UNITED STATES
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WASHINGTON, D.C. 20549
FORM
CURRENT REPORT PURSUANT TO
SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
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Item 2.02 – Results of Operations and Financial Condition
On March 1, 2021, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal year ended December 31, 2020. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on March 1, 2021 to discuss its full year results of operations. A copy of the conference call presentation materials is attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.
In accordance with General Instruction B.2. of Form 8-K, the information in this Current Report on Form 8-K and the Exhibits 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 liability of that section, nor shall it be deemed 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 March 1, 2021 |
| 99.2 | March 1, 2021 Conference Call 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: March 1, 2021 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
Amicus Therapeutics Announces Full-Year 2020 Financial Results and Corporate Updates
2020 Total Galafold Revenue of $260.9M; Increased 43 Percent Year-on-Year
Continued Strong Global Growth of Galafold Expected in 2021 with Revenue of $300M-$315M
AT-GAA Rolling BLA Submission in Pompe Disease Planned for Completion in 2Q21 and Other Global Submissions Expected Throughout 2021
CLN6 Batten Disease Gene Therapy Granted Fast Track Designation by U.S. FDA
Cash Position Sufficient to Achieve Self-Sustainability
Conference Call and Webcast Today at 8:30 a.m. ET
PHILADELPHIA, PA, Mar. 1, 2021 – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced financial results for the full year ended December 31, 2020.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “During 2020, Amicus remained resilient on our journey to becoming a leading global rare disease biotechnology company. Despite the challenges of COVID, Amicus emerged from 2020 a better and stronger company organizationally, strategically, scientifically and financially. Galafold continues its path to becoming the worldwide standard of care for Fabry patients with amenable variants. And we are steadfast and passionate in our commitment to advancing AT-GAA to global regulatory submissions as fast as possible for the benefit of all people living with Pompe disease. The data from the Phase 3 PROPEL study we believe continue to show the overwhelmingly positive benefits of AT-GAA compared to the only approved medicine in this devastating disease. And the unmet need in Pompe is so great. We have the team, the resources and the programs that strongly position us to achieve our vision of delivering groundbreaking new medicines and hopefully, one day, cures for people living with rare diseases.”
Corporate Highlights
| · | Global revenue for Galafold® (migalastat) in the full year of 2020 was $260.9 million. Full year revenue represented a year-over-year increase of 43% from total revenue of $182.2 million in the full year of 2019. On a constant currency basis, full year 2020 total revenue was $258.6 million, representing operational revenue growth measured at constant currency exchange rates of 42%, which was further benefited by a positive currency impact of $2.3 million, or 1%. Galafold performance was driven largely by strong patient demand. Global compliance and adherence rates continue to exceed 90%. |
| · | Results from the global Phase 3 PROPEL clinical study of AT-GAA in late-onset Pompe disease (LOPD) were presented at the 17th Annual WORLDSymposium™ 2021. The Company plans to complete the BLA submission in the second quarter of this year and anticipates additional regulatory submissions in the European Union and in other geographies throughout 2021. |
| · | The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to the CLN6 Batten disease gene therapy, AT-GTX-501. The Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions, which have the potential to address unmet medical needs. A drug development program with Fast Track designation is afforded greater access to the FDA for the purpose of expediting the drug's development, review and potential approval. |
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| · | Initial clinical data from the Phase 1/2 CLN3 gene therapy study were presented at the 17th Annual WORLDSymposium™ 2021. Results suggest early signs of disease stabilization and the potential to slow the neurological disease progression in children living with CLN3 Batten disease. Regulatory interactions are ongoing and the Company expects to start the next clinical study in the second half of this year. |
| · | Preclinical data from the Company’s Fabry disease gene therapy clinical candidate, AT-GTX-701, presented at the 17th Annual WORLDSymposium™ 2021. As part of the research collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn), initial data from the Fabry AAV gene therapy with an engineered GLA transgene improved for stability demonstrated greater substrate reduction than wild type constructs across all tissues and doses. These findings further validate the synergies of combining Amicus-engineered transgenes with Penn’s AAV technologies to develop next generation gene therapies. |
| · | Cash position sufficient to achieve self-sustainability without the need for any future dilutive financings. The Company continues to carefully manage expenses and investments, while executing on the Galafold launch, proceeding with AT-GAA global regulatory submissions and advancing development programs. |
Full Year 2020 Financial Results
| · | Total revenue in the full year 2020 was $260.9 million, a year-over-year increase of 43% from total revenue of $182.2 million in the full year of 2019. On a constant currency basis, full year 2020 total revenue was $258.6 million, representing operational revenue growth measured at constant currency exchange rates of 42%. | |
Reported revenue was aided by a positive currency impact of $2.3 million, or 1%.
