2018 Q1 Form 10-K Financial Statement

#000107008118000042 Filed on March 06, 2018

View on sec.gov

Income Statement

Concept 2018 Q1 2017 Q4 2017
Revenue $56.06M $57.95M $194.4M
YoY Change 111.18% 130.71% 135.04%
Cost Of Revenue $3.045M $2.400M $4.577M
YoY Change 7707.69%
Gross Profit $53.02M $75.60M $189.8M
YoY Change 100.0% 129.51%
Gross Profit Margin 94.57% 130.45% 97.65%
Selling, General & Admin $32.97M $35.50M $121.3M
YoY Change 29.29% 46.69% 24.85%
% of Gross Profit 62.19% 46.96% 63.89%
Research & Development $31.36M $29.20M $117.5M
YoY Change 14.62% 12.31% -0.15%
% of Gross Profit 59.16% 38.62% 61.88%
Depreciation & Amortization $6.022M $5.940M $17.68M
YoY Change 883.99% 633.33% 437.45%
% of Gross Profit 11.36% 7.86% 9.32%
Operating Expenses $72.81M $72.58M $258.7M
YoY Change 37.62% 44.63% 20.45%
Operating Profit -$16.74M $5.452M -$64.29M
YoY Change -36.47% -121.81% -51.32%
Interest Expense -$3.303M -$3.400M -$12.09M
YoY Change 48.85% 61.9% 46.13%
% of Operating Profit -62.36%
Other Income/Expense, Net $1.004M $100.0K -$1.279M
YoY Change -415.72% -85.71% 5.97%
Pretax Income -$19.04M $2.100M -$77.67M
YoY Change -34.09% -107.95% -45.13%
Income Tax $221.0K $800.0K $1.335M
% Of Pretax Income 38.1%
Net Earnings -$19.26M $1.270M -$79.00M
YoY Change -33.71% -104.74% -44.41%
Net Earnings / Revenue -34.36% 2.19% -40.64%
Basic Earnings Per Share -$2.02
Diluted Earnings Per Share -$463.9K $31.40K -$2.015M
COMMON SHARES
Basic Shares Outstanding 41.80M shares 41.49M shares
Diluted Shares Outstanding

Balance Sheet

Concept 2018 Q1 2017 Q4 2017
SHORT-TERM ASSETS
Cash & Short-Term Investments $178.3M $191.2M $191.2M
YoY Change -11.99% -17.48% -17.48%
Cash & Equivalents $97.68M $111.8M $111.8M
Short-Term Investments $80.60M $79.50M $79.50M
Other Short-Term Assets $5.800M $6.700M $6.700M
YoY Change 26.09% 42.55% 42.55%
Inventory $12.36M $10.75M $10.80M
Prepaid Expenses
Receivables $45.60M $40.40M $40.40M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $242.0M $249.1M $249.1M
YoY Change 0.53% -4.68% -4.67%
LONG-TERM ASSETS
Property, Plant & Equipment $8.302M $8.376M $8.400M
YoY Change 17.61% 12.75% 13.51%
Goodwill $0.00
YoY Change
Intangibles $127.6M $133.0M
YoY Change
Long-Term Investments
YoY Change
Other Assets $1.500M $1.200M $1.200M
YoY Change 66.67% 100.0% 100.0%
Total Long-Term Assets $137.4M $142.6M $142.6M
YoY Change 1635.48% 1669.33% 1682.5%
TOTAL ASSETS
Total Short-Term Assets $242.0M $249.1M $249.1M
Total Long-Term Assets $137.4M $142.6M $142.6M
Total Assets $379.4M $391.7M $391.7M
YoY Change 52.59% 45.41% 45.45%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $3.104M $15.28M $15.30M
YoY Change -13.27% 142.65% 142.86%
Accrued Expenses $9.200M $17.70M $17.70M
YoY Change 21.05% 30.15% 30.15%
Deferred Revenue $3.937M
YoY Change
Short-Term Debt $0.00 $0.00 $0.00
YoY Change
Long-Term Debt Due $0.00
YoY Change
Total Short-Term Liabilities $72.72M $82.05M $82.00M
YoY Change 60.1% 65.34% 65.32%
LONG-TERM LIABILITIES
Long-Term Debt $146.9M $145.0M $145.0M
YoY Change 47.03% 47.6% 47.66%
Other Long-Term Liabilities $209.0K $243.0K $8.200M
YoY Change -32.8% -27.46% 331.58%
Total Long-Term Liabilities $147.1M $145.2M $153.2M
YoY Change 46.78% 47.35% 53.05%
TOTAL LIABILITIES
Total Short-Term Liabilities $72.72M $82.05M $82.00M
Total Long-Term Liabilities $147.1M $145.2M $153.2M
Total Liabilities $229.1M $235.2M $235.2M
YoY Change 54.99% 57.06% 57.01%
SHAREHOLDERS EQUITY
Retained Earnings -$830.1M -$814.1M
YoY Change 8.63% 10.75%
Common Stock $42.00K $42.00K
YoY Change 23.53% 23.53%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity $150.3M $156.4M $156.4M
YoY Change
Total Liabilities & Shareholders Equity $379.4M $391.7M $391.7M
YoY Change 52.59% 45.41% 45.45%

Cashflow Statement

Concept 2018 Q1 2017 Q4 2017
OPERATING ACTIVITIES
Net Income -$19.26M $1.270M -$79.00M
YoY Change -33.71% -104.74% -44.41%
Depreciation, Depletion And Amortization $6.022M $5.940M $17.68M
YoY Change 883.99% 633.33% 437.45%
Cash From Operating Activities -$15.88M $21.29M -$10.06M
YoY Change -45.06% -259.83% -90.28%
INVESTING ACTIVITIES
Capital Expenditures $479.0K -$2.040M $3.101M
YoY Change 112.89% 64.52% 74.61%
Acquisitions $0.00
YoY Change
Other Investing Activities -$1.170M -$52.00M $16.22M
YoY Change -104.75% -313.38% -84.74%
Cash From Investing Activities -$1.648M -$54.04M $13.12M
YoY Change -106.75% -333.64% -87.45%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net $0.00 $0.00
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities $1.136M 1.850M $44.22M
YoY Change 71.34% 4525.0% 4467.98%
NET CHANGE
Cash From Operating Activities -$15.88M 21.29M -$10.06M
Cash From Investing Activities -$1.648M -54.04M $13.12M
Cash From Financing Activities $1.136M 1.850M $44.22M
Net Change In Cash -$16.39M -30.90M $53.47M
YoY Change 328.95% -413.71% 2739.67%
