Migalastat Hydrochloride

Amicus Therapeutics and Teva Pharmaceuticals have struck a deal that will allow the latter to sell a generic version of Galafold (migalastat), Amicus’ chaperone therapy for Fabry disease, to patients in the U.S.Under the agreement, Teva will have its clearance on Jan. 30, 2037, assuming the U.S. Food and Drug Administration (FDA) approves the company’s generic therapy and that no other issues arise in the meantime.The generic version should have the same active ingredient, strength, route of delivery, safety, efficacy, and intended use as its brand-name alternative. Generic medicines usually come at a significantly lower cost, however.The settlement “is a major step forward in ensuring Amicus can continue to support the Fabry community with Galafold for many years to come,” Bradley Campbell, president and CEO of Amicus, said in a company financial update.Amicus and Teva have been engaged in a legal dispute since 2020 when Teva accused Amicus of refusing to provide access to enough Galafold samples for studies that could determine the equivalence of Teva’s generic formulation.While the dispute ended the following year, Teva submitted an application to the FDA in 2022 seeking approval of its Galafold generic before Amicus’ patents on the medication had expired.With the new agreement, which was finalized in October, all litigation between the two companies has ended. Amicus is still engaged in a separate dispute with Aurobindo Pharma due to a similar issue, however.Fabry disease is caused by mutations in the gene that encodes alpha-galactosidase A (Gal A), an enzyme essential for breaking down certain fatty molecules. Without a working version of the enzyme, fatty molecules build up to toxic levels inside cells, leading to the organ damage that drives Fabry symptoms.Galafold is a chaperone therapy that’s designed to bind to the defective Gal A enzyme and stabilize it so it can function better, thereby allowing fatty molecules to clear, slowing the disease’s progression.Taken once daily, the oral medication has been approved in the U.S. for Fabry patients with certain types of disease-causing mutations since 2018. It’s also cleared in the European Union and other markets around the world. It carries a list price of $315,000 per year.“Amicus continues to be well positioned to drive sustainable shareholder value and further our mission of delivering great medicines for people living with rare diseases,” Campbell said.

Q3 2024 Total Revenue of $141.5M, a 37% Increase Year-over-Year Galafold® Q3 Revenue of $120.4M, up 20% Year-over-Year Pombiliti® + Opfolda® Q3 Revenue of $21.1M, up 33% from Q2 2024 Raising 2024 Total Revenue Growth Guidance to 30%-32% at CER Reducing non-GAAP Operating Expense Guidance to $340M to $350M Conference Call and Webcast Today at 8:30 a.m. ET PRINCETON, N.J., Nov. 06, 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 third quarter ended September 30, 2024. “The third quarter of the year was marked by the excellent commercial performance of our two approved therapies and continued financial discipline,” said Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc. “Strong patient demand for Galafold drove double digit revenue growth, while the commercial launch of Pombiliti and Opfolda continues to build momentum. We also announced a settlement of the Galafold (migalastat) patent litigation with Teva, which is a major step forward in ensuring Amicus can continue to support the Fabry community with Galafold for many years to come. Importantly, throughout the first nine months of the year, we’ve exceeded expectations, which resulted in the achievement of non-GAAP profitability for the full year 2024 as we closed the third quarter. Amicus continues to be well positioned to drive sustainable shareholder value and further our mission of delivering great medicines for people living with rare diseases.” Financial and Corporate Highlights: Total revenue in the third quarter 2024 was $141.5 million, a year-over-year increase of 37% from total revenue of $103.5 million in the third quarter 2023. On a constant currency basis (CER)1, third quarter 2024 total revenue growth was 36%. (in thousands)Three Months Ended September 30, Year over Year % Growth Nine Months Ended September 30, Year over Year % Growth 2024 2023 Reported at CER1 2024 2023 Reported at CER1Galafold®$120,381 $100,733 20% 19% $330,557 $281,177 18% 18%Pombiliti® + Opfolda®$21,136 $2,768 664% 658% $48,032 $3,097 1451% 1442%Net Product Revenues$141,517 $103,501 37% 36% $378,589 $284,274 33% 33% Galafold (migalastat) net product sales were $120.4 million in the third quarter 2024, a year-over-year increase of 20%, or 19% at constant exchange rates1, reflecting continued strong demand. Given strong performance