$PTCT
PTC Therapeutics Inc

2022 Q1 Form 10-Q Financial Statement

#000107008122000017 Filed on May 03, 2022

View on sec.gov

Income Statement

Concept 2022 Q1 2021 Q1
Revenue $148.7M $117.9M
YoY Change 26.11% 72.79%
Cost Of Revenue $10.14M $9.100M
YoY Change 11.37% 121.95%
Gross Profit $138.6M $108.8M
YoY Change 27.39% 69.47%
Gross Profit Margin 93.19% 92.25%
Selling, General & Admin $73.27M $61.10M
YoY Change 19.93% 4.96%
% of Gross Profit 52.87% 56.15%
Research & Development $140.1M $134.5M
YoY Change 4.14% 49.28%
% of Gross Profit 101.07% 123.63%
Depreciation & Amortization $26.31M $13.59M
YoY Change 93.67% 47.13%
% of Gross Profit 18.99% 12.49%
Operating Expenses $235.3M $216.1M
YoY Change 8.87% 34.01%
Operating Profit -$86.52M -$98.15M
YoY Change -11.85% 5.55%
Interest Expense -$23.51M -$19.16M
YoY Change 22.73% 239.58%
% of Operating Profit
Other Income/Expense, Net -$11.86M -$10.88M
YoY Change 8.92% -21.31%
Pretax Income -$121.9M -$128.2M
YoY Change -4.91% 13.98%
Income Tax $4.835M $451.0K
% Of Pretax Income
Net Earnings -$126.7M -$128.6M
YoY Change -1.49% 14.16%
Net Earnings / Revenue -85.2% -109.07%
Basic Earnings Per Share -$1.78 -$1.83
Diluted Earnings Per Share -$1.78 -$1.83
COMMON SHARES
Basic Shares Outstanding 71.22M 70.19M
Diluted Shares Outstanding 71.22M 70.19M

Balance Sheet

Concept 2022 Q1 2021 Q1
SHORT-TERM ASSETS
Cash & Short-Term Investments $587.8M $988.4M
YoY Change -40.53% 65.87%
Cash & Equivalents $144.2M $146.6M
Short-Term Investments $443.6M $841.8M
Other Short-Term Assets $29.65M $23.60M
YoY Change 25.62% -2.88%
Inventory $15.28M $16.23M
Prepaid Expenses
Receivables $141.3M $94.10M
Other Receivables $0.00 $0.00
Total Short-Term Assets $774.0M $1.122B
YoY Change -31.04% 62.21%
LONG-TERM ASSETS
Property, Plant & Equipment $59.09M $37.12M
YoY Change 59.17% 41.86%
Goodwill $82.30M $82.30M
YoY Change 0.0% 0.0%
Intangibles $763.7M $712.6M
YoY Change 7.17% 1.03%
Long-Term Investments
YoY Change
Other Assets $44.40M $75.20M
YoY Change -40.96% 238.74%
Total Long-Term Assets $1.026B $989.6M
YoY Change 3.64% 15.92%
TOTAL ASSETS
Total Short-Term Assets $774.0M $1.122B
Total Long-Term Assets $1.026B $989.6M
Total Assets $1.800B $2.112B
YoY Change -14.79% 36.64%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $18.37M $19.11M
YoY Change -3.92% 18.91%
Accrued Expenses $79.52M $68.50M
YoY Change 16.08% 31.23%
Deferred Revenue $1.887M
YoY Change -78.15%
Short-Term Debt $0.00 $0.00
YoY Change
Long-Term Debt Due $149.7M $34.80M
YoY Change 330.23% 74.0%
Total Short-Term Liabilities $476.8M $250.9M
YoY Change 89.99% 15.29%
LONG-TERM LIABILITIES
Long-Term Debt $282.2M $430.0M
YoY Change -34.38% 45.99%
Other Long-Term Liabilities $300.6M $1.392M
YoY Change 21497.13% -90.59%
Total Long-Term Liabilities $282.2M $431.4M
YoY Change -34.6% 39.46%
TOTAL LIABILITIES
Total Short-Term Liabilities $476.8M $250.9M
Total Long-Term Liabilities $282.2M $431.4M
Total Liabilities $1.890B $1.819B
YoY Change 3.89% 78.92%
SHAREHOLDERS EQUITY
Retained Earnings -$2.225B -$1.703B
YoY Change 30.66% 30.64%
Common Stock $71.00K $70.00K
YoY Change 1.43% 12.9%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity -$90.58M $292.6M
YoY Change
Total Liabilities & Shareholders Equity $1.800B $2.112B
YoY Change -14.79% 36.64%

