PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2025 Financial Results
PTC Therapeutics (NASDAQ:PTCT) reported Q2 2025 financial results and corporate updates. The quarter was marked by the European and FDA approval of Sephience鈩� for PKU treatment with broad labeling. Total revenue reached $178.9 million, including $96 million from the DMD franchise. The company reported a net loss of $64.8 million, improved from $99.2 million in Q2 2024.
Key highlights include cash position of $1.99 billion, positive Phase 2 PIVOT-HD study results for votoplam in Huntington's Disease, and ongoing NDA reviews for vatiquinone and Translarna. PTC updated its full-year 2025 revenue guidance to $650-800 million.
The company entered an agreement to purchase Sephience's annual sales obligation for $225 million upfront plus future milestone payments.PTC Therapeutics (NASDAQ:PTCT) ha comunicato i risultati finanziari del secondo trimestre 2025 e aggiornamenti aziendali. Il trimestre 猫 stato caratterizzato dall'approvazione europea e FDA di Sephience鈩� per il trattamento della PKU con un'ampia indicazione. Il fatturato totale ha raggiunto i 178,9 milioni di dollari, di cui 96 milioni derivanti dal franchise DMD. L'azienda ha registrato una perdita netta di 64,8 milioni di dollari, migliorata rispetto ai 99,2 milioni del secondo trimestre 2024.
I punti salienti includono una posizione di cassa di 1,99 miliardi di dollari, risultati positivi dello studio di Fase 2 PIVOT-HD per votoplam nella malattia di Huntington e revisioni NDA in corso per vatiquinone e Translarna. PTC ha aggiornato la previsione di ricavi per l'intero 2025 a 650-800 milioni di dollari.
L'azienda ha stipulato un accordo per acquistare l'obbligo di vendita annuale di Sephience per un pagamento iniziale di 225 milioni di dollari pi霉 futuri pagamenti legati a milestone.
PTC Therapeutics (NASDAQ:PTCT) inform贸 los resultados financieros del segundo trimestre de 2025 y actualizaciones corporativas. El trimestre estuvo marcado por la aprobaci贸n europea y de la FDA de Sephience鈩� para el tratamiento de PKU con una indicaci贸n amplia. Los ingresos totales alcanzaron los
Los aspectos clave incluyen una posici贸n de efectivo de 1.99 mil millones de d贸lares, resultados positivos del estudio de Fase 2 PIVOT-HD para votoplam en la enfermedad de Huntington, y revisiones NDA en curso para vatiquinone y Translarna. PTC actualiz贸 su pron贸stico de ingresos para todo 2025 a 650-800 millones de d贸lares.
La compa帽铆a firm贸 un acuerdo para comprar la obligaci贸n anual de ventas de Sephience por un pago inicial de 225 millones de d贸lares m谩s pagos futuros por hitos.
PTC Therapeutics (NASDAQ:PTCT)電� 2025雲� 2攵勱赴 鞛 鞁れ爜 氚� 旮办梾 鞐呺嵃鞚错姼毳� 氚滍憸頄堨姷雼堧嫟. 鞚措矆 攵勱赴電� PKU 旃橂鞖� Sephience鈩㈧潣 鞙犽熃 氚� FDA 鞀轨澑瓿� 頃粯 甏戨矓鞙勴暅 鞝侅潙歃濎潉 韸轨鞙茧 頄堨姷雼堧嫟. 齑� 毵れ稖鞚� 1鞏� 7,890毵� 雼煬鞐� 雼枅鞙茧┌, 鞚� 欷� DMD 頂勲灉彀澊歃堨棎靹� 9,600毵� 雼煬毳� 旮半頄堨姷雼堧嫟. 須岇偓電� 6,480毵� 雼煬鞚� 靾滌啇鞁�鞚� 氤搓碃頄堨溂氅�, 鞚措姅 2024雲� 2攵勱赴 9,920毵� 雼煬鞐愳劀 臧滌劆霅� 靾橃箻鞛呺媹雼�.
欤检殧 雮挫毄鞙茧電� 19鞏� 9,000毵� 雼煬鞚� 順勱笀 氤挫湢瓿�, 項岉寘韯措硲鞐� 雽�頃� 氤错啝頂岆瀸鞚� 2靸� PIVOT-HD 鞐瓣惮 旮嶌爼鞝� 瓴瓣臣, 攴鸽Μ瓿� 氚旐嫲韤措吋瓿� 韸鸽灉鞀澕雮橃棎 雽�頃� NDA 鞁偓 歆勴枆 欷戩澊 韽暔霅╇媹雼�. PTC電� 2025雲� 鞝勳泊 毵れ稖 臧�鞚措崢鞀るゼ 6鞏� 5,000毵寏8鞏� 雼煬搿� 鞐呺嵃鞚错姼頄堨姷雼堧嫟.
