Amicus Therapeutics Announces Third Quarter 2017 Financial Results and Corporate Updates
260+ Fabry Patients on Reimbursed Galafold™ (Migalastat) and
On Target to Reach 300 Patients by Year-End 2017
Pompe Retrospective and Prospective Data Collection Studies Initiated
Additional Pompe Clinical Data to be Presented at WORLDSymposium™ in
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
3) Continued advancements with the Pompe program, including now ongoing collaborative discussions with regulators to determine the best and fastest pathway; and
4) A strong balance sheet with more than 18 months of cash at year-end.
Third Quarter 2017 Financial Results
- Total revenue in the third quarter 2017 was approximately
$10.9 million , a sequential increase of 51.4% from total revenue of$7.2 million in the second quarter 2017, and a year-over-year increase of 419% from total revenue of$2.1 million in the third quarter 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
$426.6 million atSeptember 30, 2017 compared to$330.4 million atDecember 31, 2016 . - Total operating expenses increased to
$284.3 million compared to$46.7 million for the third quarter 2016 primarily from non-cash charges related to the Phase 3 ESSENCE study in epidermolysis bullosa (EB). - Operating expenses, as adjusted, excludes the impact of the non-cash charges related to the ESSENCE study, were
$73.5 million , representing a$26.8 million increase over the third quarter of 2016 primarily due to increased investments in the Pompe and EB programs as well as increased investment in the Galafold commercial launch. - Net cash spend was
$147.3 million for the nine months endingSeptember 30, 2017 . - Net loss was
$111.7 million , or$0.69 per share, compared to a net loss of$46.7 million , or$0.33 per share, for the third quarter 2016. Net loss, as adjusted excludes the impact of the non-cash charges related to the ESSENCE study was$65.6 million or$0.41 per share.
2017 Financial Guidance
Cash, cash equivalents, and marketable securities totaled
Amicus continues to expect full-year 2017 net operating cash spend of between
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
International Launch and Expanded Access Programs (EAP) Updates:
- More than 260 patients (naïve and ERT-switch) on reimbursed Galafold as of
October 31, 2017 - 13 countries with reimbursement (commercial or EAP) including the top four largest EU countries
- Reimbursement dossiers submitted and pricing discussions are now underway in 12 countries
- Target of 300 patients treated with reimbursed Galafold on track for year-end 2017
Global Regulatory Updates:
- Four additional approvals secured outside the EU (
Switzerland ,Israel ,Canada andAustralia ) - Regulatory submissions completed in seven additional countries outside the EU, including
Japan - NDA submission to
U.S. FDA to be based on existing data on track for 4Q17
Anticipated Upcoming Fabry Disease Program Milestones:
- Commercial launch and EAPs in additional international countries
- Additional regulatory submissions including a
U.S. NDA (4Q17) - Regulatory decision in
Japan (1H18) - Final preclinical data and announcement of path forward for novel Amicus Fabry ERT cell line for Fabry patients with non-amenable mutations (1Q18)
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
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:
- A retrospective natural history study of Pompe patients treated with approved standard of care ERT has been initiated at leading global Pompe disease treatment centers (POM-002 Study)
- A prospective observational study has also been initiated in Pompe patients currently receiving approved standard of care ERT at leading global Pompe disease treatment centers (POM-003 Study)
- All engineering runs successfully completed and GMP production of ATB200 has commenced at the large commercial scale (1,000 Liters)
- Analytical and in vivo (preclinical) comparability studies completed between the 250 Liter and 1,000 Liter scale
Anticipated Upcoming Pompe Disease Program Milestones:
- Ongoing discussions with
U.S. and EU regulators - Additional data from ATB200-02 clinical study at
14th Annual WORLDSymposium™ (
February 5-9, 2018 ) - Pompe regulatory pathway update (1H18)
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 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, 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
CONTACTS:
Investors/Media:
Senior Director, Investor Relations
spellegrino@amicusrx.com
(609) 662-5044
Media:
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(212) 257-6725
TABLE 1 | |||||||||||||||
Amicus Therapeutics, Inc. | |||||||||||||||
Consolidated Statements of Operations | |||||||||||||||
(Unaudited) | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
Three Months Ended | Nine Months Ended | ||||||||||||||
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) | |||||||||
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 | 9,641 | 9,816 | |||||||
In-process research & development | 23,000 | 486,700 | |||||||
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, | 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 | |||
Reconciliation of Non-GAAP Financial Measures | |||
Dollars in Thousands Except Per Share Data | |||
Three Months | |||
Ended | |||
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
FOLD-G
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