in the first nine months of 2024, the Company is raising its full year 2024 revenue growth guidance for Galafold to +16% to +18% on a constant currency basis (CER)1.Pombiliti (cipaglucosidase alfa-atga) + Opfolda (miglustat) net product sales were $21.1 million in the third quarter 2024, a 33% increase from the second quarter of 2024. As of the end of October, 203 patients have been treated or are scheduled to be treated with commercial product in five markets (USA, Germany, UK, Spain, and Austria). Given strong launch momentum, the Company is raising its full year 2024 revenue guidance for Pombiliti + Opfolda to $69 million to $71 million on a constant currency basis (CER)1.Total GAAP operating expenses of $106.6 million for the third quarter 2024 decreased by 4% as compared to $110.6 million for the third quarter 2023. Total non-GAAP operating expenses of $82.6 million for the third quarter 2024 decreased by 8% as compared to $89.8 million for the third quarter 2023. Given continued financial discipline in the first nine months of 2024, the Company is reducing its non-GAAP Operating Expense guidance3 to $340 million to $350 million.GAAP net loss was $6.7 million, or $0.02 per share, for the third quarter 2024, and was reduced compared to a net loss of $21.6 million, or $0.07 per share, for the third quarter 2023.Non-GAAP net income was $30.8 million, or $0.10 per share, for the third quarter 2024, compared to a non-GAAP net loss of $4.0 million, or $0.01 per share, for the third quarter 20232. Non-GAAP profitability was also achieved in the first nine months of 2024.Cash, cash equivalents, and marketable securities totaled $249.8 million at September 30, 2024, compared to $286.2 million at December 31, 2023.In October 2024, the Company announced that it has entered into a License Agreement with Teva Pharmaceuticals USA, Inc. and Teva Pharmaceuticals, Inc. allowing Teva to market a generic version of Galafold® in the United States beginning on January 30, 2037, if approved by the U.S. Food and Drug Administration (FDA) and unless certain limited circumstances customarily included in these types of agreements occur. Similar patent litigation previously disclosed by the Company will continue against Aurobindo (Aurobindo Pharma LTD and Aurobindo Pharma USA, Inc.) as the remaining active party and the litigation stay remains in place for Lupin (Lupin LTD and Lupin Pharmaceuticals, Inc.). 2024 Financial Guidance: Previous UpdatedTotal Revenue Growth126% to 31%→30% to 32%Galafold Revenue Growth114% to 18%→16% to 18%Pombiliti + Opfolda Revenue1$62M to $67M→$69M to $71MNon-GAAP Operating Expense3$345M to $360M→$340M to $350M Amicus is focused on the following key strategic priorities in 2024: Delivering double-digit Galafold revenue growthExecuting multiple successful launches of Pombiliti + OpfoldaAdvancing ongoing studies to support medical and scientific leadership in Fabry and Pompe diseasesAchieving full-year non-GAAP profitability4 1 At constant exchange rates (CER). In order to illustrate underlying performance, Amicus discusses its results in terms of CER growth. This represents growth calculated as if the exchange rates had remained unchanged from those used in the comparative period. Full-year revenue guidance utilizes actual exchange rate as of December 31, 2023.2 Full reconciliation of GAAP results to the Company’s non-GAAP adjusted measures for the reporting period(s) appear in the tables to this press release.3 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.4 Based on projections of Amicus’ non-GAAP Net (Loss) Income under current operating plans, which includes successful Pombiliti + Opfolda launch 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. Conference Call and Webcast Amicus Therapeutics will host a conference call and audio webcast today, November 6, 2024, at 8:30 a.m. ET to discuss the third quarter 2024 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 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 USAGEGalafold 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 REACTIONSThe most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia. USE IN SPECIFIC POPULATIONSThere 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 + OpfoldaPombiliti + 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 USAGEPOMBILITI 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, and on Form 10-Q for the quarter ended September 30, 2024, 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 FaughnanVice President, Investor Relationsafaughnan@amicusrx.com(609) 662-3809 Media: Amicus Therapeutics Diana Moore Head of Global Corporate Affairs and Communicationsdmoore@amicusrx.com(609) 662-5079 FOLD-G TABLE 1 Amicus Therapeutics, Inc.Consolidated Statements of Operations(Unaudited)(in