8-K
AMICUS THERAPEUTICS, INC. (FOLD)
8-K
2023-09-27
For: 2023-09-27
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April 04, 2026
UNITEDSTATES
SECURITIESAND EXCHANGE COMMISSION
WASHINGTON,D.C. 20549
FORM
8-K
CURRENTREPORT PURSUANT TO
SECTION13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of earliest event reported): September 27, 2023
AMICUS THERAPEUTICS, INC.
(Exact Name of Registrant as Specified in Its Charter)
| Delaware | 001-33497 | 71-0869350 |
|---|---|---|
| (State or Other Jurisdiction<br><br> <br>of Incorporation) | (Commission<br><br> <br>File Number) | (I.R.S. Employer<br><br> <br>Identification No.) |
3675 Market Street,
Philadelphia, PA 19104
(Address of PrincipalExecutive Offices, and Zip Code)
215-921-7600
Registrant’sTelephone Number, Including Area Code
(Former Name or Former Address, if Changed Since Last Report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ¨ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| ¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| ¨ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
Securities registered pursuant to Section 12(b) of the Act:
| Title<br> of each class | Trading<br> Symbol(s) | Name<br> of each exchange on which registered |
|---|---|---|
| Common<br> Stock Par Value $0.01 | FOLD | NASDAQ |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company ¨
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 7.01 – Regulation FD Disclosure.
On September 27, 2023, Amicus Therapeutics, Inc. (the “Company”) released an updated corporate overview presentation that it plans to use in meetings with investors and analysts. A copy of this presentation is attached hereto as Exhibit 99.1 and incorporated herein by reference.
The information in this Item 7.01, including Exhibit 99.1, is being furnished and shall not be deemed “filed” for purposes of Section 18 of the Act, or otherwise subject to the liabilities of that Section. The information in this Item 7.01, including Exhibit 99.1, shall not be incorporated by reference into any registration statement or other document pursuant to the Act.
Item 9.01 Financial Statements and Exhibits
(d) Exhibits:
| Exhibit No. | Description |
|---|---|
| 99.1 | September 2023 Corporate Overview Presentation |
| 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: September 27, 2023 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
| AT THE FOREFRONT OF<br>THERAPIES FOR RARE DISEASES<br>Corporate Overview<br>September 2023 |
|---|
| 2<br>Forward-Looking Statements<br>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<br>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,<br>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<br>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<br>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<br>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<br>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,<br>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<br>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,<br>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<br>patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, MHRA, and PMDA, may not grant or may delay approval for our product candidates; the potential<br>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 commercializing Galafold in<br>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<br>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<br>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<br>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<br>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<br>Form 10-K for the year ended December 31, 2022, and on Form 10-Q for the quarter ended June 30, 2023. You are cautioned not to place undue reliance on these forward-looking statements,<br>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<br>release to reflect events or circumstances after the date hereof.<br>Non-GAAP Financial Measures<br>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<br>supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial<br>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<br>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<br>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<br>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<br>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<br>unpredictable, impact on our future GAAP results. |
