Alexion Pharmaceuticals : Q2 Earnings Call July 30 2020

Second Quarter

2020

Earnings Call

July 30, 2020

BUNNY WITH HER HUSBAND, JOE LIVING WITH PNH

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

2 | 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 .

This presentation contains forward-looking statements, including statements related to: guidance regarding anticipated financial results for 2020 (and all of the assumptions, judgments and estimates related to such guidance); Alexion's updated capital allocation strategy; ability of Portola acquisition to expend our presence in the hospital setting; conversion of aHUS patients from SOLIRIS to ULTOMIRIS; potential 10 launches by 2023 and ability of product launches to enable capital return; the 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; potential of ULTOMIRIS subcutaneous formulation to be first subcutaneous option for both PNH and aHUS; ambition to quadruple the number of neurology (NMOSD and gMG) patients in the US by 2025; ability to enhance ANDEXXA's launch; the impacts of COVID-19 on Alexion's business, including its revenue, expenses and commercial and clinical programs; potential launches of ULTOMIRIS for additional indications; estimated addressable patient populations for our current and pipeline products; Alexion's diverse and growing pipeline and ability to return capital shareholders; future plans to expand treated patient population; future expansion of treated patient populations with potential product launches; expected benefits of subcutaneous products, including potential flexibility for patients; potential indications that can be treated by product candidates; plans to make future 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; clear potential benefits (and transformative impact) of current products and products under development and in clinical trials (including further extended dosing intervals). Forward-looking statements are subject to factors that may cause Alexion's results and plans to differ materially from those forward-looking statements, including for example: our dependence on sales from our C5 products (SOLIRIS and ULTOMIRIS); the impact of the COVID-19 pandemic on our business; 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; our success in integrating Portola; 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 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; the Phase 4 study regarding ANDEXXA does not meet its designated endpoints and/or is not deemed safe and effective by the FDA or other regulatory agencies (and commercial sales are prohibited or limited); 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); 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 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; 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 March 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 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. 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.

Amounts may not foot due to rounding.

Q 2 E A R N I N G S C A L L A G E N D A

3 | A G E N D A

INTRODUCTION

CEO OPENING REMARKS

FINANCIAL UPDATE & OUTLOOK

R&D HIGHLIGHTS

COMMERCIAL INSIGHT

STRATEGIC OUTLOOK & CLOSING

Q&A

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 .

Chris Stevo, Head of Investor Relations

Ludwig Hantson, Ph.D., Chief Executive Officer

Aradhana Sarin, M.D., Chief Financial Officer

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

Brian Goff, Chief Commercial & Global Operations Officer

Ludwig Hantson, Ph.D., Chief Executive Officer

All Participants

CEO Opening Remarks

Ludwig Hantson, Ph.D.

Chief Executive Officer

Tristan Living with LAL-D

C O M M I T T E D T O O U R M I S S I O N

5 | C E O O P E N I N G R E M A R K 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 .

ALBIE

LIVING WITH LAL-D

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

BUNNY

LIVING WITH PNH

AIRA

LIVING WITH HPP

ROBERTA LIVING	SUMAIRA	DONNAN
WITH GMG	LIVING WITH NMOSD	LIVING WITH aHUS

O P E R A T I O N A L E X C E L L E N C E E N A B L I N G R E P U R C H A S E

C O M M I T M E N T

6 | C E O O P E N I N G R E M A R K 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 .

Strong financial			Raising 2020 revenue guidance to		Achieved			Key Pipeline Progress in 2Q20:
execution,			$5,550 to $5,600M and		Best-in-class
				•	ULTOMIRIS aHUS EU approval
		non-GAAP(1) EPS to $10.65 to $10.95		Conversion
demonstrated
				>70% for PNH
					•	Positive ULTOMIRIS Ph3 SC data
commercial excellence
		GAAP(1) EPS of $0.96 to $1.30		ULTOMIRIS in
and growing pipeline
		negatively impacted by impairments		18 months		•	Positive CAEL-101 Ph2 dose-ranging

Closed Portola			Shifting ANDEXXA focus to a multi-faceted
Acquisition with strong
		hospital system approach for success, amid a COVID era
strategic and
operational plan

Returning Capital to			Minimum of $500 - $550M in share repurchases in 2020
Shareholders			Increasing to at least 1/3 of FCF(2) on average annually 2021 - 2023

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

(2)Free Cash Flow (FCF) defined as cash flow from operations less purchases of property, plant and equipment

C O N T I N U E D P R O G R E S S A G A I N S T O U R S T R A T E G Y

7 | C E O O P E N I N G R E M A R K 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 .

