$PTCT
PTC Therapeutics Inc

2024 Q2 Form 10-Q Financial Statement

#000155837024005782 Filed on April 25, 2024

View on sec.gov

Income Statement

Concept 2024 Q2 2024 Q1
Revenue $186.7M $210.1M
YoY Change -12.68% -4.66%
Cost Of Revenue $15.53M $14.74M
YoY Change 21.96% 4.21%
Gross Profit $171.2M $195.4M
YoY Change -14.87% -5.27%
Gross Profit Margin 91.68% 92.98%
Selling, General & Admin $69.50M $73.27M
YoY Change -21.42% -15.7%
% of Gross Profit 40.6% 37.5%
Research & Development $132.2M $116.1M
YoY Change -28.89% -40.48%
% of Gross Profit 77.21% 59.44%
Depreciation & Amortization $7.018M $55.40M
YoY Change -86.24% 29.35%
% of Gross Profit 4.1% 28.35%
Operating Expenses $226.9M $240.8M
YoY Change 17.69% -28.75%
Operating Profit -$40.22M -$45.45M
YoY Change -587.08% -61.35%
Interest Expense -$43.49M -$39.23M
YoY Change 35.42% 43.55%
% of Operating Profit
Other Income/Expense, Net -$2.025M -$9.000K
YoY Change -148.46% -100.09%
Pretax Income -$85.73M -$84.70M
YoY Change -63.9% -37.26%
Income Tax $13.45M $6.880M
% Of Pretax Income
Net Earnings -$99.18M -$91.58M
YoY Change -50.13% -34.1%
Net Earnings / Revenue -53.12% -43.58%
Basic Earnings Per Share -$1.29 -$1.20
Diluted Earnings Per Share -$1.29 -$1.20
COMMON SHARES
Basic Shares Outstanding 76.70M 76.61M
Diluted Shares Outstanding 76.73M 76.50M

Balance Sheet

Concept 2024 Q2 2024 Q1
SHORT-TERM ASSETS
Cash & Short-Term Investments $1.093B $884.8M
YoY Change 223.51% 209.05%
Cash & Equivalents $654.8M $548.4M
Short-Term Investments $438.5M $336.5M
Other Short-Term Assets $45.64M $56.21M
YoY Change 8.08% -42.98%
Inventory $31.83M $30.06M
Prepaid Expenses
Receivables $187.2M $197.7M
Other Receivables $0.00 $0.00
Total Short-Term Assets $1.358B $1.169B
YoY Change 130.27% 90.54%
LONG-TERM ASSETS
Property, Plant & Equipment $65.99M $184.0M
YoY Change -63.1% 131.43%
Goodwill $82.30M $82.30M
YoY Change 0.0% 0.0%
Intangibles $329.9M $330.0M
YoY Change -26.96% -51.9%
Long-Term Investments
YoY Change
Other Assets $23.10M $24.55M
YoY Change -35.19% -48.73%
Total Long-Term Assets $558.4M $620.9M
YoY Change -25.38% -37.63%
TOTAL ASSETS
Total Short-Term Assets $1.358B $1.169B
Total Long-Term Assets $558.4M $620.9M
Total Assets $1.916B $1.790B
YoY Change 43.21% 11.24%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $24.28M $94.53M
YoY Change -75.15% 259.29%
Accrued Expenses $86.66M $88.13M
YoY Change -13.16% 7.45%
Deferred Revenue
YoY Change
Short-Term Debt $0.00 $0.00
YoY Change
Long-Term Debt Due $257.3M $229.1M
YoY Change 139.16% 129.67%
Total Short-Term Liabilities $609.8M $574.5M
YoY Change 28.02% 30.1%
LONG-TERM LIABILITIES
Long-Term Debt $284.8M $1.912B
YoY Change -77.23% 234.2%
Other Long-Term Liabilities $141.0K $141.2M
YoY Change -99.9% 100026.24%
Total Long-Term Liabilities $284.9M $2.053B
YoY Change -79.49% 258.79%
TOTAL LIABILITIES
Total Short-Term Liabilities $609.8M $574.5M
Total Long-Term Liabilities $284.9M $2.053B
Total Liabilities $2.897B $2.684B
YoY Change 51.07% 29.87%
SHAREHOLDERS EQUITY
Retained Earnings -$3.474B -$3.375B
YoY Change 16.01% 20.72%
Common Stock $76.00K $76.00K
YoY Change 1.33% 4.11%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity -$980.2M -$893.9M
YoY Change
Total Liabilities & Shareholders Equity $1.916B $1.790B
YoY Change 43.21% 11.24%