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| 3<br>A Rare Company<br>Patient-dedicated, rare disease biotechnology company with sustained double-digit revenue<br>growth, a global commercial infrastructure, and late-stage development capabilities<br>GLOBAL<br>COMMERCIAL<br>ORGANIZATION<br>EMPLOYEES<br>in 20 Countries<br>GALAFOLD<br> &<br>POMBILITI +<br>OPFOLDA<br>Gene Therapy<br>Platform<br>Leveraging<br>Experience in Protein<br>Engineering<br> & Glycobiology<br>14-18%<br>FY23 Galafold<br>Revenue Growth<br>at CER<br>Non-GAAP<br>PROFITABILITY<br>expected in<br>2H 2023<br>Cumulative $1.5B-<br>$2B Peak Potential<br>AT-GAA<br>Under U.S.<br>Regulatory Review for<br>Pompe Disease<br>$266M<br>Cash<br>as of 6/30/23<br>World-class<br>Clinical<br>Development<br>Capabilities |
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| 2023<br>Strategic<br>Priorities<br>Sustain double-digit Galafold revenue growth of 14-18% at CER1<br>Secure FDA, EMA, and MHRA approvals for AT-GAA<br>Initiate successful global launches of AT-GAA<br>Advance best-in-class, next-generation Fabry and Pompe pipeline<br>programs and capabilities<br>Maintain strong financial position on path to profitability<br>4<br>1<br>2<br>3<br>4<br>5<br>1 CER: Constant Exchange Rates; 2023 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2022 |
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| 5<br>Amicus Pipeline<br>Streamlined rare disease pipeline with focus on Fabry disease and Pompe disease franchises<br>INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL<br>FABRY FRANCHISE<br>Galafold®<br>(migalastat)<br>Fabry Gene Therapy<br>Next-Generation Chaperone<br>POMPE FRANCHISE<br>PombilitiTM + OpfoldaTM*<br>(cipaglucosidase alfa) + (miglustat)<br>Pompe Gene Therapy<br>OTHER<br>CLN3 Batten Disease Gene Therapy<br>Next-Generation Research Programs<br>ODD<br>BTD ODD<br>ODD - Orphan Drug Designation BTD - Breakthrough Therapy Designation *Approved only in the European Union and U.K. |
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| 6<br>Galafold® (migalastat)<br>Continued Growth<br>Building a leadership position in the<br>treatment of Fabry disease |
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| 7<br>Global Fabry Market<br> Believed to be significantly underdiagnosed<br> – Newborn screening studies suggest Fabry is one<br>of the more prevalent genetic diseases<br>(~1:1,000 to ~1:4,000 incidence)<br> In 2021 and 2022, Galafold was the fastest<br>growing Fabry treatment and the greatest<br>contributor to market growth<br> – Galafold has led to market expansion with<br> >1,000+ naïve patients treated<br>Global Fabry disease market growth continues to be driven by diagnosis of new patients<br>(millions)<br>1 Global market measured by reported sales of approved therapies for Fabry disease – 2027 sales projected using ~7% CAGR<br>$0<br>$500<br>$1,000<br>$1,500<br>$2,000<br>$2,500<br>$3,000<br>2017 2022 2027E<br>Global Fabry Market of ~$1.9B in 2022 and<br>Tracking toward ~$2.6B+ by 20271<br>// // |
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| 8<br>2023 Galafold Success (as of June 30, 2023)<br>Building on Galafold’s success and leveraging leadership position to drive continued growth<br>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%)<br>were headache, nasopharyngitis, urinary tract infection, nausea, and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please<br>visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings,<br>drug interactions, and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.<br>Galafold is the first and only approved oral<br>treatment option with a unique mechanism of<br>action for Fabry patients with amenable variants<br>35-50%<br>Fabry Patients<br>Amenable to<br>Galafold 40+<br>Countries with<br>Regulatory<br>Approvals<br>53<br>Orange Book<br>Listed Patents<br>$94.3M<br>2Q23 Galafold<br>Revenue<br>14-18%<br>2023 Galafold<br>Operational Growth<br>at CER<br>60%<br>Share of Treated<br>Amenable<br>Patients |