須岇偓電� Sephience鞚� 鞐瓣皠 韺愲Г 鞚橂毳� 2鞏� 2,500毵� 雼煬 靹犽秷 歆�旮� 氚� 頄ロ泟 毵堨澕鞀ろ啢 歆�旮夑臣 頃粯 鞚胳垬頃橂姅 瓿勳暯鞚� 觳搓舶頄堨姷雼堧嫟.
PTC Therapeutics (NASDAQ:PTCT) a publi茅 les r茅sultats financiers du deuxi猫me trimestre 2025 ainsi que des mises 脿 jour d'entreprise. Le trimestre a 茅t茅 marqu茅 par l'approbation europ茅enne et FDA de Sephience鈩� pour le traitement de la PKU avec un large libell茅. Le chiffre d'affaires total a atteint 178,9 millions de dollars, dont 96 millions provenant de la franchise DMD. La soci茅t茅 a enregistr茅 une perte nette de 64,8 millions de dollars, en am茅lioration par rapport aux 99,2 millions du deuxi猫me trimestre 2024.
Les points cl茅s incluent une tr茅sorerie de 1,99 milliard de dollars, des r茅sultats positifs de l'茅tude de phase 2 PIVOT-HD pour le votoplam dans la maladie de Huntington, ainsi que des examens NDA en cours pour le vatiquinone et Translarna. PTC a mis 脿 jour ses pr茅visions de revenus pour l'ann茅e 2025 脿 650-800 millions de dollars.
La soci茅t茅 a conclu un accord pour acqu茅rir l'obligation annuelle de vente de Sephience pour un paiement initial de 225 millions de dollars plus des paiements futurs li茅s 脿 des jalons.
PTC Therapeutics (NASDAQ:PTCT) ver枚ffentlichte die Finanzergebnisse f眉r das zweite Quartal 2025 sowie Unternehmensupdates. Das Quartal war gepr盲gt von der europ盲ischen und FDA-Zulassung von Sephience鈩� zur Behandlung von PKU mit breiter Indikation. Der Gesamtumsatz erreichte 178,9 Millionen US-Dollar, davon 96 Millionen US-Dollar aus dem DMD-Gesch盲ft. Das Unternehmen meldete einen Nettoverlust von 64,8 Millionen US-Dollar, verbessert gegen眉ber 99,2 Millionen im zweiten Quartal 2024.
Zu den wichtigsten Highlights z盲hlen eine Barmittelposition von 1,99 Milliarden US-Dollar, positive Ergebnisse der Phase-2-Studie PIVOT-HD f眉r Votoplam bei Huntington-Krankheit sowie laufende NDA-Pr眉fungen f眉r Vatiquinon und Translarna. PTC aktualisierte seine Umsatzprognose f眉r das Gesamtjahr 2025 auf 650-800 Millionen US-Dollar.
Das Unternehmen schloss eine Vereinbarung zum Kauf der j盲hrlichen Verkaufsverpflichtung von Sephience f眉r eine Vorabzahlung von 225 Millionen US-Dollar plus zuk眉nftige Meilensteinzahlungen ab.
- FDA and European approval of Sephience with broad labeling for PKU treatment
- Strong cash position of $1.99 billion, up from $1.14 billion in December 2024
- Positive Phase 2 PIVOT-HD study results for votoplam in Huntington's Disease
- Evrysdi royalty revenue increased to $57.6M from $53.2M year-over-year
- Total revenue declined to $178.9M from $186.7M year-over-year
- Net loss of $64.8M in Q2 2025
- DMD franchise revenue declined with Translarna dropping to $59.5M from $70.4M
- SG&A expenses increased to $85.3M from $69.5M year-over-year
Insights
PTC's Sephience PKU drug launches with regulatory wins, but Q2 results show revenue declines despite new approval catalysts.
PTC Therapeutics' Q2 2025 results tell a story of transition as the company pivots toward its newly-approved PKU treatment Sephience as a growth driver. Total revenue came in at
The bright spots in the quarter were regulatory wins for Sephience (sepiapterin), securing both European Commission approval on June 19 and FDA approval on July 28. Notably, both approvals came with broad labels covering all disease subtypes across age groups. The global launch has already begun in the US and Germany, with management positioning Sephience as the "foundational product for PTC's future growth and path to profitability."
The Evrysdi royalty revenue stream continues showing strength, with
PTC's financial position appears solid with
Operating expenses show mixed trends: R&D expenses decreased
The pipeline continues advancing with several regulatory decisions expected in the coming months, including vatiquinone for Friedreich's ataxia (August 19) and Translarna for nonsense mutation DMD. The positive Phase 2 PIVOT-HD results for votoplam in Huntington's Disease and upcoming FDA discussions about a potential accelerated approval pathway represent significant potential value drivers.
