UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
FORM
CURRENT REPORT PURSUANT TO
SECTION 13 OR 15(d) OF THE
SECURITIES EXCHANGE ACT OF 1934
Date
of Report (Date of earliest event reported):
(Exact Name of Registrant as Specified in Its Charter)
Delaware
(State or Other Jurisdiction of
Incorporation)
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If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02 Results of Operations and Financial Condition
On November 5, 2020, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended September 30, 2020. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on November 5, 2020 to discuss its third 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 5, 2020 | |
| 99.2 | November 5, 2020 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 5, 2020 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary | |
Exhibit 99.1
Amicus Therapeutics Announces Third Quarter 2020 Financial Results and Corporate Updates
Galafold 3Q20 Revenue of $67.4 Million, On-Track to Achieve Revenue Guidance of $250M-$260M
AT-GAA Phase 3 PROPEL Study Readout in 1Q21 and Rolling BLA Submission to Begin in 4Q20
Positive CLN6 Batten Disease Gene Therapy Data Presented in October
Fabry Disease Gene Therapy Clinical Candidate Selected
Conference Call and Webcast Today at 8:30 a.m. ET
CRANBURY, NJ, Nov. 5, 2020 – 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 third quarter ended September 30, 2020. The Company also summarized recent program updates and reiterated its full-year 2020 guidance.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “During the third quarter, we made tremendous progress advancing our mission for patients and are on track to achieve our 2020 key strategic priorities, including our global Fabry commercial launch, Pompe late-stage development program, and advancing our industry-leading gene therapy pipeline. Through these efforts, we remain strongly positioned 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 third quarter of 2020 was $67.4M. Third quarter revenue represented a year-over-year increase of 38% from total revenue of $48.8 million in the third quarter of 2019. On a constant currency basis, third quarter 2020 total revenue was $65.7 million, representing operational revenue growth measured at constant currency exchange rates of 35%, which was further benefited by a positive currency impact of $1.7 million, or 3%. Galafold performance was driven largely by strong patient demand. Global compliance and adherence rates continue to exceed 90%. |
| · | Global Phase 3 PROPEL clinical study of AT-GAA in late-onset Pompe disease (LOPD) on track for top-line data in 1Q21. To date, 97%+ of the 3,100+ planned infusions and assessments for the ongoing PROPEL study have been completed on schedule. The Company plans to initiate a rolling BLA for AT-GAA in the fourth quarter of 2020, completing final submission in the first half of 2021. |
| · | To be highlighted on today’s call, new natural history data from the Amicus POM-002 chart review study in people living with LOPD treated long-term with current standard of care, alglucosidase alfa. Data are consistent with the medical literature and further supports PROPEL design assumptions. |
| · | Additional Phase 1/2 CLN6 data presented at the Child Neurology Society Annual Meeting in October. The data shows a meaningful effect in slowing disease progression out to 24 months compared to natural history. Regulatory interactions are ongoing, and the Company expects to provide feedback on the path forward in 2021. |
| · | Initial data from the Phase 1/2 CLN3 study expected in early 2021. Regulatory interactions are ongoing and the Company expects to provide feedback on the path forward in 2021. |
| · | A gene therapy clinical candidate has been selected for IND enabling studies in Fabry Disease. Initial data from the Company’s AAV gene therapy with an engineered GLA transgene demonstrated significantly better GL-3 reduction than one with wild-type GLA. Full set of preclinical data to be presented at a medical conference in early 2021. |
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| · | 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 and advancing development programs. |
Third Quarter 2020 Financial Results
| · | Total revenue in the third quarter 2020 was $67.4 million, a year-over-year increase of 38% from total revenue of $48.8 million in the third quarter of 2019. On a constant currency basis, third quarter 2020 total revenue was $65.7 million, representing operational revenue growth measured at constant currency exchange rates of 35%, Reported revenue was aided by a positive currency impact of $1.7 million, or 3%. | |
| · | Cash, cash equivalents, and marketable securities totaled $509.1 million at September 30, 2020, compared to $452.7 million at December 31, 2019. |
| · | Total GAAP operating expenses of $111.8 million for the third quarter 2020 increased as compared to $100.5 million for the third quarter 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs. |
| · | Total non-GAAP operating expenses of $92.4 million for the third quarter of 2020 increased as compared to $89.7 million in the third quarter of 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs.1 |
| · | Net loss was $64.0 million, or $0.25 per share, compared to a net loss of $61.8 million, or $0.24 per share, for the third quarter 2019. The third quarter 2020 net loss included an impact of $7.3 million relating to extinguishment of debt. |
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.
