8-K
AMICUS THERAPEUTICS, INC. (FOLD)
UNITEDSTATES
SECURITIESAND EXCHANGE COMMISSION
WASHINGTON,D.C. 20549
FORM
8-K
CURRENTREPORT PURSUANT TO
SECTION13 OR 15(d) OF THE
SECURITIESEXCHANGE ACT OF 1934
Date of Report (Date of earliest event reported): May 7, 2020
AMICUS THERAPEUTICS, INC.
(Exact Name of Registrant as Specified in Its Charter)
Delaware
(State or Other Jurisdiction of
Incorporation)
| Delaware | 001-33497 | 71-0869350 |
|---|---|---|
| (State or Other Jurisdiction<br><br> <br>of Incorporation) | (Commission<br><br> <br>File Number) | (I.R.S. Employer<br><br> <br>Identification No.) |
1 Cedar Brook Drive,
Cranbury, NJ 08512
(Address of PrincipalExecutive Offices, and Zip Code)
609-662-2000
Registrant’sTelephone Number, Including Area Code
(Former Name or Former Address, if Changed Since Last Report.)
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
| ¨ | Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425) |
|---|---|
| ¨ | Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12) |
| --- | --- |
| ¨ | Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b)) |
| --- | --- |
| ¨ | Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c)) |
| --- | --- |
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered |
|---|---|---|
| Common Stock Par Value $0.01 | FOLD | NASDAQ |
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (17 CFR §230.405) or Rule 12b-2 of the Securities Exchange Act of 1934 (17 CFR §240.12b-2). Emerging growth company ¨
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ¨
Item 2.02 Results of Operations and Financial Condition
On May 7, 2020, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended March 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 May 7, 2020 to discuss its first 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 May 7, 2020 |
| --- | --- |
| 99.2 | May 7, 2020 Conference Call Presentation Materials |
| --- | --- |
| 104 | Cover Page Interactive Data File (embedded within the<br>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: May 7, 2020 | By: | /s/ Ellen S. Rosenberg |
| Name: | Ellen S. Rosenberg | |
| Title: | Chief Legal Officer and Corporate Secretary |
Exhibit 99.1
Amicus Therapeutics Announces First Quarter2020 Financial Results and Corporate Updates
Galafold 1Q2020 Revenue of $60.5Million Reflects Continued Strong Adoption in All Key Global Regions, On Track to Achieve 2020 Revenue Guidance of $250M-$260M
AT-GAA in Late-Onset Pompe DiseaseGranted Rolling BLA Submission by U.S. FDA
CLN3 Batten Disease Gene Therapy GrantedFast Track Designation by U.S. FDA
Infantile-Onset Pompe Disease ExpandedAccess Program Underway
Cash Runway Now Well into 2H2022
Conference Call and Webcast Todayat 8:30 a.m. ET
CRANBURY, NJ, May 7, 2020 – Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced financial results for the first quarter ended March 31, 2020. The Company also summarized recent program updates and reiterated its full-year 2020 guidance.
Revenue
| · | Global revenue for Galafold^®^(migalastat) in the first quarter of 2020 was $60.5M and continues to track toward full-year 2020 revenue guidance of $250 millionto $260 million. First quarter revenue represented a year-over-year increase of 78% from total revenue of $34.0 million in<br>the first quarter of 2019**.** On a constant currency basis, first quarter 2020 total revenue was $61.4 million, representing<br>operational revenue growth measured at constant currency exchange rates of 81%, which was offset by a negative currency impact<br>of $0.9 million, or 3%. |
|---|---|
| · | First quarter revenue represents thecontinued performance across the global business, including new patient starts from switch and naïve patients throughout thequarter in all major regions, including those hardest hit by COVID-19. Performance driven largely by strong patient demand<br>with a minimal contribution from ordering patterns as healthcare organizations in some countries adjusted to the impact of the<br>COVID-19 virus. Global compliance and adherence rates continue to exceed 90%. |
| --- | --- |
Supply Chain
| · | Global supply chain for Galafold maintainscontinuous supply during the COVID-19 pandemic. With sufficient inventory on hand and product effectively moving down the supply<br>chain into individual countries, Amicus continues to fulfill the needs of Fabry patients without interruption. The company expects<br>to be able to continuously supply this oral precision medicine in all global geographies for any and all people living with Fabry<br>disease who have an amenable variant throughout the current COVID-19 pandemic and beyond. |
|---|---|