PTC secures major PKU drug approvals with optimal labeling but faces revenue challenges as it transitions to new growth drivers.
The regulatory success of Sephience (sepiapterin) represents a significant market access achievement for PTC Therapeutics. Both European and FDA approvals were secured with remarkably broad labels - covering all disease subtypes and all age groups (from 1 month upward in the US). This optimal labeling outcome eliminates potential market fragmentation and maximizes the addressable patient population. The rapid transition to commercial launch activities in Germany and the US demonstrates effective launch readiness planning.
The strategic decision to purchase the percentage-based sales obligation from former Censa shareholders for
PTC's revised full-year 2025 revenue guidance of
The increase in SG&A expenses (
The continued strong performance of Evrysdi royalties (
鈥� European and FDA approval of Sephience鈩� (sepiapterin) with broad labeling for PKU 鈥�
鈥� Global launch underway in
鈥� Total Q2 Revenue of
"We had another strong quarter highlighted by the first approvals of Sephience for the treatment of children and adults with PKU," said Matthew B. Klein, M.D., Chief Executive Officer. "We have initiated the global launch and expect Sephience to be the foundational product for PTC's future growth and path to profitability."
Key Corporate Updates:听
- Second quarter 2025 total net product, collaboration and royalty revenue of
$179 million - Second quarter 2025 revenue for the DMD franchise of
$96 million $59 million $36 million - Initiated global launch of Sephience鈩� in the
U.S. andGermany as well as in other countries through early access and named patient programs - Entered into agreement to purchase the Sephience annual percentage-based global net sales obligation owed to former Censa shareholders in exchange for an upfront payment of
$225 million
Key Clinical and Regulatory Milestones:听
- Sephience鈩� (sepiapterin)
- Marketing authorization granted by the听EC on June 19, 2025 with broad label inclusive of all disease subtypes and all ages 听
- FDA approval on July 28, 2025 with broad label inclusive of all disease subtypes and all ages, from 1 month of age upwards
- Japan NDA review is ongoing with decision expected in Q4 2025
- NDA reviews for听vatiquinone (Friedreich's ataxia) and Translarna (nonsense mutation DMD) are ongoing, with regulatory action date of August 19, 2025 for vatiquinone
- In May 2025, reported positive Phase 2 PIVOT-HD study results for votoplam (PTC518) in Huntington's Disease patients. PTC continues to collaborate with Novartis on next steps and aims to meet with FDA in Q4 2025 to discuss Phase 3 clinical trial design and potential accelerated approval pathway.
Second Quarter 2025 Financial Highlights:
- Total revenues were
$178.9 million $186.7 million - Total revenue includes net product revenue across the commercial portfolio of
$118.3 million $133.2 million $60.5 million $53.5 million - Translarna net product revenues were
$59.5 million $70.4 million - Emflaza net product revenues were
$36.4 million $47.3 million - Roche reported Evrysdi庐 first half 2025 sales of approximately
869 CHF million, resulting in royalty revenue of$57.6 million $53.2 million - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$113.0 million $132.2 million - Non-GAAP R&D expenses were
$104.0 million $9.0 million $122.7 million $9.4 million - GAAP SG&A expenses were
$85.3 million $69.5 million - Non-GAAP SG&A expenses were
$75.7 million $9.5 million $59.7 million $9.8 million - Net loss was
$64.8 million $99.2 million - Cash, cash equivalents, and marketable securities were
$1,989.2 million $1,139.7 million - Shares issued and outstanding as of June 30, 2025, were 79,378,145.
