PTC Therapeutics (PTCT)
Market Price (9/23/2026): $65.93 | Market Cap: $5.5 BilSector: Health Care | Industry: Pharmaceuticals
PTC Therapeutics (PTCT)
Market Price (9/23/2026): $65.93Market Cap: $5.5 BilSector: Health CareIndustry: Pharmaceuticals
Investment Highlights Why It Matters Detailed financial logic regarding cash flow yields vs trend-riding momentum.
Cash is significant % of market capNet D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -28% Low stock price volatilityVol 12M is 40% Megatrend and thematic driversMegatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more. | Meaningful short interestShort Interest Days-to-CoverDTC = (Short Interest Share Quantity) / (Average Daily Trading Volume). Reflects how many days it would take to cover (close out) the short interest based on average volumes. High DTC can signify an increased risk of a short squeeze. is 12.13, Short Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 12% | Expensive valuation multiplesP/EBITPrice/EBIT or Price/(Operating Income) ratio is 46x Weak revenue growthRev Chg LTMRevenue Change % Last Twelve Months (LTM) is -43% Not cash flow generativeCFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -3.0%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -33% Yield minus risk free rate is negativeERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -4.8% Key risksPTCT key risks include [1] significant revenue concentration in a few key products, Show more. |
| Cash is significant % of market capNet D/ENet Debt/Equity. Debt net of cash. Negative indicates net cash. Equity is taken as the Market Capitalization is -28% |
| Low stock price volatilityVol 12M is 40% |
| Megatrend and thematic driversMegatrends include Biotechnology & Genomics, and Precision Medicine. Themes include Gene Editing & Therapy, Targeted Therapies, Show more. |
| Meaningful short interestShort Interest Days-to-CoverDTC = (Short Interest Share Quantity) / (Average Daily Trading Volume). Reflects how many days it would take to cover (close out) the short interest based on average volumes. High DTC can signify an increased risk of a short squeeze. is 12.13, Short Interest % of Basic SharesShort Interest % of Basic Shares = (Short Interest Quantity) / (Basic Shares Outstanding). A high fraction of short interest can indicate potential risk of a short squeeze. is 12% |
| Expensive valuation multiplesP/EBITPrice/EBIT or Price/(Operating Income) ratio is 46x |
| Weak revenue growthRev Chg LTMRevenue Change % Last Twelve Months (LTM) is -43% |
| Not cash flow generativeCFO/Rev LTMCash Flow from Operations / Revenue (Sales), Last Twelve Months (LTM) is -3.0%, FCF/Rev LTMFree Cash Flow / Revenue (Sales), Last Twelve Months (LTM) is -33% |
| Yield minus risk free rate is negativeERPEquity Risk Premium (ERP) = Total Yield - Risk Free Rate, Reflects the premium above risk free assets offered by the investment. is -4.8% |
| Key risksPTCT key risks include [1] significant revenue concentration in a few key products, Show more. |
Qualitative Assessment
AI Analysis | Feedback
Updated on 9/11/2026
PTC Therapeutics (PTCT) stock has lost about 10% since 5/31/2026 because of the following key factors:
1. Decline in Legacy Product Revenue Exceeding Expectations.
Despite overall strong Q2 2026 financial results reported on July 30, 2026, PTC Therapeutics experienced notable declines in revenue from key established products. Translarna (ataluren) net product revenues decreased to $42.2 million in fiscal Q2 2026, compared to $59.5 million in fiscal Q2 2025. Similarly, Emflaza (deflazacort) net product revenues fell to $24.6 million in fiscal Q2 2026, from $36.4 million in fiscal Q2 2025, primarily due to continued generic erosion. This erosion of revenue from mature products likely contributed to investor concerns regarding the long-term stability of the company's existing portfolio.
2. Regulatory Setback and Delayed Approval for Vatiquinone.
The company announced that despite the FDA indicating that the data package for vatiquinone in Friedreich's ataxia was supportive of approval, the agency required an additional study due to a "strict statistical view". PTC Therapeutics plans to initiate a new open-label study in fiscal Q3 2026, enrolling approximately 120 individuals. This requirement for further clinical trials delays the potential market entry and revenue generation from this pipeline candidate, impacting future growth projections.
Show more
PTC Therapeutics (PTCT) stock has lost about 10% since 5/31/2026 because of the following key factors:
1. Decline in Legacy Product Revenue Exceeding Expectations.
Despite overall strong Q2 2026 financial results reported on July 30, 2026, PTC Therapeutics experienced notable declines in revenue from key established products. Translarna (ataluren) net product revenues decreased to $42.2 million in fiscal Q2 2026, compared to $59.5 million in fiscal Q2 2025. Similarly, Emflaza (deflazacort) net product revenues fell to $24.6 million in fiscal Q2 2026, from $36.4 million in fiscal Q2 2025, primarily due to continued generic erosion. This erosion of revenue from mature products likely contributed to investor concerns regarding the long-term stability of the company's existing portfolio.
2. Regulatory Setback and Delayed Approval for Vatiquinone.
The company announced that despite the FDA indicating that the data package for vatiquinone in Friedreich's ataxia was supportive of approval, the agency required an additional study due to a "strict statistical view". PTC Therapeutics plans to initiate a new open-label study in fiscal Q3 2026, enrolling approximately 120 individuals. This requirement for further clinical trials delays the potential market entry and revenue generation from this pipeline candidate, impacting future growth projections.
Show more
Updated on 9/11/2026
PTC Therapeutics (PTCT) stock has lost about 10% since 5/31/2026 because of the following key factors:
1. Decline in Legacy Product Revenue Exceeding Expectations.
Despite overall strong Q2 2026 financial results reported on July 30, 2026, PTC Therapeutics experienced notable declines in revenue from key established products. Translarna (ataluren) net product revenues decreased to $42.2 million in fiscal Q2 2026, compared to $59.5 million in fiscal Q2 2025. Similarly, Emflaza (deflazacort) net product revenues fell to $24.6 million in fiscal Q2 2026, from $36.4 million in fiscal Q2 2025, primarily due to continued generic erosion. This erosion of revenue from mature products likely contributed to investor concerns regarding the long-term stability of the company's existing portfolio.
2. Regulatory Setback and Delayed Approval for Vatiquinone.
The company announced that despite the FDA indicating that the data package for vatiquinone in Friedreich's ataxia was supportive of approval, the agency required an additional study due to a "strict statistical view". PTC Therapeutics plans to initiate a new open-label study in fiscal Q3 2026, enrolling approximately 120 individuals. This requirement for further clinical trials delays the potential market entry and revenue generation from this pipeline candidate, impacting future growth projections.
3. Negative Market Reaction Post-Q2 2026 Earnings Despite Beats.
On July 30, 2026, the day PTC Therapeutics reported its fiscal Q2 2026 earnings, the stock experienced a significant drop of 9.94%. This occurred even though the company announced an EPS of $0.92, which substantially beat the consensus estimate of $0.24, and quarterly revenue of $360.52 million, exceeding analysts' expectations of $298.63 million. The market's negative reaction, despite headline beats and raised full-year 2026 total revenue guidance to $1.18 to $1.28 billion, suggests investors focused on underlying concerns, such as the declines in legacy product sales and the setback with the vatiquinone program.
Show less
PTC Therapeutics (PTCT) stock has lost about 10% since 5/31/2026 because of the following key factors:
1. Decline in Legacy Product Revenue Exceeding Expectations.
Despite overall strong Q2 2026 financial results reported on July 30, 2026, PTC Therapeutics experienced notable declines in revenue from key established products. Translarna (ataluren) net product revenues decreased to $42.2 million in fiscal Q2 2026, compared to $59.5 million in fiscal Q2 2025. Similarly, Emflaza (deflazacort) net product revenues fell to $24.6 million in fiscal Q2 2026, from $36.4 million in fiscal Q2 2025, primarily due to continued generic erosion. This erosion of revenue from mature products likely contributed to investor concerns regarding the long-term stability of the company's existing portfolio.