| · | Manufacturing and supply of AT-GAAremain intact globally. Amicus maintains focus on providing a continuous supply of AT-GAA to all patients and continues to<br>coordinate drug delivery per a site-by-site basis in order to address the needs of each patient. The Company has a high degree<br>of confidence that there is ample supply of AT-GAA to support all ongoing clinical studies in the Pompe program, including the<br>Phase 3 PROPEL study, which continues to track towards completing on schedule. |
| --- | --- |
Research and Development
| · | Global Phase 3 PROPEL clinical study of AT-GAA in late-onset Pompedisease remains on track for top line data in 1H21. To date, 97% out of the 2,250 planned infusions for the ongoing PROPEL<br>study have been completed on schedule. Process performance qualification (PPQ) runs have been successfully completed for the drug<br>substance with key strategic partner, WuXi Biologics, and will serve as the foundation for the Chemistry, Manufacturing, and Control<br>(CMC) module for a biologics license application (BLA) submission. The Company plans to initiate a rolling BLA for AT-GAA in the<br>second half of 2020, completing final submission in the first half of 2021. |
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| · | Expanded Access Program for infantile-onsetPompe patients underway. Amicus has initiated an expanded access program for its investigational medicine AT-GAA for young<br>children living with infantile-onset Pompe disease (IOPD). |
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| · | Plan to advance regulatory discussionsto finalize clinical and regulatory path in both CLN6 and CLN3 this year. Additional data in the CLN6 Phase 1/2 study, as well<br>as, initial CLN3 Phase 1/2 data expected later this year. |
|---|---|
| · | The U.S. Food and Drug Administration (FDA) has granted Fast TrackDesignation to the CLN3 Batten disease gene therapy, AT-GTX-502, for the treatment of pediatric patients less than 18 years ofage. The Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions, which<br>have the potential to address unmet medical needs. A drug development program with Fast Track designation is afforded greater access<br>to the FDA for the purpose of expediting the drug's development, review and potential approval. |
| --- | --- |
| · | Additional preclinical gene therapy data expected across multiple programs in industry leading gene therapy portfolio. As previously announced, one oral presentation and two posters<br> accepted at the American Society of Gene & Cell Therapy 23rd Annual Meeting<br> being held virtually on May 12–15. |
| --- | --- |
Financial Strength
| · | Cash runway now extended to well into 2H2022. Amicus continues<br>to carefully manage expenses and investments, while executing on the Galafold launch and advancing development programs. Extension<br>of the cash runway will be achieved through continued careful expense management, prioritization of very early stage research programs<br>and more measured capital expenditures. |
|---|---|
| · | First quarter non-GAAP operating expense on track for full-yearguidance. First quarter operating expenses driven by the timing of charges related to payments of the process performance qualification<br>runs of AT-GAA and initiation of the tech transfer to our gene therapy contract manufacturing partners. Non-GAAP operating expense<br>to decline throughout 2020 on path to achieving the 2020 non-GAAP operating expense guidance of $410 million to $420 million. |
| --- | --- |
2020 Key Strategic Priorities
| · | Achieve $250 million to $260 million of<br>global product revenue for Galafold |
|---|---|
| · | Complete Pompe Phase 3 PROPEL study, enroll<br>pediatric studies and advance manufacturing to support 2021 BLA and MAA |
| --- | --- |
| · | Advance clinical development, manufacturing<br>and regulatory discussions for CLN6 and CLN3 Batten programs |
| --- | --- |
| · | Progress Pompe gene therapy towards Investigational<br>New Drug (IND) application and disclose up to two additional IND candidates |
| --- | --- |
| · | Maintain strong financial position |
| --- | --- |
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “During the first quarter, we have made tremendous progress advancing our mission for patients. Following success in the first quarter, we are on track and well-capitalized to achieve our 2020 key strategic priorities including our global Fabry launch, Pompe late-stage development program, and advancing our industry-leading gene therapy pipeline. In addition, we have taken steps to prioritize our spend and now see our cash runway lasting well into the second half of 2022. 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.”