2020 Financial Guidance
| · | For the full-year 2020, the Company anticipates total Galafold revenue of $250 million to $260 million based on the average exchange rates for 2019. |
| · | Non-GAAP operating expense guidance for the full-year 2020 is $410 million to $420 million, driven by continued investment in the global Galafold launch, AT-GAA clinical studies, and advancing our gene therapy pipeline.2 |
| · | Cash, cash equivalents, and marketable securities totaled $509.1 million at September 30, 2020. Based on current operating models, the Company believes that the current cash position, along with 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 Milestones by Program
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
| · | On track to meet full-year 2020 revenue guidance range of $250 million to $260 million |
| · | Continued geographic expansion |
| · | Registry and other Phase 4 studies underway |
AT-GAA for Pompe Disease
| · | Plans to initiate a Rolling Biologics License Application (BLA) for AT-GAA in 2020, with addition of complete clinical results for PROPEL in 1H2021 to support full approval |
| · | Additional supportive studies, including an open-label study in 12- to <18-year-olds living with Pompe |
Gene Therapy Portfolio
| · | Advance regulatory discussions to finalize clinical and regulatory path for the CLN6 Batten disease gene therapy development program |
| · | Initial data from the CLN3 Batten disease Phase 1/2 study expected in early 2021 and advance regulatory discussions to finalize clinical and regulatory path |
| · | Continue IND-enabling toxicology work in Pompe disease and progress towards IND |
| · | Additional preclinical data expected across multiple programs |
| · | Manufacturing advancements across portfolio |
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Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, November 5, 2020 at 8:30 a.m. ET to discuss the third quarter 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: 7788189.
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 5, 2020. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 7788189.
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 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 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. |
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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. | |
| · | 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.
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, including as they are impacted by COVID-19 related disruption, are based on current information. The potential impact on operations from the COVID-19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID-19, such as travel bans, shelter in place orders and third-party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product. In addition to the impact of the COVID-19 pandemic, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products; and the potential that we will need additional funding to complete all of our studies and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. Statements regarding corporate financial guidance and financial goals and the attainment of such goals. With respect to statements regarding projections of the Company's revenue and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2019, the Quarterly Report filed on Form 10-Q for the quarter ended June 30, 2020, and the Quarterly Report filed on Form 10-Q 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:
Amicus Therapeutics
Andrew Faughnan
Director, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Communications
dmoore@amicusrx.com
(609) 662-5079
FOLD–G
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TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per share amounts)
| Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Net product sales | $ | 67,437 | $ | 48,768 | $ | 190,315 | $ | 126,944 | ||||||||
| Cost of goods sold | 8,399 | 5,596 | 21,627 | 15,018 | ||||||||||||
| Gross profit | 59,038 | 43,172 | 168,688 | 111,926 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 70,419 | 58,892 | 229,150 | 194,466 | ||||||||||||
| Selling, general, and administrative | 37,850 | 39,680 | 112,722 | 126,561 | ||||||||||||
| Changes in fair value of contingent consideration payable | 1,034 | 789 | 2,680 | 2,652 | ||||||||||||