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| 9<br>Galafold Performance<br>Raising FY23 revenue growth guidance to 14% to 18% at CER<br> Global mix of switch (~45%) and previously<br>untreated patients (~55%)3<br> Compliance and adherence over 90%+<br> Expect non-linear quarterly growth to<br>continue due to uneven ordering patterns<br>and FX fluctuations<br>FY16 FY17 FY18 FY19 FY20 FY21 FY22 FY23<br>$5M<br>Q1<br>$79M<br>Q2<br>$81M<br>Q3<br>$82M<br>Q4<br>$88M<br>$37M<br>$91M<br>$182M<br>$261M<br>$306M<br>1H23 reported revenue growth of +13% to $180M with strong operational growth of +16%<br>$329M1<br>Q1<br>$86M<br>$375M - $388M2<br>Q2<br>$94M<br>1 FY22 reported revenue growth of +8% to $329M with strong operational growth of +16% at CER – FY22 negative currency impact YoY of ~$26M 2 At constant exchange rate (CER)<br>3 Data on file |
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| 10<br>Galafold Global Commercial Momentum (as of June 30, 2023)<br>Strong patient demand and performance against key metrics lay the foundation<br>for continued double-digit growth in 2023<br>10<br>Continued penetration into existing markets<br>Further uptake in diagnosed untreated population<br>Continued geographic expansion and label extensions<br>Maintaining compliance and adherence<br>Driving reimbursement and access<br>Sustained Growth in 2023 Driven by: |
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| 11<br>Pombiliti® (cipaglucosidase alfa)<br> +<br>Opfolda® (miglustat)<br>Potential to establish a new standard of care<br>for people living with Pompe disease |
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| 12<br>Deficiency of GAA leading to lysosomal<br>glycogen accumulation and cellular<br>dysfunction<br>Diagnosed at different stages of life,<br>from childhood to adulthood<br>Symptoms include progressive muscle<br>weakness, particularly skeletal and respiratory<br>muscles, that worsens over time<br>Respiratory failure is a major<br>cause of mortality<br>~5,000-10,000 people diagnosed<br>globally; Significant underdiagnosis<br>~$1.2B+ global Pompe ERT<br>sales1<br>Majority of patients on current<br>standard of care decline after<br>~2 years<br>Late-Onset Pompe Disease (LOPD) Overview<br>1 Based on 12 months ended December 31, 2022. Source: Sanofi Press Release<br>Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by<br>a deficiency of the enzyme acid alpha-glucosidase (GAA) |
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| 13<br>Global Pompe Market<br>Global Pompe disease market growth continues to be driven by the diagnosis of new patients<br> An estimated 3,500-4,000 Pompe patients<br>globally are being treated by ERT3<br>Global Pompe Market of ~$1.2B in 2022<br>and Tracking toward $1.8B+ by 20271<br>(millions)<br>$0<br>$200<br>$400<br>$600<br>$800<br>$1,000<br>$1,200<br>$1,400<br>$1,600<br>$1,800<br>$2,000<br>2017 2022 2027E<br>1 Global market measured by reported sales of approved therapies for Pompe disease – 2027 sales projected using ~8% CAGR 2 As reported FY2022<br>3 Amicus Data on File from Market Mapping<br>// //<br>United States<br>41%<br>Europe<br>37%<br>Rest of World<br>22%<br>Global Pompe Market Sales Split<br>FY20222 |
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| 14<br>AT-GAA: An Innovative Approach to Pompe Disease<br>Our scientists created a uniquely glycosylated and highly phosphorylated ERT<br>(cipaglucosidase alfa) that significantly enhances targeting to key affected muscles<br> AT-GAA is a two-component therapy combining<br>cipaglucosidase alfa, an ERT, with miglustat, an orally<br>administered enzyme stabilizer<br> Consists of a unique cell line producing a naturally<br>glycosylated enzyme that can be properly processed<br>within the lysosome to its mature form which is required to<br>optimally break down glycogen1<br>cipaglucosidase alfa<br>miglustat<br>1 Selvan et al. 2021, J Biol Chem 2021 Jan-Jun;296:100769<br>ERT: Enzyme Replacement Therapy |
