PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2025 Financial Results
– Initiated US and EU launch of Sephience™ (sepiapterin) –
– Strong initial Sephience uptake with global revenue of
– Robust Q3 performance with total revenue of
– Full-year 2025 revenue guidance narrowed to
"We achieved an outstanding quarter, highlighted by the strong start to the global Sephience launch," said
Key Corporate Updates
- Third quarter 2025 total revenue of
$211.0 million - Global launch of Sephience™ initiated in US and
Europe - Third quarter 2025 revenue of
$19.6 million , including$14.4 million revenue in the US and$5.2 million revenue ex-US - 521 patient start forms received from 141 unique prescribers in the US as of
September 30 - 341 total patients on commercial therapy worldwide as of
September 30
- Third quarter 2025 revenue of
- Third quarter 2025 revenue for the DMD franchise of
$85.9 million , including net product revenue for Translarna™ of$50.7 million and for Emflaza® of$35.2 million - R&D Day planned for
Tuesday, December 2 nd inNew York
Key Clinical and Regulatory Updates
- Additional Sephience marketing authorization reviews ongoing including in
Japan where decision is expected in Q4 2025 - FDA meeting for votoplam Huntington's disease program planned for Q4
- FDA meeting planned for vatiquinone Friedreich's ataxia program in Q4
- Translarna NDA remains under FDA review
Third Quarter 2025 Financial Highlights
- Total revenue was
$211.0 million for the third quarter of 2025, compared to$196.8 million for the third quarter of 2024. - Total revenue includes net product revenue across the commercial portfolio of
$131.0 million for the third quarter of 2025, compared to$135.4 million for the third quarter of 2024. Total revenue also includes royalty, collaboration and license revenue of$80.1 million for the third quarter of 2025, compared to$61.4 million for the third quarter of 2024. - Sephience net product revenue was
$19.6 million for the third quarter of 2025. - Translarna net product revenue was
$50.7 million for the third quarter of 2025, compared to$72.3 million for the third quarter of 2024. - Emflaza net product revenue was
$35.2 million for the third quarter of 2025, compared to$51.9 million for the third quarter of 2024. - Roche reported Evrysdi® 2025 year-to-date sales of approximately
CHF 1,293 million , resulting in royalty revenue of$70.8 million to PTC for the third quarter of 2025, as compared to$61.4 million for the third quarter of 2024. - Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expense was
$100.2 million for the third quarter of 2025, compared to$161.4 million for the third quarter of 2024. - Non-GAAP R&D expense was
$91.0 million for the third quarter of 2025, excluding$9.1 million in non-cash, stock-based compensation expense, compared to$152.0 million for the third quarter of 2024, excluding$9.4 million in non-cash, stock-based compensation expense. - GAAP SG&A expense was
$84.0 million for the third quarter of 2025, compared to$73.5 million for the third quarter of 2024. - Non-GAAP SG&A expense was
$73.8 million for the third quarter of 2025, excluding$10.3 million in non-cash, stock-based compensation expense, compared to$63.1 million for the third quarter of 2024, excluding$10.3 million in non-cash, stock-based compensation expense. - Net income was
$15.9 million for the third quarter of 2025, compared to net loss of$106.7 million for the third quarter of 2024. - Cash, cash equivalents, and marketable securities were
$1,687.8 million as ofSeptember 30, 2025 , compared to$1,139.7 million as ofDecember 31, 2024 . The third quarter cash balance reflects the PTC agreement to purchase approximately 90% of the Sephience annual percentage-based global net sales obligation owed to former Censa shareholders in exchange for an upfront payment of$225.0 million and future sales-based milestone payments. - Shares issued and outstanding as of
September 30, 2025 , were 79,931,766.
PTC Full-Year 2025 Financial Guidance
For the full year 2025, PTC anticipates:
- Total revenue of
$750 to$800 million , which includes in-line products and royalty revenue from Evrysdi. - GAAP R&D and SG&A expense of
$805 to$835 million . - Non-GAAP R&D and SG&A expense of
$730 to$760 million , excluding estimated non-cash, stock-based compensation expense of$75 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.
