UNITED STATES
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WASHINGTON, D.C. 20549
FORM
CURRENT REPORT PURSUANT TO
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Item 2.02 – Results of Operations and Financial Condition
On March 1, 2023, Amicus Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the fiscal year ended December 31, 2022. A copy of this press release is attached hereto as Exhibit 99.1. The Company will host a conference call and webcast on March 1, 2023 to discuss its full year results of operations. A copy of the conference call presentation materials is attached hereto as Exhibit 99.2. Both exhibits are incorporated herein by reference.
In accordance with General Instruction B.2. of Form 8-K, the information in this Current Report on Form 8-K and the Exhibits shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits
(d) Exhibits:
| Exhibit No. | Description |
| 99.1 | Press Release, dated March 1, 2023 |
| 99.2 | March 1, 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: March 1, 2023 | By: | /s/ Ellen S. Rosenberg |
| Name: Ellen S. Rosenberg | ||
| Title: Chief Legal Officer and Corporate Secretary | ||
Exhibit 99.1
Amicus Therapeutics Announces Full-Year 2022 Financial Results and Corporate Updates
2022 Revenue Growth of 16% at CER to $329M
Projecting Galafold Revenue Growth in 2023 of 12-17% at CER
U.S. FDA Pre-approval Inspection for AT-GAA Now Scheduled; Approval expected in 3Q 2023
EU and U.K. AT-GAA Regulatory Reviews On-Track; Approvals expected in 3Q 2023
Non-GAAP Profitability Anticipated in 2H 2023
Conference Call and Webcast Today at 8:30 a.m. ET
PHILADELPHIA, PA, Mar. 1, 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 full-year ended December 31, 2022.
Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “In 2022, we made significant progress across the business heading into what we believe will be a pivotal year for Amicus. We delivered strong growth in Galafold, highlighted by the over 2,000 people around the world who are now on treatment. We set a strong foundation for the anticipated global launch of our second product. And finally, we streamlined our pipeline while continuing our judicious management of resources. In 2023, we remain laser focused on continuing to grow Galafold globally at double-digit rates, preparing for the expected approvals and launches of AT-GAA for Pompe disease in multiple major markets, and maintaining our financial discipline in order to achieve non-GAAP profitability in the second half of this year. These strategic priorities align with our mission to deliver innovative treatments that impart a meaningful difference in the lives of people living with rare diseases.”
Corporate Highlights:
| · | Global revenue in the full-year 2022 was $329.2 million. Full-year revenue represented a year-over-year increase of 8% from total revenue of $305.5 million in the full-year of 2021. Full-year operational revenue growth measured at constant exchange rates (CER)1 was 16%. |
| Three Months Ended December 31, |
Year over Year % Growth |
Twelve Months Ended December 31, |
Year over Year % Growth |
|||||||||||||||||||||||||||||
| (in thousands) | 2022 | 2021 | As Reported | at CER1 | 2022 | 2021 | As Reported | at CER1 | ||||||||||||||||||||||||
| Net Product Revenues | $ | 88,096 | $ | 82,154 | 7 | % | 16 | % | $ | 329,233 | $ | 305,514 | 8 | % | 16 | % | ||||||||||||||||
| · | For the full-year 2023, the Company anticipates double-digit Galafold revenue growth of 12-17% at CER1. 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. |
| · | The U.S. Food and Drug Administration (FDA) pre-approval inspection for AT-GAA is scheduled. Regulatory approval in the U.S. is expected during the third quarter of 2023, pending a successful inspection. |
| · | The EU and U.K. AT-GAA regulatory reviews remain on-track with approvals expected in 3Q 2023. The Committee for Medicinal Products for Human Use (CHMP) previously adopted a positive opinion of Pombiliti™, also known as cipaglucosidase alfa. A CHMP opinion for miglustat, the enzyme stabilizer component of AT-GAA is expected in the second quarter 2023. The regulatory submission process for AT-GAA in the U.K. was initiated in December 2022, with final approval expected in the third quarter of 2023. |
| · | Expanded access programs continue to meet the growing demand for AT-GAA across multiple countries. In the U.K., under the Early Access to Medicines Scheme (EAMS), multiple physicians have requested access from each of the leading Pompe centers in the country. Many patients with Pompe disease are participating in additional expanded access programs in the U.S., Germany, France, and Japan. |
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| · | Two oral presentations and 11 posters highlighting Amicus’ development programs in Fabry disease and Pompe disease presented at the 19th Annual WORLDSymposium™ 2023. Updated long-term efficacy and safety data from the global Phase 3 open-label extension study of AT-GAA in late-onset Pompe disease (LOPD) demonstrated consistency and durability of effect in patients out to two years, suggesting long-term benefit of treatment for people living with LOPD. Initial results from the FollowME Fabry Pathfinders registry was presented showing stable renal function out to 3-years for patients on Galafold. |