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| 15<br>WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD<br>Number of patients (n)<br>Mean (SD) baseline 6MWD (% predicted):<br>Cipa/mig–cipa/mig = 56.1 (16.03)<br>Alg/pla–cipa/mig = 54.8 (17.68)<br>Mean (SD) week 104 6MWD (% predicted):<br>Cipa/mig–cipa/mig = 60.8 (18.07)<br>Alg/pla–cipa/mig = 54.3 (21.86)<br>6 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>Baseline<br>Week<br>12 26 38 52 64 78 104<br> –1<br> –2<br>0<br>1<br>2<br>3<br>4<br>5<br>Cipa/mig–cipa/mig 62 61 54 58 61 55 56 56<br>Alg/pla–cipa/mig 29 29 27 28 29 27 26 26 Mean (+/– SE) of change from ATB200-03 baseline (% predicted 6MWD)<br>Mean (SD) baseline FVC (% predicted):<br>Cipa/mig–cipa/mig = 67.7 (19.48)<br>Alg/pla–cipa/mig = 67.2 (21.29)<br>Mean (SD) week 104 FVC (% predicted):<br>Cipa/mig–cipa/mig = 66.2 (20.00)<br>Alg/pla–cipa/mig = 61.0 (18.20)<br> –7<br> –5<br> –3<br> –1<br>0<br>1<br>2<br>3 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br> –4<br> –2<br> –6<br>Number of patients (n) Week<br>12 26 38 52 64 78 104<br>Cipa/mig–cipa/mig 62 61 53 54 55 50 51 53<br>Alg/pla–cipa/mig 29 29 26 27 26 25 26 24<br>Baseline<br>Mean (+/– SE) of change from<br>ATB200-03 baseline (sitting % predicted FVC<br>Phase 3 open-label extension study data demonstrate that treatment with AT-GAA up to 2 years<br>was associated with a durable effect, supporting the long-term benefits<br> ERT-experienced and -naïve patients treated with AT-GAA throughout PROPEL<br>showed durable improvements in % predicted 6MWD that were maintained<br>throughout to week 104<br> ERT-experienced and -naïve patients who received alglucosidase alfa/placebo in<br>PROPEL and switched to AT-GAA in the OLE showed stability in % predicted 6MWD<br>throughout the OLE study<br> ERT-experienced patients treated with AT-GAA throughout PROPEL remained stable,<br>while patients who received alglucosidase alfa/placebo experienced a decline in<br>sitting % predicted FVC that stabilized after switching to AT-GAA in the OLE study<br>ERT-Experienced 6MWD (%): Change<br>from baseline<br>ERT-Experienced FVC (%): Change<br>from baseline |
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| 16<br>SSIEM Update – PROPEL Effect Size Analysis in ERT-Experienced Adults<br>Post hoc analysis shows majority of study parameters demonstrated stabilization or improvement<br>after switching from SoC to Pombiliti + Opfolda<br> Significant improvement was only observed for (unadjusted mean<br>CFBL) QoL: PROMIS–Dyspnea (-1.53).<br> Significant worsening was observed for (unadjusted mean CFBL):<br>sitting forced vital capacity (FVC, -4.02%); supine FVC (-2.63%);<br>sitting slow vital capacity (SVC, -6.52%); maximum expiratory<br>pressure (MEP, -3.85%); creatine kinase (CK, 79.6 IU/L); and hexose<br>tetrasaccharide (Hex4, 1.89 mmol/molCr).<br> Significant improvement was observed for (unadjusted mean<br>CFBL): 6MWD (16.89 m); 6MWD % predicted (3.20); manual muscle<br>test (MMT) lower extremities (1.63); MMT upper extremities (1.76);<br>MMT overall score (3.38); CK (-118.0 IU/L); Hex4 (-1.69<br>mmol/molCr); QoL: EuroQol Dimensions-5 Levels Instrument (EQ-5D-5L)–Pain/Discomfort (-0.19); QoL: PROMIS–Fatigue (-1.87); QoL:<br>Subject’s Global Impression of Change (SGIC)–Overall (0.34); QoL:<br>SGIC–Ability to Move Around (0.21); QoL: SGIC–Muscle Function<br>(0.20); QoL: SGIC–Daily Living (0.28); QoL: SGIC–Energy Level (0.33);<br>and QoL: other–Physician’s Global Impression of Change (PGIC)–<br>Overall (0.27).<br> Significant worsening was not observed for any assessments.