2. Regulatory Setback and Delayed Approval for Vatiquinone.
The company announced that despite the FDA indicating that the data package for vatiquinone in Friedreich's ataxia was supportive of approval, the agency required an additional study due to a "strict statistical view". PTC Therapeutics plans to initiate a new open-label study in fiscal Q3 2026, enrolling approximately 120 individuals. This requirement for further clinical trials delays the potential market entry and revenue generation from this pipeline candidate, impacting future growth projections.
3. Negative Market Reaction Post-Q2 2026 Earnings Despite Beats.
On July 30, 2026, the day PTC Therapeutics reported its fiscal Q2 2026 earnings, the stock experienced a significant drop of 9.94%. This occurred even though the company announced an EPS of $0.92, which substantially beat the consensus estimate of $0.24, and quarterly revenue of $360.52 million, exceeding analysts' expectations of $298.63 million. The market's negative reaction, despite headline beats and raised full-year 2026 total revenue guidance to $1.18 to $1.28 billion, suggests investors focused on underlying concerns, such as the declines in legacy product sales and the setback with the vatiquinone program.
Show less
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Stock Movement Drivers
Fundamental Drivers
The -10.7% change in PTCT stock from 5/31/2026 to 9/22/2026 was primarily driven by a -26.3% change in the company's P/S Multiple.| (LTM values as of) | 5312026 | 9222026 | Change |
|---|---|---|---|
| Stock Price ($) | 73.85 | 65.96 | -10.7% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 827 | 1,009 | 22.0% |
| P/S Multiple | 7.4 | 5.4 | -26.3% |
| Shares Outstanding (Mil) | 83 | 83 | -0.6% |
| Cumulative Contribution | -10.7% |
Market Drivers
5/31/2026 to 9/22/2026| Return | Correlation | |
|---|---|---|
| PTCT | -10.7% | |
| Market (SPY) | 2.5% | -1.9% |
| Sector (XLV) | 14.2% | 31.5% |
Fundamental Drivers
The -3.3% change in PTCT stock from 2/28/2026 to 9/22/2026 was primarily driven by a -41.7% change in the company's Total Revenues ($ Mil).| (LTM values as of) | 2282026 | 9222026 | Change |
|---|---|---|---|
| Stock Price ($) | 68.19 | 65.96 | -3.3% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 1,731 | 1,009 | -41.7% |
| P/S Multiple | 3.2 | 5.4 | 67.6% |
| Shares Outstanding (Mil) | 82 | 83 | -1.0% |
| Cumulative Contribution | -3.3% |
Market Drivers
2/28/2026 to 9/22/2026| Return | Correlation | |
|---|---|---|
| PTCT | -3.3% | |
| Market (SPY) | 13.3% | 21.6% |
| Sector (XLV) | 7.0% | 27.2% |
Fundamental Drivers
The 33.7% change in PTCT stock from 8/31/2025 to 9/22/2026 was primarily driven by a 148.5% change in the company's P/S Multiple.| (LTM values as of) | 8312025 | 9222026 | Change |
|---|---|---|---|
| Stock Price ($) | 49.33 | 65.96 | 33.7% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 1,765 | 1,009 | -42.8% |
| P/S Multiple | 2.2 | 5.4 | 148.5% |
| Shares Outstanding (Mil) | 78 | 83 | -5.8% |
| Cumulative Contribution | 33.7% |
Market Drivers
8/31/2025 to 9/22/2026| Return | Correlation | |
|---|---|---|
| PTCT | 33.7% | |
| Market (SPY) | 21.2% | 22.7% |
| Sector (XLV) | 25.8% | 33.0% |
Fundamental Drivers
The 67.0% change in PTCT stock from 8/31/2023 to 9/22/2026 was primarily driven by a 50.5% change in the company's P/S Multiple.| (LTM values as of) | 8312023 | 9222026 | Change |
|---|---|---|---|
| Stock Price ($) | 39.50 | 65.96 | 67.0% |
| Change Contribution By: | |||
| Total Revenues ($ Mil) | 819 | 1,009 | 23.2% |
| P/S Multiple | 3.6 | 5.4 | 50.5% |
| Shares Outstanding (Mil) | 75 | 83 | -10.0% |
| Cumulative Contribution | 67.0% |
Market Drivers
8/31/2023 to 9/22/2026| Return | Correlation | |
|---|---|---|
| PTCT | 67.0% | |
| Market (SPY) | 78.3% | 27.8% |
| Sector (XLV) | 34.1% | 29.6% |
Price Returns Compared
| 2021 | 2022 | 2023 | 2024 | 2025 | 2026 | Total [1] | |
|---|---|---|---|---|---|---|---|
| Returns | |||||||
| PTCT Return | -35% | -4% | -28% | 64% | 68% | -14% | 7% |
| Peers Return | -19% | 13% | 1% | -12% | -22% | 0% | -36% |
| S&P 500 Return | 27% | -19% | 24% | 23% | 16% | 13% | 107% |
Monthly Win Rates [3] | |||||||
| PTCT Win Rate | 33% | 58% | 42% | 83% | 58% | 33% | |
| Peers Win Rate | 45% | 57% | 43% | 40% | 53% | 51% | |
| S&P 500 Win Rate | 75% | 42% | 67% | 75% | 67% | 56% | |
Max Drawdowns [4] | |||||||
| PTCT Max Drawdown | -49% | -43% | -69% | -24% | -37% | -27% | |
| Peers Max Drawdown | -42% | -31% | -35% | -34% | -46% | -31% | |
| S&P 500 Max Drawdown | -5% | -25% | -10% | -8% | -19% | -9% | |
[1] Cumulative total returns since the beginning of 2021
[2] Peers: SRPT, BIIB, BMRN, RARE, VRTX.
[3] Win Rate = % of calendar months in which monthly returns were positive
[4] Max drawdown represents maximum peak-to-trough decline within a year
[5] 2026 data is for the year up to 9/22/2026 (YTD)
How Low Can It Go
| Event | PTCT | S&P 500 |
|---|---|---|
| 2025 US Tariff Shock | ||
| % Loss | -28.2% | -18.8% |
| % Gain to Breakeven | 39.3% | 23.1% |
| Time to Breakeven | 28 days | 79 days |
| Summer-Fall 2023 Five Percent Yield Shock | ||
| % Loss | -53.5% | -9.5% |
| % Gain to Breakeven | 114.8% | 10.5% |
| Time to Breakeven | 200 days | 24 days |
| 2023 SVB Regional Banking Crisis | ||
| % Loss | -12.5% | -6.7% |
| % Gain to Breakeven | 14.2% | 7.1% |
| Time to Breakeven | 503 days | 31 days |
| 2022 Inflation Shock & Fed Tightening | ||
| % Loss | -39.9% | -24.5% |
| % Gain to Breakeven | 66.3% | 32.4% |
| Time to Breakeven | 22 days | 427 days |
| 2020 COVID-19 Crash | ||
| % Loss | -45.5% | -33.7% |
| % Gain to Breakeven | 83.4% | 50.9% |
| Time to Breakeven | 233 days | 140 days |
| Q4 2018 Fed Policy Error / Growth Scare | ||
| % Loss | -34.1% | -19.2% |
| % Gain to Breakeven | 51.8% | 23.8% |
| Time to Breakeven | 212 days | 105 days |
In The Past
PTC Therapeutics's stock fell -28.2% during the 2025 US Tariff Shock. Such a loss loss requires a 39.3% gain to breakeven.
