2020 Q2 Form 10-Q Financial Statement

#000107008120000096 Filed on April 30, 2020

View on sec.gov

Income Statement

Concept 2020 Q2 2020 Q1 2019 Q1
Revenue $75.24M $68.26M $53.58M
YoY Change -12.02% 27.39% -4.42%
Cost Of Revenue $5.300M $4.100M $2.376M
YoY Change 65.06% 72.56% -21.97%
Gross Profit $69.90M $64.20M $51.21M
YoY Change -15.08% 25.37% -3.41%
Gross Profit Margin 92.9% 94.05% 95.57%
Selling, General & Admin $53.66M $58.21M $40.54M
YoY Change 9.03% 43.57% 22.98%
% of Gross Profit 76.77% 90.67% 79.18%
Research & Development $176.5M $90.11M $52.57M
YoY Change 194.31% 71.42% 67.61%
% of Gross Profit 252.54% 140.35% 102.65%
Depreciation & Amortization $10.21M $9.235M $7.059M
YoY Change 32.6% 30.83% 17.22%
% of Gross Profit 14.61% 14.38% 13.79%
Operating Expenses $262.5M $161.3M $122.7M
YoY Change 111.23% 31.39% 68.56%
Operating Profit -$187.3M -$92.99M -$69.14M
YoY Change 383.19% 34.5% 312.95%
Interest Expense -$5.379M -$5.642M -$2.288M
YoY Change 159.35% 146.59% -30.73%
% of Operating Profit
Other Income/Expense, Net $11.31M -$13.83M -$109.0K
YoY Change -6279.78% 12589.91% -110.86%
Pretax Income -$181.3M -$112.5M -$71.54M
YoY Change 342.14% 57.21% 275.68%
Income Tax $84.00K $222.0K $576.0K
% Of Pretax Income
Net Earnings -$181.4M -$112.7M -$72.11M
YoY Change 334.15% 56.26% 274.36%
Net Earnings / Revenue -241.13% -165.09% -134.58%
Basic Earnings Per Share
Diluted Earnings Per Share -$2.782M -$1.806M -$1.290M
COMMON SHARES
Basic Shares Outstanding 62.88M shares 62.63M shares 58.40M shares
Diluted Shares Outstanding

Balance Sheet

Concept 2020 Q2 2020 Q1 2019 Q1
SHORT-TERM ASSETS
Cash & Short-Term Investments $498.9M $595.9M $407.2M
YoY Change 37.25% 46.34% 128.38%
Cash & Equivalents $158.5M $123.3M $201.1M
Short-Term Investments $340.4M $472.6M $206.0M
Other Short-Term Assets $34.10M $24.30M $9.300M
YoY Change 95.98% 161.29% 60.34%
Inventory $18.37M $19.13M $16.22M
Prepaid Expenses
Receivables $53.60M $52.60M $46.40M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $605.0M $691.9M $479.1M
YoY Change 27.38% 44.43% 97.97%
LONG-TERM ASSETS
Property, Plant & Equipment $26.81M $26.17M $14.54M
YoY Change 74.17% 79.98% 75.14%
Goodwill $82.30M $82.30M $82.34M
YoY Change 0.0% -0.05%
Intangibles $708.8M $705.3M $695.0M
YoY Change 0.87% 1.49% 444.79%
Long-Term Investments
YoY Change
Other Assets $48.80M $22.20M $2.300M
YoY Change 741.38% 865.22% 53.33%
Total Long-Term Assets $871.6M $853.7M $804.8M
YoY Change 6.74% 6.07% 485.69%
TOTAL ASSETS
Total Short-Term Assets $605.0M $691.9M $479.1M
Total Long-Term Assets $871.6M $853.7M $804.8M
Total Assets $1.477B $1.546B $1.284B
YoY Change 14.33% 20.38% 238.4%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $15.47M $16.07M $9.786M
YoY Change 51.34% 64.26% 215.27%
Accrued Expenses $51.10M $52.20M $24.00M
YoY Change 43.14% 117.5% 160.87%
Deferred Revenue $7.702M $8.638M $5.497M
YoY Change -3.79% 57.14%
Short-Term Debt $0.00 $0.00 $0.00
YoY Change
Long-Term Debt Due $18.33M $20.00M $16.67M
YoY Change -8.34% 20.0%
Total Short-Term Liabilities $201.8M $217.7M $143.0M
YoY Change 16.03% 52.17% 96.72%
LONG-TERM LIABILITIES
Long-Term Debt $297.0M $294.6M $138.5M
YoY Change 118.91% 112.74% -5.73%
Other Long-Term Liabilities $26.68M $14.79M $8.770M
YoY Change 216.99% 68.6% 4096.17%
Total Long-Term Liabilities $323.7M $309.4M $147.2M
YoY Change 124.64% 110.11% 0.1%
TOTAL LIABILITIES
Total Short-Term Liabilities $201.8M $217.7M $143.0M
Total Long-Term Liabilities $323.7M $309.4M $147.2M
Total Liabilities $904.1M $1.017B $771.0M
YoY Change 12.61% 31.89% 236.52%
SHAREHOLDERS EQUITY
Retained Earnings -$1.485B -$1.303B -$1.011B
YoY Change 41.03% 28.92% 21.79%
Common Stock $67.00K $62.00K $58.00K
YoY Change 15.52% 6.9% 38.1%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity $572.5M $528.7M $512.9M
YoY Change
Total Liabilities & Shareholders Equity $1.477B $1.546B $1.284B
YoY Change 14.33% 20.38% 238.4%

Cashflow Statement

Concept 2020 Q2 2020 Q1 2019 Q1
OPERATING ACTIVITIES
Net Income -$181.4M -$112.7M -$72.11M
YoY Change 334.15% 56.26% 274.36%