Cashflow Statement

Concept 2022 Q1 2021 Q1
OPERATING ACTIVITIES
Net Income -$126.7M -$128.6M
YoY Change -1.49% 14.16%
Depreciation, Depletion And Amortization $26.31M $13.59M
YoY Change 93.67% 47.13%
Cash From Operating Activities -$97.40M -$100.2M
YoY Change -2.75% 22.81%
INVESTING ACTIVITIES
Capital Expenditures $9.312M $5.669M
YoY Change 64.26% -5.88%
Acquisitions
YoY Change
Other Investing Activities $58.36M $35.82M
YoY Change 62.91% -137.61%
Cash From Investing Activities $49.04M $30.15M
YoY Change 62.68% -129.77%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 1.168M 9.531M
YoY Change -87.75% -48.45%
NET CHANGE
Cash From Operating Activities -97.40M -100.2M
Cash From Investing Activities 49.04M 30.15M
Cash From Financing Activities 1.168M 9.531M
Net Change In Cash -45.54M -62.18M
YoY Change -26.76% -62.26%
FREE CASH FLOW
Cash From Operating Activities -$97.40M -$100.2M
Capital Expenditures $9.312M $5.669M
Free Cash Flow -$106.7M -$105.8M
YoY Change 0.84% 20.84%

