Amicus Therapeutics, Inc.: Amicus Therapeutics Announces Full-Year 2023 Financial Results and Corporate Updates

2023 Total Revenue of $399.4M, a 21% Increase Year-over-Year

Strong Patient Demand Continues for Pombiliti + Opfolda in the U.S., U.K., and Germany

Projecting 2024 Galafold^® Revenue Growth of 11-16% at CER

Anticipating Full-Year Non-GAAP Profitability in 2024

Conference Call and Webcast Today at 8:30 a.m. ET

PRINCETON, N.J., Feb. 28, 2024 (GLOBE NEWSWIRE) -- 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, 2023.

"In 2023, Amicus made tremendous progress across all our strategic priorities," said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. "We strengthened our leadership position in Fabry and Pompe disease globally and achieved our goal of non-GAAP profitability in the fourth quarter. Patient demand for Galafold exceeded our expectations and grew at the highest rate seen in the last four years and we continue to be excited by the long-term growth of this important medicine. We also successfully launched our second commercial therapy, Pombiliti + Opfolda, in the three largest Pompe disease markets. In 2024, we will continue to drive significant top line revenue growth supported by sustained double-digit Galafold performance and the successful ongoing global commercial launch of Pombiliti + Opfolda putting us on track for our first full year of non-GAAP profitability. Amicus is at a major inflection point and strongly positioned to continue to advance our mission of delivering groundbreaking new medicines to thousands of people living with rare diseases and creating value for our shareholders."

Corporate Highlights:

• Total revenues for the full-year 2023 were $399.4 million, up 21%, reflecting operational growth measured at constant exchange rates (CER)¹ of 20% and favorable currency impact of $2.7 million or 1%. Fourth quarter total revenues were $115.1 million, up 31%, or 27% at CER.
  

(in thousands)	Three Months Ended December 31,				Year over Year % Growth				Twelve Months Ended December 31,				Year over Year % Growth
	2023		2022		Reported		at CER1		2023		2022		Reported		at CER1
Galafold®	106,600		87,989		21%		18%		387,777		329,046		18%		17%
Pombiliti+ Opfolda	8,482		107		n/a		n/a		11,579		187		n/a		n/a
Net Product Revenues	$115,082		$88,096		31%		27%		$399,356		$329,233		21%		20%

• Galafold (migalastat) net product sales for the full-year 2023 were $387.8 million, representing a year-over-year increase of 18%, or 17% at CER. Fourth quarter net product sales were $106.6 million. At the end of 2023, there were >2,400 people living with Fabry disease on Galafold following a year of increased demand.
• Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales for the full-year 2023 were $11.6 million. Fourth quarter net product sales were $8.5 million. The commercial launch of Pombiliti + Opfolda is underway in the three largest markets with 120 patients on treatment with commercial product or scheduled to be treated as of early January and continued strong patient demand.
• Eleven posters and an oral presentation highlighting Amicus' development programs in Fabry disease and Pompe disease presented at the 20th Annual WORLDSymposium. Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) honored with the 2024 New Treatment Award, which recognizes important achievements in advancing new treatments approved for lysosomal diseases.
• On a GAAP basis, net loss in the fourth quarter of 2023 was $33.8 million. The Company achieved non-GAAP profitability³ in the fourth quarter of 2023 of $2.6 million.

Full-Year 2023 Financial Results

• Total revenue in the full-year 2023 was $399.4 million, a year-over-year increase of 21% from total revenue of $329.2 million in the full-year 2022. On a constant currency basis, full-year 2023 total revenue growth was 20%. Reported revenue had a favorable currency impact of approximately $2.7 million, or 1%.
• Total GAAP operating expenses of $439.2 million for the full-year 2023 decreased by 13% as compared to $502.8 million for the full-year 2022.
• Total non-GAAP operating expenses of $341.6 million for the full-year 2023 decreased by 17% as compared to $413.2 million for the full-year 2022.
• GAAP net loss was $151.6 million, or $0.51 per share, for the full-year 2023, and was reduced compared to a net loss of $236.6 million, or $0.82 per share, for the full-year 2022.
• Non-GAAP net loss was $38.5 million, or $0.13 per share, for the full-year 2023, and was reduced compared to a net loss of $152.5 million, or $0.53 per share, for the full-year 2022.
• Cash, cash equivalents, and marketable securities totaled $286.2 million at December 31, 2023, compared to $293.6 million at December 31, 2022.

2024 Financial Guidance

• For the full-year 2024, the Company anticipates total Galafold revenue growth between 11% and 16% 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 2024 is $345 million to $365 million, driven by disciplined expense management offset by continued investment in Galafold, Pombiliti + Opfolda clinical studies, as well as global launch activities⁴.