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| Event | PTCT | S&P 500 |
|---|---|---|
| 2025 US Tariff Shock | ||
| % Loss | -28.2% | -18.8% |
| % Gain to Breakeven | 39.3% | 23.1% |
| Time to Breakeven | 28 days | 79 days |
| Summer-Fall 2023 Five Percent Yield Shock | ||
| % Loss | -53.5% | -9.5% |
| % Gain to Breakeven | 114.8% | 10.5% |
| Time to Breakeven | 200 days | 24 days |
| 2022 Inflation Shock & Fed Tightening | ||
| % Loss | -39.9% | -24.5% |
| % Gain to Breakeven | 66.3% | 32.4% |
| Time to Breakeven | 22 days | 427 days |
| 2020 COVID-19 Crash | ||
| % Loss | -45.5% | -33.7% |
| % Gain to Breakeven | 83.4% | 50.9% |
| Time to Breakeven | 233 days | 140 days |
| Q4 2018 Fed Policy Error / Growth Scare | ||
| % Loss | -34.1% | -19.2% |
| % Gain to Breakeven | 51.8% | 23.8% |
| Time to Breakeven | 212 days | 105 days |
| 2016-2017 Trump Reflation Bond Selloff | ||
| % Loss | -64.3% | -3.7% |
| % Gain to Breakeven | 180.0% | 3.9% |
| Time to Breakeven | 11 days | 6 days |
| 2015-2016 China Devaluation / Global Growth Scare | ||
| % Loss | -86.7% | -12.2% |
| % Gain to Breakeven | 649.1% | 13.9% |
| Time to Breakeven | 824 days | 62 days |
| 2014-2016 Oil Price Collapse | ||
| % Loss | -28.4% | -6.8% |
| % Gain to Breakeven | 39.7% | 7.3% |
| Time to Breakeven | 797 days | 15 days |
In The Past
PTC Therapeutics's stock fell -28.2% during the 2025 US Tariff Shock. Such a loss loss requires a 39.3% gain to breakeven.
Preserve Wealth
Limiting losses and compounding gains is essential to preserving wealth.
Asset Allocation
Actively managed asset allocation strategies protect wealth. Learn more.
About PTC Therapeutics (PTCT)
PTC Therapeutics, Inc. is a biopharmaceutical company specializing in the discovery, development, and commercialization of medicines for patients suffering from rare genetic disorders. The company maintains a comprehensive portfolio ranging from commercialized therapies to candidates in various stages of clinical and preclinical development, focusing on addressing significant unmet medical needs.
The core of PTC's commercial offerings includes treatments for Duchenne muscular dystrophy (DMD): Translarna, available in the European Economic Area, Brazil, and Russia, and Emflaza, marketed in the United States. Additionally, PTC commercializes Tegsedi and Waylivra for other rare diseases across Latin America and the Caribbean, and Evrysdi for spinal muscular atrophy (SMA) in Brazil, often leveraging strategic collaborations to expand market access.
Beyond its current commercial products, PTC Therapeutics is actively advancing its pipeline, notably with PTC518, a promising candidate for Huntington's disease, developed using its innovative splicing platform. Through its commitment to scientific innovation and global collaborations, PTC Therapeutics aims to provide life-changing therapies to patients with rare diseases across diverse geographic markets.
AI Analysis | Feedback
Analogy 1: A BioMarin Pharmaceutical for a diverse set of rare diseases.
Analogy 2: A Vertex Pharmaceuticals, specializing in a wider range of rare genetic disorders.
AI Analysis | Feedback
- Translarna: A medicine for the treatment of Duchenne muscular dystrophy, including nonsense mutation Duchenne muscular dystrophy.
- Emflaza: A commercial product used for the treatment of Duchenne muscular dystrophy.
- Tegsedi: Commercialized for the treatment of various rare diseases.
- Waylivra: Commercialized for the treatment of various rare diseases.
- Evrysdi: Marketed for the treatment of spinal muscular atrophy in adults and children.
- PTC518: A product candidate under development for the treatment of Huntington's disease.
AI Analysis | Feedback
PTC Therapeutics, Inc. (PTCT) primarily sells its pharmaceutical products to individuals (patients) suffering from rare disorders. Based on the company description, its major customer categories are:
- Individuals with Duchenne muscular dystrophy (DMD)
- Individuals with Spinal Muscular Atrophy (SMA)
- Individuals with other rare diseases targeted by commercialized products such as Tegsedi and Waylivra
AI Analysis | Feedback
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Matthew B. Klein, M.D., M.S., F.A.C.S. Chief Executive Officer
Dr. Klein has served as Chief Executive Officer of PTC Therapeutics since March 2023. He joined PTC in October 2019, holding various leadership roles including Global Head, Gene and Mitochondrial Therapies, Global Head, Clinical Development, Chief Development Officer, and Chief Operating Officer. Before joining PTC, Dr. Klein was the Chief Executive Officer and Chief Medical Officer of BioElectron Technology Corporation from 2018 to 2019, a biotechnology company focused on rare diseases, which was subsequently acquired by PTC in 2019. He also previously held the position of Senior Vice President, Clinical Science at BioElectron from 2012 to 2013. Dr. Klein has served on the board of directors of ClearPoint Neuro, Inc. since 2020.
Pierre Gravier Chief Financial Officer
Mr. Gravier was appointed Chief Financial Officer of PTC Therapeutics in July 2023. He brings over 17 years of experience as an investment banker, venture capitalist, and scientist. Prior to joining PTC, Mr. Gravier was a managing director in the healthcare group of Perella Weinberg Partners from 2013 to 2023, where he advised companies in the biopharmaceutical and pharmaceutical sectors on finance strategy and corporate development. Before Perella Weinberg, he worked as a healthcare investment banker at Barclays Capital in London from 2009 to 2013. He also served as a venture capital analyst at Société Générale Asset Management in Paris, focusing on early-stage biotechnology investments, and began his career as a scientist at Ferring Pharmaceuticals.
Eric Pauwels Chief Business Officer
Mr. Pauwels became Chief Business Officer in 2020, having initially joined PTC in March 2015 as Senior Vice President and General Manager of the Americas. He has more than 40 years of healthcare experience and has launched over 30 biopharmaceutical products globally, with a significant focus on rare diseases in the last 18 years. Prior to his tenure at PTC, Mr. Pauwels was the Chief Commercial Officer and President of International for NPS Pharmaceuticals from 2011 to 2015. NPS Pharmaceuticals was acquired by Shire in 2015, around the time Mr. Pauwels transitioned to PTC. Before NPS, he was the first Chief Commercial Officer for Shire Human Genetic Therapies Rare Disease Business from 2005 to 2010. He also held global marketing and management roles at companies such as Bayer, Fournier, and Johnson & Johnson.
Lee Golden, M.D. Executive Vice President and Chief Medical Officer
Dr. Golden joined PTC Therapeutics in 2020 as Senior Vice President, Head of Global Clinical Development, and was appointed Chief Medical Officer in 2022, becoming Executive Vice President and Chief Medical Officer in April 2023. He leads global clinical development for PTC's pipeline assets. Prior to PTC, Dr. Golden served as Chief Medical Officer at Espero BioPharma, Inc., a privately held clinical-stage biopharmaceutical company, and as Chief Medical Officer at Gemphire Therapeutics, a clinical-stage biopharmaceutical company. His industry experience spans over 20 years, including senior roles at large pharmaceutical and biotech companies such as Pfizer, Actelion, Elan, Eisai, Mesoblast, and Novartis. He also serves as Chairman of the Advisory Board for Coagulation Sciences LLC.
Neil Almstead, Ph.D. Chief Technical Operations Officer
Dr. Almstead has been with PTC Therapeutics for over 20 years, holding various executive positions since joining in 2000. He was promoted to Chief Technical Operations Officer in January 2019. In this role, he is responsible for overseeing research, manufacturing, and the supply chain for both small-molecule and gene-therapy programs, and is leading the establishment of in-house gene therapy manufacturing. Before joining PTC, Dr. Almstead worked as a project manager at Procter & Gamble.
AI Analysis | Feedback
The key risks to PTC Therapeutics' business are primarily centered on regulatory challenges, pipeline development setbacks, and competitive pressures.
1. **Regulatory Challenges and Loss of Product Approvals:** PTC Therapeutics faces significant and ongoing regulatory hurdles, most notably with its Duchenne muscular dystrophy (DMD) drug, Translarna (ataluren). The company recently withdrew its New Drug Application (NDA) resubmission for Translarna in the United States after the FDA indicated that the submitted data was unlikely to meet the threshold for substantial evidence of effectiveness to support approval. This decision followed a major setback in Europe, where Translarna's conditional marketing authorization was not renewed in late 2024, despite its initial approval in 2014. These developments are highly impactful as Translarna, alongside Emflaza, constituted PTC's top-performing products, and the loss of key market access significantly jeopardizes revenue streams. Furthermore, the company previously faced an FDA denial for its Friedreich's ataxia candidate, vatiquinone, due to insufficient evidence of efficacy. The reliance on successful regulatory approvals means that negative outcomes can materially impact the company's ability to commercialize products and affect its financial health.
