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U.S. Galafold® (Migalastat) Fabry Launch Tracking Significantly Ahead of Expectations- 100+ Patients Prescribed Galafold Since August Launch
3Q18 Global Galafold Net Product Sales of $20.6M Driven by Continued International Growth - 500+ International Fabry Patients Now on Galafold
Reaffirming Higher End of FY18 Revenue Guidance of $80M-$90M –
Balance Sheet Strength Sufficient to Fund Operations into at least 2021
Gene Therapy Pipeline Provides 14 New Programs and Future Growth Platform
Conference Call and Webcast Today at 8:30am ET
CRANBURY, N.J., Nov. 05, 2018 (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, 2018. The Company also summarized recent program updates, reiterated its full-year 2018 revenue guidance and reduced its net cash spend guidance for the year.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “The third quarter marked a major transformation for Amicus that brings us several steps closer toward our 2023 vision to treat at least 5,000 patients and to achieve $1 billion in annual global revenue. In the last three months, we received our first U.S. drug approval for Galafold, expanded our pipeline to include 14 new gene therapy programs for rare metabolic diseases, and presented positive 18-month data for our differentiated Pompe treatment paradigm. With a strong revenue base and $500M+ peak sales opportunity for Galafold, as well as $564 million in cash, we have never been in a stronger position to advance our robust portfolio to drive significant value for shareholders and the patient communities that we serve.”
Third Quarter 2018 Financial Results and Full-Year 2018 Financial Guidance
“We are very pleased with the momentum of the global Galafold launch,” said Bradley L. Campbell, President and Chief Operating Officer of Amicus Therapeutics. “We see continued strong uptake and growth, along with very high compliance and adherence to this new oral Fabry treatment option, in both patients who are ERT-experienced and an increasing number who are ERT-treatment naïve in Europe. As anticipated, quarter-over-quarter revenue reflect some uneven ordering patterns as well as a rising number of extended, 90-day prescriptions as we headed into the summer months, which have normalized since the start of the fourth quarter. These adoption trends may reflect the emerging Fabry treatment paradigm with a stable oral medication that can be taken during summer travels and on holidays. Japan is also off to a solid start, now with a double-digit number of patient prescriptions. Importantly, the first 12 weeks of the U.S. launch have significantly exceeded our expectations. With more than 100 individual prescriptions for Galafold in the U.S., we are seeing robust patient demand from a broad prescriber base of more than 40 Fabry physicians. Given this global momentum, we are confident in meeting the higher end of our full-year 2018 guidance and setting a solid foundation for 2019.”
2018 Financial Guidance
For the full-year 2018 the Company reiterated its total Galafold revenue guidance to $80 to $90 million. This reflects global revenue from all expected 2018 commercial markets. The Company is lowering its full-year 2018 net cash spend to $190 to $210 million from the previous range of $220 to $250 million. The current cash position, including Galafold revenues, is sufficient to fund ongoing operations into at least 2021. Potential future business development collaborations, pipeline expansion, and investment in manufacturing capabilities could impact the Company’s future capital requirements.
Galafold (Migalastat) Oral Precision Medicine for Fabry Disease
Galafold is an oral precision medicine for Fabry disease approved in the EU and other geographies to treat Fabry disease in patients 16 years or older who have amenable genetic mutations. The U.S. FDA approved Galafold under Subpart H for the treatment of adult patients with a confirmed diagnosis of Fabry disease and an amenable genetic variant. For patients who are not suitable for treatment with Galafold on the basis of their genetic mutations, or variants, Amicus is advancing a next-generation gene therapy.
Global Galafold Updates:
AT-GAA for Pompe Disease
AT-GAA is a novel treatment paradigm that consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose 6-phosphate (M6P), to enhance uptake, co-administered with AT2221, a pharmacological chaperone to stabilize ATB200 while in the circulation to deliver active therapeutic enzyme.
Positive results from a global Phase 1/2 clinical study (ATB200-02) have shown consistent and durable responses across key measures of safety, functional outcomes and biomarkers in both ERT-switch and ERT-naïve Pompe patients following up to 18 months of treatment with AT-GAA. The Company’s strategy is to enhance the body of clinical data for AT-GAA in ongoing studies and the upcoming pivotal study (PROPEL, also referred to as ATB200-03) to deliver this potential new therapy to as many people living with Pompe disease as soon as possible.
Recent and Anticipated AT-GAA Program Milestones:
As part of the Company’s long-term commitment to provide multiple solutions to address the significant unmet needs of the Pompe community, Amicus is also advancing a next-generation gene therapy as a potential cure for Pompe disease.
Gene Therapy Portfolio: 14 New Programs for Rare Metabolic Diseases
During the third quarter and early fourth quarter, Amicus expanded its pipeline [link here] to include 14 new gene therapy programs and future growth platform for rare metabolic diseases. The Company acquired 10 preclinical and clinical stage adeno associated virus (AAV) programs (intrathecal delivery) for neurologic lysosomal storage disorders (LSDs) currently in development at Nationwide Children’s Hospital. In collaboration with the University of Pennsylvania, the Company is advancing four next-generation AAV gene therapies for Fabry disease, Pompe disease, CDKL5 deficiency disorder (CDD) and one additional undisclosed rare metabolic disorder.