Cashflow Statement

Concept 2024 Q2 2024 Q1
OPERATING ACTIVITIES
Net Income -$99.18M -$91.58M
YoY Change -50.13% -34.1%
Depreciation, Depletion And Amortization $7.018M $55.40M
YoY Change -86.24% 29.35%
Cash From Operating Activities -$71.45M $70.76M
YoY Change 406.04% -339.94%
INVESTING ACTIVITIES
Capital Expenditures -$7.375M $9.588M
YoY Change -218.09% -6.64%
Acquisitions
YoY Change
Other Investing Activities -$73.30M -$105.3M
YoY Change 318.68% 463.31%
Cash From Investing Activities -$65.92M -$114.9M
YoY Change 177.55% 296.71%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 246.5M 540.0K
YoY Change 1235.22% -86.81%
NET CHANGE
Cash From Operating Activities -71.45M 70.76M
Cash From Investing Activities -65.92M -114.9M
Cash From Financing Activities 246.5M 540.0K
Net Change In Cash 109.1M -43.63M
YoY Change -662.05% -30.0%
FREE CASH FLOW
Cash From Operating Activities -$71.45M $70.76M
Capital Expenditures -$7.375M $9.588M
Free Cash Flow -$64.08M $61.17M
YoY Change 214.65% -253.85%