| · | Galafold U.S. intellectual property estate strengthened following the issuance of 19 new patents in 2022. Galafold is protected by orphan drug regulatory exclusivities and a broad U.S. intellectual property portfolio of 46 orange book-listed patents, including 5 composition of matter patents, 30 of which provide protection through at least 2038. |
| · | Full-year 2023 non-GAAP operating expense guidance of $340 million to $360 million, driven by prudent expense management while maintaining AT-GAA manufacturing and pre-launch activities. |
| · | Based on the current operating plan, the timing of AT-GAA approvals, and through careful management of expenses, the Company is on-track to achieve non-GAAP profitability2 in the second half of 2023. |
Full-Year 2022 Financial Results
| · | Total revenue in the full-year 2022 was $329.2 million, a year-over-year increase of 8% from total revenue of $305.5 million in the full-year 2021. On a constant currency basis, full-year 2022 total revenue growth was 16%. Reported revenue was offset by a negative currency impact of $26.1 million, or 8%. |
| · | Cash, cash equivalents, and marketable securities totaled $293.6 million at December 31, 2022, compared to $482.5 million at December 31, 2021. |
| · | Total GAAP operating expenses of $502.8 million for the full-year 2022 increased as compared to $477.5 million for the full-year 2021. |
| · | Total non-GAAP operating expenses of $413.2 million for the full-year 2022 increased slightly as compared to $406.9 million for the full-year 2021, reflecting decreased program spend offset by non-recurring expenses related to the reprioritization of the gene therapy portfolio.3 |
| · | Net loss was $236.6 million, or $0.82 per share, for the full-year 2022, and was reduced compared to a net loss of $250.5 million, or $0.92 per share, for the full-year 2021. |
2023 Financial Guidance
| · | For the full-year 2023, the Company anticipates total Galafold revenue growth between 12 and 17% at CER1 driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, 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. |
| · | Non-GAAP operating expense guidance for the full-year 2023 is $340 million to $360 million, driven by prudent expense management offset by continued investment in the global Galafold launch, AT-GAA clinical studies and pre-launch activities, in addition to certain non-recurring costs for manufacturing to support the global launch of AT-GAA4. |
| · | 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 (12-17% at CER1) |
| · | Secure FDA, EMA, and MHRA approvals for AT-GAA |
| · | Initiate successful global launches of AT-GAA |
| · | 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 2022 Galafold revenue guidance utilizes the average actual exchange rates for 2021.
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, March 1, 2023 at 8:30 a.m. ET to discuss the full-year 2022 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 number 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 Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
| · | Galafold is not intended for concomitant use with enzyme replacement therapy. |
| · | Galafold is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m2). The safety and efficacy of Galafold in children less than 12 years of age have not yet been established. No data are available. |
| · | No dosage adjustments are required in patients with hepatic impairment or in the elderly population. |
| · | There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse. |
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| · | While taking Galafold, effective birth control should be used. It is not known whether Galafold is excreted in human milk. |
| · | Contraindications to Galafold include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION. |
| · | Galafold 123 mg capsules are not for children (≥12 years) weighing less than 45 kg. |
| · | It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on Galafold or switched to Galafold. |
| · | OVERDOSE: General medical care is recommended in the case of Galafold overdose. |
| · | The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received Galafold. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS. |
| · | Call your doctor for medical advice about side effects. |
For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.
About Fabry Disease
Fabry disease is an inherited lysosomal disorder caused by deficiency of an enzyme called alpha-galactosidase A (alpha-Gal A), which results from mutations in the GLA gene. The primary biological function of alpha-Gal A is to degrade specific lipids in lysosomes, including globotriaosylceramide (referred to here as GL-3 and also known as Gb3). Lipids that can be degraded by the action of alpha-Gal A are called "substrates" of the enzyme. Reduced or absent levels of alpha-Gal A activity lead to the accumulation of GL-3 in the affected tissues, including heart, kidneys, and skin. Accumulation of GL-3 and progressive deterioration of organ function is believed to lead to the morbidity and mortality of Fabry disease. The symptoms can be severe, differ from person to person, and begin at an early age.
