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Item 2.02 – Results of Operations and Financial Condition.
On January 9, 2023, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing preliminary 2022 revenue and its 2023 strategic outlook, along with various business updates. A copy of the press release is attached hereto as Exhibit 99.1. As previously announced, the Company will also be presenting at the 41st Annual J.P. Morgan Healthcare Conference on January 9th, 2023. A copy of the presentation materials management will be using at the conference is also attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.
The information furnished pursuant to this Item 2.02, including Exhibits 99.1 and 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, and shall not be incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d) Exhibits:
| Exhibit No. | Description | |
| 99.1 | Press Release dated January 9, 2023 | |
| 99.2 | Presentation Materials – 41st Annual J.P. Morgan Healthcare Conference | |
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) |
Signature Page
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.
| AMICUS THERAPEUTICS, INC. | |||
| Date: January 9, 2023 | By: | /s/ Ellen S. Rosenberg | |
| Name: Ellen S. Rosenberg | |||
| Title: Chief Legal Officer and Corporate Secretary | |||
Exhibit 99.1
Amicus Therapeutics
Reports Preliminary 2022 Revenue
and Provides 2023 Strategic Outlook
Significant Growth in Demand with
More Than 2,000 People Living with Fabry Disease on Galafold by End of 2022
2022 Full Year Revenue of ~$329M, Representing 16% YoY Growth at CER
Continued Double-Digit Growth in Galafold Revenue of 12-17% at CER Expected in 2023
Multiple Approvals and Launches Expected in 2023 for AT-GAA in Pompe Disease
On-Track to Achieve non-GAAP Profitability in 2H2023
PHILADELPHIA, PA, January 9, 2023 – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today provided its preliminary and unaudited 2022 revenue, corporate updates, and full-year 2023 outlook.
Corporate Highlights:
| · | Global revenue in 2022 reached $329 million (preliminary and unaudited) driven by strong new patient accruals and sustained patient adherence, representing a year-over-year operational revenue growth measured at constant exchange rates (CER)1 of 16%. Full-year revenue growth measured at actual exchange rates was 8% reflecting a negative currency impact of approximately $26 million, or 8%, in 2022. Fourth quarter revenue was approximately $88 million (preliminary and unaudited). |
| · | For the full-year 2023, the Company anticipates double-digit Galafold revenue growth of 12-17% at CER. Growth is expected to be driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, continued diagnosis of new Fabry patients, and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. |
| · | Multiple approvals and launches expected in 2023 for AT-GAA in Late-onset Pompe disease. In Europe, the Committee for Medicinal Products for Human Use (CHMP) adopted a positive opinion of Pombiliti™, also known as cipaglucosidase alfa. A CHMP opinion for miglustat, the enzyme stabilizer component of AT-GAA is expected in the second quarter 2023. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022, with final approval expected in the second half of 2023. As previously announced, in the U.S., the Food and Drug Administration (FDA) deferred action on AT-GAA. Amicus remains actively engaged with the Agency on developing a plan and logistics for the pre-approval inspection and once there is more clarity, will provide expected approval timing. |
| · | Expanded access programs continue to meet the growing demand for AT-GAA across multiple countries. In the U.K., under the Early Access to Medicines Scheme (EAMS), multiple physicians have requested access from each of the leading Pompe centers in the country. Many patients with Pompe disease are participating in additional expanded access programs in the U.S., France, Germany, and Japan. |
| · | Galafold U.S. intellectual property estate strengthened following the issuance of 19 new patents in 2022. Galafold is protected by orphan drug regulatory exclusivities and a broad U.S. intellectual property portfolio of 46 orange book-listed patents, including 5 composition of matter patents, 30 of which provide protection through at least 2038. |
| · | Based on the current operating plan, the timing of AT-GAA approvals, and through careful management of expenses, the Company is on track to achieve non-GAAP profitability2 in the second half of 2023. |
Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “In 2022, Amicus remained steadfast in our mission to transform the lives of people living with rare diseases with excellent progress made across our strategic priorities. The Galafold business remained very strong last year, delivering double-digit operational revenue growth and finishing the year with over 2,000 patients on Galafold. We continue to expect robust growth again for 2023 driven by patient demand across the globe for this precision medicine for Fabry disease. In Pompe disease, we eagerly anticipate multiple AT-GAA regulatory approvals in key geographies including the EU, U.K., and U.S. following completion of the FDA inspection, and look forward to launching our second commercial product. We remain excited for the opportunity to offer a new and innovative treatment option, one that we believe has the potential to be the new standard of care, to people living with Pompe disease around the world. We believe we have the opportunity to deliver significant value for our shareholders as Amicus transforms into a leading global rare disease biotechnology company with two innovative therapies that can make a significant impact on the lives of people living with Fabry disease and Pompe disease.”
