Amicus Therapeutics Announces Second Quarter 2017 Financial Results and Corporate Updates
179 Fabry Disease Patients on Reimbursed Galafold (migalastat) as of
Migalastat NDA Submission Targeted for 4Q17
Phase 3 EB Topline Data and Complete Phase 1/2 Pompe Data on Track for Late 3Q17
Second Quarter 2017 Financial Results
- Total revenue in the second quarter 2017 was approximately
$7.2 million , a sequential increase of 71.4% from total revenue of$4.2 million in the first quarter 2017. 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
$227.2 million atJune 30, 2017 compared to$330.4 million atDecember 31, 2016 . - Total operating expenses increased to
$53.2 million compared to$48.5 million for the second quarter 2016 primarily due to increases in manufacturing scale-up investments on the Pompe program. - Net cash spend was
$103.5 million for the six months endingJune 30, 2017 . - Net loss was
$48.1 million , or$0.34 per share, compared to a net loss of$51.1 million , or$0.40 per share, for the second quarter 2016.
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. The
International Launch and Expanded Access Programs (EAP) Updates:
- 179 patients (naïve and ERT-switch) on reimbursed Galafold as of
July 31, 2017 - 12 countries with reimbursement (commercial or EAP) including the top four largest EU markets
- Reimbursement dossiers submitted and pricing discussions are now underway in 13 countries
- Target of 300 patients treated with reimbursed Galafold by year-end 2017
Global Regulatory Updates:
- Two additional approvals secured outside the EU (
Switzerland andIsrael ) - Regulatory submissions completed in seven additional territories outside the EU, including
Japan ,Canada andAustralia U.S. FDA confirmed NDA submission may be based on existing data (no additional gastrointestinal symptoms study required)
Anticipated Upcoming Fabry Disease Program Milestones:
- International commercial launch and EAPs in additional countries
- Additional regulatory
submissions including a
U.S. NDA (4Q17) - Regulatory decision in
Japan (1H18) - Fabry ERT cell line development and optimization
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. Positive functional data in initial patients were reported during the second quarter 2017 from an ongoing global Phase 1/2 clinical study (ATB200-02) to evaluate safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of ATB200/AT2221. The study enrolled a total of 20 patients across three cohorts, including ambulatory ERT-switch patients (Cohort 1), non-ambulatory ERT-switch patients (Cohort 2), and ERT-naïve patients (Cohort 3).
Anticipated Upcoming Pompe Disease Program Milestones:
- Full ATB200-02 study data, including 6-month functional data in all patients (late 3Q17)
- Meetings with US and EU regulators
SD-101 for Epidermolysis Bullosa (EB)
SD-101 is a novel, late-stage, proprietary topical treatment with the potential to be the first approved therapy for EB. SD-101 is currently being investigated in a registration-directed Phase 3 study ( ESSENCE, also known as SD-005) to support global regulatory submissions. As previously announced, Amicus has completed the analysis plan for the primary endpoints in the blinded ongoing ESSENCE study, and top-line Phase 3 data are on track for late in the third quarter of 2017. More than 95% of patients completing the primary treatment period have elected to continue in the open-label extension study
SD-101 was the first investigational treatment to show improvements in wound closure across all major EB types in completed Phase 2 clinical studies. SD-101 has been granted FDA Breakthrough Therapy designation, rare pediatric disease designation, and orphan drug designation.
