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Item 2.02 Results of Operations and Financial Condition
On November 9, 2021, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended September 30, 2021. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on November 9, 2021 to discuss its second quarter 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 November 9, 2021 |
| 99.2 | November 9, 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: November 9, 2021 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
Amicus Therapeutics Announces Third Quarter 2021 Financial Results and Corporate Updates
3Q21 Total Galafold® Revenue of $79.5M – an 18% increase over 3Q20
Reiterating 2021 Revenue Guidance of $300M-$315M
AT-GAA BLA and NDA for Pompe Disease Accepted for Review by the U.S. FDA;
Marketing Authorization Applications Submitted to European Medicines Agency
On Track to Complete Planned Business Combination of Amicus Gene Therapy Business
with ARYA IV Resulting in the Launch of Caritas Therapeutics in Late 2021/Early 2022
Conference Call and Webcast Today at 8:30 a.m. ET
PHILADELPHIA, PA, Nov. 9, 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 quarter ended September 30, 2021.
“In the second half of 2021, we have furthered our mission for people living with devastating rare diseases through the commercial execution of Galafold and advancement of the global regulatory filings and launch preparations for AT-GAA,” stated, John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. “As previously announced, our teams are proceeding with the business combination of our leading gene therapy portfolio with ARYA IV to launch Caritas Therapeutics, a next-generation genetic medicines company. This transaction will serve patients and shareholders well by accelerating funding for our gene therapy pipeline, while simultaneously strengthening the financial profile of Amicus. We are immensely excited for what the future of science and biotechnology holds as we accelerate our commitment to extraordinary patient dedication.”
Corporate Highlights
| · | Global revenue for Galafold® (migalastat) in the third quarter of 2021 reached $79.5 million, representing a year-over-year increase of 18% from total revenue of $67.4 million in the third quarter of 2020. |
| · | Long-term Galafold data published in the September 2021 Issue of Molecular Genetics and Metabolism Reports showing generally stable renal function in patients with an amenable variant during long-term treatment, up to 8.6 years, of Galafold, irrespective of treatment status, gender or phenotype. |
| · | U.S. Food and Drug Administration (FDA) accepted for review the Biologics License Application (BLA) for cipaglucosidase alfa and the New Drug Application (NDA) for miglustat, the two components of AT-GAA. The FDA has set a Prescription Drug User Fee Act (PDUFA) action date of May 29, 2022 for the NDA and July 29, 2022 for the BLA. |
| · | Marketing Authorization Applications (MAA) submitted to the European Medicines Agency (EMA) for AT-GAA in Pompe disease. The filings are based on the evaluation of the effects of AT-GAA in people living with Pompe disease and its safety profile, which include data from the Phase 1/2 and Phase 3 PROPEL studies as well as data from the long-term open-label extension study. |
| · | Amicus and ARYA Sciences Acquisition Corp IV (“ARYA IV”), a special purpose acquisition company, announced their intent to launch a next-generation genetic medicine company, Caritas Therapeutics, Inc. Through a definitive business combination agreement, the Amicus gene therapy business will be acquired by ARYA IV. The transaction will result in two independent publicly traded companies with attractive stand-alone investment profiles. Amicus will become the largest shareholder in Caritas with a ~36% ownership stake (assuming no redemptions by ARYA’s shareholders) and retain co-development and commercialization rights to the Fabry and Pompe gene therapy programs, as well as negotiation rights on select future muscular dystrophy programs. The Form S-4 is now filed with the Securities and Exchange Commission and the launch of Caritas Therapeutics is expected in late 2021 or early 2022. |
| · | Cash position sufficient to achieve self-sustainability and profitability in 2023. Following the transaction with ARYA IV, the previously announced ~$200 million private investment from leading biotechnology investors, and through careful management of expenses, the Company is on the path to achieve self-sustainability and profitability by 2023 as it executes on the global Galafold launch and AT-GAA global regulatory filings. |
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Third Quarter 2021 Financial Results
| · | Total revenue in the third quarter of 2021 was $79.5 million, a year-over-year increase of 18% from total revenue of $67.4 million in the third quarter of 2020. On a constant currency basis, third quarter 2021 total revenue was $78.5 million, representing operational revenue growth measured at constant currency exchange rates of 16.5%. Reported revenue was aided by a positive currency impact of $1.0 million, or 1.5%. |
| · | Cash, cash equivalents, and marketable securities totaled $557.0 million at September 30, 2021, compared to $483.3 million at December 31, 2020. |
| · | Total GAAP operating expenses of $110.2 million for the third quarter of 2021 decreased as compared to $111.8 million for the third quarter 2020, reflecting the timing of investments in our pipeline. |
| · | Total non-GAAP operating expenses of $93.6 million for the third quarter of 2021 increase as compared to $92.4 million in the third quarter of 2020.1 |
| · | Net loss was reduced to $50.3 million, or $0.19 per share, compared to a net loss of $64.0 million, or $0.25 per share, for the third quarter 2020. |
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 $300 million to $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, UK 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 |
| · | Based on current operating models, the Company believes that the current cash position 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.