Depreciation, Depletion And Amortization $10.21M $9.235M $7.059M
YoY Change 32.6% 30.83% 17.22%
Cash From Operating Activities -$74.81M -$81.55M -$42.41M
YoY Change 145.12% 92.3% 167.09%
INVESTING ACTIVITIES
Capital Expenditures -$2.160M $6.023M $2.865M
YoY Change 9.09% 110.23% 498.12%
Acquisitions
YoY Change
Other Investing Activities $144.2M -$95.25M -$147.6M
YoY Change -498.51% -35.48% 12517.95%
Cash From Investing Activities $142.1M -$101.3M -$150.5M
YoY Change -472.15% -32.71% 9032.16%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net $13.50M $224.4M
YoY Change -93.98%
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities -32.92M 18.49M $225.7M
YoY Change -1255.09% -91.81% 19769.89%
NET CHANGE
Cash From Operating Activities -74.81M -81.55M -$42.41M
Cash From Investing Activities 142.1M -101.3M -$150.5M
Cash From Financing Activities -32.92M 18.49M $225.7M
Net Change In Cash 34.32M -164.7M $31.65M
YoY Change -152.13% -620.57% -293.08%
FREE CASH FLOW
Cash From Operating Activities -$74.81M -$81.55M -$42.41M
Capital Expenditures -$2.160M $6.023M $2.865M
Free Cash Flow -$72.65M -$87.58M -$45.27M
YoY Change 154.56% 93.44% 176.79%

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<div style="line-height:120%;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;font-weight:bold;">The Company</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">The Company has two products, Translarna</span><span style="font-family:inherit;font-size:8pt;">™ </span><span style="font-family:inherit;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, or 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. Emflaza is approved in the United States for the treatment of DMD in patients two years and older.</span></div><div style="line-height:120%;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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, or 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, or BLA, for PTC-AADC for the treatment of AADC deficiency in the United States, which it anticipates submitting to the United States Food and Drug Administration, or FDA, in the second half of 2020. In January 2020, the Company submitted a marketing authorization application, or 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 Medicinal Products for Human Use by the end of 2020.</span></div><div style="line-height:120%;padding-bottom:6px;padding-top:6px;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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, or hATTR amyloidosis. Waylivra has received marketing authorization in the EU for the treatment of familial chylomicronemia syndrome, or FCS. The Company anticipates filing for marketing authorization for Waylivra with ANVISA, the Brazilian health regulatory authority, in the second half of 2020.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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, or SMA Foundation. The lead compound in the SMA program is risdiplam (RG7916, RO7034067). Roche submitted an NDA for risdiplam to the FDA in the fourth quarter of 2019. In April 2020, the FDA extended the Prescription Drug User Fee Act ("PDUFA") date for a decision from May 24, 2020 to August 24, 2020 as a result of additional data that Roche submitted, including comprehensive data from the Sunfish part 2 study. Risdiplam is expected to be indicated in the United States for SMA type 1, 2 and 3 patients, if approved. Roche anticipates submitting an MAA for risdiplam in the EEA in mid-year 2020.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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 accounted for as an asset acquisition. In 2020, the Company plans to initiate three trials in this platform with two unique compounds that regulate inflammation and oxidative stress.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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 </span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">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.