LEAD AND EXPAND

OUR COMPLEMENT BASE BUSINESS

LEAD		EXPAND

DIVERSIFY

INTO NEW GROWTH AREAS

DIVERSIFY

Set a New Standard for Best in Class Conversion

70% PNH ambition met in U.S.

aHUS Conversion on Track

aHUS ULTOMIRIS EMA approval

Innovating for Patients Positive topline Ph3 data for ULTOMIRIS SC program

Paving Way for Future ULTOMIRIS Conversion

IPR settlement provides clarity on potential biosimilar entry

Committed to Neurology and Growing the Addressable Population we Serve

189 net U.S. patient adds in 2Q amidst COVID-19 pandemic

Enrollment on track for gMG and NMOSD ULTOMIRIS Ph3 trials

ALS Ph3 trial enrolling

Increasing ULTOMIRIS

Addressable Population HSCT-TMAPh3 trial on track to initiate in 2H 2020

Diversifying our Commercial Portfolio

Portola acquisition expands our presence in the hospital setting

Diversifying our R&D Pipeline

ALXN1830 Ph1 SC dosing signal

Positive Ph2 dose-ranging for CAEL-101

ALXN2040 Ph2 C3G data supports proof of mechanism

Financial Update

Aradhana Sarin, M.D.

Chief Financial Officer

Roberta

Living with gMG

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

9 | F I N A N C I A L U P D A T E	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 .

Total Revenues

GAAP(1) Operating

Margin

Non-GAAP(1)

Operating Margin

• C5 (SOLIRIS + ULTOMIRIS) sales grew 19% YoY driven by

$1.445B	+20% vs 2Q19	growth in neurology & continued strength in the base PNH
		and atypical HUS business

• Metabolic sales grew 30% YoY driven by increase in volume

(95%)	NM*	vs 2Q19	▪ GAAP operating margin impacted by 2Q20 impairment of
			intangible assets
58%	+4 bps	vs 2Q19	▪ Non-GAAP operating margin strength continued in 2Q20

GAAP(1) EPS

Non-GAAP(1) EPS

($4.84)

$3.11

-337%	vs 2Q19
+18%	vs 2Q19

• GAAP EPS impacted by 2Q20 impairment of intangible assets, partially offset by a net increase in gains recorded on our strategic equity investments
• Non-GAAPEPS primarily driven by topline strength YoY

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

Provided July 30, 2020, 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. *Percentage not meaningful

10 | F I N A N C I A L U P D A T E

	$1,203
	100
($)	124
Millions	307

672

2Q19

N E T P R O D U C T S A L E S B Y G E O G R A P H Y

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 .

	$1,445	$1,445
	$1,384
$1,263	125	113
126	158
128	159
156

137		329	307
	309
302

	793	832	868
696

3Q19	4Q19	1Q20	2Q20
US Europe	APAC	ROW

Provided July 30, 2020, 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.

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

11 | F I N A N C I A L U P D A T E

SOLIRIS Net Product Sales

	$981	$991	$1,013	$1,023	$976
	94	120			112	116	92
($)	110	118			94	87	82
280	256			250	264	248
Millions
496	497			557	556	553
	2Q19	3Q19	4Q19	1Q20	2Q20
		US	Europe	APAC	ROW
		ULTOMIRISNet Product Sales
						$223	$251
						60
				$170	57
				32
($)
				46	34
	$90
Millions				31
$54	21				158
		93	131
		65
	54
	2Q19	3Q19	4Q19	1Q20	2Q20
		US	Europe	APAC	ROW

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 .

Total C5 Franchise Net Product Sales

			$1,183	$1,246	$1,227
		$1,080
	$1,035	170	223	251
	54	90
($)
Millions	981	991	1013	1023	976

2Q19	3Q19	4Q19	1Q20	2Q20
	SOLIRIS	ULTOMIRIS

SOLIRIS: YoY revenue flat as strong growth in Neurology is offset by ULTOMIRIS conversion in PNH and atypical HUS; QoQ revenue impacted by timing of tender market orders in ROW

ULTOMIRIS: Continued strength driven by conversion from SOLIRIS in PNH and atypical HUS; 70% PNH conversion ambition in U.S. achieved

Provided July 30, 2020 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.

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

12 | F I N A N C I A L U P D A T E

MetabolicNet Product Sales

			$201	$199	$218
		$183	34
	$168	34	27
	28
($)	26
Millions		154	167	172	184
	141

2Q19	3Q19	4Q19	1Q20	2Q20
	STRENSIQ KANUMA

Metabolics:

• +30% YoY revenue growth; driven by volume

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 .