2021 Milestones by Program
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
| · | Continue revenue growth in 2021 |
| · | EU label expanded to cover adolescent population |
| · | Continue geographic expansion |
| · | Registry and other Phase 4 studies |
AT-GAA for Pompe Disease
| · | BLA and NDA submissions accepted for review in 3Q21; EU MAA submissions completed in 4Q21 |
| · | Ongoing supportive studies, including pediatric and extension studies |
Gene Therapy Portfolio
| · | Reported initial data from the CLN3 Batten disease gene therapy Phase 1/2 study; advance manufacturing activities and regulatory discussions to enable dosing additional patients with Good Manufacturing Practice (GMP) clinical-grade material |
| · | Advance manufacturing activities and regulatory discussions for the CLN6 Batten disease gene therapy program |
| · | Continue to progress IND-enabling studies, manufacturing activities and regulatory discussions in both Fabry and Pompe gene therapies |
| · | Disclose additional preclinical data and potential IND candidate declarations across multiple preclinical programs |
| · | Manufacturing advancements and updates across the portfolio |
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Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, November 9, 2021, at 8:30 a.m. ET to discuss the third quarter 2021 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: 3686792.
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 November 9, 2021. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 3686792.
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.
U.S. INDICATIONS AND USAGE
Galafold is indicated for the treatment of adults with a confirmed diagnosis of Fabry disease and an amenable galactosidase alpha gene (GLA) variant based on in vitro assay data.
This indication is approved under accelerated approval based on reduction in kidney interstitial capillary cell globotriaosylceramide (KIC GL-3) substrate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trials.
U.S. IMPORTANT SAFETY INFORMATION
ADVERSE REACTIONS
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been established in pediatric patients.
To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
| · | Galafold is not intended for concomitant use with enzyme replacement therapy. |
| · | Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m2). The safety and efficacy of Galafold in children less than 12 years of age have not yet been established. No data are available. |
| · | No dosage adjustments are required in patients with hepatic impairment or in the elderly population. |
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| · | There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse. |
| · | While taking Galafold, effective birth control should be used. It is not known whether Galafold is excreted in human milk. |
| · | Contraindications to Galafold include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION. |
| · | Galafold 123 mg capsules are not for children (≥12 years) weighing less than 45 kg. |
| · | It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold. |
| · | OVERDOSE: General medical care is recommended in the case of Galafold overdose. |
| · | The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received Galafold. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS. |
| · | Call your doctor for medical advice about side effects. |
For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.
About 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.
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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 and Quarterly Report 10-Q for the quarter ended September 30, 2021, 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.