FREE CASH FLOW
Cash From Operating Activities -$15.88M $21.29M -$10.06M
Capital Expenditures $479.0K -$2.040M $3.101M
Free Cash Flow -$16.36M $23.33M -$13.16M
YoY Change -43.84% -293.13% -87.5%

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CY2017 us-gaap Deferred Income Tax Expense Benefit
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199000 USD
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CY2017Q4 us-gaap Deferred Income Tax Liabilities
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0 USD
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3937000 USD
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1587000 USD
CY2017Q4 us-gaap Deferred Revenue Noncurrent
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7954000 USD
CY2015 us-gaap Deferred State And Local Income Tax Expense Benefit
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0 USD
CY2016 us-gaap Deferred State And Local Income Tax Expense Benefit
DeferredStateAndLocalIncomeTaxExpenseBenefit
0 USD
CY2017 us-gaap Deferred State And Local Income Tax Expense Benefit
DeferredStateAndLocalIncomeTaxExpenseBenefit
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CY2017Q4 us-gaap Deferred Tax Assets Gross
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0 USD
CY2017Q4 us-gaap Deferred Tax Assets Liabilities Net
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0 USD
CY2016Q4 us-gaap Deferred Tax Assets Net
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19425000 USD
CY2017Q4 us-gaap Deferred Tax Assets Net
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10067000 USD
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231500000 USD
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80061000 USD
CY2017Q4 us-gaap Deferred Tax Assets Operating Loss Carryforwards Domestic
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61068000 USD
CY2017Q4 us-gaap Deferred Tax Assets Operating Loss Carryforwards Not Subject To Expiration
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169200000 USD
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9672000 USD
CY2017Q4 us-gaap Deferred Tax Assets Operating Loss Carryforwards State And Local
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11884000 USD
CY2017Q4 us-gaap Deferred Tax Assets Operating Loss Carryforwards Subject To Expiration
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62300000 USD
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22125000 USD
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18815000 USD
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2968000 USD
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1749000 USD
CY2017Q4 us-gaap Deferred Tax Assets Tax Credit Carryforwards General Business
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67800000 USD
CY2016Q4 us-gaap Deferred Tax Assets Tax Deferred Expense Reserves And Accruals Accrued Liabilities
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849000 USD
CY2017Q4 us-gaap Deferred Tax Assets Tax Deferred Expense Reserves And Accruals Accrued Liabilities
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625000 USD
CY2016Q4 us-gaap Deferred Tax Assets Valuation Allowance
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183000000 USD
CY2016Q4 us-gaap Deferred Tax Assets Valuation Allowance
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CY2017Q4 us-gaap Deferred Tax Assets Valuation Allowance
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CY2017Q4 us-gaap Deferred Tax Assets Valuation Allowance
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235000 USD
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140000 USD
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700000 USD
CY2016 us-gaap Defined Contribution Plan Cost Recognized
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1100000 USD
CY2017 us-gaap Defined Contribution Plan Cost Recognized
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1600000 USD
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0.060
CY2017 us-gaap Defined Contribution Plan Employer Matching Contribution Percent Of Match
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0.84
CY2015 us-gaap Depreciation
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2900000 USD
CY2016 us-gaap Depreciation
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3300000 USD
CY2017 us-gaap Depreciation
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2300000 USD
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2876000 USD
CY2016 us-gaap Depreciation Depletion And Amortization
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3290000 USD
CY2017 us-gaap Depreciation Depletion And Amortization
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17682000 USD
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-639000 USD
