$PTCT
PTC Therapeutics Inc Stock $ptct

$ptct Overview Financials Valuation SEC Filings Company Website

2020 Q4 Form 10-Q Financial Statement

#000107008120000139 Filed on October 29, 2020

Income Statement

Concept	2020 Q4	2020 Q3	2019 Q3
Revenue	$118.9M	$118.4M	$71.42M
YoY Change	23.21%	65.79%	33.26%
Cost Of Revenue	$4.900M	$4.700M	$3.006M
YoY Change	40.0%	56.35%	-8.69%
Gross Profit	$114.0M	$113.7M	$68.41M
YoY Change	22.71%	66.2%	36.01%
Gross Profit Margin	95.88%	96.03%	95.79%
Selling, General & Admin	$75.50M	$57.84M	$49.28M
YoY Change	18.9%	17.36%	28.45%
% of Gross Profit	66.23%	50.87%	72.04%
Research & Development	$118.0M	$93.00M	$63.08M
YoY Change	44.25%	47.44%	16.02%
% of Gross Profit	103.51%	81.79%	92.2%
Depreciation & Amortization	$12.40M	$11.64M	$8.140M
YoY Change	33.62%	43.0%	14.97%
% of Gross Profit	10.88%	10.24%	11.9%
Operating Expenses	$215.2M	$173.5M	$131.9M
YoY Change	27.18%	31.57%	29.53%
Operating Profit	-$96.37M	-$55.13M	-$60.48M
YoY Change	32.41%	-8.84%	25.39%
Interest Expense	-$24.30M	-$21.04M	-$2.666M
YoY Change	341.82%	689.16%	-14.5%
% of Operating Profit
Other Income/Expense, Net	$58.90M	$28.77M	$2.800M
YoY Change	425.89%	927.36%	281.47%
Pretax Income	-$61.70M	-$47.40M	-$60.34M
YoY Change	-7.91%	-21.44%	19.22%
Income Tax	$12.60M	$22.29M	-$344.0K
% Of Pretax Income
Net Earnings	-$74.35M	-$69.69M	-$60.00M
YoY Change	-4.28%	16.16%	17.71%
Net Earnings / Revenue	-62.53%	-58.86%	-84.01%
Basic Earnings Per Share
Diluted Earnings Per Share	-$1.080M	-$1.031M	-$1.062M
COMMON SHARES
Basic Shares Outstanding	68.24M	67.70M shares	58.95M shares
Diluted Shares Outstanding

Balance Sheet

Concept	2020 Q4	2020 Q3	2019 Q3
SHORT-TERM ASSETS
Cash & Short-Term Investments	$1.104B	$1.141B	$708.6M
YoY Change	60.75%	61.02%	184.12%
Cash & Equivalents	$208.8M	$434.0M	$523.6M
Short-Term Investments	$894.8M	$707.0M	$185.1M
Other Short-Term Assets	$39.50M	$26.90M	$20.70M
YoY Change	120.67%	29.95%	158.75%
Inventory	$18.70M	$19.48M	$17.11M
Prepaid Expenses
Receivables	$69.90M	$56.80M	$60.40M
Other Receivables	$0.00	$0.00	$0.00
Total Short-Term Assets	$1.232B	$1.244B	$806.9M
YoY Change	58.06%	54.2%	157.55%
LONG-TERM ASSETS
Property, Plant & Equipment	$33.83M	$28.14M	$18.48M
YoY Change	57.0%	52.25%	109.93%
Goodwill	$82.34M	$82.30M	$82.30M
YoY Change	0.0%	0.0%	0.0%
Intangibles	$715.3M	$712.5M	$702.9M
YoY Change	0.68%	1.37%	16.26%
Long-Term Investments
YoY Change
Other Assets	$60.60M	$28.10M	$17.20M
YoY Change	269.51%	63.37%	975.0%
Total Long-Term Assets	$976.5M	$936.9M	$834.6M
YoY Change	15.63%	12.25%	16.67%
TOTAL ASSETS
Total Short-Term Assets	$1.232B	$1.244B	$806.9M
Total Long-Term Assets	$976.5M	$936.9M	$834.6M
Total Assets	$2.208B	$2.181B	$1.641B
YoY Change	36.0%	32.87%	59.58%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable	$18.67M	$15.66M	$8.959M
YoY Change	80.79%	74.83%	37.03%
Accrued Expenses	$92.00M	$68.00M	$50.70M
YoY Change	31.43%	34.12%	181.67%
Deferred Revenue	$4.151M	$5.992M	$8.810M
YoY Change	-49.64%	-31.99%	339.62%
Short-Term Debt	$0.00	$0.00	$0.00
YoY Change
Long-Term Debt Due	$22.30M	$0.00	$20.00M
YoY Change	11.5%	-100.0%	199.99%
Total Short-Term Liabilities	$277.3M	$253.8M	$183.5M
YoY Change	17.59%	38.32%	59.67%
LONG-TERM LIABILITIES
Long-Term Debt	$309.1M	$303.0M	$293.1M
YoY Change	5.2%	3.35%	103.21%
Other Long-Term Liabilities	$1.392M	$1.968M	$9.455M
YoY Change	887.23%	-79.19%	9261.39%
Total Long-Term Liabilities	$310.5M	$304.9M	$302.6M
YoY Change	5.62%	0.77%	109.62%
TOTAL LIABILITIES
Total Short-Term Liabilities	$277.3M	$253.8M	$183.5M
Total Long-Term Liabilities	$310.5M	$304.9M	$302.6M
Total Liabilities	$1.726B	$1.670B	$977.8M
YoY Change	67.69%	70.8%	52.18%
SHAREHOLDERS EQUITY
Retained Earnings	-$1.629B	-$1.555B	-$1.113B
YoY Change	36.82%	39.69%	24.95%
Common Stock	$70.00K	$68.00K	$61.00K
YoY Change	12.9%	11.48%	19.61%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity	$482.0M	$511.0M	$663.7M
YoY Change
Total Liabilities & Shareholders Equity	$2.208B	$2.181B	$1.641B
YoY Change	36.0%	32.87%	59.58%

