At a glance
PTC Therapeutics Inc is worth $5.26B, about 8 times its fiscal 2025 earnings. Revenue grew 114.5% to $1.73B in fiscal 2025, with 39.4% kept as profit; over 5 years it has grown 35.4% a year. It pays no dividend. The stock returned −6.6% over the past year with dividends reinvested and +11.6% a year over five years, against +11.5% and +11.7% for the S&P 500. At its worst it fell 45.5% in the 2020 crash (S&P 500: 33.7%) and 43.4% in the 2022 bear market (S&P 500: 24.5%), and was back at its earlier high 8 months and 2 months after the lows.
The business in plain English
PTC Therapeutics discovers, develops and sells medicines for rare diseases in children and adults, mainly in neurology and metabolism. Sephience (sepiapterin) treats phenylketonuria, or PKU, an inherited disorder in which the body cannot break down an amino acid called phenylalanine. Left untreated, PKU can cause permanent intellectual disability. Sephience won approval in Europe and the US in mid-2025, Japan in December 2025 and Brazil in February 2026. An estimated 58,000 people live with PKU worldwide.
Its other medicines include Translarna and Emflaza for Duchenne muscular dystrophy, a muscle-wasting disease that mainly affects boys. It also sells the gene therapy Upstaza, called Kebilidi in the US, for AADC deficiency, a rare nervous system disorder. PTC holds the rights to sell two Ionis medicines, Tegsedi and Waylivra, in Latin America and the Caribbean. Evrysdi, a spinal muscular atrophy treatment, came out of its collaboration with Roche.
In the year ended December 31, 2025, most of PTC's $1.73 billion in revenue came from partners. Collaboration and license payments made up 57.7%, and royalties (a cut of partners' sales) made up 14.1%. For example, Novartis licensed the Huntington's disease candidate votoplam in November 2024. Product sales made up the rest: Translarna 13.6%, Emflaza 8.5%, Upstaza and Kebilidi 3.3% and other products 2.2%. A significant share of product sales comes from outside the US.
Price history
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PTCT -6.6% over the year to Oct 28, 2026, total return with dividends reinvested.
How it makes money
By product or service
| Collaboration And License Revenue | $998.4M | 57.7% |
| Royalty | $244.2M | 14.1% |
| Translarna1 | $235.3M | 13.6% |
| Emflaza | $146.4M | 8.5% |
| Upstaza And Kebilidi | $56.6M | 3.3% |
| Other Products | $37.3M | 2.2% |
Latest results
- In Q3 FY2026 (ended Sep 30, 2026), revenue was $357.8M, up 69.6% from a year earlier, and net income was $-51.7M (-425.2%).
- For fiscal 2025, revenue was $1.73B (+114.5% year over year; +35.4% a year from FY2020 to FY2025) with a 39.4% net margin.
Financial health check
| Revenue growth a year (FY2020–FY2025) | +35.4% good |
|---|---|
| Operating margin | 50.1% good |
| Free cash flow margin | 40.6% good |
| Return on assets (FY2025) | 23.5% good |
| Net cash (cash minus debt) | $1.59B good |
| Fiscal year | Revenue | Net income | Free cash flow |
|---|---|---|---|
| FY2020 | $380.8M | $-438.2M | $-211.9M |
| FY2021 | $538.6M | $-523.9M | $-279.5M |
| FY2022 | $698.8M | $-559.0M | $-388.7M |
| FY2023 | $937.8M | $-626.6M | $-186.9M |
| FY2024 | $806.8M | $-363.3M | $-114.2M |
| FY2025 | $1.73B | $682.6M | $702.3M |
Valuation snapshot
| Market value | $5.26B |
|---|---|
| P/E ratio | 8.1 |
| Dividend yield | None |
Rank among 1514 US companies
Market value at each year end
PTC Therapeutics’s market value went from $374.4M at the end of 2016 to $5.26B on Oct 28, 2026, about 14.1 times as much; since the end of 2025 it is down 14%.
