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
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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 August 8, 2023, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal quarter ended June 30, 2023. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on August 8, 2023 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 Item 2.02 of 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 5.02. – Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officer.
The Company announced today that Daphne Quimi, the Company’s Chief Financial Officer, notified the Company on August 3, 2023 that she intends to retire before the end of 2023. Both the Company and Ms. Quimi expect that she will remain employed by the Company as Chief Financial Officer until a successor Chief Financial Officer is appointed. The Company and Ms. Quimi also expect that Ms. Quimi will remain employed by the Company for a transition period following the appointment of such successor to ensure an orderly transition of duties and responsibilities.
The Company is conducting an external search for its next Chief Financial Officer, and through this search the Company has already identified a number of highly qualified candidates.
Forward Looking Statements
This Current Report on Form 8-K contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. 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. All forward-looking statements are subject to risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2022 and the Quarterly Report filed on Form 10-Q for the quarter ended March 31, 2023. 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.
Item 9.01 Financial Statements and Exhibits
(d) Exhibits:
| Exhibit No. | Description |
| 99.1 | Press Release dated August 8, 2023 |
| 99.2 | August 8, 2023 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: August 8, 2023 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
Amicus Therapeutics Announces Second Quarter 2023 Financial Results and Corporate Updates
1H 2023 Revenue Growth of 16% at CER to $180.8M
Raising FY 2023 Galafold® Revenue Growth Guidance to 14%-18% at CER
EU Launch of Pombiliti® + Opfolda® Underway; U.S. and U.K. Approvals Expected 3Q 2023
Reducing 2023 Non-GAAP Operating Expense Guidance to $330M-$350M
Non-GAAP Profitability Projected in 2H 2023
Conference Call and Webcast Today at 8:30 a.m. ET
PHILADELPHIA, PA, Aug. 8, 2023 – Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused on developing and commercializing novel medicines for rare diseases, today announced financial results for the second quarter ended June 30, 2023.
Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Amicus has made tremendous progress through the first half of 2023, highlighted by the strong growth of Galafold and the regulatory approval of Pombiliti and Opfolda in the EU. We are confident in our trajectory towards delivering on our core objectives this year, including the continued growth of Galafold and the anticipated approvals of Pombiliti and Opfolda in the U.S. and U.K. this quarter. I am also very pleased to share we have revised our revenue guidance upwards and are reducing operating expense guidance for the year further supporting our path to non-GAAP profitability. Amicus is well positioned to deliver sustainable value for our shareholders while advancing our mission to deliver great medicines for people living with rare diseases.”
Corporate Highlights:
| · | Global revenue in the second quarter 2023 was $94.5 million. Second quarter revenue represented a year-over-year increase of 17% from total revenue of $80.7 million in the second quarter 2022. Second quarter performance reflected a strong operational revenue growth of 17% measured at constant exchange rates (CER)1 and a negligible currency impact of $0.2 million, or 0%. |
| · | Global revenue in the first half 2023 was $180.8 million. First half revenue represented a year-over-year increase of 13% from total revenue of $159.4 million in the first half 2022. First half performance reflected strong operational revenue growth of 16% measured at constant exchange rates (CER)1 and a negative currency impact of $4.1 million, or 3%. |
| Three Months Ended June 30, | Year over Year % Growth | Six Months Ended June 30, | Year over Year % Growth | |||||||||||||||||||||||||||||
| (in thousands) | 2023 | 2022 | As Reported | at CER1 | 2023 | 2022 | As Reported | at CER1 | ||||||||||||||||||||||||
| Net Product Revenues | $ | 94,503 | $ | 80,731 | 17 | % | 17 | % | $ | 180,773 | $ | 159,446 | 13 | % | 16 | % | ||||||||||||||||
| · | Given strong operational performance in the first half of 2023, the Company now anticipates Galafold® revenue growth of 14-18% at CER1 for the full-year 2023. Growth is expected to be driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, label extensions, continued diagnosis of new Fabry patients, and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. |
