2020 Galafold Acquirement Admonition of $250M-$260M
Focused on Pompe Phase 3 PROPEL abstraction and accomplishment to abutment 2021 BLA and MAA
Continued Advance Beyond Industry Arch Attenuate Ache Gene Analysis Portfolio
Strong Balance Sheet with $450M Banknote – Banknote Runway Able-bodied into 2022
CRANBURY, N.J., Jan. 13, 2020 (GLOBE NEWSWIRE) — Amicus Therapeutics (FOLD), a global, accommodating axial biotechnology aggregation focused on discovering, developing and carrying atypical medicines for attenuate diseases, today provided its full-year 2020 cardinal angle and banking guidance.
Over the beforehand of 2019, Amicus essentially met or exceeded its bristles key cardinal priorities:
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “During 2019, Amicus connected our adventure of acceptable a arch all-around attenuate ache company. Afterward connected drive and able acceptance beyond the apple for our Fabry attention anesthetic Galafold, we accept exceeded our 2019 guidance. Patients are additionally now actuality brash in assorted Amicus analytic studies, including our Phase 1/2 and Phase 3 abstraction for AT-GAA in Pompe disease, as able-bodied as Phase 1/2 studies of our investigational gene therapies for CLN3 and CLN6 Batten disease. Our aboriginal analytic abstracts from our gene analysis action appropriate that our CLN6 gene analysis has the abeyant to balance progression in this adverse adolescence disease. In addition, our preclinical gene analysis assignment with our ally at the University of Pennsylvania gives us amazing affiance in the adeptness to beforehand abutting bearing gene therapies for the abounding of patients alive with a attenuate disease. Amicus is in a stronger position than anytime and charcoal focused on transforming the lives of bodies alive with these rare, life-threatening altitude and creating cogent amount for our shareholders.”
Amicus is focused on the afterward bristles key cardinal priorities in 2020:
Mr. Crowley will altercate Amicus’ accumulated objectives and key milestones in a presentation at the 38th Anniversary J.P. Morgan Healthcare Conference on Tuesday, January 14, 2020 at 8:30 a.m. PT (11:30 a.m. ET). A alive webcast of the presentation can be accessed through the Investors area of the Amicus Therapeutics accumulated web armpit at http://ir.amicusrx.com/events.cfm, and will be archived for 90 days.
Full-Year 2019 Banking Summary and 2020 Admonition
Amicus ahead appear full-year 2019 acquirement of about $181 actor (preliminary and unaudited) from bartering sales of Galafold.
For the full-year 2020, the Aggregation anticipates absolute Galafold acquirement of $250 actor to $260 actor based on the boilerplate barter ante for 2019. Beforehand in 2020 is accepted to be apprenticed by connected beforehand in EU markets, added success from launches in the U.S. and Japan, as able-bodied as agreement in added markets. Non-GAAP operating amount admonition for the year is $410 actor to $420 million, apprenticed by connected beforehand in the all-around Galafold launch, AT-GAA analytic studies, and advancing our gene analysis pipeline.
Cash, banknote equivalents, and bankable balance totaled over $450 actor (preliminary and unaudited) at December 31, 2019. The accepted banknote position is advancing to armamentarium advancing operations able-bodied into 2022.
Galafold (Migalastat) Articulate Attention Anesthetic for Fabry DiseaseGalafold is an articulate attention anesthetic for the analysis of Fabry ache in adults who accept amenable GLA variants and is accustomed in over 40 countries about the world, including the U.S., EU, Japan and others.
Global Galafold Updates:
AT-GAA for Pompe DiseaseAT-GAA is an investigational analysis in Phase 3 development that consists of cipaglucosidase alfa (ATB200), a altered recombinant animal acerbic alpha-glucosidase (rhGAA) agitator with optimized carbohydrate structures, decidedly mannose 6-phosphate (M6P), to enhance uptake into cells, co-administered with miglustat (AT2221), a pharmacological chaperone. Positive after-effects from a all-around Phase 1/2 analytic abstraction (ATB200-02) accept apparent constant and abiding responses beyond key measures of safety, anatomic outcomes and biomarkers in both ERT-switch and ERT-naïve Pompe patients afterward up to 24 months of analysis with AT-GAA.