| · | Cash, cash equivalents, and marketable securities totaled $483.3 million at December 31, 2020, compared to $452.7 million at December 31, 2019. | |
| · | Total GAAP operating expenses of $476.8 million for the full year 2020 increased as compared to $464.3 million for the full year 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs. | |
| · | Total non-GAAP operating expenses of $415.7 million for the full year of 2020 increased as compared to $411.8 million in the full year of 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs.1 | |
| · | Net loss was $276.9 million, or $1.07 per share, compared to a net loss of $356.4 million, or $1.48 per share, for the full year 2019. |
1 Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for all reporting periods appear in the tables to this press release.
2021 Financial Guidance
| · | For the full-year 2021, the Company anticipates total Galafold revenue of at least $300-$315 million. Double-digit revenue growth in 2021 is expected to be driven by continued operational growth and commercial execution across all major markets, including the U.S., EU, U.K. and Japan. | |
| · | Non-GAAP operating expense guidance for the full-year 2021 is $410 million to $420 million, driven by continued investment in the global Galafold launch, AT-GAA clinical studies and pre-launch activities, and advancing our gene therapy pipeline.2 | |
| · | Cash, cash equivalents, and marketable securities totaled $483.3 million at December 31, 2020. Based on current operating models, the Company believes that the current cash position, which includes the net proceeds from the 2020 Senior Secured Term Loan, and expected future revenues are sufficient to fund the Company’s operations and ongoing research programs through to self-sustainability. |
2 A reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure is not available without unreasonable effort due to high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure.
Anticipated 2021 Milestones by Program
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
| · | Continued revenue growth in 2021 |
| · | Plans to expand EU label to cover adolescent population |
| · | Continued geographic expansion |
| · | Registry and other Phase 4 studies |
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AT-GAA for Pompe Disease
| · | Complete the BLA submission in 1H2021 and the EU MAA submission to be completed in 2H2021 |
| · | Ongoing supportive studies, including pediatric and extension studies |
Gene Therapy Portfolio
| · | Advance manufacturing and regulatory discussions for the CLN6 Batten disease gene therapy program and begin dosing additional patients with GMP grade material |
| · | Report initial data from the CLN3 Batten disease Phase 1/2 study, advance manufacturing and regulatory discussions, and submit protocol for next clinical study to the IND |
| · | Continue to progress IND-enabling work in both Pompe and Fabry gene therapy |
| · | Disclose additional preclinical data and potential IND candidate declarations across multiple preclinical programs |
| · | Manufacturing advancements and updates across the portfolio |
Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, March 1, 2021 at 8:30 a.m. ET to discuss the full year 2020 financial results and corporate updates. Interested participants and investors may access the conference call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international), conference ID: 6959755.
A live audio webcast and related presentation materials can also be accessed via the Investors section of the Amicus Therapeutics corporate website at ir.amicusrx.com. Web participants are encouraged to register on the website 15 minutes prior to the start of the call. A replay of the call will be available for seven days beginning at 11:30 a.m. ET on March 1, 2021. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 6959755.
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 over 40 countries around the world, including the U.S., EU, U.K., Japan and others.