2. **Clinical Trial and Regulatory Risks for Pipeline Products:** The successful development and approval of pipeline candidates are critical for PTC Therapeutics' future growth, but this area carries substantial risk. For instance, while PTC518 for Huntington's disease met its primary endpoint in a Phase 2 study by significantly reducing Huntingtin protein levels, the clinical data generated considerable skepticism from investors regarding its ability to demonstrate a clear correlation between protein reduction and clinical outcomes, and its potential to significantly slow disease progression. The mixed results between Stage 2 and Stage 3 patients also suggest complexities in treatment effect, casting doubt on the path to accelerated approval. This investor skepticism led to a notable drop in the company's share price, highlighting the high stakes associated with pipeline development and regulatory alignment.
3. **Competition and Loss of Market Exclusivity:** PTC Therapeutics operates within a highly competitive biopharmaceutical industry, particularly in the rare disease segment. A significant risk is the loss of market exclusivity for key products, such as Emflaza, which has already occurred. This exposes the product to generic competition, potentially eroding market share and pricing power. For new product launches, such as sepiapterin for phenylketonuria (PKU), the company must effectively differentiate its offerings from existing treatments to gain market access and achieve favorable reimbursement. The ongoing threat of new entrants and the development of similar or superior products by competitors necessitate continuous innovation and strong market execution to maintain a competitive edge and ensure long-term revenue potential.
AI Analysis | Feedback
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AI Analysis | Feedback
PTC Therapeutics focuses on developing treatments for rare disorders, with several key products addressing significant addressable markets globally and in specific regions.
Translarna (ataluren) for Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)
The global Duchenne muscular dystrophy (DMD) treatment market, which includes nmDMD, was valued at approximately USD 4.79 billion in 2025 and is projected to increase to around USD 19.46 billion by 2034. Translarna specifically targets nonsense mutations, which are applicable to about 10-15% of DMD patients. In 2023, the DMD market in the seven major markets (7MM: U.S., France, Germany, Italy, Spain, U.K., and Japan) was valued at USD 2.3 billion, with projections to reach USD 5.2 billion by 2033. The U.S. alone accounted for approximately USD 1.9 billion of the DMD market in 2024. Translarna generated around USD 235.5 million in sales in Europe in 2025.Emflaza (deflazacort) for Duchenne Muscular Dystrophy (DMD)
Emflaza is a corticosteroid therapy for the broader Duchenne muscular dystrophy (DMD) market. The global DMD treatment market was valued at approximately USD 4.79 billion in 2025 and is expected to grow to about USD 19.46 billion by 2034. North America held approximately 46% of the global DMD market share in 2024. Emflaza's net product revenue for the full year 2023 was USD 255.1 million.Waylivra (volanesorsen) for Familial Chylomicronemia Syndrome (FCS)
The global Familial Chylomicronemia Syndrome (FCS) treatment market was valued at approximately USD 15.30 million in 2024 and is projected to reach USD 19.9 million by 2035. Another estimate places the global market at USD 14.23 million in 2024, expected to reach USD 20.83 million by 2032. Waylivra is currently approved in Europe but not in the U.S. The 7MM (U.S., EU4, U.K., and Japan) market for FCS was approximately USD 15 million in 2022.Evrysdi (risdiplam) for Spinal Muscular Atrophy (SMA)
The global spinal muscular atrophy (SMA) treatment market was estimated at USD 4.40 billion in 2024 and is projected to reach USD 13.09 billion by 2030. Another source projects the market to reach nearly USD 20.06 billion by 2033. North America held the largest revenue share in the SMA treatment market, accounting for 68.4% in 2024. Evrysdi's full-year sales for 2024 were approximately USD 1.8 billion, with forecasts suggesting sales could reach USD 2.9 billion by 2031.PTC518 for Huntington's Disease (HD)
The global Huntington's disease (HD) treatment market was valued at approximately USD 1.39 billion in 2025 and is projected to grow to USD 4.33 billion by 2034. North America accounted for a significant share of the HD treatment market, with 40.85% in 2024. The U.S. Huntington's Disease treatment market is expected to reach USD 552.5 million by 2033, growing from USD 154.38 million in 2024.Tegsedi (inotersen) for Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis) with Polyneuropathy
Information regarding the specific addressable market size for Tegsedi for hereditary transthyretin amyloidosis with polyneuropathy was not explicitly available in the provided search results.AI Analysis | Feedback
PTC Therapeutics (PTCT) is expected to drive future revenue growth over the next 2-3 years through several key initiatives:
- Global Expansion and Uptake of Sepiapterin (Sephience) for PKU: The successful launch and continued global expansion of Sephience (sepiapterin) for phenylketonuria (PKU) is a significant revenue driver. The drug was launched in the United States and Europe, with additional launches anticipated in Japan and Brazil. Management projects substantial revenue from this product, which generated approximately $111 million in its initial months post-launch in 2025 and is expected to be the primary contributor to product revenue growth in 2026.
- Advancement and Potential Accelerated Approval of PTC518 for Huntington's Disease: PTC518, an investigational therapy for Huntington's disease (HD), has shown promising Phase 2 results, meeting its primary endpoint by reducing blood Huntingtin protein levels. The company plans to work with the FDA on an accelerated approval pathway for this treatment. A collaboration agreement with Novartis for the PTC518 program includes potential milestone payments of up to $1.9 billion, indicating its significant financial impact.
- Continued Growth and Geographic Expansion of the Duchenne Muscular Dystrophy (DMD) Franchise: Despite facing regulatory setbacks for Translarna in the U.S. and potential generic competition for Emflaza, the DMD franchise continues to be a meaningful revenue contributor. The company's strategy involves driving growth through new patients in existing markets and ongoing geographic expansion for its Duchenne muscular dystrophy therapies, Translarna and Emflaza.
- Global Rollout and Expansion of Gene Therapy for AADC Deficiency (Upstaza): The ongoing global rollout of PTC's gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, Upstaza, is another anticipated growth driver. The company expects to expand this therapy into new territories in the coming years, thereby increasing its patient reach and diversifying revenue streams.
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Share Issuance
- PTC Therapeutics has issued restricted stock units (RSUs) and stock options as inducement grants to new employees, including 14,550 stock options and 17,515 RSUs approved on March 10, 2026, for 18 new hires.
- A "Post IPO" funding round occurred on May 16, 2023, though the specific dollar amount of shares issued was not disclosed.
- The company maintains an "Amended and Restated 2016 Employee Stock Purchase Plan" which allows for the issuance of up to 2,000,000 shares of common stock to eligible employees.
Inbound Investments
- In December 2025, PTC Therapeutics sold the remainder of its Evrysdi® royalty to Royalty Pharma for an upfront payment of $240 million, with potential for up to an additional $60 million in sales-based milestones.
- A collaboration and license agreement with Novartis for votoplam, which closed in January 2025, generated $998.4 million in collaboration and license revenue for PTC Therapeutics in 2025.
- Several institutional investors acquired significant stakes in PTC Therapeutics, including Jefferies Financial Group, which purchased approximately $97.2 million worth of shares in Q3 2026, and Palo Alto Investors, which increased its position by approximately $33.1 million in Q3 2025.
Outbound Investments
- In 2024, PTC Therapeutics acquired Censa Pharmaceuticals for $250 million, adding the phase 3-ready asset CTRX-100 for phenylketonuria to its pipeline.
Capital Expenditures
- PTC Therapeutics' capital expenditures were approximately $8.79 million in fiscal year 2025.
- In the last 12 months, the company reported capital expenditures of -$8.86 million.
- Annual capital expenditures were $28 million in 2021, $32 million in 2022, and $28 million in 2023.
