2019 Galafold Revenue Nearly Doubled to $182.2M

On Track to Achieve 2020 Revenue Guidance of $250M-$260M

Focused on Pompe Phase 3 PROPEL Study, Manufacturing to Support 2021 BLA and MAA, and Accelerating Expanded Access Program for Infantile-Onset Patients

Advancing Industry-Leading Rare Disease Gene Therapy Portfolio

Strong Balance Sheet with $450M+ Cash – Cash Runway Well into 2022

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

CRANBURY, N.J., March 02, 2020 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq: FOLD), a global, patient-dedicated biotechnology company focused on discovering, developing and delivering novel medicines for rare diseases, today announced financial results for the full-year ended December 31, 2019. The Company also summarized recent program updates and reiterated its full-year 2020 guidance.

Corporate Highlights for Full-Year 2019 and Year-to-Date 2020

• Galafold® (migalastat), the first oral treatment option for people living with Fabry and who have an amenable variant, revenue grew from $91.2 million in full-year 2018 to $182.2 million in full-year 2019, exceeding the high end of the full-year 2019 guidance range of $170 million to $180 million. Over the course of 2019, Amicus received key marketing authorizations around the globe, including Argentina, Brazil, Colombia and Taiwan.
• Global Phase 3 PROPEL clinical trial of AT-GAA in late-onset Pompe disease exceeded enrollment and inventory build remains on track. As previously announced, 59 clinical sites enrolled 123 participants globally in the Phase 3 PROPEL study. Process performance qualification (PPQ) runs nearing successful completion with key strategic partner, WuXi Biologics, and will serve as the foundation for the Chemistry, Manufacturing, and Control (CMC) module for a biologics license application (BLA) submission.
• The Company plans to apply for and initiate a rolling BLA for AT-GAA, completing final submission in the first half of 2021.
• Focus on an Expanded Access Program for infantile-onset Pompe patients. Amicus intends to offer an expanded access program for infantile-onset patients.
• Presented positive interim results in ongoing Phase 1/2 clinical study for CLN6 Batten disease. Data on motor, language, seizure and vision sub scores suggest stabilization of these individual components in most patients, in particular those children treated at a younger age.
• Amicus continues to carefully manage expenses and investments, while executing on the Galafold launch and advancing development programs. The current cash position is expected to fund ongoing operations well into 2022.

2020 Key Strategic Priorities

• Achieve $250 million to $260 million of global product revenue for Galafold
• Complete Pompe Phase 3 PROPEL study, enroll pediatric studies and advance manufacturing to support 2021 BLA and MAA
• Advance clinical development, manufacturing and regulatory discussions for CLN6 and CLN3 Batten programs
• Progress Pompe gene therapy towards IND and disclose up to two additional IND candidates
• Maintain strong financial position

John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “Amicus has made great strides in our continued evolution as a leading global rare disease biotechnology company. We are on track and well-capitalized to achieve all our 2020 key strategic priorities including our global Fabry launch, Pompe late-stage development program, and gene therapy pipeline. With a very successful, commercial product in Fabry disease, a late stage program with Breakthrough Therapy Designation in late onset Pompe disease and 14 gene therapy programs for rare diseases in development, including two in the clinic, we are now, strongly positioned to achieve our vision of delivering groundbreaking new medicines and hopefully one day cures for people living with rare metabolic diseases.”

Full-Year 2019 Financial Results

• Total revenue in the full-year 2019 was $182.2 million, an increase from total revenue of $91.2 million in the full-year 2018.
• Cash, cash equivalents, and marketable securities totaled $452.7 million at December 31, 2019, compared to $504.2 million at December 31, 2018.
• Total GAAP operating expenses were $464.3 million for the full-year 2019, compared to $405.6 million in the full-year 2018. Operating expenses reflecting increased investments in the Galafold launch, Pompe program, and gene therapy pipeline.
• Total non-GAAP operating expenses of $411.8 million for the full-year 2019 increased as compared to $268.8 million for the full-year 2018, reflecting continued investments in the Galafold launch, Pompe program, and gene therapy pipeline. Non-GAAP operating expenses came in at the lower end of the guidance range of $410 million to $420 million. 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.
• Net loss was $356.4 million, or $1.48 per share, compared to a net loss of $349.0 million, or $1.88 per share, for the full-year 2018.

2020 Financial Guidance

• For the full-year 2020, the Company anticipates total Galafold revenue of $250 million to $260 million based on the average exchange rates for 2019.
• Non-GAAP operating expense guidance for the full-year 2020 is $410 million to $420 million, driven by continued investment in the global Galafold launch, AT-GAA clinical studies, and advancing our gene therapy pipeline. 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.
• Cash, cash equivalents, and marketable securities totaled $452.7 million at December 31, 2019. The current cash position is anticipated to fund ongoing operations well into 2022.

Anticipated 2020 Milestones by Program

Amicus previously announced 2020 program milestones in early January 2020. All anticipated milestones remain on track as follows:

Galafold (migalastat) Oral Precision Medicine for Fabry Disease

• On track to meet full-year 2020 revenue guidance range of $250 million to $260 million
• Registry and other Phase 4 supportive studies underway

AT-GAA for Pompe Disease

• Plans to apply for and initiate a Rolling Biologics License Application (BLA) for AT-GAA in 2020, with addition of full clinical results in 1H2021 to support full approval under Fast Track Designation
• Retrospective natural history study data in approximately 100 ERT-treated Pompe patients
• Additional supportive studies, including an open-label study in 12 to 18-year-old patients

Gene Therapy Portfolio

• Dose additional patients in CLN6 Phase 1/2 study and plan to advance regulatory discussions to finalize clinical and regulatory path
• Initiate long-term follow-up of initial participants in the CLN6 Phase 1/2 study in 1H2020 to obtain long-term safety and efficacy data
• Plan to advance regulatory discussions to finalize clinical and regulatory path in CLN3
• Report initial data on patients enrolled in CLN3 Phase 1/2 study
• Complete IND-enabling toxicology work in Pompe disease and progress towards IND
• Additional preclinical data expected in multiple programs
• Disclose up to two additional IND candidates
• Manufacturing advancements across portfolio

Conference Call and WebcastAmicus Therapeutics will host a conference call and audio webcast today, March 2, 2020, at 8:30 a.m. ET to discuss the full-year 2019 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: 2782337.

A live audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/, and will be archived for 30 days. 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 March 2, 2020. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 2782337.

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 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 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.

E.U. and U.K. Important Safety InformationTreatment 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 (