| Depreciation and amortization | 2,496 | 1,116 | 6,299 | 3,261 | ||||||||||||
| Total operating expenses | 111,799 | 100,477 | 350,851 | 326,940 | ||||||||||||
| Loss from operations | (52,761 | ) | (57,305 | ) | (182,163 | ) | (215,014 | ) | ||||||||
| Other income (expense): | ||||||||||||||||
| Interest income | 518 | 2,752 | 2,898 | 7,990 | ||||||||||||
| Interest expense | (6,784 | ) | (4,026 | ) | (14,148 | ) | (15,105 | ) | ||||||||
| Loss on exchange of convertible notes | — | — | — | (40,624 | ) | |||||||||||
| Loss on extinguishment of debt | (7,276 | ) | — | (7,276 | ) | — | ||||||||||
| Other income (expense) | 3,019 | (3,481 | ) | 29 | (3,272 | ) | ||||||||||
| Loss before income tax | (63,284 | ) | (62,060 | ) | (200,660 | ) | (266,025 | ) | ||||||||
| Income tax (expense) benefit | (727 | ) | 251 | (4,791 | ) | (634 | ) | |||||||||
| Net loss attributable to common stockholders | $ | (64,011 | ) | $ | (61,809 | ) | $ | (205,451 | ) | $ | (266,659 | ) | ||||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.25 | ) | $ | (0.24 | ) | $ | (0.80 | ) | $ | (1.13 | ) | ||||
| Weighted-average common shares outstanding — basic and diluted | 259,161,799 | 254,674,422 | 258,091,170 | 235,527,540 | ||||||||||||
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
| September 30, 2020 | December 31, 2019 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 210,631 | $ | 142,837 | ||||
| Investments in marketable securities | 298,451 | 309,903 | ||||||
| Accounts receivable | 44,828 | 33,284 | ||||||
| Inventories | 15,767 | 14,041 | ||||||
| Prepaid expenses and other current assets | 15,600 | 20,008 | ||||||
| Total current assets | 585,277 | 520,073 | ||||||
| Operating lease right-of-use assets, less accumulated amortization of $6,850 and $5,342 at September 30, 2020 and December 31, 2019, respectively | 23,397 | 33,315 | ||||||
| Property and equipment, less accumulated depreciation of $23,582 and $17,604 at September 30, 2020 and December 31, 2019, respectively | 44,618 | 47,705 | ||||||
| In-process research & development | 23,000 | 23,000 | ||||||
| Goodwill | 197,797 | 197,797 | ||||||
| Other non-current assets | 26,453 | 28,317 | ||||||
| Total Assets | $ | 900,542 | $ | 850,207 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 14,764 | $ | 21,722 | ||||
| Accrued expenses and other current liabilities | 89,595 | 99,901 | ||||||
| Operating lease liabilities | 7,368 | 7,189 | ||||||
| Total current liabilities | 111,727 | 128,812 | ||||||
| Deferred reimbursements | 8,906 | 8,906 | ||||||
| Long-term debt | 388,584 | 149,505 | ||||||
| Contingent consideration payable | 16,561 | 22,681 | ||||||
| Deferred income taxes | 5,051 | 5,051 | ||||||
| Operating lease liabilities | 44,627 | 53,531 | ||||||
| Other non-current liabilities | 4,817 | 5,296 | ||||||
| Total liabilities | 580,273 | 373,782 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders’ equity: | ||||||||
| Common stock, $0.01 par value, 500,000,000 shares authorized, 259,600,650 and 255,417,869 shares issued and outstanding at September 30, 2020 and December 31, 2019, respectively | 2,626 | 2,598 | ||||||
| Additional paid-in capital | 2,274,797 | 2,227,225 | ||||||
| Accumulated other comprehensive loss: | ||||||||
| Foreign currency translation adjustment | 4,576 | 2,785 | ||||||
| Unrealized (loss) gain on available-for-sale securities | (56 | ) | 40 | |||||
| Warrants | 12,387 | 12,387 | ||||||
| Accumulated deficit | (1,974,061 | ) | (1,768,610 | ) | ||||
| Total stockholders’ equity | 320,269 | 476,425 | ||||||
| Total Liabilities and Stockholders’ Equity | $ | 900,542 | $ | 850,207 | ||||
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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, | |||||||||||||||
| 2020 | 2019 | 2020 | 2019 | |||||||||||||
| Total operating expenses - as reported GAAP | $ | 111,799 | $ | 100,477 | $ | 350,851 | $ | 326,940 | ||||||||
| Research and development: | ||||||||||||||||
| Share-based compensation | 8,626 | 3,106 | 17,241 | 12,090 | ||||||||||||
| Selling, general and administrative: | ||||||||||||||||
| Share-based compensation | 7,282 | 5,737 | 19,671 | 19,432 | ||||||||||||
| Changes in fair value of contingent consideration payable | 1,034 | 789 | 2,680 | 2,652 | ||||||||||||
| Depreciation and amortization | 2,496 | 1,116 | 6,299 | 3,261 | ||||||||||||
