Amicus Therapeutics Announces Full-Year 2017 Financial Results and 2018 Corporate Updates
Galafold Expansion Continues with 360+ Fabry Patients Treated Today - on Track to Achieve FY18 Revenue Guidance of
Significant Progress Across Pompe Clinical, Regulatory and Manufacturing Activities - Successfully Completed First GMP Manufacturing Campaign of ATB200 Drug Substance at 1000L Scale
Conference Call and Webcast Today at
Corporate Highlights for Full-Year 2017 and Year-to-Date 2018
- Full-year 2017 revenue for Fabry oral precision medicine Galafold (migalastat) totaled
$36.9 million . On track to achieve full-year 2018 revenue guidance of$75M-$85M with more than 360 patients treated today. - Regulatory decisions for migalastat anticipated in 1H18 in
Japan (J-NDA) and in 3Q18 in the U.S. (NDA filed under priority review; PDUFA action dateAugust 13, 2018 ). - Novel, highly differentiated Pompe treatment regimen ATB200/AT2221 continues to show persistent and durable improvements on functional outcomes and key disease biomarkers, with favorable safety profile, following up to 12 months of treatment in Pompe patients.
- Significant Pompe biologics manufacturing milestones achieved. Agreement reached with
FDA on biocomparability between 250L GMP and 1000L engineering batches in 4Q17. Initial 1000L GMP drug substance manufacturing campaign now complete in 1Q18. - Building robust Pompe clinical data package to include current and additional patients in ongoing Phase 1/2 clinical study, as well as a supportive retrospective natural history study and prospective observational study. Regulatory update expected in 2Q18.
- Cash, cash equivalents and marketable securities totaled
$358.6 million atDecember 31, 2017 . Balance sheet further strengthened with$300M in gross proceeds from a follow on public offering inFebruary 2018 .
2018 Key Strategic Priorities
- Double global revenue for Galafold (
$75 million - $85 million ) - Secure approvals for migalastat in
Japan and the U.S. - Achieve clinical, manufacturing and regulatory milestones to advance ATB200/AT2221 toward global regulatory submissions and approvals as soon as possible
- Develop and expand preclinical pipeline to ensure at least one new clinical program in 2019
- Maintain a strong balance sheet
Full-Year 2017 Financial Results
- Total revenue in the full-year 2017 was
$36.9 million , an increase from total revenue of$5.0 million in the full-year 2016. Total revenue represents commercial sales of Galafold (migalastat) which commenced inMay 2016 , as well as reimbursed Expanded Access Programs (EAPs). - Cash, cash equivalents, and marketable securities totaled
$358.6 million atDecember 31, 2017 compared to$330.4 million atDecember 31, 2016 . - Total operating expenses increased to
$472.7 million for the full-year 2017 compared to$186.0 million in the full-year 2016. Operating expenses in 2017 included$210.8 million of non-cash charges related to the Phase 3 ESSENCE study in epidermolysis bullosa (EB). - Operating expenses, as adjusted, excluding the impact of non-cash charges related to the ESSENCE study, were
$261.9 million for the full-year 2017, reflecting increased investments in the Pompe and EB programs as well as increased investment in the Galafold commercial launch. - Net cash spend was
$216.5 million for the full-year 2017, in-line with full-year 2017 net cash spend guidance of$200 million to $225 million . - Net loss was
$284.0 million , or$1.85 per share, compared to a net loss of$200.0 million , or$1.49 per share, for the full-year 2016. Net loss, as adjusted, excludes the impact of the non-cash charges related to the ESSENCE study and was$237.9 million or$1.55 per share.