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">The marketing authorization in the EEA was last renewed in July 2019 and is effective, unless extended, through August 5, 2020. 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. In February 2020, the Company submitted a marketing authorization renewal request to the EMA.</span></div><div style="line-height:120%;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span 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 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 Company expects that data for Study 045 will be available in the third quarter of 2020 followed by a potential re-submission of an NDA 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.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;"><br/></span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">On April 20, 2017, the Company completed its acquisition of all rights to Emflaza, or the Transaction. 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.</span></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">Upon the closing of the Transaction, the Company paid to Marathon total upfront consideration comprised of </span><span style="font-family:inherit;font-size:10pt;"><span>$75.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> in cash, funded through cash on hand, and </span><span style="font-family:inherit;font-size:10pt;"><span>6,683,598</span></span><span style="font-family:inherit;font-size:10pt;"> shares of the Company’s common stock. The number of shares of common stock issued at closing was determined by dividing </span><span style="font-family:inherit;font-size:10pt;"><span>$65.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> by the volume-weighted average price per share of the Company’s common stock on the Nasdaq Stock Market for the </span><span style="font-family:inherit;font-size:10pt;"><span>15</span></span><span style="font-family:inherit;font-size:10pt;"> trading-day period ending on the third trading day immediately preceding the closing. Marathon is entitled to receive contingent payments from the Company based on annual net sales of Emflaza, up to a specified aggregate maximum amount over the expected commercial life of the asset, and a single </span><span style="font-family:inherit;font-size:10pt;"><span>$50.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> sales-based milestone, in each case subject to the terms and conditions of the Asset Purchase Agreement.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">On August 23, 2018, the Company completed its acquisition of Agilis Biotherapeutics, Inc., or Agilis, pursuant to an Agreement and Plan of Merger, dated as of July 19, 2018 (the “Merger Agreement”), by and among the Company, Agility 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").</span></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">Upon the closing of the Merger, the Company paid to Agilis equityholders total upfront consideration comprised of </span><span style="font-family:inherit;font-size:10pt;"><span>$49.2 million</span></span><span style="font-family:inherit;font-size:10pt;"> in cash and </span><span style="font-family:inherit;font-size:10pt;"><span>3,500,907</span></span><span style="font-family:inherit;font-size:10pt;"> shares of the Company’s common stock (the “Closing Stock Consideration”). The Closing Stock Consideration was determined by dividing </span><span style="font-family:inherit;font-size:10pt;"><span>$150.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> by the volume-weighted average price per share of the Company’s common stock on the Nasdaq Global Select Market for the </span><span style="font-family:inherit;font-size:10pt;"><span>10</span></span><span style="font-family:inherit;font-size:10pt;"> consecutive trading-day period ending on the second trading-day immediately preceding the closing of the Merger. Agilis equityholders may become 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. Under the Merger Agreement, the Company is required to pay </span><span style="font-family:inherit;font-size:10pt;"><span>$40.