| Total operating expense adjustments to reported GAAP | 19,438 | 10,748 | 45,891 | 37,435 | ||||||||||||
| Total operating expenses - as adjusted | $ | 92,361 | $ | 89,729 | $ | 304,960 | $ | 289,505 | ||||||||
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Exhibit 99.2
3Q20 Financial Results Conference Call & Webcast November 5, 2020
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 press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties . For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, including as they are impacted by COVID - 19 related disruption, are based on current information . The potential impact on operations from the COVID - 19 pandemic is inherently unknown and cannot be predicted with confidence and may cause actual results and performance to differ materially from the statements in this release, including without limitation, because of the impact on general political and economic conditions, including as a result of efforts by governmental authorities to mitigate COVID - 19 , such as travel bans, shelter in place orders and third - party business closures and resource allocations, manufacturing and supply chain disruptions and limitations on patient access to commercial or clinical product . In addition to the impact of the COVID - 19 pandemic, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation : the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective ; the potential that it may be difficult to enroll patients in our clinical trials ; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates ; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or our other product candidates if and when approved ; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues ; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products ; and the potential that we will need additional funding to complete all of our studies and manufacturing . Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results . Statements regarding corporate financial guidance and financial goals and the attainment of such goals . With respect to statements regarding projections of the Company's revenue and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans . In addition, all forward - looking statements are subject to other risks detailed in our Annual Report on Form 10 - K for the year ended December 31 , 2019 , the Quarterly Report filed on Form 10 - Q for the quarter ended June 30 , 2020 , and the Quarterly Report filed on Form 10 - Q 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 . 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 . Full reconciliations of GAAP results to the comparable non - GAAP measures for the reported periods appear in the financial tables section of this presentation . When we provide our expectation for non - GAAP operating expenses on a forward - looking basis, a reconciliation of the differences between the non - GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses . The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results .
3 A leading fully integrated, global rare disease biotechnology company Introduction Gene Therapy PLATFORM Protein Engineering & Glycobiology $509.1M Cash as of 9 /30/20 Two Clinical - Stage Gene Therapies AT - GAA Phase 3 in Pompe Disease GLOBAL COMMERCIAL ORGANIZATION World Class BIOLOGICS Capabilities Robust R&D Engine Nearly 50+ Lysosomal Disorders and More Prevalent Rare Diseases 3 EMPLOYEES in 27 Countries
4 2020 Key Strategic Priorities Introduction 1 2 3 4 5 Achieve global product revenue for Galafold of $250M - $260M Complete Pompe Phase 3 PROPEL study, enroll pediatric studies and advance manufacturing to support 2021 BLA and MAA Advance clinical development, manufacturing and regulatory discussions for CLN6 and CLN3 Batten programs Progress Pompe gene therapy towards IND and disclose up to two additional IND candidates Maintain strong financial position
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
6 Galafold Snapshot (as of September 30, 2020) 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, Japan, and other Countries Continued Expansion in 2020 $ 67.4M 3Q20 Galafold Revenue $ 250 - 260M FY20 Global Galafold Rev. Guidance 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