PTC Full-Year 2025 Financial Guidance:
- PTC anticipates full-year 2025 revenue to be between
$650 million $800 million - PTC anticipates full-year 2025 GAAP R&D and SG&A expense to be between
$805 $835 million - PTC anticipates full-year 2025 non-GAAP R&D and SG&A expense to be between
$730 $760 million $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 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 | ||||||||||
2025 | 2024 | 2025 | 2024 | ||||||||
Revenues: | |||||||||||
Net product revenue | $ | 118,329 | $ | 133,220 | $ | 271,755 | $ | 310,824 | |||
Collaboration and license revenue | 2,941 | - | 989,172 | - | |||||||
Royalty revenue | 57,605 | 53,183 | 94,044 | 84,337 | |||||||
Manufacturing revenue | - | 301 | - | 1,661 | |||||||
Total revenues | 178,875 | 186,704 | 1,354,971 | 396,822 | |||||||
Operating expenses: | |||||||||||
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets | 11,420 | 15,527 | 24,282 | 30,267 | |||||||
Amortization of acquired intangible assets | 4,061 | 2,865 | 7,859 | 54,395 | |||||||
Research and development (1) | 112,990 | 132,169 | 221,963 | 248,298 | |||||||
Selling, general and administrative (2) | 85,262 | 69,500 | 166,223 | 142,772 | |||||||
Change in the fair value of contingent consideration | - | 5,100 | (800) | 5,000 | |||||||
Tangible asset impairment and losses (gains) on transactions, net | 99 | 1,761 | 176 | 1,761 | |||||||
Total operating expenses | 213,832 | 226,922 | 419,703 | 482,493 | |||||||
(Loss) income from operations | (34,957) | (40,218) | 935,268 | (85,671) | |||||||
Interest expense, net | (30,358) | (43,490) | (64,450) | (84,324) | |||||||
Other expense, net | (5,737) | (2,025) | (12,042) | (434) | |||||||
(Loss) income before income tax benefit (expense) | (71,052) | (85,733) | 858,776 | (170,429) | |||||||
Income tax benefit (expense) | 6,203 | (13,446) | (57,063) | (20,326) | |||||||
Net (loss) income attributable to common stockholders | $ | (64,849) | $ | (99,179) | $ | 801,713 | $ | (190,755) | |||
Weighted-average shares outstanding: | |||||||||||
Basic (in shares) | 78,151,240 | 76,725,070 | 78,438,830 | 76,610,598 | |||||||
Diluted (in shares) | 78,151,240 | 76,725,070 | 86,502,578 | 76,610,598 | |||||||
Net (loss) income per share鈥攂asic (in dollars per share) | $ | (0.83) | $ | (1.29) | $ | 10.22 | $ | (2.49) | |||
Net (loss) income per share鈥攄iluted (in dollars per share) | $ | (0.83) | $ | (1.29) | $ | 9.29 | $ | (2.49) | |||
(1) Research and development reconciliation | |||||||||||
GAAP research and development | $ | 112,990 | $ | 132,169 | $ | 221,963 | $ | 248,298 | |||
Less: share-based compensation expense | 9,030 | 9,428 | 17,693 | 18,395 | |||||||
Non-GAAP research and development | $ | 103,960 | $ | 122,741 | $ | 204,270 | $ | 229,903 | |||
(2) Selling, general and administrative reconciliation | |||||||||||
GAAP selling, general and administrative | $ | 85,262 | $ | 69,500 | $ | 166,223 | $ | 142,772 | |||
Less: share-based compensation expense | 9,513 | 9,815 | 18,910 | 19,226 | |||||||
Non-GAAP selling, general and administrative | $ | 75,749 | $ | 59,685 | $ | 147,313 | $ | 123,546 | |||
听
PTC Therapeutics, Inc. | |||||
June 30, 2025 | December 31, 2024 | ||||
Cash, cash equivalents and marketable securities | $ | 1,989,150 | $ | 1,139,696 | |
Total Assets | $ | 2,634,155 | $ | 1,705,024 | |
Total debt | $ | 286,013 | $ | 285,412 | |
Total deferred revenue | 9,760 | 5,505 | |||
Total liability for sale of future royalties | 2,096,006 | 2,081,776 | |||
Total liabilities | $ | 2,840,701 | $ | 2,803,095 | |
Total stockholders' deficit (79,378,145 and 77,704,188 common | $ | (206,546) | $ | (1,098,071) | |
Total liabilities and stockholders' deficit | $ | 2,634,155 | $ | 1,705,024 | |
听
PTC Therapeutics, Inc. Reconciliation of GAAP to Non-GAAP Projected Full Year 2025 R&D and听SG&A Expense | |||||
Low End of Range | High End of Range | ||||
Projected GAAP R&D and SG&A Expense | $ | 805 | $ | 835 | |
Less: projected non-cash, stock-based compensation expense | 75 | 75 | |||
Projected non-GAAP R&D and SG&A expense | $ | 730 | $ | 760 | |
听
Acronyms:
CHF: Confoederatio Helvetica Francs (Swiss francs)
DMD: Duchenne Muscular Dystrophy
EC: European Commission
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:听
To access the call by phone, please 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 . A replay of the call will be available approximately two hours after completion of听the call and will be archived on the company's website for 30 days.
About PTC Therapeutics, Inc.听
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听www.ptcbio.com听and follow on Facebook, X, and LinkedIn.
For more information please contact:
Investors:
Ellen Cavaleri
+1 (615) 618-8228
[email protected]
Media:
Jeanine Clemente
+1 (908) 912-9406
[email protected]
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, 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; 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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FAQ
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