2018 Financial Guidance
Amicus recorded
Cash, cash equivalents, and marketable securities totaled
Program Highlights
Migalastat for Fabry Disease
Migalastat is an oral precision medicine intended to treat Fabry disease in patients who have amenable genetic mutations. Regulatory authorities in the
Amicus is committed to advancing the highest quality therapies for all people living with Fabry disease. 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:
- 360+ patients (naïve and ERT-switch) on reimbursed Galafold as of
February 28, 2017 - Total full-year 2017 revenue of
$36.9 million from global commercial sales and expanded access programs (EAPs) - Pricing and reimbursement secured in 18 countries
- Approvals secured in EU,
Australia ,Canada ,Israel ,South Korea andSwitzerland - Approvals pending in
Japan , U.S. andTaiwan
Anticipated Milestones:
- Regulatory decisions on Japanese J-NDA (1H18) and U.S. NDA (3Q18)
- Total full-year 2018 revenue guidance of
$75 million to $85 million - ATB101 co-formulated with migalastat advancing toward the clinic in 2019
ATB200/AT2221 for Pompe Disease
ATB200/AT2221 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. During WORLDSymposium™ in
The Company is in the midst of a series of collaborative discussions with U.S. and EU regulators regarding the best and fastest pathway forward for this novel treatment option. Amicus expects to provide a Pompe regulatory pathway update in the second quarter of 2018. While these discussions are underway, Amicus continues to make progress with clinical and manufacturing activities to support the needs of the Pompe community.
Pompe clinical activities:
- Phase 1/2 clinical study includes 19 current patients treated for up to 12 months, with four to six additional patients to be enrolled
- Retrospective study (POM-002) on the natural history of Pompe disease in up to 100 ERT-treated Pompe patients to help provide context for the ATB200-02 clinical study results
- Prospective observational study (POM-003) to assess safety and functional outcomes in patients currently treated with standard of care ERT, and to serve as a potential run-in for a registration study
Pompe manufacturing activities:
FDA agreed on comparability between 250L scale and 1000L engineering batches, as well as the testing strategy for demonstrating comparability between 250L scale and 1000L GMP batches- Initial GMP production runs of ATB200 drug substance completed at the 1,000 liter commercial scale
Anticipated Upcoming Pompe Program Milestones:
- Expansion of ongoing ATB200-02 clinical study to include four to six additional ambulatory ERT-switch patients
- Final demonstration of comparability between 1,000L GMP material and 250L material and release for clinic of 1,000L GMP material
- Pompe regulatory pathway update (2Q18)
- Initiation of larger registration-directed study (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.
About
Forward-Looking Statements
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
CONTACTS:
Investors/Media:
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
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(347) 658-8290
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TABLE 1 | |||||||||||
Amicus Therapeutics, Inc. Consolidated Statements of Operations (in thousands, except share and per share amounts) |
|||||||||||
Years Ended December 31, | |||||||||||
2017 | 2016 | 2015 | |||||||||
Revenue: | |||||||||||
Net Product Sales | $ 36,930 | $ 4,958 | $ — | ||||||||
Total revenue | 36,930 | 4,958 | — | ||||||||
Cost of goods sold | 6,236 | 833 | — | ||||||||
Gross Profit | 30,694 | 4,125 | — | ||||||||
Operating Expenses: | |||||||||||
Research and development | 149,310 | 104,793 | 76,943 | ||||||||
Selling, general and administrative | 88,671 | 71,151 | 47,269 | ||||||||
Changes in fair value of contingent consideration payable | (234,322 | ) | 6,760 | 4,377 | |||||||
Loss on impairment of assets | 465,427 | — | — | ||||||||
Restructuring charges | — | 69 | 15 | ||||||||
Depreciation | 3,593 | 3,242 | 1,833 | ||||||||
Total operating expenses | 472,679 | 186,015 | 130,437 | ||||||||
Loss from operations | (441,985 | ) | (181,890 | ) | (130,437 | ) | |||||
Other income (expenses): | |||||||||||
Interest income | 4,096 | 1,602 | 929 | ||||||||
Interest expense | (17,240 | ) | (5,398 | ) | (1,578 | ) | |||||
