CRANBURY, N.J., Nov. 05, 2020 (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 third quarter ended September 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 third 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 commercial launch, Pompe late-stage development program, and advancing our industry-leading gene therapy pipeline. 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 third quarter of 2020 was $67.4M. Third quarter revenue represented a year-over-year increase of 38% from total revenue of $48.8 million in the third quarter of 2019. On a constant currency basis, third quarter 2020 total revenue was $65.7 million, representing operational revenue growth measured at constant currency exchange rates of 35%, which was further benefited by a positive currency impact of $1.7 million, or 3%. Galafold performance was driven largely by strong patient demand. Global compliance and adherence rates continue to exceed 90%.
• Global Phase 3 PROPEL clinical study of AT-GAA in late-onset Pompe disease (LOPD) on track for top-line data in 1Q21. To date, 97%+ of the 3,100+ 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 fourth quarter of 2020, completing final submission in the first half of 2021.
• To be highlighted on today’s call, new natural history data from the Amicus POM-002 chart review study in people living with LOPD treated long-term with current standard of care, alglucosidase alfa. Data are consistent with the medical literature and further supports PROPEL design assumptions.
• Additional Phase 1/2 CLN6 data presented at the Child Neurology Society Annual Meeting in October. The data shows a meaningful effect in slowing disease progression out to 24 months compared to natural history. Regulatory interactions are ongoing, and the Company expects to provide feedback on the path forward in 2021.
• Initial data from the Phase 1/2 CLN3 study expected in early 2021. Regulatory interactions are ongoing and the Company expects to provide feedback on the path forward in 2021.
• A gene therapy clinical candidate has been selected for IND enabling studies in Fabry Disease. Initial data from the Company’s AAV gene therapy with an engineered GLA transgene demonstrated significantly better GL-3 reduction than one with wild-type GLA. Full set of preclinical data to be presented at a medical conference in early 2021.
• 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 and advancing development programs.

Third Quarter 2020 Financial Results

• Total revenue in the third quarter 2020 was $67.4 million, a year-over-year increase of 38% from total revenue of $48.8 million in the third quarter of 2019. On a constant currency basis, third quarter 2020 total revenue was $65.7 million, representing operational revenue growth measured at constant currency exchange rates of 35%. Reported revenue was aided by a positive currency impact of $1.7 million, or 3%.
• Cash, cash equivalents, and marketable securities totaled $509.1 million at September 30, 2020, compared to $452.7 million at December 31, 2019.
• Total GAAP operating expenses of $111.8 million for the third quarter 2020 increased as compared to $100.5 million for the third quarter 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs.
• Total non-GAAP operating expenses of $92.4 million for the third quarter of 2020 increased as compared to $89.7 million in the third quarter of 2019, reflecting continued investments in our pipeline offset by decreased travel and third-party costs.¹
• Net loss was $64.0 million, or $0.25 per share, compared to a net loss of $61.8 million, or $0.24 per share, for the third quarter 2019. The third quarter 2020 net loss included an impact of $7.3 million relating to extinguishment of debt.

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.

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 $509.1 million at September 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 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 Milestones by Program

Galafold (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 underway

AT-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
• Additional supportive studies, including an open-label study in 12- to <18-year-olds living with Pompe

Gene Therapy Portfolio

• Advance regulatory discussions to finalize clinical and regulatory path for the CLN6 Batten disease gene therapy development program
• Initial data from the CLN3 Batten disease Phase 1/2 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
• Manufacturing advancements across portfolio

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.

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.