7 $48.8M $ 67.4 M 3Q19 Operational Growth FX Impact 3Q20 Year - over - Year Sales Growth Galafold Quarterly Performance Growth remains steady with Q3 revenue of $67.4M; $65.7M on a constant currency basis Galafold: Precision Medicine for Fabry Disease 3Q19 vs 3Q20 +81% $ 48.8 M $ 55.3 M $ 60.5 M $ 62.4 M $ 67.4 M 3Q19 4Q19 1Q20 2Q20 3Q20 Quarterly Galafold Sales +35% +3%
8 Galafold Growth Trajectory Galafold: Precision Medicine for Fabry Disease FY18 FY19 FY20 FY21 FY22 FY23 $91M $182M $500M+ ~$1B Projected Cumulative Revenues (2020 - 2022) $1B Annual Revenue Galafold is on track to generate $1B+ in projected cumulative revenues from 2020 - 2022 and is on an anticipated path to $500M+ in annual sales in 2023 and $1B+ annual sales at peak Peak $ 250M - $260M
AT - GAA: Next Potential Standard of Care for Pompe Disease “We encourage and embrace constant innovation” - Amicus Belief Statement
10 AT - GAA: Foundation in Protein Engineering AT - GAA for Pompe Disease Amicus scientists created a uniquely glycosylated and highly phosphorylated ERT (AT - GAA) that significantly enhances targeting to key muscles affected
11 Amicus POM - 002 Natural History Study Data from the Amicus POM - 002 chart review study in patients treated long - term with alglucosidase alfa is consistent with the medical literature and further supports PROPEL design assumptions Note: The blue solid curve is the estimated trendline obtained using a Loess regression with the smooth parameter at 0.8. Time (in months) on the X - axis was calculated from the actual assessment dates relative to the first dose of ERT. POM - 002: Longitudinal 6MWD van der Ploeg 2017: Longitudinal 6MWD Note: Data were integrated from 68 patients from two sequential clinical studies (Late - Onset Treatment Study [LOTS; NCT00158600] and LOTS Extension [NCT00455195]2) and the Pompe Registry; Ans T. van der Ploeg et al. Poster presented at the 13th Annual WORLD Symposium™ 2017, February 13 – 17, 2017, San Diego, CA, USA AT - GAA for Pompe Disease 0 20 40 60 80 100 120 140 Month 0 20 40 60 80 100 120 % P r e d i c t e d 6 M W D 0 20 40 60 80 100 120 140 Month 0 20 40 60 80 100 120 % P r e d i c t e d 6 M W D n=70 N=70 N=22 6MWT % Predicted 6MWT % Predicted Time from first infusion, months Time from first infusion, months 11
12 AT - GAA: Key Takeaways AT - GAA for Pompe Disease AT - GAA for Pompe Advances Toward Approval as “Crown Jewel” of Amicus Portfolio • PROPEL study timelines are on track with data expected 1Q2021 – To date, 97%+ of the 3,100+ planned infusions and assessments for the ongoing PROPEL study have been completed on schedule • Breakthrough Therapy Designation and the Promising Innovative Medicine designation highlight unmet need in Pompe disease • U.S. FDA grants rolling BLA submission and on - track to initiate in 4Q2020 • Expanded Access Program for infantile - onset Pompe patients underway • Process performance qualification (PPQ) runs with our partners at WuXi have been successfully completed for the drug substance and drug product • Peak revenue potential of $1B - $2B, with exclusivity well into 2030s
“We have a duty to obsolete our own technologies” - Amicus Belief Statement Next Generation Gene Therapy Platform
14 DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL Fabry Franchise Galafold ® (migalastat) Monotherapy Fabry Gene Therapy PENN Pompe Franchise AT - GAA ( Novel ERT + Chaperone) Pompe Gene Therapy PENN Batten Franchise – Gene Therapies CLN6 Batten Disease NCH CLN3 Batten Disease NCH CLN1 Batten Disease NCH Next Generation Research Programs and CNS Gene Therapies CDKL5 Deficiency Disorder GTx / ERT PENN Others NCH / PENN MPS Franchise Mepsevii ™ (vestronidase alfa) (Japan Only)* Next Generation MPSIIIA PENN MPSIIIB PENN Next Generation Gene Therapy Platform *Exclusive license from Ultragenyx for Japanese rights to Mepsevii™, investigator - sponsored trial in Japan underway ODD ODD ODD ODD RPD RPD PRIME LEGEND ODD - Orphan Drug Designation RPD - Rare Pediatric Disease Designation PRIME – Priority Medicines Designation
