PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results
Rhea-AI Summary
PTC Therapeutics (NASDAQ: PTCT) reported Q2 2026 total revenue of $360.5 million, including $238.8 million in net product revenue and $71.1 million in Evrysdi royalties. Sephience™ generated $151.3 million with 1,647 patients on commercial therapy worldwide. The company recognized a $50 million milestone from Novartis for first dosing in the Phase 3 INVEST-HD study and posted GAAP net income of $83.5 million versus a prior-year loss. Cash, cash equivalents and marketable securities reached $2.23 billion, supported by a $550 million 0% senior convertible notes issue due 2031, alongside repurchase of most 1.5% notes due 2026.
PTC raised 2026 expected total revenue to $1.18–$1.28 billion, increasing product revenue guidance to $850–$950 million while maintaining GAAP and non-GAAP R&D and SG&A ranges. Pipeline updates included positive long-term votoplam data in Huntington’s disease, initiation of Phase 1 PTC612, expected Phase 2a start for PTC844 in Q3 2026, and naming PTC303 as a development candidate.
Positive
- Total Q2 2026 revenue $360.5M, up from $178.9M in Q2 2025
- Net product revenue $238.8M in Q2 2026, more than 100% YoY increase
- Q2 2026 net income $83.5M versus $64.8M net loss in Q2 2025
- 2026 total revenue guidance raised to $1.18–$1.28B; product $850–$950M
- Cash and marketable securities $2.23B as of June 30, 2026
- $50M Novartis milestone recognized from INVEST-HD Phase 3 initiation
Negative
- Translarna Q2 2026 revenue $42.2M, down from $59.5M in Q2 2025
- Emflaza Q2 2026 revenue $24.6M, down from $36.4M due to generic erosion
- Total debt $591.0M at June 30, 2026, up from $286.6M at year-end 2025
- Interest expense $48.5M in Q2 2026, up from $30.4M in Q2 2025
- Stockholders’ deficit $165.4M as of June 30, 2026
News Explained
As of June 30, PTC reported $2,229,082 of liquidity against $3,415,840 of liabilities and a $165,363 stockholders’ deficit.
PTC Therapeutics has reported results for the quarter ended
The June financing is reported as
The next specified regulatory checkpoint is a planned FDA discussion in the second half of 2026 concerning the finalized 24-month PIVOT-HD results.
Key Figures
Previous Earnings Reports
| Date | Event | Sentiment | 24h Move | Catalyst |
|---|---|---|---|---|
| May 07 | Q1 2026 earnings | Positive | +14.3% | Revenue growth, raised guidance, and positive PIVOT-HD clinical update |
| Feb 19 | FY2025 earnings | Positive | +1.1% | Annual revenue, Sephience launch, royalty transaction, and collaboration-driven net income |
| Nov 04 | Q3 2025 earnings | Positive | +6.0% | Sephience launch, positive net income, and updated revenue guidance |
| Aug 07 | Q2 2025 earnings | Positive | -9.9% | Sephience approvals, PIVOT-HD results, and updated revenue guidance |
| May 06 | Q1 2025 earnings | Positive | +12.6% | Collaboration agreement, net income, and positive regulatory progress |
24h Move is the share-price change in the day after each event; other market factors may also have contributed.
Four of five tag-specific earnings events had positive 24-hour reactions; Q2 2025 was the sole divergence.
Key Terms
nda regulatory
gaap financial
AI-generated analysis. How Rhea-AI works. Not financial advice.
– Robust Q2 performance with total revenue of
– Full-year 2026 expected total revenue increased to
– Sephience™ (sepiapterin) Q2 2026 revenue of
– Strong cash position of
"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
- Q2 2026 total revenue of
$361 million $239 million - Sephience global launch continues with strong momentum
- Q2 2026 revenue of
$151 million - 1,647 patients on commercial therapy worldwide as of June 30, 2026
- Q2 2026 revenue of
- Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing
- PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching
52% at 10 mg dose level - Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized
- INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease
- INVEST-HD initiation triggered a
$50 million
- PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching
- Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026
- Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21
- Study includes open-label treatment arm with matched natural history control group
- PTC pipeline continues to advance
- PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027
- Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026
- Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026
- Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026.
Second Quarter 2026 Financial Highlights
- Total net product revenue and royalty revenue was
$309.9 million $175.9 million - Total net product revenue across the commercial portfolio was
$238.8 million $118.3 million 100% increase. - Sephience net product revenues were
$151.3 million 21% growth compared to the first quarter of 2026. - Translarna™ (ataluren) net product revenues were
$42.2 million $59.5 million - Emflaza® (deflazacort) net product revenues were
$24.6 million $36.4 million - Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately
968 CHF million, resulting in royalty revenue of$71.1 million $57.6 million - In the second quarter of 2026, PTC recorded a development milestone of
$50.0 million - Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were
$99.2 million $113.0 million - Non-GAAP R&D expenses were
$88.6 million $10.5 million $104.0 million $9.0 million - GAAP SG&A expenses were
$80.6 million $85.3 million - Non-GAAP SG&A expenses were
$67.9 million $12.7 million $75.7 million $9.5 million - Net income was
$83.5 million $64.8 million - In June 2026, PTC issued
$550.0 million 0% coupon and a conversion price representing a40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its1.5% senior convertible notes due in September 2026. - Cash, cash equivalents, and marketable securities were
$2,229.1 million $1,945.4 million - 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 $1.28 billion $850 $950 million $750 $850 million - GAAP R&D and SG&A expense guidance remains
$775 $815 million - Non-GAAP R&D and SG&A expense guidance remains
$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. | |||||||||||||||||||||
Three Months Ended June | Six Months Ended June | ||||||||||||||||||||
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 | 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. | ||||||||||
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 | $ | (165,363) | $ | (205,313) | ||||||
Total liabilities and stockholders' deficit | $ | 3,250,477 | $ | 2,898,767 | ||||||
PTC Therapeutics, Inc. | |||||
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.
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SOURCE PTC Therapeutics, Inc.