Legacy Portola: ANDEXXA/ONDEXXYA Net Product Sales

$36

		3	$28
	$27		$26
		5
($)			3	$22
				2
Millions				23
27	33		20
		24
	2Q19(1)	3Q19(1)	4Q19(1)	1Q20(1)	2Q20 (2)
		United States	ROW

ANDEXXA / ONDEXXYA:

• YoY & QoQ revenue decline driven primarily by COVID-19 related demand reductions

1. Net product revenues as previously reported by Portola
2. Net product revenues recognized by Portola in 2Q20 have not been adjusted for consistency with Alexion accounting policies and are not included in Alexion's 2Q 2020 quarterly results. Alexion has not audited these net product revenues and has relied upon the amounts as publicly reported by Portola for all periods prior to the acquisition and, with respect to the second quarter of 2020 upon information that was made available to Alexion in the accounting records of Portola.

Provided July 30, 2020 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.

2 Q 2 0 2 0 F I N A N C I A L P E R F O R M A N C E - Y o Y C O M P A R I S O N

13 | F I N A N C I A L U P D A T ER 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 .

			2Q '20			2Q '19
$ Millions, Except EPS		GAAP (1)	Non-GAAP(1)		GAAP (1)	Non-GAAP(1)	∆ Non-GAAP(1)
Total Revenue	$1,445	$1,445		$1,203	$1,203		+20%
SOLIRIS® Revenue	$976	$976		$981	$981		<-1%
ULTOMIRIS® Revenue	$251	$251		$54	$54		+363%
STRENSIQ® Revenue	$184	$184		$141	$141		+30%
KANUMA® Revenue	$34	$34		$26	$26		+28%
COGS	$145	$142		$99	$96		+186 bps
% of Total Revenue	10%	10%		8%	8%
R&D	$221	$205		$188	$149		+181 bps
% of Total Revenue	15%	14%		16%	12%
SG&A	$301	$254		$299	$256		-370 bps
% of Total Revenue	21%	18%		25%	21%
Impairment of Intangible Assets	$2,053	-		-	-		-
Operating (Loss) Income	($1,370)	$845		$533	$703		+20%
Operating Margin	(95%)	58%		44%	58%		+4 bps
Effective Tax Rate	21%	15%		8%	13%		+219 bps
Earnings (Loss) Per Share	($4.84)	$3.11		$2.04	$2.64		+18%

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

Provided July 30, 2020, 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.

U P D A T I N G F Y 2 0 O U T L O O K T O R E F L E C T T H E S T R E N G T H O F

T H E B U S I N E S S D E S P I T E C U R R E N T E N V I R O N M E N T

14 | F I N A N C I A L U P D A T E	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 .

$ Millions, Except EPS

Total Revenue

SOLIRIS®/ULTOMIRIS®

Metabolic

ANDEXXA/ONDEXXYA

R&D (% of Total Revenue)(3)

GAAP

Non-GAAP

SG&A (% of Total Revenue)(3)

GAAP

Non-GAAP

Operating Margin(3)

GAAP

Non-GAAP

Earnings Per Share(3)

GAAP

Non-GAAP

Previous	Updated	YoY Growth
Guidance	Guidance(1)(2)
$5,230 to $5,330	$5,550 to $5,600	12%
$4,495 to $4,570	$4,725 to $4,755	11%
$735 to $760	$785 to $800	13%
-	$40 to $45	N/A
17.5% to 18.6%	18.1% to 19.2%	+90 bps
16.0% to 17.0%	16.5% to 17.5%	+256 bps
22.2% to 23.5%	24.5% to 25.7%	-17 bps
18.5% to 19.5%	21.0% to 22.0%	-54 bps
42.4% to 43.8%	3.8% to 5.4%	NM*
55.0% to 56.0%	53.0% to 54.0%	-240bps
$8.14 to $8.47	$0.96 to $1.30	-89%
$10.45 to $10.75	$10.65 to $10.95	3%

Adjusted 2020 Guidance - Key Assumptions

Increased Revenue Guidance Reflects Business Strength & Durability

• Strong compliance rates sustained across indications in 1H; potential risk for reduced compliance in 2H
• Lesser impact to new patient starts vs anticipated in 1H; however new patient initiation queue build is slowing
• Strong ULTOMIRIS conversion continues (particularly atypical HUS)
• Payer mix & institutional budget impact not yet seen; monitoring for 2H impact

Focus on Portola Integration and Cost Management in COVID-19 Environment

• R&D delays from COVID less than anticipated, resulting in increased spend
• Includes Portola Opex of ~$125M for 2H20(2)
• Portola ~$0.32 dilutive to non-GAAP EPS FY20(2)
• Strong, best-in-class margins maintained despite COVID & Portola integration

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 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 the previously announced activities that may occur after the issuance of this presentation.
2. Updated 2020 financial guidance includes the impact of the recent 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.
3. A reconciliation of GAAP to non-GAAP financial guidance is provided in the appendix and is available at www.alexion.com.

*Percentage not meaningful

Provided July 30, 2020, 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.

