E - S T A G E P I P E L I N E



gMG NMOSDHSCT-TMA

CAEL-101ALXN1840FcRn

ALXN1830 & ABY-039

ACH-4471 ACH-5228


Elamipretide

SC PNH / aHUS


aHUSALS TBD C3G

PNH with Clinical

EVHPNH TBD

Wilson’s Disease

ALAmyloidosis WAIHA gMG TBD ATTRGA in

AMDLHONBarth SyndromePMM

Estimated Addressable Patients in the U.S. by Late-Stage PipelineMultiple rare opportunities

Multiple rare opportunities

Multiple rare opportunities

~1,000,000

Illustrative

Moderate/ Severe

Mild/ Moderate

Ultra-rare (<6k)

Rare(6k-50k)

Rare(50k+)

Moderate

Severe

Moderate/ Severe

Mild/ Moderate

Ultra-rare (<6k)

Rare(6k-50k)

Rare(50k+)


A L E X I O N T O A C Q U I R E A C H I L L I O N

S T R A T E G I C R A T I O N A L E

Further builds diversified pipeline with potential to treat numerous additional complement-mediated diseases

Potential in Numerous Rare Diseases

Factor D is a critical control point for the complement system’s alternative pathway (AP)

• Implicated in numerous rare diseases with significant unmet needs; platform for pipeline expansion

• Inhibits AP but leaves Lectin and Classical pathways to fight infection• Ability to target with oral small molecules – leverages Achillion small

molecule chemistry expertise and library

Acquisition adds two clinical stage oral Factor D inhibitors to Alexion pipeline

• Danicopan (ACH-4471) in Phase 2 (TID) for:• Add-on for PNH patients with clinical EVH • C3 glomerulopathy (C3G)

• ACH-5228 completed successful Phase 1; Potential best-in-class oral Factor D inhibitor (BID) for PNH and numerous additional complement-mediated diseases

• IP through at least mid-2030s

Aligned with disciplined BD strategy• Initial consideration of ~$930M or ~$700M net with ~$230M* cash on

Achillion’s balance sheet• Potential for additional financial benefit from NOLs

Strategic Rationale

*Cash and marketable securities as of September 30th, 2019; Actual amount will be determined as of the transaction closeSubject to transaction close

Source: Achillion

Gastroenterology• Non-alcoholic

steatohepatitis (NASH)• Fatty liver disease• Cirrhosis

NeurologyOphthalmology• AMD• Diabetic retinopathy• Geographic atrophy

Dermatology

Pulmonology

Hematology• PNH with EVH – in Ph 2• HUS• aHUS

Nephrology• C3G – in Ph 2• IC-MPGN



O P T I O N T O C O - D E V E L O P A N D C O M M E R C I A L I Z E E L A M I P R E T I D E


P I P E L I N E

Primary Mitochondrial Myopathy (PMM)

• Lead indication PMM: Mitochondrial disorder characterized by debilitating muscle weakness, chronic fatigue, and exercise intolerance. Caused by mitochondrial dysfunction resulting from cardiolipin peroxidation

• No currently approved treatments for PMM. Standard of care is supportive, largely managed by neurologists / neuromuscular specialists

Elamipretide

• Small molecule that binds to and stabilizes cardiolipin, improving mitochondrial function

• Daily subcutaneous auto-injector

• Phase 2 MMPOWER2 showed improvement in 6 minute-walk test among ‘low walker’ patients

• Phase 3 MMPOWER3 Trial underway, top line results expected Q1 2020

Additional Indications

• Barth’s Syndrome: Ultra-rare genetic mitochondrial disease with high infant mortality and no approved treatments. Completed Phase 2/3 development

• Leber’s Hereditary Optic Neuropathy (LHON): Mitochondrial disease leading to vision loss. Phase 2 trial complete and Phase 3 planning underway

• Geographic atrophy in AMD: Devastating complication of age-related macular degeneration. Ongoing Phase 2 trial

ALXN option to co-develop and commercialize Elamipretide for mitochondrial diseases