Show as a table
| Year end | Market value | Change |
|---|---|---|
| Oct 28, 2026 | $5.26B | -13.7% |
| 2025 | $6.10B | +75.2% |
| 2024 | $3.48B | +67.4% |
| 2023 | $2.08B | -24.2% |
| 2022 | $2.74B | -2.6% |
| 2021 | $2.82B | -32.4% |
| 2020 | $4.16B | +40.7% |
| 2019 | $2.96B | +70.9% |
| 2018 | $1.73B | +150.2% |
| 2017 | $692.0M | +84.8% |
| 2016 | $374.4M |
Price behavior
| YTD | 1 yr | 3 yr | 5 yr | 10 yr | |
|---|---|---|---|---|---|
| PTCT price data to Oct 28, 2026 | -17.0% | -6.6% | +50.4% | +11.6% | +17.3% |
Show as a table
| Year | Price data |
|---|---|
| 2026 (to date) | -17.0% |
| 2025 | +68.3% |
| 2024 | +63.8% |
| 2023 | -27.8% |
| 2022 | -4.2% |
| 2021 | -34.7% |
| 2020 | +27.1% |
| 2019 | +40.0% |
| 2018 | +105.8% |
| 2017 | +52.9% |
| 2016 | -83.2% |
| 2015 | -37.4% |
| 2014 | +205.1% |
| 2008 financial crisis stocks fell Oct 9, 2007 – Mar 9, 2009 | Before this security existed |
|---|---|
| 2020 COVID crash stocks fell Feb 19, 2020 – Mar 23, 2020 | Lost 31.3% over that stretch · deepest drop along the way 45.5%, back to its old high 7.7 months after the low |
| 2022 bear market stocks fell Jan 3, 2022 – Oct 12, 2022 | Gained 20.3% over that stretch · deepest drop along the way 43.4%, back to its old high 1.6 months after the low |
Insider activity
Form 4 filings from 2025-10-30 to 2026-10-30 (25 read): 0 open-market purchases and 71 sales ($24.8M), 71 of them under pre-arranged 10b5-1 trading plans.
| Date | Insider | Type | Value |
|---|---|---|---|
| Jun 17, 2026 | Christine Marie Utter SVP, CHIEF ACCOUNTING OFFICER | sell (plan) | $1.5M |
| Jun 10, 2026 | Eric Pauwels CHIEF BUSINESS OFFICER | sell (plan) | $1.3M |
| Jul 7, 2026 | Neil Gregory Almstead CHIEF TECHNICAL OPS OFFICER | sell (plan) | $1.0M |
| Jun 25, 2026 | Neil Gregory Almstead CHIEF TECHNICAL OPS OFFICER | sell (plan) | $981K |
| Jun 22, 2026 | Neil Gregory Almstead CHIEF TECHNICAL OPS OFFICER | sell (plan) | $978K |
Who owns it
Large shareholders (13G filings)
| BlackRock, Inc. | 14.8% | Nov 30, 2025 |
| JANUS HENDERSON GROUP PLC | 8.7% | Mar 31, 2026 |
| Vanguard Portfolio Management | 5.88% | Mar 31, 2026 |
| STATE STREET CORPORATION | 5.2% | Dec 31, 2025 |
| Vanguard Capital Management | 5.17% | Mar 31, 2026 |
| Wellington Management Group LLP | 4.8% | Dec 31, 2025 |
| Camber Capital Management LP | 4.15% | Dec 31, 2024 |
| Armistice Capital, LLC | 4.08% | Sep 30, 2025 |
| The Toronto-Dominion Bank | 3.7% | Mar 31, 2026 |
ETFs with the most weight in PTCT
| IBRN iShares Neuroscience and Healthcare ETF | 4.24% |
| SBIO ALPS Medical Breakthroughs ETF | 3.37% |
| PBE Invesco Biotechnology & Genome ETF | 2.55% |
| HAPS Harbor Human Capital Factor US Small Cap ETF | 1.52% |
| MNTL Tema Neuroscience and Mental Health ETF | 1.4% |
| MMSC First Trust Multi-Manager Small Cap Opportunities ETF | 0.54% |
| BRNY Burney U.S. Factor Rotation ETF | 0.5% |
| QQQS Invesco NASDAQ Future Gen 200 ETF | 0.49% |
What could go wrong
- Translarna's European sales depend on each country. In March 2025, the European Commission declined to renew Translarna's conditional approval, and its US application was withdrawn. European sales depend on individual countries allowing continued use. Translarna supplied 13.6% of 2025 revenue.
- Most revenue depends on partners. Collaboration and license payments (57.7%) and royalties (14.1%) made up most of 2025 revenue. Novartis leads votoplam's development. If partnerships fall short, or large license payments do not recur, revenue could drop sharply.
