Amicus Therapeutics Announces Full-Year 2020 Financial Results and Corporate Updates

           2020 Total Galafold Revenue of $260.9M; Increased 43 Percent Year-on-Year
Continued Strong Global Growth of Galafold Expected in 2021 with Revenue of $300M-$315MAT-GAA Rolling BLA Submission in Pompe Disease Planned for Completion in 2Q21 and Other Global Submissions Expected Throughout 2021CLN6 Batten Disease Gene Therapy Granted Fast Track Designation by U.S. FDACash Position Sufficient to Achieve Self-SustainabilityConference Call and Webcast Today at 8:30 a.m. ETPHILADELPHIA, March 01, 2021 (GLOBE NEWSWIRE) — 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 full year ended December 31, 2020.John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, “During 2020, Amicus remained resilient on our journey to becoming a leading global rare disease biotechnology company. Despite the challenges of COVID, Amicus emerged from 2020 a better and stronger company organizationally, strategically, scientifically and financially. Galafold continues its path to becoming the worldwide standard of care for Fabry patients with amenable variants. And we are steadfast and passionate in our commitment to advancing AT-GAA to global regulatory submissions as fast as possible for the benefit of all people living with Pompe disease. The data from the Phase 3 PROPEL study we believe continue to show the overwhelmingly positive benefits of AT-GAA compared to the only approved medicine in this devastating disease. And the unmet need in Pompe is so great. We have the team, the resources and the programs that strongly position us to achieve our vision of delivering groundbreaking new medicines and hopefully, one day, cures for people living with rare diseases.”Corporate HighlightsGlobal revenue for Galafold^® (migalastat) in the full year of 2020 was $260.9 million. Full year revenue represented a year-over-year increase of 43% from total revenue of $182.2 million in the full year of 2019. On a constant currency basis, full year 2020 total revenue was $258.6 million, representing operational revenue growth measured at constant currency exchange rates of 42%, which was further benefited by a positive currency impact of $2.3 million, or 1%. Galafold performance was driven largely by strong patient demand. Global compliance and adherence rates continue to exceed 90%.Results from the global Phase 3 PROPEL clinical study of AT-GAA in late-onset Pompe disease (LOPD) were presented at the 17th Annual WORLDSymposium™ 2021. The Company plans to complete the BLA submission in the second quarter of this year and anticipates additional regulatory submissions in the European Union and in other geographies throughout 2021.The U.S. Food and Drug Administration (FDA) granted Fast Track Designation to the CLN6 Batten disease gene therapy, AT-GTX-501. The Fast Track program facilitates the development and accelerates the review of new drugs for serious conditions, which have the potential to address unmet medical needs. A drug development program with Fast Track designation is afforded greater access to the FDA for the purpose of expediting the drug’s development, review and potential approval.

Initial clinical data from the Phase 1/2 CLN3 gene therapy study were presented at the 17th Annual WORLDSymposium™ 2021. Results suggest early signs of disease stabilization and the potential to slow the neurological disease progression in children living with CLN3 Batten disease. Regulatory interactions are ongoing and the Company expects to submit the protocol for the next clinical study in the second half of this year.Preclinical data from the Company’s Fabry disease gene therapy clinical candidate, AT-GTX-701, presented at the 17th Annual WORLDSymposium™ 2021. As part of the research collaboration with the Gene Therapy Program of the Perelman School of Medicine at the University of Pennsylvania (Penn), initial data from the Fabry AAV gene therapy with an engineered GLA transgene improved for stability demonstrated greater substrate reduction than wild type constructs across all tissues and doses. These findings further validate the synergies of combining Amicus-engineered transgenes with Penn’s AAV technologies to develop next generation gene therapies.Cash position sufficient to achieve self-sustainability without the need for any future dilutive financings. The Company continues to carefully manage expenses and investments, while executing on the Galafold launch, proceeding with AT-GAA global regulatory submissions and advancing development programs. Full Year 2020 Financial ResultsTotal revenue in the full year 2020 was $260.9 million, a year-over-year increase of 43% from total revenue of $182.2 million in the full year of 2019. On a constant currency basis, full year 2020 total revenue was $258.6 million, representing operational revenue growth measured at constant currency exchange rates of 42%. Reported revenue was aided by a positive currency impact of $2.3 million, or 1%.Cash, cash equivalents, and marketable securities totaled $483.3 million at December 31, 2020, compared to $452.7 million at December 31, 2019.Total GAAP operating expenses of $476.8 million for the full year 2020 increased as compared to $464.3 million for the full year 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs.Total non-GAAP operating expenses of $415.7 million for the full year of 2020 increased as compared to $411.8 million in the full year of 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs.¹Net loss was $276.9 million, or $1.07 per share, compared to a net loss of $356.4 million, or $1.48 per share, for the full year 2019._{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 GuidanceFor the full-year 2021, the Company anticipates total Galafold revenue of at least $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.²Cash, cash equivalents, and marketable securities totaled $483.3 million at December 31, 2020. Based on current operating models, the Company believes that the current cash position, which includes the net proceeds from the 2020 Senior Secured Term Loan, and expected future revenues are sufficient to fund the Company’s operations and ongoing research programs through to self-sustainability._{² 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.}Anticipated 2021 Milestones by ProgramGalafold (migalastat) Oral Precision Medicine for Fabry DiseaseContinued revenue growth in 2021Plans to expand EU label to cover adolescent populationContinued geographic expansionRegistry and other Phase 4 studiesAT-GAA for Pompe DiseaseComplete the BLA submission in 1H2021 and the EU MAA submission to be completed in 2H2021Ongoing supportive studies, including pediatric and extension studiesGene Therapy PortfolioAdvance manufacturing and regulatory discussions for the CLN6 Batten disease gene therapy program and begin dosing additional patients with GMP grade materialReport initial data from the CLN3 Batten disease Phase 1/2 study, advance manufacturing and regulatory discussions, and submit protocol for next clinical studyContinue to progress IND-enabling work in both Pompe and Fabry gene therapyDisclose additional preclinical data and potential IND candidate declarations across multiple preclinical programsManufacturing advancements and updates across the portfolioConference Call and Webcast