About Pompe Disease
Pompe disease is an inherited lysosomal disorder caused by deficiency of the enzyme acid alpha-glucosidase (GAA). Reduced or absent levels of GAA lead to accumulation of glycogen in cells, which is believed to result in the clinical manifestations of Pompe disease. Pompe disease ranges from a rapidly fatal infantile form with significant impacts to heart function, to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement. Late-onset Pompe disease can be severe and debilitating, including progressive muscle weakness throughout the body, particularly the skeletal muscles and muscles controlling breathing, that worsens over time.
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, 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 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
(in thousands, except share and per share amounts)
| Years Ended December 31, | ||||||||||||
| 2022 | 2021 | 2020 | ||||||||||
| Net product sales | $ | 329,233 | $ | 305,514 | $ | 260,886 | ||||||
| Cost of goods sold | 38,599 | 34,466 | 31,044 | |||||||||
| Gross profit | 290,634 | 271,048 | 229,842 | |||||||||
| Operating expenses: | ||||||||||||
| Research and development | 276,677 | 272,049 | 308,443 | |||||||||
| Selling, general, and administrative | 213,041 | 192,710 | 156,407 | |||||||||
| Changes in fair value of contingent consideration payable | 1,078 | 6,514 | 3,144 | |||||||||
| Loss on impairment of assets | 6,616 | — | — | |||||||||
| Depreciation and amortization | 5,342 | 6,209 | 8,846 | |||||||||
| Total operating expenses | 502,754 | 477,482 | 476,840 | |||||||||
| Loss from operations | (212,120 | ) | (206,434 | ) | (246,998 | ) | ||||||
| Other income (expense): | ||||||||||||
| Interest income | 3,024 | 509 | 3,226 | |||||||||
| Interest expense | (37,119 | ) | (32,471 | ) | (22,425 | ) | ||||||
| Loss on extinguishment of debt | — | (257 | ) | (7,276 | ) | |||||||
| Other income (expense) | 4,176 | (2,901 | ) | (781 | ) | |||||||
| Loss before income tax | (242,039 | ) | (241,554 | ) | (274,254 | ) | ||||||
| Income tax benefit (expense) | 5,471 | (8,906 | ) | (2,598 | ) | |||||||
| Net loss attributable to common stockholders | $ | (236,568 | ) | $ | (250,460 | ) | $ | (276,852 | ) | |||
| Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.82 | ) | $ | (0.92 | ) | $ | (1.07 | ) | |||
| Weighted-average common shares outstanding — basic and diluted | 289,057,198 | 271,421,986 | 258,867,380 | |||||||||
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TABLE 2
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
| December 31, | ||||||||
| 2022 | 2021 | |||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 148,813 | $ | 245,197 | ||||
| Investments in marketable securities | 144,782 | 237,299 | ||||||
| Accounts receivable | 66,196 | 52,672 | ||||||
| Inventories | 23,816 | 26,818 | ||||||
| Prepaid expenses and other current assets | 40,209 | 34,848 | ||||||
| Total current assets | 423,816 | 596,834 | ||||||
| Operating lease right-of-use assets, net | 29,534 | 20,586 | ||||||
| Property and equipment, less accumulated depreciation of $22,281 and $19,882 at December 31, 2022 and 2021, respectively | 30,778 | 42,496 | ||||||
| In-process research & development | 23,000 | 23,000 | ||||||
| Goodwill | 197,797 | 197,797 | ||||||
| Other non-current assets | 19,242 | 24,427 | ||||||
| Total Assets | $ | 724,167 | $ | 905,140 | ||||
| Liabilities and Stockholders' Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 15,413 | $ | 21,513 | ||||
| Accrued expenses and other current liabilities | 93,636 | 98,153 | ||||||
| Contingent consideration payable | 21,417 | 18,900 | ||||||
| Operating lease liabilities | 8,552 | 7,409 | ||||||
| Total current liabilities | 139,018 | 145,975 | ||||||
| Long-term debt | 391,990 | 389,357 | ||||||
| Operating lease liabilities | 51,578 | 43,363 | ||||||
| Deferred income taxes | 4,939 | 4,930 | ||||||
| Deferred reimbursements | 4,656 | 5,906 | ||||||
| Other non-current liabilities | 8,939 | 8,240 | ||||||
| Total liabilities | 601,120 | 597,771 | ||||||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Common stock, $0.01 par value, 500,000,000 shares authorized, 281,108,273 and 278,912,800 shares issued and outstanding at December 31, 2022 and 2021, respectively | 2,815 | 2,808 | ||||||
| Additional paid-in capital | 2,664,744 | 2,595,419 | ||||||
| Accumulated other comprehensive (loss) gain: | ||||||||
| Foreign currency translation adjustment | (11,989 | ) | 5,251 | |||||
| Unrealized loss on available-for-sale securities | (116 | ) | (270 | ) | ||||
| Warrants | 83 | 83 | ||||||
| Accumulated deficit | (2,532,490 | ) | (2,295,922 | ) | ||||
| Total stockholders' equity | 123,047 | 307,369 | ||||||
| Total Liabilities and Stockholders' Equity | $ | 724,167 | $ | 905,140 | ||||
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TABLE 3
Amicus Therapeutics, Inc.