CONTACTS:
Investors:
Andrew Faughnan
Executive 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)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Net product sales | $ | 79,545 | $ | 67,437 | $ | 223,360 | $ | 190,315 | ||||||||
| Cost of goods sold | 11,696 | 8,399 | 26,615 | 21,627 | ||||||||||||
| Gross profit | 67,849 | 59,038 | 196,745 | 168,688 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 59,333 | 70,419 | 186,453 | 229,150 | ||||||||||||
| Selling, general, and administrative | 46,107 | 37,850 | 135,109 | 112,722 | ||||||||||||
| Changes in fair value of contingent consideration payable | 3,288 | 1,034 | 4,780 | 2,680 | ||||||||||||
| Depreciation and amortization | 1,520 | 2,496 | 4,691 | 6,299 | ||||||||||||
| Total operating expenses | 110,248 | 111,799 | 331,033 | 350,851 | ||||||||||||
| Loss from operations | (42,399 | ) | (52,761 | ) | (134,288 | ) | (182,163 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income | 108 | 518 | 323 | 2,898 | ||||||||||||
| Interest expense | (8,165 | ) | (6,784 | ) | (24,307 | ) | (14,148 | ) | ||||||||
| Loss on extinguishment of debt | (257 | ) | (7,276 | ) | (257 | ) | (7,276 | ) | ||||||||
| Other income (expense) | 237 | 3,019 | (2,729 | ) | 29 | |||||||||||
| Loss before income tax | (50,476 | ) | (63,284 | ) | (161,258 | ) | (200,660 | ) | ||||||||
| Income tax benefit (expense) | 182 | (727 | ) | (5,925 | ) | (4,791 | ) | |||||||||
| Net loss attributable to common stockholders | $ | (50,294 | ) | $ | (64,011 | ) | $ | (167,183 | ) | $ | (205,451 | ) | ||||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.19 | ) | $ | (0.25 | ) | $ | (0.63 | ) | $ | (0.80 | ) | ||||
| Weighted-average common shares outstanding — basic and diluted | 267,464,637 | 259,161,799 | 266,085,788 | 258,091,170 | ||||||||||||
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
September 30, 2021 | December 31, 2020 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 385,903 | $ | 163,240 | ||||
| Investments in marketable securities | 171,057 | 320,029 | ||||||
| Accounts receivable | 51,427 | 46,923 | ||||||
| Inventories | 22,072 | 19,556 | ||||||
| Prepaid expenses and other current assets | 20,081 | 29,721 | ||||||
| Total current assets | 650,540 | 579,469 | ||||||
| Operating lease right-of-use assets, net | 21,270 | 23,296 | ||||||
| Property and equipment, less accumulated depreciation of $18,789 and $14,487 at September 30, 2021 and December 31, 2020, respectively | 41,991 | 43,863 | ||||||
| In-process research & development | 23,000 | 23,000 | ||||||
| Goodwill | 197,797 | 197,797 | ||||||
| Other non-current assets | 22,077 | 19,095 | ||||||
| Total Assets | $ | 956,675 | $ | 886,520 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 24,474 | $ | 17,063 | ||||
| Accrued expenses and other current liabilities | 72,453 | 96,841 | ||||||
| Contingent consideration payable | 17,000 | 8,900 | ||||||
| Operating lease liabilities | 7,175 | 6,872 | ||||||
| Total current liabilities | 121,102 | 129,676 | ||||||
| Deferred reimbursements | 7,406 | 7,406 | ||||||
| Long-term debt | 388,719 | 389,254 | ||||||
| Contingent consideration payable | 7,605 | 16,925 | ||||||
| Deferred income taxes | 4,896 | 4,896 | ||||||
| Operating lease liabilities | 43,495 | 45,604 | ||||||
| Other non-current liabilities | 6,823 | 6,379 | ||||||
| Total liabilities | 580,046 | 600,140 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Common stock, $0.01 par value, 500,000,000 shares authorized, 278,585,092 and 262,063,461 shares issued and outstanding at September 30, 2021 and December 31, 2020, respectively | 2,805 | 2,650 | ||||||
| Additional paid-in capital | 2,579,953 | 2,308,578 | ||||||
| Accumulated other comprehensive income (loss): | ||||||||
| Foreign currency translation adjustment | 6,617 | 8,412 | ||||||
| Unrealized loss on available-for-sale securities | (184 | ) | (185 | ) | ||||
| Warrants | 83 | 12,387 | ||||||
| Accumulated deficit | (2,212,645 | ) | (2,045,462 | ) | ||||
| Total stockholders’ equity | 376,629 | 286,380 | ||||||
| Total Liabilities and Stockholders’ Equity | $ | 956,675 | $ | 886,520 | ||||
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2021 | 2020 | 2021 | 2020 | |||||||||||||
| Total operating expenses - as reported GAAP | $ | 110,248 | $ | 111,799 | $ | 331,033 | $ | 350,851 | ||||||||
| Research and development: | ||||||||||||||||
| Share-based compensation | 3,775 | 8,626 | 13,232 | 17,241 | ||||||||||||
| Selling, general and administrative: | ||||||||||||||||
| Share-based compensation | 8,066 | 7,282 | 30,699 | 19,671 | ||||||||||||
| Changes in fair value of contingent consideration payable | 3,288 | 1,034 | 4,780 | 2,680 | ||||||||||||
| Depreciation and amortization | 1,520 | 2,496 | 4,691 | 6,299 | ||||||||||||
| Total operating expense adjustments to reported GAAP | 16,649 | 19,438 | 53,402 | 45,891 | ||||||||||||
| Total operating expenses - as adjusted | $ | 93,599 | $ | 92,361 | $ | 277,631 | $ | 304,960 | ||||||||