Amicus is focused on the following five key strategic priorities in 2023:
| · | Sustain double-digit Galafold revenue growth (12-17% at CER) |
| · | Secure FDA, EMA, and MHRA approvals for AT-GAA |
| · | Initiate successful global launches of AT-GAA |
| · | Advance next generation pipeline programs (Fabry GTx, Fabry Next-Generation Chaperone, Pompe GTx) |
| · | Maintain strong financial position on path to profitability |
Mr. Campbell will discuss the Amicus corporate objectives and key milestones in a presentation at the 41st Annual J.P. Morgan Healthcare Conference on Monday, January 9, 2023, at 2:15 p.m. PT. A live webcast of the presentation can be accessed through the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days.
1 In order to illustrate underlying performance, Amicus discusses its results in terms of constant exchange rate (CER) growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period. Full-year 2023 Galafold revenue guidance utilizes actual exchange rate at December 31, 2022.
2 Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. We define non-GAAP Net Income as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on impairment of assets, restructuring charges, and income taxes.
About Galafold
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.
U.S. INDICATIONS AND USAGE
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
U.S. IMPORTANT SAFETY INFORMATION
ADVERSE REACTIONS
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been established in pediatric patients.
To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
| · | Galafold is not intended for concomitant use with enzyme replacement therapy. |
| · | Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m2). The safety and efficacy of Galafold in children less than 12 years of age have not yet been established. No data are available. |
| · | No dosage adjustments are required in patients with hepatic impairment or in the elderly population. |
| · | There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse. |
| · | While taking Galafold, effective birth control should be used. It is not known whether Galafold is excreted in human milk. |
| · | Contraindications to Galafold include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION. |
| · | Galafold 123 mg capsules are not for children (≥12 years) weighing less than 45 kg. |
| · | It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold. |
| · | OVERDOSE: General medical care is recommended in the case of Galafold overdose. |
| · | The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received Galafold. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS. |
| · | Call your doctor for medical advice about side effects. |
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.
About Fabry Disease
Fabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which results from mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including heart, kidneys, and skin. Accumulation of GL-3 and progressive deterioration of organ function is believed to lead to the morbidity and mortality of Fabry disease. The symptoms can be severe, differ from person to person, and begin at an early age.
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel high-quality medicines for people living with rare diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on Twitter and LinkedIn.
Non-GAAP Financial Measures
In addition to financial information prepared in accordance with U.S. GAAP, this press release 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.
Forward Looking Statement
This press release 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, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations 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. 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, Japan, the US and other geographies or AT-GAA 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 and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. Statements regarding corporate financial guidance and financial goals and the attainment of such goals. With respect to 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, 2021 and the Quarterly Report filed on Form 10-Q for the quarter ended September 30, 2022. 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.