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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 0–15 years of age have not yet been established. |
| · | 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. |
| · | 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. |
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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 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 metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic 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. Full reconciliations of GAAP results to the comparable non-GAAP measures for the reported periods appear in the financial tables section of this press release. 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 Statements
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, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, and revenue goals, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations and/or revenue from the COVID-19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID-19, such as travel bans, shelter in place orders and third-party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product or to treatment sites. In addition to the impact of the COVID-19 pandemic, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, UK, 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, commercialization and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding corporate financial guidance and financial goals and the attainment of such goals and statements regarding projections of the Company's revenue and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2020 to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
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CONTACTS:
Investors:
Andrew Faughnan
Sr. Director, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Diana Moore
Head of Global Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
FOLD–G
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TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
| Years Ended December 31, | ||||||||||||
| 2020 | 2019 | 2018 | ||||||||||
| Net product sales | $ | 260,886 | $ | 182,237 | $ | 91,245 | ||||||
| Cost of goods sold | 31,044 | 21,963 | 14,404 | |||||||||
| Gross profit | 229,842 | 160,274 | 76,841 | |||||||||
| Operating expenses: | ||||||||||||
| Research and development | 308,443 | 286,378 | 270,902 | |||||||||
| Selling, general, and administrative | 156,407 | 169,861 | 127,200 | |||||||||
| Changes in fair value of contingent consideration payable | 3,144 | 3,297 | 3,300 | |||||||||
| Depreciation and amortization | 8,846 | 4,775 | 4,216 | |||||||||
| Total operating expenses | 476,840 | 464,311 | 405,618 | |||||||||
| Loss from operations | (246,998 | ) | (304,037 | ) | (328,777 | ) | ||||||
| Other (expense) income: | ||||||||||||
| Interest income | 3,226 | 10,249 | 10,461 | |||||||||
| Interest expense | (22,425 | ) | (18,872 | ) | (22,402 | ) | ||||||
| Loss on exchange of convertible notes | — | (40,624 | ) | — | ||||||||
| Loss on extinguishment of debt | (7,276 | ) | — | — | ||||||||
| Change in fair value of derivatives | — | — | (2,739 | ) | ||||||||
| Other expense | (781 | ) | (2,626 | ) | (5,632 | ) | ||||||
| Loss before income tax | (274,254 | ) | (355,910 | ) | (349,089 | ) | ||||||
| Income tax (expense) benefit | (2,598 | ) | (478 | ) | 94 | |||||||
| Net loss attributable to common stockholders | $ | (276,852 | ) | $ | (356,388 | ) | $ | (348,995 | ) | |||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (1.07 | ) | $ | (1.48 | ) | $ | (1.88 | ) | |||
| Weighted-average common shares outstanding — basic and diluted | 258,867,380 | 240,421,001 | 185,790,021 | |||||||||
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
| December 31, | ||||||||
| 2020 | 2019 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 163,240 | $ | 142,837 | ||||
| Investments in marketable securities | 320,029 | 309,903 | ||||||
| Accounts receivable | 46,923 | 33,284 | ||||||
| Inventories | 19,556 | 14,041 | ||||||
| Prepaid expenses and other current assets | 29,721 | 20,008 | ||||||
| Total current assets | 579,469 | 520,073 | ||||||
| Operating lease right-of-use assets, less accumulated amortization of $7,574 and $5,342 at December 31, 2020 and December 31, 2019, respectively | 23,296 | 33,315 | ||||||
| Property and equipment, less accumulated depreciation of $14,487 and $17,604 at December 31, 2020 and December 31, 2019, respectively | 43,863 | 47,705 | ||||||
| In-process research & development | 23,000 | 23,000 | ||||||
| Goodwill | 197,797 | 197,797 | ||||||
| Other non-current assets | 19,095 | 28,317 | ||||||