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Exhibit 99.2
3Q21 Financial Results Conference Call & Webcast November 9, 2021
2 Forward - Looking Statements Introduction This presentation contains "forward - looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company . The inclusion of forward - looking statements should not be regarded as a representation by us that any of our plans will be achieved . Any or all of the forward - looking statements in this presentation may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties . For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, and revenue goals, including as they are impacted by COVID - 19 related disruption, are based on current information . The potential impact on operations and/or revenue from the COVID - 19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID - 19 , such as travel bans, shelter in place orders and third - party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product or to treatment sites . In addition to the impact of the COVID - 19 pandemic, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation : the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective ; the potential that it may be difficult to enroll patients in our clinical trials ; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates ; the potential that we may not be successful in commercializing Galafold in Europe, 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 , and on Form 10 - Q for the quarter ended September 30 , 2021 , 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 .
3 Amicus has built a leading, fully integrated, global rare disease biotechnology company Gene Therapy PLATFORM Protein Engineering & Glycobiology $557M Cash as of 9/30/21 Two Clinical - Stage Gene Therapies AT - GAA Registration in Pompe Disease GLOBAL COMMERCIAL ORGANIZATION World Class BIOLOGICS Capabilities Robust R&D Engine 50+ Lysosomal Disorders and More Prevalent Rare Diseases EMPLOYEES in 27 Countries Introduction
4 2021 Key Strategic Priorities Introduction 1 2 3 4 5 Achieve double - digit Galafold growth and revenue of $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
5 On - Track to Complete Planned Business Combination of Amicus Gene Therapy Business with ARYA IV Resulting in the Launch of Caritas Therapeutics in Late 2021/Early 2022 Caritas Strategic Rationale Introduction Enhancing the ability of both Amicus and Caritas to meet the unmet needs of patients living with rare diseases Accelerating Amicus’ path to profitability, expected in 2023 Significantly strengthening the financial profile of each company Accelerating the development and broadening the scope of our gene therapy portfolio Reinforcing management focus on key strategic and financial goals Unlocking value while creating a more targeted investment thesis for shareholders
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
7 Galafold Snapshot (as of September 30, 2021) Galafold: Precision Medicine for Fabry Disease One of the Most Successful Rare Disease Launches 348 Amenable Variants in U.S. Label 40+ Countries with Regulatory Approvals: Including U.S., EU,U.K., Japan and Other Countries Continued Expansion in 2021 $ 79.5M + 18 % YoY 3Q21 Galafold Revenue Galafold is indicated for adults with a confirmed diagnosis of Fabry Disease and an amenable mutation/variant. The most commo n a dverse 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, dru g i nteractions 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 1,384 Amenable Mutations Included in the EU Label $ 300 M - $ 315M FY21 Global Galafold Rev enue
8 Galafold Success and FY21 Revenue Guidance Galafold momentum remains on track to deliver within full year 2021 revenue guidance Galafold: Precision Medicine for Fabry Disease FY16 FY17 FY18 FY19 FY20 FY21 $36.9M $91.2M $260.9M FY21 Rev. Guidance $300M - $315M $182.2M $5.0M Q1 $66.4M Q2 $77.4M Q3 $ 79.5 M YTD $223.3M
9 Galafold Long - Term Treatment Publication Key Findings 1,2 • Migalastat - treated patients had generally stable renal function for up to 8.6 years • Migalastat stabilized eGFR in ERT - naive and ERT - experienced males and females compared to historical untreated controls • Migalastat generally stabilized eGFR in ERT - naïve male patients with the classic phenotype Published in the September 2021 Issue of Molecular Genetics and Metabolism Reports showing generally stable renal function during long - term treatment Galafold: Precision Medicine for Fabry Disease 1. Study limitations: Post hoc design, small sample sizes in some subgroups, lack of statistical comparisons with untreated o r E RT - treated historical cohorts, assessment of renal function using eGFR, and the heterogeneity of statistical methods used to est imate eGFR slopes in the literature limited direct comparisons. 2. The study includes data that are not in the FDA - approved Prescribing Information (PI) for Galafold and the clinical relevance of the changes in eGFR in the treatment of Fabry disease has not been established.
