UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT PURSUANT TO
SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date of Report (Date of
earliest event reported):
(Exact Name of Registrant as Specified in Its Charter)
(State or Other Jurisdiction of Incorporation) |
(Commission File Number) |
(I.R.S. Employer Identification No.) |
(Address of Principal Executive Offices, and Zip Code)
Registrant’s Telephone 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 of each class | Trading Symbol(s) | Name of each exchange on which registered |
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 8.01 – Other Events
On September 14, 2022, Amicus Therapeutics, Inc. (the “Company”) released presentation materials that it will be using in meetings with investors and analysts. A copy of the presentation materials is attached hereto as Exhibit 99.1 and is incorporated herein by reference.
Item 9.01 Financial Statements and Exhibits
(d) Exhibits:
| Exhibit No. | Description | |
| 99.1 | September 14, 2022 Presentation Materials | |
| 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 14, 2022 | By: | /s/ Ellen S. Rosenberg | |
| Name: Ellen S. Rosenberg | |||
| Title: Chief Legal Officer and Corporate Secretary | |||
Exhibit 99.1
Corporate Overview September 2022 At the Forefront of Therapies for Rare Diseases
2 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, expenses, cash position, and future profitability 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 in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, 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 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, manufacturing and launch preparations . Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results . With respect to statements regarding projections of the Company's revenue, expenses, cash position, and future profitability, 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 Form 10 - Q for the quarter ended June 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 . 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 .
3 A Rare Company Patient - dedicated, Rare Disease Biotechnology Company with Sustained Double - digit Revenue Growth, a Global Commercial Infrastructure, and Late - stage Development Capabilities AT - GAA a Two - component Therapy Under Global Regulatory Reviews for Pompe Disease GLOBAL COMMERCIAL ORGANIZATION World - class CLINICAL DEVELOPMENT Capabilities EMPLOYEES in 27 Countries GALAFOLD & AT - GAA Gene Therapy PLATFORM Leveraging Experience in Protein Engineering & Glycobiology $350M - $365M FY22 Global Galafold Revenue at CER Cumulative $2B Peak Potential $386.8M Cash as of 6/30/22 Non - GAAP PROFITABILITY expected in 2023
2022 Strategic Priorities to Drive Value 1 2 3 4 5 Double - digit Galafold growth (15 - 20%) with revenue of $350M to $365M at CER 1 Secure FDA approval and positive CHMP opinion for AT - GAA Initiate successful, rapid launch in U.S. for AT - GAA Advance best - in - class, next - generation genetic medicines and capabilities Maintain strong financial position on path to profitability 4 1 CER: Constant Exchange Rates; 2022 Galafold revenue guidance is based on the average exchange rates for 2021
5 INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL FABRY FRANCHISE Galafold ® (migalastat) Fabry Gene Therapy Next - Generation Chaperone POMPE FRANCHISE AT - GAA (cipaglucosidase alfa + miglustat) Pompe Gene Therapy OTHER CLN3 Batten Disease Gene Therapy Next - Generation Research Programs Amicus Pipeline Streamlined Rare Disease Pipeline with Focus on Fabry Disease and Pompe Disease ODD ODD BTD ODD - Orphan Drug Designation BTD - Breakthrough Therapy Designation
6 Positioned for Significant Value Growth Focused on Execution and Driving Sustainable Double - digit Revenue Growth on Path to Profitability Continue to bring Galafold ® to as many patients as possible, sustain double - digit revenue growth Successful launch of AT - GAA for people living with Pompe disease Advance next - generation gene therapies in Fabry and Pompe diseases Fully leverage global capabilities and infrastructure as a leader in rare diseases Achieve non - GAAP profitability in 2023
7 Galafold® (migalastat) Continued Growth … building a leadership position in the treatment of Fabry disease
8 Global Fabry Market ▪ Fabry Disease is believed to be significantly underdiagnosed – Newborn screening studies suggest Fabry could be one of the more prevalent human genetic diseases (~1:1,000 to ~1:4,000 incidence) ▪ In 2021, Galafold was the fastest growing medicine for Fabry disease and the greatest contributor to Fabry market growth – Introduction of Galafold has led to market expansion with 800+ naive patients diagnosed and treated for the first time Global Fabry Disease Market Growth Continues to be Driven by Diagnosing New Patients in Addition to the Introduction of Galafold (millions) 1 Global market measured by reported sales of approved therapies for Fabry disease – 2025 sales projected using 8% CAGR $0 $500 $1,000 $1,500 $2,000 $2,500 $3,000 2017 2018 2019 2020 2021E 2022E 2023E 2024E 2025E Global Fabry market to exceed $1.9B in 2021 and tracking toward ~$2.6B by 2025 1