Cashflow Statement

Concept	2020 Q4	2020 Q3	2019 Q3
OPERATING ACTIVITIES
Net Income	-$74.35M	-$69.69M	-$60.00M
YoY Change	-4.28%	16.16%	17.71%
Depreciation, Depletion And Amortization	$12.40M	$11.64M	$8.140M
YoY Change	33.62%	43.0%	14.97%
Cash From Operating Activities	-$64.36M	$26.65M	-$15.50M
YoY Change	530.36%	-271.94%	-558.58%
INVESTING ACTIVITIES
Capital Expenditures	-$6.350M	-$3.310M	-$4.250M
YoY Change	36.27%	-22.12%	226.92%
Acquisitions
YoY Change
Other Investing Activities	-$201.8M	-$390.9M	$34.68M
YoY Change	-10.06%	-1227.19%	-252.04%
Cash From Investing Activities	-$208.1M	-$394.2M	$30.43M
YoY Change	-9.12%	-1395.47%	-226.16%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities	40.09M	643.1M	382.4M
YoY Change	1658.33%	68.18%	7426.77%
NET CHANGE
Cash From Operating Activities	-64.36M	26.65M	-15.50M
Cash From Investing Activities	-208.1M	-394.2M	30.43M
Cash From Financing Activities	40.09M	643.1M	382.4M
Net Change In Cash	-232.4M	275.5M	397.3M
YoY Change	-1.92%	-30.66%	-2636.97%
FREE CASH FLOW
Cash From Operating Activities	-$64.36M	$26.65M	-$15.50M
Capital Expenditures	-$6.350M	-$3.310M	-$4.250M
Free Cash Flow	-$58.01M	$29.96M	-$11.25M
YoY Change	945.23%	-366.31%	-340.38%

