Amicus Therapeutics Announces Second Quarter 2019 Financial Results and Corporate Updates.
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Release date- 08082019 - CRANBURY - 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 millionand 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 millionin 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.
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.
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.
Non-GAAP Financial Measures
Operational revenue growth excluding the impact of foreign currency exchange rates is a non-GAAP financial measure and should not be considered a replacement for, and should be read together with, the most comparable GAAP financial measure.
This press release contains 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995 relating to preclinical and clinical development of our product candidates, the timing and reporting of results from preclinical studies and clinical trials, the prospects and timing of the potential regulatory approval of our product candidates, commercialization plans, manufacturing and supply plans, financing plans, and the projected revenues and cash position for the Company. The inclusion of forward-looking statements should not be regarded as a representation by us that any of our plans will be achieved. Any or all of the forward-looking statements in this press release may turn out to be wrong and can be affected by inaccurate assumptions we might make or by known or unknown risks and uncertainties. For example, with respect to statements regarding the goals, progress, timing, and outcomes of discussions with regulatory authorities, and in particular the potential goals, progress, timing, and results of preclinical studies and clinical trials, actual results may differ materially from those set forth in this release due to the risks and uncertainties inherent in our business, including, without limitation: the potential that results of clinical or preclinical studies indicate that the product candidates are unsafe or ineffective; the potential that it may be difficult to enroll patients in our clinical trials; the potential that regulatory authorities, including the FDA, EMA, and PMDA, may not grant or may delay approval for our product candidates; the potential that we may not be successful in commercializing Galafold in Europe, Japan, the US and other geographies or our other product candidates if and when approved; the potential that preclinical and clinical studies could be delayed because we identify serious side effects or other safety issues; the potential that we may not be able to manufacture or supply sufficient clinical or commercial products and the potential that we will need additional funding to complete all of our studies and manufacturing. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results. With respect to statements regarding projections of the Company's revenue and cash position, actual results may differ based on market factors and the Company's ability to execute its operational and budget plans. In addition, all forward-looking statements are subject to other risks detailed in our Annual Report on Form 10-K for the year ended December 31, 2018. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, and we undertake no obligation to revise or update this news release to reflect events or circumstances after the date hereof.
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|Date:||Aug 9, 2019|
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