Gene Therapy Pipeline Highlights:
Upcoming Gene Therapy Pipeline Milestones in 2018 and 2019:
Conference Call and Webcast
Amicus Therapeutics will host a conference call and audio webcast today, November 5, 2018, at 8:30 a.m. ET to discuss the third quarter 2018 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: 2292105.
An audio webcast and slide presentation can also be accessed via the Investors section of the Amicus Therapeutics corporate website at http://ir.amicusrx.com/ and will be archived for 30 days. Web participants are encouraged to go to the website 15 minutes prior to the start of the call to register, download, and install any necessary software. A telephonic replay of the call will be available for seven days beginning at 11:30 a.m. ET on November 5, 2018. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 2292105.
GalafoldTM (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 Australia, Canada, European Union, Israel, Japan, South Korea, Switzerland and the U.S.
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.
U.S. IMPORTANT SAFETY INFORMATION
The most common adverse reactions reported with Galafold (≥10%) were headache, nasopharyngitis, urinary tract infection, nausea and pyrexia.
USE IN SPECIFIC POPULATIONS
There is insufficient clinical data on Galafold use in pregnant women to inform a drug-associated risk for major birth defects and miscarriage. Advise women of the potential risk to a fetus.
It is not known if Galafold is present in human milk. Therefore, the developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for Galafold and any potential adverse effects on the breastfed child from Galafold or from the underlying maternal condition.
Galafold is not recommended for use in patients with severe renal impairment or end-stage renal disease requiring dialysis.
The safety and effectiveness of Galafold have not been established in pediatric patients.
To report Suspected Adverse Reactions, contact Amicus Therapeutics at 1-877-4AMICUS or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
For additional information about Galafold, including the full U.S. Prescribing Information, please visit https://www.amicusrx.com/pi/Galafold.pdf.
EU Important Safety Information
Treatment with Galafold should be initiated and supervised by specialists experienced in the diagnosis and treatment of Fabry disease. Galafold is not recommended for use in patients with a nonamenable mutation.
For further important safety information for Galafold, including posology and method of administration, special warnings, drug interactions and adverse drug reactions, please see the European SmPC for Galafold available from the EMA website at www.ema.europa.eu.
About Amicus Therapeutics
Amicus Therapeutics (Nasdaq: FOLD) is a global, patient-centric 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 us on Twitter and LinkedIn.
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 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, 2017 as well as our Quarterly Report on Form 10-Q for the quarter September 30, 2018 to be filed November 6, 2018 with the Securities and Exchange Commission. 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.
Sara Pellegrino, IRC
Vice President, Investor Relations & Corporate Communications
Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
|Three Months Ended September 30,||Nine Months Ended September 30,|
|Net product sales||$||20,596||$||10,874||$||58,601||$||22,201|
|Cost of goods sold||4,310||1,790||10,060||3,626|
|Research and development||138,227||40,641||213,685||103,502|
|Selling, general and administrative||31,867||21,647||88,435||60,090|
|Changes in fair value of contingent consideration payable||1,300||(244,250||)||2,700||(238,622||)|
|Loss on impairment of assets||—||465,427||—||465,427|
|Total operating expenses||172,467||284,316||307,835||392,883|
|Loss from operations||(156,181||)||(275,232||)||(259,294||)||(374,308||)|
|Other income (expense):|
|Change in fair value of derivatives||—||—||(2,739||)||163|
|Other (expense) income||(1,039||)||2,044||(3,593||)||4,891|
|Loss before income tax||(159,214||)||(276,349||)||(272,018||)||(379,372||)|
|Income tax benefit||51||164,683||1,104||164,578|
|Net loss attributable to common stockholders||$||(159,163||)||$||(111,666||)||$||(270,914||)||$||(214,794||)|
|Net loss attributable to common stockholders per common share — basic and diluted||$||(0.84||)||$||(0.69||)||$||(1.47||)||$||(1.44||)|
|Weighted-average common shares outstanding — basic and diluted||189,162,841||160,796,841||184,606,790||148,963,864|
Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|September 30,||December 31,|
|Cash and cash equivalents||$||201,827||$||49,060|
|Investments in marketable securities||362,556||309,502|
|Prepaid expenses and other current assets||16,151||19,316|
|Total current assets||601,034||391,965|
|Property and equipment, less accumulated depreciation of $15,483 and $12,515 at September 30, 2018 and December 31, 2017, respectively||10,659||9,062|
|In-process research & development||23,000||23,000|
|Other non-current assets||6,099||5,200|
|Liabilities and Stockholders’ Equity|
|Accounts payable, accrued expenses, and other current liabilities||$||54,330||$||53,890|
|Contingent consideration payable||8,800||8,400|
|Total current liabilities||65,880||70,040|
|Senior secured term loan||146,622||—|
|Contingent consideration payable||19,300||17,000|
|Deferred income taxes||6,465||6,465|
|Other non-current liabilities||3,029||2,346|
|Commitments and contingencies|
|Common stock, $0.01 par value, 500,000,000 and 250,000,000 shares authorized, 189,254,341 and 166,989,790 shares issued and outstanding at September 30, 2018 and December 31, 2017, respectively||1,941||1,721|
|Additional paid-in capital||1,731,174||1,400,758|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment||(875||)||(1,659||)|
|Unrealized gain on available-for-sale securities||(211||)||(436||)|
|Total stockholders’ equity||410,951||352,850|
|Total Liabilities and Stockholders’ Equity||$||838,589||$||627,024|
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