Facts In Submission

Frame	Concept Type	Concept / XBRL Key	Value
	dei	Entity Central Index Key EntityCentralIndexKey	0001070081
	dei	Current Fiscal Year End Date CurrentFiscalYearEndDate	--12-31
	dei	Document Fiscal Year Focus DocumentFiscalYearFocus	2020
	dei	Document Fiscal Period Focus DocumentFiscalPeriodFocus	Q3
	dei	Amendment Flag AmendmentFlag	false
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CY2019Q4	us-gaap	Common Stock Shares Outstanding CommonStockSharesOutstanding	61935870
CY2020Q3	us-gaap	Operating Lease Weighted Average Remaining Lease Term1 OperatingLeaseWeightedAverageRemainingLeaseTerm1	P5Y10M13D
CY2019Q4	us-gaap	Operating Lease Weighted Average Remaining Lease Term1 OperatingLeaseWeightedAverageRemainingLeaseTerm1	P3Y4M17D
CY2020Q3	us-gaap	Finance Lease Weighted Average Remaining Lease Term1 FinanceLeaseWeightedAverageRemainingLeaseTerm1	P12Y6M
CY2019Q4	us-gaap	Revenue Remaining Performance Obligation Expected Timing Of Satisfaction Period1 RevenueRemainingPerformanceObligationExpectedTimingOfSatisfactionPeriod1	P1Y
	dei	Document Type DocumentType	10-Q
	dei	Document Quarterly Report DocumentQuarterlyReport	true
	dei	Document Period End Date DocumentPeriodEndDate	2020-09-30
	dei	Document Transition Report DocumentTransitionReport	false
	dei	Entity File Number EntityFileNumber	001-35969
	dei	Entity Registrant Name EntityRegistrantName	PTC Therapeutics, Inc.
	dei	Entity Incorporation State Country Code EntityIncorporationStateCountryCode	DE
	dei	Entity Tax Identification Number EntityTaxIdentificationNumber	04-3416587
	dei	Entity Address Address Line1 EntityAddressAddressLine1	100 Corporate Court
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	us-gaap	Adjustments To Additional Paid In Capital Sharebased Compensation Requisite Service Period Recognition Value AdjustmentsToAdditionalPaidInCapitalSharebasedCompensationRequisiteServicePeriodRecognitionValue	48909000
	dei	Entity Address City Or Town EntityAddressCityOrTown	South Plainfield,
	dei	Entity Address State Or Province EntityAddressStateOrProvince	NJ
	dei	Entity Address Postal Zip Code EntityAddressPostalZipCode	07080
	dei	City Area Code CityAreaCode	908
	dei	Local Phone Number LocalPhoneNumber	222-7000
	dei	Security12b Title Security12bTitle	Common Stock, $0.001 par value per share
	dei	Trading Symbol TradingSymbol	PTCT
	dei	Security Exchange Name SecurityExchangeName	NASDAQ
	dei	Entity Current Reporting Status EntityCurrentReportingStatus	Yes
	dei	Entity Interactive Data Current EntityInteractiveDataCurrent	Yes
	dei	Entity Filer Category EntityFilerCategory	Large Accelerated Filer
	dei	Entity Small Business EntitySmallBusiness	false
	dei	Entity Emerging Growth Company EntityEmergingGrowthCompany	false
	dei	Entity Shell Company EntityShellCompany	false
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CY2020Q3	us-gaap	Stockholders Equity StockholdersEquity	510954000
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	us-gaap	Revenue From Contract With Customer Excluding Assessed Tax RevenueFromContractWithCustomerExcludingAssessedTax	261902000
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	us-gaap	Research And Development Expense ResearchAndDevelopmentExpense	359630000
	us-gaap	Research And Development Expense ResearchAndDevelopmentExpense	175621000
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	us-gaap	Selling General And Administrative Expense SellingGeneralAndAdministrativeExpense	169708000
	us-gaap	Selling General And Administrative Expense SellingGeneralAndAdministrativeExpense	139044000
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	us-gaap	Operating Income Loss OperatingIncomeLoss	-335394000
	us-gaap	Operating Income Loss OperatingIncomeLoss	-168374000
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	us-gaap	Interest Income Expense Nonoperating Net InterestIncomeExpenseNonoperatingNet	-7028000
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	us-gaap	Other Nonoperating Income Expense OtherNonoperatingIncomeExpense	2509000
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	us-gaap	Income Loss From Continuing Operations Before Income Taxes Extraordinary Items Noncontrolling Interest IncomeLossFromContinuingOperationsBeforeIncomeTaxesExtraordinaryItemsNoncontrollingInterest	-341212000
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	us-gaap	Net Income Loss NetIncomeLoss	-363806000
	us-gaap	Net Income Loss NetIncomeLoss	-173899000
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	us-gaap	Other Comprehensive Income Availableforsale Securities Adjustment Net Of Tax Portion Attributable To Parent OtherComprehensiveIncomeAvailableforsaleSecuritiesAdjustmentNetOfTaxPortionAttributableToParent	435000
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	us-gaap	Other Comprehensive Income Foreign Currency Transaction And Translation Adjustment Net Of Tax Portion Attributable To Parent OtherComprehensiveIncomeForeignCurrencyTransactionAndTranslationAdjustmentNetOfTaxPortionAttributableToParent	-26782000
	us-gaap	Other Comprehensive Income Foreign Currency Transaction And Translation Adjustment Net Of Tax Portion Attributable To Parent OtherComprehensiveIncomeForeignCurrencyTransactionAndTranslationAdjustmentNetOfTaxPortionAttributableToParent	-1281000
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	us-gaap	Comprehensive Income Net Of Tax ComprehensiveIncomeNetOfTax	-388949000
	us-gaap	Comprehensive Income Net Of Tax ComprehensiveIncomeNetOfTax	-174745000
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	us-gaap	Stock Issued During Period Value Stock Options Exercised StockIssuedDuringPeriodValueStockOptionsExercised	32987000
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	us-gaap	Stock Issued During Period Value New Issues StockIssuedDuringPeriodValueNewIssues	323756000
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	us-gaap	Stock Issued During Period Value Stock Options Exercised StockIssuedDuringPeriodValueStockOptionsExercised	12195000
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	us-gaap	Net Income Loss NetIncomeLoss	-173899000
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	us-gaap	Depreciation Depreciation	22903000
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	ptct	Noncash Royalty Revenue Sale Of Future Royalties NoncashRoyaltyRevenueSaleOfFutureRoyalties	0
