3Q19 Galafold® (migalastat) Revenue of $48.8M
and 1,000+ Patients on Therapy Reflects Continued Strong Global UptakeReiterating Upwardly Revised FY19 Revenue Guidance of $170M-$180MComplete Enrollment of 120+ Patients in AT-GAA Pompe Pivotal Study on Track by YE19Continued Progress Across Industry Leading Rare Disease Gene Therapy PortfolioStrong Balance Sheet with $514M+ Cash Provides Cash Runway Well into 1H22Conference Call and Webcast Today at 8:30 a.m. ETCRANBURY, N.J., Nov. 11, 2019 (GLOBE NEWSWIRE) — Amicus Therapeutics (Nasdaq: FOLD), a global biotechnology company focused on discovering, developing and delivering novel medicines for rare metabolic diseases, today announced financial results for the third quarter ended September 30, 2019. The Company also summarized recent program updates and reiterated its full-year 2019 guidance.John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “The third quarter represented another consecutive period of significant growth and adoption for Galafold across all geographies, as well as continued momentum in our Pompe pivotal study and our gene therapy pipeline. We are on track to meet or exceed each of our key strategic priorities for the year as we lay the foundation for our long-term success. Importantly, we have significantly strengthened our financial outlook with careful management of our expenses and investments. With confidence in our base business and overall financial outlook, we are well capitalized to continue to grow our revenues, advance our pipeline, and move toward self-sustainability and profitability as we continue to build Amicus into a leading global rare disease biotechnology company delivering on our mission for patients and shareholders.”Corporate Highlights for 3Q19 and Early 4Q19Global revenue for Fabry precision medicine Galafold in the third quarter of 2019 was $48.8 million and continues to track toward the upwardly revised full-year 2019 revenue guidance of $170 million to $180 million. Third quarter revenue represented a year-over-year increase of 137% from total revenue of $20.6 million in the third quarter of 2018, and a quarter over-quarter increase of 11% from total revenue of $44.1 million in the second quarter of 2019. As of September 30, 2019, Galafold represented an estimated 30% of global market share of treated amenable patients. Global compliance and adherence rates continue to exceed 90%.
Financial outlook strengthened with current cash runway now revised to well into 1H 2022 through major portfolio milestones and global growth.
Positive Phase 2 clinical data for AT-GAA in Pompe disease. Amicus presented initial six-month data in additional ERT-switch patients (Cohort 4) and full 24-month data from the first three cohorts in Phase 1/2 ATB200-02 clinical study at World Muscle Society.
Pompe Pivotal PROPEL study is expected to over-enroll (~120 Patients) by YE 2019. Given the strong global interest among the Pompe patient and physician community for AT-GAA, which has U.S. Breakthrough Therapy designation, this global study is now expected to enroll ~120 patients by year-end 2019. Pompe manufacturing also continues to advance with PPQ runs now initiated at WuXi.
Positive interim Phase 1/2 clinical data for gene therapy in CLN6 Batten disease. Initial results as well as additional supportive data at Child Neurology Society showed AAV-CLN6 gene therapy has the potential to halt the progression of a devastating disease that causes loss of brain function and is fatal in childhood.
Pompe gene therapy clinical candidate declared to move into IND-enabling studies. Dose-ranging preclinical studies are currently underway to build off the initial preclinical results showing robust uptake and glycogen reduction in multiple tissues, including brain and spinal cord.
Robust portfolio of gene therapy programs and technologies provides foundation for future, including two clinical-stage programs (CLN6 and CLN3), and eight preclinical gene therapies.3Q19 Financial ResultsTotal revenue in the third quarter 2019 was $48.8 million, a year-over-year increase of 137% from total revenue of $20.6 million in the third quarter of 2018, and a quarter over-quarter increase of 11% from total revenue of $44.1 million in the second quarter of 2019. On a constant currency basis, third quarter 2019 total revenue was $50.3 million, representing operational revenue growth measured at constant currency exchange rates of 143%, which was offset by a negative currency impact of $1.3 million, or 6%.Cash, cash equivalents, and marketable securities totaled $514.2 million at September 30, 2019, compared to $504.2 million at December 31, 2018.Total GAAP operating expenses of $100.5 million for the third quarter of 2019 decreased as compared to $172.5 million in the third quarter of 2018. The decrease is primarily due to an upfront payment of $100 million for the Celenex asset acquisition in 2018 partially offset by continued investments in the Galafold launch, Pompe clinical study program, and our gene therapy pipeline.Total non-GAAP operating expenses of $89.7 million for the third quarter of 2019 increased as compared to $63.0 million in the third quarter of 2018, reflecting continued investments in the Galafold launch, Pompe clinical study program, and our gene therapy pipeline. 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 $61.8 million, or $0.24 per share, compared to a net loss of $159.2 million, or $0.84 per share, for the third quarter 2018.2019 Financial GuidanceFollowing the success in the first three quarters of the year, in addition to the strength in global Galafold launch metrics across all major geographies, Amicus raised the lower end of the full-year 2019 Galafold revenue guidance from $160 to $180 million to $170 to $180 million. The Company anticipates full-year 2019 non-GAAP operating expense of $410 million to $420 million. 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 $514.2 million at September 30, 2019. Following a diligent review of current and outer year operating and capital expense projections, and robust outlook for Galafold revenue, Amicus now expects to end 2019 with more than $420 million in cash on hand and has extended the cash runway projection from 2021 to well into the first half of 2022.2019 Key Strategic PrioritiesNearly double annual worldwide revenue for Galafold with over 1,000 Fabry patients on Galafold by year end.Complete enrollment in pivotal Phase 3 PROPEL clinical study in Pompe disease and report additional Phase 2 data.Report additional two-year results from Phase 1/2 clinical study in CLN6 Batten disease and complete enrollment in ongoing CLN3 Batten disease Phase 1/2 study.Establish preclinical proof of concept for Fabry and Pompe gene therapies.Maintain a strong financial position.Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, November 11, 2019 at 8:30 a.m. ET to discuss the third quarter 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: 8654236.
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