Facts In Submission

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<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. PTC’s ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC’s mission is to provide access to best-in-class treatments for patients who have few or no treatment options. PTC’s strategy is to leverage its strong scientific and clinical expertise and global commercial infrastructure to bring therapies to patients.  PTC believes that this allows it to maximize value for all of its stakeholders. </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">PTC has a portfolio pipeline that includes several commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focused on the development of new treatments for multiple therapeutic areas for rare diseases.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company has two products, Translarna™ (ataluren) and Emflaza® (deflazacort), for the treatment of Duchenne muscular dystrophy (“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 Russia for the treatment of nmDMD in patients aged two years and older. 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. Translarna also has marketing authorization in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become non-ambulatory. Emflaza is approved in the United States for the treatment of DMD in patients two years and older. </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 Collaboration and License Agreement (the “Tegsedi-Waylivra Agreement”), dated August 1, 2018, by and between the Company and Akcea Therapeutics, Inc. (“Akcea”), a subsidiary of Ionis Pharmaceuticals, Inc. 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”). The Company began to make commercial sales of Tegsedi for the treatment of hATTR amyloidosis in Brazil in the second quarter of 2022 and it continues to make Tegsedi available in certain other countries within Latin America and the Caribbean through early access programs (“EAP Programs”). In August 2021, ANVISA, the Brazilian health regulatory authority, approved Waylivra as the first treatment for familial chylomicronemia syndrome (“FCS”) in Brazil, and the Company has initiated its commercial launch in Brazil while continuing to make Waylivra available in certain other countries within Latin America and the Caribbean through EAP Programs. Waylivra has also received marketing authorization in the EU for the treatment of FCS. Additionally, the Company submitted an application to ANVISA in December 2021 for the approval of Waylivra for the treatment of familial partial lipodystrophy, and it expects a regulatory decision on approval in the second half of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin: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 by the United States Food and Drug Administration (“FDA”) in August 2020 for the treatment of SMA in adults and children two months and older and by the European Commission in March 2021 for the treatment of 5q SMA in patients two months and older with a clinical diagnosis of SMA Type 1, Type 2 or Type 3 or with one to four SMN2 copies. Evrysdi also received marketing authorization for the treatment of SMA in Brazil in October 2020 and Japan in June 2021. In January 2022, the FDA granted priority review of a supplemental new drug application for Evrysdi to expand the indication to include pre-symptomatic infants under two months old with SMA and a regulatory decision on approval is expected in May 2022. In </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">addition to the Company’s SMA program, the Company’s splicing platform also includes PTC518, which is being developed for the treatment of Huntington’s disease (“HD”). The Company announced the results from its Phase 1 study of PTC518 in healthy volunteers in September 2021 demonstrating dose-dependent lowering of huntingtin messenger ribonucleic acid and protein levels, that PTC518 efficiently crosses the blood brain barrier at significant levels and that PTC518 was well tolerated. The Company initiated a Phase 2 study of PTC518 for the treatment of HD in the first quarter of 2022, which consists of an initial 12-week placebo-controlled phase focused on safety, pharmacology and pharmacodynamic effects followed by a nine-month placebo-controlled phase focused on PTC518 biomarker effect. The Company expects results from the initial 12-week phase of the Phase 2 study by the end of 2022. </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 (“AADC deficiency”), a rare CNS disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In January 2020, the Company submitted a marketing authorization application (“MAA”) for PTC-AADC for the treatment of AADC deficiency in the EEA to the European Medicines Agency (“EMA”).  In April 2022, the Company completed the Scientific Advisory Group and Oral Explanation meetings for PTC-AADC with the EMA’s Committee for Advanced Therapies. We expect an opinion from the Committee for Medicinal Products for Human Use (“CHMP”) in May 2022. The Company is also preparing a biologics license application (“BLA”) for PTC-AADC for the treatment of AADC deficiency in the United States. In response to discussions with the FDA, the Company intends to provide additional information concerning the use of the commercial cannula for PTC-AADC in young patients. The Company expects to submit a BLA to the FDA in the third quarter of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company’s Bio-e platform consists of small molecule compounds that target oxidoreductase enzymes that regulate oxidative stress and inflammatory pathways central to the pathology of a number of CNS diseases. 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 children with mitochondrial disease associated seizures in the third quarter of 2020. The Company previously experienced delays in enrolling this trial due to the COVID-19 pandemic and anticipates results from this trial to be available in the fourth quarter of 2022  The Company also initiated a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia in the fourth quarter of 2020 and anticipates results from this trial to be available in the second quarter of 2023. In the third quarter of 2021, the Company completed a Phase 1 trial in healthy volunteers to evaluate the safety and pharmacology of PTC857. PTC857 was found to be well-tolerated with no reported serious adverse events while demonstrating predictable pharmacology. The Company initiated a Phase 2 trial of PTC857 for amyotrophic lateral sclerosis in the first quarter of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The most advanced molecule in the Company’s metabolic platform is PTC923, an oral formulation of synthetic sepiapterin, a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in metabolism and synthesis of numerous metabolic products, for orphan diseases. The Company initiated a registration-directed Phase 3 trial for PTC923 for phenylketonuria (“PKU”) in the third quarter of 2021 and expects results from this trial to be available by the end of 2022.