PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results
Rhea-AI Summary
PTC Therapeutics (NASDAQ: PTCT) reported full-year 2025 product and royalty revenue of $831M, total 2025 revenues of $1.731B, and cash of $1.945B at year-end. Sephience launched in 2H 2025, generating $111M in 2025 and $92M in Q4. PTC closed a royalty sale for Evrysdi for $240M upfront and up to $60M in milestones. Full-year 2025 net income was $682.6M driven by a Novartis collaboration payment; GAAP R&D and SG&A guidance given for 2026.
Positive
- Sephience $111M total net revenue in 2025 since launch
- Full-year 2025 total revenues of $1.731B
- Year-end cash and marketable securities of $1.945B
- Royalty sale upfront proceeds of $240M from Royalty Pharma
Negative
- Q4 2025 total revenues down to $164.7M from $213.2M year-ago
- GAAP R&D expense of $455.2M for full-year 2025
- Net loss of $135.0M in Q4 2025 (quarterly volatility)
Market Reaction
Following this news, PTCT has declined 4.01%, reflecting a moderate negative market reaction. Argus tracked a trough of -6.2% from its starting point during tracking. Our momentum scanner has triggered 4 alerts so far, indicating moderate trading interest and price volatility. The stock is currently trading at $67.10. This price movement has removed approximately $234M from the company's valuation.
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Key Figures
Market Reality Check
Peers on Argus
PTCT showed a small gain of 1.26% pre-release while peers were mixed: CRSP, TGTX, and KRYS posted modest gains, MRUS declined, and RNA was flat. Moves do not clearly cluster in one direction.
Previous Earnings Reports
| Date | Event | Sentiment | Move | Catalyst |
|---|---|---|---|---|
| Nov 04 | Q3 2025 earnings | Positive | -0.3% | Reported Q3 2025 profit and initial Sephience launch metrics. |
| Aug 07 | Q2 2025 earnings | Positive | -9.9% | Sephience approvals and improved net loss with higher revenue. |
| May 06 | Q1 2025 earnings | Positive | +12.6% | Large Novartis deal drove net income and boosted cash. |
| Feb 27 | FY 2024 results | Positive | +9.3% | Strong 2024 revenue and major Novartis Huntington’s deal. |
| Nov 07 | Q3 2024 earnings | Positive | +3.1% | Guidance raised with higher DMD revenue and improved loss. |
Earnings updates have generally been received positively, but there are notable instances where strong operational news coincided with negative price reactions.
Over the past five earnings cycles, PTC reported growing revenues, major collaborations, and regulatory wins, including Sephience approvals and the Novartis Huntington’s disease deal. Several quarters, such as Q1 2025 and full-year 2024, saw substantial positive moves, while Q2 and Q3 2025 produced mild to sharp declines despite constructive updates. Today’s full-year 2025 results and guidance extend this narrative of scaling revenue, new product uptake, and a strengthened balance sheet.
Historical Comparison
In the past five earnings updates, PTCT moved about 2.95% on average, with mostly positive reactions but occasional selloffs even on strong fundamentals.
Earnings releases show a transition from heavy losses toward profitability, driven by the Novartis collaboration, growing Sephience contributions, and sustained DMD franchise and royalty revenue.
Market Pulse Summary
This announcement highlights a step-change year for PTC, with full-year 2025 revenues, net income, and cash all strengthened by Sephience uptake and the Novartis collaboration. Guidance for 2026 product revenue and operating spend frames expectations as the business shifts toward a broader commercial base. Historically, earnings events have produced mixed price reactions, so investors may track execution on Sephience growth, DMD franchise durability, and progress of late-stage programs against the outlined expense plans.
Key Terms
pku medical
fda regulatory
nda regulatory
gaap financial
r&d financial
AI-generated analysis. Not financial advice.
– Full-year 2025 product and royalty revenue of
– Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of
– Cash of
"We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven."
