Amicus Therapeutics : View presentation

40th Annual J.P. Morgan Healthcare Conference

At the Forefront of Therapies for Rare Diseases

January 12, 2022

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 presentation 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 in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, and revenue goals, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations and/or revenue from the COVID-19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID-19, such as travel bans, shelter in place orders and third-party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product or to treatment sites. In addition to the impact of the COVID-19 pandemic, 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, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, U.K., Japan, the U.S. and other geographies or our other product candidates if and when approved; 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, commercialization and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding corporate financial guidance and financial goals and the attainment of such goals and statements regarding projections of the Company's revenue 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, 2020, and on Form 10-Q for the quarter ended September 30, 2021. 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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Amicus

Definition:

əˈmēkəs (noun) Latin Friend

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Our Passion is for Patients

Our Mission:

We seek to deliver the highest quality therapies for people living with rare diseases

Our Vision:

Be a leader in rare disease drug development and commercialization leveraging our expertise in bringing life-changing therapies to patients

A Rare Company

Patient Dedicated, Rare Disease Biotechnology Company with Sustained Double-Digit Revenue

Growth, a Global Commercial Infrastructure, and Late-stage Development Capabilities

EMPLOYEES	AT-GAA
in 27 Countries

a Two-Component Therapy

under Regulatory Review

GLOBALfor Pompe Disease

COMMERCIAL

ORGANIZATION

Gene Therapy

PLATFORM

Protein Engineering

& Glycobiology

$557M

Cash

as of 9/30/21

World-Class

CLINICAL DEVELOPMENT

Capabilities

Robust R&D Engine

Nearly 50+ Lysosomal

Disorders and More Prevalent

Rare Diseases

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2021 Strategic Priorities Accomplished:

Setting the Stage for a Successful 2022

1 Achieve double-digit Galafold growth and revenue of $300M to $315M

2	Report data from the AT-GAA Phase 3 PROPEL study and complete
BLA and MAA filings for regulatory approvals
3	Advance clinical studies, regulatory discussions, and scientific data
across industry leading gene therapy pipeline
4	Further manufacturing capabilities and capacity to build world-class
technical operations to support all gene therapy programs
5	Maintain strong financial position

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