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-1584000 USD
CY2017 us-gaap Effect Of Exchange Rate On Cash And Cash Equivalents
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6199000 USD
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CY2016 us-gaap Effective Income Tax Rate Continuing Operations
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-0.0040
CY2017 us-gaap Effective Income Tax Rate Continuing Operations
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-0.0172
CY2015 us-gaap Effective Income Tax Rate Reconciliation At Federal Statutory Income Tax Rate
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0.34
CY2016 us-gaap Effective Income Tax Rate Reconciliation At Federal Statutory Income Tax Rate
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0.34
CY2017 us-gaap Effective Income Tax Rate Reconciliation At Federal Statutory Income Tax Rate
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0.34
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-0.2087
CY2016 us-gaap Effective Income Tax Rate Reconciliation Change In Deferred Tax Assets Valuation Allowance
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-0.3072
CY2017 us-gaap Effective Income Tax Rate Reconciliation Change In Deferred Tax Assets Valuation Allowance
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0.2910
CY2015 us-gaap Effective Income Tax Rate Reconciliation Foreign Income Tax Rate Differential
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-0.2406
CY2016 us-gaap Effective Income Tax Rate Reconciliation Foreign Income Tax Rate Differential
EffectiveIncomeTaxRateReconciliationForeignIncomeTaxRateDifferential
-0.1984
CY2017 us-gaap Effective Income Tax Rate Reconciliation Foreign Income Tax Rate Differential
EffectiveIncomeTaxRateReconciliationForeignIncomeTaxRateDifferential
-0.1033
CY2015 us-gaap Effective Income Tax Rate Reconciliation Nondeductible Expense Research And Development
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0.1950
CY2016 us-gaap Effective Income Tax Rate Reconciliation Nondeductible Expense Research And Development
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0.1666
CY2017 us-gaap Effective Income Tax Rate Reconciliation Nondeductible Expense Research And Development
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0.1953
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-0.0182
CY2016 us-gaap Effective Income Tax Rate Reconciliation Other Adjustments
EffectiveIncomeTaxRateReconciliationOtherAdjustments
0.0001
CY2017 us-gaap Effective Income Tax Rate Reconciliation Other Adjustments
EffectiveIncomeTaxRateReconciliationOtherAdjustments
-0.0015
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EffectiveIncomeTaxRateReconciliationOtherReconcilingItemsPercent
0.0000
CY2016 us-gaap Effective Income Tax Rate Reconciliation Other Reconciling Items Percent
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0.0014
CY2017 us-gaap Effective Income Tax Rate Reconciliation Other Reconciling Items Percent
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-0.0026
CY2015 us-gaap Effective Income Tax Rate Reconciliation State And Local Income Taxes
EffectiveIncomeTaxRateReconciliationStateAndLocalIncomeTaxes
-0.0043
CY2016 us-gaap Effective Income Tax Rate Reconciliation State And Local Income Taxes
EffectiveIncomeTaxRateReconciliationStateAndLocalIncomeTaxes
0.0305
CY2017 us-gaap Effective Income Tax Rate Reconciliation State And Local Income Taxes
EffectiveIncomeTaxRateReconciliationStateAndLocalIncomeTaxes
-0.0101
CY2016Q4 us-gaap Employee Related Liabilities Current
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17711000 USD
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38200000 USD
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EmployeeServiceShareBasedCompensationNonvestedAwardsTotalCompensationCostNotYetRecognizedPeriodForRecognition1
P1Y10M28D
CY2015 us-gaap Fair Value Adjustment Of Warrants
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-140000 USD
CY2016 us-gaap Fair Value Adjustment Of Warrants
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-47000 USD
CY2017 us-gaap Fair Value Adjustment Of Warrants
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0 USD
CY2015 us-gaap Foreign Currency Transaction Gain Loss Unrealized
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0 USD
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CY2017 us-gaap Foreign Currency Transaction Gain Loss Unrealized
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459000 USD
CY2015 us-gaap Gain Loss On Disposition Of Assets1
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37000 USD
CY2016 us-gaap Gain Loss On Disposition Of Assets1
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0 USD
CY2017 us-gaap Gain Loss On Disposition Of Assets1