Pompe Affairs Updates:
Anticipated Pompe Affairs Milestones in 2020:
Gene Analysis PortfolioAmicus has accustomed an industry arch gene analysis portfolio of abeyant therapies for bodies alive with attenuate diseases, through a authorization with Nationwide Children’s Hospital and an broadcast accord with the University of Pennsylvania. Our action includes gene analysis programs in rare, neurologic lysosomal disorders, specifically: CLN6, CLN3, CLN8 and CLN1 Batten disease, Pompe disease, Fabry disease, CDKL5 absence disorder, Niemann-Pick Type C, Mucopolysaccharidosis Type IIIB, as able-bodied as a abutting bearing affairs in Mucopolysaccharidosis Type IIIA. The broadcast accord with Penn additionally provides us with absolute disease-specific admission and the advantage rights to beforehand potentially confusing new gene analysis belvedere technologies and programs for best lysosomal disorders and a broader portfolio of attenuate diseases, including Rett Syndrome, Angelman Syndrome, Myotonic Dystrophy, and baddest added able-bodied dystrophies.
Gene Analysis Affairs Updates:
Anticipated Gene Analysis Action Milestones in 2020:
About Galafold Galafold® (migalastat) 123 mg capsules is an articulate pharmacological babysitter of alpha-Galactosidase A (alpha-Gal A) for the analysis of Fabry ache in adults who accept amenable GLA variants. In these patients, Galafold works by stabilizing the body’s own abortive agitator so that it can bright the accession of ache substrate. Globally, Amicus Therapeutics estimates that about 35 to 50 percent of Fabry patients may accept amenable GLA variants, admitting acquiescence ante aural this ambit alter by geography. Galafold is accustomed in over 40 countries about the world, including the U.S., EU, Japan and others.
U. S. INDICATIONS AND USAGEGalafold is adumbrated for the analysis of adults with a accepted analysis of Fabry ache and an acquiescent galactosidase alpha gene (GLA) alternative based on in vitro assay data.
This adumbration is accustomed beneath accelerated approval based on abridgement in branch interstitial capillary corpuscle globotriaosylceramide (KIC GL-3) substrate. Connected approval for this adumbration may be accidental aloft analysis and description of analytic account in acknowledging trials.
U.S. IMPORTANT SAFETY INFORMATION
ADVERSE REACTIONSThe best accepted adverse reactions appear with Galafold (≥10%) were headache, nasopharyngitis, urinary amplitude infection, abhorrence and pyrexia.
USE IN SPECIFIC POPULATIONSThere is bereft analytic abstracts on Galafold use in abundant women to acquaint a drug-associated accident for above bearing defects and miscarriage. Advise women of the abeyant accident to a fetus.
It is not accepted if Galafold is present in animal milk. Therefore, the adorning and bloom allowances of breastfeeding should be brash alternating with the mother’s analytic charge for Galafold and any abeyant adverse furnishings on the breastfed adolescent from Galafold or from the basal affectionate condition.
Galafold is not recommended for use in patients with astringent renal crime or end-stage renal ache acute dialysis.
The assurance and capability of Galafold accept not been accustomed in pediatric patients.
To address Suspected Adverse Reactions, acquaintance Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For added admonition about Galafold, including the abounding U.S. Prescribing Information, amuse visit https://www.amicusrx.com/pi/Galafold.pdf.
EU Important Assurance InformationTreatment with Galafold should be accomplished and supervised by specialists accomplished in the analysis and analysis of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
For added important assurance admonition for Galafold, including posology and adjustment of administration, appropriate warnings, biologic interactions and adverse biologic reactions, amuse see the European SmPC for Galafold accessible from the EMA website at www.ema.europa.eu.
About Fabry Disease
Fabry ache is an affiliated lysosomal ataxia acquired by absence of an agitator alleged alpha-galactosidase A (alpha-Gal A), which after-effects from mutations in the GLA gene. The primary biological action of alpha-Gal A is to abase specific lipids in lysosomes, including globotriaosylceramide (referred to actuality as GL-3 and additionally accepted as Gb3). Lipids that can be base by the action of alpha-Gal A are alleged “substrates” of the enzyme. Reduced or absent levels of alpha-Gal A action advance to the accession of GL-3 in the afflicted tissues, including heart, kidneys, and skin. Accession of GL-3 and accelerating abasement of agency action is believed to advance to the anguish and bloodshed of Fabry disease. The affection can be severe, alter from being to person, and activate at an aboriginal age.