</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 two oncology agents that are in clinical development, unesbulin and emvododstat. The Company completed its Phase 1 trials evaluating unesbulin in leiomyosarcoma (“LMS”) and diffuse intrinsic pontine glioma (“DIPG”) in the fourth quarter of 2021. The Company initiated a registration-directed Phase 2/3 trial of unesbulin for the treatment of LMS in the first quarter of 2022, and it expects to initiate a registration-directed Phase 2 trial of unesbulin for the treatment of DIPG in the third quarter of 2022. The Company completed its Phase 1 trial evaluating emvododstat in acute myelogenous leukemia (“AML”), in the fourth quarter of 2021. The Company expects to provide further updates regarding its emvododstat program at a later date.</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 Company initiated a Phase 2/3 clinical trial evaluating the efficacy and safety of emvododstat in patients hospitalized with COVID-19. In February 2021, the Company announced the completion of the first stage of the Phase 2/3 trial. The Company expects results from this trial to be available in the second quarter of 2022.</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 for rare diseases.</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. The marketing authorization in the EEA was last renewed in June 2021 and is effective, unless extended, through August 5, 2022. In February 2022, the Company submitted a marketing authorization renewal request to the EMA and, in April 2022, the CHMP issued an opinion recommending the renewal. 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 Company refers to the trial and open-label extension together as Study 041. The Company anticipates reporting results from the placebo-controlled trial by the end of the second quarter of 2022 after data analysis is completed. The Company then expects to submit a report on the placebo-controlled trial and the open-label extension data that has been collected to date to the EMA by the end of the third quarter of 2022, as required.</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 PTC’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 followed the FDA’s recommendation and collected, using newer technologies via procedures and methods that the Company designed, such dystrophin data in a new study, Study 045, and announced the results of Study 045 in February 2021. Study 045 did not meet its pre-specified primary endpoint. The Company anticipates reporting results from the placebo-controlled trial of Study 041 by the end of the second quarter of 2022 after data analysis is completed, and subject to a positive outcome in that study, the Company expects to re-submit the NDA.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">As of March 31, 2022, the Company had an accumulated deficit of approximately $2,224.7 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 9), public offerings of common stock in February 2014, October 2014, April 2018, January 2019, and September 2019, "at the market offering" of its common stock, its initial public offering of common stock in June 2013, proceeds from the Royalty Purchase Agreement dated as of July 17, 2020, by and among the Company, RPI 2019 Intermediate Finance Trust (“RPI”), and, solely for the limited purposes set forth therein, Royalty Pharma PLC (the “Royalty Purchase Agreement”) (see Note 2), private placements of its convertible preferred stock, collaborations, bank and institutional lender debt, 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 has also relied on revenue associated with milestone and royalty payments from Roche pursuant to the License and Collaboration Agreement (the “SMA License Agreement”) dated as of November 23, 2011, by and among the Company, Roche and, for the limited purposes set forth therein, the SMA Foundation, under its SMA program. 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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<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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CY2022Q1 us-gaap Finance Lease Weighted Average Remaining Lease Term1
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P10Y9M
CY2021Q4 us-gaap Finance Lease Weighted Average Remaining Lease Term1
FinanceLeaseWeightedAverageRemainingLeaseTerm1
P11Y
CY2022Q1 us-gaap Finance Lease Weighted Average Discount Rate Percent
FinanceLeaseWeightedAverageDiscountRatePercent
0.0780
CY2021Q4 us-gaap Finance Lease Weighted Average Discount Rate Percent
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CY2022Q1 us-gaap Operating Lease Payments
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CY2021Q1 us-gaap Operating Lease Payments
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CY2021Q1 us-gaap Finance Lease Principal Payments
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CY2022Q1 us-gaap Finance Lease Interest Payment On Liability
FinanceLeaseInterestPaymentOnLiability
1724000
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FinanceLeaseInterestPaymentOnLiability
776000
CY2022Q1 us-gaap Right Of Use Asset Obtained In Exchange For Operating Lease Liability
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587000
CY2021Q1 us-gaap Right Of Use Asset Obtained In Exchange For Operating Lease Liability
RightOfUseAssetObtainedInExchangeForOperatingLeaseLiability
13000
CY2022Q1 us-gaap Lessee Operating Lease Liability Payments Remainder Of Fiscal Year
LesseeOperatingLeaseLiabilityPaymentsRemainderOfFiscalYear
10154000
CY2022Q1 us-gaap Lessee Operating Lease Liability Payments Due Next Twelve Months
LesseeOperatingLeaseLiabilityPaymentsDueNextTwelveMonths
13298000