| · | Commercial Launch of Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) underway in the EU. In the EU, the European Commission granted full approval of Pombiliti + Opfolda for the treatment of adults with late-onset Pompe disease (LOPD). The Company has initiated the commercial launch of Pombiliti + Opfolda in Germany and reimbursement discussions with healthcare authorities in additional European countries are underway. |
1
| · | U.S. and U.K. regulatory reviews of AT-GAA remain on-track. The Company continues to expect regulatory approvals of AT-GAA in both the U.S. and U.K. in the third quarter of 2023. |
| · | Galafold U.S. intellectual property estate further strengthened following the issuance of multiple new patents in 2023. Galafold is protected by orphan drug regulatory exclusivities and a broad U.S. intellectual property portfolio of 53 orange book-listed patents, including 9 composition of matter patents, 37 of which provide protection through 2038 and beyond. |
| · | Full-year 2023 non-GAAP operating expense guidance reduced to $330 million to $350 million, driven by prudent expense management while investing in AT-GAA manufacturing and launch activities. |
| · | Based on the current operating plan and the timing of AT-GAA approvals, the Company is on-track to achieve non-GAAP profitability2 in the second half of 2023. |
| · | Amicus announces retirement of Chief Financial Officer, Daphne Quimi. After 15 years of distinguished leadership at Amicus, Daphne Quimi has decided to retire. Ms. Quimi will remain in her role as CFO until her successor is appointed and will remain with Amicus through the end of the year in order to support a smooth transition. |
Second Quarter 2023 Financial Results
| · | Total revenue in the second quarter 2023 was $94.5 million, a year-over-year increase of 17% from total revenue of $80.7 million in the second quarter 2022. On a constant currency basis, second quarter 2023 total revenue growth was 17%. Currency impact on reported revenue in the second quarter of 2023 represented a negligible amount of $0.2 million, or 0%. | |
| · | Total GAAP operating expenses of $104.2 million for the second quarter 2023 decreased as compared to $133.1 million for the second quarter 2022. | |
| · | Total non-GAAP operating expenses of $84.0 million for the second quarter 2023 decreased as compared to $119.2 million for the second quarter 2022, primarily reflecting decreased program spend.3 | |
| · | Net loss was $43.2 million, or $0.15 per share in the second quarter 2023, and was reduced compared to a net loss of $62.2 million, or $0.21 per share, for the second quarter 2022. | |
| · | Cash, cash equivalents, and marketable securities totaled $265.6 million at June 30, 2023, compared to $293.6 million at December 31, 2022. |
2023 Financial Guidance
| · | For the full-year 2023, the Company now anticipates total Galafold revenue growth between 14 and 18% at CER1 driven by several factors including continued strong underlying demand from both switch and treatment-naïve patients further geographic expansion and label extensions, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. | |
| · | Amicus is reducing its non-GAAP operating expense guidance for the full-year 2023 to $330 million to $350 million, driven by prudent expense management offset by continued investment in Galafold, AT-GAA clinical studies, non-recurring costs for manufacturing as well as global launch activities4. | |
| · | The Company is on-track to achieve non-GAAP profitability2 in the second half of 2023. |
Amicus is focused on the following five key strategic priorities in 2023:
| · | Sustain double-digit Galafold revenue growth (14-18% at CER1) | |
| · | Secure EMA, MHRA and FDA approvals for Pombiliti + Opfolda | |
| · | Initiate successful global launches of Pombiliti + Opfolda | |
| · | Advance next generation pipeline programs (Fabry GTx, Fabry Next-Generation Chaperone, Pompe GTx) | |
| · | Maintain strong financial position on path to profitability |
1 In order to illustrate underlying performance, Amicus discusses its results in terms of constant exchange rate (CER) growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period. Full-year 2023 Galafold revenue guidance utilizes the actual exchange rates at December 31, 2022.
2 Based on projections of Amicus’ non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. Amicus defines non-GAAP Net Income as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes.
3 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.
4 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.
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Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, August 8, 2023, at 8:30 a.m. ET to discuss the second quarter 2023 financial results and corporate updates. Participants and investors interested in accessing the call by phone will need to register using the online registration form. After registering, all phone participants will receive a dial-in number along with a personal PIN to access the event.
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. An archived webcast and accompanying slides will be available on the Company's website shortly after the conclusion of the live event.