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First Quarter 2020 Financial Results
| · | Total revenue in the first quarter 2020<br>was $60.5 million, a year-over-year increase of 78% from total revenue of $34.0 million in the first quarter of 2019. On a constant<br>currency basis, first quarter 2020 total revenue was $61.4 million, representing operational revenue growth measured at constant<br>currency exchange rates of 81%, which was offset by a negative currency impact of $0.9 million, or 3%. |
|---|---|
| · | Cash, cash equivalents, and marketable<br>securities totaled $338.9 million at March 31, 2020, compared to $452.7 million at December 31, 2019. |
| --- | --- |
| · | Total GAAP operating expenses of $132.0<br>million for the first quarter 2020 increased as compared to $111.3 million for the first quarter 2019, reflecting continued investments<br>in the Galafold launch, Pompe program, and gene therapy pipeline. |
| --- | --- |
| · | Total non-GAAP operating expenses of $116.7<br>million for the first quarter of 2020 increased as compared to $96.2 million in the first quarter of 2019, reflecting continued<br>investments in the Galafold launch, Pompe clinical study program, and gene therapy pipeline. First quarter non-GAAP operating expense<br>on track for full-year guidance. Non-GAAP operating expense to decline throughout 2020 on path to achieving the 2020 expense guidance<br>of $410 million to $420 million.^1^ |
| --- | --- |
| · | Net loss was $88.9 million, or $0.35 per<br>share, compared to a net loss of $120.3 million, or $0.56 per share, for the first quarter 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.
2020 Financial Guidance
| · | For the full-year 2020, the Company anticipates<br>total Galafold revenue of $250 million to $260 million based on the average exchange rates for 2019. |
|---|---|
| · | Non-GAAP operating expense guidance for<br>the full-year 2020 is $410 million to $420 million, driven by continued investment in the global Galafold launch, AT-GAA clinical<br>studies, and advancing our gene therapy pipeline.^2^ |
| --- | --- |
| · | Cash, cash equivalents, and marketable<br>securities totaled $338.9 million at March 31, 2020. The current cash position is anticipated to fund ongoing operations now well<br>into 2H2022. |
| --- | --- |
^^
^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 2020 Milestones by Program
Galafold (migalastat) Oral PrecisionMedicine for Fabry Disease
| · | On track to meet full-year 2020 revenue<br>guidance range of $250 million to $260 million |
|---|---|
| · | Continued geographic expansion |
| --- | --- |
| · | Registry and other Phase 4 supportive<br>studies underway |
| --- | --- |
AT-GAA for Pompe Disease
| · | Plans to initiate a Rolling Biologics<br>License Application (BLA) for AT-GAA in 2020, with addition of complete clinical results for PROPEL in 1H2021 to support full approval |
|---|---|
| · | Retrospective natural history study data<br>in approximately 100 enzyme replacement therapy treated Pompe patients |
| --- | --- |
| · | Additional supportive studies, including<br>an open-label study in 12- to <18-year-old patients |
| --- | --- |
Gene Therapy Portfolio
| · | Report further safety and efficacy data<br>in the CLN6 Batten disease Phase 1/2 study and advance regulatory discussions to finalize clinical and regulatory path |
|---|---|
| · | Report initial data from the CLN3 Batten<br>disease Phase 1/2 study and advance regulatory discussions to finalize clinical and regulatory path |
| --- | --- |
| · | Continue IND-enabling toxicology work<br>in Pompe disease and progress towards IND |
| --- | --- |
| · | Additional preclinical data expected across<br>multiple programs with disclosure of up to two additional IND candidates |
| --- | --- |
| · | Manufacturing advancements across portfolio |
| --- | --- |
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Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, May 7, 2020 at 8:30 a.m. ET to discuss the first 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: 1287896.
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 May 7, 2020. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 1287896.
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, 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<br>therapy. |
|---|---|
| · | Galafold is not recommended for use in patients with Fabry disease<br>who have severe renal impairment (<30 mL/min/1.73 m^2^). The safety and efficacy of Galafold in children 0–15<br>years of age have not yet been established. |
| --- | --- |
| · | No dosage adjustments are required in patients with hepatic impairment<br>or in the elderly population. |
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| · | There is very limited experience with the use of this medicine in<br>pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until<br>you have checked with your doctor, pharmacist, or nurse. |
| --- | --- |
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| · | While taking Galafold, effective birth control should be used. It<br>is not known whether Galafold is excreted in human milk. |
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| · | Contraindications to Galafold include hypersensitivity to the active<br>substance or to any of the excipients listed in the PRESCRIBING INFORMATION. |
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| · | It is advised to periodically monitor renal function, echocardiographic<br>parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold. |
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| · | OVERDOSE: General medical care is recommended in the case of Galafold<br>overdose. |
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| · | The most common adverse reaction reported was headache, which was<br>experienced by approximately 10% of patients who received Galafold. For a complete list of adverse reactions, please review the<br>SUMMARY OF PRODUCT CHARACTERISTICS. |
| --- | --- |
| · | Call your doctor for medical advice about<br>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, 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 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.