<br>ERT-experienced patients remaining on alg+pbo(n=30) generally showed worsening (d<-0.2) or stability (-0.2≤d<+0.2) across most outcomes, with significant worsening for various lung function assessments and biomarkers<br>ERT-experienced patients switching to cipa+migmostly showed improvement or stability, with significant improvements for various assessments of motor function, muscle strength, biomarker, and global impression of change scales<br>*<br>Indicates nominal statistical significance; ✝For these endpoints, a negative CFBL value indicated a better result. For visualization purposes, the direction of the results was reversed so that positive CFBL values indicate better results. GSGC, Gait, Stairs, Gowers’ maneuver, and Chair; 16 LOCF, last observation carried forward; MIP, maximum inspiratory pressure, QMT, quantitative muscle test, SNIP, sniff nasal inspiratory pressure, TUG, timed up and go; VAS, visual analogue scale. |
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| 17<br>17 1Data on file<br>Global Regulatory Status<br>Expect regulatory approvals and launch into the three largest Pompe markets in 2023<br> Pombiliti® + Opfolda® now approved in the U.K.<br> Pombiliti® + Opfolda® now approved in the EU<br> Based on recent engagements with the U.S. FDA,<br>approval expected in the coming days<br>17 |
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| 18<br>Ongoing Clinical Studies and Expanded Access Mechanisms<br> Ongoing clinical studies in children and adolescents1 with<br>LOPD and infantile-onset Pompe disease (IOPD)<br> Multiple expanded access mechanisms in place, including in<br>the U.S., U.K., Germany, France, Japan, and others<br> At time of first regulatory approval, ~200 people living with<br>Pompe disease on AT-GAA across extension studies and<br>expanded access programs<br> ~75 centers worldwide currently participating in clinical trials<br>and access programs<br>Advancing science though ongoing clinical studies and providing expanded access<br>through multiple mechanisms<br>1 Children and adolescents aged 0 to <18 years old |
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| 19<br>Pombiliti + Opfolda EU Opportunity<br> Strong indication statement:<br> – Pombiliti® (cipaglucosidase alfa) is a long-term enzyme replacement<br>therapy used in combination with the enzyme stabiliser Opfolda®<br>(miglustat) for the treatment of adults with late-onset Pompe disease<br>(acid α glucosidase [GAA] deficiency)<br> >1,300 patients are estimated to be treated in Europe1<br> – ~60 Patients throughout EU currently on Pombiliti + Opfolda,<br>including ~20 in Germany and Austria<br> Launch underway in Germany<br> – 6 month “free pricing” period and AMNOG reimbursement process<br> – First patients dosed and additional patients scheduled to start infusions<br>1 Amicus data on file from market mapping<br>EU Pompe market currently represents a sizeable market opportunity of $450M+ |
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| 20<br>Launch of Pombiliti + Opfolda Underway in the EU<br>Experienced and passionate rare disease commercial and medical organization<br>supporting early days of launch<br>Access and<br>Reimbursement<br>Positive Interactions<br>with Payors<br>Focus on broad patient access<br>Country-by-country<br>reimbursement process<br>Active discussions<br>to demonstrate value<br>Patient Demand<br>Initial focus on clinical trial<br>and expanded access patients<br>First patients dosed; Multiple<br>scheduled for infusion<br>On-track to transition all trial<br>and expanded access patients<br>in Germany within 90 days<br>KOL and Patient<br>Outreach<br>Promotion and Education<br>Efforts<br>Existing relationships with<br>HCPs at key treatment centers<br>Engaging top prescribers<br>within first 30 days<br>Ongoing disease education<br>Performance |
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| 21<br>Corporate Outlook<br>Delivering on our mission for patients and<br>shareholders |