Key Corporate Highlights
- Full-year 2025 product and royalty revenue of
$831 million - Global launch of Sephience off to strong start
- Q4 2025 revenue of
$92 million $81 million $11 million - Sephience total net revenue of
$111 million - 946 total patients on commercial therapy worldwide as of December 31, 2025
- 1,134 patient start forms received in the US as of December 31, 2025
- Approval in
Japan in December 2025; approval inBrazil in February 2026 - Sephience global footprint expected to increase to 20 to 30 countries by end of 2026
- Q4 2025 revenue of
- In December 2025, PTC sold the remainder of its Evrysdi® (risdiplam) royalty to Royalty Pharma for
$240 million $60 million $150 million $2.5 billion - End-of-Phase 2 meeting with FDA held in Q4 2025 to discuss the votoplam Huntington's disease (HD) program
- Alignment reached on design of global Phase 3 trial, INVEST-HD, which is planned to initiate in 1H 2026
- FDA confirmed openness for potential Accelerated Approval pathway given significant unmet need
- Type C meeting with FDA held in December 2025 to discuss the vatiquinone Friedreich's ataxia program; FDA indicated that an additional study would be necessary to support NDA resubmission and meeting minutes stated that this could be an open-label study with a natural history control group
Fourth Quarter and Full Year 2025 Financial Highlights
- Total revenues were
$164.7 million $213.2 million $1,730.7 million $806.8 million $998.4 million - Total net product revenues were
$184.0 million $150.1 million $586.7 million $582.1 million - Translarna™ (ataluren) net product revenues were
$39.0 million $89.1 million $235.3 million $321.1 million - Emflaza® (deflazacort) net product revenues were
$27.1 million $50.5 million $146.4 million $207.2 million - Roche reported Evrysdi full year 2025 sales of approximately
1,757 CHF million, resulting in royalty revenue of$244.2 million $203.9 million - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$133.1 million $124.8 million $455.2 million $534.5 million - Non-GAAP R&D expenses were
$124.3 million $8.8 million $116.0 million $8.8 million $419.6 million $35.7 million $497.9 million $36.6 million - GAAP SG&A expenses were
$96.9 million $84.7 million $347.1 million $300.9 million - Non-GAAP SG&A expenses were
$87.2 million $9.7 million $76.3 million $8.4 million $308.3 million $38.9 million $262.9 million $38.0 million - Net loss was
$135.0 million $65.9 million $682.6 million $363.3 million - Cash, cash equivalents, and marketable securities were
$1,945.4 million $1,139.7 million - Shares issued and outstanding as of December 31, 2025, were 81,474,366.
Full Year 2026 Financial Guidance
- Total product revenue of
$700 $800 million 36% increase from 2025, with the majority from Sephience - GAAP R&D and SG&A expense of
$775 $815 million - Non-GAAP R&D and SG&A expense of
$680 $720 million $95 million
Non-GAAP Financial Measures
In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.
PTC Therapeutics, Inc. Consolidated Statements of Operations (In thousands, except share and per share data) | ||||||||||||
Three Months Ended | Twelve Months Ended | |||||||||||
2025 | 2024 | 2025 | 2024 | |||||||||
Revenues: | ||||||||||||
Net product revenue | $ | 183,992 | $ | 150,088 | $ | 586,703 | $ | 582,145 | ||||
Collaboration and license revenue | (73) | 304 | 998,357 | 304 | ||||||||
Royalty revenue | 79,387 | 58,162 | 244,224 | 203,864 | ||||||||
Translarna France* | (98,629) | 4,618 | (98,629) | 18,806 | ||||||||
Manufacturing revenue | - | - | - | 1,661 | ||||||||
Total revenues | 164,677 | 213,172 | 1,730,655 | 806,780 | ||||||||
Operating expenses: | ||||||||||||
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets | 6,950 | 16,283 | 47,013 | 57,398 | ||||||||
Amortization of acquired intangible assets | 9,349 | 3,307 | 24,742 | 60,738 | ||||||||
Research and development (1) | 133,128 | 124,770 | 455,249 | 534,480 | ||||||||
Selling, general and administrative (2) | 96,874 | 84,683 | 347,143 | 300,911 | ||||||||
Change in the fair value of contingent consideration | - | (10,175) | (800) | (4,475) | ||||||||
Intangible asset impairment | - | 159,548 | - | 159,548 | ||||||||
Tangible asset impairment and (gains) losses on transactions, net | (10,275) | (2,855) | (9,627) | 750 | ||||||||
Total operating expenses | 236,026 | 375,561 | 863,720 | 1,109,350 | ||||||||
(Loss) income from operations | (71,349) | (162,389) | 866,935 | (302,570) | ||||||||