CONTACT:
Investors:
Amicus Therapeutics
Andrew Faughnan
Executive Director, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
FOLD–G
| AT THE FOREFRONT OF THERAPIES FOR RARE DISEASES 41STAnnual J.P. Morgan Healthcare ConferenceJanuary 9, 2023 |
| 2
Forward-Looking Statements
Thispresentationcontains"forward-lookingstatements" |
| Definition: \əˈmēkəs(noun) LatinFriend Our Passion is for PatientsOur Mission: We seek to deliver the highest quality therapies for people living with rare diseasesOur Vision: Be a leader in rare disease drug development and commercialization leveraging our global capabilities in bringing life-changing therapies to patients 3 |
| 4 A Rare CompanyPatient-dedicated, rare disease biotechnology company with sustained double-digit revenue growth, a global commercial infrastructure, and late-stage development capabilities GLOBAL COMMERCIAL ORGANIZATIONEMPLOYEESin 20 Countries GALAFOLD&AT-GAA Gene TherapyPlatformLeveraging Experience in Protein Engineering & Glycobiology 12%-17%FY23 Galafold Revenue Growth at CER Non-GAAP PROFITABILITY expected in 2H2023 Cumulative $1.5B-$2B Peak Potential AT-GAAUnder Global Regulatory Reviews forPompe Disease $355MCashas of 9/30/22 World-class Clinical Development Capabilities |
| 5 2022: A Year in Headlines Positive Long-Term Data from Phase 1/2 Study of AT-GAA in Pompe Disease |
| 2023 Strategic Priorities Sustain double-digit Galafold revenue growth of 12-17% at CER1 Secure FDA, EMA, and MHRA approvals for AT-GAA Initiate successful global launches of AT-GAA Advance best-in-class, next-generation Fabry and Pompe pipeline programs and capabilities Maintain strong financial position on path to profitability 6 1 2 3 4 5 1CER: Constant Exchange Rates;2023 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2022 |
| 7 Galafold® (migalastat)Continued GrowthBuilding a leadership position in the treatment of Fabry disease |
| 8 Fabry Disease Overview Deficiency of α-Gal A enzyme leading to GL-3 accumulation 1,000+ known variants 16,000+ diagnosed WW (51% female/49% male4)Fabry is a rare inherited genetic disorder caused by the deficiency of the GLA enzyme Leading Causes of DeathLife-Limiting Symptoms TRANSIENT ISCHEMIC ATTACK (TIA) & STROKE1 KIDNEY DISEASE3•Protein in the urine•Decreased kidney function•Kidney failure HEART DISEASE2•Irregular heartbeat (fast or slow)•Heart attack or heart failure•Enlarged heart GASTROINTESTINAL3•Nausea, vomiting, cramping, diarrhea•Pain/bloating after eating, feeling full •Constipation•Difficulty managing weight1.DesnickR, et al. Ann Intern Med. 2003 2.Yousef Z, et al. EurHeart J. 2013 3.Germain D. OrphanetJ Rare Dis. 2010 4.Data on file |
| 9 Global Fabry Market Believed to be significantly underdiagnosed–Newborn screening studies suggest Fabry is one of the more prevalent genetic diseases (~1:1,000 to ~1:4,000 incidence) In 2021 and 2022, Galafold was the fastest growing Fabry treatment and the greatest contributor to market growth–Galafold has led to market expansion with >1,000+ naïve patients treated Global Fabry disease market growth continues to be driven by diagnosis of new patients(millions)1Global market measured by reported sales of approved therapies for Fabry disease –2027 sales projected using ~8% CAGR $0$500$1,000$1,500$2,000$2,500 $3,00020172022E2027EGlobal Fabry Marketof ~$1.9B in 2022 and Tracking toward ~$2.6B+ by 20271 // // |
| 10 2022 Galafold Success (as of December 31, 2022) Building on Galafold’s success and leveragingleadership position to drive continued growth Galafold is indicated for adults with a confirmed diagnosis of Fabry diseaseand 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 visithttps://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 atwww.ema.europa.eu ..Galafold is the first and only approved oral treatment option with a unique mechanism of action for Fabry patients with amenable variants 350Amenable Variants in U.S. Label45Countries with Regulatory Approvals 35Countries withReimbursed Sales$329M2022 Galafold Revenue116%Operational Growth1,386Amenable Mutations in EU Label1 Preliminary and unaudited |
