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Share Name | Share Symbol | Market | Type |
---|---|---|---|
Amicus Therapeutics Inc | NASDAQ:FOLD | NASDAQ | Common Stock |
Price Change | % Change | Share Price | Bid Price | Offer Price | High Price | Low Price | Open Price | Shares Traded | Last Trade | |
---|---|---|---|---|---|---|---|---|---|---|
0.06 | 0.58% | 10.48 | 10.48 | 10.49 | 10.79 | 10.47 | 10.74 | 578,027 | 19:48:27 |
260+ Fabry Patients on Reimbursed Galafold™ (Migalastat) and On Target to Reach 300 Patients by Year-End 2017
John F. Crowley, Chairman and Chief Executive Officer of Amicus Therapeutics, stated, “During the third quarter we continued to successfully execute across our strategic priorities for our core programs in Fabry and Pompe. We are pleased with the significant growth and expansion of our international Galafold launch, and the number of patients treated with this precision medicine for Fabry disease. In Pompe disease, the data cascade for our novel treatment paradigm ATB200/AT2221 has continued to exceed our expectations in terms of the consistency, durability and magnitude of effect across patients and across functional outcomes, key disease biomarkers and safety. There has been extraordinary patient and physician demand for this important potential treatment option. We are committed to increasing access to this investigational medicine for as many people as possible living with Pompe as soon as we can.”
Amicus is on track to achieve four key strategic priorities between now and the end of 2017:
1) Target of 300 patients on reimbursed Galafold;2) New Drug Application (NDA) submission for migalastat to the U.S. FDA;3) Continued advancements with the Pompe program, including now ongoing collaborative discussions with regulators to determine the best and fastest pathway; and4) A strong balance sheet with more than 18 months of cash at year-end.
Third Quarter 2017 Financial Results
2017 Financial Guidance
Cash, cash equivalents, and marketable securities totaled $426.6 million at September 30, 2017 compared to $330.4 million at December 31, 2016. The current cash position includes $243.0 million in net proceeds from a follow on public offering in July 2017.
Amicus continues to expect full-year 2017 net operating cash spend of between $175 million to $200 million and full-year 2017 total net cash spend (including third-party milestone payments and capital expenditures) of between $200 million and $225 million. The current cash position is anticipated to fund ongoing operations into at least the second half of 2019.
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 European Union, Switzerland, Israel, Canada and Australia have granted full approval for migalastat under the trade name Galafold. The EU approval may serve as the basis for regulatory approvals in more than two-thirds of the global Fabry market that is outside the U.S. In the U.S., as previously announced, the FDA has confirmed that Amicus may submit a new drug application (NDA) for migalastat.
International Launch and Expanded Access Programs (EAP) Updates:
Global Regulatory Updates:
Anticipated Upcoming Fabry Disease Program Milestones:
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. Additional positive data were reported in October 2017 from an ongoing global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221 across three cohorts, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3). The Company has commenced collaborative discussions with U.S. and EU regulators regarding the best and fastest pathway forward for this novel treatment option, and continues to anticipate a Pompe regulatory pathway update in the first half of 2018.
In order to continue to build a robust data set and to meet the needs of the Pompe community, Amicus announced the following four key status updates for this important program:
Anticipated Upcoming Pompe Disease Program Milestones:
SD-101 for Epidermolysis Bullosa (EB)
During the third quarter Amicus reported that top-line data from the randomized, double-blind, placebo-controlled Phase 3 clinical study (ESSENCE, SD-005) to assess the efficacy and safety of the novel topical wound-healing agent SD-101 did not meet the primary endpoints or secondary endpoints in participants with EB. Based on these top-line data Amicus has no current plans to invest in any additional clinical studies or commercial preparation activities for SD-101. The Company continues to make SD-101 available to all patients currently enrolled in the ongoing extension study (SD-006).
Conference Call and WebcastAmicus Therapeutics will host a conference call and audio webcast today, November 8, 2017 at 8:30 a.m. ET to discuss third quarter 2017 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: 5995789.
An audio webcast can also be accessed via the Investors section of the Amicus Therapeutics corporate web site at http://ir.amicusrx.com/ and will be archived for 30 days. Web participants are encouraged to go to the web site 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 today. Access numbers for this replay are 855-859-2056 (U.S./Canada) and 404-537-3406 (international); conference ID: 5995789.