| Total Assets | $ | 886,520 | $ | 850,207 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 17,063 | $ | 21,722 | ||||
| Accrued expenses and other current liabilities | 96,841 | 99,901 | ||||||
| Contingent consideration payable | 8,900 | — | ||||||
| Operating lease liabilities | 6,872 | 7,189 | ||||||
| Total current liabilities | 129,676 | 128,812 | ||||||
| Deferred reimbursements | 7,406 | 8,906 | ||||||
| Long-term debt | 389,254 | 149,505 | ||||||
| Contingent consideration payable | 16,925 | 22,681 | ||||||
| Deferred income taxes | 4,896 | 5,051 | ||||||
| Operating lease liabilities | 45,604 | 53,531 | ||||||
| Other non-current liabilities | 6,379 | 5,296 | ||||||
| Total liabilities | 600,140 | 373,782 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Common stock, $0.01 par value, 500,000,000 shares authorized, 262,063,461 and 255,417,869 shares issued and outstanding at December 31, 2020 and December 31, 2019, respectively | 2,650 | 2,598 | ||||||
| Additional paid-in capital | 2,308,578 | 2,227,225 | ||||||
| Accumulated other comprehensive loss: | ||||||||
| Foreign currency translation adjustment | 8,412 | 2,785 | ||||||
| Unrealized (loss) gain on available-for securities | (185 | ) | 40 | |||||
| Warrants | 12,387 | 12,387 | ||||||
| Accumulated deficit | (2,045,462 | ) | (1,768,610 | ) | ||||
| Total stockholders' equity | 286,380 | 476,425 | ||||||
| Total Liabilities and Stockholders' Equity | $ | 886,520 | $ | 850,207 | ||||
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
| December 31 | ||||||||||||
| 2020 | 2019 | 2018 | ||||||||||
| Total operating expenses - as reported GAAP | $ | 476,840 | $ | 464,311 | $ | 405,618 | ||||||
| Research and development: | ||||||||||||
| Share-based compensation | 20,817 | 17,575 | 11,740 | |||||||||
| Asset acquisition related expenses for in-process R&D | - | - | 100,000 | |||||||||
| Selling, general and administrative: | ||||||||||||
| Share-based compensation | 28,334 | 26,855 | 17,520 | |||||||||
| Changes in fair value of contingent consideration payable | 3,144 | 3,297 | 3,300 | |||||||||
| Depreciation and amortization | 8,846 | 4,775 | 4,216 | |||||||||
| Total operating expense adjustments to reported GAAP | 61,141 | 52,502 | 136,776 | |||||||||
| Total operating expenses - as adjusted | $ | 415,699 | $ | 411,809 | $ | 268,842 | ||||||
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Exhibit 99.2
 |
Exhibit 99.2 Full Year 2020 Financial Results Conference Call & Webcast March 1, 2021 |
 |
Introduction 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 and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, and revenue goals, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations and/or revenue from the COVID-19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID-19, such as travel bans, shelter in place orders and third-party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product or to treatment sites. In addition to the impact of the COVID-19 pandemic, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, UK, 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, commercialization and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding corporate financial guidance and financial goals and the attainment of such goals and statements regarding projections of the Company's revenue and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2020 to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof. Non-GAAP Financial Measures In addition to financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results. |
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Introduction 4 PLATFORM & Glycobiology AT-GAA Phase 3 in Pompe Disease Two Clinical-Stage Gene Therapies $483.3M Cash as of 12/31/20 GLOBAL COMMERCIAL ORGANIZATION Robust R&D Engine Nearly 50+ Lysosomal Disorders and More Prevalent Rare Diseases EMPLYEES in 27untries Gene Therapy Protein Engineering World Class BIOLOGICS Capabilities A leading fully integrated, global rare disease biotechnology company |
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Introduction 5 2021 Key Strategic Priorities 1 2 3 4 5 Achieve double-digit Galafold growth and revenue $300M to $315M Report data from the AT-GAA Phase 3 PROPEL study and complete BLA and MAA filings for regulatory approvals Advance clinical studies, regulatory discussions and scientific data across industry leading gene therapy pipeline Further manufacturing capabilities and capacity to build world-class technical operations to support all gene therapy programs Maintain strong financial position |
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Galafold® (migalastat) Global Launch… …taking a leadership role in the treatment of Fabry disease “We push ideas as far and as fast as possible” - Amicus Belief Statement |
