$PTCT
PTC THERAPEUTICS, INC.

2023 Q4 Form 10-Q Financial Statement

#000107008123000065 Filed on October 26, 2023

View on sec.gov

Income Statement

Concept 2023 Q4 2023 Q3
Revenue $307.1M $196.6M
YoY Change 83.41% -9.46%
Cost Of Revenue $29.12M $9.500M
YoY Change 167.31% -32.2%
Gross Profit $277.9M $187.1M
YoY Change 77.57% -7.89%
Gross Profit Margin 90.52% 95.18%
Selling, General & Admin $76.29M $80.89M
YoY Change -17.72% 0.96%
% of Gross Profit 27.45% 43.23%
Research & Development $121.4M $164.2M
YoY Change -35.69% -0.76%
% of Gross Profit 43.66% 87.77%
Depreciation & Amortization $80.71M $62.06M
YoY Change 107.65% 80.0%
% of Gross Profit 29.04% 33.17%
Operating Expenses $272.1M $314.7M
YoY Change -15.88% 10.31%
Operating Profit $5.820M -$118.2M
YoY Change -103.49% 73.29%
Interest Expense -$40.28M -$28.16M
YoY Change 1.45% 34.87%
% of Operating Profit -692.01%
Other Income/Expense, Net $14.96M -$20.27M
YoY Change -70.28% -46.87%
Pretax Income -$157.1M -$166.6M
YoY Change -17.21% 30.96%
Income Tax -$1.259M -$33.62M
% Of Pretax Income
Net Earnings -$155.8M -$133.0M
YoY Change -8.83% 21.64%
Net Earnings / Revenue -50.74% -67.64%
Basic Earnings Per Share -$1.76
Diluted Earnings Per Share -$2.06 -$1.76
COMMON SHARES
Basic Shares Outstanding 75.46M 75.34M
Diluted Shares Outstanding 75.38M

Balance Sheet

Concept 2023 Q4 2023 Q3
SHORT-TERM ASSETS
Cash & Short-Term Investments $876.7M $294.8M
YoY Change 113.47% 2.21%
Cash & Equivalents $594.0M $165.2M
Short-Term Investments $282.7M $129.7M
Other Short-Term Assets $150.5M $44.10M
YoY Change 42.43% -50.76%
Inventory $30.58M $35.75M
Prepaid Expenses
Receivables $160.8M $165.7M
Other Receivables $0.00 $0.00
Total Short-Term Assets $1.219B $540.4M
YoY Change 75.65% 4.47%
LONG-TERM ASSETS
Property, Plant & Equipment $87.09M $85.03M
YoY Change 19.97% 27.12%
Goodwill $82.34M $82.30M
YoY Change 0.0% 0.0%
Intangibles $379.5M $423.3M
YoY Change -46.24% -43.55%
Long-Term Investments
YoY Change
Other Assets $36.25M $34.70M
YoY Change -25.39% -37.04%
Total Long-Term Assets $677.1M $719.5M
YoY Change -33.08% -32.07%
TOTAL ASSETS
Total Short-Term Assets $1.219B $540.4M
Total Long-Term Assets $677.1M $719.5M
Total Assets $1.896B $1.260B
YoY Change 11.14% -20.08%
SHORT-TERM LIABILITIES
YoY Change
Accounts Payable $6.045M $19.15M
YoY Change -77.83% -8.2%
Accrued Expenses $116.3M $116.2M
YoY Change -7.73% 17.23%
Deferred Revenue $801.0K $1.224M
YoY Change -40.71%
Short-Term Debt $0.00 $0.00
YoY Change
Long-Term Debt Due $197.3M $108.5M
YoY Change 162.56% 34.7%
Total Short-Term Liabilities $603.1M $492.1M
YoY Change 48.46% 17.15%
LONG-TERM LIABILITIES
Long-Term Debt $284.2M $573.2M
YoY Change -50.29% 102.71%
Other Long-Term Liabilities $141.0K $141.0K
YoY Change -94.66% -96.99%
Total Long-Term Liabilities $284.4M $573.3M
YoY Change -50.49% 99.46%
TOTAL LIABILITIES
Total Short-Term Liabilities $603.1M $492.1M
Total Long-Term Liabilities $284.4M $573.3M
Total Liabilities $2.714B $1.931B
YoY Change 32.23% 7.06%
SHAREHOLDERS EQUITY
Retained Earnings -$3.284B -$3.128B
YoY Change 23.58% 25.81%
Common Stock $75.00K $75.00K
YoY Change 4.17% 5.63%
Preferred Stock
YoY Change
Treasury Stock (at cost)
YoY Change
Treasury Stock Shares
Shareholders Equity -$818.6M -$670.8M
YoY Change
Total Liabilities & Shareholders Equity $1.896B $1.260B
YoY Change 11.14% -20.08%