About Galafold
Galafold® (migalastat) 123 mg capsules is an oral pharmacological chaperone of alpha-Galactosidase A (alpha-Gal A) for the treatment of Fabry disease in adults who have amenable galactosidase alpha gene (GLA) variants. In these patients, Galafold works by stabilizing the body’s own dysfunctional enzyme so that it can clear the accumulation of disease substrate. Globally, Amicus Therapeutics estimates that approximately 35 to 50 percent of Fabry patients may have amenable GLA variants, though amenability rates within this range vary by geography. Galafold is approved in more than 40 countries around the world, including the U.S., EU, U.K., and Japan.
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 Therapeutic Indication
Galafold® (migalastat) is indicated for long-term treatment of adults and adolescents aged 12 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.
EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialist physicians experienced in the diagnosis and treatment of Fabry disease. Galafold is not intended for concomitant use with enzyme replacement therapy.
The safety and efficacy of Galafold in children aged less than 12 years have not been established. No data are available.
Galafold is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients listed in the Summary of Product Characteristics (SmPC).
Galafold 123 mg capsules are not for children (≥12 years) weighing less than 45 kg.
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It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on or switched to Galafold. In case of meaningful clinical deterioration, further clinical evaluation or discontinuation of treatment with Galafold should be considered.
Galafold is not indicated for use in patients with non-amenable mutations.
Galafold is not recommended for use in patients with severe renal insufficiency, defined as estimated GRF less than 30 mL/min/1.73m2.
Food and caffeine should not be consumed at least 2 hours before and 2 hours after taking Galafold to give a minimum 4 hours fast.
Galafold is not recommended in women of childbearing potential not using contraception. Galafold is not recommended during pregnancy. It is not known whether Galafold is secreted in human milk.
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 SmPC.
OVERDOSE: General medical care is recommended in the case of Galafold overdose.
For complete information please see the EU SmPC available at https://www.ema.europa.eu/en/medicines/human/EPAR/galafold
About Pombiliti® + Opfolda®
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa, a bis-M6P-enriched rhGAA that facilitates high-affinity uptake through the M6P receptor while retaining its capacity for processing into the most active form of the enzyme, and the oral enzyme stabilizer, miglustat, that’s designed to reduce loss of enzyme activity in the blood. In clinical studies, Pombiliti + Opfolda was associated with demonstrated improvements in both musculoskeletal and respiratory measures.
Important Safety Information
Pombiliti (cipaglucosidase alfa) Important Safety Information
Posology and Method of Administration: Pombiliti must be used in combination with miglustat 65 mg hard capsules. The recommended dose of Pombiliti is 20 mg/kg of body weight every other week. The Pombiliti infusion should start 1 hour after taking miglustat capsules. Paediatric population: The safety and efficacy of Pombiliti in combination with miglustat therapy in paediatric patients less than 18 years old have not yet been established. No data are available. Contraindications: Life-threatening hypersensitivity to the active substance, or to any of the excipients. Contraindication to miglustat. Anaphylaxis and infusion-associated reactions (IARs): Serious anaphylaxis and IARs have occurred in some patients during infusion and following infusion with Pombiliti. Premedication with oral antihistamine, antipyretics, and/or corticosteroids may be administered to assist with signs and symptoms related to IARs experienced with prior enzyme replacement therapy (ERT) treatment. Reduction of the infusion rate, temporary interruption of the infusion, symptomatic treatment with oral antihistamine, or antipyretics, and appropriate resuscitation measures should be considered to manage serious IARs. If anaphylaxis or severe allergic reactions occur, infusion should be immediately paused, and appropriate medical treatment should be initiated. The current medical standards for emergency treatment of anaphylactic reactions are to be observed and cardiopulmonary resuscitation equipment should be readily available. The risks and benefits of re-administering Pombiliti following anaphylaxis or severe allergic reaction should be carefully considered, and appropriate resuscitation measures made available. Risk of acute cardiorespiratory failure in susceptible patients: Patients with acute underlying respiratory illness or compromised cardiac and/or respiratory function may be at risk of serious exacerbation of their cardiac or respiratory compromise during infusions. Appropriate medical support and monitoring measures should be readily available during Pombiliti infusion. Immune complex-related reactions: Immune complex-related reactions have been reported with other ERTs in patients who had high IgG antibody titres, including severe cutaneous reactions and nephrotic syndrome. If immune complex-related reactions occur, discontinuation of the administration of Pombiliti should be considered and appropriate medical treatment should be initiated. The risks and benefits of re-administering Pombiliti following an immune complex-related reaction should be reconsidered for each individual patient. Contraception in females: Reliable contraceptive measures must be used by women of childbearing potential during treatment with Pombiliti in combination with miglustat, and for 4 weeks after discontinuing treatment. Pregnancy: Pombiliti in combination with miglustat therapy is not recommended during pregnancy. Breast feeding: It is not known if Pombiliti and miglustat are secreted in human breast milk. A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from Pombiliti in combination with miglustat therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman. Summary of the safety profile: The most commonly reported adverse reactions only attributable to Pombiliti were chills (4.0%), dizziness (2.6%), flushing (2.0%), somnolence (2.0%), chest discomfort (1.3%), cough, (1.3%), infusion site swelling (1.3%), and pain (1.3%). Reported serious adverse reactions only attributable to Pombiliti were urticaria (2.0%), anaphylaxis (1.3%), pyrexia (0.7%), presyncope (0.7%), dyspnoea (0.7%), pharyngeal oedema (0.7%), wheezing (0.7%), and hypotension (0.7%). Refer to SmPC for full list.