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	us-gaap	Unrealized Gain Loss On Investments UnrealizedGainLossOnInvestments	2529000
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	ptct	Unrealized Gain Loss On Convertible Securities UnrealizedGainLossOnConvertibleSecurities	0
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	us-gaap	Paid In Kind Interest PaidInKindInterest	6583000
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	us-gaap	Amortization Of Financing Costs AmortizationOfFinancingCosts	434000
	us-gaap	Share Based Compensation ShareBasedCompensation	48909000
	us-gaap	Share Based Compensation ShareBasedCompensation	30668000
	us-gaap	Foreign Currency Transaction Gain Loss Unrealized ForeignCurrencyTransactionGainLossUnrealized	27077000
	us-gaap	Foreign Currency Transaction Gain Loss Unrealized ForeignCurrencyTransactionGainLossUnrealized	-205000
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	us-gaap	Increase Decrease In Inventories IncreaseDecreaseInInventories	1574000
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	us-gaap	Increase Decrease In Receivables IncreaseDecreaseInReceivables	-5879000
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	us-gaap	Payments To Acquire Intangible Assets PaymentsToAcquireIntangibleAssets	20250000
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	us-gaap	Cash Cash Equivalents Restricted Cash And Restricted Cash Equivalents Period Increase Decrease Including Exchange Rate Effect CashCashEquivalentsRestrictedCashAndRestrictedCashEquivalentsPeriodIncreaseDecreaseIncludingExchangeRateEffect	361579000
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	us-gaap	Interest Paid Net InterestPaidNet	9802000
	us-gaap	Interest Paid Net InterestPaidNet	7042000
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	ptct	Non Cash Public Offering Transaction Costs NonCashPublicOfferingTransactionCosts	506000
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	us-gaap	Nature Of Operations NatureOfOperations	<p style="font-family:'Times New Roman','Times','serif';font-size:10pt;font-weight:bold;margin:0pt;">PTC Therapeutics, Inc.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;font-weight:bold;margin:0pt;">Notes to Consolidated Financial Statements (unaudited)</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;font-weight:bold;margin:0pt;">September 30, 2020</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;font-weight:bold;margin:0pt 0pt 12pt 0pt;">In thousands (except per share data unless otherwise noted)</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;font-weight:bold;margin:0pt 0pt 12pt 0pt;">1. The Company</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">PTC Therapeutics, Inc. (the “Company” or “PTC”) is a science-driven global biopharmaceutical company focused on the discovery, development and commercialization of clinically-differentiated medicines that provide benefits to patients with rare disorders. The Company’s ability to globally commercialize products is the foundation that drives its continued investment in a robust diversified pipeline of transformative medicines and its mission to provide access to best-in-class treatments for patients who have an unmet medical need. The Company’s strategy is to bring best-in-class therapies with differentiated clinical benefit to patients affected by rare disorders and to leverage its global commercial infrastructure to maximize value for its patients and other stakeholders.</p><p style="font-family:'Times New Roman','Times','serif';font-size:8pt;text-align:justify;margin:0pt 0pt 12pt 0pt;"><span style="font-size:10pt;">The Company has two products, Translarna</span>™ <span style="font-size:10pt;">(ataluren) and Emflaza™ (deflazacort), for the treatment of Duchenne muscular dystrophy, or DMD, a rare, life threatening disorder. Translarna has marketing authorization in the European Economic Area (the “EEA”) for the treatment of nonsense mutation Duchenne muscular dystrophy (“nmDMD”), in ambulatory patients aged 2 years and older and in Brazil for the treatment of nmDMD in ambulatory patients aged 5 years and older, subject to annual renewal and other conditions. In July 2020, the European Commission approved the removal of the statement “efficacy has not been demonstrated in non-ambulatory patients” from the indication statement for Translarna. Emflaza is approved in the United States for the treatment of DMD in patients two years and older.</span></p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company has a pipeline of gene therapy product candidates for rare monogenic diseases that affect the central nervous system (“CNS”) including PTC-AADC for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC deficiency”), a rare CNS disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. The Company is preparing a biologics license application ("BLA") for PTC-AADC for the treatment of AADC deficiency in the United States and it anticipates submitting a BLA to the United States Food and Drug Administration ("FDA") in the first half of 2021. In January 2020, the Company submitted a marketing authorization application ("MAA") to the European Medicines Agency (“EMA”) for PTC-AADC for the treatment of AADC deficiency in the EEA, and the Company expects an opinion from the Committee for Medical Products for Human Use (“CHMP”) in the first half of 2021.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company holds the rights for the commercialization of Tegsedi™ (inotersen) and Waylivra™ (volanesorsen) for the treatment of rare diseases in countries in Latin America and the Caribbean pursuant to the Company’s Collaboration and License Agreement with Akcea Therapeutics, Inc. (“Akcea”). Tegsedi has received marketing authorization in the United States, the European Union (the “EU”) and Brazil for the treatment of stage 1 or stage 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (“hATTR amyloidosis”). Waylivra has received marketing authorization in the EU for the treatment of familial chylomicronemia syndrome (“FCS”). The Company filed for marketing authorization for Waylivra for the treatment of FCS with ANVISA, the Brazilian health regulatory authority, in June 2020 and, subject to potential delays in the review process related to the COVID-19 pandemic, expects a regulatory decision on approval from ANVISA in 2021.