Reconciliation of Non-GAAP Financial Measures
(in thousands)
| December 31 | ||||||||||||
| 2022 | 2021 | 2020 | ||||||||||
| Total operating expenses - as reported GAAP | $ | 502,754 | $ | 477,482 | $ | 476,840 | ||||||
| Research and development: | ||||||||||||
| Share-based compensation | 25,089 | 17,340 | 20,817 | |||||||||
| Selling, general and administrative: | ||||||||||||
| Share-based compensation | 51,423 | 40,498 | 28,334 | |||||||||
| Loss on impairment of assets | 6,616 | — | — | |||||||||
| Changes in fair value of contingent consideration payable | 1,078 | 6,514 | 3,144 | |||||||||
| Depreciation and amortization | 5,342 | 6,209 | 8,846 | |||||||||
| Total operating expense adjustments to reported GAAP | 89,548 | 70,561 | 61,141 | |||||||||
| Total operating expenses - as adjusted | $ | 413,206 | $ | 406,921 | $ | 415,699 | ||||||
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| A T T H E F O R E F R O N T O F T H E R A P I E S F O R R A R E D I S E A S E S FY22 Financial Results Conference Call & Webcast March 1, 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 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, 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 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 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. |
| 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 & AT-GAA Gene Therapy Platform Leveraging Experience in Protein Engineering & Glycobiology 12-17% 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 $294M Cash as of 12/31/22 World-class Clinical Development Capabilities |
| 2023 Strategic Priorities Sustain double-digit Galafold revenue growth of 12-17% 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 1CER: 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 2022 Galafold Success (as of December 31, 2022) 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 350 Amenable Variants in U.S. Label 40+ Countries with Regulatory Approvals 46 Orange Book Listed Patents $329M 2022 Galafold Revenue 16% 2022 Galafold Operational Growth 1,386 Amenable Mutations in EU Label |
| 7 Galafold Performance 2022 reported revenue growth of +8% to $329M – Strong operational growth of +16% at CER ▪ Global 3-month net new patients trend highest in 2 years ▪ ~50% share of treated amenable patients ▪ Interactions with HCPs increasing from same period last year ▪ Healthy global mix of switch (~55%) and previously untreated patients (~45%)1 ▪ Compliance and adherence over 90%+ $306M $329M -8% +16% 2021 Operational Growth FX Impact 2022 Year-over-Year Sales Growth 1Data on file FY22 Strength Reflects Increasing Demand with >2,000 Individuals Treated |
| 8 Galafold Quarterly Trends Growth remains strong with Q4 revenue of $88M and FY22 revenue of $329M $79M $82M $79M $81M $82M $88M 3Q21 4Q21 1Q22 2Q22 3Q22 4Q22 Quarterly Galafold Sales ▪ Expect non-linear quarterly growth to continue due to uneven ordering patterns and FX fluctuations Distribution of Galafold Revenue by Quarter in Past 5 years: Q1 Q2 Q3 Q4 5 Year Avg. 22% 24% 26% 28% |
| 9 Galafold Global Launch Momentum (as of December 31, 2022) Strong patient demand with 2,000+ individuals treated with Galafold and performance against key metrics lay the foundation for continued double-digit growth in 2023 9 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: |
