CRANBURY, N.J., Aug. 08, 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 second quarter ended June 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, “2019 has been a great period of growth and achievement at Amicus as we continue to build a leading global rare disease biotechnology company. We are on track to meet or exceed all of our key strategic priorities for the year - continued success for Galafold, tremendous momentum in our Phase 3 Pompe clinical study, positive interim Phase 1/2 clinical data for our first gene therapy for CLN6 Batten disease, and an industry leading rare disease gene therapy portfolio. We are in a very strong position to deliver on our mission for patients and shareholders.”

Corporate Highlights for 2Q19 and Early 3Q19

• Global revenue for Fabry precision medicine Galafold in the second quarter of 2019 was $44.1 million and continues to track toward full-year 2019 revenue guidance of $160 million to $180 million. Now expecting to exceed 1,000 patients on therapy well before year-end. Second quarter revenue represented a year-over-year increase of 107% from total revenue of $21.3 million in the second quarter of 2018, and a quarter over-quarter increase of 30% from total revenue of $34.0 million in the first quarter of 2019. As of June 30, 2019, Galafold represented an estimated 24% of global market share of treated amenable patients. Global compliance and adherence rates continue to exceed 90%.
• Enrollment momentum in ongoing pivotal PROPEL study in Pompe disease at participating sites in 29 countries. This global study at the leading Pompe centers in the world is on track to achieve full enrollment of ~100 patients by year-end 2019. The study is now more than a majority enrolled. The Pompe patient and physician community around the world continues to show strong interest in AT-GAA, which has U.S. Breakthrough Therapy Designation, as the next potential standard of care.
• Positive interim Phase 1/2 clinical data for gene therapy in CLN6 Batten disease. The results for our AAV-CLN6 gene therapy showed a positive impact on motor and language function in seven of eight patients with available efficacy data (treated from 16 to 25 months), compared to a natural history dataset, as well as in comparisons within sibling pairs.
• Strategic partnerships with Catalent Biologics and Thermo Fisher for clinical and commercial gene therapy manufacturing. Brammer Bio, a part of Thermo Fisher, is assisting with clinical and commercial-scale capabilities for Amicus gene therapy programs in CLN6 and additional forms of Batten disease. Paragon Gene Therapy, a unit of Catalent Biologics, will support clinical manufacturing capabilities and capacity for multiple active preclinical lysosomal disorder programs in collaboration with Penn.
• Major expansion of gene therapy collaboration with the Perelman School of Medicine at the University of Pennsylvania (Penn). Amicus now has exclusive worldwide rights to Penn’s next generation gene therapy technologies for a majority of lysosomal disorders and 12 additional rare diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy and select other muscular dystrophies.
• Positive initial proof-of-concept for preclinical Pompe gene therapy data at American Society of Cell and Gene Therapy meeting. Positive preclinical results showed robust uptake and glycogen reduction in multiple tissues, including brain and spinal cord, and were a key driver for the expanded Amicus-Penn collaboration. Additional Pompe preclinical studies and selection of a clinical candidate are on track in 2019.
• Exclusive license for Japanese rights to Ultragenyx’s (Nasdaq: RARE) Mepsevii™ (vestronidase alfa), an enzyme replacement therapy (ERT) for the lysosomal disorder Mucopolysaccharidosis type VII (MPS VII, Sly syndrome). Amicus will leverage its existing infrastructure, relationships, and experience in clinical development, regulatory approvals, and commercialization in Japan. Mepsevii^™ is currently approved for the treatment of children and adults with MPS VII in the U.S., EU, and Brazil. Amicus plans to use the existing Mepsevii data package, as well as data from an ongoing investigator-sponsored trial in Japan, to support a J-NDA submission.
• Strong financial position to continue executing the Galafold launch, advance development programs and build leadership position in gene therapy science, development and manufacturing capabilities and capacity. The current cash position of $575.7 million at June 30, 2019, which includes $189.0 million in net proceeds raised from our June 2019 public offering of common stock, is expected to fund ongoing operations into 2021.

2Q19 Financial Results

• Total revenue in the second quarter 2019 was $44.1 million, a year-over-year increase of 107% from total revenue of $21.3 million in the second quarter of 2018, and a quarter over-quarter increase of 30% from total revenue of $34.0 million in the first quarter of 2019. This includes year-over-year operational revenue growth measured at constant currency exchange rates of 115%, offset by a negative currency impact of $1.7M, or 8%.
• Cash, cash equivalents, and marketable securities totaled $575.7 million at June 30, 2019, compared to $504.2 million at December 31, 2018.
• Total operating expenses of $115.2 million for the second quarter of 2019 increased as compared to $65.1 million in the second quarter of 2018, reflecting continued investments in the Galafold launch, Pompe clinical study program, and our gene therapy pipeline.
• Net loss was $84.6 million, or $0.36 per share, compared to a net loss of $61.8 million, or $0.33 per share, for the second quarter 2018.

2019 Key Strategic Priorities

• Nearly double annual worldwide revenue for Galafold (FY19 guidance of $160 million to $180 million) 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.

2019 Financial Guidance

For the full-year 2019 the Company anticipates total Galafold revenue of $160 million to $180 million. Growth in 2019 is expected to be driven by continued growth in EU markets, further geographic expansion, and successful adoption in the first full year of launch in the U.S. and Japan. For the remainder of the year, consistent with Galafold adoption trends and ordering patterns seen in previous years, Amicus expects flat to moderate revenue growth in the third quarter and higher revenue growth in the fourth quarter of 2019.

Cash, cash equivalents, and marketable securities totaled $575.7 million at June 30, 2019. The Company expects to end 2019 with approximately $400 million in cash on hand. The current cash position is anticipated to fund ongoing operations into 2021.

Anticipated 2019 Milestones by Program

Galafold Oral Precision Medicine for Fabry Disease

• Continued growth in global market share of treated amenable patients.
• Geographic expansion following recent approval in Argentina
• On track to meet full-year 2019 revenue guidance range of $160 million to $180 million.
• Target of 1,000+ patients on Galafold before year-end.

AT-GAA for Pompe Disease

• Initial 6-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, October 1-5, 2019 (Copenhagen, Denmark)
• Retrospective natural history study data in approximately 100 ERT-treated Pompe patients.
• Additional supportive studies, including an open-label study in pediatric patients.
• Full enrollment in Phase 3 PROPEL clinical study.
• Advance agreed upon CMC requirements to support BLA.

Gene Therapy Pipeline:

• Detailed data presentation of interim clinical results in CLN6 Batten disease at Amicus Analyst Day on October 10, 2019 (New York, NY) and in a poster at Child Neurology Society Annual Meeting, October 23-26, 2019 (Charlotte, NC).
• Advancement of clinical, regulatory and manufacturing activities for CLN6 Batten disease
• High dose cohort on track to commence in Phase 1/2 clinical study of CLN3 Batten disease with full enrollment by year end.
• Additional preclinical data including next-generation gene therapies for Fabry and Pompe.
• Continued advancement of preclinical gene therapy programs in CLN8 and CLN1 Batten disease.
• Selection of Pompe AAV gene therapy clinical candidate to move into IND-enabling studies.
• Selection of Amicus site for late process development and manufacturing facilities.

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 Argentina, Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland and the U.S.