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Amicus Therapeutics Announces Second Quarter 2018 Financial Results and Corporate Updates
2Q18 Net Product Sales of $21.3M Driven by Galafold® (Migalastat) Expansion
Company Increases FY18 Global Galafold Revenue Guidance to $80M-$90M
Pompe Clinical, Regulatory and Manufacturing Activities Continue to Advance
Conference Call and Webcast Today at 8:30am ET
CRANBURY, N.J., Aug. 07, 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 second quarter ended June 30, 2018. The Company also summarized recent program updates, raised its full-year 2018 revenue guidance and updated its net cash spend guidance for the year.
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, Inc. stated, “In the first half of 2018, we have made tremendous progress advancing Galafold, expanding access to patients with amenable mutations across the world. Given the continued strong momentum of the Galafold launch, the early commercial launch in Japan, and the pending accelerated approval in the U.S., we are raising our 2018 revenue guidance to $80 million to $90 million. We have also continued to advance our Pompe clinical, manufacturing and regulatory activities and look forward to providing an update later this quarter on the pivotal study design and best and fastest regulatory path in the United States for this important program. Additionally, we are also extraordinarily focused on assembling a portfolio of technologies, programs and partnerships in the gene therapy space. Our goal beginning in the second half of this year is a bold one: to build one of the most robust gene therapy pipelines in the field of rare, metabolic disorders. We are well positioned to achieve our vision to impact as many people as possible who are living with rare metabolic diseases as we continue to build a leading global rare disease biotech company.”
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 million in the second quarter of 2017.
- Cash, cash equivalents, and marketable securities totaled $552.8 million at June 30, 2018, compared to $358.6 million at December 31, 2017.
- Total operating expenses increased to $65.1 million for the second quarter 2018 compared to $53.2 million in 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 million for the second quarter 2018. Net loss was $61.8 million, or $0.33 per share, for the second quarter 2018 compared to a net loss of $48.1 million, or $0.34 per share, for the second quarter 2017.
“The second quarter of 2018 marked another period of continued growth for Galafold,” said Bradley L. Campbell, President and COO of Amicus Therapeutics. “We are raising our full-year 2018 Galafold global revenue guidance given the current and anticipated increase in patient and physician adoption in our existing markets, the ongoing launch in Japan, and our anticipated PDUFA date and launch in the U.S. this quarter. And we’re pleased to announce that we’ve hired 100% of our US launch team comprised of passionate professionals with significant rare disease experience.”
2018 Financial Guidance
For the full-year 2018 the Company is increasing its total Galafold revenue guidance to $80 million to $90 million from the previous range of $75 million to $85 million. This reflects global revenue from all expected 2018 commercial markets.
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 $220 to $250 million from the previous range of $230 to $260 million. The current cash position, including proceeds from the February 2018 equity offering and expected Galafold revenues, is sufficient to fund ongoing Fabry and Pompe program operations into at least 2021. Potential future business development collaborations, pipeline expansion, and investment in biologics manufacturing capabilities could impact the Company’s future capital requirements.
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 Australia, Canada, EU, Israel, Japan, South Korea and Switzerland have granted full approval for migalastat under the trade name Galafold to treat Fabry disease in patients 16 years or older who have amenable genetic mutations. In the U.S., the FDA accepted the Company’s new drug application (NDA) for migalastat under Subpart H priority review with a six-month PDUFA goal date of August 13, 2018.
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 Korea and Switzerland
- Commercial launch initiated in Japan during the second quarter
- U.S. leadership and field team now in place to support planned U.S. launch
- U.S. FDA regulatory decision (3Q18)
AT-GAA for Pompe Disease
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.
- 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 Congress of the World Muscle Society (4Q18).
- Commence pivotal study in 2H18.
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.
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. The cornerstone of the Amicus portfolio is Galafold, an oral precision medicine for people living with Fabry disease who have amenable genetic mutations. The lead biologics program in the Amicus pipeline is AT-GAA, a novel, late-stage treatment for Pompe disease. Amicus is committed to advancing and expanding a robust pipeline of cutting-edge, first- or best-in-class medicines for rare metabolic diseases.
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|