Non-GAAP Financial Measures
In addition to the United States generally accepted accounting principles (GAAP) results, this earnings release contains non-GAAP financial measures that we believe, when considered together with the GAAP information, provides useful information to investors that promotes a more complete understanding of our operating results and financial position for the current period. Management uses these non-GAAP financial measures internally for planning, forecasting, evaluating and allocating resources to the Company's programs. The non-GAAP results exclude the impact of the following GAAP items specifically related to the Phase 3 ESSENCE study in EB: changes in fair value of contingent consideration, impairment of assets and adjustments to income tax expense. These non-GAAP financial measures should be considered in addition to, and not as replacements for, or superior to, measures of financial performance prepared in accordance with GAAP. Please refer to the attached Reconciliation of Non-GAAP Financial Measures for explanations of the amounts adjusted to arrive at non-GAAP total operating expense, net loss attributable to common stockholders and net loss attributable to common stockholders per common share – basic and diluted for the three month period ended September 30, 2017.
EU Important Safety InformationTreatment 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 biotechnology company at the forefront of therapies for rare and orphan diseases. The Company has a robust pipeline of advanced therapies for a broad range of human genetic diseases. Amicus’ lead programs in development include the small molecule pharmacological chaperone migalastat as a monotherapy for Fabry disease, as well as novel enzyme replacement therapy (ERT) and biologic products for Fabry disease, Pompe disease, and other rare and devastating diseases.
Forward-Looking StatementsThis 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, financing plans, and the projected 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 or 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; and the potential that we will need additional funding to complete all of our studies. Further, the results of earlier preclinical studies and/or clinical trials may not be predictive of future results for any of our product candidates. With respect to statements regarding projections of the Company's 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 previous filings with the SEC and in our Annual Report on Form 10-K for the year ended December 31, 2016 and our Quarterly Report on Form 10-Q for the quarter ended September 30, 2017, filed today. 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.
CONTACTS:
Investors/Media:Amicus TherapeuticsSara Pellegrino, IRCSenior Director, Investor Relationsspellegrino@amicusrx.com (609) 662-5044
Media: W2O GroupBrian Reidbreid@w2ogroup.com(212) 257-6725
TABLE 1 | |||||||||||||||
Amicus Therapeutics, Inc. | |||||||||||||||
Consolidated Statements of Operations | |||||||||||||||
(Unaudited) | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
Three Months Ended September 30, | Nine Months Ended September 30, | ||||||||||||||
2017 | 2016 | 2017 | 2016 | ||||||||||||
Revenue: | |||||||||||||||
Net product sales | $ | 10,874 | $ | 2,127 | $ | 22,201 | $ | 2,127 | |||||||
Cost of goods sold | 1,790 | 344 | 3,626 | 344 | |||||||||||
Gross Profit | 9,084 | 1,783 | 18,575 | 1,783 | |||||||||||
Operating Expenses: | |||||||||||||||
Research and development | 40,641 | 32,457 | 103,502 | 74,163 | |||||||||||
Selling, general and administrative | 21,647 | 17,469 | 60,090 | 52,470 | |||||||||||
Changes in fair value of contingent consideration payable | (244,250 | ) | (4,110 | ) | (238,622 | ) | 9,228 | ||||||||
Loss on impairment of assets | 465,427 | - | 465,427 | - | |||||||||||
Restructuring charges | - | 11 | - | 69 | |||||||||||
Depreciation | 851 | 896 | 2,486 | 2,336 | |||||||||||
Total operating expenses | 284,316 | 46,723 | 392,883 | 138,266 | |||||||||||
Loss from operations | (275,232 | ) | (44,940 | ) | (374,308 | ) | (136,483 | ) | |||||||
Other income (expenses): | |||||||||||||||
Interest income | 1,190 | 460 | 2,702 | 1,098 | |||||||||||