| 10 AT-GAA Launch Preparations Experienced and passionate rare disease medical and commercial organization poised for second successful launch Key Strengths Commitment to patient access Clear focus on launch Identification of key Pompe disease treatment centers Development of educational materials Planning Access Scientific Exchange Team Great experience and passion Eagerness to introduce a new therapy upon approvals Highly leverageable team in place, few new hires needed Published Phase 3 PROPEL data in The Lancet Neurology Active medical conference and publication schedule Multiple expanded access programs in place Continued education on biology of disease and diagnosis Demonstrating value to payors including parity pricing strategy |
| 11 AT-GAA (cipaglucosidase alfa + miglustat) Potential to establish a new standard of care for people living with Pompe disease |
| 12 Deficiency of GAA leading to lysosomal glycogen accumulation and cellular dysfunction Age of onset ranges from infancy to adulthood Symptoms include muscle weakness, respiratory failure, and cardiomyopathy Respiratory and cardiac failure are leading causes of morbidity and mortality Estimated incidence of ~1:28,000; Significant underdiagnosis ~$1.2B+ global Pompe ERT sales1 Majority of patients on current standard of care decline after ~2 years Pompe Disease Overview 1Based on 12 months ended December 31, 2022. Source: Sanofi Press Release Pompe is a severe and fatal neuromuscular disease caused by the deficiency of lysosomal enzyme GAA |
| 13 13 1Data on file AT-GAA: Global Regulatory Status Anticipate regulatory approvals and launch into the three largest Pompe markets in 2023 ▪ Pombiliti™ (cipaglucosidase alfa) European Commission (EC) decision expected in 1Q 2023 ▪ Miglustat CHMP opinion expected in 2Q 2023 with EC decision anticipated in 3Q 2023 ▪ Pre-approval inspection now scheduled ▪ Anticipating 3Q 2023 FDA approval ▪ U.K. MAA submitted via recognition procedure based on CHMP opinion ▪ Anticipating 3Q 2023 MHRA approval 13 |
| 14 AT-GAA: Ongoing Clinical Studies and Expanded Access Mechanisms ▪ Ongoing clinical studies in children and adolescents1 with LOPD as well as in Infantile-Onset Pompe Disease (IOPD) ▪ Multiple expanded access mechanisms in place, including in the U.S., U.K., Germany, France, Japan, and others ▪ ~200 people living with Pompe disease are now 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 |
| 15 WORLDSymposium Update Number of patients (n) Mean (SD) baseline 6MWD (% predicted): Cipa/mig–cipa/mig = 56.1 (16.03) Alg/pla–cipa/mig = 54.8 (17.68) Mean (SD) week 104 6MWD (% predicted): Cipa/mig–cipa/mig = 60.8 (18.07) Alg/pla–cipa/mig = 54.3 (21.86) 6 Cipa/mig–cipa/mig Cipa/mig Alg/pla Baseline Week 12 26 38 52 64 78 104 –1 –2 0 1 2 3 4 5 Cipa/mig–cipa/mig 62 61 54 58 61 55 56 56 Alg/pla–cipa/mig 29 29 27 28 29 27 26 26 Mean (+/– SE) of change from ATB200-03 baseline (% predicted 6MWD) Mean (SD) baseline FVC (% predicted): Cipa/mig–cipa/mig = 67.7 (19.48) Alg/pla–cipa/mig = 67.2 (21.29) Mean (SD) week 104 FVC (% predicted): Cipa/mig–cipa/mig = 66.2 (20.00) Alg/pla–cipa/mig = 61.0 (18.20) –7 –5 –3 –1 0 1 2 3 Cipa/mig–cipa/mig Cipa/mig Alg/pla –4 –2 –6 Number of patients (n) Week 12 26 38 52 64 78 104 Cipa/mig–cipa/mig 62 61 53 54 55 50 51 53 Alg/pla–cipa/mig 29 29 26 27 26 25 26 24 Baseline Mean (+/– SE) of change from ATB200-03 baseline (sitting % predicted FVC Ph 3 open-label extension study data demonstrate that treatment with AT-GAA up to 2 years was associated with a durable effect, supporting the long-term benefits ▪ ERT-experienced and -naïve patients treated with AT-GAA throughout PROPEL showed durable improvements in % predicted 6MWD that were maintained throughout to week 104 ▪ ERT-experienced and -naïve patients who received alglucosidase alfa/placebo in PROPEL and switched to AT-GAA in the OLE showed stability in % predicted 6MWD throughout the OLE study ▪ ERT-experienced patients treated with AT-GAA throughout PROPEL remained stable, while patients who received alglucosidase alfa/placebo experienced a decline in sitting % predicted FVC that stabilized after switching to AT-GAA in the OLE study ERT-Experienced 6MWD (%): Change from baseline ERT-Experienced FVC (%): Change from baseline |