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CY2017 us-gaap General And Administrative Expense
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121271000 USD
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-169962000 USD
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CY2016 us-gaap Income Loss From Continuing Operations Before Income Taxes Foreign
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CY2017 us-gaap Income Loss From Continuing Operations Before Income Taxes Foreign
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-169962000 USD
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-141541000 USD
CY2017 us-gaap Income Loss From Continuing Operations Before Income Taxes Minority Interest And Income Loss From Equity Method Investments
IncomeLossFromContinuingOperationsBeforeIncomeTaxesMinorityInterestAndIncomeLossFromEquityMethodInvestments
-77665000 USD
CY2015 us-gaap Income Tax Expense Benefit
IncomeTaxExpenseBenefit
485000 USD
CY2016 us-gaap Income Tax Expense Benefit
IncomeTaxExpenseBenefit
569000 USD
CY2017 us-gaap Income Tax Expense Benefit
IncomeTaxExpenseBenefit
1335000 USD
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111000 USD
CY2016 us-gaap Income Taxes Paid Net
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943000 USD
CY2017 us-gaap Income Taxes Paid Net
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1101000 USD
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16981000 USD
CY2016 us-gaap Increase Decrease In Accounts Payable And Accrued Liabilities
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4259000 USD
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24011000 USD
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-3307000 USD
CY2016 us-gaap Increase Decrease In Deferred Revenue
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1526000 USD
CY2017 us-gaap Increase Decrease In Deferred Revenue
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9469000 USD
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-131000 USD
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278000 USD
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544000 USD
CY2015 us-gaap Increase Decrease In Inventories
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0 USD
CY2016 us-gaap Increase Decrease In Inventories
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0 USD
CY2017 us-gaap Increase Decrease In Inventories
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6454000 USD
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-92000 USD
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733000 USD
CY2015 us-gaap Increase Decrease In Prepaid Deferred Expense And Other Assets
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2107000 USD
CY2016 us-gaap Increase Decrease In Prepaid Deferred Expense And Other Assets
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-1171000 USD
CY2017 us-gaap Increase Decrease In Prepaid Deferred Expense And Other Assets
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1784000 USD
CY2015 us-gaap Increase Decrease In Receivables
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6907000 USD
CY2016 us-gaap Increase Decrease In Receivables
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14842000 USD
CY2017 us-gaap Increase Decrease In Receivables
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12203000 USD
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0 USD
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CY2017 us-gaap Interest Income Expense Nonoperating Net
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CY2015 us-gaap Interest Paid
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CY2016 us-gaap Interest Paid
InterestPaid
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CY2017 us-gaap Interest Paid
InterestPaid
6271000 USD
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0 USD
CY2017Q4 us-gaap Inventory Finished Goods
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6390000 USD
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CY2017Q4 us-gaap Inventory Net
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10754000 USD
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0 USD
CY2017Q4 us-gaap Inventory Raw Materials
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452000 USD
CY2016Q4 us-gaap Inventory Work In Process
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0 USD
CY2017Q4 us-gaap Inventory Work In Process
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3912000 USD
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149762000 USD
CY2017Q4 us-gaap Liabilities
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235216000 USD
CY2016Q4 us-gaap Liabilities And Stockholders Equity
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269345000 USD