Peer Outperformance in Pharmaceuticals
PTCT has outperformed 76% of its 62 Pharmaceuticals peers over 5Y. Among the peers that beat it is CORT. Pharmaceuticals ranks 7th of 10 industries in Health Care by median 5Y return. The sector’s gains have largely come from elsewhere.
Share of Pharmaceuticals constituents that PTCT has outperformed, by holding period. Peers without a full price history over a given window are excluded from that window.
1Y
57%
of 74 industry peers · 8.4% return
3Y
86%
of 69 industry peers · 175.8% return
5Y
76%
of 62 industry peers · 67.1% return
Pharmaceuticals peers with revenue growth within 4pp of PTCT's that beat it on at least 2 of 3 windows, and by at least 20pp over 5Y. Growth is matched by construction, so the gap is largely a re-rating gap. The multiple column is the context for it.
| Ticker | Name | Rev Growth 3Y Avg | P/E | 1Y | 3Y | 5Y | 5Y Gap |
|---|---|---|---|---|---|---|---|
| PTCT | PTC Therapeutics | 21.0% | -142.6x | 8.4% | 175.8% | 67.1% | — |
| CORT | Corcept Therapeutics | 24.9% | 224.4x | 46.9% | 271.4% | 473.5% | +406pp |
Price returns, excluding dividends. P/E shown for context only and not used for matching.
Median price return by industry across the Health Care sector, ranked by 5Y. Pharmaceuticals is PTCT's own industry. Use this to see which corners of the sector have carried the returns.
| Industry | Names | 1Y med | 3Y med | 5Y med | Top 3 by 5Y |
|---|---|---|---|---|---|
| Health Care Distributors | 6 | 19.0% | 46.5% | 91.6% | CAH 382% · MCK 351% · COR 171% |
| Managed Health Care | 8 | 37.7% | -6.6% | 0.6% | OSCR 79% · HQY 41% · ELV 14% |
| Health Care Services | 20 | 19.1% | 35.3% | -0.5% | HIMS 269% · RDNT 159% · DGX 69% |
| Health Care Facilities | 24 | 5.9% | 13.0% | -4.0% | NHC 269% · THC 261% · ENSG 135% |
| Health Care Equipment | 50 | -2.1% | -0.4% | -21.5% | POCI 11363% · UFPT 354% · GKOS 204% |
| Health Care Supplies | 12 | 19.5% | -6.4% | -38.8% | LNTH 270% · HAE 56% · MMSI 19% |
| Pharmaceuticals ← | 63 | -6.9% | 13.1% | -42.8% | LQDA 2369% · INDV 1106% · ETON 1024% |
| Life Sciences Tools & Services | 113 | 2.5% | -8.4% | -67.1% | SNWV 1794% · MEDP 223% · NTRA 216% |
| Biotechnology | 378 | 0.3% | -19.9% | -78.2% | CELZ 1243% · DNTH 1189% · TRVI 1055% |
| Health Care Technology | 21 | -24.3% | -49.3% | -78.8% | SPOK 71% · HSTM 2% · VEEV -13% |
Median of constituents with a full price history over each window. Top names are unfiltered by size in this render.
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Peer Comparisons
| Peers to compare with: |
Financials
| Median | |
|---|---|
| Name | |
| Mkt Price | 64.66 |
| Mkt Cap | 8.9 |
| Rev LTM | 2,690 |
| Op Inc LTM | 133 |
| FCF LTM | 400 |
| FCF 3Y Avg | 290 |
| CFO LTM | 484 |
| CFO 3Y Avg | 419 |
Growth & Margins
| Median | |
|---|---|
| Name | |
| Rev Chg LTM | 5.3% |
| Rev Chg 3Y Avg | 18.1% |
| Rev Chg Q | 16.2% |
| QoQ Delta Rev Chg LTM | 4.0% |
| Op Inc Chg LTM | -51.2% |
| Op Inc Chg 3Y Avg | 43.8% |
| Op Mgn LTM | 8.0% |
| Op Mgn 3Y Avg | 13.4% |
| QoQ Delta Op Mgn LTM | -1.7% |
| CFO/Rev LTM | 15.4% |
| CFO/Rev 3Y Avg | 13.8% |
| FCF/Rev LTM | 12.5% |
| FCF/Rev 3Y Avg | 4.4% |
Valuation
| Median | |
|---|---|
| Name | |
| Mkt Cap | 8.9 |
| P/S | 3.5 |
| P/Op Inc | 22.3 |
| P/EBIT | 25.8 |
| P/E | 13.5 |
| P/CFO | 12.9 |
| Total Yield | -0.1% |
| Dividend Yield | 0.0% |
| FCF Yield 3Y Avg | 1.3% |
| D/E | 0.2 |
| Net D/E | 0.0 |
FDA Approved Drugs Data
Expand for More| Post-Approval Fwd Returns | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| FDA App # | Brand Name | Generic Name | Dosage Form | FDA Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| NDA219666 | SEPHIENCE | sepiapterin | powder | 7282025 | 51.3% | 71.9% | 70.0% | 48.8% | 48.8% |
| NDA208684 | EMFLAZA | deflazacort | tablet | 2092017 | -6.2% | 32.8% | 66.4% | 115.1% | 377.3% |
| NDA208685 | EMFLAZA | deflazacort | suspension | 2092017 | -6.2% | 32.8% | 66.4% | 115.1% | 377.3% |
FDA-Licensed Biologics (CBER · Purple Book)
| Post-Approval Fwd Returns | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| FDA BLA # | Brand Name | Generic Name | License Type | First Approval | Latest Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| 125722 | KEBILIDI | eladocagene exuparvovec-tneq | 351(a) | 11/13/2024 | 11/13/2024 | 11.1% | 6.2% | 70.2% | 49.8% | 49.8% |
| Post-Approval Fwd Returns | |||||||||
|---|---|---|---|---|---|---|---|---|---|
| FDA App # | Brand Name | Generic Name | Dosage Form | FDA Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| NDA219666 | SEPHIENCE | sepiapterin | powder | 7282025 | 51.3% | 71.9% | 70.0% | 48.8% | 48.8% |
| NDA208684 | EMFLAZA | deflazacort | tablet | 2092017 | -6.2% | 32.8% | 66.4% | 115.1% | 377.3% |
| NDA208685 | EMFLAZA | deflazacort | suspension | 2092017 | -6.2% | 32.8% | 66.4% | 115.1% | 377.3% |
FDA-Licensed Biologics (CBER · Purple Book)
| Post-Approval Fwd Returns | ||||||||||
|---|---|---|---|---|---|---|---|---|---|---|
| FDA BLA # | Brand Name | Generic Name | License Type | First Approval | Latest Approval | 3M Rtn | 6M Rtn | 1Y Rtn | 2Y Rtn | Total Rtn |
| 125722 | KEBILIDI | eladocagene exuparvovec-tneq | 351(a) | 11/13/2024 | 11/13/2024 | 11.1% | 6.2% | 70.2% | 49.8% | 49.8% |
Clinical Pipeline
| Drug Candidate | Indication | Phase 3 Trials | Est. Primary Completion |
|---|---|---|---|
| Vatiquinone (PTC743) | Friedreich Ataxia | 1 | Dec 2027 |
| Sepiapterin (PTC923) | Phenylketonuria | 2 | Feb 2031 |
Active Phase 3 studies led by the company. Source: ClinicalTrials.gov · Updated 8/28/2026
Segment Financials
Revenue by Segment| $ Mil | 2025 | 2024 | 2023 | 2022 | 2021 |
|---|---|---|---|---|---|
| Collaboration and license revenue | 998 | 0 | 100 | 55 | |
| Royalty revenue | 244 | 204 | 169 | 55 | |
| Translarna | 235 | 321 | 333 | ||
| Emflaza | 146 | 207 | 255 | ||
| Sephience | 111 | 0 | 0 | ||
| Upstaza/Kebilidi | 57 | 17 | 19 | ||
| All other products | 37 | 37 | 31 | ||
| Manufacturing revenue | 0 | 2 | 8 | 0 | |