| 11 Galafold Global Launch Momentum (as of December 31, 2022) Global 3-month net new patients trend highest in 2 years ~50% share of treated amenable patients Healthy mix of switch (55%) and previously untreated patients (45%)1 Compliance and adherence >90% Growing prescriber base Continued penetration into existing marketsFurther uptake in diagnosed untreated populationContinued geographic expansionMaintaining compliance and adherenceDriving reimbursement and access FY22 Strength Reflects Increasing Demand with >2,000 Individuals TreatedSustained Growth in 2023 Driven by: Strong patient demand with 2,000+ individuals treated with Galafold and performance against key metrics lay the foundation for continued double-digit growth in 20231Data on file 11 |
| 12 Galafold Studies and Real-World Evidence Growing body of evidence for Galafold on compliance, impacts on quality of life, long-term efficacy and importance of early treatment 12 |
| 13 Galafold Long-Term Growth Opportunity In the next 5 years, the Fabry market is expected to surpass $2.5B with ~$1B estimated to have amenable mutations Galafold has successfully switched 80%-90% of treated amenable patients in its most mature markets1Assuming 35%-50% amenability 2Assuming ~7% CAGR Market Amenable to Galafold1Market Amenable to Galafold:~$1.1B1 Fabry Market Today Estimated Fabry Market in 20272 ~$1.9B ~$2.6B |
| 14 AT-GAA (cipaglucosidase alfa + miglustat)Potential to establish a new standard of care for people living with Pompe disease |
| 15 Deficiency of GAA leading to lysosomal glycogen accumulation and cellular dysfunction NBS studies shows higher incidence than medical literature suggests (~1:10,000 to ~1:30,000) Symptoms include muscle weakness, respiratory failure, and cardiomyopathy Majority of patients on current standard of care decline after ~2 years Estimated incidence of ~1:28,000; Significant underdiagnosis Respiratory and cardiac failure are leading causes of morbidity and mortality Age of onset ranges from infancy to adulthoodPompe Disease Overview NBS: Newborn ScreeningPompe is a severe and fatal neuromusculardisease causedby the deficiency of lysosomal enzymeGAA |
| 16 Global Pompe Market Global Pompe disease market growth continues to be driven by the diagnosis of new patients An estimated 3,500-4,000 Pompe patients globally are being treated by ERT3Global Pompe Market of ~$1.2B in 2022and Tracking toward $1.8B+ by 20271(millions) $0$200$400 $600 $800$1,000 $1,200 $1,400 $1,600 $1,800 $2,000 2017 2022E 2027E 1Global market measured by reported sales of approved therapies for Pompe disease –2027 sales projected using ~8% CAGR2As reported YTD September 2022 3Amicus Data on File from Market Mapping // // United States 41% Europe 36% Rest of World 23% Global Pompe Market Sales SplitYTD 9M 20222 |
| 17 AT-GAA: An Innovative Approach to Pompe Disease Our scientists created a uniquely glycosylated and highly phosphorylated ERT (cipaglucosidase alfa) that significantly enhances targeting to key affected muscles AT-GAA is a two-component therapy combining cipaglucosidase alfa, an ERT, with miglustat, an orally administered enzyme stabilizer Consists of a unique cell line producing a naturally glycosylated enzyme that can be properly processed within the lysosome to its mature form which is required to optimally break down glycogen1 cipaglucosidase alfamiglustat 1Selvan et al. 2021, J Biol Chem 2021 Jan-Jun;296:100769ERT: Enzyme Replacement Therapy |
| 18 Phase 3 PROPEL Study Results:Overall Population (n=122*) Primary and first key secondary endpoint showed greater improvement with AT-GAA vs. alglucosidase alfa in the overall population of ERT-naïve and ERT-experienced patients6MWD=6-minute walk distance;; FVC=forced vital capacity; SE=standard error. P values are nominal 2-sided; FVC data normally distributedand P value is from ANCOVA. 6MWD data not normally distributed and P value is for nonparametric ANCOVA; *Results exclude one outlier subject 6MWD (m): Change from baseline (n=85, n=37) FVC (% predicted): Change from baseline(n=85, n=37) |
| 19 Phase 3 PROPEL Topline Results:ERT Experienced Population (n=95)NOTE: Baseline is Mean (STDEV); CFBL is Mean (SE); P-values are nominal 2-sided; FVC data normally distributed and p–values are from ANCOVA6MWD data not normally distributed and 6MWD p-value is for non-parametric ANCOVA; 6MWD parametric MMRM p-value was p=0.078 ERT-experienced patients treated with AT-GAA demonstrated improvements over time in 6MWD and stabilization over time in FVC versus alglucosidase alfa 6MWD (m): Change from baseline (n=65, n=30) FVC (% predicted): Change from baseline(n=65, n=30) |