A G 1 0 D E V E L O P E D T O S T A B I L I Z E T T R I N AT T R P AT I E N T S



Transthyretin (TTR) Amyloidosis AG10

Small molecule designed to treat the root cause of ATTR by binding and stabilizing TTR in the blood

• Eidos currently evaluating AG10 in a Phase 3 study in the US and Europe for ATTR cardiomyopathy (N=510)

• Demonstrated greater than 90% average TTR stabilization at Day 28 in Phase 2 study

• AG10 is the only TTR stabilizer in development that has been observed to mimic the stabilizing structure of the rescue mutation

Initiating Japan Phase 3 Early 2020 Pending

Regulatory Discussion

Rare, fatal cardiovascular and neurodegenerative disease

• Dissociated and misfolded TTR proteins that aggregate and deposit in tissues forming amyloid fibrils

• Survival in untreated patients ~3 years post-diagnosis

• ~100k patients in the US, ~400k worldwide• Additional ~40k with Mutant-type TTR Amyloidosis

ALXN agreement for exclusive license to develop and commercialize AG10 in Japan


Commercial UpdateBrian GoffChief Commercial Officer


Japan Launch Update

U L T O M I R I S ® P N H O N T R A C K F O R B E S T - I N - C L A S S C O N V E R S I O N


C O M M E R C I A L H I G H L I G H T S

Launch reach moving beyond centers of excellence and into broader treater base

Permanent J-Code in place as of Oct. 1st

Loading dose impacts quarter of ULTOMIRIS initiation, maintenance dosing thereafter

U.S. Launch Update EU Launch Update

Strong conversion continues in Germany with over 45% conversion as of Oct. 21st

Now launched in Germany, Austria, Denmark, and Finland

Reimbursement planning & discussions in other countries ongoing with launches planned for 2020+

Ambition for best-in-class 70% conversion two years from launch

Japanese approval June 18, 2019 Reimbursement secured and launch

kicked off in September 2019 Strong conversion in Japan with >15%

conversion as of Oct. 21st

Similar best-in-class conversion ambition

Launch off to promising start, tracking above early progress in the U.S.

Encouraging feedback across all stakeholders

3%

8%

14%

22%

38%

47%51%


U L T O M I R I S ® A P P R O V E D F O R AT Y P I C A L H U S I N T H E U S


U.S. Approval October 18thPotential EU approval 1H2020

Best in class conversion ambition of stable aHUS patients Positive feedback thus far from patients and payers Pending regulatory review, EU launch expected in first half of

2020 Atypical HUS-specific dynamics:

– Duration of therapy is shorter than in PNH and varies depending on etiology; Opportunity for ULTOMIRIS to potentially improve persistency

– Newly diagnosed patients generally present in acute setting– Maintenance dosing ~33% discount to SOLIRIS® annually

Leverage PNH Launch Experience To Drive Rapid aHUS Conversion

U.S. Launch & Facilitated Conversion Underway



G O A L T O E S T A B L I S H U L T O M I R I S ® A S N E W S T A N D A R D O F C A R E

Provided October 23, 2019, as part of an oral presentation and is qualified by such, contains forward-looking statements, actual results may vary materially; Alexion disclaims any duty to update. *Alexion internal estimate


PNH

aHUS

gMG

2019 2020 2021 2022 2023 2024 2025 2026 2027US Conversion Ambition

70% conversion by mid-year 2020

70% conversion of stable patients by late 2021

70% conversion by late 2024

NMOSD70% conversion by late 2024

IllustrativeEarliest possible biosimilar launch*

ALXN 1210 SC launch ambition

Orphan drug designation We have ample time to convert patients to ULTOMIRIS before a potential SOLIRIS® biosimilar launch