CONTACTS:
Amicus Therapeutics
Andrew Faughnan
Director, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
FOLD–G
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TABLE 1
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(Unaudited)
(in thousands, except share and per shareamounts)
| Three Months Ended March 31, | ||||||
|---|---|---|---|---|---|---|
| 2020 | 2019 | |||||
| Net product sales | $ | 60,525 | $ | 34,046 | ||
| Cost of goods sold | 6,552 | 4,055 | ||||
| Gross profit | 53,973 | 29,991 | ||||
| Operating expenses: | ||||||
| Research and development | 89,120 | 64,593 | ||||
| Selling, general, and administrative | 40,215 | 44,303 | ||||
| Changes in fair value of contingent consideration payable | 931 | 1,383 | ||||
| Depreciation and amortization | 1,764 | 991 | ||||
| Total operating expenses | 132,030 | 111,270 | ||||
| Loss from operations | (78,057 | ) | (81,279 | ) | ||
| Other income (expense): | ||||||
| Interest income | 1,515 | 2,639 | ||||
| Interest expense | (3,729 | ) | (6,454 | ) | ||
| Loss on exchange of convertible notes | — | (36,123 | ) | |||
| Other (expense) income | (8,316 | ) | 1,086 | |||
| Loss before income tax | (88,587 | ) | (120,131 | ) | ||
| Income tax expense | (361 | ) | (168 | ) | ||
| Net loss attributable to common stockholders | $ | (88,948 | ) | $ | (120,299 | ) |
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.35 | ) | $ | (0.56 | ) |
| Weighted-average common shares outstanding — basic and diluted | 256,968,248 | 213,519,287 |
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per shareamounts)
| December 31, 2019 | |||||
|---|---|---|---|---|---|
| Assets | |||||
| Current assets: | |||||
| Cash and cash equivalents | 123,231 | $ | 142,837 | ||
| Investments in marketable securities | 215,642 | 309,903 | |||
| Accounts receivable | 40,555 | 33,284 | |||
| Inventories | 12,831 | 14,041 | |||
| Prepaid expenses and other current assets | 15,690 | 20,008 | |||
| Total current assets | 407,949 | 520,073 | |||
| Operating lease right-of-use assets, less accumulated amortization of 6,260 and 5,342 at March 31, 2020 and December 31, 2019, respectively | 32,501 | 33,315 | |||
| Property and equipment, less accumulated depreciation of 19,260 and 17,604 at March 31, 2020 and December 31, 2019, respectively | 47,688 | 47,705 | |||
| In-process research & development | 23,000 | 23,000 | |||
| Goodwill | 197,797 | 197,797 | |||
| Other non-current assets | 29,414 | 28,317 | |||
| Total Assets | 738,349 | $ | 850,207 | ||
| Liabilities and Stockholders’ Equity | |||||
| Current liabilities: | |||||
| Accounts payable | 16,239 | $ | 21,722 | ||
| Accrued expenses and other current liabilities | 66,722 | 98,651 | |||
| Deferred reimbursements | 1,250 | 1,250 | |||
| Operating lease liabilities | 7,503 | 7,189 | |||
| Total current liabilities | 91,714 | 128,812 | |||
| Deferred reimbursements | 8,906 | 8,906 | |||
| Convertible notes | 2,167 | 2,131 | |||
| Senior secured term loan | 147,569 | 147,374 | |||
| Contingent consideration payable | 23,612 | 22,681 | |||
| Deferred income taxes | 5,051 | 5,051 | |||
| Operating lease liabilities | 52,522 | 53,531 | |||
| Other non-current liabilities | 4,214 | 5,296 | |||
| Total liabilities | 335,755 | 373,782 | |||
| Commitments and contingencies | |||||
| Stockholders’ equity: | |||||
| Common stock, 0.01 par value, 500,000,000 shares authorized, 257,449,955 and 255,417,869 shares issued and outstanding at March 31, 2020 and December 31, 2019, respectively | 2,607 | 2,598 | |||
| Additional paid-in capital | 2,238,346 | 2,227,225 | |||
| Accumulated other comprehensive loss: | |||||
| Foreign currency translation adjustment | 6,981 | 2,785 | |||
| Unrealized (loss) gain on available-for-sale securities | (169 | ) | 40 | ||
| Warrants | 12,387 | 12,387 | |||
| Accumulated deficit | (1,857,558 | ) | (1,768,610 | ) | |
| Total stockholders’ equity | 402,594 | 476,425 | |||
| Total Liabilities and Stockholders’ Equity | 738,349 | $ | 850,207 |
All values are in US Dollars.