| 20 1Data on file 20 AT-GAA: Global Regulatory Status Anticipate regulatory approvals and launch into the three largest Pompe markets in 2023 1 FDA has not provided anticipated action dates as they continue to monitor the public health situation and travel restrictionsinChina; The Company expects the FDA to approve the NDA and BLA applications together Pombiliti™(cipaglucosidase alfa) received a positive CHMP opinion in December 2022 Miglustat CHMP opinion is expected in 2Q 2023 PDUFA action deferred due to inability to conduct pre-approval manufacturing inspection1 In discussion with the FDA to develop plans and logistics for inspection U.K. MAA submitted via recognition procedure based on CHMP opinion 20 |
| 21 AT-GAA: EU Opportunity Strong indication statement:–Pombiliti™(cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabilisermiglustat for the treatment of adults with late-onset Pompe disease (acid α glucosidase [GAA] deficiency) >1,300 patients are estimated to be treated in Europe1 Broad experience with AT-GAA from a wide set of KOLs through clinical trials and early access programs EU regulatory outcome and label to be leveraged in other ex-U.S. geographies1 Amicus Data on File from Market Mapping EU Pompe market currently represents a sizeable market opportunity of $450M+ |
| 22 AT-GAA: U.S. Opportunity AlaskaHawaiiUniversally screenedNot screened Pompe disease Newborn Screening in 34 U.S. States1U.S. Pompe Market Sales SplitYTD 9M 20222 >800 patients are estimated to be treated in the USA3 alglucosidase alfa 69% avalaglucosidase alfa 31% 1 https://www.newsteps.org/resources/data-visualizations/newborn-screening-status-all-disorders2As reported in YTD September 20223Amicus Data on File from Market Mapping U.S. Pompe market currently represents a sizeable market opportunity of >$500M 22 |
| 23 AT-GAA: U.K. Opportunity U.K. submission via recognition procedure based on CHMP opinion Significant demand through EAMS underscores unmet need:–Dozens of patients on treatment today–All leading centers have requested access–Requests for additional patients being received every month >200 people with Pompe disease are estimated to be treated in the U.K.11Amicus Data on File from Market Mapping U.K. represents the third largest Pompe disease market 23 |
| 24 AT-GAA: Ongoing Clinical Studies and Expanded Access Mechanisms Ongoing clinical studies in children and adolescents1with LOPD as well as in Infantile-Onset Pompe Disease (IOPD) Multiple expanded access mechanisms in place, including in the U.S., U.K., Germany, France, Japan, and others ~190 people living with Pompe disease are now on AT-GAA across extension studies and expanded access programs ~75 centers worldwide currently participating in clinical trials and access programs Advancing science though ongoing clinical studies and providing expanded accessthrough multiple mechanisms1 Children and adolescents aged 0 to <18 years old |
| 25 AT-GAA Launch Preparations Experienced and passionate rare disease medical and commercial organization poised for secondsuccessful launch Key Strengths Commitment to patient accessClear focus on launchIdentification of key Pompe disease treatment centersDevelopment of educational materials Planning Access Scientific Exchange Team Great experience and passionEagerness to introduce a new therapy upon approvalsHighly leverageable team in place, few new hires neededPublished Phase 3 PROPEL data in The Lancet NeurologyActive medical conference and publication scheduleMultiple expanded accessprograms in placeContinued education on biology of disease and diagnosisDemonstrating value to payors including parity pricing strategy |
| 26 Corporate OutlookDelivering on our mission for patients and shareholders |
| 27 INDICATIONDISCOVERYPRECLINICALPHASE 1/2PHASE 3REGULATORYCOMMERCIALFABRY FRANCHISEGalafold®(migalastat) Fabry Gene TherapyNext-Generation ChaperonePOMPE FRANCHISEAT-GAA (cipaglucosidase alfa + miglustat)Pompe Gene TherapyOTHERCLN3 Batten Disease Gene Therapy Next-Generation Research ProgramsAmicus Pipeline Streamlined rare disease pipeline with focus on Fabry disease and Pompe disease franchises ODD ODD BTDODD-Orphan Drug Designation BTD -Breakthrough Therapy Designation |