G R O W I N G A N D A D V A N C I N G O U R I N N O V A T I V E P I P E L I N E

15 | F I N A N C I A L U P D A T E

	2018(3)	2019
2017
$736M Non-GAAP(1)	$646M Non-GAAP(1)	$721M Non-GAAP(1)
$878M GAAP(1)	$730M GAAP(1)	$886M GAAP(1)
R&D Expense	R&D Expense	R&D Expense
2 Assets(2)	3 Assets	8 Assets
2 Internal	1 Internal	2 Internal
	2 Through BD	6 Through BD
4 Clinical Programs	8 Clinical Programs	14 Clinical Programs

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 .

2020

$948M Non-GAAP(1)

$1,040M GAAP(1)

R&D Expense(4)

11 Assets

3 Internal 8 Through BD

20+ Clinical Programs

Significant Progress Made Developing A Robust, Value-Creating Pipeline

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

(2)Asset defined as a singular molecular entity; (3)2018 decrease in R&D spend related to pipeline strategy refocus; (4)Mid point of 2020 guidance issued 7/30/20

U P D A T E D A P P R O A C H T O C A P I T A L A L L O C A T I O N

16 | F I N A N C I A L U P D A T E	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 .

Significant Progress Since 2017 Allows an Updated and More Flexible Approach

•	Alexion constantly evaluates
	our capital allocation approach
•	R&D portfolio has grown
	substantially since 2017
	• 20 total programs
	• confidence in potential of 10
	launches by 2023 enabling
	capital return
•	Free cash flow generation has
	improved over the last three

Funding Expanding R&D Portfolio

Capital Allocation Priorities

Share Repurchase	Balance Sheet	Disciplined BD
(Reduce Net Debt)

years, with high FCF

conversion(1)

• Allows Alexion to be more

flexible on capital allocation

strategy

Committing to $500 - $550M of repurchases in 2020; increasing to at least 1/3 of FCF on average annually from 2021 - 2023

No near-term plan to pay a dividend given life-cycle

Near-term focus on successful Portola integration

Remain open to future BD that meet criteria for value creation and strategic fit

(1)Free Cash Flow (FCF) defined as cash flow from operations less purchases of property, plant and equipment; FCF conversion defined as FCF divided by net income

R&D Highlights

John Orloff, M.D.

Global Head of R&D

Joe Living with PNH

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

2 0 D E V E L O P M E N T P R O G R A M S

18 | R & D H I G H L I G H T SR 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 .

End of 2017

2Q 2020

PHASE

PHASE 1

PHASE 2

PHASE 3

SOLIRIS

IV (GBS)

IV (NMOSD)

Ph3

Approved

IV (PNH)

Ph3

Approved

IV (aHUS)

Ph3

Approved

SC Weekly

Ph1

IV (gMG)

ULTOMIRIS

IV (NMOSD)

IV (ALS)

IV (HSCT-TMA)

IV (CM-TMA)

IV (COVID-19)1

IV (Renal Basket)

ALXN1720

SC (Anti-C5Bi-specific)

ALXN1840

Oral (Wilson)

ALXN1830

SC FcRn (WAIHA)

SC FcRn (gMG)

CAEL-101

IV (AL Amyloidosis)2

AG10

Oral (ATTR-CM) Japan Only 3

ALXN2040

Oral (PNH with EVH)

ALXN2050

Oral (PNH Monotherapy)

Oral (Renal Basket)

ANDEXXA

ANDEXXA-S (Urgent Surgery)

CERDULATINIB

Lymphoma (CTCL, PTCL, and FL)

Hematology

Nephrology

Metabolics

Neurology

Cardiology

Other/TBD

1Adults with COVID-19 who are hospitalized with severe pneumonia or acute respiratory distress syndrome (ARDS); 2Structured as an option to acquire Caelum; 3Exclusive license to develop and commercialize in Japan; See appendix for further description of each program

E N H A N C I N G C A P A B I L I T I E S T O S U P P O R T

G R O W I N G P I P E L I N E

19 | R & D H I G H L I G H T 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 .

Building on Existing Capabilities to Accelerate Towards Launches

Accelerate	Bring the	Enable
PoC and	Digital	Increase
Trial to the
Pivotal	Clinical	Capacity
Patient
Trials	Trials

• Focus on innovative study designs with real-worlddata-driven startup and enrollment plans
• Generalize home treatment, telemedicine, and real- world data collection
• Build capabilities and empowerment at country level

• Enhance real-time and predictive risk identification via central monitoring
• Establish artificial intelligence- and machine learning- driven decision support
• Balancing of internal & external resources based on trial risk and productivity

Transforming our clinical trial programs through digital and enhanced organizational capacity