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> of the development milestone payments upon the passing of the second anniversary of the closing of the Merger, regardless of whether the applicable milestones have been achieved.</span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">Upon the closing of the Asset Acquisition, the Company paid to BioElectron total upfront consideration of </span><span style="font-family:inherit;font-size:10pt;"><span>$10.0 million</span></span><span style="font-family:inherit;font-size:10pt;">, funded with cash on hand, less (i) transaction expenses incurred by BioElectron, (ii) the amount of outstanding indebtedness of BioElectron </span></div><div style="line-height:120%;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">including a </span><span style="font-family:inherit;font-size:10pt;"><span>$4.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> loan advance to BioElectron plus accrued and unpaid interest thereon and (iii) </span><span style="font-family:inherit;font-size:10pt;"><span>$1.5 million</span></span><span style="font-family:inherit;font-size:10pt;"> held in an escrow account to secure potential indemnification obligations owed to the Company. Subject to the terms and conditions of the Asset Acquisition Agreement, BioElectron may become entitled to receive contingent milestone payments of up to </span><span style="font-family:inherit;font-size:10pt;"><span>$200.0 million</span></span><span style="font-family:inherit;font-size:10pt;"> (in cash or in shares of the Company’s common stock, as determined by the Company) from the Company based on the achievement of certain regulatory and net sales milestones. Subject to the terms and conditions of the Asset Acquisition Agreement, BioElectron may also become entitled to receive contingent payments based on a percentage of net sales of certain products.</span></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;">As of </span><span style="font-family:inherit;font-size:10pt;">March 31, 2020</span><span style="font-family:inherit;font-size:10pt;">, the Company had an accumulated deficit of approximately </span><span style="font-family:inherit;font-size:10pt;"><span>$1,303.4 million</span></span><span style="font-family:inherit;font-size:10pt;">. The Company has financed its operations to date primarily through the private offerings in September 2019 of </span><span style="font-family:inherit;font-size:10pt;"><span>1.50%</span></span><span style="font-family:inherit;font-size:10pt;"> convertible senior notes due 2026 and in August 2015 of </span><span style="font-family:inherit;font-size:10pt;"><span>3.00%</span></span><span style="font-family:inherit;font-size:10pt;"> convertible senior notes due 2022 (see Note 10), 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, private placements of its convertible preferred stock, collaborations, bank 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 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.</span></div>
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<div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;font-size:10pt;"><span style="font-family:inherit;font-size:10pt;font-weight:bold;">Use of estimates</span></div><div style="line-height:120%;padding-bottom:10px;padding-top:10px;text-align:justify;font-size:10pt;"><span 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’s research and development expenses, 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.</span></div>
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CY2019Q4 us-gaap Inventory Net
InventoryNet
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CY2020Q1 us-gaap Inventory Write Down
InventoryWriteDown
200000 USD
CY2019Q1 us-gaap Inventory Write Down
InventoryWriteDown
0 USD
CY2020Q1 us-gaap Allowance For Doubtful Accounts Receivable
AllowanceForDoubtfulAccountsReceivable
200000 USD
CY2019Q4 us-gaap Allowance For Doubtful Accounts Receivable
AllowanceForDoubtfulAccountsReceivable
300000 USD
CY2020Q1 us-gaap Allowance For Doubtful Accounts Receivable Period Increase Decrease