| 28 Expanding Our Leadership Position in Fabry and Pompe Differentiated gene therapy approach for greater potency and optimized cross correction through transgene engineering for stability and targeting Fabry Gene TherapyPompe Gene Therapy Proprietary pantropic AAV capsid and ubiquitous promoter Engineered hGLAtransgene for improved stability and optimized cross correction Preclinical data demonstrate robust substrate reduction across all Fabry disease relevant tissues Preclinical and manufacturing work underway Proprietary pantropic AAV capsid and ubiquitous promoter Engineered hGAA transgene for improved uptake and optimized cross correction Preclinical data demonstrate robust glycogen reduction in all key Pompe disease relevant tissues Preclinical and manufacturing work underway Wild-typeVehicleAAV.hGLA nat - LDAAV.hGLA nat - MDAAV.hGLA nat- HDAAV.hGLA eng - LDAAV.hGLA eng- MDAAV.hGLA eng- HD 0510152025 % tubes with storage **** *-3 Quadriceps: Histopathscore |
| 29 2023 Key Milestones Galafold: Fabry Disease AT-GAA: Pompe Disease Financial Strength 2023 revenue growth of 12-17% at CER First data published from followMeRegistry Regulatory approvals in multiple key geographies Initial sales of AT-GAA in 2H2023 Long-term data from Phase 3 PROPEL study at WORLDSymposium Non-GAAP profitability1expected in 2H20231Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. We define non-GAAP Net Income as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on impairment of assets, restructuring charges, and income taxes. |
| 30 True Measure of Success: Impacting the Lives of People Living with Rare Diseases YE17 2023+Thousands of Patients*>2,000 Patients* YE22 >350 Patients* *Clinical & commercial, all figures approximate |
| Thank You |
| 32 Environmental, Social, & Governance (ESG) Snapshot Board of Directors Eco-friendly decision-making has unearthed economic efficiencies while continuing to bolster our standing as a good corporate citizen.Committed to ongoing Board refreshment and diversity of background, gender, skills, and experience:Contributions allocated:$1,677,000 U.S.$832,976 Intl.770Volunteer hours (U.S.):20+Amicus supported community programs: 80%Board Independence60%Overall Board DiversityAddress a rare genetic disease First-in-class or best-in-classImpart meaningful benefit for patients 496Global Employees58%% female employees Whom We ServeEnvironmental Management Green building design Energy & water conservation Hazardous waste management Pledge for a CureDesignate a portion of product revenue back into R&D for that specific disease until there is a cure.Pricing PROMISE Programs we invest in have 3 key characteristicsCommitted to never raising the annual price of our products more than consumer inflation.Diversity, Equity, & Inclusion (DEI) 2023 and Beyond:•Maintain strength in global gender diversity•Increase US diversity through intentional and ongoing action•Continuously evaluate compensation practices to ensure pay parity Pledge to support a more inclusive culture to impact our employees, our communities, and society. 3 Female2 Veteran Status1 African AmericanDirector Diversity % Hiring Slate Diversity 82% Leverage employee capabilities and expertise to provide a culture that drives performance and ultimately attracts, energizes, and retains critical talent.Employee Recruitment, Engagement, & Retention Pulse surveys reveal employees feel high personal satisfaction in their job, are proud of their work and what they contribute to the communityCharitable Giving(as of 12/31/21)74 patients /20countriesExpanded Access through Jan 2023: Career DevelopmentReimagined performance management process to measure the what and the how, rewarding those who role-model our Mission-Focused Behaviors. |
| 33 FX Sensitivity and Galafold Distribution of Quarterly Sales Impact from Foreign Currency Q4 2022Currency Variances: USD/Q4 2021Q4 2022VarianceEUR1.1441.021(10.7%)GBP1.3481.174(12.9%)JPY0.0090.007(19.5%) Full Year 2023 Revenue SensitivityGiven the high proportion of Amicus revenue Ex-US, a change in exchange rates of +/-5% compared to year end 2022 rates could lead to a $11M-$12M change in global reported revenues in 2023.Distribution of Galafold Revenue by Quarter in Past 5 years: Q1 Q2Q3Q45 Year Avg.22%24%26%28% |
| 34 AT-GAA Phase 3 PROPEL Study Results Clinically meaningful outcomes from Phase 3 PROPEL study provide the basis for global regulatory submissions of AT-GAA |