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Galafold: Precision Medicine for Fabry Disease 7 Galafold Snapshot (as of December 31, 2020) One of the Most Successful $260.9M FY20 Galafold Revenue Rare Disease Launches $300M-315M FY21 Global Galafold Revenue 1,384 Amenable Mutations Included in the Label 348 nable Variants in U.S. Label Continue Expan in 2021 40+ Countries with Regulatory Approvals: including U.S., EU, Japan, and other Countries Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf. For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu. Galafold is an orally delivered small molecule precision medicine with a unique mechanism of action for Fabry patients with amenable variants that replaces the need for intravenously delivered ERT |
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Galafold: Precision Medicine for Fabry Disease 8 2020 Galafold Success Year-over-Year Sales Growth Quarterly Galafold Sales 2019 Operational Growth FX Impact 2020 3Q19 4Q19 1Q20 2Q20 3Q20 4Q20 2019 vs 2020 $70.9M $67M $62M $61M $55M $49M +1% $260.9M $182M +42% Growth remains strong with Q4 revenue of $70.9M and FY2020 revenue of $260.9M |
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Galafold: Precision Medicine for Fabry Disease 9 Galafold Global Commercial Momentum FY20 Strength Reflects Continued Strength with 1,400+ Treated Patients Strong patient demand and several new launch countries anticipated in 2021 Global mix of switch (60%) and previously untreated patients (40%) Continued growing prescriber base in the U.S. to nearly 200 physicians Continue to support diagnostic initiatives to drive a shorter pathway to diagnosis ▪ Co pliance and adherence 0% 10 success in renegotiatin ▪ Int national reimbursement agreements Time from PRF to shipment don to 21 days US ▪ US insurance re-authorizatio ne s ▪ Strong global demand supported by high compliance and adherence rates laying foundation for continued growth anticipated in 2021 |
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Galafold: Precision Medicine for Fabry Disease 10 Galafold Geographic Growth Expect to add at least 5 additional countries in 2021 Expansion into multiple new markets throughout 2020 including: Poland, Iceland, Luxembourg, Argentina, Greece Regulatory approval in 40+ countries and commercial sales in 30+ of those today |
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Galafold: Precision Medicine for Fabry Disease 11 Outlook for 2021 • 2020 execution lays a solid foundation and global demand remains strong with continued growth anticipated in 2021 and beyond New Galafold patient additions slowed in Q4 due to COVID reemergence and resulting in increased lag time between patient identification and treatment initiation • • In 2021, project double-digit revenue growth with starts expected to be greater than in 2020 Expect higher patient adds and revenue growth in 2021 as COVID impact eases Continue to see >90% compliance and adherence net new patient • the second half of • rates globally Galafold Continues Strong Launch Performance & Cornerstone of Amicus Success Continued double-digit Galafold revenue growth to $300M-$315M in 2021 |
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Galafold: Precision Medicine for Fabry Disease 12 Galafold Opportunity $1B+ Opportunity With inherent Fabry market growth and our work to improve screening and diagnosis, Galafold has the potential to drive $1B+ $500M+ Projected Revenue Peak annual revenue at peak $261M 2023 Durable growth and increasing investment in Fabry disease diagnosis drives longer-term market potential $91M 2020 Driven by: Market penetration in existing and new markets Continued uptake into diagnosed, untreated market 2018 Strong IP protection through orphan drug acts in U.S. and EU, as well as multiple patents Continued momentum across geographies 1st year of launch in major geographies |
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AT-GAA: Next Potential Standard of Care for Pompe Disease “We encourage and embrace constant innovation” - Amicus Belief Statement |
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AT-GAA for Pompe Disease 1144 Unmet Need in Pompe Disease Today There is an initial positive effect on the most important outcome measures, however, a more limited long-term benefit of stabilization of the clinical course under ERT for many patients over a long period. Though, accordingto our data, this long-term therapeutic efficacy is and consecutively the support over time. As weakest for the lung capacity need of additional ventilatory respiratory insufficiency is the most frequent cause of death in Pompe disease, it is important to further improve this organ function in particular. The fast approval of novel therapeutic options is a great unmet need for Pompe patients.” Modified from Goodschmidt et. al., 2021, STIG study: real-world data of long-term outcomes of adults with Pompe disease under enzyme replacement therapy with alglucosidase alfa Natural history studies and publications in Pompe disease continue to highlight the unmet need and the continued decline on key measures of disease |