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Opfolda (miglustat) 65 mg hard capsules Important Safety Information
Posology and Method of Administration: Opfolda must be used in combination with Pombiliti. The recommended dose is to be taken orally every other week and is based on body weight. Opfolda should be taken approximately 1 hour but no more than 3 hours before the start of the Pombiliti infusion. Paediatric population: The safety and efficacy of Opfolda in combination with Pombiliti therapy in paediatric patients less than 18 years old have not yet been established. No data are available. Contraindications: Hypersensitivity to the active substance or to any of the excipients. Contraindication to cipaglucosidase alfa. Food Interaction: Patients should fast for 2 hours before and 2 hours after taking Opfolda. Contraception in females: Reliable contraceptive measures must be used by women of childbearing potential during treatment with Opfolda in combination with Pombiliti, and for 4 weeks after discontinuing treatment. Pregnancy: Opfolda crosses the placenta. Opfolda in combination with Pombiliti therapy is not recommended during pregnancy. Breast feeding: It is not known if Opfolda and Pombiliti are secreted in human breast milk. A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from Opfolda in combination with Pombiliti therapy taking into account the benefit of breastfeeding for the child and the benefit of therapy for the woman. Summary of the safety profile: The most commonly reported adverse reaction only attributable to Opfolda 65 mg was constipation (1.3%). Refer to SmPC for full list.
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 diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a pipeline of cutting-edge, first- or best-in-class medicines for rare 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. 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 Statement
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, 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, MHRA, and PMDA, may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or AT-GAA 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, 2022, and on Form 10-Q for the quarter ended June 30, 2023, 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.
CONTACT:
Investors:
Amicus Therapeutics
Andrew Faughnan
Vice President, 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 June 30, | Six Months Ended June 30, | |||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Net product sales | $ | 94,503 | $ | 80,731 | $ | 180,773 | $ | 159,446 | ||||||||
| Cost of goods sold | 9,114 | 8,197 | 16,056 | 15,779 | ||||||||||||
| Gross profit | 85,389 | 72,534 | 164,717 | 143,667 | ||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | 35,149 | 78,319 | 76,648 | 159,836 | ||||||||||||
| Selling, general, and administrative | 65,423 | 53,379 | 139,380 | 111,495 | ||||||||||||
| Changes in fair value of contingent consideration payable | 337 | 115 | 588 | (1,073 | ) | |||||||||||
| Loss on impairment of assets | 1,134 | — | 1,134 | 6,616 | ||||||||||||
| Depreciation and amortization | 2,206 | 1,334 | 3,463 | 2,745 | ||||||||||||
| Total operating expenses | 104,249 | 133,147 | 221,213 | 279,619 | ||||||||||||
| Loss from operations | (18,860 | ) | (60,613 | ) | (56,496 | ) | (135,952 | ) | ||||||||
| Other (expense) income: | ||||||||||||||||
| Interest income | 1,737 | 356 | 3,936 | 489 | ||||||||||||
| Interest expense | (12,492 | ) | (8,257 | ) | (24,336 | ) | (16,404 | ) | ||||||||
| Other (expense) income | (10,902 | ) | 7,268 | (16,840 | ) | 9,170 | ||||||||||
| Loss before income tax | (40,517 | ) | (61,246 | ) | (93,736 | ) | (142,697 | ) | ||||||||
| Income tax expense | (2,715 | ) | (911 | ) | (2,428 | ) | (4,720 | ) | ||||||||
| Net loss attributable to common stockholders | $ | (43,232 | ) | $ | (62,157 | ) | $ | (96,164 | ) | $ | (147,417 | ) | ||||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.15 | ) | $ | (0.21 | ) | $ | (0.33 | ) | $ | (0.51 | ) | ||||
| Weighted-average common shares outstanding — basic and diluted | 292,797,002 | 291,970,562 | 292,071,201 | 288,646,587 | ||||||||||||
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(Unaudited)