10 Outlook for 2021 • Global demand remains strong with 3Q21 revenue growth rate of 18% and continued growth anticipated in 2021 and beyond • In 2021, project double - digit revenue growth with net new patient starts expected to be greater than in 2020 • Expanded EU label following the European Commission approval for use in adolescents • COVID continues to impact time between patient identification and treatment initiation • Expect higher patient adds and revenue growth in 2H2021 • Continue to see >90% compliance and adherence rates globally Galafold: Precision Medicine for Fabry Disease Continued double - digit Galafold revenue growth to within $300M - $315M in 2021 Galafold Continues Strong Launch Performance & Cornerstone of Amicus Success
11 $91M ~$500M Projected Revenue Galafold Opportunity Galafold: Precision Medicine for Fabry Disease With inherent Fabry market growth and our work to improve screening and diagnosis, Galafold has the potential to drive $1B+ annual revenue at peak $1B+ Opportunity Durable growth and increasing investment in Fabry disease diagnosis drives longer term market potential Strong IP protection through orphan drug acts in U.S. and EU, as well as multiple patents 1 st year of launch in major geographies Driven by: Market penetration in existing and new markets Continued uptake into diagnosed, untreated market Anticipated acceleration of growth as COVID impact eases 2018 Peak 2023 2020 $261M Continued momentum across geographies
AT - GAA: Next Potential Standard of Care for Pompe Disease “We encourage and embrace constant innovation” - Amicus Belief Statement
13 Pompe Disease Overview Pompe disease is a severe and fatal neuromuscular disease and one of the most prevalent lysosomal disorders with very high unmet medical need AT - GAA for Pompe Disease Deficiency of GAA leading to 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 5,000 – 10,000+ patients diagnosed WW 1 ; newborn screening suggests underdiagnosis ~$1.1B+ global Pompe ERT sales 2 1. National Institute of Neurological Disorders and Stroke (NIH). 2. Based on year ending June 30, 2021. Source: Sanofi Press Re leases Patients on current standard of care decline after ~2 years
14 Phase 3 PROPEL Study Primary, Key Secondary and Biomarker Endpoint Heat Map AT - GAA for Pompe Disease Endpoints across motor function, pulmonary function, muscle strength, PROs and biomarkers favored AT - GAA over alglucosidase alfa Source: Presented at the 16th International Congress on Neuromuscular Diseases (ICNMD) May 2021. Note: * Nominal P - value <0.05; based on LOCF means
15 AT - GAA: Highlights AT - GAA for Pompe Disease AT - GAA for Pompe Advances Toward Approval • U.S. FDA accepted for review the BLA and NDA for AT - GAA • MAA for AT - GAA submitted with the EMA • Granted positive scientific opinion through the Early Access to Medicines Scheme (EAMS) by the United Kingdom’s MHRA • 150+ patients worldwide now being treated with AT - GAA, including adults, adolescents and infants • Pediatric study for adolescents up to 17 years with late - onset Pompe disease ongoing • Clinical study for Pompe patients with infantile - onset disease expected to begin this year • Expanded access program for infantile - and adult - onset patients open and has enrolled multiple patients with Pompe. Further expanded access for all Pompe patients being considered.