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AT-GAA for Pompe Disease 15 PROPEL Topline Results: Overall Population (n=122) 6MWD (m) FVC (% predicted) NOTES: Baseline is Mean (STDEV); CFBL is Mean LOCF (SE); P-values are nominal 2-sided; FVC data normally distributed and p–values are from ANCOVA. Results exclude one clinically implausible patient who used an investigational anabolic steroid ostarine (selective androgen receptor modulator) just prior to study start. 6MWD data not normally distributed and 6MWD p-value is for non-parametric ANCOVA; 6MWD parametric MMRM p-value was p=0.097. Treatment Baseline CFBL at Week 52 Difference P-Value AT-GAA (n=85) 70.7 (19.6) -0.9(0.7) +3.0 (1.2) p=0.023 Alglucosidase alfa (n=37) 69.7 (21.5) -4.0 (0.8) Treatment Baseline CFBL at Week 52 Difference P-Value AT-GAA (n=85) 357.9 (111.8) +20.8 (4.6) +13.6 (8.3) p=0.072 Alglucosidase alfa (n=37) 351.0 (121.3) +7.2 (6.6) 6MWD showed greater improvement with AT-GAA versus alglucosidase alfa but did not demonstrate statistical superiority; FVC demonstrated clinically significant improvement with AT-GAA over alglucosidase alfa |
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AT-GAA for Pompe Disease 16 PROPEL Topline Results ERT Experienced Population (n=95) 6MWD (m): Change from baseline (n=65, n=30) FVC (% predicted): Change from baseline (n=65, n=30) 2.0 1.5 30 1.0 25 0.5 0.0 20 -0.5 -1.0 -1.5 15 10 -2.0 -2.5 5 -3.0 -3.5 -4.0 0 -2 -4 -6 -8 -4.5 -5.0 Bas eline Week 12 Week 26 Visit Week 38 Week 52 LOCF Bas eline Week 12 Week 26 Visit Week 38 Week 52 LOCF 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 Mean(SE) Actual Change in 6MWD (m) Mean(SE) Actual Change in Sitting % Predicted FVC Treatment:ATB200/AT2221Alglucos idas e Treatment: ATB200/AT2221 Alglucos idas e p=0.046 16.9 0.0 p=0.006 0.1 -4.0 ERT experienced patients treated with AT-GAA demonstrated improvements over time in 6MWD and stabilization over time in FVC versus alglucosidase alfa |
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AT-GAA for Pompe Disease 17 Primary, Key Secondary and Biomarker Endpoint Heat Map All Patients & ERT Experienced Patients Overall Population ERT Experienced Population Note: * Nominal P-value <0.05; based on LOCF means Alglucosidase alfa AT-GAA Motor Function 6MWD* GSGC* Pulmonary Function FVC* Muscle Strength Lower MMT PROs PROMIS-Physical PROMIS-Fatigue Biomarker Hex4* CK* Alglucosidase alfa AT-GAA Motor Function 6MWD GSGC* Pulmonary Function FVC* Muscle Strength Lower MMT PROs PROMIS-Physical PROMIS-Fatigue Biomarker Hex4* CK* Endpoints across motor function, pulmonary function, muscle strength, PROs and biomarkers favored AT-GAA over alglucosidase alfa in both the overall and ERT experienced populations |
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AT-GAA for Pompe Disease 18 Post Hoc Non-Inferiority Analyses p-value NOTE1: 6MWD results are based on MMRM analysis using observed cases and FV results are based on ANCOVA using LOCF. NOTE2: As Lumizyme did not show statistical significance versus placebo for 6MWD, the NI margins were not statistically justified. Results exclude one clinically implausible patient who used an investigational anabolic steroid ostarine (selective androgen receptor modulator) just prior to study start. Parameter Non-Inferiority Margin 1-sided 6MWD 15 meters 0.0004 10 meters 0.0026 5 meters 0.0127 3 meters 0.0224 % predicted FVC 1.1% 0.0008 Post hoc non-inferiority analyses of 6MWD and FVC are highly statistically significant |
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AT-GAA for Pompe Disease 19 AT-GAA: Key Takeaways • • Rolling BLA submission expected to complete in Q2 Other key regulatory submissions for approval throughout 2021 including MAA in Europe Potential for early approval under EAMS framework with Priority Innovative Medicines Designation in UK 150+ patients worldwide now being treated with AT-GAA including adults, adolescents and infants Pediatric study for Pompe patients aged 12 to <18 with late-onset Pompe disease ongoing Clinical study for Pompe patients with infantile onset disease expected to begin this year Expanded access program for Pompe infantile patients and adult-onset patients open and has enrolled multiple patients with Pompe.Further expanded access for all Pompe patients being considered. • • • • • AT-GAA for Pompe Advances Toward Approval |