AllowanceForDoubtfulAccountsReceivablePeriodIncreaseDecrease
0 USD
CY2019Q4 us-gaap Deferred Tax Liabilities
DeferredTaxLiabilities
122000000.0 USD
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NumberOfOperatingLeases
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CY2020Q1 us-gaap Lessee Operating Lease Renewal Term
LesseeOperatingLeaseRenewalTerm
P10Y
CY2020Q1 us-gaap Operating Lease Expense
OperatingLeaseExpense
2400000 USD
CY2019Q1 us-gaap Operating Lease Expense
OperatingLeaseExpense
1000000.0 USD
CY2020Q1 us-gaap Operating Lease Cost
OperatingLeaseCost
2118000 USD
CY2019Q1 us-gaap Operating Lease Cost
OperatingLeaseCost
812000 USD
CY2020Q1 us-gaap Variable Lease Cost
VariableLeaseCost
228000 USD
CY2019Q1 us-gaap Variable Lease Cost
VariableLeaseCost
143000 USD
CY2020Q1 us-gaap Short Term Lease Cost
ShortTermLeaseCost
77000 USD
CY2019Q1 us-gaap Short Term Lease Cost
ShortTermLeaseCost
53000 USD
CY2020Q1 us-gaap Lease Cost
LeaseCost
2423000 USD
CY2019Q1 us-gaap Lease Cost
LeaseCost
1008000 USD
CY2020Q1 us-gaap Operating Lease Right Of Use Asset
OperatingLeaseRightOfUseAsset
17671000 USD
CY2019Q4 us-gaap Operating Lease Right Of Use Asset
OperatingLeaseRightOfUseAsset
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3589000 USD
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OperatingLeaseLiabilityCurrent
5153000 USD
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OperatingLeaseLiabilityNoncurrent
14645000 USD
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OperatingLeaseLiabilityNoncurrent
9018000 USD
CY2020Q1 us-gaap Operating Lease Liability
OperatingLeaseLiability
18234000 USD
CY2019Q4 us-gaap Operating Lease Liability
OperatingLeaseLiability
14171000 USD
CY2020Q1 us-gaap Operating Lease Weighted Average Remaining Lease Term1
OperatingLeaseWeightedAverageRemainingLeaseTerm1
P5Y4M6D
CY2019Q1 us-gaap Operating Lease Weighted Average Remaining Lease Term1
OperatingLeaseWeightedAverageRemainingLeaseTerm1
P4Y8M15D
CY2020Q1 us-gaap Operating Lease Weighted Average Discount Rate Percent
OperatingLeaseWeightedAverageDiscountRatePercent
0.0734
CY2019Q1 us-gaap Operating Lease Weighted Average Discount Rate Percent
OperatingLeaseWeightedAverageDiscountRatePercent
0.0702
CY2020Q1 us-gaap Operating Lease Payments
OperatingLeasePayments
1971000 USD
CY2019Q1 us-gaap Operating Lease Payments
OperatingLeasePayments
732000 USD
CY2020Q1 us-gaap Right Of Use Asset Obtained In Exchange For Operating Lease Liability
RightOfUseAssetObtainedInExchangeForOperatingLeaseLiability
22642000 USD
CY2019Q1 us-gaap Right Of Use Asset Obtained In Exchange For Operating Lease Liability
RightOfUseAssetObtainedInExchangeForOperatingLeaseLiability
11314000 USD
CY2020Q1 us-gaap Lessee Operating Lease Liability Payments Remainder Of Fiscal Year
LesseeOperatingLeaseLiabilityPaymentsRemainderOfFiscalYear
3543000 USD
CY2020Q1 us-gaap Lessee Operating Lease Liability Payments Due Year Two
LesseeOperatingLeaseLiabilityPaymentsDueYearTwo
4372000 USD
CY2020Q1 us-gaap Lessee Operating Lease Liability Payments Due Year Three
LesseeOperatingLeaseLiabilityPaymentsDueYearThree
3846000 USD
CY2020Q1 us-gaap Lessee Operating Lease Liability Payments Due Year Four
LesseeOperatingLeaseLiabilityPaymentsDueYearFour
3435000 USD
CY2020Q1 ptct Lessee Operating Lease Liability Payments Due Year Fiveand Thereafter
LesseeOperatingLeaseLiabilityPaymentsDueYearFiveandThereafter
6900000 USD
CY2020Q1 us-gaap Lessee Operating Lease Liability Payments Due
LesseeOperatingLeaseLiabilityPaymentsDue
22096000 USD
CY2020Q1 us-gaap Lessee Operating Lease Liability Undiscounted Excess Amount
LesseeOperatingLeaseLiabilityUndiscountedExcessAmount
3862000 USD
CY2020Q1 us-gaap Operating Lease Liability
OperatingLeaseLiability
18234000 USD
CY2020Q1 ptct Number Of Operating Lease Lease Notyet Commenced
NumberOfOperatingLeaseLeaseNotyetCommenced
2 operating_lease
CY2019Q4 us-gaap Area Of Real Estate Property
AreaOfRealEstateProperty
185000 sqft
CY2020Q1 us-gaap Area Of Real Estate Property
AreaOfRealEstateProperty
220500 sqft