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company also has a spinal muscular atrophy ("SMA") collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., referred to collectively as Roche, and the Spinal Muscular Atrophy Foundation ("SMA Foundation"). The SMA program has one approved product, Evrysdi™ (risdiplam), which was approved in August 2020 by the FDA for the treatment of SMA in adults and children two months and older. Evrysdi also received marketing authorization for the treatment of SMA in Brazil in October 2020 and the EMA accepted the MAA filed by Roche for Evrysdi for the treatment of SMA in August 2020.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">On October 25, 2019, the Company completed the acquisition of substantially all of the assets of BioElectron Technology Corporation (“BioElectron”), a Delaware corporation, including certain compounds that the Company has begun to develop as part of its Bio-e platform, (the “Asset Acquisition”) pursuant to an asset purchase agreement by and between the Company and BioElectron, dated October 1, 2019 (the “Asset Acquisition Agreement”). The transaction was </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">accounted for as an asset acquisition. The two most advanced molecules in the Company’s Bio-e platform are vatiquinone and PTC857. The Company initiated a registration-directed Phase 2/3 placebo-controlled trial of vatiquinone in approximately 60 children with mitochondrial disease and associated refractory epilepsy in the third quarter of 2020. The Company expects to initiate a registration-directed Phase 3 trial of vatiquinone in approximately 100 patients with Friedrich ataxia in the fourth quarter of 2020. In the second quarter of 2020, the Company initiated a Phase 1 trial in healthy volunteers to evaluate the safety and pharmacology of PTC857, which the Company believes has the potential to address multiple CNS disorders with glucocerebrosidase Parkinson’s disease as its first planned indication. The Company completed dosing within the single ascending dose study and expects to complete dosing within the multiple ascending dose study from the Phase 1 trial by the end of 2020.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">In June 2020, the FDA authorized the initiation of a Phase 2/3 clinical trial evaluating PTC299, a dihydroorotate dehydrogenase inhibitor that the Company has also been developing in oncological indications, as a potential treatment for COVID-19. The integrated Phase 2/3 study, which has been initiated and is being conducted in two stages, will evaluate the efficacy and safety of PTC299 in patients hospitalized with SARS-CoV-2 infection. The Company expects Stage 1 of the clinical trial to be completed in the fourth quarter of 2020 and it anticipates reporting top-line results from both stages in the first half of 2021.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">In addition, the Company has a pipeline of product candidates and discovery programs that are in early clinical, pre-clinical and research and development stages focused on the development of new treatments for multiple therapeutic areas, including rare diseases and oncology.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company’s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the European Commission following reassessment by the 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. 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 2022. The Company refers to the trial and open-label extension together as Study 041.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The marketing authorization in the EEA was last renewed in June 2020 and is effective, unless extended, through August 5, 2021. The renewal was based on the Company’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.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">Translarna is an investigational new drug in the United States. During the first quarter of 2017, the Company filed a New Drug Application (“NDA”), over protest with 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 Company filed 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 the Company’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’ muscles. The Company intends to follow the FDA’s recommendation and will collect, using newer technologies via procedures and methods that the Company designed, such dystrophin data in a new study, Study 045, which the Company initiated in the fourth quarter of 2018. The final study muscle biopsies for the patients in Study 045 were completed in October 2020 and the Company has begun the process of analyzing the samples according to its protocol. The Company expects to report top-line results from Study 045 in the first quarter of 2021 followed by a potential resubmission of the NDA for Translarna for the treatment of nmDMD thereafter. 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.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">On August 23, 2018, the Company completed its acquisition of Agilis Biotherapeutics, Inc. (“Agilis”), pursuant to an Agreement and Plan of Merger, dated as of July 19, 2018 (the “Merger Agreement”), by and among the Company, Agility </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">Merger Sub, Inc., a Delaware corporation and the Company’s wholly owned, indirect subsidiary, Agilis and, solely in its capacity as the representative, agent and attorney-in-fact of the equityholders of Agilis, Shareholder Representative Services LLC, (the "Merger").</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">Upon the closing of the Merger, the Company paid to Agilis equityholders total upfront consideration comprised of $49.2 million in cash and 3,500,907 shares of the Company’s common stock (the “Closing Stock Consideration”). The Closing Stock Consideration was determined by dividing $150.0 million by the volume-weighted average price per share of the Company’s common stock on the Nasdaq Global Select Market for the <span style="-sec-ix-hidden:Hidden_6bGeDJgUIkyP7oopAFXUlQ"><span style="color:#000000;font-family:'Times New Roman','Times','serif';font-size:10pt;font-style:normal;font-weight:normal;text-align:justify;">10</span></span> consecutive trading-day period ending on the second trading-day immediately preceding the closing of the Merger. Agilis equityholders are entitled to receive contingent payments from the Company based on the achievement of certain development, regulatory and net sales milestones as well as based upon a percentage of net sales of certain products.