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Three Months Ended |
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Nine Months Ended |
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2025 |
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2024 |
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2025 |
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2024 |
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Revenues: |
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Net product revenue |
$ |
130,956 |
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$ |
135,421 |
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$ |
402,711 |
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$ |
446,245 |
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Collaboration and license revenue |
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9,258 |
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- |
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998,430 |
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- |
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Royalty revenue |
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70,793 |
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61,365 |
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164,837 |
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145,702 |
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Manufacturing revenue |
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- |
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- |
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- |
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1,661 |
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Total revenues |
211,007 |
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196,786 |
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1,565,978 |
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593,608 |
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Operating expenses: |
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Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets |
15,781 |
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10,848 |
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40,063 |
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41,115 |
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Amortization of acquired intangible assets |
7,534 |
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3,036 |
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15,393 |
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57,431 |
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Research and development (1) |
100,158 |
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161,412 |
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322,121 |
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409,710 |
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Selling, general and administrative (2) |
84,046 |
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73,456 |
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250,269 |
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216,228 |
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Change in the fair value of contingent consideration |
- |
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700 |
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(800) |
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5,700 |
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Tangible asset impairment and losses (gains) on transactions, net |
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472 |
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1,844 |
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648 |
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3,605 |
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Total operating expenses |
207,991 |
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251,296 |
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627,694 |
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733,789 |
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Income (loss) from operations |
3,016 |
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(54,510) |
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938,284 |
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(140,181) |
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Interest expense, net |
(32,592) |
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(41,609) |
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(97,042) |
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(125,933) |
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Other income (expense), net |
6,502 |
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(1,872) |
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(5,540) |
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(2,306) |
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(Loss) income before income tax benefit (expense) |
(23,074) |
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(97,991) |
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835,702 |
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(268,420) |
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Income tax benefit (expense) |
38,970 |
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(8,663) |
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(18,093) |
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(28,989) |
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Net income (loss) attributable to common stockholders |
$ |
15,896 |
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$ |
(106,654) |
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$ |
817,609 |
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$ |
(297,409) |
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Weighted-average shares outstanding: |
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Basic (in shares) |
78,471,119 |
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76,925,523 |
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78,642,478 |
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76,716,340 |
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Diluted (in shares) |
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87,115,369 |
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76,925,523 |
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86,899,727 |
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76,716,340 |
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Net income (loss) per share—basic (in dollars per share) |
$ |
0.20 |
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$ |
(1.39) |
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$ |
10.40 |
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$ |
(3.88) |
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Net income (loss) per share—diluted (in dollars per share) |
$ |
0.20 |
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$ |
(1.39) |
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$ |
9.45 |
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$ |
(3.88) |
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(1) Research and development reconciliation |
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GAAP research and development |
$ |
100,158 |
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$ |
161,412 |
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$ |
322,121 |
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$ |
409,710 |
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Less: share-based compensation expense |
9,133 |
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9,416 |
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26,827 |
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27,810 |
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Non-GAAP research and development |
$ |
91,025 |
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$ |
151,996 |
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$ |
295,294 |
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$ |
381,900 |
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(2) Selling, general and administrative reconciliation |
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GAAP selling, general and administrative |
$ |
84,046 |
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$ |
73,456 |
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$ |
250,269 |
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$ |
216,228 |
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Less: share-based compensation expense |
10,293 |
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10,339 |
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29,202 |
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29,566 |
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Non-GAAP selling, general and administrative |
$ |
73,753 |
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$ |
63,117 |
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$ |
221,067 |
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$ |
186,662 |
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Cash, cash equivalents and marketable securities |
$ |
1,687,817 |
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$ |
1,139,696 |
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Total Assets |
$ |
2,643,700 |
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$ |
1,705,024 |
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Total debt |
$ |
286,319 |
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$ |
285,412 |
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Total deferred revenue |
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1,691 |
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5,505 |
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Total liability for sale of future royalties |
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2,081,921 |
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2,081,776 |
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Total liabilities |
$ |
2,799,458 |
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$ |
2,803,095 |
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Total stockholders' deficit (79,931,766 and 77,704,188 common shares |
$ |
(155,758) |
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$ |
(1,098,071) |
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Total liabilities and stockholders' deficit |
$ |
2,643,700 |
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$ |
1,705,024 |
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Low End of Range |
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High End of Range |
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Projected GAAP R&D and SG&A Expense |
$ |
805 |
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$ |
835 |
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Less: projected non-cash, stock-based compensation expense |
75 |
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75 |
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Projected non-GAAP R&D and SG&A expense |
$ |
730 |
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$ |
760 |
Acronyms:
CHF: Confoederatio Helvetica Francs (Swiss francs)
DMD: Duchenne Muscular Dystrophy
FDA:
GAAP: Generally Accepted Accounting Principles
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. To avoid delays, we recommend participants dial in for the conference call 15 minutes prior to the start of the call. 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 approximately two hours after completion and will be archived on the company's website for 30 days.
About
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 and Facebook.
For more information please contact:
Investors:
+1 (615) 618-8228
ecavaleri@ptcbio.com
Media:
+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 Full-Year 2025 Financial Guidance", including with respect to (i) 2025 total revenue guidance and (ii) 2025 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 regulatory and 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
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, Waylivra or vatiquinone.
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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