CY2022Q1 us-gaap Finance Lease Liability Payments Due Next Twelve Months
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3000000
CY2022Q1 us-gaap Lessee Operating Lease Liability Payments Due Year Two
LesseeOperatingLeaseLiabilityPaymentsDueYearTwo
12619000
CY2022Q1 us-gaap Finance Lease Liability Payments Due Year Two
FinanceLeaseLiabilityPaymentsDueYearTwo
3000000
CY2022Q1 us-gaap Lessee Operating Lease Liability Payments Due Year Three
LesseeOperatingLeaseLiabilityPaymentsDueYearThree
11231000
CY2022Q1 us-gaap Finance Lease Liability Payments Due Year Three
FinanceLeaseLiabilityPaymentsDueYearThree
3000000
CY2022Q1 ptct Lessee Operating Lease Liability To Be Paid Year Four And After
LesseeOperatingLeaseLiabilityToBePaidYearFourAndAfter
80769000
CY2022Q1 ptct Finance Lease Liability To Be Paid Year Four And After
FinanceLeaseLiabilityToBePaidYearFourAndAfter
21000000
CY2022Q1 us-gaap Lessee Operating Lease Liability Payments Due
LesseeOperatingLeaseLiabilityPaymentsDue
128071000
CY2022Q1 us-gaap Finance Lease Liability Payments Due
FinanceLeaseLiabilityPaymentsDue
30000000
CY2022Q1 us-gaap Lessee Operating Lease Liability Undiscounted Excess Amount
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48270000
CY2022Q1 us-gaap Finance Lease Liability Undiscounted Excess Amount
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9553000
CY2022Q1 us-gaap Operating Lease Liability
OperatingLeaseLiability
79801000
CY2022Q1 us-gaap Finance Lease Liability
FinanceLeaseLiability
20447000
CY2021Q4 ptct Fair Value Assets Transfers Between Level1 Level2 And Level3 Amount
FairValueAssetsTransfersBetweenLevel1Level2AndLevel3Amount
0
CY2022Q1 ptct Fair Value Assets Transfers Between Level1 Level2 And Level3 Amount
FairValueAssetsTransfersBetweenLevel1Level2AndLevel3Amount
0
CY2022Q1 us-gaap Available For Sale Debt Securities Amortized Cost Basis
AvailableForSaleDebtSecuritiesAmortizedCostBasis
248335000
CY2022Q1 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax
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30000
CY2022Q1 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
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3545000
CY2022Q1 us-gaap Available For Sale Securities Debt Securities
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CY2021Q4 us-gaap Available For Sale Debt Securities Amortized Cost Basis
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CY2021Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax
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107000
CY2021Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
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CY2021Q4 us-gaap Available For Sale Securities Debt Securities
AvailableForSaleSecuritiesDebtSecurities
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CY2022Q1 us-gaap Debt Securities Available For Sale Allowance For Credit Loss Writeoff
DebtSecuritiesAvailableForSaleAllowanceForCreditLossWriteoff
0
CY2021Q1 us-gaap Debt Securities Available For Sale Allowance For Credit Loss Period Increase Decrease
DebtSecuritiesAvailableForSaleAllowanceForCreditLossPeriodIncreaseDecrease
0
CY2022Q1 us-gaap Debt Securities Available For Sale Allowance For Credit Loss Period Increase Decrease
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0
CY2022Q1 us-gaap Marketable Securities Realized Gain Loss
MarketableSecuritiesRealizedGainLoss
-100000
CY2021Q1 us-gaap Marketable Securities Realized Gain Loss
MarketableSecuritiesRealizedGainLoss
700000
CY2022Q1 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position Less Than12 Months Accumulated Loss
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3402000
CY2022Q1 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position Less Than12 Months
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPositionLessThan12Months
227918000
CY2022Q1 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position12 Months Or Longer Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPosition12MonthsOrLongerAccumulatedLoss
143000
CY2022Q1 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position12 Months Or Longer
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPosition12MonthsOrLonger
4876000
CY2022Q1 us-gaap Debt Securities Available For Sale Unrealized Loss Position Accumulated Loss
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3545000
CY2022Q1 us-gaap Debt Securities Available For Sale Unrealized Loss Position
DebtSecuritiesAvailableForSaleUnrealizedLossPosition
232794000
CY2021Q4 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position Less Than12 Months Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPositionLessThan12MonthsAccumulatedLoss
673000
CY2021Q4 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position Less Than12 Months
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPositionLessThan12Months
256801000
CY2021Q4 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position12 Months Or Longer Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPosition12MonthsOrLongerAccumulatedLoss
36000
CY2021Q4 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position12 Months Or Longer
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPosition12MonthsOrLonger
4985000
CY2021Q4 us-gaap Debt Securities Available For Sale Unrealized Loss Position Accumulated Loss
DebtSecuritiesAvailableForSaleUnrealizedLossPositionAccumulatedLoss
709000
CY2021Q4 us-gaap Debt Securities Available For Sale Unrealized Loss Position
DebtSecuritiesAvailableForSaleUnrealizedLossPosition
261786000
CY2022Q1 us-gaap Available For Sale Securities Debt Maturities Within One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesWithinOneYearFairValue
111442000
CY2022Q1 ptct Available For Sale Securities Debt Maturities Over One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesOverOneYearFairValue
133378000
CY2021Q4 us-gaap Available For Sale Securities Debt Maturities Within One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesWithinOneYearFairValue
221611000
CY2021Q4 ptct Available For Sale Securities Debt Maturities Over One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesOverOneYearFairValue
155074000
CY2022Q1 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
29485000
CY2021Q4 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
55733000
CY2022Q1 us-gaap Accrued Income Taxes Current
AccruedIncomeTaxesCurrent
4549000