CY2020Q1 us-gaap Lessee Operating Lease Term Of Contract
LesseeOperatingLeaseTermOfContract
P15Y
CY2020Q1 ptct Percent Of Market Rate
PercentOfMarketRate
0.95
CY2020Q1 ptct Investment In Mri
InvestmentInMRI
4600000 USD
CY2020Q1 ptct Payments To Acquire Convertible Debt Investment
PaymentsToAcquireConvertibleDebtInvestment
10000000.0 USD
CY2020Q1 us-gaap Debt Instrument Convertible Conversion Price1
DebtInstrumentConvertibleConversionPrice1
6.00
CY2020Q1 ptct Debt Instrument Convertible Period
DebtInstrumentConvertiblePeriod
P5Y
CY2020Q1 us-gaap Available For Sale Debt Securities Gross Unrealized Loss
AvailableForSaleDebtSecuritiesGrossUnrealizedLoss
2800000 USD
CY2020Q1 us-gaap Available For Sale Debt Securities Amortized Cost Basis
AvailableForSaleDebtSecuritiesAmortizedCostBasis
471963000 USD
CY2020Q1 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
1374000 USD
CY2020Q1 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
765000 USD
CY2020Q1 us-gaap Available For Sale Securities Debt Securities
AvailableForSaleSecuritiesDebtSecurities
472572000 USD
CY2019Q4 us-gaap Available For Sale Debt Securities Amortized Cost Basis
AvailableForSaleDebtSecuritiesAmortizedCostBasis
397776000 USD
CY2019Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Gain Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedGainBeforeTax
787000 USD
CY2019Q4 us-gaap Available For Sale Debt Securities Accumulated Gross Unrealized Loss Before Tax
AvailableForSaleDebtSecuritiesAccumulatedGrossUnrealizedLossBeforeTax
28000 USD
CY2019Q4 us-gaap Available For Sale Securities Debt Securities
AvailableForSaleSecuritiesDebtSecurities
398535000 USD
CY2020Q1 us-gaap Debt Securities Available For Sale Allowance For Credit Loss Writeoff
DebtSecuritiesAvailableForSaleAllowanceForCreditLossWriteoff
0 USD
CY2020Q1 us-gaap Debt Securities Available For Sale Allowance For Credit Loss Period Increase Decrease
DebtSecuritiesAvailableForSaleAllowanceForCreditLossPeriodIncreaseDecrease
0 USD
CY2020Q1 us-gaap Marketable Securities Realized Gain Loss
MarketableSecuritiesRealizedGainLoss
0 USD
CY2020Q1 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position Less Than12 Months Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPositionLessThan12MonthsAccumulatedLoss
765000 USD
CY2020Q1 us-gaap Held To Maturity Securities Continuous Unrealized Loss Position Less Than Twelve Months Fair Value
HeldToMaturitySecuritiesContinuousUnrealizedLossPositionLessThanTwelveMonthsFairValue
207619000 USD
CY2020Q1 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position12 Months Or Longer Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPosition12MonthsOrLongerAccumulatedLoss
0 USD
CY2020Q1 us-gaap Held To Maturity Securities Continuous Unrealized Loss Position Twelve Months Or Longer Fair Value
HeldToMaturitySecuritiesContinuousUnrealizedLossPositionTwelveMonthsOrLongerFairValue
0 USD
CY2020Q1 us-gaap Debt Securities Available For Sale Unrealized Loss Position Accumulated Loss
DebtSecuritiesAvailableForSaleUnrealizedLossPositionAccumulatedLoss
765000 USD
CY2020Q1 us-gaap Held To Maturity Securities Continuous Unrealized Loss Position Fair Value
HeldToMaturitySecuritiesContinuousUnrealizedLossPositionFairValue
207619000 USD
CY2019Q4 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position Less Than12 Months Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPositionLessThan12MonthsAccumulatedLoss
28000 USD
CY2019Q4 us-gaap Held To Maturity Securities Continuous Unrealized Loss Position Less Than Twelve Months Fair Value
HeldToMaturitySecuritiesContinuousUnrealizedLossPositionLessThanTwelveMonthsFairValue
95990000 USD
CY2019Q4 us-gaap Debt Securities Available For Sale Continuous Unrealized Loss Position12 Months Or Longer Accumulated Loss
DebtSecuritiesAvailableForSaleContinuousUnrealizedLossPosition12MonthsOrLongerAccumulatedLoss
0 USD
CY2019Q4 us-gaap Held To Maturity Securities Continuous Unrealized Loss Position Twelve Months Or Longer Fair Value
HeldToMaturitySecuritiesContinuousUnrealizedLossPositionTwelveMonthsOrLongerFairValue
0 USD