Interest expense | (4,351 | ) | (1,517 | ) | (12,820 | ) | (3,517 | ) | |||||||
Other income (expense) | 2,044 | (910 | ) | 5,054 | (3,199 | ) | |||||||||
Loss before income tax benefit | (276,349 | ) | (46,907 | ) | (379,372 | ) | (142,101 | ) | |||||||
Income tax benefit | 164,683 | 253 | 164,578 | 706 | |||||||||||
Net loss attributable to common stockholders | $ | (111,666 | ) | $ | (46,654 | ) | $ | ( 214,794 | ) | $ | (141,395 | ) | |||
Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.69 | ) | $ | (0.33 | ) | $ | (1.44 | ) | $ | (1.07 | ) | |||
Weighted‑average common shares outstanding — basic and diluted | 160,796,841 | 140,656,109 | 148,963,864 | 131,675,690 | |||||||||||
TABLE 2 | |||||||||
Amicus Therapeutics, Inc. | |||||||||
Consolidated Balance Sheets | |||||||||
(Unaudited) | |||||||||
(in thousands, except share and per share amounts) | |||||||||
September 30, | December 31, | ||||||||
2017 | 2016 | ||||||||
Assets | |||||||||
Current assets: | |||||||||
Cash and cash equivalents | $ | 64,133 | $ | 187,026 | |||||
Investments in marketable securities, current portion | 347,388 | 143,325 | |||||||
Accounts receivable | 5,974 | 1,304 | |||||||
Inventories | 7,272 | 3,416 | |||||||
Prepaid expenses and other current assets | 6,246 | 4,993 | |||||||
Total current assets | 431,013 | 340,064 | |||||||
Investments in marketable securities | 15,109 | - | |||||||
Property and equipment, less accumulated depreciation of $13,273 and $12,495 at September 30, 2017 and December 31, 2016, respectively | 9,641 | 9,816 | |||||||
In-process research & development | 23,000 | 486,700 | |||||||
Goodwill | 197,797 | 197,797 | |||||||
Other non-current assets | 4,219 | 2,468 | |||||||
Total Assets | $ | 680,779 | $ | 1,036,845 | |||||
Liabilities and Stockholders’ Equity | |||||||||
Current liabilities: | |||||||||
Accounts payable, accrued expenses, and other current liabilities | $ | 53,709 | $ | 41,008 | |||||
Deferred reimbursements, current portion | 6,250 | 13,850 | |||||||
Contingent consideration payable, current portion | 8,200 | 56,101 | |||||||
Total current liabilities | 68,159 | 110,959 | |||||||
Deferred reimbursements | 16,906 | 21,906 | |||||||
Convertible notes | 161,635 | 154,464 | |||||||
Contingent consideration payable | 12,900 | 213,621 | |||||||
Deferred income taxes | 9,186 | 173,771 | |||||||
Other non-current liability | 2,313 | 1,973 | |||||||
Commitments and contingencies | |||||||||
Stockholders’ equity: | |||||||||
Common stock, $0.01 par value, 250,000,000 shares authorized 165,491,141 and 142,691,986 shares issued and outstanding at September 30, 2017 and December 31, 2016, respectively | 1,707 | 1,480 | |||||||
Additional paid-in capital | 1,387,767 | 1,120,156 | |||||||
Accumulated other comprehensive loss: | |||||||||
Foreign currency translation adjustment | (1,367 | ) | 1,945 | ||||||
Unrealized gain on available-for-sale securities | (101 | ) | 102 | ||||||
Warrants | 16,076 | 16,076 | |||||||
Accumulated deficit | (994,402 | ) | (779,608 | ) | |||||
Total stockholders’ equity | 409,680 | 360,151 | |||||||
Total Liabilities and Stockholders’ Equity | $ | 680,779 | $ | 1,036,845 |
TABLE 3 | |||
Amicus Therapeutics, Inc. | |||
Reconciliation of Non-GAAP Financial Measures | |||
Dollars in Thousands Except Per Share Data | |||
Three Months | |||
Ended | |||
September 30, 2017 | |||
Total operating expenses – as reported | $ | 284,316 | |
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 | $ | 73,539 | |
Net loss attributable to common stockholders – as reported | $ | (111,666 | ) |
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 | $ | (65,572 | ) |
Net loss attributable to common stockholders per common | |||
share – basic and diluted – as reported | $ | (0.69 | ) |
Net loss attributable to common stockholders per common | |||
share – basic and diluted – as adjusted | $ | (0.41 | ) |
Weighted-average common shares outstanding – basic and | |||
diluted – as reported and adjusted | 160,796,841 | ||
(1) Related to the reversal of the deferred tax liability associated with the Scioderm in process research and development asset.
FOLD–G
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