Anticipated EB Program Milestones:
- Top-line Phase 3 data (late 3Q17)
Conference Call and Webcast
An audio webcast can also be accessed via the Investors section of the
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
TABLE 1 | |||||||||||||||
Amicus Therapeutics, Inc. | |||||||||||||||
Consolidated Statements of Operations | |||||||||||||||
(Unaudited) | |||||||||||||||
(in thousands, except share and per share amounts) | |||||||||||||||
Three Months Ended | Six Months Ended | ||||||||||||||
2017 | 2016 | 2017 | 2016 | ||||||||||||
Revenue: | |||||||||||||||
Net product sales | $ | 7,158 | $ | — | $ | 11,327 | $ | — | |||||||
Cost of goods sold | 1,061 | — | 1,836 | — | |||||||||||
Gross Profit | 6,097 | — | 9,491 | — | |||||||||||
Operating Expenses: | |||||||||||||||
Research and development | 31,985 | 18,281 | 62,861 | 41,706 | |||||||||||
Selling, general and administrative | 19,311 | 19,300 | 38,443 | 35,001 | |||||||||||
Changes in fair value of contingent consideration payable | 1,050 | 10,186 | 5,628 | 13,338 | |||||||||||
Restructuring charges | — | 8 | — | 58 | |||||||||||
Depreciation | 812 | 767 | 1,636 | 1,440 | |||||||||||
Total operating expenses | 53,158 | 48,542 | 108,568 | 91,543 | |||||||||||
Loss from operations | (47,061 | ) | (48,542 | ) | (99,077 | ) | (91,543 | ) | |||||||
Other income (expenses): | |||||||||||||||
Interest income | 753 | 331 | 1,512 | 638 | |||||||||||
Interest expense | (4,179 | ) | (1,055 | ) | (8,469 | ) | (2,000 | ) | |||||||
Other income (expense) | 2,400 | (2,237 | ) | 3,010 | (2,289 | ) | |||||||||
Loss before income tax (expense)/benefit | (48,087 | ) | (51,503 | ) | (103,024 | ) | (95,194 | ) | |||||||
Income tax (expense)/ benefit | (49 | ) | 453 | (105 | ) | 453 | |||||||||
Net loss attributable to common stockholders | $ | (48,136 | ) | $ | (51,050 | ) | $ | (103,129 | ) | (94,741 | ) | ||||
Net loss attributable to common stockholders per common share — basic and diluted | $ | (0.34 | ) | $ | (0.40 | ) | $ | (0.72 | ) | (0.75 | ) | ||||
Weighted‑average common shares outstanding — basic and diluted | 143,000,718 | 129,122,175 | 142,886,614 | 127,160,943 |
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 | $ | 37,394 | $ | 187,026 | |||||
Investments in marketable securities | 189,838 | 143,325 | |||||||
Accounts receivable | 3,786 | 1,304 | |||||||
Inventories | 3,948 | 3,416 | |||||||
Prepaid expenses and other current assets | 6,023 | 4,993 | |||||||
Total current assets | 240,989 | 340,064 | |||||||
Property and equipment, less accumulated depreciation of | 10,471 | 9,816 | |||||||
In-process research & development | 486,700 | 486,700 | |||||||
197,797 | 197,797 | ||||||||
Other non-current assets | 3,009 | 2,468 | |||||||
Total Assets | $ | 938,966 | $ | 1,036,845 | |||||
Liabilities and Stockholders' Equity | |||||||||
Current liabilities: | |||||||||
Accounts payable, accrued expenses, and other current liabilities | $ | 35,645 | $ | 41,008 | |||||
Deferred reimbursements, current portion | 18,850 | 13,850 | |||||||
Contingent consideration payable, current portion | 46,188 | 56,101 | |||||||
Total current liabilities | 100,683 | 110,959 | |||||||
Deferred reimbursements | 16,906 | 21,906 | |||||||
Convertible notes | 159,171 | 154,464 | |||||||
Contingent consideration payable | 219,162 | 213,621 | |||||||
Deferred income taxes | 173,869 | 173,771 | |||||||
Other non-current liability | 2,283 | 1,973 | |||||||
Commitments and contingencies | |||||||||
Stockholders' equity: | |||||||||
Common stock, 143,371,243 and 142,691,986 shares issued and outstanding at | 1,485 | 1,480 | |||||||
Additional paid-in capital | 1,132,229 | 1,120,156 | |||||||
Accumulated other comprehensive loss: | |||||||||
Foreign currency translation adjustment, less tax expense of | (192 | ) | 1,945 | ||||||
Unrealized gain on available-for securities | 31 | 102 | |||||||
Warrants | 16,076 | 16,076 | |||||||
Accumulated deficit | (882,737 | ) | (779,608 | ) | |||||
Total stockholders' equity | 266,892 | 360,151 | |||||||
Total Liabilities and Stockholders' Equity | $ | 938,966 | $ | 1,036,845 |
FOLD-G
CONTACTS: Investors/Media:Source:Amicus Therapeutics Sara Pellegrino , IRC Senior Director, Investor Relations spellegrino@amicusrx.com (609) 662-5044 Media:W2O Group Brian Reid breid@w2ogroup.com (212) 257-6725
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