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 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 little to 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. PTC has a diversified therapeutic 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 relating to neurology and metabolism.</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. Translarna also has marketing authorization in Russia for the treatment of nmDMD in patients aged two years and older and in Brazil for the treatment of nmDMD in ambulatory patients two years and older and for continued treatment of patients that become non-ambulatory, as well as in various other countries. 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’s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the European Commission (“EC”) following reassessment by the European Medicines Agency (“EMA”) of the benefit-risk balance of the authorization, which the Company refers to as the annual EMA reassessment. In September 2022, the Company submitted a Type II variation to the EMA to support conversion of the conditional marketing authorization for Translarna to a standard marketing authorization, which included a report on the placebo-controlled trial of Study 041 and data from the open-label extension. In February 2023, the Company also submitted an annual marketing authorization renewal request to the EMA. In September 2023, the Committee for Medicinal Products for Human Use (“CHMP”), gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna for the treatment of nmDMD and a negative opinion on the renewal of the existing conditional marketing authorization of Translarna for the treatment of nmDMD. On January 25, 2024, the CHMP issued a negative opinion for the renewal of the conditional marketing authorization following a re-examination procedure. In accordance with EMA regulations, the EC has approximately 67 days to adopt the opinion. At this time, the EC has not yet adopted the negative opinion. If the EC adopts the negative opinion, Translarna would no longer have marketing authorization in the member states of the EEA.</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. Following the Company’s announcement of top-line results from the placebo-controlled trial of Study 041 in June 2022, the Company submitted a meeting request to the U.S. Food and Drug Administration (“FDA”) to gain clarity on the regulatory pathway for a potential re-submission of a New Drug Application (“NDA”) for Translarna. The FDA provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness to support an NDA re-submission. The Company held a Type C meeting with the FDA in the fourth quarter of 2023 to discuss the totality of Translarna data. <span style="background:#ffffff;">Based on feedback from the FDA, the Company plans to re-submit the NDA based on results from Study 041 and from the Company’s international drug registry study for nmDMD patients receiving Translarna by mid-year 2024.</span></p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">The Company has developed Upstaza (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic L-Amino Acid Decarboxylase (“AADC”) deficiency (“AADC deficiency”), a rare central nervous system (“CNS”) disorder arising from reductions in the enzyme AADC that results from mutations in the dopa decarboxylase gene. In July 2022, </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">the EC approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the EEA. In November 2022, the Medicines and Healthcare Products Regulatory Agency approved Upstaza for the treatment of AADC deficiency for patients 18 months and older within the United Kingdom. In March 2024, the Company submitted a biologics license application (“BLA”) for Upstaza for the treatment of AADC deficiency in the United States.</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<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”). In August 2021, ANVISA, the Brazilian health regulatory authority, approved Waylivra as the first treatment for familial chylomicronemia syndrome (“FCS”) in Brazil. In December 2022, ANVISA approved Waylivra for the treatment of familial partial lipodystrophy (“FPL”). Waylivra has also received marketing authorization in the EU for the treatment of FCS.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company also has a spinal muscular atrophy (“SMA”) collaboration with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc. (referred to collectively as “Roche”) and the Spinal Muscular Atrophy Foundation (“SMA Foundation”). The SMA program has one approved product, Evrysdi® (risdiplam), which was approved by the FDA in August 2020 for the treatment of SMA in adults and children two months and older and by the EC 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 has also received marketing authorization for the treatment of SMA in over 100 countries. In May <span style="letter-spacing:-0.1pt;">2022, the FDA approved a label expansion for Evrysdi to include infants under two months old with SMA. In August 2023, the EC approved an extension of the Evrysdi marketing authorization to include infants under two months old in the EU.</span></p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">One of the Company’s most advanced clinical stage molecules is sepiapterin. Sepiapterin is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in metabolism and synthesis of numerous metabolic products. In May 2023, the Company announced that the primary endpoint was achieved in its registration-directed Phase 3 trial for sepiapterin for phenylketonuria (“PKU”). The primary endpoint of the study was the achievement of statistically-significant reduction in blood Phe level. In March 2024, the Company submitted a marketing authorization application (“MAA”) to the EMA for sepiapterin for the treatment of PKU in the EEA. Additionally, the Company participated in a pre-NDA meeting with the FDA in the third quarter of 2023. At that meeting, the FDA stated that the sepiapterin clinical safety and efficacy data supported NDA submission for the treatment of pediatric and adult PKU patients. However, the FDA has requested that PTC completes a 26-week nonclinical mouse study to assess sepiapterin carcinogenicity potential prior to NDA submission. Based on the timing to complete this study, PTC expects to submit an NDA to the FDA for sepiapterin for the treatment of PKU no later than the third quarter of 2024 and PTC intends to discuss with the FDA the potential for an earlier submission if PTC is permitted to submit the 26-week mouse study report during the NDA review. PTC also expects to make regulatory submissions for sepiapterin for the treatment of PKU in Japan and Brazil in 2024.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">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 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. In June 2023, the Company announced interim data from the 12-week placebo-controlled phase. The Phase 2 study is actively ongoing outside the United States, while it has been paused within the United States as the FDA requested additional data to allow the Phase 2 study to proceed. The Company expects results from the 12-month interim data from the Phase 2 study of PTC518 for the treatment of HD in the second quarter of 2024. The Company expects to submit a safety data update to the FDA in the second quarter of 2024 to support lifting of the partial clinical hold on the program.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">The Company’s ferroptosis and inflammation 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 ferroptosis and inflammation platform are vatiquinone and utreloxastat. The Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia, called MOVE-FA, in May 2023. While the study did not meet its primary </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">endpoint, vatiquinone treatment did demonstrate significant benefit on key disease subscales, including the upright stability subscale, as well as on other disease relevant endpoints. In the first quarter of 2024, the Company met with the FDA, who expressed willingness to review an NDA for vatiquinone for the treatment of Friedreich ataxia based on the MOVE-FA trial as well as data from the ongoing open label extension study following the MOVE-FA trial, potentially allowing for the submission of an NDA in late 2024. In the first quarter of 2024, the Company also received scientific advice from the EMA on the MOVE-FA trial results, in which the EMA stated that the MOVE-FA data would likely not be sufficient for conditional authorization. The Company initiated a Phase 2 registration directed trial of utreloxastat for amyotrophic lateral sclerosis, or ALS, in the first quarter of 2022. The Company expects topline results from this trial in the fourth quarter of 2024.</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;">As of March 31, 2024, the Company had an accumulated deficit of approximately $3,375.2 million. The Company has financed its operations to date primarily through the private offerings of convertible senior notes (see Note 9), public and “at the market offerings” of common stock, proceeds from royalty purchase agreements (see Note 2), net proceeds from the Company’s’ borrowings under its credit agreement with Blackstone (see Note 9), private placements of its convertible preferred stock and common stock, collaborations, bank and institutional lender debt, other convertible 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. 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 since 2014, Emflaza for the treatment of DMD in the United States since 2017, and Upstaza for the treatment of AADC deficiency in the EEA since May 2022. 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, milestone and royalty payments from Roche, 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, 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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