2021 Q4 Form 10-K Financial Statement

#000107008122000011 Filed on February 22, 2022

View on sec.gov

Income Statement

Concept 2021 Q4 2021 2020 Q4
Revenue $165.2M $538.6M $118.9M
YoY Change 38.96% 41.45% 23.21%
Cost Of Revenue $9.327M $32.33M $4.900M
YoY Change 90.35% 71.05% 40.0%
Gross Profit $155.9M $506.3M $114.0M
YoY Change 36.76% 39.93% 22.71%
Gross Profit Margin 94.36% 94.0% 95.88%
Selling, General & Admin $86.55M $285.8M $75.50M
YoY Change 14.63% 16.56% 18.9%
% of Gross Profit 55.51% 56.45% 66.23%
Research & Development $149.8M $540.7M $118.0M
YoY Change 26.99% 13.2% 44.25%
% of Gross Profit 96.11% 106.8% 103.51%
Depreciation & Amortization $18.81M $64.13M $12.40M
YoY Change 51.7% 47.47% 33.62%
% of Gross Profit 12.07% 12.67% 10.88%
Operating Expenses $240.6M $913.0M $215.2M
YoY Change 11.8% 12.37% 27.18%
Operating Profit -$84.73M -$374.4M -$96.37M
YoY Change -12.08% -13.28% 32.41%
Interest Expense -$41.40M -$86.02M -$24.30M
YoY Change 70.38% 52.65% 341.82%
% of Operating Profit
Other Income/Expense, Net -$12.48M -$57.88M $58.90M
YoY Change -121.18% -167.94% 425.89%
Pretax Income -$138.6M -$518.3M -$61.70M
YoY Change 124.65% 28.64% -7.91%
Income Tax $4.657M $5.561M $12.60M
% Of Pretax Income
Net Earnings -$143.3M -$523.9M -$74.35M
YoY Change 92.68% 19.57% -4.28%
Net Earnings / Revenue -86.71% -97.27% -62.53%
Basic Earnings Per Share -$7.43
Diluted Earnings Per Share -$2.027M -$7.43 -$1.080M
COMMON SHARES
Basic Shares Outstanding 70.70M 70.47M 68.24M
Diluted Shares Outstanding 70.47M

Balance Sheet

Concept 2021 Q4 2021 2020 Q4
SHORT-TERM ASSETS
Cash & Short-Term Investments $773.4M $773.4M $1.104B
YoY Change -29.93% -29.93% 60.75%
Cash & Equivalents $189.7M $189.7M $208.8M
Short-Term Investments $583.7M $583.7M $894.8M
Other Short-Term Assets $54.68M $54.68M $39.50M
YoY Change 38.43% 38.43% 120.67%
Inventory $15.86M $15.86M $18.70M
Prepaid Expenses
Receivables $110.5M $110.5M $69.90M
Other Receivables $0.00 $0.00 $0.00
Total Short-Term Assets $954.4M $954.4M $1.232B
YoY Change -22.52% -22.52% 58.06%
LONG-TERM ASSETS
Property, Plant & Equipment $52.59M $130.0M $33.83M
YoY Change 55.43% 9.99% 57.0%
Goodwill $82.34M $82.34M
YoY Change 0.0% 0.0%
Intangibles $724.8M $715.3M
YoY Change 1.33% 0.68%
Long-Term Investments
YoY Change
Other Assets $46.50M $46.50M $60.60M
YoY Change -23.27% -23.27% 269.51%
Total Long-Term Assets $983.7M $983.7M $976.5M
YoY Change 0.73% 0.73% 15.63%
TOTAL ASSETS
Total Short-Term Assets $954.4M $954.4M $1.232B
Total Long-Term Assets $983.7M $983.7M $976.5M
Total Assets $1.938B $1.938B $2.208B
YoY Change -12.24% -12.24% 36.0%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $23.03M $23.03M $18.67M
YoY Change 23.4% 23.17% 80.79%
Accrued Expenses $105.6M $105.6M $92.00M
YoY Change 14.81% 14.81% 31.43%
Deferred Revenue $4.151M
YoY Change -49.64%
Short-Term Debt $0.00 $0.00 $0.00
YoY Change
Long-Term Debt Due $149.5M $211.8M $22.30M
YoY Change 570.58% 849.91% 11.5%
Total Short-Term Liabilities $509.3M $509.3M $277.3M
YoY Change 83.66% 83.68% 17.59%
LONG-TERM LIABILITIES
Long-Term Debt $281.9M $976.6M $309.1M
YoY Change -8.81% -1.44% 5.2%
Other Long-Term Liabilities $313.5M $313.5M $1.392M
YoY Change 22422.92% -2.42% 887.23%
Total Long-Term Liabilities $281.9M $1.290B $310.5M
YoY Change -9.22% -1.68% 5.62%
TOTAL LIABILITIES
Total Short-Term Liabilities $509.3M $509.3M $277.3M
Total Long-Term Liabilities $281.9M $1.290B $310.5M
Total Liabilities $1.937B $1.937B $1.726B
YoY Change 12.18% 12.18% 67.69%
SHAREHOLDERS EQUITY
Retained Earnings -$2.098B -$1.629B
YoY Change 28.8% 36.82%
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 $1.438M $1.438M $482.0M
YoY Change
Total Liabilities & Shareholders Equity $1.938B $1.938B $2.208B
YoY Change -12.24% -12.24% 36.0%

