PTC Therapeutics posts Q2 profit, lifts 2026 outlook
PTC Therapeutics reported strong second-quarter 2026 results, with total revenue of $361 million, including $239 million of product revenue.
Rhea-AI Filing Summary
PTC Therapeutics reported strong second-quarter 2026 results, with total revenue of $361 million, including $239 million of product revenue. Sephience generated $151 million of Q2 2026 revenue and had 1,647 patients on commercial therapy worldwide as of June 30, 2026. Total net product and royalty revenue was $309.9 million versus $175.9 million a year earlier, and net income attributable to common stockholders was $83.5 million, compared with a $64.8 million loss in Q2 2025; diluted EPS was $0.92.
The company raised full-year 2026 guidance, targeting total revenue of $1.18 to $1.28 billion and product revenue of $850 to $950 million. Projected 2026 GAAP R&D and SG&A expense is $775 to $815 million, with non-GAAP expected at $680 to $720 million. Cash and marketable securities were about $2.2 billion as of June 30, 2026, with total debt of $590.97 million and a $2.32 billion liability for sale of future royalties.
Pipeline updates included positive 24‑month PIVOT‑HD data for votoplam showing 52% dose‑dependent disease slowing on the cUHDRS at the 10 mg dose in Stage 2 participants, and a Novartis-funded Phase 3 INVEST‑HD trial (~770 patients) that triggered a $50 million milestone payment. PTC named PTC303 as its next MSH3 splicing candidate, initiated Phase 1 for PTC612 and plans a Phase 2a study of PTC844 in Q3 2026. The company also appointed Hege Sollie‑Zetlmayer to its board as a Class III director, granting equity awards under its director compensation policy.
Positive
- Return to profitability and strong revenue growth: Q2 2026 net income was $83.5 million versus a $64.8 million loss in Q2 2025, with total net product and royalty revenue rising to $309.9 million from $175.9 million.
- Raised 2026 revenue guidance: full-year 2026 total revenue outlook increased to $1.18 to $1.28 billion, with product revenue guidance lifted to $850 to $950 million, signaling higher expected commercial performance.
- Robust cash position: cash, cash equivalents and marketable securities totaled approximately $2.2 billion as of June 30, 2026, providing substantial financial flexibility alongside expanding revenues and profitability.
Negative
- None.
8-K Event Classification
Key Figures
Key Terms
Composite Unified Huntington's Disease Rating Scale medical
New Drug Application regulatory
non-GAAP financial
sale of future royalties financial
NLRP3 inhibitor medical
Offering Details
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
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FORM
CURRENT REPORT
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Item 2.02. Results of Operations and Financial Condition.
On July 30, 2026, PTC Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended June 30, 2026. The full text of the press release issued in connection with the announcement is furnished as Exhibit 99.1 to this Current Report on Form 8-K (this “Report”) and is incorporated by reference into this Item 2.02.
Item 5.02. Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers.
On July 28, 2026, the Company appointed Hege Sollie-Zetlmayer to its board of directors (the “Board”), effective immediately, filling a vacancy on the Board. Ms. Sollie-Zetlmayer will serve as a Class III director with a term expiring at the annual meeting of stockholders to be held in 2028.
Ms. Sollie-Zetlmayer has 30 years of experience in the biopharmaceutical and medical device industry, including the last eight with PTC Therapeutics, Inc. Ms. Sollie-Zetlmayer joined the Company in June 2018 as VP Human Resources Europe, Asia Pacific, and became VP Global Talent Management & Global HR Business Partners in November 2018, SVP, Global Human Resources in November 2020 and Chief Human Resources Officer from November 2023, a role she held until her retirement in June 2026. She serves as a director of PTC Therapeutics International Limited, our international headquarters and indirect wholly owned subsidiary, and previously served as a director of PTC Therapeutics Switzerland GmbH, our indirect wholly owned subsidiary. Ms. Sollie-Zetlmayer founded and manages HSZ Consulting GmbH, an executive advisory company, and previously served as the first female chairman of the Norwegian Medical Device Association. Ms. Sollie-Zetlmayer studied Human Resources at the Norwegian School of Business BI in Oslo and holds a certificate from Wharton Executive Education in Corporate Governance.
In connection with Ms. Sollie-Zetlmayer’s appointment to the Board, she will be entitled to compensation in accordance with the Company’s outside director compensation policy, under which each non-employee director receives a base annual retainer of $50,000 per year for service as a Board member. The Company will also reimburse Ms. Sollie-Zetlmayer for reasonable travel and other expenses incurred in connection with attending meetings of the Board and any committee on which she serves.