CY2019Q4 us-gaap Debt Securities Available For Sale Unrealized Loss Position Accumulated Loss
DebtSecuritiesAvailableForSaleUnrealizedLossPositionAccumulatedLoss
28000 USD
CY2019Q4 us-gaap Held To Maturity Securities Continuous Unrealized Loss Position Fair Value
HeldToMaturitySecuritiesContinuousUnrealizedLossPositionFairValue
95990000 USD
CY2020Q1 us-gaap Available For Sale Securities Debt Maturities Within One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesWithinOneYearFairValue
380312000 USD
CY2020Q1 ptct Available For Sale Securities Debt Maturities Over One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesOverOneYearFairValue
92260000 USD
CY2019Q4 us-gaap Available For Sale Securities Debt Maturities Within One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesWithinOneYearFairValue
342418000 USD
CY2019Q4 ptct Available For Sale Securities Debt Maturities Over One Year Fair Value
AvailableForSaleSecuritiesDebtMaturitiesOverOneYearFairValue
56117000 USD
CY2020Q1 ptct Business Combination Consideration Liability Deferred Consideration Payable Current
BusinessCombinationConsiderationLiabilityDeferredConsiderationPayableCurrent
40000000.0 USD
CY2020Q1 us-gaap Fair Value Measurement With Unobservable Inputs Reconciliation Recurring Basis Liability Issues
FairValueMeasurementWithUnobservableInputsReconciliationRecurringBasisLiabilityIssues
0 USD
CY2019Q1 us-gaap Fair Value Measurement With Unobservable Inputs Reconciliation Recurring Basis Liability Issues
FairValueMeasurementWithUnobservableInputsReconciliationRecurringBasisLiabilityIssues
0 USD
CY2020Q1 us-gaap Other Comprehensive Income Loss Before Reclassifications Net Of Tax
OtherComprehensiveIncomeLossBeforeReclassificationsNetOfTax
8599000 USD
CY2020Q1 us-gaap Reclassification From Aoci Current Period Net Of Tax Attributable To Parent
ReclassificationFromAociCurrentPeriodNetOfTaxAttributableToParent
0 USD
CY2020Q1 us-gaap Other Comprehensive Income Loss Net Of Tax
OtherComprehensiveIncomeLossNetOfTax
8599000 USD
CY2020Q1 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
21770000 USD
CY2019Q4 us-gaap Employee Related Liabilities Current
EmployeeRelatedLiabilitiesCurrent
38889000 USD
CY2020Q1 ptct Accrued Consulting And Contracted Research Current
AccruedConsultingAndContractedResearchCurrent
15227000 USD
CY2019Q4 ptct Accrued Consulting And Contracted Research Current
AccruedConsultingAndContractedResearchCurrent
12969000 USD
CY2020Q1 us-gaap Accrued Professional Fees Current
AccruedProfessionalFeesCurrent
5026000 USD
CY2019Q4 us-gaap Accrued Professional Fees Current
AccruedProfessionalFeesCurrent
3562000 USD
CY2020Q1 ptct Sales Allowance And Other Related Costs
SalesAllowanceAndOtherRelatedCosts
43229000 USD
CY2019Q4 ptct Sales Allowance And Other Related Costs
SalesAllowanceAndOtherRelatedCosts
41155000 USD
CY2020Q1 ptct Sales Rebates And Royalties Current
SalesRebatesAndRoyaltiesCurrent
37511000 USD
CY2019Q4 ptct Sales Rebates And Royalties Current
SalesRebatesAndRoyaltiesCurrent
42997000 USD
CY2020Q1 us-gaap Accounts Payable Current
AccountsPayableCurrent
16074000 USD
CY2019Q4 us-gaap Accounts Payable Current
AccountsPayableCurrent
10324000 USD
CY2020Q1 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
6549000 USD
CY2019Q4 us-gaap Other Accrued Liabilities Current
OtherAccruedLiabilitiesCurrent
9380000 USD
CY2020Q1 us-gaap Accounts Payable And Accrued Liabilities Current
AccountsPayableAndAccruedLiabilitiesCurrent
145386000 USD
CY2019Q4 us-gaap Accounts Payable And Accrued Liabilities Current
AccountsPayableAndAccruedLiabilitiesCurrent
159276000 USD
CY2020Q1 us-gaap Sale Of Stock Number Of Shares Issued In Transaction
SaleOfStockNumberOfSharesIssuedInTransaction
262205 shares
CY2020Q1 us-gaap Sale Of Stock Price Per Share
SaleOfStockPricePerShare
52.81
CY2020Q1 us-gaap Sale Of Stock Consideration Received On Transaction
SaleOfStockConsiderationReceivedOnTransaction
13500000 USD
CY2020Q1 us-gaap Net Income Loss Available To Common Stockholders Basic
NetIncomeLossAvailableToCommonStockholdersBasic
-112687000 USD