PoC = Proof of Concept

M U L T I P L E S H O T S O N G O A L F O R 1 0 L A U N C H E S B Y 2 0 2 3

20 | R & D H I G H L I G H T 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 .
	LEAD and EXPAND our complement base business		DIVERSIFY into new growth areas

Weekly SC

~20K

U.S. Target

Population1

~15K

U.S. Diagnosed

Population2

ALXN1840 ALXN2040

<6K Each

U.S. Ultra-Rare

Population

Ph3 Trial

To File

3Q '21

First in

Class

SC Infusion C5 Inhibitor

PNH / aHUS

				~5K
				U.S. Diagnosed
				Population3
Ph3 Trial		Ph3 Trial		Ph3 Trial
>65%		Enrollment		To Initiate
Enrolled		On-Going		2H '20
Best in		First in		First and
Class		Class		Only
C5 Inhibitor		C5 Inhibitor		C5 Inhibitor
gMG		ALS		HSCT-TMA

							~10K
							U.S. Diagnosed
							Population7		<6K
~4.5K
							JP Diagnosed
U.S. Target	~2K	<2K				Population8
Population4
		Addt U.S.		JP Diagnosed
		Opportunity5		Population6
Ph3 Trial		Ph3 Trial		Ph3 Trial		Ph3 Trial		Ph3 Trial
>25%		To Initiate		To Initiate		To Initiate		To Initiate
Enrolled		1H '21		1H '21		2H '20		2H '20
Best in		Best in		First and		First		Best in
Class		Class		Only		Targeted		Class
C5 Inhibitor		C5 Inhibitor		C5 Inhibitor		Therapy		Small
				New				Molecule
NMOSD	CM-TMA	GBS		AL	ATTR-CM
				Japan Only	Amyloidosis	Japan Only

~5K

U.S. Diagnosed	<10%
Population9
		U.S. PNH
		Population10
Ph3 Trial		Ph3 Trial
On-Going;		To Initiate
Data 1H '21		2H '20
Potential		Address
Superiority		Patients
vs Standard		Suffering
of Care		from EVH
Wilson		PNH
Disease		with EVH

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

1. 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

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

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

ALXN2040 C3G

PK/PD results support proof of mechanism

• Reductions in proteinuria observed in subset of patients
• ALXN2040 PK and AP inhibition insufficient for robust disease control

Exploring next steps for C3G, including ALXN2050 which has improved PK/PD and inhibition of the AP

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 .

ALXN1210 & ALXN2050

Pursuing a Proof of Concept/Portfolio Strategy in Renal

• Planning PoC studies across several renal indications
• Strategically assessing routes of administration and unmet patient needs
• Initiation of ALXN1210 Study 2H2020
• Initiation of ALXN2050 Study 1H 2021

ALXN1830 gMG & WAIHA

SC single doses suggest meaningful IgG-lowering potential prior to study pause due to COVID-19

• Preliminary PK/PD modeling suggests weekly SC infusions of 1500mg may have the potential to provide >70% IgG lowering
• Dosing would be compatible with convenient SC delivery via on-body device

Potential Indications

LupusIgAN Nephritis

Primary MN

C3G

& Others

	Assets
ALXN1210	Q8W IV; Q1W SC Dosing
	(in development)
ALXN2050	BID Oral Dosing

Intend to re-start program 1H21; Continue gMG SC development and shift WAIHA to SC formulation

After PoC trials, proceed with the most promising

asset/indication combinations for further

development in Phase 3 trials

AP = Alternative Pathway; WAIHA = Warm Autoimmune Hemolytic Anemia; PoC = Proof of Concept

Commercial Insight

Brian Goff

Chief Commercial

& Operations Officer

Donnan

Living with aHUS

	U L T O M I R I S I N P N H I S T H E N E W S T A N D A R D
	F O R B E S T - I N - C L A S S C O N V E R S I O N
23 | C O M M E R C I A L I N S I G H T	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 .

Continuing to enhance ULTOMIRIS value proposition by shortening infusion time (100mg/mL)

and offering infusion independence (subcutaneous formulation)

Jul 2020

PNH ULTOMIRIS 70% Conversion

In US Achieved

aHUS ULTOMIRIS Conversion

On Track T0 70% Ambition*

Dec		2018
			Jul 2019
U.S. PNH ULTOMIRIS		EU PNH ULTOMIRIS
Approval			Approval
Jun 2019	Oct 2019
			JP PNH ULTOMIRIS	U.S. aHUS ULTOMIRIS
			Approval			Approval

Jun 2020	2H	2020	3Q 2021
Positive Topline Ph3	100mg/mL ULTOMIRIS	SC ULTOMIRIS
ULTOMIRIS SC Data	Formulation Launch	Formulation Filing
EU aHUS ULTOMIRIS	JP aHUS ULTOMIRIS
Approval	Approval