About Amicus Therapeutics
Amicus Therapeutics (FOLD) is a global, patient-dedicated biotechnology aggregation focused on discovering, developing and carrying atypical high-quality medicines for bodies alive with attenuate metabolic diseases. With amazing accommodating focus, Amicus Therapeutics is committed to advancing and accretion a able-bodied action of cutting-edge, first- or best-in-class medicines for attenuate metabolic diseases. For added admonition amuse appointment the company’s website at www.amicusrx.com, and chase us on Twitter and LinkedIn.
Forward Looking Statement
This columnist absolution contains “forward-looking statements” aural the acceptation of the Private Balance Litigation Reform Act of 1995 apropos to preclinical and analytic development of our artefact candidates, the timing and advertisement of after-effects from preclinical studies and analytic trials, the affairs and timing of the abeyant authoritative approval of our artefact candidates, commercialization plans, accomplishment and accumulation plans, costs plans, business development affairs and the projected revenues, sales, costs and banknote position for the Company. The admittance of advanced statements should not be admired as a representation by us that any of our affairs or projections will be achieved. Any or all of the advanced statements in this columnist absolution may about-face out to be amiss and can be afflicted by inaccurate assumptions we adeptness accomplish or by accepted or alien risks and uncertainties. For example, with account to statements apropos the goals, progress, timing, and outcomes of discussions with authoritative authorities, and in accurate the abeyant goals, progress, timing, and after-effects of preclinical studies and analytic trials, absolute after-effects may alter materially from those set alternating in this absolution due to the risks and uncertainties inherent in our business, including, afterwards limitation: the abeyant that after-effects of analytic or preclinical studies announce that the artefact candidates are alarming or ineffective; the abeyant that it may be difficult to accept patients in our analytic trials; the abeyant that authoritative authorities, including the FDA, EMA, and PMDA, may not admission or may adjournment approval for our artefact candidates; the abeyant that we may not be acknowledged in commercializing Galafold in Europe, Japan, the US and added geographies or our added artefact candidates if and back approved; the abeyant that preclinical and analytic studies could be delayed because we analyze austere ancillary furnishings or added assurance issues; the abeyant that we may not be able to accomplish or accumulation acceptable analytic or bartering products; and the abeyant that we will charge added allotment to complete all of our studies and manufacturing. Further, the after-effects of beforehand preclinical studies and/or analytic trials may not be predictive of approaching results. With account to statements apropos projections of the Company’s revenue, sales, costs and banknote position, absolute after-effects may alter based on bazaar factors and the Company’s adeptness to assassinate its operational and account affairs and strategies. In addition, all advanced statements are accountable to added risks abundant in our Anniversary Address on Form 10-K for the year concluded December 31, 2018. You are cautioned not to abode disproportionate assurance on these advanced statements, which allege alone as of the date hereof. All advanced statements are able in their absoluteness by this cautionary statement, and we undertake no obligation to alter or amend this columnist absolution to reflect contest or affairs afterwards the date hereof.
Non-GAAP Banking Measures
In accession to banking admonition able in accordance with U.S. GAAP, this presentation additionally contains adapted banking measures that we accept accommodate investors and administration with added admonition apropos to operating achievement and trends that facilitate comparisons amid periods and with account to projected information. These adapted banking measures are non-GAAP measures and should be brash in accession to, but not as a acting for, the admonition able in accordance with U.S. GAAP. We about exclude assertive GAAP items that administration does not accept affect our basal operations and that do not accommodated the GAAP analogue of abnormal or non-recurring items. Added companies may ascertain these measures in altered ways. Back we accommodate our apprehension for non-GAAP operating costs on a advanced basis, a adaptation of the differences amid the non-GAAP apprehension and the agnate GAAP admeasurement about is not accessible afterwards absurd accomplishment due to potentially aerial variability, complication and low afterimage as to the items that would be afar from the GAAP admeasurement in the accordant approaching period, such as abnormal assets or losses. The airheadedness of the afar items may accept a significant, and potentially unpredictable, appulse on our approaching GAAP results.
Andrew FaughnanDirector, Investor [email protected] (609) 662-3809
Media:Christopher ByrneExecutive Director, Accumulated [email protected] (609) 662-2798
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