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">On April 29, 2020, the Company, certain of the former equity holders of Agilis, and, for the limited purposes set forth in the agreement, Shareholder Representative Services LLC, entered into a Rights Exchange Agreement (the “Rights Exchange Agreement”). Pursuant to the Rights Exchange Agreement, the Company issued 2,821,176 shares of its common stock (the “Common Stock Consideration”) and paid $36.9 million (the “Cash Consideration”), in the aggregate, to such former equityholders of Agilis (the “Participating Rightholders”) in exchange for the cancellation and forfeiture by the Participating Rightholders of their rights to receive certain milestone-based contingent payments under the Merger Agreement, pursuant to which the Company completed the Merger.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">Pursuant to the terms of the Rights Exchange Agreement, the Participating Rightholders have canceled and forfeited their rights under the Merger Agreement to receive (i) $174.0 million, in the aggregate, of potential milestone payments based on the achievement of certain regulatory milestones and (ii) $37.6 million, in the aggregate, of $40.0 million in development milestone payments that would have been due upon the passing of the second anniversary of the closing of the Merger, regardless of whether the milestones were achieved.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Rights Exchange Agreement has no effect on the Merger Agreement other than to provide for the cancellation and forfeiture of the Participating Rightholders’ rights to receive $211.6 million, in the aggregate, of the milestone payments described above. As a result, all other rights and obligations under the Merger Agreement remain in effect pursuant to their terms, including the Company’s obligation to pay up to an aggregate maximum amount of $20.0 million upon the achievement of certain development milestones (representing the remaining portion of potential development milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement while excluding the remaining $2.4 million milestone payment that was due and paid in the three month period ended September 30, 2020 upon the passing of the second anniversary of the closing of the Merger), up to an aggregate maximum amount of $361.0 million upon the achievement of certain regulatory milestones (representing the remaining portion of potential regulatory milestone payments for which rights were not canceled and forfeited pursuant to the Rights Exchange Agreement), up to a maximum aggregate amount of $150.0 million upon the achievement of certain net sales milestones and a percentage of annual net sales for Friedreich ataxia and Angelman syndrome during specified terms, ranging from 2% to 6%, pursuant to the terms of the Merger Agreement. Refer to Note 5 for further details regarding the Rights Exchange Agreement.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">On May 29, 2020, the Company completed its acquisition of Censa Pharmaceuticals, Inc. (“Censa”) pursuant to an Agreement and Plan of Merger, dated as of May 5, 2020 (the "Censa Merger Agreement"), by and among the Company, Hydro Merger Sub, Inc., the Company’s wholly owned, indirect subsidiary, and, solely in its capacity as the representative, agent and attorney-in-fact of the securityholders of Censa, Shareholder Representative Services LLC (the "Censa Merger"). The transaction was accounted for as an asset acquisition. In connection with the Censa Merger, the Company acquired PTC923, which is being pursued as a possible treatment for orphan metabolic diseases associated with defects in the tetrahydrobiopterin biochemical pathways, including phenylketonuria ("PKU"). Refer to Note 3 for further details.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">As of September 30, 2020, the Company had an accumulated deficit of approximately $1,554.5 million. The Company has financed its operations to date primarily through the private offerings in September 2019 of 1.50% convertible senior notes due 2026 and in August 2015 of 3.00% convertible senior notes due 2022 (see Note 11), public offerings of common stock in February 2014, October 2014, April 2018, January 2019, and September 2019, "at the market offerings" of its common stock, its initial public offering of common stock in June 2013, proceeds from the Royalty Purchase Agreement </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">(see Note 2), private placements of its convertible preferred stock, collaborations, bank debt, the Company’s credit and security agreement (the "Credit Agreement"), with MidCap Financial Trust, or MidCap Financial, as administrative agent and MidCap Financial and other certain institutions as lenders thereto (see Note 11), grant funding and clinical trial support from governmental and philanthropic organizations and patient advocacy groups in the disease area addressed by the Company’s product candidates. Since 2014, the Company has also relied on revenue generated from net sales of Translarna for the treatment of nmDMD in territories outside of the United States, and since May 2017, the Company has generated revenue from net sales of Emflaza for the treatment of DMD in the United States. The Company expects that cash flows from the sales of its products, together with the Company’s cash, cash equivalents and marketable securities, will be sufficient to fund its operations for at least the next twelve months.</p>
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	us-gaap	Use Of Estimates UseOfEstimates	<p style="font-family:'Times New Roman','Times','serif';font-size:10pt;font-weight:bold;margin:0pt 0pt 12pt 0pt;">Use of estimates</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">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, royalty revenue, certain accruals related to the Company’s research and development expenses, valuation procedures for liability for sale of future royalties, valuation procedures for convertible notes, fair value of the contingent consideration, 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.</p>
