Full Press Release Details
Amicus Therapeutics Announces Second Quarter
Financial Results and Corporate Updates
2Q21 Total Galafold (migalastat)
Revenue of $77.4M, a 24% increase over 2Q20
On-Track to Achieve Revenue Guidance of $300M-$315M
Completed the Rolling BLA and NDA Submissions
to the U.S. FDA for AT-GAA in Pompe Disease
Positive EMA Rapporteur and Co-Rapporteur
Meeting Support the MAA Submissions for AT-GAA;
Global Submissions On-Track in 2021
Conference Call and Webcast Today at 8:30
PA, Aug. 5, 2021 - Amicus Therapeutics (Nasdaq: FOLD), a patient-dedicated global biotechnology company focused
on discovering, developing and delivering novel medicines for rare diseases, today announced financial results for the quarter ended
Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, "Throughout this year and into the third quarter,
the global Amicus team has continued to advance our mission for patients and made significant strides towards achieving our 2021 strategic
priorities, including continued commercial execution of Galafold, the completion of our rolling BLA submission with the U.S. FDA and progression
of additional global regulatory work for AT-GAA, as well as advancing our industry-leading gene therapy pipeline. Through our efforts,
we remain well positioned to deliver on our mission for patients and shareholders, and to continue building Amicus into a leading global
rare disease biotechnology company. We are especially excited for and confident in our Pompe program now moving through regulatory reviews
around the world and hopeful that it will reach many more people living with Pompe disease as soon as possible."
Corporate Highlights
Second Quarter 2021 Financial Results
| Total revenue in the second quarter of 2021 was $77.4 million, a year-over-year increase of 24.0% from total revenue of $62.4 million in the second quarter of 2020. On a constant currency basis, second quarter 2021 total revenue was $73.1 million, representing operational revenue growth measured at constant currency exchange rates of 17.2%. Reported revenue was aided by a positive currency impact of $4.3 million, or 6.8%. | ||
| Cash, cash equivalents, and marketable securities totaled $383.1 million at June 30, 2021, compared to $483.3 million at December 31, 2020. | ||
| Total GAAP operating expenses of $107.9 million for the second quarter of 2021 increased as compared to $107.0 million for the second quarter 2020. | ||
| Total non-GAAP operating expenses of $93.5 million for the second quarter of 2021 decreased as compared to $95.9 million in the second quarter of 2020, reflecting the timing of investments in our pipeline. 1 | ||
| Net loss was $51.2 million, or $0.19 per share, compared to a net loss of $52.5 million, or $0.20 per share, for the second quarter 2020. |
1 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.
2021 Financial Guidance
| For the full-year 2021, the Company anticipates total Galafold revenue of $300 million to $315 million. Double-digit revenue growth in 2021 is expected to be driven by continued operational growth 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 2021 is $410 million to $420 million, driven by continued investment in the global Galafold launch, AT-GAA clinical studies and pre-launch activities, and advancing our gene therapy pipeline. 2 | ||
| Based on current operating models, the Company believes that the current cash position and expected future revenues are sufficient to fund the Company's operations and ongoing research programs through to self-sustainability. |
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.
2021 Milestones by Program
Galafold (migalastat) Oral Precision Medicine
| Continue revenue growth in 2021 | ||
| EU label expanded to cover adolescent population | ||
| Continue geographic expansion | ||
| Registry and other Phase 4 studies |
AT-GAA for Pompe Disease
| Completed the BLA and NDA submissions in 3Q21; EU MAA submissions to be completed in 2H2021 | ||
| Ongoing supportive studies, including pediatric and extension studies |
Gene Therapy Portfolio
| Advance manufacturing activities and regulatory discussions for the CLN6 Batten disease gene therapy program to enable dosing of additional patients with GMP clinical grade material | ||
| Reported initial data from the CLN3 Batten disease gene therapy Phase 1/2 study; advance manufacturing activities and regulatory discussions to enable dosing additional patients with GMP clinical-grade material | ||
| Continue to progress IND-enabling work in both Pompe and Fabry gene therapies | ||
| Disclose additional preclinical data and potential IND candidate declarations across multiple preclinical programs | ||
| Manufacturing advancements and updates across the portfolio |
Conference Call and Webcast
will host a conference call and audio webcast today, August 5, 2021 at 8:30 a.m. ET to discuss
the second quarter 2021 financial results and corporate updates. Interested participants and investors may access the conference
call by dialing 877-303-5859 (U.S./Canada) or 678-224-7784 (international), conference ID: 7374935.
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. A replay of the call will be available for seven
days beginning at 11:30 a.m. ET on August 5, 2021. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international);
conference ID: 7374935.
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 over 40 countries around the world, including the U.S., EU, U.K., Japan and others.
U.S. INDICATIONS AND USAGE
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
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. |
| 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.
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. Full reconciliations of GAAP results to the comparable non-GAAP measures for the reported periods
appear in the financial tables section of this press release. 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