Cashflow Statement

Concept 2021 Q4 2021 2020 Q4
OPERATING ACTIVITIES
Net Income -$143.3M -$523.9M -$74.35M
YoY Change 92.68% 19.57% -4.28%
Depreciation, Depletion And Amortization $18.81M $64.13M $12.40M
YoY Change 51.7% 47.47% 33.62%
Cash From Operating Activities -$68.84M -$251.3M -$64.36M
YoY Change 6.96% 29.51% 530.36%
INVESTING ACTIVITIES
Capital Expenditures -$8.726M $28.21M -$6.350M
YoY Change 37.42% 58.12% 36.27%
Acquisitions
YoY Change
Other Investing Activities $77.00M $247.4M -$201.8M
YoY Change -138.16% -145.5% -10.06%
Cash From Investing Activities $68.27M $219.2M -$208.1M
YoY Change -132.81% -139.03% -9.12%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 4.354M 20.88M 40.09M
YoY Change -89.14% -96.88% 1658.33%
NET CHANGE
Cash From Operating Activities -68.84M -251.3M -64.36M
Cash From Investing Activities 68.27M 219.2M -208.1M
Cash From Financing Activities 4.354M 20.88M 40.09M
Net Change In Cash 3.788M -19.09M -232.4M
YoY Change -101.63% -75.9% -1.92%
FREE CASH FLOW
Cash From Operating Activities -$68.84M -$251.3M -$64.36M
Capital Expenditures -$8.726M $28.21M -$6.350M
Free Cash Flow -$60.11M -$279.5M -$58.01M
YoY Change 3.63% 31.91% 945.23%

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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;text-indent:18pt;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;text-indent:18pt;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”) and Brazil 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. 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;text-indent:18pt;margin:0pt 0pt 12pt 0pt;">The Company holds the rights for the commercialization of Tegsedi<sup style="font-size:7.5pt;line-height:100%;top:0pt;vertical-align:top;">®</sup> (inotersen) and Waylivra<sup style="font-size:7.5pt;line-height:100%;top:0pt;vertical-align:top;">®</sup> (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 has initiated its commercial launch for Tegsedi for the treatment of hATTR amyloidosis in Brazil and it continues to make Tegsedi available in certain other countries within Latin America and the Caribbean through early access 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<span style="background:#ffffff;"> continuing to make Waylivra available in certain other countries within Latin America and the Caribbean through EAP programs</span>. 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;text-indent:18pt;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<sup style="font-size:7.5pt;line-height:100%;top:0pt;vertical-align:top;">®</sup> (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. In 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 </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">blood brain barrier at significant levels and that PTC518 was well tolerated. The Company expects to initiate a Phase 2 study of PTC518 in the first quarter of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;text-indent:18pt;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”) 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 (“CHMP”) in April 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 second quarter of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;text-indent:18pt;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 has experienced delays in enrolling this trial due to the COVID-19 pandemic and now 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 expects to initiate a Phase 2 trial of PTC857 for amyotrophic lateral sclerosis in the second quarter of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;text-indent:18pt;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. 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;text-indent:18pt;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 expects to initiate a registration-directed Phase 2/3 trial of unesbulin for the treatment of LMS in the second 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, a small molecule dihydrooratate dehydrogenase inhibitor that inhibits de novo pyrimidine nucleotide synthesis, 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;text-indent:18pt;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 first half of 2022.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;text-indent:18pt;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;text-indent:18pt;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. 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 expects results from the placebo-controlled trial to be available in mid-2022. 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;text-indent:18pt;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. <span style="background:#ffffff;">Study 045 did not meet its pre-specified primary endpoint. The Company expects results from the placebo-controlled trial of Study 041 to be available in mid-2022, and subject to a positive outcome in that study, the Company expects to re-submit the NDA.</span></p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;text-indent:18pt;margin:0pt 0pt 12pt 0pt;">As of December 31, 2021, the Company had an accumulated deficit of approximately $2,098.0 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 8), 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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