| 16 Corporate Outlook Delivering on our mission for patients and shareholders |
| 17 2022 Select Financial Results 2022 revenue of $329M and growth rate of 16% at CER from global Galafold sales (in thousands, except per share data) Dec. 31, 2022 Dec. 31, 2021 Product Revenue $329,233 $305,514 Cost of Goods Sold 38,599 34,466 R&D Expense 276,677 272,049 SG&A Expense 213,041 192,710 Changes in Fair Value of Contingent Consideration 1,078 6,514 Loss on Impairment of Assets 6,616 — Depreciation and Amortization 5,342 6,209 Loss from Operations (212,120) (206,434) Income Tax Benefit (Expense) 5,471 (8,906) Net Loss (236,568) (250,460) Net Loss Per Share (0.82) (0.92) |
| Financial Outlook and Path to Profitability Clear strategy to build our business, advance our portfolio, and achieve profitability 18 Sustain Galafold Revenue Growth Deliver on Financial Goals Secure Approvals of AT-GAA $329M FY22 revenue, +16% YoY operational growth 2023 Galafold revenue growth guidance of +12-17% 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 1Based 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 $340M-$360M |
| 19 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 |
| 21 Appendix |
| 22 Environmental, Social, & Governance (ESG) Snapshot Board of Directors Eco-friendly decision-making has unearthed economic efficiencies while continuing to bolster our standing as a good corporate citizen. Committed to ongoing Board refreshment and diversity of background, gender, skills, and experience: Contributions allocated: $1,677,000 U.S. $832,976 Intl. 770 Volunteer hours (U.S.): 20+ Amicus supported community programs: 80% Board Independence 60% Overall Board Diversity Address a rare genetic disease First-in-class or best-in-class Impart meaningful benefit for patients 496 Global Employees 58% % female employees Whom We Serve Environmental Management Green building design Energy & water conservation Hazardous waste management Pledge for a Cure Designate a portion of product revenue back into R&D for that specific disease until there is a cure. Pricing PROMISE Programs we invest in have 3 key characteristics Committed to never raising the annual price of our products more than consumer inflation. Diversity, Equity, & Inclusion (DEI) 2023 and Beyond: • Maintain strength in global gender diversity • Increase US diversity through intentional and ongoing action • Continuously evaluate compensation practices to ensure pay parity Pledge to support a more inclusive culture to impact our employees, our communities, and society. 3 Female 2 Veteran Status 1 African American Director Diversity % Hiring Slate Diversity 82% 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 Charitable Giving (as of 12/31/21) 74 patients /20countries Expanded Access through Jan 2023: Career Development Reimagined performance management process to measure the what and the how, rewarding those who role-model our Mission-Focused Behaviors. |
| 23 FX Sensitivity and Galafold Distribution of Quarterly Sales Impact from Foreign Currency Q4 2022 Currency Variances: USD/ Q4 2021 Q4 2022 Variance EUR 1.144 1.021 (10.7%) GBP 1.348 1.174 (12.9%) JPY 0.009 0.007 (19.5%) Full Year 2023 Revenue Sensitivity Given the high proportion of Amicus revenue 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% |