9 Galafold Success (as of June 30, 2022) Building on Galafold’s Success and Leveraging Leadership Position to Drive Continued Growth Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable variant. The most common adverse re actions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, inclu din g 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 . Galafold is the first and only approved oral treatment option with a unique mechanism of action for Fabry patients with amenable variants 350 Amenable Variants in U.S. Label 40+ Countries with Regulatory Approvals Continued Geographic Expansion in 2022 $ 80.7M 2Q22 Galafold Revenue $ 350M - $ 365M 2022 Galafold Revenue Guidance at CER 1,384 Amenable Mutations in EU Label
10 Galafold Performance 1H22 Reported Revenue Growth of +10.9% to $159.4M - Operational Growth of +17.8% at CER ▪ Global 3 - month net new patients trend greater than 6 - month and 12 - month ▪ In the U.S., the month of June saw highest net new patients and PRFs since April 2021 ▪ Global mix of switch (~55%) and previously untreated patients (~45%) ▪ Compliance and adherence over 90%+ ▪ Expect non - linear quarterly growth to continue due to uneven ordering patterns $143.8M $159.4M - 6.9% +17.8% 1H21 Operational Growth FX Impact 1H22 Year - over - Year Sales Growth
11 Penetration of the diagnosed untreated population Increase in newborn screening and diagnostic initiatives Strong IP rights, including COM protection through 2038 Continued penetration into existing markets Expansion into new geographies Broadening of labels Galafold Growth Opportunity $1B Annual Sales Opportunity at Peak Sustained double - digit revenue growth: Near - term growth to $500M driven by: Long - term growth towards peak sales potential driven by: 1H operational revenue growth of +17.8% COM: Composition of Matter
12 Galafold Initiatives Building the Body of the Evidence around Galafold Broadening Labels: Adolescents and Additional Variants Publications and Medical Presentations Ongoing and Planned Phase IV Studies Over 500 Patients Enrolled in a Global Registry Strengthening our IP Portfolio
13 AT - GAA (cipaglucosidase alfa + miglustat) … potential to establish a new standard of care for people living with Pompe disease
14 Deficiency of GAA leading to lysosomal glycogen accumulation and cellular dysfunction Age of onset ranges from infancy to adulthood Symptoms include muscle weakness, respiratory failure, and cardiomyopathy Respiratory and cardiac failure are leading causes of morbidity and mortality Estimated incidence of ~1:28,000; newborn screening suggests significant underdiagnosis ~$1.2B+ global Pompe ERT sales 1 Majority of patients on current standard of care decline after ~2 years Pompe Disease Overview 1. Based on 12 months ended December 31, 2021. Source: Sanofi Press Release Pompe is a Severe and Fatal Neuromuscular Disease Caused by the Deficiency of Lysosomal Enzyme GAA
15 AT - GAA: An Innovative Approach to Pompe Disease Our Scientists Created a Uniquely Glycosylated and Highly Phosphorylated ERT (ATB200) that Significantly Enhances Targeting to Key Affected Muscles ▪ AT - GAA is a two - component therapy combining ATB200, an ERT, with AT2221, an orally administered enzyme stabilizer ▪ Consists of a naturally occurring cell line that can be properly processed within the lysosome to its mature form which is required to optimally break down glycogen 1 ATB200 AT2221 1 Selvan et al. 2021, J Biol Chem 2021 Jan - Jun;296:100769 ERT: Enzyme Replacement Therapy
16 Phase 3 PROPEL Study Results Primary, Key Secondary and Biomarker Endpoint Heat Map Endpoints Across Motor Function, Pulmonary Function, Muscle Strength, PROs, and Biomarkers Favored AT - GAA over alglucosidase alfa
17 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 Patients 6MWD=6 - minute walk distance;; FVC=forced vital capacity; SE=standard error. P values are nominal 2 - sided; FVC data normally distributed and 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)
18 Phase 3 PROPEL Topline Results: ERT Experienced Population (n=95) ERT Experienced Patients Treated with AT - GAA Demonstrated Improvements over Time in 6MWD and Stabilization over Time in FVC Versus alglucosidase alfa NOTE: Baseline is Mean (STDEV); CFBL is Mean (SE); P - values are nominal 2 - sided; FVC data normally distributed and p – values are from ANCOVA 6MWD data not normally distributed and 6MWD p - value is for non - parametric ANCOVA; 6MWD parametric MMRM p - value was p=0.078 6MWD (m): Change from baseline (n=65, n=30) FVC (% predicted): Change from baseline (n=65, n=30)
19 Phase 3 PROPEL Study Publication Clinically Meaningful Outcomes from Phase 3 PROPEL Study Provide the Basis for Global Regulatory Submissions of AT - GAA ▪ Peer - reviewed results from PROPEL suggest that treatment with AT - GAA provided clinically meaningful improvements over standard of care, including ERT - experienced patients with high unmet need ▪ The authors deemed AT - GAA to provide a differentiated mechanism of action and potential alternative treatment option for people living with late - onset Pompe Disease Schoser et al. 2021 , The Lancet Neurology, Volume 20, Issue12, P1027 - 1037
20 Long - Term Data from Phase 1/2 Clinical Study (ATB200 - 02) Persistent and Durable Improvements in Motor and Respiratory Function and Reductions in Biomarkers of Muscle Damage and Disease Substrate Observed in Patients out to 36 Months NOTE: * One patient in the ERT - naïve cohort experienced a large drop in % predicted FVC at month 21, which returned to previous levels at the following visit (month 24). CFBL in 6MWD in (A) ERT - Experienced and (B) ERT - Naïve Patients CFBL in FVC in (A) ERT - Experienced and (B) ERT - Naïve Patients