- Vatiquinone setback shows regulatory risk. In August 2025, the FDA said vatiquinone for Friedreich's ataxia lacked substantial evidence that it works and asked for another study. Delays like this add costs and push back possible new revenue.
- Sephience launch needs prices, coverage and patients. Sephience was first approved for PKU in 2025 and serves a small market of an estimated 58,000 people worldwide. Sales depend on winning prices and insurance coverage country by country and on finding eligible patients.
- Hard-to-make products and outside manufacturers. Some products and candidates may be difficult to produce, and PTC depends on outside manufacturers for supply. Problems could delay approvals or interrupt sales. PTC must also complete further FDA requirements for Kebilidi after its approval.
- Overseas sales and drug-pricing pressure. A significant share of product sales occurs outside the US, which brings foreign rules, sanctions and export limits into play. Government price controls, reimbursement limits or healthcare reform could also cut what PTC earns per patient.
Recent events
- Quarterly results released; Investor presentation or announcement
- Investor presentation or announcement; Other company announcement
- Major agreement signed
- Investor presentation or announcement; Other company announcement
- Quarterly results released; Executive or board change; Investor presentation or announcement
- Major agreement signed; New debt or financial obligation; Unregistered sale of shares; Other company announcement
- Other company announcement
- Executive or board change
- Shareholder vote results
- Quarterly results released; Investor presentation or announcement
For income and retirement investors
PTC Therapeutics pays no dividend and bought back no stock from fiscal 2021 through fiscal 2025. All returns come from the share price, so an investor who needs income would have to sell shares to raise cash. The share count rose 18.1%, from about 70.7 million on October 27, 2021, to 83.5 million on July 29, 2026, which spreads ownership more thinly. After losses every year from 2020 through 2024, PTC earned $682.6 million in 2025 and produced $702.3 million of free cash flow (cash left after running and investing in the business). None of it went back to shareholders.
The shares swing hard. They fell 45.5% in the 2020 COVID crash, from February 19 to March 16, 2020, and recovered by November 4, 2020. Over the 2022 bear market they gained 20.3% overall, but they dropped 43.4% between April and June 2022 along the way. Single stocks like this carry more risk than diversified funds.
Questions investors ask
How does PTC Therapeutics make money?
Mostly through partners. In the year ended December 31, 2025, collaboration and license revenue made up 57.7% of PTC's $1.73 billion in revenue, and royalties made up another 14.1%. The rest came from its own product sales. Translarna (13.6%) and Emflaza (8.5%) treat Duchenne muscular dystrophy. The gene therapy Upstaza/Kebilidi added 3.3% and other products 2.2%. Its PKU drug, Sephience, was approved in Europe, the US and Japan in 2025.
Does PTC Therapeutics pay a dividend?
No. PTC Therapeutics paid no dividends and made no share buybacks from fiscal 2021 through fiscal 2025. Instead, its share count rose 18.1% between October 2021 and July 2026, from about 70.7 million to 83.5 million. Returns depend entirely on the share price, so the stock may not suit investors who need regular income.
Is PTC Therapeutics profitable?
Yes, for fiscal 2025. After losses every year from 2020 through 2024, PTC earned $682.6 million on $1.73 billion of revenue in 2025, or $7.78 per diluted share. It also earned $83.5 million in the quarter ended June 30, 2026. Collaboration and license payments made up 57.7% of 2025 revenue, and such payments can be uneven. The stock's price-to-earnings ratio was about 8.1, based on 2025 earnings, as of October 28, 2026.
What happened to Translarna in Europe?
In March 2025, the European Commission declined to renew Translarna's conditional approval for nonsense mutation Duchenne muscular dystrophy. It followed a negative opinion from the European Medicines Agency's drug committee. The Commission said individual EU countries can use provisions of EU law to allow continued commercial use, so sales depend on decisions country by country. Translarna still brought in $235.3 million, or 13.6% of revenue, in 2025. PTC's US application for Translarna was withdrawn.
What drugs is PTC Therapeutics developing?
Its two most advanced candidates are votoplam and vatiquinone. In May 2025, PTC said votoplam, for Huntington's disease, met its Phase 2 (mid-stage) goals: it lowered a disease-linked protein called HTT in the blood and was safe. Partner Novartis leads its development. PTC's 10-K, filed February 19, 2026, said it expected Novartis to start a Phase 3 (late-stage) trial in the first half of 2026. The FDA turned down vatiquinone, for Friedreich's ataxia, in August 2025 and asked for another study.