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| Financial Outlook and Path to Profitability<br>Clear strategy to build our business, advance our portfolio, and achieve profitability<br>22<br>Sustain Revenue<br>Growth<br>Deliver on<br>Financial Goals<br>Secure Approvals<br>of AT-GAA<br>$180.8M 1H 2023 revenue,<br>+16% YoY<br>operational growth<br>2023 Galafold revenue<br>growth guidance of<br>+14-18% YoY at CER<br>Galafold and<br>AT-GAA expected to<br>drive strong double-digit growth long term<br>Focused on prudent<br>expense management<br>Achieve profitability1<br>in 2H 2023<br>1 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, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on impairment of assets, restructuring charges, and income taxes.<br>2023 non-GAAP operating<br>expense guidance of<br>$330M-$350M |
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| 23<br>Positioned for Significant Value Growth<br>Focused on execution and driving sustainable double-digit revenue growth on path to profitability<br>Continue to<br>bring Galafold®<br>to as many<br>patients as<br>possible, sustain<br>double-digit<br>operational<br>revenue growth<br>Successful<br>launch of AT-GAA for people<br>living with<br>Pompe disease<br>Advance<br>next-generation<br>gene therapies in<br>Fabry and<br>Pompe diseases<br>Fully leverage<br>global<br>capabilities and<br>infrastructure as<br>a leader in rare<br>diseases<br>Achieve<br>non-GAAP<br>profitability<br>in 2H 20231<br>1 Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. Non-GAAP Net Income defined as GAAP Net Income excluding the impact of share-based<br>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, restructuring charges and income taxes. |
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| 24<br>True Measure of Success:<br>Impacting the Lives of People Living with Rare Diseases<br>YE17 2023+<br> >2,000 Patients* Thousands of Patients*<br>YE22<br> >350 Patients*<br>*Clinical & commercial, all figures approximate |
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| Appendix |
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| 26<br>Appendix |
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| 27<br>2023 Environmental, Social, & Governance (ESG) Snapshot<br>Board of Directors<br>Committed to ongoing Board refreshment and diversity of<br>background, gender, skills, and experience:<br>80% Board<br>Independence<br>60% Overall Board<br>Diversity<br>Address a rare genetic disease<br>First-in-class or best-in-class<br>Impart meaningful benefit for<br>patients<br>484<br>Global Employees<br>57%<br>% Female Employees<br>Who We Serve Our mission is to<br>drive sustainability<br>with our partners<br>by incorporating<br>environmental and<br>sustainability<br>principles into all<br>our commercial<br>relationships<br>Pledge for a Cure<br>Designate a portion of product revenue back into<br>R&D for that specific disease until there is a cure.<br>Programs we<br>invest in<br>have 3 key<br>characteristics<br>3 Female<br>2 Veteran Status<br>1 African American<br>Director Diversity<br>% Hiring Slate Diversity 97%<br>Leverage employee capabilities and expertise to provide a<br>culture that drives performance and ultimately attracts,<br>energizes, and retains critical talent.<br>Employee Recruitment,<br>Engagement, & Retention<br>Pulse surveys reveal employees feel high personal<br>satisfaction in their job, are proud of their work<br>and what they contribute to the community<br>Career Development<br>Reimagined performance management process to<br>measure the what and the how, rewarding those who<br>role-model our Mission-Focused Behaviors.<br>Committed to producing<br>transformative medicines for patients<br>while practicing environmental<br>responsibility and adhering to<br>sustainability best practices in our<br>operations.<br>Environmental<br>Management<br>0% Amicus Owned Direct Manufacturing and<br>Related GHG Emissions<br>Diversity, Equity, &<br>Inclusion (DEI)<br>Pledge to support a more inclusive culture to impact<br>our employees, our communities, and society.<br>Goal of maintaining gender diversity and<br>increasing overall diversity throughout<br>our global workforce.<br>580<br>Volunteer hours<br>(U.S.):<br>22<br>Amicus supported<br>community programs:<br>79 patients /19countries<br>Expanded Access through Feb 2023:<br>Pricing PROMISE<br>Contributions allocated:<br>$2,288,998 U.S.<br>$954,349 Intl.<br>Charitable Giving<br>Committed to never raising the annual price of our<br>products more than consumer inflation. |