CY2017Q4 us-gaap Liabilities And Stockholders Equity
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391653000 USD
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49624000 USD
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82048000 USD
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0 USD
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144971000 USD
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79454000 USD
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104481000 USD
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CY2016Q4 us-gaap Net Income Loss
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CY2016 us-gaap Net Income Loss
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CY2017Q1 us-gaap Net Income Loss
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CY2017Q2 us-gaap Net Income Loss
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CY2017Q3 us-gaap Net Income Loss
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CY2017Q4 us-gaap Net Income Loss
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1270000 USD
CY2017 us-gaap Net Income Loss
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1 segment
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203896000 USD
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57337000 USD
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CY2016Q3 us-gaap Operating Expenses
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CY2016Q4 us-gaap Operating Expenses
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CY2016 us-gaap Operating Expenses
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CY2017Q3 us-gaap Operating Expenses
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CY2017Q4 us-gaap Operating Expenses
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72578000 USD
CY2017 us-gaap Operating Expenses
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258684000 USD
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652000 USD
CY2016Q2 us-gaap Operating Income Loss
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CY2016Q3 us-gaap Operating Income Loss
OperatingIncomeLoss
-32064000 USD
CY2016Q4 us-gaap Operating Income Loss
OperatingIncomeLoss
-24992000 USD
CY2016 us-gaap Operating Income Loss
OperatingIncomeLoss
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CY2017Q1 us-gaap Operating Income Loss
OperatingIncomeLoss
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CY2017Q2 us-gaap Operating Income Loss
OperatingIncomeLoss
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CY2017Q3 us-gaap Operating Income Loss
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CY2017Q4 us-gaap Operating Income Loss
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5452000 USD
CY2017 us-gaap Operating Income Loss
OperatingIncomeLoss
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6688000 USD
CY2017Q4 us-gaap Operating Leases Future Minimum Payments Due Current
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2110000 USD
CY2017Q4 us-gaap Operating Leases Future Minimum Payments Due In Four Years
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712000 USD
CY2017Q4 us-gaap Operating Leases Future Minimum Payments Due In Three Years
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845000 USD
CY2017Q4 us-gaap Operating Leases Future Minimum Payments Due In Two Years
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1095000 USD
CY2017Q4 us-gaap Operating Leases Future Minimum Payments Due Thereafter
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1274000 USD
CY2015 us-gaap Operating Leases Rent Expense Net
OperatingLeasesRentExpenseNet
1800000 USD
CY2016 us-gaap Operating Leases Rent Expense Net
OperatingLeasesRentExpenseNet
2200000 USD
CY2017 us-gaap Operating Leases Rent Expense Net
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2200000 USD
CY2016Q4 us-gaap Other Accrued Liabilities Current
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2825000 USD
CY2017Q4 us-gaap Other Accrued Liabilities Current
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2286000 USD
CY2015 us-gaap Other Comprehensive Income Availableforsale Securities Adjustment Net Of Tax Portion Attributable To Parent
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-202000 USD
CY2016 us-gaap Other Comprehensive Income Availableforsale Securities Adjustment Net Of Tax Portion Attributable To Parent
OtherComprehensiveIncomeAvailableforsaleSecuritiesAdjustmentNetOfTaxPortionAttributableToParent
386000 USD
CY2017 us-gaap Other Comprehensive Income Availableforsale Securities Adjustment Net Of Tax Portion Attributable To Parent
OtherComprehensiveIncomeAvailableforsaleSecuritiesAdjustmentNetOfTaxPortionAttributableToParent
225000 USD
CY2015 us-gaap Other Comprehensive Income Foreign Currency Transaction And Translation Adjustment Net Of Tax Portion Attributable To Parent
