2Q18 Net Product Sales of
Company Increases FY18 Global Galafold Revenue Guidance to
Pompe Clinical, Regulatory and Manufacturing Activities Continue to Advance
Conference Call and Webcast Today at
Second Quarter 2018 Financial Results and Full-Year 2018 Financial Guidance
- Total revenue in the second quarter 2018 was
$21.3 million, a year-over-year increase of 198% from total revenue of $7.2 millionin the second quarter of 2017.
- Cash, cash equivalents, and marketable securities totaled
$552.8 millionat June 30, 2018, compared to $358.6 millionat December 31, 2017.
- Total operating expenses increased to
$65.1 millionfor the second quarter 2018 compared to $53.2 millionin the second quarter 2017, reflecting an increased investment in Pompe clinical and manufacturing activities as well as Galafold commercial launch and launch preparations.
- Net cash spend was
$51.0 millionfor the second quarter 2018. Net loss was $61.8 million, or $0.33per share, for the second quarter 2018 compared to a net loss of $48.1 million, or $0.34per share, for the second quarter 2017.
“The second quarter of 2018 marked another period of continued growth for Galafold,” said
2018 Financial Guidance
For the full-year 2018 the Company is increasing its total Galafold revenue guidance to
Based on the increase in expected 2018 revenue and operating expenses that are trending favorably to budget, the Company is updating its full-year 2018 net cash spend to
Migalastat for Fabry Disease
Amicus is committed to advancing the highest quality therapies for all people living with Fabry disease. Migalastat, the Company’s first therapy, is an oral precision medicine. Regulatory authorities in
For people with non-amenable mutations who are not eligible for migalastat as an oral precision medicine, the strategy is to advance next-generation therapies such as a novel Fabry ERT (ATB101) co-formulated with migalastat or other innovative technologies that continue to be evaluated.
Global Fabry Updates:
- Pricing and reimbursement secured in 19 countries with first commercial patients treated in multiple new countries in 2018
- Approvals secured in
Australia, Canada, EU, Israel, Japan, South Koreaand Switzerland
- Commercial launch initiated in
Japanduring the second quarter
- U.S. leadership and field team now in place to support planned U.S. launch
FDAregulatory decision (3Q18)
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.
The Company is engaged in collaborative discussions with U.S. and EU regulators regarding a number of key topics including a registration-directed study for full approval, manufacturing activities, and the best and fastest pathway forward for this novel treatment regimen. The Company believes that the evolving regulatory paths in both the U.S. and EU will include a series of iterative discussions with regulators as the program advances and as additional data are collected, including data from existing patients in ongoing studies, data from new patients being enrolled into ongoing studies, and the results of a formal retrospective natural history study of Pompe patients receiving current standard of care ERT. Amicus expects to provide an
Manufacturing activities to support the needs of the Pompe community are also ongoing.
Pompe Manufacturing Updates:
- Release of 1,000L GMP material for use in the planned pivotal study.
- Feedback from German regulatory authorities (BfArM) indicating general agreement with the manufacturing strategy for ATB200, including the strategy to demonstrate comparability of drug substance and drug product between the 1,000 liter scale and the 250 liter scale.
Anticipated Pompe Program Milestones in 2H18:
- Pompe US regulatory update (3Q18).
- Completion of a retrospective natural history study in ~100 ERT-treated Pompe patients.
- 18-month data from ATB200-02 clinical study to be presented at the 23rd
International Congressof the World Muscle Society(4Q18).
- Commence pivotal study in 2H18.
Conference Call and Webcast
An audio webcast can also be accessed via the Investors section of the
Non-GAAP Financial Measures
In addition to
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.
- 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 m2). 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.
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.
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
Associate Director, Investor Relations
|Amicus Therapeutics, Inc.
Consolidated Statements of Operations
(in thousands, except share and per share amounts)
|Three Months Ended June 30,||Six Months Ended June 30,|
|Net product sales||$||21,309||$||7,158||$||38,005||$||11,327|
|Cost of goods sold||3,135||1,061||5,750||1,836|
|Research and development||34,660||31,985||75,458||62,861|
|Selling, general and administrative||29,172||19,311||56,568||38,443|
|Changes in fair value of contingent consideration payable||300||1,050||1,400||5,628|
|Total operating expenses||65,105||53,158||135,368||108,568|
|Loss from operations||(46,931||)||(47,061||)||(103,113||)||(99,077||)|
|Other income (expense):|
|Change in fair value of derivatives||(7,600||)||—||(2,739||)||—|
|Other (expense) income||(5,316||)||2,400||(2,554||)||3,010|
|Loss before income tax||(61,494||)||(48,087||)||(112,804||)||(103,024||)|
|Income tax benefit (expense)||(339||)||(49||)||1,053||(105||)|
|Net loss attributable to common stockholders||$||(61,833||)||$||(48,136||)||$||(111,751||)||$||(103,129||)|
|Net loss attributable to common stockholders per common share — basic and diluted||$||(0.33||)||$||(0.34||)||$||(0.61||)||$||(0.72||)|
|Weighted-average common shares outstanding — basic and diluted||188,621,423||143,000,718||182,303,128||142,886,614|
|Amicus Therapeutics, Inc.
Consolidated Balance Sheets
(in thousands, except share and per share amounts)
|June 30,||December 31,|
|Cash and cash equivalents||$||73,311||$||49,060|
|Investments in marketable securities||465,641||309,502|
|Prepaid expenses and other current assets||14,432||19,316|
|Total current assets||576,230||391,965|
|Investments in marketable securities||13,836||—|
|Property and equipment, less accumulated depreciation of $14,415 and $12,515 at June 30, 2018 and December 31, 2017, respectively||9,111||9,062|
|In-process research & development||23,000||23,000|
|Other non-current assets||5,915||5,200|
|Liabilities and Stockholders’ Equity|
|Accounts payable, accrued expenses, and other current liabilities||$||41,613||$||53,890|
|Contingent consideration payable||8,600||8,400|
|Total current liabilities||52,963||70,040|
|Contingent consideration payable||18,200||17,000|
|Deferred income taxes||6,465||6,465|
|Other non-current liability||2,770||2,346|
|Commitments and contingencies|
|Common stock, $0.01 par value, 500,000,000 and 250,000,000 shares authorized, 189,053,214 and 166,989,790 shares issued and outstanding at June 30, 2018 and December 31, 2017, respectively||1,939||1,721|
|Additional paid-in capital||1,723,865||1,400,758|
|Accumulated other comprehensive loss:|
|Foreign currency translation adjustment||(1,539||)||(1,659||)|
|Unrealized gain on available-for-sale securities||(455||)||(436||)|
|Total stockholders’ equity||561,895||352,850|
|Total Liabilities and Stockholders’ Equity||$||825,889||$||627,024|
Source: Amicus Therapeutics, Inc.