Interest expense, net | (55,188) | (41,060) | (152,230) | (166,993) | ||||||||
Other (expense) income, net | (12,546) | 8,850 | (18,086) | 6,544 | ||||||||
Gain on sale of priority review voucher | - | 99,900 | - | 99,900 | ||||||||
(Loss) income before income tax benefit (expense) | (139,083) | (94,699) | 696,619 | (363,119) | ||||||||
Income tax benefit (expense) | 4,118 | 28,813 | (13,975) | (176) | ||||||||
Net (loss) income attributable to common stockholders | $ | (134,965) | $ | (65,886) | $ | 682,644 | $ | (363,295) | ||||
Weighted-average shares outstanding: | ||||||||||||
Basic (in shares) | 80,689,810 | 77,201,783 | 79,534,290 | 76,845,055 | ||||||||
Diluted (in shares) | 80,689,810 | 77,201,783 | 88,311,494 | 76,845,055 | ||||||||
Net (loss) income per share—basic (in dollars per share) | $ | (1.67) | $ | (0.85) | $ | 8.58 | $ | (4.73) | ||||
Net (loss) income per share—diluted (in dollars per share) | $ | (1.67) | $ | (0.85) | $ | 7.78 | $ | (4.73) | ||||
(1) Research and development reconciliation | ||||||||||||
GAAP research and development | $ | 133,128 | $ | 124,770 | $ | 455,249 | $ | 534,480 | ||||
Less: share-based compensation expense | 8,843 | 8,818 | 35,669 | 36,629 | ||||||||
Non-GAAP research and development | $ | 124,285 | $ | 115,952 | $ | 419,580 | $ | 497,851 | ||||
(2) Selling, general and administrative reconciliation | ||||||||||||
GAAP selling, general and administrative | $ | 96,874 | $ | 84,683 | $ | 347,143 | $ | 300,911 | ||||
Less: share-based compensation expense | 9,675 | 8,420 | 38,878 | 37,986 | ||||||||
Non-GAAP selling, general and administrative | $ | 87,199 | $ | 76,263 | $ | 308,265 | $ | 262,925 | ||||
*In the fourth quarter of 2025, PTC changed its estimates for its sales allowance related to Translarna revenues in | ||||||||||||
PTC Therapeutics, Inc.
| |||||
December 31, 2025 | December 31, 2024 | ||||
Cash, cash equivalents and marketable securities | $ | 1,945,371 | $ | 1,139,696 | |
Total assets | $ | 2,898,767 | $ | 1,705,024 | |
Total debt | $ | 286,631 | $ | 285,412 | |
Total deferred revenue | 2,040 | 5,505 | |||
Total liability for sale of future royalties | 2,308,366 | 2,081,776 | |||
Total liabilities | $ | 3,104,080 | $ | 2,803,095 | |
Total stockholders' deficit (81,474,366 and 77,704,188 common shares issued | $ | (205,313) | $ | (1,098,071) | |
Total liabilities and stockholders' deficit | $ | 2,898,767 | $ | 1,705,024 | |
PTC Therapeutics, Inc. Reconciliation of GAAP to Non-GAAP Projected Full Year 2026 R&D and SG&A Expense
| |||||
Low End of Range | High End of Range | ||||
Projected GAAP R&D and SG&A Expense | $ | 775 | $ | 815 | |
Less: projected non-cash, stock-based compensation expense | 95 | 95 | |||
Projected non-GAAP R&D and SG&A expense | $ | 680 | $ | 720 | |
Acronyms
CHF: Confoederatio Helvetica Francs (Swiss francs)
DMD: Duchenne muscular dystrophy
FA: Friedreich's ataxia
FDA:
GAAP: Generally Accepted Accounting Principles
HD: Huntington's disease
NDA: New Drug Application
nmDMD: Nonsense mutation Duchenne muscular dystrophy
PKU: Phenylketonuria
R&D: Research and Development
SG&A: Selling, General, and Administrative
Today's Conference Call and Webcast Reminder
To access the call by phone, please click here to register and you will be provided with dial-in details. The webcast conference call can be accessed on the Investors section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call will be available after completion of the call and will be archived on the company's website.
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit www.ptcbio.com and follow on LinkedIn, X, Instagram and Facebook.
For more Information
Investors
Ellen Cavaleri
+1 (615) 618-6228
ecavaleri@ptcbio.com
Media
Jeanine Clemente
+1 (908) 912-9406
jclemente@ptcbio.com
Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "Full Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions.
PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the U.S. on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra.
The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.
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SOURCE PTC Therapeutics, Inc.
FAQ
How much revenue did PTC Therapeutics (PTCT) report for full-year 2025?
What were Sephience sales for PTCT in 2025 and Q4 2025?
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What material transaction did PTCT complete related to Evrysdi royalties?
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