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-261000 USD
CY2016 us-gaap Other Comprehensive Income Foreign Currency Transaction And Translation Adjustment Net Of Tax Portion Attributable To Parent
OtherComprehensiveIncomeForeignCurrencyTransactionAndTranslationAdjustmentNetOfTaxPortionAttributableToParent
-671000 USD
CY2017 us-gaap Other Comprehensive Income Foreign Currency Transaction And Translation Adjustment Net Of Tax Portion Attributable To Parent
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CY2017 us-gaap Concentration Risk Credit Risk
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<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:10px;padding-top:10px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Concentration of credit risk</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-indent:32px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The Company&#8217;s financial instruments that are exposed to credit risks consist primarily of cash and cash equivalents, available-for-sale marketable securities and accounts receivable. The Company maintains its cash and cash equivalents in bank accounts, which, at times, exceed federally insured limits. The Company has not experienced any credit losses in these accounts and does not believe it is exposed to any significant credit risk on these funds. The Company&#8217;s investment policy includes guidelines on the quality of the financial institutions and financial instruments the Company is allowed to invest in, which the Company believes minimizes the exposure to concentration of credit risk.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-indent:32px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The Company is subject to credit risk from its accounts receivable related to its product sales. The payment terms are predetermined and the Company evaluates the creditworthiness of each customer or distributor on a regular basis. The Company reserves all uninsured amounts billed directly to a patient until the time of cash receipt as collectability is not reasonably assured at the time the product is received. To date, the Company has not incurred any credit losses.</font></div></div>
CY2017 us-gaap Nature Of Operations
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<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:10px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">The Company</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">PTC Therapeutics,&#160;Inc. (the &#8220;Company&#8221; or &#8220;PTC&#8221;) are a science-led global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. The Company has launched two rare disease products and has a global commercial footprint. The Company&#8217;s recent ability to commercialize products is the foundation that drives its continued investment in a robust pipeline of transformative medicines and its mission to provide access to best-in-class treatments for patients who have an unmet medical need.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The Company has two products, Translarna</font><font style="font-family:inherit;font-size:8pt;">&#8482; </font><font style="font-family:inherit;font-size:10pt;">(ataluren) and Emflaza&#8482; (deflazacort), for the treatment of Duchenne muscular dystrophy, or DMD, a rare, life threatening disorder. Translarna received marketing authorization from the European Commission in August 2014 for the treatment of nonsense mutation Duchenne muscular dystrophy, or nmDMD, in ambulatory patients aged </font><font style="font-family:inherit;font-size:10pt;">5</font><font style="font-family:inherit;font-size:10pt;">&#160;years and over in the </font><font style="font-family:inherit;font-size:10pt;">31</font><font style="font-family:inherit;font-size:10pt;"> member states of the European Economic Area, or EEA. Emflaza is approved in the United States for the treatment of DMD in patients five years and older. </font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The Company&#8217;s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the European Commission following reassessment by the European Medicines Agency, or EMA, of the benefit-risk balance of the authorization, which the Company refers to as the annual EMA reassessment. This marketing authorization is further subject to the specific obligation to conduct and submit the results of a multi-center, randomized, double-blind, 18-month, placebo-controlled trial, followed by an 18-month open-label extension, according to an agreed protocol, in order to confirm the efficacy and safety of Translarna in the approved patient population. The final report on the trial and open-label extension is to be submitted by the Company to the EMA by the end of the third quarter of 2021. The Company refers to the trial and open-label extension together as Study 041.