			ULTOMIRIS SC Formulation
ULTOMIRIS 100mg/mL High Concentration
• Reduces infusion time by >50% to ~45 minutes		•	Weekly SC infusion with rapid, patient-friendly device
		•	Potential to be first SC option for both PNH & aHUS

*aHUS ambition of 70% of total patients on ULTOMIRIS within 2 years of launch

E X P A N D I N G L E A D E R S H I P W I T H C O N T I N U O U S

N E U R O L O G Y G R O W T H

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

US Neurology Patients: gMG and NMOSD

4x growth ambition	2,341
by 2025 set

2,152

1,885

1,530

1,193

968

788

560

375

194

43

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 .

gMG: Significant Opportunity for Growth In Target Market

4x growth ambition by 2025**

5-8K

~20K

Opportunity to expand target population by moving earlier in the treatment journey

Novel entrants expand treated patient population

60-80K Total US gMG Patients

On Track to Achieve Ambition of 4x Expansion of US Neurology Treated Patients*

*Ambition Baseline - 12/31/19 1,885 patients; **4X growth ambition for both gMG and NMOSD, graphic above only reflects gMG population

A N D E X X A F I R S T A P P R O V E D T H E R A P Y F O R F A C T O R X A

I N H I B I T O R M A J O R B L E E D R E V E R S A L

25 | C O M M E R C I A L I N S I G H T	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 .

Patients With Life- Threatening

or Severe Bleeds

While On Factor Xa Inhibitors

Face Potentially

Devastating Consequences

>40%

Intracranial Hemorrhage (ICH)

30-day mortality rate

~3-12%

Gastrointestinal (GI) bleeding

30-day mortality rate

Value Proposition Is Strong

• Targeted and rapid reversal of anti-FXa activity
• Proven efficacy in clinical trials
• Demonstrated safety profile
• Expanding body of real-world evidence, including compelling HEOR data
• Use built into 19 guidelines of medical societies in North America and Europe

In line with Alexion's mission, ANDEXXA is a transformative medicine for Factor Xa patients in need

P R O V E N S U C C E S S L E V E R A G I N G H O S P I T A L P L A T F O R M

26 | C O M M E R C I A L I N S I G H T	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 .

Journey of a Patient with aHUS

Keys to Success

Presentation Diagnosis Initiation Maintenance

✓ Strong value proposition of Soliris/ULTOMIRIS in treating aHUS

90% of aHUS initiations occur in the hospital setting

Continued growth in aHUS patient initiations with highest to date in 2Q

• 10+ years of experienced account managers focused on system- wide approach
• Dedicated access and field reimbursement support who work with financial decisionmakers in the hospital setting
• Existing trusted relationships with large hospital systems across the country

2011

2020

L E V E R A G I N G A L E X I O N ' S C O M M E R C I A L C O R E

C O M P E T E N C I E S T O E N H A N C E A N D E X X A ' S L A U N C H

27 | C O M M E R C I A L I N S I G H T	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 .

Continued Progress Over Past 90 Days

• NTAP renewal proposed through ANDEXXA DRG inclusion in Oct 2021
• J-Codewent live for "drip and ship" or out-patient use in July 2020
• ACC consensus guidelines published in July 2020

Ramping Up ANDEXXA Through More Comprehensive Account Support

		Thought Leader	Mobilize champions
		Liaisons
Market Access
	Contracting
Field Medical
	Health	Enable system level access
		Economics
		Market Access
		Field Medical	Data generation, KOL engagement
Field Sales
		Field Sales	HCP/community demand generation

illustrative

Portola Deployment	Alexion Deployment
(Launch to Date)	(Post Integration)

Focus will shift from HCP pull through to a multi-faceted approach across the hospital system

CEO Closing Remarks

Ludwig Hantson, Ph.D.

Chief Executive Officer

Tristan Living with LAL-D

D R I V I N G S H A R E H O L D E R V A L U E T H R O U G H A L L A V E N U E S

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

Strong Financial Execution

+16% Revenue CAGR (2016-2020*)

$5,575

$4,991

$4,131

$3,551

$3,084

2016 2017 2018 2019 2020*

+24% Non-GAAP(-17% GAAP)(1)

EPS CAGR (2016-2020*)(1)

$10.70 $10.80 $10.53

$7.92

$5.86

$4.62

$1.76	$1.97				$1.13
			$0.35
2016	2017	2018		2019	2020*
		Non-GAAP EPS(1)		GAAP EPS(1)

Commercial Launch Excellence

• • Diverse, Growing Pipeline
• Set new standard for best-in-class conversion with ULTOMIRIS PNH
• • >70% US conversion in ~18 months
• aHUS remains on track for 70% conversion ambition in 2 years
• US neurology patient volume expansion in 2 years post-launch
• Expanding hospital presence post- Portola acquisition with ANDEXXA
• • Multi-facetedsystem approach
• 20 pipeline programs spanning Rare Hematology, Nephrology, Neurology, Cardiology, Metabolics

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 .