| 24 I N D I C A T I O N D I S C O V E R Y P R E C L I N I C A L P H A S E 1 / 2 P H A S E 3 R E G U L A T O R Y C O M M E R C I A L F A B R Y F R A N C H I S E Galafold®(migalastat) Fabry Gene Therapy Next-Generation Chaperone P O M P E F R A N C H I S E AT-GAA (cipaglucosidase alfa + miglustat) Pompe Gene Therapy O T H E R CLN3 Batten Disease Gene Therapy Next-Generation Research Programs Amicus Pipeline Streamlined rare disease pipeline with focus on Fabry disease and Pompe disease franchises ODD BTD ODD ODD - Orphan Drug Designation BTD - Breakthrough Therapy Designation |
| 25 AT-GAA Phase 3 PROPEL Study Results Clinically meaningful outcomes from Phase 3 PROPEL study provide the basis for global regulatory submissions of AT-GAA |
| 26 WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD Improvement from the PROPEL baseline in % predicted 6MWD for the cipa/mig group was maintained throughout the OLE for ERT-experienced and ERT-naïve patients • ERT-experienced and -naïve patients treated with cipa/mig throughout showed durable improvements in % predicted 6MWD in PROPEL that were maintained throughout the OLE to week 104 • ERT-experienced and -naïve patients who received alg/pla in PROPEL and switched to cipa/mig in the OLE showed stability in % predicted 6MWD throughout the OLE *Excludes one outlier. SE, standard error. Number of patients (n) Mean (SD) baseline 6MWD (% predicted): Cipa/mig–cipa/mig = 56.1 (16.03) Alg/pla–cipa/mig = 54.8 (17.68) Mean (SD) week 104 6MWD (% predicted): Cipa/mig–cipa/mig = 60.8 (18.07) Alg/pla–cipa/mig = 54.3 (21.86) 6 Cipa/mig–cipa/mig Cipa/mig Alg/pla Baseline Week 12 26 38 52 64 78 104 Number of patients (n) Baseline Week 12 26 38 52 64 78 104 Mean (SD) baseline 6MWD (% predicted): Cipa/mig–cipa/mig = 61.9 (15.33) Alg/pla–cipa/mig = 61.4 (17.11) Mean (SD) week 104 6MWD (% predicted): Cipa/mig–cipa/mig = 70.9 (16.31) Alg/pla–cipa/mig = 70.3 (8.57) –2 –4 0 2 4 6 8 12 10 14 16 –1 –2 0 1 2 3 4 5 Cipa/mig–cipa/mig Cipa/mig Alg/pla Cipa/mig–cipa/mig 62 61 54 58 61 55 56 56 29 27 26 26 Cipa/mig–cipa/mig 20 19 17 20 20 18 19 18 Alg/pla–cipa/mig 29 29 27 28 Alg/pla–cipa/mig 7 7 6 7 7 5 6 7 Mean (+/ – SE) of change from ATB200-03 baseline (% predicted 6MWD) Mean (+/ – SE) of change from ATB200-03 baseline (% predicted 6MWD) ERT experienced ERT naïve* |
| 27 WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD Sitting % predicted FVC remained stable in ERT-experienced and ERT-naïve patients throughout the OLE for both PROPEL treatment groups • ERT-experienced patients treated with cipa/mig throughout remained stable, while patients who received alg/pla in PROPEL experienced a decline in sitting % predicted FVC that stabilized after switching to cipa/mig in the OLE • ERT-naïve patients in both treatment groups experienced some decline in PROPEL that stabilized in the OLE with no further decline in FVC to week 104 *Excludes one outlier. Mean (SD) baseline FVC (% predicted): Cipa/mig–cipa/mig = 67.7 (19.48) Alg/pla–cipa/mig = 67.2 (21.29) Mean (SD) week 104 FVC (% predicted): Cipa/mig–cipa/mig = 66.2 (20.00) Alg/pla–cipa/mig = 61.0 (18.20) –7 –5 –3 –1 0 1 2 3 Cipa/mig–cipa/mig Cipa/mig Alg/pla –4 –2 –6 Mean (SD) baseline FVC (% predicted): Cipa/mig–cipa/mig = 80.2 (18.69) Alg/pla–cipa/mig = 79.1 (22.58) Mean (SD) week 104 FVC (% predicted): Cipa/mig–cipa/mig = 73.5 (21.24) Alg/pla–cipa/mig = 79.6 (17.92) –7 –10 –4 0 –6 –9 –2 1 –5 –8 –1 2 3 Cipa/mig–cipa/mig Cipa/mig Alg/pla 104 –3 Number of patients (n) Week 12 26 38 52 64 78 104 Number of patients (n) Week 12 26 38 52 64 78 Cipa/mig–cipa/mig 62 61 53 54 55 50 51 53 26 25 26 24 Cipa/mig–cipa/mig 20 18 18 17 19 18 18 17 Alg/pla–cipa/mig 29 29 26 27 Alg/pla–cipa/mig 7 7 5 5 6 4 4 5 Baseline Baseline ERT experienced ERT naïve* Mean (+/ – SE) of change from ATB200-03 baseline (sitting % predicted FVC Mean (+/ – SE) of change from ATB200-03 baseline (sitting % predicted FVC |