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP FinancialMeasures
(in thousands)
| Three Months Ended March 31, | ||||
|---|---|---|---|---|
| 2020 | 2019 | |||
| Total operating expenses - as reported GAAP | $ | 132,030 | $ | 111,270 |
| Research and development: | ||||
| Share-based compensation | 5,253 | 5,032 | ||
| Selling, general and administrative: | ||||
| Share-based compensation | 7,343 | 7,712 | ||
| Changes in fair value of contingent consideration payable | 931 | 1,383 | ||
| Depreciation and amortization | 1,764 | 991 | ||
| Total operating expense adjustments to reported GAAP | 15,291 | 15,118 | ||
| Total operating expenses - as adjusted | $ | 116,739 | $ | 96,152 |
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Exhibit 99.2
| 1Q20 Financial Results Conference Call & Webcast May 7, 2020 |
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| 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, 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 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<br>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. |
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| Introduction 3 PLATFORM & Glycobiology AT-GAA Phase 3 in Pompe Disease Two Clinical-Stage Gene Therapies $338M+ Cash as of 3/31/20 GLOBAL COMMERCIAL ORGANIZATION Robust R&D Engine Nearly 50+ Lysosomal Disorders and More Prevalent Rare Diseases EMPLYEES in 27ountries Gene Therapy Protein Engineering World Class BIOLOGICS Capabilities A leading fully-integrated, global rare disease biotechnology company |
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| Introduction 4 2020 Key Strategic Priorities 1 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 2 3 4 5 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 |
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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 6 Galafold Snapshot (as of March 31, 2020) One of the Most Successful $250-260M FY20 Global $60.5M 1Q20 Galafold R Rare Disease Launches nce Continue Expansio in 2020 348 ble Variants in U.S. Label 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 the cornerstone of Amicus’ success.It 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 enzyme replacement therapy |
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| Galafold: Precision Medicine for Fabry Disease 7 Galafold Quarterly Performance Quarterly Galafold Sales Year-over-Year Galafold Sales Growth $60.5M 1Q19 2Q19 3Q19 4Q19 1Q20 1Q19 Operational Growth FX Impact 1Q20 1Q19 vs 1Q20 $55M $49M $44M $34M -3% $60.5M $34M +81% Quarterly Growth Remains Steady with 1Q20 Revenue of $60.5M, Growing 78% Year-over-Year |
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| Galafold: Precision Medicine for Fabry Disease 8 Galafold Growth Trajectory $1B Annual Revenue $500M+ $250M-$260M $91M $182M FY18 FY19 FY20 FY21 ~$1B FY22 FY23 Peak Projected Cumulative Revenues (2020-2022) 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 |
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| Amicus Global Supply Chain 9 Global Supply Chain Galafold Carbogen, Switzerland Existing Supply Chain Strategy: • Galafold: Hold multiple years of inventory in API and Drug Product • AT-GAA: Built inventory ahead of time and move drug product to UK AT-GAA Wuxi, China AL M AC C L IN IC A L S ERV I C ES United Kingdom W OR L D C OU R I E R D E P OT Japan Post COVID-19: • Galafold: Push inventory into the supply chain as far as possible down to country and pharmacy level • AT-GAA: Push inventory into supply chain as far as possible and coordinate site by site for delivery AL M AC C L I N I C AL S ERV I C ES USA Z UE LLI G P H A R M A D E P O T Taiwan W O R L D Argentina C O U R I E R DE P O T F L I N DE R S DE P O T Australia |
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| AT-GAA:Next Potential Standardof Care for Pompe Disease “We encourage and embrace constant innovation” - Amicus Belief Statement |
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| AT-GAA for Pompe Disease 11 AT-GAA: Key Takeaways • PROPEL study timelines are on track with data expected 1H2021 – To date, 97% of the 2,250 planned infusions 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 today U.S. FDA grants rolling BLA submission and company plans to initiate in 2H2020 Expanded Access Program for infantile-onset Pompe patients underway • • • Process performance qualification (PPQ) runs with our partners WuXi have been successfully completed for the drug substance at • Peak revenue potential of $1B-$2B, with exclusivity well into 2030s AT-GAA for Pompe Advances Toward Approval as “Crown Jewel” of Amicus Portfolio |