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	us-gaap	Short Term Lease Cost ShortTermLeaseCost	340000
	us-gaap	Short Term Lease Cost ShortTermLeaseCost	235000
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CY2019Q3	us-gaap	Earnings Per Share Basic And Diluted EarningsPerShareBasicAndDiluted	-1.06
	us-gaap	Earnings Per Share Basic And Diluted EarningsPerShareBasicAndDiluted	-5.59
	us-gaap	Earnings Per Share Basic And Diluted EarningsPerShareBasicAndDiluted	-3.01
	us-gaap	Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount	12490036
	us-gaap	Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount	11393284
CY2020Q3	us-gaap	Allocated Share Based Compensation Expense AllocatedShareBasedCompensationExpense	16779000
CY2019Q3	us-gaap	Allocated Share Based Compensation Expense AllocatedShareBasedCompensationExpense	10484000
CY2020Q3	us-gaap	Amortization Of Intangible Assets AmortizationOfIntangibleAssets	9600000
	us-gaap	Allocated Share Based Compensation Expense AllocatedShareBasedCompensationExpense	30668000
CY2020Q3	us-gaap	Employee Service Share Based Compensation Nonvested Awards Total Compensation Cost Not Yet Recognized EmployeeServiceShareBasedCompensationNonvestedAwardsTotalCompensationCostNotYetRecognized	173200000
	us-gaap	Employee Service Share Based Compensation Nonvested Awards Total Compensation Cost Not Yet Recognized Period For Recognition1 EmployeeServiceShareBasedCompensationNonvestedAwardsTotalCompensationCostNotYetRecognizedPeriodForRecognition1	P2Y10M28D
CY2020Q3	ptct	Proceeds From Sale Of Future Royalties ProceedsFromSaleOfFutureRoyalties	650000000.0
CY2020Q3	us-gaap	Debt Instrument Interest Rate Effective Percentage DebtInstrumentInterestRateEffectivePercentage	0.110
CY2020Q3	ptct	Liability Sale Of Future Royalties Obligation Incurred LiabilitySaleOfFutureRoyaltiesObligationIncurred	650000000
	ptct	Liability Sale Of Future Royalties Obligation Incurred LiabilitySaleOfFutureRoyaltiesObligationIncurred	650000000
CY2020Q3	ptct	Liability Sale Of Future Royalties Royalty Revenue Payable LiabilitySaleOfFutureRoyaltiesRoyaltyRevenuePayable	299000
	ptct	Liability Sale Of Future Royalties Royalty Revenue Payable LiabilitySaleOfFutureRoyaltiesRoyaltyRevenuePayable	299000
CY2020Q3	ptct	Liability Sale Of Future Royalties Interest Expense LiabilitySaleOfFutureRoyaltiesInterestExpense	14557000
	ptct	Liability Sale Of Future Royalties Interest Expense LiabilitySaleOfFutureRoyaltiesInterestExpense	14557000
CY2020Q3	ptct	Liability Sale Of Future Royalties LiabilitySaleOfFutureRoyalties	664258000
CY2020Q3	ptct	Liability Sale Of Future Royalties LiabilitySaleOfFutureRoyalties	664258000
CY2020Q3	us-gaap	Debt Instrument Interest Rate Effective Percentage DebtInstrumentInterestRateEffectivePercentage	0.110
CY2020Q3	us-gaap	Debt Instrument Interest Rate Effective Percentage DebtInstrumentInterestRateEffectivePercentage	0.110
	ptct	Debt Instrument Additional Amount Available For Repurchase DebtInstrumentAdditionalAmountAvailableForRepurchase	37500000
	us-gaap	Number Of Operating Segments NumberOfOperatingSegments	1
CY2020Q3	ptct	Numberofdistributors Numberofdistributors	2
CY2019Q3	ptct	Numberofdistributors Numberofdistributors	2
CY2019Q3	ptct	Percentageofnetproductsales Percentageofnetproductsales	0.10
CY2020Q3	ptct	Percentageofnetproductsales Percentageofnetproductsales	0.10
	ptct	Numberofdistributors Numberofdistributors	2
	ptct	Numberofdistributors Numberofdistributors	2
	ptct	Percentageofnetproductsales Percentageofnetproductsales	0.10
	ptct	Percentageofnetproductsales Percentageofnetproductsales	0.10
CY2020Q3	us-gaap	Contract With Customer Liability ContractWithCustomerLiability	6000000.0
CY2019Q4	us-gaap	Contract With Customer Liability ContractWithCustomerLiability	11700000
CY2020Q3	us-gaap	Contract With Customer Liability Revenue Recognized ContractWithCustomerLiabilityRevenueRecognized	2000000.0
	us-gaap	Contract With Customer Liability Revenue Recognized ContractWithCustomerLiabilityRevenueRecognized	6000000.0
CY2019Q3	us-gaap	Contract With Customer Liability Revenue Recognized ContractWithCustomerLiabilityRevenueRecognized	2300000
	us-gaap	Contract With Customer Liability Revenue Recognized ContractWithCustomerLiabilityRevenueRecognized	2300000
CY2020Q3	us-gaap	Revenue Remaining Performance Obligation RevenueRemainingPerformanceObligation	6000000.0
CY2019Q4	us-gaap	Revenue Remaining Performance Obligation RevenueRemainingPerformanceObligation	11700000
CY2020Q3	us-gaap	Revenue Remaining Performance Obligation Expected Timing Of Satisfaction Period1 RevenueRemainingPerformanceObligationExpectedTimingOfSatisfactionPeriod1	P1Y
	us-gaap	Finite Lived Intangible Asset Useful Life FiniteLivedIntangibleAssetUsefulLife	P7Y
CY2019Q3	us-gaap	Amortization Of Intangible Assets AmortizationOfIntangibleAssets	7000000.0
	us-gaap	Amortization Of Intangible Assets AmortizationOfIntangibleAssets	26300000
	us-gaap	Amortization Of Intangible Assets AmortizationOfIntangibleAssets	19700000
CY2020Q3	us-gaap	Finite Lived Intangible Assets Amortization Expense Remainder Of Fiscal Year FiniteLivedIntangibleAssetsAmortizationExpenseRemainderOfFiscalYear	9627000
CY2020Q3	us-gaap	Finite Lived Intangible Assets Amortization Expense Next Twelve Months FiniteLivedIntangibleAssetsAmortizationExpenseNextTwelveMonths	38506000
CY2020Q3	us-gaap	Finite Lived Intangible Assets Amortization Expense Year Two FiniteLivedIntangibleAssetsAmortizationExpenseYearTwo	38506000
CY2020Q3	us-gaap	Finite Lived Intangible Assets Amortization Expense Year Three FiniteLivedIntangibleAssetsAmortizationExpenseYearThree	38506000
CY2020Q3	ptct	Finite Lived Intangible Asset Expected Amortization Year Four And Thereafter FiniteLivedIntangibleAssetExpectedAmortizationYearFourAndThereafter	10891000
CY2020Q3	us-gaap	Finite Lived Intangible Assets Net FiniteLivedIntangibleAssetsNet	136036000
	us-gaap	Indefinitelived Intangible Assets Acquired IndefinitelivedIntangibleAssetsAcquired	576500000
	us-gaap	Indefinite Lived Intangible Assets Period Increase Decrease IndefiniteLivedIntangibleAssetsPeriodIncreaseDecrease	0
CY2020Q3	us-gaap	Goodwill Goodwill	82300000
	us-gaap	Goodwill Period Increase Decrease GoodwillPeriodIncreaseDecrease	0
CY2020Q3	us-gaap	Goodwill Period Increase Decrease GoodwillPeriodIncreaseDecrease	0