| Translarna France | -99 | 19 | 23 | ||
| Life science | 699 | ||||
| Net product revenue | 429 | ||||
| Total | 1,731 | 807 | 938 | 699 | 539 |
| $ Mil | 2024 | 2022 |
|---|---|---|
| Life science | -363 | -559 |
| Total | -363 | -559 |
Price Behavior
Price Behavior
| Market Price | $65.96 | |
| Market Cap ($ Bil) | 5.5 | |
| First Trading Date | 06/20/2013 | |
| Distance from 52W High | -26.3% | |
| 50 Days | 200 Days | |
| DMA Price | $71.88 | $72.56 |
| DMA Trend | indeterminate | down |
| Distance from DMA | -8.2% | -9.1% |
| 3M | 1YR | |
| Volatility | 37.3% | 39.9% |
| Downside Capture | 33.33 | 85.34 |
| Upside Capture | -82.36 | 79.12 |
| Correlation (SPY) | -1.1% | 22.3% |
PTCT Betas & Captures as of 8/31/2026
[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
| 1M | 2M | 3M | 6M | 1Y | 3Y | |
|---|---|---|---|---|---|---|
| Beta | 0.84 | 0.32 | -0.07 | 0.77 | 0.72 | 1.06 |
| Up Beta | 0.48 | 1.34 | 1.23 | 1.50 | 1.42 | 1.00 |
| Down Beta | 4.85 | 1.67 | 0.21 | 0.05 | -0.23 | 0.76 |
| Up Capture | 64% | -90% | -65% | 57% | 93% | 199% |
| Bmk +ve Days | 10 | 21 | 32 | 68 | 138 | 427 |
| Stock +ve Days | 10 | 19 | 32 | 64 | 130 | 375 |
| Down Capture | 51% | 25% | -63% | 80% | 77% | 106% |
| Bmk -ve Days | 11 | 21 | 32 | 59 | 113 | 324 |
| Stock -ve Days | 11 | 23 | 31 | 62 | 119 | 371 |
[1] Upside and downside betas calculated using positive and negative benchmark daily returns respectively
Based On 1-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with PTCT | |
|---|---|---|---|---|
| PTCT | 9.1% | 39.8% | 0.31 | - |
| Sector ETF (XLV) | 26.0% | 16.1% | 1.24 | 33.1% |
| Equity (SPY) | 17.6% | 13.0% | 0.98 | 22.3% |
| Gold (GLD) | 18.0% | 29.3% | 0.56 | 18.6% |
| Commodities (DBC) | 46.6% | 20.7% | 1.75 | -17.2% |
| Real Estate (VNQ) | 5.2% | 13.6% | 0.12 | 16.0% |
| Bitcoin (BTCUSD) | -26.0% | 44.9% | -0.54 | 15.1% |
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 5-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with PTCT | |
|---|---|---|---|---|
| PTCT | 10.5% | 55.1% | 0.39 | - |
| Sector ETF (XLV) | 6.8% | 15.2% | 0.26 | 29.7% |
| Equity (SPY) | 13.3% | 17.2% | 0.59 | 29.8% |
| Gold (GLD) | 18.9% | 18.8% | 0.81 | 3.9% |
| Commodities (DBC) | 10.8% | 19.6% | 0.43 | -3.2% |
| Real Estate (VNQ) | 1.0% | 18.9% | -0.05 | 27.2% |
| Bitcoin (BTCUSD) | 12.7% | 52.6% | 0.42 | 19.5% |
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Based On 10-Year Data
| Annualized Return | Annualized Volatility | Sharpe Ratio | Correlation with PTCT | |
|---|---|---|---|---|
| PTCT | 23.1% | 65.4% | 0.59 | - |
| Sector ETF (XLV) | 10.8% | 16.7% | 0.52 | 28.9% |
| Equity (SPY) | 15.4% | 17.9% | 0.73 | 28.9% |
| Gold (GLD) | 12.2% | 16.4% | 0.61 | 0.9% |
| Commodities (DBC) | 8.3% | 18.1% | 0.37 | 3.4% |
| Real Estate (VNQ) | 4.7% | 20.7% | 0.19 | 22.7% |
| Bitcoin (BTCUSD) | 64.0% | 66.2% | 1.04 | 9.7% |
Smart multi-asset allocation framework can stack odds in your favor. Learn How
Returns Analyses
Earnings Returns History
Updated 9/1/2026| Forward Returns | |||
|---|---|---|---|
| Earnings Date | 1D Returns | 5D Returns | 21D Returns |
| 7/30/2026 | -9.9% | -6.2% | -7.2% |
| 5/7/2026 | 14.3% | 12.8% | 9.9% |
| 2/19/2026 | 1.1% | -2.6% | -6.6% |
| 11/4/2025 | 6.0% | 9.0% | 14.1% |
| 8/7/2025 | -9.9% | -3.3% | 13.1% |
| 5/6/2025 | 12.6% | 28.2% | 42.9% |
| 2/27/2025 | 9.3% | 4.4% | 7.3% |
| 11/7/2024 | 3.1% | -1.4% | 11.9% |
| ... | |||
| SUMMARY STATS | |||
| # Positive | 16 | 15 | 14 |
| # Negative | 9 | 10 | 11 |
| Median Positive | 6.1% | 6.1% | 10.3% |
| Median Negative | -7.3% | -4.7% | -7.2% |
| Max Positive | 14.3% | 31.9% | 45.9% |
| Max Negative | -22.7% | -24.6% | -24.0% |
| Forward Returns | |||
|---|---|---|---|
| Earnings Date | 1D Returns | 5D Returns | 21D Returns |
| 7/30/2026 | -9.9% | -6.2% | -7.2% |
| 5/7/2026 | 14.3% | 12.8% | 9.9% |
| 2/19/2026 | 1.1% | -2.6% | -6.6% |
| 11/4/2025 | 6.0% | 9.0% | 14.1% |
| 8/7/2025 | -9.9% | -3.3% | 13.1% |
| 5/6/2025 | 12.6% | 28.2% | 42.9% |
| 2/27/2025 | 9.3% | 4.4% | 7.3% |
| 11/7/2024 | 3.1% | -1.4% | 11.9% |
| 8/8/2024 | -3.6% | 1.8% | -3.2% |
| 4/25/2024 | 12.1% | 31.9% | 45.9% |
| 2/29/2024 | 13.3% | 9.1% | 1.4% |
| 1/8/2024 | 7.5% | -2.0% | -4.0% |
| 10/26/2023 | -22.7% | -24.6% | -10.3% |
| 8/3/2023 | -4.0% | 0.8% | -0.6% |
| 4/27/2023 | 4.7% | 6.1% | -19.0% |
| 2/21/2023 | -1.1% | -2.3% | 3.6% |
| 10/27/2022 | -13.7% | -18.7% | -9.2% |
| 8/4/2022 | 8.7% | 5.6% | 5.4% |
| 5/3/2022 | 2.7% | -7.0% | -24.0% |
| 2/22/2022 | -7.3% | -10.8% | -4.2% |
| 10/28/2021 | 4.1% | 14.1% | 1.2% |
| 7/29/2021 | 0.2% | 4.3% | 10.7% |
| 5/4/2021 | 6.3% | 0.7% | 0.1% |
| 2/25/2021 | 3.5% | 4.9% | -10.8% |
| 10/29/2020 | -3.2% | 10.1% | 16.1% |
| SUMMARY STATS | |||
| # Positive | 16 | 15 | 14 |
| # Negative | 9 | 10 | 11 |
| Median Positive | 6.1% | 6.1% | 10.3% |
| Median Negative | -7.3% | -4.7% | -7.2% |
| Max Positive | 14.3% | 31.9% | 45.9% |
| Max Negative | -22.7% | -24.6% | -24.0% |
SEC Filings
Expand for More| Report Date | Filing Date | Filing |
|---|---|---|
| 06/30/2026 | 07/30/2026 | 10-Q |
| 03/31/2026 | 05/07/2026 | 10-Q |
| 12/31/2025 | 02/19/2026 | 10-K |
| 09/30/2025 | 11/04/2025 | 10-Q |
| 06/30/2025 | 08/07/2025 | 10-Q |
| 03/31/2025 | 05/06/2025 | 10-Q |
| 12/31/2024 | 02/27/2025 | 10-K |
| 09/30/2024 | 11/07/2024 | 10-Q |
| 06/30/2024 | 08/08/2024 | 10-Q |
| 03/31/2024 | 04/25/2024 | 10-Q |
| 12/31/2023 | 02/29/2024 | 10-K |
| 09/30/2023 | 10/26/2023 | 10-Q |
| 06/30/2023 | 08/03/2023 | 10-Q |
| 03/31/2023 | 04/27/2023 | 10-Q |
| 12/31/2022 | 02/21/2023 | 10-K |
| 09/30/2022 | 10/27/2022 | 10-Q |
| Report Date | Filing Date | Filing |
|---|---|---|
| 06/30/2026 | 07/30/2026 | 10-Q |
| 03/31/2026 | 05/07/2026 | 10-Q |
| 12/31/2025 | 02/19/2026 | 10-K |
| 09/30/2025 | 11/04/2025 | 10-Q |
| 06/30/2025 | 08/07/2025 | 10-Q |
| 03/31/2025 | 05/06/2025 | 10-Q |
| 12/31/2024 | 02/27/2025 | 10-K |
| 09/30/2024 | 11/07/2024 | 10-Q |
| 06/30/2024 | 08/08/2024 | 10-Q |
| 03/31/2024 | 04/25/2024 | 10-Q |
| 12/31/2023 | 02/29/2024 | 10-K |