Returning Capital to

Shareholders

• Updating capital allocation strategy given continued strength in our FCF conversion and robust progress on our pipeline
• Commitment to share buybacks
• • Minimum of $500 - $550M of repurchases in 2020
  • Increasing to at least 1/3 of FCF on average annually from 2021 - 2023
• Retain financial flexibility for disciplined business development

(1)A reconciliation of GAAP to non-GAAP financial results is provided in the appendix and is available at www.alexion.com. *Represents mid-point of 2020 guidance ($5,550 to $5,600M revenue; $10.65 to $10.95 non-GAAP EPS; $0.96 to $1.30 GAAP EPS)

Q&A

JUSTICE	CHELSEY
LIVING WITH aHUS	LIVING WITH NMOSD

MONIKA WITH HER HUSBAND LIVING WITH gMG

APPENDIX

ALEXION CURRENT INDICATIONS

32 |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 .

	Indication	Description	Links
PNH	Paroxysmal Nocturnal Hemoglobinuria	Chronic, debilitating, and potentially life-threateningultra-rare blood	more info
		disorder, with an average age of onset in the early 30s
aHUS	atypical Hemolytic Uremic Syndrome	Ultra-rare, genetic, chronic, potentially life-threatening disease.	more info
		Chronic uncontrolled complement activation results in thrombotic
		microangiopathy (TMA)
gMG	Generalized Myasthenia Gravis	Debilitating, chronic, and progressive autoimmune neuromuscular	more info
		disease.
NMOSD	Neuromyelitis Optica Spectrum Disorder	Rare, devastating, complement-mediated disorder of the central	more info
		nervous system characterized by relapses where each individual
		attack results in cumulative disability including blindness and
		paralysis, and sometimes premature death (primarily affects women)
HPP	Hypophosphatemia	Inherited, progressive, ultra-rare metabolic disease in which	more info
		patients experience devastating effects on multiple systems of the
		body, and face debilitating or life-threatening complications
LAL-D	Liposomal Acid Lipase Deficiency	Genetic, chronic, and progressive ultra-rare metabolic disease in	more info
		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
ANDEXXA	Coagulation factor Xa reversal	Reversal agent for life-threatening bleeds induced by factor Xa	more info
	(recombinant)	inhibitors

PIPELINE COMPOUNDS

33 |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 .

Identifier	Name (INN)	MOA	ROA	Other	Indications	Disclosed Launch Timing
				Names	(commercialized & in development)
ALXN1210	ULTOMIRIS	C5 inhibitor antibody with	IV	N/A	•	Paroxsymal Nocturnal Hemoglobinuria (PNH)	• Launched (US, EU, JP)
	(ravulizumab)	extended half-life			•	Atypical Hemolytic Uremic Syndrome (aHUS)	•	Launched (US, EU)
					•	Generalized Myasthenia Gravis (gMG)	• Launch exp late '22 / early '23
					• Neuromyelitis Optica Spectrum Disorder (NMOSD)	• Launch exp late '22 / early '23
					• Amyotrophic Lateral Sclerosis (ALS)	•	Not disclosed
					• Hematopoetic Stem Cell Transplant Thrombotic	•	Not disclosed
						Microangiopathy (HSCT-TMA)
					•	Complement Mediated Thrombotic	•	Not disclosed
						Microangiopathy (CM-TMA)
					• Adults with COVID-19 who are hospitalized with	•	Not disclosed
						severe pneumonia or acute respiratory distress
						syndrome (ARDS)
					•	Renal Basket Study	•	Not disclosed
ALXN1720	N/A	Bispecific C5 Albumin	SC	N/A	•	Undisclosed complement-mediated indications	•	Not disclosed
		minibody
ALXN1840	N/A	Copper chelator	Oral	WTX-101	•	Wilson's Disease	•	Not disclosed
ALXN1830	N/A	Anti-FcRn antibody	IV & SC	SYNT001	•	Warm Autoimmune Hemolytic Anemia (WAIHA)	•	Not disclosed
					•	Generalized Myasthenia Gravis (gMG)	•	Not disclosed
CAEL-101	N/A	ALκ/ALλ fibril reactive	IV	N/A	•	Amyloid Light-Chain (AL) Amyloidosis	•	Not disclosed
		antibody
AG10	N/A	TTR tetramers stabilizer	Oral	N/A	•	Transthyretin Amyloid Cardiomyopathy (ATTR-CM)	•	Not disclosed
		(small molecule)
ALXN2040	(danicopan)	Factor D inhibitor	Oral	ACH-4471	•	PNH with Extravascular Hemolysis (PNH w/ EVH)	•	Not disclosed
		(small molecule)
ALXN2050	N/A	Factor D inhibitor	Oral	ACH-5228	•	Paroxsymal Nocturnal Hemoglobinuria (PNH)	•	Not disclosed
		(small molecule)			•	Renal Basket Study	•	Not disclosed
ANNEXA-S	ANDEXXA	Factor Xa Reversal	IV	N/A	•	Major bleed patients on rivaroxaban/ apixaban	•	Launched (US, EU)
					•	Urgent Surgery	•	Not disclosed
CERDULATINIB	N/A	SYK/JAK kinase inhibitor	Oral	N/A	•	Lymphoma (CTCL, PTCL, FL)	•	Not disclosed