CY2019Q1 us-gaap Net Income Loss Available To Common Stockholders Basic
NetIncomeLossAvailableToCommonStockholdersBasic
-72113000 USD
CY2020Q1 us-gaap Weighted Average Number Of Share Outstanding Basic And Diluted
WeightedAverageNumberOfShareOutstandingBasicAndDiluted
62389158 shares
CY2019Q1 us-gaap Weighted Average Number Of Share Outstanding Basic And Diluted
WeightedAverageNumberOfShareOutstandingBasicAndDiluted
55855111 shares
CY2020Q1 us-gaap Earnings Per Share Basic And Diluted
EarningsPerShareBasicAndDiluted
-1.81
CY2019Q1 us-gaap Earnings Per Share Basic And Diluted
EarningsPerShareBasicAndDiluted
-1.29
CY2020Q1 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
13597219 shares
CY2019Q1 us-gaap Antidilutive Securities Excluded From Computation Of Earnings Per Share Amount
AntidilutiveSecuritiesExcludedFromComputationOfEarningsPerShareAmount
11506722 shares
CY2020Q1 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
15220000 USD
CY2019Q1 us-gaap Allocated Share Based Compensation Expense
AllocatedShareBasedCompensationExpense
9263000 USD
CY2020Q1 us-gaap Employee Service Share Based Compensation Nonvested Awards Total Compensation Cost Not Yet Recognized
EmployeeServiceShareBasedCompensationNonvestedAwardsTotalCompensationCostNotYetRecognized
200700000 USD
CY2020Q1 us-gaap Employee Service Share Based Compensation Nonvested Awards Total Compensation Cost Not Yet Recognized Period For Recognition1
EmployeeServiceShareBasedCompensationNonvestedAwardsTotalCompensationCostNotYetRecognizedPeriodForRecognition1
P3Y2M23D
CY2020Q1 ptct Debt Instrument Additional Amount Available For Repurchase
DebtInstrumentAdditionalAmountAvailableForRepurchase
37500000 USD
CY2020Q1 us-gaap Number Of Operating Segments
NumberOfOperatingSegments
1 segment
CY2020Q1 ptct Numberofdistributors
Numberofdistributors
2 Number_Distributor
CY2020Q1 ptct Percentageofnetproductsales
Percentageofnetproductsales
0.10
CY2019Q1 ptct Numberofdistributors
Numberofdistributors
3 Number_Distributor
CY2020Q1 ptct Percentageofnetproductsales
Percentageofnetproductsales
0.10
CY2020Q1 us-gaap Contract With Customer Liability
ContractWithCustomerLiability
10400000 USD
CY2019Q4 us-gaap Contract With Customer Liability
ContractWithCustomerLiability
11700000 USD
CY2020Q1 us-gaap Contract With Customer Liability Revenue Recognized
ContractWithCustomerLiabilityRevenueRecognized
2000000.0 USD
CY2020Q1 us-gaap Contract With Customer Liability
ContractWithCustomerLiability
10400000 USD
CY2019Q4 us-gaap Contract With Customer Liability
ContractWithCustomerLiability
11700000 USD
CY2019 us-gaap Finite Lived Intangible Asset Useful Life
FiniteLivedIntangibleAssetUsefulLife
P7Y
CY2020Q1 us-gaap Amortization Of Intangible Assets
AmortizationOfIntangibleAssets
7900000 USD
CY2019Q1 us-gaap Amortization Of Intangible Assets
AmortizationOfIntangibleAssets
6100000 USD
CY2020Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Remainder Of Fiscal Year
FiniteLivedIntangibleAssetsAmortizationExpenseRemainderOfFiscalYear
23851000 USD
CY2020Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Year Two
FiniteLivedIntangibleAssetsAmortizationExpenseYearTwo
31801000 USD
CY2020Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Year Three
FiniteLivedIntangibleAssetsAmortizationExpenseYearThree
31801000 USD
CY2020Q1 us-gaap Finite Lived Intangible Assets Amortization Expense Year Four
FiniteLivedIntangibleAssetsAmortizationExpenseYearFour
31801000 USD
CY2020Q1 ptct Finite Lived Intangible Assets Amortization Expense Year Five And Thereafter
FiniteLivedIntangibleAssetsAmortizationExpenseYearFiveAndThereafter
9540000 USD
CY2020Q1 us-gaap Finite Lived Intangible Assets Net
FiniteLivedIntangibleAssetsNet
128794000 USD
CY2020Q1 us-gaap Finite Lived Intangible Assets Remaining Amortization Period1
FiniteLivedIntangibleAssetsRemainingAmortizationPeriod1
P4Y2M12D
CY2020Q1 us-gaap Indefinitelived Intangible Assets Acquired
IndefinitelivedIntangibleAssetsAcquired
576500000 USD
CY2020Q1 us-gaap Goodwill
Goodwill
82300000 USD

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