| 09/30/2023 | 10/26/2023 | 10-Q |
| 06/30/2023 | 08/03/2023 | 10-Q |
| 03/31/2023 | 04/27/2023 | 10-Q |
| 12/31/2022 | 02/21/2023 | 10-K |
| 09/30/2022 | 10/27/2022 | 10-Q |
| 06/30/2022 | 08/04/2022 | 10-Q |
| 03/31/2022 | 05/03/2022 | 10-Q |
| 12/31/2021 | 02/22/2022 | 10-K |
| 09/30/2021 | 10/28/2021 | 10-Q |
| 06/30/2021 | 07/29/2021 | 10-Q |
| 03/31/2021 | 05/04/2021 | 10-Q |
| 12/31/2020 | 02/25/2021 | 10-K |
| 09/30/2020 | 10/29/2020 | 10-Q |
| 06/30/2020 | 08/05/2020 | 10-Q |
| 03/31/2020 | 04/30/2020 | 10-Q |
| 12/31/2019 | 03/02/2020 | 10-K |
| 09/30/2019 | 10/30/2019 | 10-Q |
Recent Forward Guidance
Updated 7/31/2026Latest: Q2 2026 Earnings Reported 7/30/2026
| Forward Guidance | Guidance Change | |||||||
|---|---|---|---|---|---|---|---|---|
| Metric | Source | Low | Mid | High | % Chg | % Delta | Change | Prior |
| 2026 Total Revenue | Reported | 1.18 Bil | 1.23 Bil | 1.28 Bil | 8.8% | Raised | Guidance: 1.13 Bil for 2026 | |
| 2026 Total Product Revenue | Reported | 850.00 Mil | 900.00 Mil | 950.00 Mil | 12.5% | Raised | Guidance: 800.00 Mil for 2026 | |
| 2026 GAAP R&D and SG&A Expense | Reported | 775.00 Mil | 795.00 Mil | 815.00 Mil | 0 | Affirmed | Guidance: 795.00 Mil for 2026 | |
| 2026 Non-GAAP R&D and SG&A Expense | Reported | 680.00 Mil | 700.00 Mil | 720.00 Mil | 0 | Affirmed | Guidance: 700.00 Mil for 2026 | |
Insider Activity
Updated 9/18/2026| # | Owner | Title | Holding | Action | Filing Date | Price | Shares | Transacted Value | Value of Held Shares | Form |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Schmertzler, Michael | See footnote | Sell | 9182026 | 67.85 | 10,000 | 678,533 | 87,978,197 | Form | |
| 2 | Schmertzler, Michael | See footnote | Sell | 9182026 | 67.12 | 10,000 | 671,195 | 87,697,886 | Form | |
| 3 | Schmertzler, Michael | See footnote | Sell | 9112026 | 67.34 | 10,000 | 673,400 | 88,659,440 | Form | |
| 4 | Schmertzler, Michael | See footnote | Sell | 9112026 | 67.84 | 10,000 | 678,403 | 89,996,583 | Form | |
| 5 | Schmertzler, Michael | See footnote | Sell | 8282026 | 71.58 | 12,500 | 894,784 | 95,677,002 | Form |
| # | Owner | Title | Holding | Action | Filing Date | Price | Shares | Transacted Value | Value of Held Shares | Form |
|---|---|---|---|---|---|---|---|---|---|---|
| 1 | Schmertzler, Michael | See footnote | Sell | 9182026 | 67.85 | 10,000 | 678,533 | 87,978,197 | Form | |
| 2 | Schmertzler, Michael | See footnote | Sell | 9182026 | 67.12 | 10,000 | 671,195 | 87,697,886 | Form | |
| 3 | Schmertzler, Michael | See footnote | Sell | 9112026 | 67.34 | 10,000 | 673,400 | 88,659,440 | Form | |
| 4 | Schmertzler, Michael | See footnote | Sell | 9112026 | 67.84 | 10,000 | 678,403 | 89,996,583 | Form | |
| 5 | Schmertzler, Michael | See footnote | Sell | 8282026 | 71.58 | 12,500 | 894,784 | 95,677,002 | Form | |
| 6 | Schmertzler, Michael | See footnote | Sell | 8282026 | 72.94 | 12,500 | 911,725 | 98,400,266 | Form | |
| 7 | Schmertzler, Michael | See footnote | Sell | 8212026 | 71.11 | 12,500 | 888,858 | 96,821,069 | Form | |
| 8 | Schmertzler, Michael | See footnote | Sell | 8212026 | 73.32 | 12,500 | 916,554 | 100,754,470 | Form | |
| 9 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 8192026 | 72.57 | 2,813 | 204,127 | 7,639,134 | Form |
| 10 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 8062026 | 68.57 | 10,292 | 705,722 | 26,321,829 | Form |
| 11 | Gravier, Pierre | CHIEF FINANCIAL OFFICER | Direct | Sell | 7162026 | 80.35 | 3,494 | 280,743 | 6,748,275 | Form |
| 12 | Smith, Mary L | Direct | Sell | 7132026 | 90.00 | 15,834 | 1,425,060 | 1,963,170 | Form | |
| 13 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 7132026 | 90.25 | 2,464 | 222,376 | 5,441,985 | Form |
| 14 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 7082026 | 88.89 | 24,613 | 2,187,745 | 5,359,723 | Form |
| 15 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 7082026 | 85.08 | 5,079 | 432,118 | 8,956,463 | Form |
| 16 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 7082026 | 86.00 | 22,161 | 1,905,736 | 5,185,416 | Form |
| 17 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 6302026 | 85.26 | 8,130 | 693,151 | 5,127,189 | Form |
| 18 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 6262026 | 85.97 | 20,960 | 1,801,857 | 5,169,764 | Form |
| 19 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 6262026 | 85.03 | 5,323 | 452,607 | 5,113,359 | Form |
| 20 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 6242026 | 80.90 | 23,749 | 1,921,378 | 4,865,296 | Form |
| 21 | Utter, Christine Marie | SVP, CHIEF ACCOUNTING OFFICER | Direct | Sell | 6222026 | 75.56 | 19,686 | 1,487,474 | 5,304,236 | Form |
| 22 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 6222026 | 78.65 | 10,462 | 822,868 | 8,279,959 | Form |
| 23 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 6122026 | 75.00 | 21,277 | 1,595,775 | 5,505,825 | Form |
| 24 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 5192026 | 72.18 | 2,812 | 202,958 | 7,593,747 | Form |
| 25 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 5152026 | 72.95 | 12,572 | 917,154 | 28,742,973 | Form |
| 26 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 5122026 | 78.97 | 3,540 | 279,570 | 8,309,079 | Form |
| 27 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 4232026 | 70.91 | 2,850 | 202,094 | 27,943,504 | Form |
| 28 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 4082026 | 69.45 | 2,265 | 157,293 | 7,306,463 | Form |
| 29 | Golden, Lee Scott | EVP & CHIEF MEDICAL OFFICER | Direct | Sell | 4062026 | 67.99 | 829 | 56,364 | 5,379,029 | Form |
| 30 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 4032026 | 69.04 | 52,003 | 3,590,243 | 4,151,807 | Form |
| 31 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 3132026 | 64.08 | 2,662 | 170,581 | 25,434,634 | Form |
| 32 | Okey, Stephanie | Direct | Sell | 3122026 | 70.00 | 15,167 | 1,061,690 | 560,000 | Form | |
| 33 | Okey, Stephanie | Direct | Sell | 3092026 | 63.38 | 6,333 | 401,386 | 507,040 | Form | |