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| 28<br>WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD<br>Improvement from the PROPEL baseline in % predicted 6MWD for the cipa/mig group was<br>maintained throughout the OLE for ERT-experienced and ERT-naïve patients<br> ERT-experienced and -naïve patients treated with cipa/mig throughout showed durable improvements in % predicted 6MWD in PROPEL that were maintained<br>throughout the OLE to week 104<br> ERT-experienced and -naïve patients who received alg/pla in PROPEL and switched to cipa/mig in the OLE showed stability in<br>% predicted 6MWD throughout the OLE<br>*Excludes one outlier. SE, standard error.<br>Number of patients (n)<br>Mean (SD) baseline 6MWD (% predicted):<br>Cipa/mig–cipa/mig = 56.1 (16.03)<br>Alg/pla–cipa/mig = 54.8 (17.68)<br>Mean (SD) week 104 6MWD (% predicted):<br>Cipa/mig–cipa/mig = 60.8 (18.07)<br>Alg/pla–cipa/mig = 54.3 (21.86)<br>6 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>Baseline<br>Week<br>12 26 38 52 64 78 104<br>Number of patients (n)<br>Baseline<br>Week<br>12 26 38 52 64 78 104<br>Mean (SD) baseline 6MWD (% predicted):<br>Cipa/mig–cipa/mig = 61.9 (15.33)<br>Alg/pla–cipa/mig = 61.4 (17.11)<br>Mean (SD) week 104 6MWD (% predicted):<br>Cipa/mig–cipa/mig = 70.9 (16.31)<br>Alg/pla–cipa/mig = 70.3 (8.57)<br> –2<br> –4<br>0<br>2<br>4<br>6<br>8<br>12<br>10<br>14<br>16<br> –1<br> –2<br>0<br>1<br>2<br>3<br>4<br>5<br>Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>Cipa/mig–cipa/mig 62 61 54 58 61 55 56 56<br>29 27 26 26<br>Cipa/mig–cipa/mig 20 19 17 20 20 18 19 18<br>Alg/pla–cipa/mig 29 29 27 28 Alg/pla–cipa/mig 7 7 6 7 7 5 6 7 Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (% predicted 6MWD)<br>Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (% predicted 6MWD)<br>ERT experienced ERT naïve* |
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| 29<br>WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD<br>Sitting % predicted FVC remained stable in ERT-experienced and ERT-naïve patients throughout<br>the OLE for both PROPEL treatment groups<br> ERT-experienced patients treated with cipa/mig throughout remained stable, while patients who received alg/pla in PROPEL experienced a decline in sitting %<br>predicted FVC that stabilized after switching to cipa/mig in the OLE<br> ERT-naïve patients in both treatment groups experienced some decline in PROPEL that stabilized in the OLE with no further decline in FVC to week 104<br>Mean (SD) baseline FVC (% predicted):<br>Cipa/mig–cipa/mig = 67.7 (19.48)<br>Alg/pla–cipa/mig = 67.2 (21.29)<br>Mean (SD) week 104 FVC (% predicted):<br>Cipa/mig–cipa/mig = 66.2 (20.00)<br>Alg/pla–cipa/mig = 61.0 (18.20)<br> –7<br> –5<br> –3<br> –1<br>0<br>1<br>2<br>3 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br> –4<br> –2<br> –6<br>Mean (SD) baseline FVC (% predicted):<br>Cipa/mig–cipa/mig = 80.2 (18.69)<br>Alg/pla–cipa/mig = 79.1 (22.58)<br>Mean (SD) week 104 FVC (% predicted):<br>Cipa/mig–cipa/mig = 73.5 (21.24)<br>Alg/pla–cipa/mig = 79.6 (17.92)<br> –7<br> –10<br> –4<br>0<br> –6<br> –9<br> –2<br>1<br> –5<br> –8<br> –1<br>2<br>3 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>104<br> –3<br>Number of patients (n) Week<br>12 26 38 52 64 78 104<br>Number of patients (n) Week<br>12 26 38 52 64 78<br>Cipa/mig–cipa/mig 62 61 53 54 55 50 51 53<br>26 25 26 24<br>Cipa/mig–cipa/mig 20 18 18 17 19 18 18 17<br>Alg/pla–cipa/mig 29 29 26 27 Alg/pla–cipa/mig 7 7 5 5 6 4 4 5<br>Baseline Baseline<br>ERT experienced ERT naïve*<br>Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (sitting % predicted FVC<br>Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (sitting % predicted FVC<br>*Excludes one outlier. |