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| Next GenerationGene TherapyPlatform “We have a duty to obsolete our own technologies” - Amicus Belief Statement |
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| Next Generation Gene Therapy Platform 13 sease Designation *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 + Chaperone) Pompe Gene Therapy PENN Batten Franchise – Gene Therapies CLN6 Batten Disease NCH CLN3 Batten Disease NCH CLN8 Batten Disease NCH CLN1 Batten Disease NCH Next Generation Research Programs and CNS Gene Therapies CDKL5 Deficiency Disorder GTx / ERT PENN Niemann-Pick Type C (NPC) NCH / PENN Others NCH / PENN MPS Franchise LEGEND ODD - Orphan Drug Designation RPD - Rare Pediatric Mepsevii™ (vestronidase alfa) (Japan Only)* Next Generation MPSIIIA PENN MPSIIIB PENN Di ODD RPD ODD RPD ODD ODD |
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| Next Generation Gene Therapy Platform 14 Gene Therapy: Updates & Key Takeaways • CLN6 Phase 1/2 interim data show profound impact with potential to become first ever approved gene therapy for fatal brain disease in children Additional patients to be dosed in Phase 1/2 study of CLN3 in 2021 with commercial supply Orphan drug designations granted in U.S. and EU for intrathecal AAV gene therapies for CLN6 and CLN3 Batten disease; CLN3 granted Fast Track designation by U.S. FDA Pompe gene therapy clinical candidate declared to move into IND-enabling studies Penn Collaboration is R&D engine, with rights to 50+ diseases 8 preclinical gene therapies in development • • • • • Portfolio of Gene Therapy Programs and Technologies Provides Foundation for Future |
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| Next Generation Gene Therapy Platform 15 Combines Amicus and Penn Expertise Across Lysosomal and Rare Diseases Protein Engineering & Glycobiology Expertise Clinical and Regulatory Expertise Global Commercial Infrastructure Team of 200+ scientists bringing expertise and experience in: Vectors, Tropisms, Capsids Safety Dosing, Immunology Manufacturing, Scalability Next-Generation Gene Therapy Platform Driving 1-2 new INDs every year starting in 2021 An R&D platform with rights to 50+ diseases, including 8 active preclinical programs |
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| Financial Summary “We are business led and science driven” - Amicus Belief Statement |
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| Financial Summary 17 1Q2020 Select Financial Results (in thousands, except per share data) Mar. 31, 2020 Mar. 31, 2019 Product Revenue 60,525 34,046 Cost of Goods Sold 6,552 4,055 R&D Expense 89,120 64,593 SG&A Expense 40,215 44,303 Changes in Fair Value of Contingent Consideration 931 1,383 Depreciation and Amortization 1,764 991 Loss from Operations (78,057) (81,279) Income Tax Expense (361) (168) Net Loss (88,948) (120,299) Net Loss Per Share (0.35) (0.56) 1Q2020 Revenue of $60.5M Primarily from Global Galafold Sales |
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| Financial Summary 18 Cash Runway Now to Well into 2H2022 (~2+ years) $338M+ Cash 1Q2020 Well into ~2+ Years Cash Runway 2H2022 Fully funded through major milestones in portfolio and continued global growth |
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| Financial Summary 19 Financial Outlook: Key Takeaways • Cash runway now well into 2H2022 – Achieved through continued careful expense management, prioritization of very early stage research programs and more measured capital expenditures • Non-GAAP quarterly operating expense expected to decline throughout 2020 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 Only modest additional capital required in the outer years to extend runway into profitability with multiple non-equity sources available as/when needed • • • |
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| ClosingRemarks “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 of Non-GAAP Financial Measures (in thousands) Three Months Ended March 31, 2020 2019 s Total operating expenses - as reported GAAP Research and development: Share-based compensation Selling, general and administrative: Share-based compensation Changes illfair value of contingent consideration payable Depreciation and amortization Total operating expense adjustments to reported GAAP Total operating expenses - as adjusted $ 132,030 1ll,270 5,253 5,032 7,343 931 1,764 7,712 1,383 991 15,291 s ]}6,739 15,118 $ 96,152 Ll..Amicus {v-Therapeutics- |
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