| 34 | Golden, Lee Scott | EVP & CHIEF MEDICAL OFFICER | Direct | Sell | 3092026 | 63.38 | 10,000 | 633,800 | 5,066,851 | Form |
| 35 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Spouse | Sell | 3092026 | 63.38 | 3,989 | 252,823 | 173,471 | Form |
| 36 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 2232026 | 69.48 | 3,019 | 209,763 | 7,310,215 | Form |
| 37 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 2192026 | 69.36 | 3,081 | 213,698 | 7,506,902 | Form |
| 38 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 2192026 | 69.29 | 2,813 | 194,914 | 7,712,875 | Form |
| 39 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 1122026 | 78.70 | 1,024 | 80,589 | 6,307,097 | Form |
| 40 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 1122026 | 77.41 | 1,722 | 133,300 | 6,282,983 | Form |
| 41 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 1122026 | 77.93 | 2,314 | 180,320 | 8,674,079 | Form |
| 42 | Utter, Christine Marie | SVP, CHIEF ACCOUNTING OFFICER | Direct | Sell | 1122026 | 76.45 | 1,034 | 79,049 | 5,557,380 | Form |
| 43 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 1122026 | 76.50 | 4,602 | 352,057 | 6,340,931 | Form |
| 44 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 1122026 | 76.45 | 2,514 | 192,195 | 30,155,932 | Form |
| 45 | Golden, Lee Scott | EVP & CHIEF MEDICAL OFFICER | Direct | Sell | 1122026 | 76.45 | 866 | 66,206 | 7,066,121 | Form |
| 46 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 1122026 | 76.56 | 4,033 | 308,760 | 8,699,029 | Form |
| 47 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 1122026 | 76.45 | 881 | 67,352 | 8,806,734 | Form |
| 48 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Spouse | Sell | 1122026 | 76.45 | 59 | 4,511 | 519,172 | Form |
| 49 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 1062026 | 75.55 | 2,266 | 171,192 | 9,133,840 | Form |
| 50 | Reeve, Emma | Direct | Sell | 1062026 | 76.00 | 734 | 55,784 | 810,616 | Form | |
| 51 | Steele, Glenn JR MD Phd | Direct | Sell | 1062026 | 76.35 | 12,000 | 916,200 | 1,412,475 | Form | |
| 52 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 1022026 | 76.64 | 8,089 | 619,941 | 26,201,300 | Form |
| 53 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 12232025 | 77.26 | 20,508 | 1,584,350 | 5,436,681 | Form |
| 54 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 12192025 | 75.06 | 3,202 | 240,351 | 5,282,383 | Form |
| 55 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 12192025 | 75.43 | 40,290 | 3,038,924 | 5,307,973 | Form |
| 56 | Reeve, Emma | Direct | Sell | 12052025 | 78.62 | 733 | 57,628 | 524,081 | Form | |
| 57 | Klein, Matthew B | CHIEF EXECUTIVE OFFICER | Direct | Sell | 12032025 | 79.07 | 3,428 | 271,052 | 27,671,653 | Form |
| 58 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 11262025 | 83.44 | 71,928 | 6,001,932 | 8,396,513 | Form |
| 59 | Jacobson, Allan Steven | Direct | Sell | 11262025 | 83.55 | 14,199 | 1,186,282 | 1,457,977 | Form | |
| 60 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 11262025 | 80.18 | 35,572 | 2,852,031 | 8,067,738 | Form |
| 61 | Jacobson, Allan Steven | Direct | Sell | 11262025 | 80.14 | 11,801 | 945,736 | 1,398,529 | Form | |
| 62 | Reeve, Emma | Direct | Sell | 11252025 | 79.50 | 10,000 | 795,000 | 529,947 | Form | |
| 63 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 11192025 | 75.99 | 2,812 | 213,687 | 7,895,548 | Form |
| 64 | Reeve, Emma | Direct | Sell | 11142025 | 74.50 | 7,333 | 546,308 | 496,617 | Form | |
| 65 | Reeve, Emma | Direct | Sell | 10312025 | 69.50 | 15,666 | 1,088,787 | 463,287 | Form | |
| 66 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 10092025 | 63.43 | 3,375 | 214,091 | 6,590,900 | Form |
| 67 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 10072025 | 65.07 | 2,266 | 147,449 | 6,760,838 | Form |
| 68 | Reeve, Emma | Direct | Sell | 10072025 | 65.96 | 25,562 | 1,686,024 | 681,480 | Form | |
| 69 | Jacobson, Allan Steven | Direct | Sell | 10072025 | 65.00 | 12,000 | 780,000 | 1,134,315 | Form | |
| 70 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 10072025 | 65.82 | 22,655 | 1,491,100 | 6,838,525 | Form |
| 71 | Almstead, Neil Gregory | CHIEF TECHNICAL OPS OFFICER | Direct | Sell | 10072025 | 66.05 | 55,000 | 3,632,977 | 6,646,696 | Form |
| 72 | Reeve, Emma | Direct | Sell | 9162025 | 59.50 | 15,705 | 934,448 | 614,754 | Form | |
| 73 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 9152025 | 61.34 | 24,585 | 1,508,083 | 6,373,451 | Form |
| 74 | Boulding, Mark Elliott | EXEC. VP AND CLO | Direct | Sell | 9152025 | 61.00 | 22,664 | 1,382,504 | 6,337,961 | Form |
| 75 | Southwell, David P | Direct | Sell | 9112025 | 58.34 | 12,000 | 700,080 | 983,029 | Form | |
| 76 | Pauwels, Eric | CHIEF BUSINESS OFFICER | Direct | Sell | 9092025 | 56.92 | 39,850 | 2,268,374 | 4,150,356 | Form |
| 77 | Jacobson, Allan Steven | Direct | Sell | 9022025 | 50.15 | 1,667 | 83,600 | 875,168 | Form |
Investor Activity (13F)
Updated Sep 23, 202613F holdings as of Mar 31, 2026 (Q1 2026)
Active managers (13F portfolio over $250M, at least 3 holdings) with a position over $5M that is either over 10% of their portfolio or held in a concentrated book of 50 or fewer total positions. Index/ETF, sovereign, bank, community-bank and charitable/donor-advised filers are excluded.
Industry Resources
| Health Care Resources |
| U.S. National Library of Medicine |
| ClinicalTrials.gov |
| Modern Healthcare |
| Healthcare Dive |
| Fierce Healthcare |
| Health Affairs |
| Health Data Management |
| FDA Tracker |
| Pharmaceuticals Resources |
| Fierce Pharma |
| Pharm Exec |
| Endpoints News |
External Quote Links
| Y Finance | Barrons |
| TradingView | Morningstar |
| SeekingAlpha | ValueLine |
| Motley Fool | Robinhood |
| CNBC | Etrade |
| MarketWatch | Unusual Whales |
| YCharts | Perplexity Finance |
| FinViz |
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Core Cache Last Updated: 9/22/2026