CY2021Q4 us-gaap Accrued Income Taxes Current
AccruedIncomeTaxesCurrent
1287000
CY2022Q1 ptct Accrued Consulting And Contracted Research Current
AccruedConsultingAndContractedResearchCurrent
28391000
CY2021Q4 ptct Accrued Consulting And Contracted Research Current
AccruedConsultingAndContractedResearchCurrent
26434000
CY2022Q1 us-gaap Accrued Professional Fees Current
AccruedProfessionalFeesCurrent
5013000
CY2021Q4 us-gaap Accrued Professional Fees Current
AccruedProfessionalFeesCurrent
3547000
CY2022Q1 ptct Sales Allowance And Other Related Costs
SalesAllowanceAndOtherRelatedCosts
63109000
CY2021Q4 ptct Sales Allowance And Other Related Costs
SalesAllowanceAndOtherRelatedCosts
61662000
CY2022Q1 ptct Sales Rebates Current
SalesRebatesCurrent
57680000
CY2021Q4 ptct Sales Rebates Current
SalesRebatesCurrent
68770000
CY2022Q1 us-gaap Accrued Royalties Current
AccruedRoyaltiesCurrent
30670000
CY2021Q4 us-gaap Accrued Royalties Current
AccruedRoyaltiesCurrent
35679000
CY2022Q1 us-gaap Accounts Payable Current
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18365000
CY2021Q4 us-gaap Accounts Payable Current
AccountsPayableCurrent
23033000
CY2022Q1 us-gaap Other Accrued Liabilities Current
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9259000
CY2021Q4 us-gaap Other Accrued Liabilities Current
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12639000
CY2022Q1 us-gaap Accounts Payable And Accrued Liabilities Current
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246521000
CY2021Q4 us-gaap Accounts Payable And Accrued Liabilities Current
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CY2022Q1 us-gaap Net Income Loss Available To Common Stockholders Basic
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-126726000
CY2021Q1 us-gaap Net Income Loss Available To Common Stockholders Basic
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-128642000
CY2022Q1 us-gaap Weighted Average Number Of Shares Outstanding Basic
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71215105
CY2021Q1 us-gaap Weighted Average Number Of Shares Outstanding Basic
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70188602
CY2022Q1 us-gaap Earnings Per Share Basic
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-1.78
CY2021Q1 us-gaap Earnings Per Share Basic
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CY2022Q1 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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14266694
CY2021Q1 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
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12483840
CY2022Q1 us-gaap Revenue Remaining Performance Obligation
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0
CY2022Q1 us-gaap Allocated Share Based Compensation Expense
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26589000
CY2021Q1 us-gaap Allocated Share Based Compensation Expense
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25707000
CY2022Q1 us-gaap Employee Service Share Based Compensation Nonvested Awards Total Compensation Cost Not Yet Recognized
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251800000
CY2022Q1 us-gaap Employee Service Share Based Compensation Nonvested Awards Total Compensation Cost Not Yet Recognized Period For Recognition1
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P2Y7M13D
CY2020Q3 ptct Proceeds From Sale Of Future Royalties
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650000000.0
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0.110
CY2021Q4 ptct Liability Sale Of Future Royalties
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733985000
CY2022Q1 ptct Liability Sale Of Future Royalties Royalty Revenue Payable
LiabilitySaleOfFutureRoyaltiesRoyaltyRevenuePayable
8113000
CY2022Q1 ptct Liability Sale Of Future Royalties Interest Expense
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18874000
CY2022Q1 ptct Liability Sale Of Future Royalties
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744746000
CY2022Q1 us-gaap Debt Instrument Interest Rate Effective Percentage
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0.101
CY2022Q1 us-gaap Number Of Operating Segments
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1
CY2022Q1 ptct Numberofdistributors
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2
CY2021Q1 ptct Numberofdistributors
Numberofdistributors
2
CY2021Q4 us-gaap Contract With Customer Liability
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0
CY2022Q1 us-gaap Contract With Customer Liability
ContractWithCustomerLiability
0
CY2022Q1 us-gaap Contract With Customer Liability Revenue Recognized
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0
CY2021Q1 us-gaap Contract With Customer Liability Revenue Recognized
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2100000
CY2021Q4 us-gaap Revenue Remaining Performance Obligation
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0
CY2022Q1 us-gaap Amortization Of Intangible Assets
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23500000
CY2021Q1 us-gaap Amortization Of Intangible Assets
AmortizationOfIntangibleAssets
11300000
CY2022Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Remainder Of Fiscal Year
FiniteLivedIntangibleAssetsAmortizationExpenseRemainderOfFiscalYear
70475000
CY2022Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Next Twelve Months
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93966000
CY2022Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Year Two
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16041000
CY2022Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Year Three
FiniteLivedIntangibleAssetsAmortizationExpenseYearThree
1542000
CY2022Q1 ptct Finite Lived Intangible Assets Amortization Expense Year Four And Thereafter
FiniteLivedIntangibleAssetsAmortizationExpenseYearFourAndThereafter
5140000
CY2022Q1 us-gaap Finite Lived Intangible Assets Net
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187164000
CY2022Q1 us-gaap Indefinitelived Intangible Assets Acquired
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576500000
us-gaap Indefinite Lived Intangible Assets Period Increase Decrease
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0
us-gaap Goodwill Period Increase Decrease
GoodwillPeriodIncreaseDecrease
0
CY2022Q1 us-gaap Goodwill
Goodwill
82300000

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