Files In Submission

Name	View Source	Status
0001070081-20-000139-index-headers.html	Edgar Link	pending
0001070081-20-000139-index.html	Edgar Link	pending
0001070081-20-000139.txt	Edgar Link	pending
0001070081-20-000139-xbrl.zip	Edgar Link	pending
FilingSummary.xml	Edgar Link	unprocessable
Financial_Report.xlsx	Edgar Link	pending
MetaLinks.json	Edgar Link	pending
ptct-20200930.xsd	Edgar Link	pending
ptct-20200930ex101c45b08.htm	Edgar Link	pending
ptct-20200930ex1029e7222.htm	Edgar Link	pending
ptct-20200930ex311067de2.htm	Edgar Link	pending
ptct-20200930ex3123c96a2.htm	Edgar Link	pending
ptct-20200930ex3212402d0.htm	Edgar Link	pending
ptct-20200930ex322c3d0ad.htm	Edgar Link	pending
ptct-20200930x10q.htm	Edgar Link	pending
ptct-20200930x10q_htm.xml	Edgar Link	completed
ptct-20200930_cal.xml	Edgar Link	unprocessable
ptct-20200930_def.xml	Edgar Link	unprocessable
ptct-20200930_lab.xml	Edgar Link	unprocessable
ptct-20200930_pre.xml	Edgar Link	unprocessable
R1.htm	Edgar Link	pending
R10.htm	Edgar Link	pending
R11.htm	Edgar Link	pending
R12.htm	Edgar Link	pending
R13.htm	Edgar Link	pending
R14.htm	Edgar Link	pending
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R2.htm	Edgar Link	pending
R20.htm	Edgar Link	pending
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R27.htm	Edgar Link	pending
R28.htm	Edgar Link	pending
R29.htm	Edgar Link	pending
R3.htm	Edgar Link	pending
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R68.htm	Edgar Link	pending
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R7.htm	Edgar Link	pending
R70.htm	Edgar Link	pending
R71.htm	Edgar Link	pending
R8.htm	Edgar Link	pending
R9.htm	Edgar Link	pending
report.css	Edgar Link	pending
Show.js	Edgar Link	pending

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$PTCT
PTC Therapeutics Inc Stock $ptct

2020 Q4 Form 10-Q Financial Statement

#000107008120000139 Filed on October 29, 2020

Income Statement

Balance Sheet

Cashflow Statement

Facts In Submission

Files In Submission

$PTCT PTC Therapeutics Inc Stock $ptct

2020 Q4 Form 10-Q Financial Statement

#000107008120000139 Filed on October 29, 2020

Income Statement

Balance Sheet

Cashflow Statement

Facts In Submission

Files In Submission

$PTCT
PTC Therapeutics Inc Stock $ptct