Cashflow Statement

Concept 2023 Q4 2023 Q3
OPERATING ACTIVITIES
Net Income -$155.8M -$133.0M
YoY Change -8.83% 21.64%
Depreciation, Depletion And Amortization $80.71M $62.06M
YoY Change 107.65% 80.0%
Cash From Operating Activities -$100.3M -$14.52M
YoY Change -39.57% -61.83%
INVESTING ACTIVITIES
Capital Expenditures $5.566M $6.350M
YoY Change -35.44% -217.99%
Acquisitions
YoY Change
Other Investing Activities -$88.85M -$23.24M
YoY Change -316.39% -116.39%
Cash From Investing Activities -$94.42M -$29.60M
YoY Change -391.06% -121.69%
FINANCING ACTIVITIES
Cash Dividend Paid
YoY Change
Common Stock Issuance & Retirement, Net
YoY Change
Debt Paid & Issued, Net
YoY Change
Cash From Financing Activities 620.5M 3.370M
YoY Change 104.84% -102.41%
NET CHANGE
Cash From Operating Activities -100.3M -14.52M
Cash From Investing Activities -94.42M -29.60M
Cash From Financing Activities 620.5M 3.370M
Net Change In Cash 425.8M -40.75M
YoY Change 151.35% -2.03%
FREE CASH FLOW
Cash From Operating Activities -$100.3M -$14.52M
Capital Expenditures $5.566M $6.350M
Free Cash Flow -$105.9M -$20.87M
YoY Change -39.37% -36.11%

Facts In Submission

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dei Entity Registrant Name
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PTC Therapeutics, Inc.
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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 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. </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 relating to neurology, metabolism and oncology.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">The Company 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. 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. In July 2020, the European Commission (“EC”) 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;margin:0pt 0pt 12pt 0pt;">The Company’s marketing authorization for Translarna in the EEA is subject to annual review and renewal by the 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. <span style="background:#ffffff;">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. The Company has requested re-examination of the CHMP opinion regarding the renewal of the existing conditional marketing authorization. In re-examination, the Company intends to address concerns raised during the previous review process on the benefit demonstrated in Translarna clinical trials and regarding the robustness of the Strategic Targeting of Registries and International Database of Excellence, or STRIDE, real world registry. In accordance with EMA guidelines, the Company expects the CHMP opinion following the re-examination process to occur in late January 2024, with EC ratification of the opinion within the following 67 days.</span></p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin: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 then had an informal meeting with the FDA, during which the Company discussed the potential path to an NDA re-submission for Translarna. Based on the meeting discussion, the Company has scheduled an additional Type C meeting with the FDA to review the totality of data </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">collected to date, including dystrophin and other mechanistic data as well as additional analyses that could support the benefit of Translarna.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 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, 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. The Company is also preparing a biologics license application (“BLA”) for Upstaza for the treatment of AADC deficiency in the United States and anticipates submitting the BLA shortly after its pre-BLA meeting that is scheduled for December 2023, pending the outcome of such meeting.</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 began to make commercial sales of Waylivra in Brazil in the third quarter of 2022 while continuing to make Waylivra available in certain other countries within Latin America and the Caribbean through EAP Programs. 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 also received marketing authorization for the treatment of SMA in Brazil in October 2020 and Japan in June 2021. In May 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. 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 Company expects the next data update from the Phase 2 study of PTC518 for the treatment of HD in the first half of 2024. In the Phase 2 study, enrollment outside of the United States remains active and ongoing while enrollment within the United States is paused as the FDA requested additional data to allow the Phase 2 study to proceed. The Company had a Type A meeting with the FDA to review the clinical safety data needed to enable resumption of enrollment in the United States. At that meeting, the FDA stated that the existing three-month safety data could support 12-week dosing and that six months of clinical safety data could support dosing in the 12-month Phase 2 study.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt;">The most advanced molecule in the Company’s metabolic platform is sepiapterin, a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in metabolism and synthesis of numerous metabolic products, for orphan diseases. In May 2023, the Company announced that the primary endpoint was achieved in its registration-directed Phase 3 trial for sepiapterin for <span style="background:#ffffff;">phenylketonuria (“PKU”)</span>. The primary endpoint of the study was the achievement of statistically-significant reduction in blood Phe level. 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 </p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">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. PTC expects to submit an NDA to the FDA for sepiapterin for the treatment of PKU by the end of 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 review process of the NDA. Additionally, PTC expects to submit a marketing authorization application (“MAA”) to the EMA for sepiapterin for the treatment of PKU in the EEA in the first half of 2024.</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 utreloxastat. The Company announced topline results from a registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia in May 2023. While the study did not meet its primary endpoint of statistically significant change, the Company participated in a Type C written response only meeting with the FDA in the third quarter of 2023 to discuss the potential for an NDA submission for vatiquinone for the treatment of Friedreich ataxia. In their written response, the FDA stated that while they see the value of upright stability as a clinically meaningful endpoint, they believed a confirmatory study would likely be needed to support NDA submission. PTC has requested a follow-up live meeting to address the issues raised by the FDA.  Additionally, PTC is participating in a scientific advice procedure with the EMA to determine if the data from the registration-directed Phase 3 trial of vatiquinone in children and young adults with Friedreich ataxia could support a conditional MAA in the EEA. The Company initiated a Phase 2 trial of utreloxastat for amyotrophic lateral sclerosis in the first quarter of 2022 and enrollment is ongoing.</p><p style="font-family:'Times New Roman','Times','serif';font-size:10pt;text-align:justify;margin:0pt 0pt 12pt 0pt;">Unesbulin is the Company’s most advanced oncology agent. 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 enrollment is ongoing. The Company is evaluating its plans for a potential initiation of a registration-directed Phase 2/3 trial of unesbulin for the treatment of DIPG.</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 September 30, 2023, the Company had an accumulated deficit of approximately $3,127.8 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 (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), 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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