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Next Generation Gene Therapy Platform “We have a duty to obsolete our own technologies” - Amicus Belief Statement |
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Next Generation Gene Therapy Platform 21 *Exclusive license from Ultragenyx for Japanese rights to Mepsevii™, investigator-sponsored trial in Japan underway DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL Fabry Franchise Galafold®(migalastat) Monotherapy Fabry Gene Therapy PENN Pompe Franchise AT-GAA (Novel ERT + Enzyme Stabilizer) Pompe Gene Therapy PENN Batten Franchise – Gene Therapies CLN6 Batten Disease NCH CLN3 Batten Disease NCH CLN1 Batten Disease PENN Next Generation Research Programs and CNS Gene Therapies LEGEND ODD - Orphan Drug Designation RPD - Rare Pediatric Disease Designation PRIME - Priority Medicines Designation BTD - Breakthrough Therapy Designation CDKL5 Deficiency Disorder GTx / ERT PENN Angelman Syndrome PENN Others NCH / PENN MPS Franchise Mepsevii™ (vestronidase alfa) (Japan Only)* Next Generation MPSIIIA PENN MPSIIIB PENN ODD RPD ODD RPD PRIME ODD BTD ODD |
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Next Generation Gene Therapy Platform 22 CLN3 Batten AT-GTX-502 Disease Gene Therapy UBDRS=Unified Batten Disease Rating Scale. a Scheduled month 12 in-person UBDRS assessment was impacted by the COVID-19 pandemic. •Batten disease is a group of disorders known as neuronal ceroid lipofuscinoses (NCLs) •Mutation in one of 13 different CLN genes leads to neuronal lysosomal dysfunction •CLN3 Batten disease is one of the most common neurodegenerative disorders affecting children leading to blindness, motor impairment, learning difficulties, epilepsy and, ultimately, premature death •UBDRS-physical is the key efficacy endpoint for this study in CLN3 Batten assessing gross and fine motor function, vision and speech Low-Dose Cohort (n=3) Compared with Patients in the Natural History Study (n=82) Early clinical data suggest early signs of disease stabilization compared to natural history; plan to submit IND for next clinical study in 2H2021 |
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Financial Summary “We are business led and science driven” - Amicus Belief Statement |
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Financial Summary 24 2020 Select Financial Results (in thousands, except per share data) Dec. 31, 2020 Dec. 31, 2019 Product Revenue $260,886 $182,237 Cost of Goods Sold 31,044 21,963 R&D Expense 308,443 286,378 SG&A Expense 156,407 169,861 Changes in Fair Value of Contingent Consideration 3,144 3,297 Depreciation and Amortization 8,846 4,775 Loss from Operations (246,998) (304,037) Loss on Extinguishment of Debt (7,276) - Income Tax (Expense) Benefit (2,598) (478) Net Loss (276,852) (356,388) Net Loss Per Share (1.07) (1.48) 2020 revenue of $260.9M from global Galafold sales |
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Financial Summary 25 Financial Outlook: Key Takeaways • Galafold revenue in 2020 was $260.9 million, exceeding the Company’s guidance • 2020 Non-GAAP Operating Expenses of $415.7M in line with guidance of $410 million to $420 million • Non-GAAP operating expense guidance for 2021 is expected to remain flat at $410 million to $420 million – Driven by disciplined expense management and continued investment in the global Galafold launch, AT-GAA clinical studies and advancing our gene therapy pipeline • Current cash position is sufficient to achieve self-sustainability without the need for future dilutive financing |
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Closing Remarks “We are business led and science driven” - Amicus Belief Statement |
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ThankYou “Our passion for making a difference unites us” -Amicus Belief Statement |
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Appendix |
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Amicus Therapeutics, Inc. Reconciliation ofNon-GAAP Financial i\•leasures (in thousands) December31 2020 s476,840 2019 2018 s Total operating expenses - as reported GAAP Research and d evelopment: Share-based compensation Asset acquisition related expenses for in- processR&D Selling, general and administrative: Share-based compensation Changes in fair value of contingent consideration payable Depreciation and amortization Total operating expense adjustments to reported GAAP Total ope.rating expenses -as adjusted $ 405,618 464,.311 20,817 17,575 11,740 100,000 28,334 3,144 17,520 3,300 26,855 3,297 8,846 4,775 4,216 61,141 52,502 136,776 $ $ $ 268,842 415,699 411,809 /vA-micus Therapeutics- |