Amicus Therapeutics : Fourth Quarter and Full Year 2023 Results Conference Call and Webcast

A T T H E F O R E F R O N T O F

T H E R A P I E S F O R R A R E D I S E A S E S

FY23 Results

Conference Call

& Webcast

February 28, 2024

Forward-Looking Statements

This presentation contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities and pricing and reimbursement authorities, are based on current information. Actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in negotiations with pricing and reimbursement authorities; the potential that we may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies, the manufacturing, and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, non-GAAP profitability and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2023 to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.

Non-GAAP Financial Measures

In addition to financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results.

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A Rare Company

A leading biotech company with >$500M of sales projected in 2024

>500 EMPLOYEES in 20+ Countries

LEVERAGEABLE	2
GLOBAL
COMMERCIAL	APPROVED
ORGANIZATION	THERAPIES

$399M

in 2023 Revenue

21%

First Two-Component Therapy	IncreaseYear-Over-Year
for Pompe Disease

World Class

Clinical

Development

Capabilities

Expect Full Year

2024

Non-GAAP

Profitability

Non-GAAP

PROFITABILITY

Q4 2023

ACHIEVED

Combined Peak

Revenue Potential

$1.5B - $2B

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2024 Strategic Priorities

1. Transformative Year Ahead for Amicus

1

2

3

4

Drive double-digit Galafold® revenue growth of 11-16% at CER1

Execute multiple successful launches of Pombiliti + Opfolda

Advance ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases

Achieve non-GAAP profitability for the full year

4	1CER: Constant Exchange Rates; 2024 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2023

Galafold® (migalastat)

Continued Growth

Building a leadership position in the treatment of Fabry disease

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2023 Galafold Success (as of December 31, 2023)

Galafold is the only approved oral treatment option in Fabry disease

A unique mechanism of action for Fabry patients with amenable variants

$388M

2023 Galafold

60-65%Revenue

Share of Treated

Amenable Patients

17%

YoY Growth at CER1

Galafold is indicated for adults with a confirmed diagnosis of Fabry disease and an amenable variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions, and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.

6	1CER: Constant Exchange Rates

2,400+

Individuals

Treated 40+

Countries with

Regulatory

Approvals

35-50%

Fabry Patients

Amenable to

Galafold

Galafold Performance

Galafold YTD reported revenue growth of +18% to $388M

$430M-450M1

$388M



Global mix of switch (~43%) and previously

untreated patients (~57%)2

$306M

$329M

Q4

Expect non-linear quarterly growth to continue

$107M



$261M

due to uneven ordering patterns

Q3

and FX fluctuations

$182M

$101M

Distribution of Galafold revenue

Q2

by quarter over previous 5 years:

$91M

$94M

Q1

Q2

Q3

Q4

$37M

Q1

$5M

5 Year Avg.

22%

24%

26%

28%

$86M

FY16

FY17

FY18

FY19

FY20

FY21

FY22

FY23

FY24

FY24 revenue growth guidance to 11% to 16% at CER

7	1 At constant exchange rate (CER)
2 Data on file

Key Growth Drivers for 2024

Building off a strong year with highest patient demand seen in last four years to lay the

groundwork for continued double-digit Galafold growth in 2024

• Increasing patient identification through ongoing medical education, screening, and improved diagnostics
• Driving market share of treated amenable patients through excellent execution
• Expanding market through uptake in naïve population as well as geographic and label expansion
• Maintaining >90% adherence and compliance through HCP and patient education and support

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Pombiliti

+

Opfolda

(cipaglucosidase alfa-atga)

(miglustat)

Potential to establish a new standard of care for people living with late-onset Pompe disease

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Successful Global Launch of Pombiliti + Opfolda Underway

FY 2023 revenue of $11.6M ($8.5M in Q4 2023) provides strong foundation for 2024

Patient Demand

As of early January 2024

~120 patients treated with commercial product or scheduled to be treated

~105 patients from clinical trials and early access

~15 new patients from competitor ERTs or naïve

Very positive early feedback from real-world experience

KOL Outreach

Successfully engaged with top prescribers in each approved country

Existing relationships with HCPs at key treatment centers

Ongoing disease education

Access and Reimbursement

Positive interactions

with US, UK, and EU payors

Focus on broad patient access

Country-by-country reimbursement process underway

Multiple launches expected in 2H 2024

Focus in 2024 is on maximizing the number of patients on therapy by year end

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