In addition, in accordance with the Company's outside director equity compensation policy, the Company granted Ms. Sollie-Zetlmayer, pursuant to the Company’s Amended and Restated 2013 Long-Term Incentive Plan, (i) an “initial director equity grant” consisting of 3,475 stock options to purchase shares of the Company’s common stock, which vest equally on a monthly basis over 36 months beginning on the one month anniversary of the grant date and 4,000 restricted stock units, which shares underlying such restricted stock units vest in three equal annual installments commencing on July 28, 2027, and (ii) an “annual director equity grant”, pro-rated for the remainder of 2026, consisting of 1,738 stock options to purchase shares of the Company’s common stock, which vest in six equal monthly installments commencing on August 2, 2026, and 2,000 restricted stock units, with the shares underlying such restricted stock units vesting on January 2, 2027. Beginning in 2027, Ms. Sollie-Zetlmayer will be eligible for equity award grants on the same terms as other continuing members of the Board.
There is no arrangement or understanding between Ms. Sollie-Zetlmayer and any other persons pursuant to which Ms. Sollie-Zetlmayer was elected as a director. In addition, Ms. Sollie-Zetlmayer is not a party to any transaction, or series of transactions, required to be disclosed pursuant to Item 404(a) of Regulation S-K.
Item 7.01. Regulation FD Disclosure.
The Company will host a conference call on July 30, 2026 at 4:30 p.m. eastern time, as previously announced. During this call the Company expects to review financial results for the quarter ended June 30, 2026, as well as other corporate highlights and updates. Instructions on how to access the conference call are included in the press release furnished as Exhibit 99.1 hereto.
The information in this Report (including Items 2.02 and 7.01 and Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.
Item 9.01. Financial Statements and Exhibits.
(d) Exhibits
Exhibit No. | | Description |
99.1 | Press Release, dated July 30, 2026 issued by PTC Therapeutics, Inc. | |
104 | The cover page from this Current Report on Form 8-K, formatted in Inline XBRL |
Signature
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.
PTC Therapeutics, Inc. | ||
Date: July 30, 2026 | By: | /s/ Pierre Gravier |
Name: | Pierre Gravier | |
Title: | Chief Financial Officer | |
Exhibit 99.1
PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results
– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue –
– Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million –
– Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion –
– Strong cash position of $2.2 billion as of June 30, 2026 –
WARREN, N.J., July 30, 2026 – PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026.
“Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. “We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington’s disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule.”
Key Corporate Updates
Second Quarter 2026 Financial Highlights
| ● | Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. |
| ● | Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. |
| ● | Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. |
| ● | Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. |
| ● | Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. |
| ● | In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. |
| ● | Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. |
| ● | Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. |
| ● | GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. |
| ● | Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. |
| ● | Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. |
| ● | In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock’s closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. |
| ● | Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. |
| ● | Shares issued and outstanding as of June 30, 2026, were 83,327,286. |
PTC Updates Full-Year 2026 Financial Guidance
| ● | Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million |
| ● | GAAP R&D and SG&A expense guidance remains $775 to $815 million |
| ● | Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $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 June 30, | | Six Months Ended June 30, | ||||||||||||||||
| 2026 | | 2025 | | 2026 | | 2025 | ||||||||||||
Revenues: | | | | | | | | ||||||||||||
Net product revenue | $ | 238,819 | | $ | 118,329 | | $ | 464,392 | | $ | 271,755 | ||||||||
Collaboration and license revenue | | 50,595 | | 2,941 | | 50,738 | | 989,172 | |||||||||||
Royalty revenue | | 71,105 | | 57,605 | | 117,940 | | 94,044 | |||||||||||
Total revenues | 360,519 | | 178,875 | | 633,070 | 1,354,971 | |||||||||||||
Operating expenses: | | | | | | | |||||||||||||