21 Global Pompe Market Global Pompe Disease Market Growth Continues to be Driven by the Diagnosis of New Patients - Only One Approved Therapy on the Market up until 2021 ▪ Pompe Disease believed to be significantly underdiagnosed – Newborn screening studies suggests Pompe to be more prevalent than medical literature suggest (~1:10,000 to ~1:30,000) – Newborn screening already occurring in 27 U.S. states with 9 additional U.S. states pursuing NBS implementation for Pompe disease Global Pompe Market to exceed $1.1B in 2021 and tracking toward $1.5B+ by 2025 1 (millions) $0 $200 $400 $600 $800 $1,000 $1,200 $1,400 $1,600 $1,800 2017 2018 2019 2020 2021 2022 2023 2024 2025 1 Global market measured by reported sales of approved therapies for Pompe disease – 2025 sales projected using 8% CAGR
22 AT - GAA: Key Takeaways ▪ U.S. Regulatory status update: – U.S. PDUFA date 2H2022 1 subject to completion of a manufacturing inspection, which has not yet been scheduled – Negotiations substantially complete for draft label ▪ International Regulatory status update: – CHMP opinion expected 4Q2022 – Planning for additional regulatory submissions ▪ Multiple expanded access mechanisms in place, including in the U.S., U.K., Germany, France, Japan, and others – First reimbursed access through the French compassionate access program ▪ 175+ people living with Pompe disease are now on AT - GAA across our clinical extension studies and expanded access programs ▪ Ongoing supportive studies: – LOPD in children and adolescents aged 0 to <18; Infantile - Onset Pompe Disease (IOPD) Focused on Advancing AT - GAA to as Many Patients as Possible through Global Regulatory Pathways and Expanded Access Mechanisms 1 FDA PDUFA date of October 29, 2022 for cipaglucosidase alfa BLA; The Company expects the FDA to approve the NDA and BLA appli cat ions together
23 AT - GAA Launch Preparations Experienced and Passionate Rare Disease Medical and Commercial Organization Poised for Second Successful Launch Key Strengths Commitment to patient access Clear focus on launch Identification of key Pompe disease treatment centers Development of educational materials Planning Access Education Team Great experience and passion Eagerness to introduce a new therapy upon approvals Highly leverageable team in place, few new hires needed Published Phase 3 PROPEL data in The Lancet Neurology Active medical conference and publication schedule Multiple Expanded Access Programs in place Continued education on biology of disease and diagnosis Demonstrating value to payors including parity pricing strategy
24 Financial & Operational Strategy … maintaining a strong financial outlook
25 Revenue Performance Q2 Revenue Growth of +4.3% to $80.7M resulting from Strong Operational Growth of +12.9% at CER Offset by Negative FX impact of - 8.6% $77.4M $80.7M - 8.6% +12.9% 2Q21 Operational Growth FX Impact 2Q22 Year - over - Year Sales Growth ▪ Significant currency exposure as 66% of Galafold revenue generated outside the U.S. ▪ Applying average July 2022 exchange rates, the negative FX impact on full - year 2022 Galafold ® reported sales would be approximately - 9%, or ~$26 million.
Financial Outlook and Path to Profitability Clear Strategy to Build Our Business, Advance Our Portfolio, and Achieve Profitability 26 Sustain Galafold Revenue Growth Deliver on Financial Goals Secure Approvals of AT - GAA $159.4M 1H2022 revenue, +17.8% YoY Operational Growth 2022 Galafold revenue guidance of $350M - $365M at CER, +15 - 20% YoY Growth Galafold and AT - GAA expected to drive strong double - digit growth long term Focused on prudent expense management Achieve profitability 1 in 2023 1 Based on projections of Amicus non - GAAP Net Income under current operating plans, which includes successful AT - GAA regulatory ap provals 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. 2022 non - GAAP operating expense guidance of $470M - $485M
27 True Measure of Success: Impacting the Lives of Patients Living with Rare Diseases YE17 2023+ Thousands of Patients* >1,900+ Patients* YE21 >350 Patients* *Clinical & commercial, all figures approximate
Thank You
Appendix
30 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 US $832,976 Intl. 770 Volunteer hours (US): 20+ Amicus supported community programs: 80% Board Independence 60 % Overall Board Diversity Address a rare genetic disease First - in - class or best - in - class Impart meaningful benefit for patients 496 Global Employees 58% % female employees Who We Serve Environmental Management Green building design Energy & water conservation Hazardous waste management Pledge for a Cure Designate 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 characteristics: Committed 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 Female 2 Veteran Status 1 African American Director 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, and Retention Pulse surveys reveal employees feel high personal satisfaction in their job, are proud of their work and what they contribute to the community Charitable Giving 52 patients / 18 countries Expanded Access through 2021: Career Development Reimagined performance management process to measure the what and the how, rewarding those who role - model our Mission - Focused Behaviors .
31 Appendix