(in thousands, except share and per share amounts)
June 30, 2023 | December 31, 2022 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 211,307 | $ | 148,813 | ||||
| Investments in marketable securities | 54,319 | 144,782 | ||||||
| Accounts receivable | 63,716 | 66,196 | ||||||
| Inventories | 51,381 | 23,816 | ||||||
| Prepaid expenses and other current assets | 52,099 | 40,209 | ||||||
| Total current assets | 432,822 | 423,816 | ||||||
| Operating lease right-of-use assets, net | 28,042 | 29,534 | ||||||
| Property and equipment, less accumulated depreciation of $24,060 and $22,281 at June 30, 2023 and December 31, 2022, respectively | 30,238 | 30,778 | ||||||
| Intangible asset, less accumulated depreciation of $855 and $0 at June 30, 2023 and December 31, 2022, respectively | 22,145 | 23,000 | ||||||
| Goodwill | 197,797 | 197,797 | ||||||
| Other non-current assets | 19,049 | 19,242 | ||||||
| Total Assets | $ | 730,093 | $ | 724,167 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 13,522 | $ | 15,413 | ||||
| Accrued expenses and other current liabilities | 124,868 | 93,636 | ||||||
| Contingent consideration payable | 13,005 | 21,417 | ||||||
| Operating lease liabilities | 7,840 | 8,552 | ||||||
| Total current liabilities | 159,235 | 139,018 | ||||||
| Long-term debt | 393,350 | 391,990 | ||||||
| Operating lease liabilities | 50,976 | 51,578 | ||||||
| Deferred reimbursements | 5,906 | 4,656 | ||||||
| Deferred income taxes | — | 4,939 | ||||||
| Other non-current liabilities | 9,045 | 8,939 | ||||||
| Total liabilities | 618,512 | 601,120 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Common stock, $0.01 par value, 500,000,000 shares authorized, 286,992,923 and 281,108,273 shares issued and outstanding at June 30, 2023 and December 31, 2022, respectively | 2,856 | 2,815 | ||||||
| Additional paid-in capital | 2,733,148 | 2,664,744 | ||||||
| Accumulated other comprehensive gain (loss): | ||||||||
| Foreign currency translation adjustment | 4,337 | (11,989 | ) | |||||
| Unrealized loss on available-for-sale securities | (177 | ) | (116 | ) | ||||
| Warrants | 71 | 83 | ||||||
| Accumulated deficit | (2,628,654 | ) | (2,532,490 | ) | ||||
| Total stockholders' equity | 111,581 | 123,047 | ||||||
| Total Liabilities and Stockholders' Equity | $ | 730,093 | $ | 724,167 | ||||
8
TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
| Three Months Ended June 30, | Six Months Ended June 30, | |||||||||||||||
| 2023 | 2022 | 2023 | 2022 | |||||||||||||
| Total operating expenses - as reported GAAP | $ | 104,249 | $ | 133,147 | $ | 221,213 | $ | 279,619 | ||||||||
| Research and development: | ||||||||||||||||
| Stock-based compensation | 4,117 | 4,379 | 12,607 | 13,744 | ||||||||||||
| Selling, general and administrative: | ||||||||||||||||
| Stock-based compensation | 12,460 | 8,084 | 38,864 | 29,370 | ||||||||||||
| Loss on impairment of assets | 1,134 | - | 1,134 | 6,616 | ||||||||||||
| Changes in fair value of contingent consideration payable | 337 | 115 | 588 | (1,073 | ) | |||||||||||
| Depreciation and amortization | 2,206 | 1,334 | 3,463 | 2,745 | ||||||||||||
| Total operating expense adjustments to reported GAAP | 20,254 | 13,912 | 56,656 | 51,402 | ||||||||||||
| Total operating expenses - as adjusted | $ | 83,995 | $ | 119,235 | $ | 164,557 | $ | 228,217 | ||||||||
9
| AT THE FOREFRONT OF THERAPIES FOR RARE DISEASES 2Q23 Results Conference Call & Webcast August 8, 2023 |
| 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 presentation may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, 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, MHRA, and PMDA, may not grant or may delay approval for our product candidates; the potential that required regulatory inspections may be delayed or not be successful and delay or prevent product approval; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or AT-GAA 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, 2022, and on Form 10-Q for the quarter ended June 30, 2023, to be filed today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof. Non-GAAP Financial Measures In addition to financial information prepared in accordance with