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets | 19,921 | | 11,420 | | 47,949 | 24,282 | |||||||||||||
Amortization of acquired intangible assets | 11,841 | | 4,061 | | 23,422 | 7,859 | |||||||||||||
Research and development (1) | 99,150 | | 112,990 | | 200,023 | 221,963 | |||||||||||||
Selling, general and administrative (2) | 80,630 | | 85,262 | | 166,813 | 166,223 | |||||||||||||
Change in the fair value of contingent consideration | - | | - | | - | (800) | |||||||||||||
Tangible asset impairment and losses on transactions, net | | - | | | 99 | | | 927 | | | 176 | ||||||||
Total operating expenses | 211,542 | | 213,832 | | 439,134 | | 419,703 | ||||||||||||
Income (loss) from operations | 148,977 | | (34,957) | | 193,936 | | 935,268 | ||||||||||||
Interest expense, net | (48,481) | | (30,358) | | (97,511) | | (64,450) | ||||||||||||
Other expense, net | (2,951) | | (5,737) | | (1,342) | | (12,042) | ||||||||||||
Income (loss) before income tax (expense) benefit | 97,545 | | (71,052) | | 95,083 | | 858,776 | ||||||||||||
Income tax (expense) benefit | (14,049) | | 6,203 | | (14,396) | | (57,063) | ||||||||||||
Net income (loss) attributable to common stockholders | $ | 83,496 | | $ | (64,849) | | $ | 80,687 | | $ | 801,713 | ||||||||
| | | | | | | | ||||||||||||
Weighted-average shares outstanding: | | | | | | | | ||||||||||||
Basic (in shares) | 83,006,808 | | 78,151,240 | | 82,765,248 | | 78,438,830 | ||||||||||||
Diluted (in shares) | 92,020,009 | 78,151,240 | | 91,824,273 | | | 86,502,578 | ||||||||||||
Net income (loss) per share—basic (in dollars per share) | $ | 1.01 | | $ | (0.83) | | $ | 0.97 | | $ | 10.22 | ||||||||
Net income (loss) per share—diluted (in dollars per share) | $ | 0.92 | | $ | (0.83) | | $ | 0.90 | | $ | 9.29 | ||||||||
| | | | | | | | ||||||||||||
(1) Research and development reconciliation | | | | | | | | ||||||||||||
GAAP research and development | $ | 99,150 | | $ | 112,990 | | $ | 200,023 | | $ | 221,963 | ||||||||
Less: share-based compensation expense | 10,547 | | 9,030 | | 21,677 | | 17,693 | ||||||||||||
Non-GAAP research and development | $ | 88,603 | | $ | 103,960 | | $ | 178,346 | | $ | 204,270 | ||||||||
| | | | | | | | ||||||||||||
(2) Selling, general and administrative reconciliation | | | | | | | | ||||||||||||
GAAP selling, general and administrative | $ | 80,630 | | $ | 85,262 | | $ | 166,813 | | $ | 166,223 | ||||||||
Less: share-based compensation expense | 12,744 | | 9,513 | | 25,035 | | 18,910 | ||||||||||||
Non-GAAP selling, general and administrative | $ | 67,886 | | $ | 75,749 | | $ | 141,778 | | $ | 147,313 | ||||||||
| | | | | | | | ||||||||||||
PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(in thousands, except share data)
| June 30, 2026 | | December 31, 2025 | |||||||
Cash, cash equivalents and marketable securities | $ | 2,229,082 | | $ | 1,945,371 | |||||
Total Assets | $ | 3,250,477 | | $ | 2,898,767 | |||||
| | | | | | |||||
Total debt | $ | 590,970 | | $ | 286,631 | |||||
Total deferred revenue | | 1,192 | | | 2,040 | |||||
Total liability for sale of future royalties | | 2,321,434 | | | 2,308,366 | |||||
Total liabilities | $ | 3,415,840 | | $ | 3,104,080 | |||||
| | | | | | |||||
Total stockholders' deficit (83,327,286 and 81,474,366 common shares issued and outstanding at June 30, 2026, and December 31, 2025, respectively) | $ | (165,363) | | $ | (205,313) | |||||
Total liabilities and stockholders' deficit | $ | 3,250,477 | | $ | 2,898,767 | |||||
| | | | | | |||||
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2026 R&D and SG&A Expense
(in millions)
| 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)
cUHDRS: Composite Unified Huntington's Disease Rating Scale
DMD: Duchenne muscular dystrophy
FA: Friedreich’s ataxia
FDA: US Food and Drug Administration
GAAP: Generally Accepted Accounting Principles
HD: Huntington’s disease
NDA: New Drug Application
nmDMD: Nonsense mutation Duchenne muscular dystrophy
R&D: Research and Development
SG&A: Selling, General, and Administrative
Today's Conference Call and Webcast Reminder
To access the live webcast, please visit the “Events & Presentations” page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details.
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, Facebook and Instagram.
For more information please contact:
Investors:
Ellen Cavaleri
+1 (615) 618-8228
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 "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total 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 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 US 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; expectations regarding PTC’s product candidates, including the timing of clinical trials and studies; 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.