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The marketing authorization in the EEA was last renewed in June 2017 and is effective, unless extended, through August 5, 2018. In February 2018, the Company submitted a marketing authorization renewal request to the EMA. The renewal was based on the Company&#8217;s commitment to conduct Study 041 and the totality of the clinical data available from its trials and studies of Translarna for the treatment of nmDMD, including the safety and efficacy results of the Phase 2b and Phase 3 clinical trials. The primary efficacy endpoint was not achieved in either trial within the pre-specified level of statistical significance.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">In June 2014, the Company initiated reimbursed early access programs, or EAP programs, for Translarna for nmDMD patients in selected territories in the EEA and recorded its first sales of Translarna in the third quarter of 2014 pursuant to an EAP program. In December 2014, the Company recorded its first commercial sales in Germany. As of </font><font style="font-family:inherit;font-size:10pt;">December&#160;31, 2017</font><font style="font-family:inherit;font-size:10pt;">, Translarna was available in </font><font style="font-family:inherit;font-size:10pt;">over 25</font><font style="font-family:inherit;font-size:10pt;"> countries on a commercial basis or pursuant to the EAP program. The Company expects to expand its launch activities across the EEA pursuant to the marketing authorization granted by the EMA throughout 2018 and future years, subject to continued renewal of its marketing authorization following annual EMA reassessments and successful completion of pricing and reimbursement negotiations. Concurrently, the Company plans to continue to pursue EAP programs in select countries where those mechanisms exist, both within the EEA and in other countries that will reference the marketing authorization in the EEA.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Translarna is an investigational new drug in the United States. During the first quarter of 2017, the Company filed a New Drug Application, or NDA, over protest with the United States Food and Drug Administration, (the "FDA"), for which the FDA granted a standard review. In October 2017, the Office of Drug Evaluation I of the FDA issued a complete response letter for the NDA, stating that it was unable to approve the application in its current form. In response, the Companyfiled a formal dispute resolution request with the Office of New Drugs of the FDA. In February 2018, the Office of New Drugs of the FDA denied PTC&#8217;s appeal of the Complete Response Letter. In its response, the Office of New Drugs recommended a possible path forward for the ataluren NDA submission based on the accelerated approval pathway. This would involve a re-submission of an NDA containing the current data on effectiveness of ataluren with new data to be generated on dystrophin production in nmDMD patients&#8217; muscles. The Company intends to follow the FDA&#8217;s recommendation and will collect such dystrophin data using newer technologies via procedures and methods that will be mutually agreeable to the Company and the FDA. Additionally, should a re-submission of an NDA receive accelerated approval, the Office of New Drugs stated that Study 041, which is currently enrolling, could serve as the confirmatory post-approval trial required in connection with the accelerated approval framework.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The NDA, which seeks approval of Translarna for the treatment of nmDMD in the United States, was initially submitted by the Company in December 2015. In February 2016, following the submission, the Company received a Refuse to File letter from the FDA regarding the NDA. The FDA stated in the Refuse to File letter that the NDA was not sufficiently complete to permit a substantive review. Specifically, the Company was notified in the letter that, in the view of the FDA, both the Phase&#160;2b and Phase&#160;3 ACT DMD trials were negative and do not provide substantial evidence of effectiveness and that the NDA did not contain adequate information regarding the abuse potential of Translarna. Additionally, the FDA stated that the Company had proposed a post-hoc adjustment of ACT DMD that eliminates data from a majority of enrolled patients. During July 2016, the Company appealed the Refuse to File decision via the formal dispute resolution process within FDA&#8217;s Center for Drug Evaluation and Research; however, this appeal was denied by the FDA&#8217;s Office of Drug Evaluation I in October 2016. </font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">On