Strong Momentum for Galafold Launch in Europe and Further Global Regulatory Submissions Initial Data from Pompe Phase 1/2 Clinical Study on Track for 4Q16 CRANBURY, N.J., Nov. 07, 2016 (GLOBE NEWSWIRE) -- Amicus Therapeutics (Nasdaq:FOLD), a global biotechnology company at the forefront of therapies for rare and orphan diseases, today announced financial results for the third quarter ended September 30, 2016. The Company also provided program updates and reiterated full-year 2016 net cash spend guidance. "During the third quarter we continued to execute toward our vision to build a leading global biotechnology company delivering meaningful benefits to people living with devastating rare diseases," stated John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. "We are most pleased with the continued strong momentum in the very early stages of our product launch in Europe. Fifty patients, most of whom have switched from existing approved ERT products, have been prescribed Galafold as of the end of October. The vast majority of these new Galafold patients are in Germany, which is the only nation in the EU where Galafold is fully commercially available. It is wonderful to be able to begin to offer a new therapeutic option to Fabry patients and physicians with our oral, precision small molecule medicine, Galafold. We also remain sharply focused on additional key strategic priorities, including: 1) further global regulatory submissions for migalastat, including a Japanese NDA and clarity on the optimal U.S. regulatory pathway; 2) the advancement of our clinical programs in Pompe and epidermolysis bullosa (EB); 3) a strong balance sheet, and 4) the expansion of our biologics pipeline. We continue to believe that we have one of the best portfolios within the rare and orphan diseases that is uniquely differentiated by our strong science, novel technology platforms, and our incorporation of the patient's perspective at every stage of the drug development process."
Third Quarter 2016 Financial Results
- Total product revenue in the third quarter of 2016 was approximately $2.1 million, which represents commercial sales of Galafold (migalastat) in Germany as well as reimbursed Expanded Access Programs (EAPs) in two countries.
- Cash, cash equivalents, and marketable securities totaled $212.4 million at September 30, 2016 compared to $214.2 million at June 30, 2016.
- Total operating expenses in the third quarter of 2016 increased to $46.7 million compared to $38.0 million for the third quarter 2015 primarily due to increases in commercial costs for the Fabry monotherapy program and the addition of the Phase 3 SD-101 program for EB.
- Net loss was $46.7 million, or $0.33 per share in the third quarter of 2016, compared to a net loss of $37.8 million, or $0.32 per share, for the third quarter of 2015.
- 50 patients (naïve and ERT-switch) on reimbursed Galafold as of October 31, 2016
- 5 countries with reimbursement (commercial or EAP)
- Reimbursement dossiers submitted and pricing discussions are now underway in 15 countries.
- Swissmedic in Switzerland approved Galafold for long-term treatment of adults and adolescents aged 16 years and older with a confirmed diagnosis of Fabry disease and who have an amenable mutation
- The Committee for Medicinal Products for Human Use (CHMP) agreed to 44 new amenable mutations and the EU label is being updated to include a total of 313 amenable mutations
- Regulatory submissions completed in six additional territories outside the EU
- EU commercial reimbursement and EAP in additional territories
- Regulatory submissions in additional territories that accept the marketing authorization application (MAA) as basis for submission
- U.S. regulatory update on optimal filing pathway for migalastat anticipated by year-end
- Fabry ERT cell line development and program update
- Japanese regulatory submission (J-NDA) targeted for 1H17 on accelerated timeline
- Data from clinical study ATB200-02 in first four ambulatory ERT-switch patients on track by year-end 2016
- Additional ATB200-02 study data in naïve and non-ambulatory patients, as well as initial extension-phase data on ambulatory ERT-switch patients, throughout 1H17
Anticipated EB Program Milestones:
- Top-line data from the Phase 3 ESSENCE study of SD-101 (1H17)
- GALAFOLD is not intended for concomitant use with enzyme replacement therapy.
- GALAFOLD is not recommended for use in patients with Fabry disease who have severe renal impairment (<30 mL/min/1.73 m 2). The safety and efficacy of GALAFOLD in children 0-15 years of age have not yet been established.
- No dosage adjustments are required in patients with hepatic impairment or in the elderly population.