“We have a duty to obsolete our own technologies” - Amicus Belief Statement Next - Generation Gene Therapy Platform
17 Harnessing the Power of Genetic Medicine The mission of Caritas is to transform the lives of children and adults living with rare genetic diseases through advanced protein engineering and innovative vector technologies Diversified portfolio of clinical and preclinical rare disease development programs with pivotal study start in CLN3, three INDs, and two IND candidates targeted through end of 2023 1 Proprietary platform technologies and protein engineering capabilities enabling innovative, clinically differentiated gene therapies 2 Exclusive relationship with Penn to develop gene therapies for rare genetic disorders 3 Fully designed, ready - to - build, state - of - the - art clinical manufacturing facility with commercial expansion capabilities 4 Attractive risk and cost - sharing partnership with Amicus provides access to an established orphan disease commercial platform 5 Experienced public company leadership coupled with fully built out gene therapy discovery, research and development team 6 Next Generation Gene Therapy Platform
18 Protein Engineering Expertise & Technologies for Gene Therapy Next Generation Gene Therapy Platform Differentiated gene therapy approach for greater potency and optimized cross correction through transgene engineering for stability and targeting ▪ Proprietary AAV capsid ▪ Pantropic capsid and ubiquitous promoter ▪ Engineered hGLA transgene at dimer interface designed for improved stability and optimized cross correction ▪ Preclinical data demonstrate robust substrate reduction across all Fabry disease relevant tissues, including first evidence of dorsal root ganglia storage reduction ▪ IND expected in 2H2022 ▪ Proprietary AAV capsid ▪ Pantropic capsid and ubiquitous promoter ▪ Engineered hGAA transgene with cell receptor binding motif designed for improved uptake and optimized cross correction ▪ Preclinical data demonstrate robust glycogen reduction in all key Pompe disease relevant tissues, including reduction in neurons of central nervous system ▪ IND - enabling work underway Pompe Gene Therapy Fabry Gene Therapy W i l d - t y p e V e h i c l e A A V . h G L A n a t - L D A A V . h G L A n a t - M D A A V . h G L A n a t - H D A A V . h G L A e n g - L D A A V . h G L A e n g - M D A A V . h G L A e n g - H D 0 5 10 15 20 25 % t u b e s w i t h s t o r a g e * * * * Quadriceps: Histopath score Kidney: % Tubules with GL - 3 Note: Data from studies in KO GLA and GAA mice Amicus and Caritas to co - develop the Fabry and Pompe gene therapies
“We are business led and science driven” - Amicus Belief Statement Financial Summary
20 3Q21 Select Financial Results Financial Summary Sep. 30, 2021 (in thousands, except per share data) Sep. 30, 2020 Product Revenue $79,545 $67,437 Cost of Goods Sold 11,696 8,399 R&D Expense 59,333 70,419 SG&A Expense 46,107 37,850 Changes in Fair Value of Contingent Consideration 3,288 1,034 Depreciation and Amortization 1,520 2,496 Loss from Operations (42,399) (52,761) Income Tax Benefit (Expense) 182 (727) Net Loss ( 50,294 ) (64,011) Net Loss Per Share (0.19) (0.25) 3Q21 revenue of $ 79.5M and growth rate of 18 % primarily from global Galafold sales
21 Financial Outlook: Key Takeaways • Reaffirming full - year Galafold revenue guidance of $300 million to $315 million • Non - GAAP operating expense guidance for 2021 is expected to remain flat at $410 million to $420 million • Balance sheet further strengthened with a ~$200 million private investment from leading biotechnology investors • Current cash position is sufficient to achieve self - sustainability and profitability by 2023 Financial Summary
Closing Remarks “We believe in our future to build long - term value for our stakeholders” - Amicus Belief Statement
Thank You “Our passion for making a difference unites us” - Amicus Belief Statement
Appendix
25 Reconciliation Appendix