 Galafold 2Q2020 Revenue of 62.4 Million, On-Track to Achieve 2020 Revenue Guidance of 250M-260MAT-GAA Phase 3 PROPEL Study Readout and Rolling BLA Submission to U.S. FDA on ScheduleAdvancing Industry-Leading Rare Disease Gene Therapy PortfolioPath to Profitability Achieved without the Need for Any Future Dilutive FinancingsConference Call and Webcast Today at 8:30 a.m. ETCRANBURY, N.J. , Aug. 10, 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 second quarter ended June 30, 2020. The Company also summarized recent program updates and reiterated its full-year 2020 guidance.John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc., stated, During the second quarter, we made tremendous progress advancing our mission for patients and are on track to achieve our 2020 key strategic priorities, including our global Fabry launch, Pompe late-stage development program, and advancing our industry-leading gene therapy pipeline. Following our strategic financing in July, Amicus is firmly on a path to profitability without the need to access the equity markets. Through these efforts, we remain strongly positioned to achieve our vision of delivering groundbreaking new medicines and hopefully, one day, cures for people living with rare diseases.Corporate Highlights Global revenue for Galafold migalastat in the second quarter of 2020 was 62.4M. Second quarter revenue represented a year-over-year increase of 41 from total revenue of 44.1 million in the second quarter of 2019. On a constant currency basis, second quarter 2020 total revenue was 63.3 million, representing operational revenue growth measured at constant currency exchange rates of 43, which was offset by a negative currency impact of 1.0 million, or 2. Strong second quarter revenue represents the continued performance across the global business, including new patient starts from switch and naïve patients throughout the quarter in all major regions, including that hardest hit by COVID-19. Performance driven largely by strong patient demand. Global compliance and adherence rates continue to exceed 90. 1,000 mutations added to the EU Galafold label. This update includes a number of new mutations identified by creating and testing all possible single-base pair point mutations in the GLA gene. Including this list will help physicians assess treatment options in an expeditious manner. Amenable mutations continue to represent up to half of all people living with Fabry disease. Global Phase 3 PROPEL clinical study of AT-GAA in late-onset Pompe disease LOPD remains on track for top line data in 1H21. To date, 97 of the 2,810 planned infusions and assessments for the ongoing PROPEL study have been completed on schedule. The Company plans to initiate a rolling BLA for AT-GAA in the second half of 2020, completing final submission in the first half of 2021. Expanded Access Program for infantile-onset Pompe patients underway. Amicus has initiated an expanded access program for its investigational medicine AT-GAA for young children living with infantile-onset Pompe disease IOPD. Additional Phase 12 CLN6 data to be presented this year at the Child Neurology Society Annual Meeting in October. Regulatory interactions are ongoing and the Company expects to provide feedback on the path forward in early 2021. Initial data from the Phase 12 CLN3 study expected in early 2021 based on changes in medical conference schedules. Regulatory interactions are ongoing and concurrent with the data, the Company expects to provide feedback on the regulatory pathway. Positive preclinical Pompe gene therapy data presented at American Society of Cell and Gene Therapy meeting in May. Positive results showed the Amicus engineered hGAA having better targeting and clearance of glycogen storage in Pompe mice. Preliminary data in non-human primates suggested therapeutically relevant expression levels in target organs. Cash position sufficient to achieve self-sustainability without the need for any future dilutive financings. The previously announced debt facility places Amicus firmly on a path to profitability, while the Company continues to carefully manage expenses and investments, while executing on the Galafold launch and advancing development programs. 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 Investigational New Drug IND application and disclose up to two additional IND candidates Maintain strong financial positionSecond Quarter 2020 Financial Results Total revenue in the second quarter 2020 was 62.4 million, a year-over-year increase of 41 from total revenue of 44.1 million in the second quarter of 2019. On a constant currency basis, second quarter 2020 total revenue was 63.3 million, representing operational revenue growth measured at constant currency exchange rates of 43, which was offset by a negative currency impact of 1.0 million, or 2. Cash, cash equivalents, and marketable securities totaled 309.6 million at June 30, 2020, compared to 452.7 million at December 31, 2019. Total GAAP operating expenses of 107.0 million for the second quarter 2020 decreased as compared to 115.2 million for the second quarter 2019, reflecting decreased travel and third-party costs, offset by continued investments in our gene therapy pipeline. Total non-GAAP operating expenses of 95.9 million for the second quarter of 2020 decreased as compared to 103.6 million in the second quarter of 2019, reflecting decreased travel and third-party costs, offset by continued investments in our gene therapy pipeline.1 Net loss was 52.5 million, or 0.20 per share, compared to a net loss of 84.6 million, or 0.36 per share, for the second quarter 2019.1 Full reconciliation of GAAP results to the Companys non-GAAP adjusted measures for all reporting periods appear in the tables to this press release.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.2 Cash, cash equivalents, and marketable securities totaled 309.6 million at June 30, 2020. Based on current operating models, the Company believes that the current cash position, along with the net proceeds from the 2020 Senior Secured Term Loan and expected revenues, is sufficient to fund the Companys operations and ongoing research programs through to profitability.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 2020 Milestones by ProgramGalafold migalastat Oral Precision Medicine for Fabry Disease On track to meet full-year 2020 revenue guidance range of 250 million to 260 million Continued geographic expansion Registry and other Phase 4 studies underwayAT-GAA for Pompe Disease Plans to initiate a Rolling Biologics License Application BLA for AT-GAA in 2020, with addition of complete clinical results for PROPEL in 1H2021 to support full approval Retrospective natural history study data in approximately 100 Pompe Patients treated with enzyme replacement therapy Additional supportive studies, including an open-label study in 12- to 18-year-olds living with PompeGene Therapy Portfolio Report further safety and efficacy data in the CLN6 Batten disease Phase 12 study and advance regulatory discussions to finalize clinical and regulatory path Initial data from the CLN3 Batten disease Phase 12 study expected in early 2021 and advance regulatory discussions to finalize clinical and regulatory path Continue IND-enabling toxicology work in Pompe disease and progress towards IND Additional preclinical data expected across multiple programs with disclosure of up to two additional IND candidates Manufacturing advancements across portfolio Conference Call and Webcast Amicus Therapeutics will host a conference call and audio webcast today, August 10, 2020 at 8:30 a.m. ET to discuss the second quarter 2020 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: 4949075.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 10, 2020. Access numbers for this replay are 855-859-2056 U.S.Canada and 404-537-3406 international; conference ID: 4949075.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. 