ULTOMIRIS CONVERSION DYNAMIC: TWO KEY CONSIDERATIONS

34 | A P P E N D I X

Conversion Loading Dose Dynamic

ULTOMIRIS vs. SOLIRIS U.S. Annual Cost Per Patient

PNH

aHUS

+10%

-10%

Year 1: Loading dose + Maintenance Dosing

	Maintenance Dosing	-20%

Note: pricing discounts are approximations, not exact

-33%

• SOLIRIS indication-specific dosing: aHUS, gMG, NMOSD labeled dose higher than PNH
• • Drives indication-specific pricing differences when comparing SOLIRIS vs. ULTOMIRIS pricing
• ULTOMIRIS weight-based dosing

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 .

Quarter-on-quarter (QoQ) Variability

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

• 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

35 | A P P E N D I X	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 .

36 | A P P E N D I X	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 .

37 | A P P E N D I X	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 .

38 | A P P E N D I X	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 .

39 | A P P E N D I X	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 .

40 | A P P E N D I X	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 .

41 | A P P E N D I X	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 .

42 | A P P E N D I X	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 .

	Reconciliation of GAAP to non-GAAP R&D Expense
		2017		2018		2019		2020
GAAP R&D Expense	$	736	$	646	$	721	$	948
Share-based compensation		76		57		62		92
Upfront and milestone payments related to licenses and collaborations		49		27		103		-
Restructuring related expenses		16		0		-		-
Non-GAAP R&D Expense	$	878	$	730	$	886	$	1,040

43 | A P P E N D I X	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 .

Reconciliation of GAAP to non-GAAP EPS

		2 0 1 6		2 0 1 7		2 0 1 8		2 0 1 9		2 0 2 0
GAAP net incom e	$	399.4	$	443.3	$	77.6	$	2,404.3	$	252.0
Before tax adjustments:
Cost of sales:
Share-based compensation		11.1		11.1		16.0		14.2		16.0
Fair value adjustment in inventory acquired		10.8		5.2		-		-		-
Restructruing related expenses		-		152.1		5.8		-		-
Research and development expense:
Share-based compensation		57.6		76.4		57.4		61.7		92.5
Upfront and milestone payments related to licenses and other strategic agreements		9.6		49.4		26.7		103.4		-
Restructruing related expenses		-		16.3		0.1		-		-
Selling, general and administrative expense:
Share-based compensation		123.7		155.7		129.6		161.1		180.0
Restructruing related expenses		-		10.9		19.4		-		-
Litigation charges		-		-		13.0		0.1		22.0
Gain on sale of asset		-		-		(3.5)		-		-
Acquired in-process research and development		-		-		1,183.0		(4.1)		-
Amortization of purchased intangible assets		322.2		320.1		320.1		309.6		202.0
Change in fair value of contingent consideration		35.7		41.0		116.5		11.6		31.0
Acquisition-related costs		2.3		-		-		-		131.0
Restructuring expenses		3.0		104.6		25.5		12.0		(1.0)
Impairment of intangible assets		85.0		31.0		-		-		2,053.0
Investment income and (expense):
(Gains) and losses related to strategic equity investments		-		-		(43.1)		(59.7)		(26.0)
Other income and (expense):
Gain related to purchase option		-		-		-		(32.0)		-
Restructuring related expenses		-		2.6		(0.1)		-		-
Adjustments to income tax expense		(6.0)		(82.2)		(145.4)		(584.9)		(517.0)
N on-GAAP net incom e	$	1,054.4	$	1,337.5	$	1,798.6	$	2,397.3	$	2,435.5
GAAP earnings per com m on share - diluted	$	1.76	$	1.97	$	0.35	$	10.70	$	1.13
N on-GAAP earnings per com m on share - diluted	$	4.62	$	5.86	$	7.92	$	10.53	$	10.80
Shares used in com puting diluted earnings per com m on share (GAAP)		226.3		225.4		224.5		224.8		222.5
Shares used in com puting diluted earnings per com m on share (non-GAAP)		228.3		228.1		227.1		227.6		225.5