Amicus is focused on the following key strategic priorities in 2024:

• Delivering double-digit Galafold revenue growth (11-16% at CER)
• Executing multiple successful launches of Pombiliti + Opfolda
• Advancing ongoing studies to support medical and scientific leadership in Fabry and Pompe diseases
• Achieving full year non-GAAP profitability²

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 2024 Galafold revenue guidance utilizes actual exchange rate as of December 31, 2023.
² Based on projections of Amicus' non-GAAP Net (Loss) Income under current operating plans, which includes successful Pombiliti + Opfolda launches and continued Galafold growth. Amicus defines non-GAAP Net (Loss) Income as GAAP Net (Loss) 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.

Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, February 28, 2024, at 8:30 a.m. ET to discuss the full-year 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 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 people living with Fabry disease 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.

About Pombiliti + Opfolda
Pombiliti + Opfolda, is a two-component therapy that consists of cipaglucosidase alfa-atga, 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.

U.S. INDICATIONS AND USAGE
POMBILITI in combination with OPFOLDA is indicated for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing =40 kg and who are not improving on their current enzyme replacement therapy (ERT).

SAFETY INFORMATION

HYPERSENSITIVITY REACTIONS INCLUDING ANAPHYLAXIS: Appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available. If a severe hypersensitivity reaction occurs, POMBILITI should be discontinued immediately and appropriate medical treatment should be initiated. INFUSION-ASSOCIATED REACTIONS (IARs): If severe IARs occur, immediately discontinue POMBILITI and initiate appropriate medical treatment. RISK OF ACUTE CARDIORESPIRATORY FAILURE IN SUSCEPTIBLE PATIENTS: Patients susceptible to fluid volume overload, or those with acute underlying respiratory illness or compromised cardiac or respiratory function, may be at risk of serious exacerbation of their cardiac or respiratory status during POMBILITI infusion. See PI for complete Boxed Warning. CONTRAINDICATION: POMBILITI in combination with Opfolda is contraindicated in pregnancy. EMBRYO-FETAL TOXICITY: May cause embryo-fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception during treatment and for at least 60 days after the last dose. Adverse Reactions: Most common adverse reactions = 5% are headache, diarrhea, fatigue, nausea, abdominal pain, and pyrexia. Please see full PRESCRIBING INFORMATION, including BOXED WARNING, for POMBILITI (cipaglucosidase alfa-atga) LINK and full PRESCRIBING INFORMATION for OPFOLDA (miglustat) LINK.

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 X 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 use these non-GAAP measures as key performance measures for the purpose of evaluating operational performance and cash requirements internally. 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 and profitability on a forward-looking basis, a reconciliation of the differences between the non-GAAP expectation and the corresponding GAAP measure generally is not available without unreasonable effort due to potentially high variability, complexity and low visibility as to the items that would be excluded from the GAAP measure in the relevant future period, such as unusual gains or losses. The variability of the excluded items may have a significant, and potentially unpredictable, impact on our future GAAP results.

Forward Looking 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 and pricing and reimbursement authorities, are based on current information. 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 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 negotiations with pricing and reimbursement authorities; the potential that we may not be successful in commercializing Galafold and/or Pombiliti and Opfolda in Europe, the UK, the US and other geographies; 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, the manufacturing, and commercialization of our products. With respect to statements regarding corporate financial guidance and financial goals and the expected attainment of such goals and projections of the Company's revenue, non-GAAP profitability 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, 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
[e-mail]
[phone]

Media:
Amicus Therapeutics
Diana Moore
Head of Global Corporate Affairs and Communications
[e-mail]
[phone]

FOLD-G

TABLE 1

Amicus Therapeutics, Inc. Consolidated Statements of Operations (in thousands, except share and per share amounts)
	Years Ended December 31,
		2023				2022				2021
Net product sales	$	399,356			$	329,233			$	305,514
Cost of goods sold		37,326				38,599				34,466
Gross profit		362,030				290,634				271,048
Operating expenses:
Research and development		152,381				276,677				272,049
Selling, general, and administrative		275,270				213,041				192,710
Changes in fair value of contingent consideration payable		2,583				1,078				6,514
Loss on impairment of assets		1,134				6,616				-
Depreciation and amortization		7,873				5,342				6,209
Total operating expenses		439,241				502,754				477,482
Loss from operations		(77,211	)			(212,120	)			(206,434	)
Other (expense) income:
Interest income		7,078				3,024				509
Interest expense		(50,149	)			(37,119	)			(32,471	)
Loss on extinguishment of debt		(13,933	)			-				(257	)
Other (expense) income		(15,886	)			4,176				(2,901	)
Loss before income tax		(150,101	)			(242,039	)			(241,554	)
Income tax (expense) benefit		(1,483	)			5,471				(8,906	)
Net loss attributable to common stockholders	$	(151,584	)		$	(236,568	)		$	(250,460	)
Net loss attributable to common stockholders per common share - basic and diluted	$	(0.51	)		$	(0.82	)		$	(0.92	)
Weighted-average common shares outstanding - basic and diluted		295,164,515				289,057,198				271,421,986