April 20, 2017, the Company completed its acquisition of all rights to Emflaza, or the Transaction. Emflaza is approved in the United States for the treatment of DMD in patients five years and older. The Transaction was completed pursuant to an asset purchase agreement, dated March 15, 2017, as amended on April 20, 2017, (the "Asset Purchase Agreement"), by and between the Company and Marathon Pharmaceuticals, LLC (now known as Complete Pharma Holdings, LLC), or Marathon. The Transaction was accounted for as an asset acquisition. The assets acquired by the Company in the Transaction include intellectual property rights related to Emflaza, inventories of Emflaza, and certain contractual rights related to Emflaza. The Company assumed certain liabilities and obligations in the Transaction arising out of, or relating to, the assets acquired in the Transaction. </font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;text-indent:48px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">Upon the closing of the Transaction, the Company paid to Marathon total upfront consideration comprised of </font><font style="font-family:inherit;font-size:10pt;">$75.0 million</font><font style="font-family:inherit;font-size:10pt;"> in cash, funded through cash on hand, and </font><font style="font-family:inherit;font-size:10pt;">6,683,598</font><font style="font-family:inherit;font-size:10pt;"> shares of the Company&#8217;s common stock. The number of shares of common stock issued at closing was determined by dividing </font><font style="font-family:inherit;font-size:10pt;">$65.0 million</font><font style="font-family:inherit;font-size:10pt;"> by the volume weighted average price per share of the Company&#8217;s common stock on the Nasdaq Stock Market for the </font><font style="font-family:inherit;font-size:10pt;">15</font><font style="font-family:inherit;font-size:10pt;"> trading-day period ending on the third trading day immediately preceding the closing. Marathon will be entitled to receive contingent payments from the Company based on annual net sales of Emflaza beginning in 2018, up to a specified aggregate maximum amount over the expected commercial life of the asset, and a single </font><font style="font-family:inherit;font-size:10pt;">$50.0 million</font><font style="font-family:inherit;font-size:10pt;"> sales-based milestone, in each case subject to the terms and conditions of the Asset Purchase Agreement.</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-indent:32px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">As of </font><font style="font-family:inherit;font-size:10pt;">December&#160;31, 2017</font><font style="font-family:inherit;font-size:10pt;">, the Company had an accumulated deficit of approximately </font><font style="font-family:inherit;font-size:10pt;">$814.1 million</font><font style="font-family:inherit;font-size:10pt;">. The Company has financed its operations to date primarily through the private offering in August 2015 of </font><font style="font-family:inherit;font-size:10pt;">3.00%</font><font style="font-family:inherit;font-size:10pt;"> convertible senior notes due 2022 (see Note&#160;7), public offerings of common stock in February 2014 and October 2014, its initial public offering of common stock in June 2013, private placements of its convertible preferred stock, collaborations, bank debt, convertible debt financings, grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company&#8217;s product candidates.</font></div></div>
CY2017 us-gaap Use Of Estimates
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<div style="font-family:Times New Roman;font-size:10pt;"><div style="line-height:120%;padding-bottom:10px;padding-top:10px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;font-weight:bold;">Use of estimates</font></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-indent:32px;font-size:10pt;"><font style="font-family:inherit;font-size:10pt;">The preparation of financial statements in conformity with GAAP requires management to make estimates and assumptions that affect the amounts reported in the financial statements and accompanying notes. Significant estimates in these consolidated financial statements have been made in connection with the calculation of net product sales, certain accruals related to the Company&#8217;s research and development expenses, stock-based compensation, valuation procedures for the convertible notes, allowance for doubtful accounts, inventory, acquired intangible assets and the provision for or benefit from income taxes. Actual results could differ from those estimates. Changes in estimates are reflected in reported results in the period in which they become known.</font></div></div>

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