15 Interim Clinical Data for CLN6 Batten Disease Gene Therapy The Hamburg Motor & Language (HML) Score, an assessment of ambulation and speech, shows a meaningful effect in slowing disease progression at 24 months Next Generation Gene Therapy Platform Proportion of Patients with a Change in the HML Aggregate Score at Month 24 a,b Time to Unreversed 2 - point Decline in the HML Aggregate Score Difference in risk of unreversed 2 - point decline: −50% (95% CI, −77.5%, −3.6%) Exact p - value =0.0335 M+L, motor and language. NH, natural history. a The efficacy analysis included all patients with 24 - month HML data (8 of 13 treated patients in the study). b 24 - month HML data are available for 16 of 17 patients in the natural history cohort derived from a retrospective CLN6 natural history study conducted by Emily de los Reyes, MD (ClinicalTrials.gov Identifier: NCT03285425) . Data cutoff March 13, 2020. 0 20 40 60 80 100 >=1 0 -1 <=-2 Proportion of patients, % Change from baseline in HML score Treated Subjects Natural History Subjects 12.5% 12.5% 6 2.5% 37 .5% 50 % 25 % 0 % 0 % 8 7 7 16 13 10 8 5 4 1 0 12 Treated Subjects NH Subjects Months 0 4 8 12 16 20 24 28 32 36 40 Hazard ratio: 0.160 ( 95% CI, 0.021 – 1.238) Log - rank p - value=0.0354 1.0 0.9 0.8 0.7 0.6 0.5 0.4 0.3 0.2 0.1 0.0 Probability of no decline in HML score Treated Subjects NH Subjects
16 Next Generation Gene Therapy Platform Team of 200+ scientists bringing expertise and experience in: Protein Engineering & Glycobiology Expertise Clinical and Regulatory Expertise Global Commercial Infrastructure Vectors, Tropisms, Capsids Safety Dosing, Immunology Manufacturing, Scalability Combines Amicus and Penn Expertise Across Lysosomal and Rare Diseases Next - Generation Gene Therapy Platform An R&D platform with rights to 50+ diseases
17 Fabry Gene Therapy IND Candidate AAV with engineered GLA transgene demonstrated significantly better GL - 3 reduction than AAV with wild - type GLA. Full set of preclinical data to be presented at a conference in early 2021 Next Generation Gene Therapy Platform GLA Engineered for Stability Activity at Neutral pH of Circulation (pH 7.4) KO Mouse GTx: Kidney GL - 3 (% Tubules) Engineered dimer interface GLA#1 GLA#2 0 1 2 3 4 5 6 7 0 50 100 150 200 Time (days) R e l a t i v e A c t i v i t y ( % ) hGLA nat hGLA eng/stabilized 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 * * * * ▪ Engineered transgene for improved stability ▪ Proprietary AAV capsid ▪ Ubiquitous promoter IND Candidate: Approach
18 Gene Therapy: Updates & Key Takeaways • CLN6 Phase 1/2 interim data show positive impact with potential to become first - ever approved gene therapy for fatal brain disease in children • Manufacturing on track to initiate next clinical studies in CLN6 and CLN3 in 2021 using material from planned commercial process • Orphan drug designations granted in U.S. and EU for intrathecal AAV gene therapies for CLN6 and CLN3 Batten disease; CLN6 granted PRIME designation by EMA; CLN3 granted Fast Track designation by U.S. FDA • Preclinical studies ongoing for gene therapies in Pompe, Fabry, CDD, MPS IIIA, MPS IIIB and CLN1 • Penn collaboration is R&D engine, with rights to 50+ diseases Next Generation Gene Therapy Platform Portfolio of Gene Therapy Programs and Technologies Provides Foundation for Future
“We are business led and science driven” - Amicus Belief Statement Financial Summary
20 3Q20 Select Financial Results Financial Summary Sept. 30, 2020 (in thousands, except per share data) Sept. 30, 2019 Product Revenue 67,437 48,768 Cost of Goods Sold 8,399 5,596 R&D Expense 70,419 58,892 SG&A Expense 37,850 39,680 Changes in Fair Value of Contingent Consideration 1,034 789 Depreciation and Amortization 2,496 1,116 Loss from Operations (52,761) (57,305) Loss on Extinguishment of Debt (7,276) - Income Tax (Expense) Benefit (727) 251 Net Loss (64,011) (61,809) Net Loss Per Share (0.25) (0.24) 3Q20 revenue of $67.4M from global Galafold sales
21 Financial Outlook: Key Takeaways • Proceeds from July debt facility places Amicus firmly on a path to self - sustainability – Achieved through continued careful expense management, prioritization of early - stage research programs, and measured capital expenditures • Current cash position of $509.1M as of September 30 th • Company fully funded through major milestones in portfolio and continued global growth • Cumulative Galafold projected revenue of $1B+ in 2020 - 2022 offsets significant majority of company spend/investments • Reaffirming full - year Galafold revenue guidance of $250M to $260M and non - GAAP operating expense guidance of $410M to $420M Financial Summary
Closing Remarks “We are business led and science driven” - Amicus Belief Statement
Thank You “Our passion for making a difference unites us” - Amicus Belief Statement
Appendix
25 Reconciliation Appendix