U.S. GAAP, this presentation also contains adjusted financial measures that we believe provide investors and management with supplemental information relating to operating performance and trends that facilitate comparisons between periods and with respect to projected information. These adjusted financial measures are non-GAAP measures and should be considered in addition to, but not as a substitute for, the information prepared in accordance with U.S. GAAP. We typically exclude certain GAAP items that management does not believe affect our basic operations and that do not meet the GAAP definition of unusual or non-recurring items. Other companies may define these measures in different ways. When we provide our expectation for non-GAAP operating expenses on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results. |
| 3 A Rare Company Patient-dedicated, rare disease biotechnology company with sustained double-digit revenue growth, a global commercial infrastructure, and late-stage development capabilities GLOBAL COMMERCIAL ORGANIZATION EMPLOYEES in 20 Countries GALAFOLD & POMBILITI + OPFOLDA Gene Therapy Platform Leveraging Experience in Protein Engineering & Glycobiology 14-18% FY23 Galafold Revenue Growth at CER Non-GAAP PROFITABILITY expected in 2H 2023 Cumulative $1.5B- $2B Peak Potential AT-GAA Under Global Regulatory Reviews for Pompe Disease $266M Cash as of 6/30/23 World-class Clinical Development Capabilities |
| 2023 Strategic Priorities Sustain double-digit Galafold revenue growth of 14-18% at CER1 Secure FDA, EMA, and MHRA approvals for AT-GAA Initiate successful global launches of AT-GAA Advance best-in-class, next-generation Fabry and Pompe pipeline programs and capabilities Maintain strong financial position on path to profitability 4 1 2 3 4 5 1 CER: Constant Exchange Rates; 2023 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2022 |
| 5 Galafold® (migalastat) Continued Growth Building a leadership position in the treatment of Fabry disease |
| 6 2023 Galafold Success (as of June 30, 2023) Building on Galafold’s success and leveraging leadership position to drive continued growth Galafold is indicated for adults with a confirmed diagnosis of Fabry disease and an amenable variant. The most common adverse 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 first and only approved oral treatment option with a unique mechanism of action for Fabry patients with amenable variants 35-50% Fabry Patients Amenable to Galafold 40+ Countries with Regulatory Approvals 53 Orange Book Listed Patents $94.3M 2Q23 Galafold Revenue 14-18% 2023 Galafold Operational Growth at CER 60% Share of Treated Amenable Patients |
| 7 Galafold Performance Raising FY23 revenue growth guidance to 14% to 18% at CER Global mix of switch (~45%) and previously untreated patients (~55%)3 Compliance and adherence over 90%+ Expect non-linear quarterly growth to continue due to uneven ordering patterns and FX fluctuations FY16 FY17 FY18 FY19 FY20 FY21 FY22 FY23 $5M Q1 $79M Q2 $81M Q3 $82M Q4 $88M $37M $91M $182M $261M $306M 1H23 reported revenue growth of +13% to $180M with strong operational growth of +16% $329M1 Q1 $86M $375M - $388M2 Q2 $94M 1 FY22 reported revenue growth of +8% to $329M with strong operational growth of +16% at CER – FY22 negative currency impact YoY of ~$26M 2 At constant exchange rate (CER) 3 Data on file |
| 8 Galafold Global Commercial Momentum (as of June 30, 2023) Strong patient demand and performance against key metrics lay the foundation for continued double-digit growth in 2023 8 Continued penetration into existing markets Further uptake in diagnosed untreated population Continued geographic expansion and label extensions Maintaining compliance and adherence Driving reimbursement and access Sustained Growth in 2023 Driven by: |
| 9 Pombiliti® (cipaglucosidase alfa) + Opfolda® (miglustat) Potential to establish a new standard of care for people living with Pompe disease |
