2023 Total Revenue of
Strong Patient Demand Continues for Pombiliti™ + Opfolda™ in the
Projecting 2024 Galafold® Revenue Growth of 11-16% at CER
Anticipating Full-Year Non-GAAP Profitability in 2024
Conference Call and Webcast Today at
“In 2023, Amicus made tremendous progress across all our strategic priorities,” said
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 | Year over Year % Growth | Twelve Months Ended | 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 | 31% | 27% | 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 atDecember 31, 2023 , compared to$293.6 million atDecember 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. , andJapan . - 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
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
A live audio webcast and related presentation materials can also be accessed via the Investors section of the
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,
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.
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
For additional information about Galafold, including the full
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.
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
Non-GAAP Financial Measures
In addition to financial information prepared in accordance with
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
CONTACT:
Investors:
Vice President, Investor Relations
afaughnan@amicusrx.com
(609) 662-3809
Media:
Head of
dmoore@amicusrx.com
(609) 662-5079
FOLD-G
TABLE 1
Amicus Therapeutics, Inc. Consolidated Statements of Operations (in thousands, except share and per share amounts) | |||||||||||
Years Ended | |||||||||||
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) | |||||||
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 | 31,667 | 30,778 | |||||
Intangible assets, less accumulated amortization of | 20,490 | 23,000 | |||||
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, | 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 | |||||||||
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 | Years Ended | |||||||||||||||
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 |
Source:
2024 GlobeNewswire, Inc., source