Loss on extinguishment of debt | — | (13,302 | ) | (952 | ) | ||||||
Other income (expense) | 6,008 | (4,793 | ) | (80 | ) | ||||||
Loss before income tax benefit | (449,121 | ) | (203,781 | ) | (132,118 | ) | |||||
Income tax benefit | 165,119 | 3,739 | — | ||||||||
Net loss attributable to common stockholders | $(284,002 | ) | $(200,042 | ) | $(132,118 | ) | |||||
Net loss attributable to common stockholders per common share — basic and diluted | $(1.85 | ) | $(1.49 | ) | $(1.20 | ) | |||||
Weighted‑average common shares outstanding — basic and diluted | 153,355,144 | 134,401,588 | 109,923,815 |
TABLE 2 | ||||||||
Amicus Therapeutics, Inc. Consolidated Balance Sheets (in thousands, except share and per share amounts) |
||||||||
December 31, | ||||||||
2017 | 2016 | |||||||
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $49,060 | $187,026 | ||||||
Investments in marketable securities | 309,502 | 143,325 | ||||||
Accounts receivable | 9,464 | 1,304 | ||||||
Inventories | 4,623 | 3,416 | ||||||
Prepaid expenses and other current assets | 19,316 | 4,993 | ||||||
Total current assets | 391,965 | 340,064 | ||||||
Property and equipment, less accumulated depreciation of $12,515 and $12,495 at December 31, 2017 and 2016, respectively | 9,062 | 9,816 | ||||||
In-process research & development | 23,000 | 486,700 | ||||||
Goodwill | 197,797 | 197,797 | ||||||
Other non-current assets | 5,200 | 2,468 | ||||||
Total Assets | $ 627,024 | $1,036,845 | ||||||
Liabilities and Stockholders’ Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable, accrued expenses, and other current liabilities | $ 53,890 | $41,008 | ||||||
Deferred reimbursements, current portion | 7,750 | 13,850 | ||||||
Contingent consideration payable, current portion | 8,400 | 56,101 | ||||||
Total current liabilities | 70,040 | 110,959 | ||||||
Deferred reimbursements | 14,156 | 21,906 | ||||||
Convertible notes | 164,167 | 154,464 | ||||||
Contingent consideration payable | 17,000 | 213,621 | ||||||
Deferred income taxes | 6,465 | 173,771 | ||||||
Other non-current liability | 2,346 | 1,973 | ||||||
Commitments and contingencies | ||||||||
Stockholders’ equity: | ||||||||
Common stock, $.01 par value, 250,000,000 shares authorized, 166,989,790 shares issued and outstanding at December 31, 2017 Common stock, $.01 par value, 250,000,000 shares authorized, 142,691,986 shares issued and outstanding at December 31, 2016 |
1,721 | 1,480 | ||||||
Additional paid-in capital | 1,400,758 | 1,120,156 | ||||||
Accumulated other comprehensive loss: | ||||||||
Foreign currency translation adjustment | (1,659 | ) | 1,945 | |||||
Unrealized gain/ (loss) on available-for securities | (436 | ) | 102 | |||||
Warrants | 16,076 | 16,076 | ||||||
Accumulated deficit | (1,063,610 | ) | (779,608 | ) | ||||
Total stockholders’ equity | 352,850 | 360,151 | ||||||
Total Liabilities and Stockholders’ Equity | $ 627,024 | $1,036,845 |
TABLE 3 | ||||
Amicus Therapeutics, Inc. Reconciliation of Non-GAAP Financial Measures (in thousands, except share and per share amounts) |
||||
Year | ||||
Ended | ||||
December 31, 2017 | ||||
Total operating expenses – as reported | $ | 472,679 | ||
Loss on impairment of assets related to the | ||||
Phase 3 ESSENCE study in EB | 465,427 | |||
Changes in fair value of contingent | ||||
consideration payable related to the | ||||
Phase 3 ESSENCE study in EB | (254,650 | ) | ||
Total operating expenses – as adjusted | $ | 261,902 | ||
Net loss attributable to common stockholders – as reported | $ | (284,002 | ) | |
Loss on impairment of assets related to the | ||||
Phase 3 ESSENCE study in EB | 465,427 | |||
Changes in fair value of contingent | ||||
consideration payable related to the | ||||
Phase 3 ESSENCE study in EB | (254,650 | ) | ||
Income tax benefit (1) | 164,683 | |||
Net loss attributable to common stockholders – as adjusted | $ | (237,908 | ) | |
Net loss attributable to common stockholders per common | ||||
share – basic and diluted – as reported | $ | (1.85 | ) | |
Net loss attributable to common stockholders per common | ||||
share – basic and diluted – as adjusted | $ | (1.55 | ) | |
Weighted-average common shares outstanding – basic and | ||||
diluted – as reported and adjusted | 153,355,144 | |||
(1) Related to the reversal of the deferred tax liability associated with the Scioderm in process research and development asset.
Source: Amicus Therapeutics, Inc.