| 10 Deficiency of GAA leading to lysosomal glycogen accumulation and cellular dysfunction Diagnosed at different stages of life, from childhood to adulthood Symptoms include progressive muscle weakness, particularly skeletal and respiratory muscles, that worsens over time Respiratory failure is a major cause of mortality ~5,000-10,000 people diagnosed globally; Significant underdiagnosis ~$1.2B+ global Pompe ERT sales1 Majority of patients on current standard of care decline after ~2 years Late-Onset Pompe Disease (LOPD) Overview 1 Based on 12 months ended December 31, 2022. Source: Sanofi Press Release Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA) |
| 11 11 1Data on file Global Regulatory Status Expect regulatory approvals and launch into the three largest Pompe markets in 2023 U.K. MAA submitted via recognition procedure based on CHMP opinion MHRA approval expected 3Q 2023 Pombiliti® + Opfolda® now approved in the EU U.S. FDA approval expected 3Q 2023 11 |
| 12 Ongoing Clinical Studies and Expanded Access Mechanisms Ongoing clinical studies in children and adolescents1 with LOPD and infantile-onset Pompe disease (IOPD) Multiple expanded access mechanisms in place, including in the U.S., U.K., Germany, France, Japan, and others At time of first regulatory approval, ~200 people living with Pompe disease on AT-GAA across extension studies and expanded access programs ~75 centers worldwide currently participating in clinical trials and access programs Advancing science though ongoing clinical studies and providing expanded access through multiple mechanisms 1 Children and adolescents aged 0 to <18 years old |
| 13 Pombiliti + Opfolda EU Opportunity Strong indication statement: – Pombiliti® (cipaglucosidase alfa) is a long-term enzyme replacement therapy used in combination with the enzyme stabiliser Opfolda® (miglustat) for the treatment of adults with late-onset Pompe disease (acid α glucosidase [GAA] deficiency) >1,300 patients are estimated to be treated in Europe1 – ~60 Patients throughout EU currently on Pombiliti + Opfolda, including ~20 in Germany and Austria Launch underway in Germany – 6 month “free pricing” period and AMNOG reimbursement process – First patients dosed and additional patients scheduled to start infusions 1 Amicus data on file from market mapping EU Pompe market currently represents a sizeable market opportunity of $450M+ |
| 14 Launch of Pombiliti + Opfolda Underway in the EU Experienced and passionate rare disease commercial and medical organization supporting early days of launch Access and Reimbursement Positive Interactions with Payors Focus on broad patient access Country-by-country reimbursement process Active discussions to demonstrate value Patient Demand Initial focus on clinical trial and expanded access patients First patients dosed; Multiple scheduled for infusion On-track to transition all trial and expanded access patients in Germany within 90 days KOL and Patient Outreach Promotion and Education Efforts Existing relationships with HCPs at key treatment centers Engaging top prescribers within first 30 days Ongoing disease education Performance |
| 15 Corporate Outlook Delivering on our mission for patients and shareholders |
| 16 2Q 2023 Select Financial Results 2Q23 revenue of $94.5M and growth rate of 17% at CER (in thousands, except per share data) Jun. 30, 2023 Jun. 30, 2022 Product Revenue $94,503 $80,731 Cost of Goods Sold 9,114 8,197 R&D Expense 35,149 78,319 SG&A Expense 65,423 53,379 Changes in Fair Value of Contingent Consideration 337 115 Loss on Impairment of Assets 1,134 — Depreciation and Amortization 2,206 1,334 Loss from Operations (18,860) (60,613) Income Tax Expense (2,715) (911) Net Loss (43,232) (62,157) Net Loss Per Share (0.15) (0.21) |
| Financial Outlook and Path to Profitability Clear strategy to build our business, advance our portfolio, and achieve profitability 17 Sustain Revenue Growth Deliver on Financial Goals Secure Approvals of AT-GAA $180.8M 1H 2023 revenue, +16% YoY operational growth 2023 Galafold revenue growth guidance of +14-18% YoY at CER Galafold and AT-GAA expected to drive strong double-digit growth long term Focused on prudent expense management Achieve profitability1 in 2H 2023 1 Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. We define non-GAAP Net Income as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on impairment of assets, restructuring charges, and income taxes. 2023 non-GAAP operating expense guidance of $330M-$350M |