43

194

375

560

788

968

1,193

1,530

S O L I R I S ® N E U R O L O G Y E X P A N S I O N C O N T I N U E S



SOLIRIS Neurology Patients in U.S. gMG

NMOSD

Neurology expected to be the largest patient franchise in the US by year end

MG growth continues – 3Q19 strongest quarter to date Developing portfolio of products in gMG to target entire spectrum of

disease Ongoing Phase 3 ULTOMIRIS® trial to enroll broader patient population

FcRn / ALXN1830 to potentially expand gMG patient options

U.S. and Germany launches underway Compelling clinical profile well-received by physicians and patients;

payer feedback positive and in line with expectations Monotherapy data showing 100% relapse prevention at 144 weeks

highlights compelling value proposition for both patients & payers


C O N T I N U E D M O M E N T U M A N D D I V E R S I F I C AT I O N F R O M M E T A B O L I C S



STRENSIQ®

KANUMA®

Continue to identify new patients Launched and reimbursed in 7 countries Ongoing work to secure additional ex-US

agreements Continue to educate laboratories on

CALIPER study findings Leveraging efficiencies in identifying

patients

Continue to identify new patients with LAL-D

Launched and reimbursed in 9 countries Improving ex-US funding agreements Leveraging efficiencies in identifying

patients

+36%

+12%

$113

$154

3Q18 3Q19

$25 $28

3Q18 3Q19


Closing RemarksLudwig Hantson, Ph.D.Chief Executive Officer


M E A N I N G F U L P R O G R E S S I N Q 3 2 0 1 9

C L O S I N G R E M A R K S

ULTOMIRIS Atypical HUS U.S. Approval

SOLIRIS® NMOSD E.U. Approval

Japanese License Agreement for Eidos’ AG10 in ATTR;Adds to Amyloidosis footprint with Caelum

Option to Co-Develop and Commercialize Stealth’s Late-Stage Therapy for Mitochondrial Diseases

Agreement to Acquire AchillionAdds Clinical Stage Oral Factor D Inhibitors to Portfolio

ULTOMIRIS PNH EU and Japan Launches

On track for best-in-class ULTOMIRIS ® PNH conversion

Neurology on track to be largest U.S. franchise by year end



2021-20232019-20202017-2018

V I S I O N T O 2 0 2 3


C L O S I N G R E M A R K SC

onve

rsio

nEx

pans

ion

Div

ersi

ficat

ion

• SOLIRIS® PNH• SOLIRIS aHUS

gMGApproval

gMG & NMOSD

PNH Conversion

NMOSD Approval

• ULTOMIRIS® gMG• ULTOMIRIS NMOSD• ULTOMIRIS SC (PNH/aHUS/gMG/NMOSD)• ULTOMIRIS ALS• ULTOMIRIS HSCT-TMA

• ALXN 1840 (Wilson’s Disease)• CAEL101 (AL Amyloidosis)• AG10 (ATTR)• Elamipretide (PMM, Barth, LHON, GA)• Factor D (PNH with Clinical EVH, C3G,

Other Complement)

Continued Growth

aHUSConversion

gMGConversion

NMOSD Conversion

Continued Growth

1 Product Across 2 Indications

New Indication/RoA Launches

New Product Launches• STRENSIQ® (HPP)• KANUMA® (LAL-D)

2 Products Across 2 Indications


Q&A3Q2019 EarningsOctober 23, 2019


Appendix3Q2019 EarningsOctober 23, 2019




PNH

aHUS

gMG

2019 2020 2021 2022 2023 2024 2025 2026 2027EU Conversion Ambition – Germany example


70% conversion of stable patients by early 2022

70% conversion by end 2024

NMOSD70% conversion by end 2024

Illustrative

ODD for SOLIRIS®

NMOSD until 3Q2029

Earliest possible biosimilar launch


Orphan drug designation





PNH

aHUS

gMG

2019 2020 2021 2022 2023 2024 2025 2026 2027Japan Conversion Ambition


70% conversion of stable patients by late 2022

70% conversion by early 2025

NMOSD70% conversion by early 2025

Illustrative

ODD for SOLIRIS® NMOSD until 4Q2029

Biosimilar launch 6-9 months after ODD

Earliest possible biosimilar launch


Orphan drug designation

SOLIRIS IP protection through 2027





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