TABLE 2

Amicus Therapeutics, Inc. Consolidated Balance Sheets (in thousands, except share and per share amounts)
	December 31,
		2023				2022
Assets
Current assets:
Cash and cash equivalents	$	246,994			$	148,813
Investments in marketable securities		39,206				144,782
Accounts receivable		87,632				66,196
Inventories		59,696				23,816
Prepaid expenses and other current assets		49,533				40,209
Total current assets		483,061				423,816
Operating lease right-of-use assets, net		26,312				29,534
Property and equipment, less accumulated depreciation of $25,429 and $22,281 at December 31, 2023 and 2022, respectively		31,667				30,778
Intangible assets, less accumulated amortization of $2,510 and $0 at December 31, 2023 and December 31, 2022, respectively		20,490				23,000
Goodwill		197,797				197,797
Other non-current assets		18,553				19,242
Total Assets	$	777,880			$	724,167
Liabilities and Stockholders' Equity
Current liabilities:
Accounts payable	$	15,120			$	15,413
Accrued expenses and other current liabilities		144,245				93,636
Contingent consideration payable		-				21,417
Operating lease liabilities		8,324				8,552
Total current liabilities		167,689				139,018
Long-term debt		387,858				391,990
Operating lease liabilities		48,877				51,578
Other non-current liabilities		13,282				18,534
Total liabilities		617,706				601,120
Commitments and contingencies
Stockholders' equity:
Common stock, $0.01 par value, 500,000,000 shares authorized, 293,594,209 and 281,108,273 shares issued and outstanding at December 31, 2023 and 2022, respectively		2,918				2,815
Additional paid-in capital		2,836,018				2,664,744
Accumulated other comprehensive gain (loss):
Foreign currency translation adjustment		5,429				(11,989	)
Unrealized loss on available-for-sale securities		(188	)			(116	)
Warrants		71				83
Accumulated deficit		(2,684,074	)			(2,532,490	)
Total stockholders' equity		160,174				123,047
Total Liabilities and Stockholders' Equity	$	777,880			$	724,167

TABLE 3

Amicus Therapeutics, Inc. Reconciliation of Non-GAAP Financial Measures (in thousands) (Unaudited)
		Years Ended December 31,
			2023			2022			2021
Total GAAP operating expenses		$	439,241		$	502,754		$	477,482
Research and development:
Share-based compensation			21,469			25,089			17,340
Selling, general and administrative:
Share-based compensation			64,608			51,423			40,498
Loss on impairment of assets			1,134			6,616			-
Changes in fair value of contingent consideration payable			2,583			1,078			6,514
Depreciation and amortization			7,873			5,342			6,209
Total Non-GAAP operating expense adjustments			97,667			89,548			70,561
Total Non-GAAP operating expenses		$	341,574		$	413,206		$	406,921

TABLE 4

Amicus Therapeutics, Inc. Reconciliation of Non-GAAP Financial Measures (in thousands, except share and per share amounts) (Unaudited)
		Three Months Ended December 31,								Years Ended December 31,
			2023				2022				2023				2022
GAAP net loss		$	(33,843	)		$	(55,865	)		$	(151,584	)		$	(236,568	)
Share-based compensation			18,095				18,626				86,077				76,512
Loss on impairment of assets			-				-				1,134				6,616
Changes in fair value of contingent consideration payable			-				1,584				2,583				1,078
Depreciation and amortization			2,182				1,311				7,873				5,342
Loss on extinguishment of debt			13,933				-				13,933				-
Income tax expense (benefit)			2,183				(14,214	)			1,483				(5,471	)
Non-GAAP net income (loss)		$	2,550			$	(48,558	)		$	(38,501	)		$	(152,491	)
Non-GAAP net income (loss) attributable to common stockholders per common share - basic and diluted		$	0.01			$	(0.17	)		$	(0.13	)		$	(0.53	)
Weighted-average common shares outstanding - basic and diluted			300,648,503				289,602,648				295,164,515				289,057,198