| 28 WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD Cipa/mig treatment was associated with a durable reduction in serum CK during PROPEL and the OLE in both ERT-experienced and ERT-naïve patients • ERT-experienced and -naïve patients treated with cipa/mig throughout showed a decline in serum CK levels during PROPEL that was maintained throughout the OLE • ERT-experienced and -naïve patients who received alg/pla in PROPEL showed a slight increase or stability in serum CK levels to week 52, and a marked decline after switching to cipa/mig in the OLE Number of patients (n) Mean (SD) baseline CK (U/L): Cipa/mig–cipa/mig = 433.0 (407.69) Alg/pla–cipa/mig = 490.6 (450.46) Mean (SD) week 104 CK (U/L): Cipa/mig–cipa/mig = 299.7 (296.92) Alg/pla–cipa/mig = 361.6 (292.67) –300 –250 –200 –150 –100 –50 0 50 100 Cipa/mig–cipa/mig Cipa/mig Alg/pla 2 4 6 12 26 38 52 54 56 58 64 78 104 Number of patients (n) Mean (SD) baseline CK (U/L): Cipa/mig–cipa/mig = 464.1 (398.11) Alg/pla–cipa/mig = 680.3 (333.13) Mean (SD) week 104 CK (U/L): Cipa/mig–cipa/mig = 283.6 (212.49) Alg/pla–cipa/mig = 461.7 (196.76) –400 –500 –300 –200 –100 200 Cipa/mig–cipa/mig Cipa/mig Alg/pla 2 4 6 12 26 38 52 54 56 58 64 78 104 100 0 Cipa/mig–cipa/mig 62 62 62 58 61 58 57 59 54 52 53 59 57 49 Cipa/mig–cipa/mig 20 20 19 20 19 19 20 18 19 19 16 18 19 18 Alg/pla–cipa/mig 29 29 28 29 29 29 28 28 28 26 27 27 26 25 Alg/pla–cipa/mig 7 7 7 7 6 7 7 7 7 5 7 7 7 7 Mean (+/ – SE) of change from ATB200-03 baseline (U/L) Mean (+/ – SE) of change from ATB200-03 baseline (U/L) ERT experienced ERT naïve* *Excludes one outlier. Baseline Baseline Week Week |
| 29 • ERT-experienced patients treated with cipa/mig throughout experienced a decline in urine Hex4 levels in PROPEL that stabilized during the OLE. ERT-experienced patients who received alg/pla in PROPEL experienced an increase in Hex4 and a marked decline after switching to cipa/mig in the OLE • ERT-naïve patients experienced a decline in Hex4 levels during PROPEL in both treatment groups that stabilized or declined further during the OLE to week 104 Number of patients (n) Mean (SD) baseline Hex4 (mmol/mol creatinine): Cipa/mig–cipa/mig = 4.5 (3.49) Alg/pla–cipa/mig = 7.3 (7.72) Mean (SD) baseline Hex4 (mmol/mol creatinine): Cipa/mig–cipa/mig = 2.7 (1.60) Alg/pla–cipa/mig = 5.0 (5.21) –4 –3 –2 –1 0 1 2 3 4 Cipa/mig–cipa/mig Cipa/mig Alg/pla Week 2 4 6 12 26 38 52 54 56 58 64 78 104 Number of patients (n) Mean (SD) baseline Hex4 (mmol/mol creatinine): Cipa/mig–cipa/mig = 4.8 (3.00) Alg/pla–cipa/mig = 5.8 (2.50) Mean (SD) baseline Hex4 (mmol/mol creatinine): Cipa/mig–cipa/mig = 2.0 (1.28) Alg/pla–cipa/mig = 2.4 (1.16) –4 –5 –3 –2 –1 0 Cipa/mig–cipa/mig Cipa/mig Alg/pla Week 2 4 6 12 26 38 52 54 56 58 64 78 104 Cipa/mig–cipa/mig 61 61 61 60 61 57 58 55 54 51 56 59 55 56 Cipa/mig–cipa/mig 20 20 19 19 19 18 18 17 18 19 16 19 19 17 Alg/pla–cipa/mig 29 29 29 29 29 28 28 28 27 26 28 27 27 26 Alg/pla–cipa/mig 7 7 7 7 7 7 7 5 7 5 7 6 6 6 Mean (+/ – SE) of change from ATB200-03 baseline (mmol/mol creatinine) Mean (+/ – SE) of change from ATB200-03 baseline (mmol/mol creatinine) WORLDSymposium Update – Phase 3 OLE of AT-GAA in LOPD Cipa/mig treatment was associated with a durable reduction in urine Hex4 during PROPEL and the OLE in both ERT-experienced and ERT-naïve patients *Excludes one outlier. Baseline Baseline ERT experienced ERT naïve* |