- There is very limited experience with the use of this medicine in pregnant women. If you are pregnant, think you may be pregnant, or are planning to have a baby, do not take this medicine until you have checked with your doctor, pharmacist, or nurse.
- While taking GALAFOLD, effective birth control should be used. It is not known whether GALAFOLD is excreted in human milk.
- Contraindications to GALAFOLD include hypersensitivity to the active substance or to any of the excipients listed in the PRESCRIBING INFORMATION.
- It is advised to periodically monitor renal function, echocardiographic parameters and biochemical markers (every 6 months) in patients initiated on GALAFOLD or switched to GALAFOLD.
- OVERDOSE: General medical care is recommended in the case of GALAFOLD overdose.
- The most common adverse reaction reported was headache, which was experienced by approximately 10% of patients who received GALAFOLD. For a complete list of adverse reactions, please review the SUMMARY OF PRODUCT CHARACTERISTICS.
- Call your doctor for medical advice about side effects.
|Amicus Therapeutics, Inc.|
|Consolidated Statements of Operations|
|(in thousands, except share and per share amounts)|
|Three Months||Nine Months|
|Ended September 30,||Ended September 30,|
|Net product sales||$||2,127||$||-||$||2,127||$||-|
|Cost of goods sold||344||-||344||-|
|Research and development||32,457||20,971||74,163||54,318|
|Selling, general and administrative||17,469||15,372||52,470||30,077|
|Changes in fair value of contingent consideration payable||(4,110||)||1,300||9,228||2,400|
|Loss on extinguishment of debt||-||-||-||952|
|Total operating expenses||46,723||38,045||138,266||89,047|
|Loss from operations||(44,940||)||(38,045||)||(136,483||)||(89,047||)|
|Other income (expenses):|
|Loss before income tax benefit||(46,907||)||(37,800||)||(142,101||)||(89,222||)|
|Income tax benefit||253||-||706||-|
|Net loss per common share - basic and diluted||$||(0.33||)||$||(0.32||)||$||(1.07||)||$||(0.85||)|
|Weighted-average common shares outstanding - basic and diluted||140,656,109||118,724,882||131,675,690||104,885,956|
|Amicus Therapeutics, Inc.|
|Consolidated Balance Sheets|
|(in thousands, except share and per share amounts)|
|September 30,||December 31,|
|Cash and cash equivalents||$||33,115||$||69,485|
|Investments in marketable securities||179,284||144,548|
|Prepaid expenses and other current assets||5,198||2,568|
|Total current assets||221,712||216,601|
|Property and equipment, less accumulated depreciation of $15,181 and $13,353 at September 30, 2016 and December 31, 2015, respectively||10,183||6,178|
|In-process research & development||486,700||486,700|
|Other non-current assets||1,788||1,108|
|Liabilities and Stockholders' Equity|
|Accounts payable and accrued expenses||$||29,013||$||32,216|
|Contingent consideration payable, current portion||55,992||41,400|
|Other current liabilities||607||-|
|Total current liabilities||85,612||73,616|
|Due to related party||44,047||41,601|
|Unsecured notes payable||21,977||-|
|Contingent consideration payable, less current portion||216,198||232,677|
|Deferred tax liability||176,219||176,219|
|Other non-current liabilities||1,816||681|
|Commitments and contingencies|
|Common stock, $.01 par value, 250,000,000 authorized, 142,273,085 shares issued and outstanding at September 30, 2016, 250,000,000 shares authorized, 125,027,034 shares issued and outstanding at December 31, 2015||1,478||1,306|
|Additional paid-in capital||1,038,613||917,454|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment, less tax benefit of $706 at September 30, 2016||1,062||-|
|Unrealized gain/ (loss) on available-for securities||287||(115||)|
|Total stockholders' equity||336,555||347,834|
|Total Liabilities and Stockholders' Equity||$||918,180||$||908,384|
CONTACTS:Investors/Media:Amicus TherapeuticsSara PellegrinoSenior Director, Investor Relationsspellegrino@amicusrx.com(609) 662-5044Media:MWW PRSean Conleysconley@mww.com(646) 381-9096