PHILADELPHIA, Aug. 04, 2022 (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 quarter ended June 30, 2022.
Bradley Campbell, President and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “Through the first half of the year and into the third quarter, we have gained great momentum towards achieving our key strategic priorities for 2022. We are pleased by the continued global uptake of Galafold and continued patient demand, which is driving strong operational growth in-line with our 2022 guidance. We are focused on gaining regulatory approvals of AT-GAA for people living with Pompe disease around the world. Importantly, we are poised for the anticipated successful launch of AT-GAA and continue to believe in the potential of this treatment regimen to become the new global standard of care in Pompe disease. These efforts, together with our careful management of expenses and the financial strength of our business, uniquely position Amicus to deliver sustainable value for shareholders while upholding our mission for people living with rare diseases.”
- Global revenue for Galafold® (migalastat) in the first half of 2022 was $159.4 million, reflecting 11% growth with operational growth of 18% offset by currency headwinds of 7%. Second quarter sales of $80.7 million represented a year-over-year increase of 13% at constant exchange rates (CER)1. Second quarter reported revenue growth was 4% given significant currency headwinds of $6.7 million, or 9%.
|(in thousands)||Three Months Ended
|Year over Year % Growth||Six Months Ended
|Year over Year % Growth|
|Galafold Net Product Revenues||$80,731||$77,413||4%||13%||$159,446||$143,815||11%||18%|
- Galafold U.S. intellectual property estate strengthened following the issuance of 17 new patents this year. The Galafold U.S. intellectual property portfolio now includes 44 orange book listed patents, 28 of which provide protection through at least 2038, including 3 newly issued composition of matter patents.
- AT-GAA regulatory reviews progressing and pre-launch activities underway. In the U.S., the Food and Drug Administration (FDA) extended the Prescription Drug User Fee Act (PDUFA) action dates to August 29, 2022, for the New Drug Application (NDA) and October 29, 2022, for the Biologic License Application (BLA), reflective of the two components of AT-GAA. The Company continues to expect the FDA to approve the applications together by the October 29, 2022, action date. In the EU, the Committee for Medicinal Products for Human Use (CHMP) opinion is expected in late 2022.
- Expanded access programs in place to meet the growing demand for AT-GAA across multiple countries. In France, the National Agency for the Medicines and Health Products Safety (ANSM) granted the first reimbursed access to AT-GAA under their compassionate access (“Accès Compassionnel”) program. In the U.K., under the Early Access to Medicines Scheme (EAMS) multiple physicians have requested access across the leading Pompe centers in the country. Additional expanded access programs are in place in Germany and Japan with multiple Pompe patients participating in each.
- Amicus announces a program to explore next-generation pharmacological chaperones for Fabry disease through academic research collaboration agreement with the Spanish National Research Council (CSIC) and the University of Seville. This new collaboration will search for innovative glycomimetics with optimal activity and pharmacokinetic properties. Lead compounds will be selected based on their potential for greater potency, expanded number of amenable mutations, and optimal dosing.
- Company leadership transition complete. As of August 1, 2022, Bradley Campbell transitioned to the role of President and Chief Executive Officer of Amicus. John F. Crowley has transitioned to the role of Executive Chairman of Amicus for a two-year term, after which he is expected to continue as the non-executive Chairman of the Board.
- On track to achieve non-GAAP profitability2 in 2023. Through careful management of expenses, based on current operating models, the Company is on the path to achieve profitability in 2023, as it executes on the global expansion of Galafold and prepares for the global launch of AT-GAA.
Second Quarter 2022 Financial Results
- Total revenue in the second quarter 2022 was $80.7 million, a year-over-year increase of 4% from total revenue of $77.4 million in the second quarter of 2021. On a constant currency basis, second quarter 2022 total revenue growth was 13%. Reported revenue was offset by a negative currency impact of $6.7 million, or 9%.
- Cash, cash equivalents, and marketable securities totaled $386.8 million at June 30, 2022, compared to $482.5 million at December 31, 2021.
- Total GAAP operating expenses of $133.1 million for the second quarter 2022 increased as compared to $107.9 million for the second quarter 2021.
- Total non-GAAP operating expenses of $119.2 million for the second quarter of 2022 increased as compared to $93.5 million in the second quarter of 2021, reflecting non-recurring expenses related to the reprioritization of the gene therapy portfolio.3
- Net loss was $62.2 million, or $0.21 per share, compared to a net loss of $51.2 million, or $0.19 per share, for the second quarter 2021.
2022 Financial Guidance
- For the full-year 2022, the Company anticipates total Galafold revenue of $350 million to $365 million at constant exchange rates1. Double-digit revenue growth between 15 and 20% at CER1 in 2022 is expected to be driven by continued underlying demand from both switch and treatment-naïve patients, geographic expansion, the continued diagnosis of new Fabry patients and commercial execution across all major markets, including the U.S., EU, U.K., and Japan. Applying average July 2022 exchange rates, the negative currency impact on full-year 2022 Galafold reported sales would be approximately 9%.
- Non-GAAP operating expense guidance for the full-year 2022 is $470 million to $485 million, driven 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-GAA and committed obligations for the gene therapy portfolio. In 2023, Amicus expects non-GAAP operating expense at a level below 2021.4
Anticipated 2022 Milestones by Program
Galafold (migalastat) Oral Precision Medicine for Fabry Disease
- Sustain double-digit revenue growth in 2022 of $350 million to $365 million at CER
- Continue geographic expansion
- Registry and other Phase 4 studies ongoing
AT-GAA for Pompe Disease
- U.S. Prescription Drug User Fee Act (PDUFA) action date of August 29, 2022, for the NDA and October 29, 2022, for the BLA
- EU Committee for Medicinal Products for Human Use (CHMP) opinion expected in late 2022
- Continue to broaden expanded access plans in the U.K., Germany, France, Japan, and other countries
- Ongoing supportive studies, including pediatric and extension studies
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. 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, loss on impairment of assets, depreciation and amortization, acquisition related income (expense), loss on extinguishment of debt, loss on impairment of assets, 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.
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
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.
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.
- 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 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 metabolic diseases. With extraordinary patient focus, Amicus Therapeutics is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases. For more information please visit the company’s website at www.amicusrx.com, and follow on Twitter and LinkedIn.