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| 30<br>WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD<br>Cipa/mig treatment was associated with a durable reduction in serum CK during PROPEL and the<br>OLE in both ERT-experienced and ERT-naïve patients<br> ERT-experienced and -naïve patients treated with cipa/mig throughout showed a decline in serum CK levels during PROPEL that was maintained throughout the<br>OLE<br> ERT-experienced and -naïve patients who received alg/pla in PROPEL showed a slight increase or stability in serum CK levels to week 52, and a marked decline after<br>switching to cipa/mig in the OLE<br>Number of patients (n)<br>Mean (SD) baseline CK (U/L):<br>Cipa/mig–cipa/mig = 433.0 (407.69)<br>Alg/pla–cipa/mig = 490.6 (450.46)<br>Mean (SD) week 104 CK (U/L):<br>Cipa/mig–cipa/mig = 299.7 (296.92)<br>Alg/pla–cipa/mig = 361.6 (292.67)<br> –300<br> –250<br> –200<br> –150<br> –100<br> –50<br>0<br>50<br>100 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>2 4 6 12 26 38 52 54 56 58 64 78 104<br>Number of patients (n)<br>Mean (SD) baseline CK (U/L):<br>Cipa/mig–cipa/mig = 464.1 (398.11)<br>Alg/pla–cipa/mig = 680.3 (333.13)<br>Mean (SD) week 104 CK (U/L):<br>Cipa/mig–cipa/mig = 283.6 (212.49)<br>Alg/pla–cipa/mig = 461.7 (196.76)<br> –400<br> –500<br> –300<br> –200<br> –100<br>200 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>2 4 6 12 26 38 52 54 56 58 64 78 104<br>100<br>0<br>Cipa/mig–cipa/mig 62 62 62 58 61 58 57 59 54 52 53 59 57 49 Cipa/mig–cipa/mig 20 20 19 20 19 19 20 18 19 19 16 18 19 18<br>Alg/pla–cipa/mig 29 29 28 29 29 29 28 28 28 26 27 27 26 25 Alg/pla–cipa/mig 7 7 7 7 6 7 7 7 7 5 7 7 7 7 Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (U/L)<br>Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (U/L)<br>ERT experienced ERT naïve*<br>Baseline Baseline<br>Week Week<br>*Excludes one outlier. |
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| 31<br> ERT-experienced patients treated with cipa/mig throughout experienced a decline in urine Hex4 levels in PROPEL that stabilized during the OLE. ERT-experienced<br>patients who received alg/pla in PROPEL experienced an increase in Hex4 and a marked decline after switching to cipa/mig in the OLE<br> ERT-naïve patients experienced a decline in Hex4 levels during PROPEL in both treatment groups that stabilized or declined further during the OLE to week 104<br>Number of patients (n)<br>Mean (SD) baseline Hex4 (mmol/mol creatinine):<br>Cipa/mig–cipa/mig = 4.5 (3.49)<br>Alg/pla–cipa/mig = 7.3 (7.72)<br>Mean (SD) baseline Hex4 (mmol/mol creatinine):<br>Cipa/mig–cipa/mig = 2.7 (1.60)<br>Alg/pla–cipa/mig = 5.0 (5.21)<br> –4<br> –3<br> –2<br> –1<br>0<br>1<br>2<br>3<br>4 Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>Week<br>2 4 6 12 26 38 52 54 56 58 64 78 104<br>Number of patients (n)<br>Mean (SD) baseline Hex4 (mmol/mol creatinine):<br>Cipa/mig–cipa/mig = 4.8 (3.00)<br>Alg/pla–cipa/mig = 5.8 (2.50)<br>Mean (SD) baseline Hex4 (mmol/mol creatinine):<br>Cipa/mig–cipa/mig = 2.0 (1.28)<br>Alg/pla–cipa/mig = 2.4 (1.16)<br> –4<br> –5<br> –3<br> –2<br> –1<br>0<br>Cipa/mig–cipa/mig Cipa/mig Alg/pla<br>Week<br>2 4 6 12 26 38 52 54 56 58 64 78 104<br>Cipa/mig–cipa/mig 61 61 61 60 61 57 58 55 54 51 56 59 55 56 Cipa/mig–cipa/mig 20 20 19 19 19 18 18 17 18 19 16 19 19 17<br>Alg/pla–cipa/mig 29 29 29 29 29 28 28 28 27 26 28 27 27 26 Alg/pla–cipa/mig 7 7 7 7 7 7 7 5 7 5 7 6 6 6 Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (mmol/mol creatinine)<br>Mean (+/<br> – SE) of change from<br>ATB200-03 baseline (mmol/mol creatinine)<br>WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD<br>Cipa/mig treatment was associated with a durable reduction in urine Hex4 during PROPEL and the<br>OLE in both ERT-experienced and ERT-naïve patients<br>Baseline Baseline<br>ERT experienced ERT naïve*<br>*Excludes one outlier. |
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| Thank you |
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