| 18 Positioned for Significant Value Growth Focused on execution and driving sustainable double-digit revenue growth on path to profitability Continue to bring Galafold® to as many patients as possible, sustain double-digit operational revenue growth Successful launch of AT-GAA for people living with Pompe disease Advance next-generation gene therapies in Fabry and Pompe diseases Fully leverage global capabilities and infrastructure as a leader in rare diseases Achieve non-GAAP profitability in 2H 20231 1 Based on projections of Amicus non-GAAP Net Income under current operating plans, which includes successful AT-GAA regulatory approvals and continued Galafold growth. Non-GAAP Net Income defined as GAAP Net Income excluding the impact of share-based compensation expense, changes in fair value of contingent consideration, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, restructuring charges and income taxes. |
| Appendix |
| 20 Appendix |
| 21 2022 Environmental, Social, & Governance (ESG) Snapshot Board of Directors Committed to ongoing Board refreshment and diversity of background, gender, skills, and experience: 80% Board Independence 60% Overall Board Diversity Address a rare genetic disease First-in-class or best-in-class Impart meaningful benefit for patients 484 Global Employees 57% % Female Employees Who We Serve Our mission is to drive sustainability with our partners by incorporating environmental and sustainability principles into all our commercial relationships Pledge for a Cure Designate a portion of product revenue back into R&D for that specific disease until there is a cure. Programs we invest in have 3 key characteristics 3 Female 2 Veteran Status 1 African American Director Diversity % Hiring Slate Diversity 97% Leverage employee capabilities and expertise to provide a culture that drives performance and ultimately attracts, energizes, and retains critical talent. Employee Recruitment, Engagement, & Retention Pulse surveys reveal employees feel high personal satisfaction in their job, are proud of their work and what they contribute to the community Career Development Reimagined performance management process to measure the what and the how, rewarding those who role-model our Mission-Focused Behaviors. Committed to producing transformative medicines for patients while practicing environmental responsibility and adhering to sustainability best practices in our operations. Environmental Management 0% Amicus Owned Direct Manufacturing and Related GHG Emissions Diversity, Equity, & Inclusion (DEI) Pledge to support a more inclusive culture to impact our employees, our communities, and society. Goal of maintaining gender diversity and increasing overall diversity throughout our global workforce. 580 Volunteer hours (U.S.): 22 Amicus supported community programs: 79 patients /19countries Expanded Access through Feb 2023: Pricing PROMISE Contributions allocated: $2,288,998 U.S. $954,349 Intl. Charitable Giving Committed to never raising the annual price of our products more than consumer inflation. |
| 22 FX Sensitivity and Galafold Distribution of Quarterly Sales Impact from Foreign Currency Q2 2023 Currency Variances: USD/ Q2 2022 Q2 2023 YoY Variance EUR 1.066 1.089 2.2% GBP 1.257 1.251 (0.5%) JPY 0.008 0.007 (5.6%) Full Year 2023 Revenue Sensitivity Given the high proportion of Amicus revenue is Ex-US, a change in exchange rates of +/- 5% compared to year end 2022 rates could lead to a $11M-$12M change in global reported revenues in 2023. Distribution of Galafold Revenue by Quarter in Past 5 years: Q1 Q2 Q3 Q4 5 Year Avg. 22% 24% 26% 28% |
| 23 Amicus Pipeline Streamlined rare disease pipeline with focus on Fabry disease and Pompe disease franchises INDICATION DISCOVERY PRECLINICAL PHASE 1/2 PHASE 3 REGULATORY COMMERCIAL FABRY FRANCHISE Galafold® (migalastat) Fabry Gene Therapy Next-Generation Chaperone POMPE FRANCHISE PombilitiTM + OpfoldaTM* (cipaglucosidase alfa) + (miglustat) Pompe Gene Therapy OTHER CLN3 Batten Disease Gene Therapy Next-Generation Research Programs ODD BTD ODD ODD - Orphan Drug Designation BTD - Breakthrough Therapy Designation *Approved only in the European Union |
| Thank you |