thousands, except share and per share amounts) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 Net product sales$141,517 $103,501 $378,589 $284,274 Cost of goods sold 13,279 9,946 38,107 26,002 Gross profit 128,238 93,555 340,482 258,272 Operating expenses: Research and development 26,160 40,704 79,172 117,352 Selling, general, and administrative 75,106 65,651 236,711 205,031 Changes in fair value of contingent consideration payable — 1,995 — 2,583 Restructuring charges 3,143 — 9,188 — Loss on impairment of assets — — — 1,134 Depreciation and amortization 2,170 2,228 6,506 5,691 Total operating expenses 106,579 110,578 331,577 331,791 Income (loss) from operations 21,659 (17,023) 8,905 (73,519)Other expense: Interest income 1,081 1,471 3,991 5,407 Interest expense (12,692) (12,986) (37,640) (37,322)Other (expense) income (3,263) 3,833 (11,946) (13,007)Income (loss) before income tax 6,785 (24,705) (36,690) (118,441)Income tax (expense) benefit (13,514) 3,128 (34,155) 700 Net loss attributable to common stockholders$(6,729) $(21,577) $(70,845) $(117,741)Net loss attributable to common stockholders per common share — basic and diluted$(0.02) $(0.07) $(0.23) $(0.40)Weighted-average common shares outstanding — basic and diluted 304,690,596 295,759,435 303,792,479 293,314,167 TABLE 2 Amicus Therapeutics, Inc.Consolidated Balance Sheets(Unaudited)(in thousands, except share and per share amounts) September 30, 2024 December 31, 2023Assets Current assets: Cash and cash equivalents$233,647 $246,994 Investments in marketable securities 16,110 39,206 Accounts receivable 98,073 87,632 Inventories 115,338 59,696 Prepaid expenses and other current assets 35,306 49,533 Total current assets 498,474 483,061 Operating lease right-of-use assets, net 23,144 26,312 Property and equipment, less accumulated depreciation of $29,324 and $25,429 at September 30, 2024 and December 31, 2023, respectively 30,438 31,667 Intangible assets, less accumulated amortization of $4,974 and $2,510 at September 30, 2024 and December 31, 2023, respectively 18,026 20,490 Goodwill 197,797 197,797 Other non-current assets 18,678 18,553 Total Assets$786,557 $777,880 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable$13,481 $15,120 Accrued expenses and other current liabilities 136,116 144,245 Operating lease liabilities 8,541 8,324 Total current liabilities 158,138 167,689 Long-term debt 389,494 387,858 Operating lease liabilities 46,623 48,877 Other non-current liabilities 13,477 13,282 Total liabilities 607,732 617,706 Commitments and contingencies Stockholders’ equity: Common stock, $0.01 par value, 500,000,000 shares authorized, 298,691,094 and 293,594,209 shares issued and outstanding at September 30, 2024 and December 31, 2023, respectively 2,942 2,918 Additional paid-in capital 2,905,760 2,836,018 Accumulated other comprehensive income (loss): Foreign currency translation adjustment 25,159 5,429 Unrealized loss on available-for-sale securities (188) (188)Warrants 71 71 Accumulated deficit (2,754,919) (2,684,074)Total stockholders’ equity 178,825 160,174 Total Liabilities and Stockholders’ Equity$786,557 $777,880 TABLE 3 Amicus Therapeutics, Inc.Reconciliation of Non-GAAP Financial Measures(in thousands)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023Total operating expenses - as reported GAAP$ 106,579 $ 110,578 $ 331,577 $ 331,791Research and development: Stock-based compensation 4,397 4,380 12,329 16,987Selling, general and administrative: Stock-based compensation 14,291 12,131 53,359 50,995Loss on impairment of assets — — — 1,134Changes in fair value of contingent consideration payable — 1,995 — 2,583Restructuring Charges 3,143 — 9,188 —Depreciation and amortization 2,170 2,228 6,506 5,691Total operating expense adjustments to reported GAAP 24,001 20,734 81,382 77,390Total operating expenses - as adjusted$82,578 $89,844 $250,195 $254,401 TABLE 4 Amicus Therapeutics, Inc.Reconciliation of Non-GAAP Financial Measures(in thousands, except share and per share amounts)(Unaudited) Three Months Ended September 30, Nine Months Ended September 30, 2024 2023 2024 2023 GAAP net loss $(6,729) $(21,577) $(70,845) $(117,741)Share-based compensation 18,688 16,511 65,688 67,982 Changes in fair value of contingent consideration payable — 1,995 — 2,583 Depreciation and amortization 2,170 2,228 6,506 5,691 Loss on impairment of assets — — — 1,134 Restructuring charges 3,143 — 9,188 — Income tax expense (benefit) 13,514 (3,128) 34,155 (700)Non-GAAP net income (loss) $30,786 $(3,971) $44,692 $(41,051) Non-GAAP net income (loss) attributable to common stockholders per common share — basic and diluted $0.10 $(0.01) $0.15 $(0.14)Weighted-average common shares outstanding — basic and diluted 304,690,596 295,759,435 303,792,479 293,314,167