PTC Therapeutics Provides Corporate Update and Reports First Quarter 2025 Financial Results
- DMD franchise revenue of $134 million (Translarna: $86M, Emflaza: $48M)
- Strong cash position of $2.0 billion as of March 31, 2025
- Positive CHMP opinion for Sephience™ in April 2025, with FDA decision expected July 29, 2025
- Updated 2025 revenue guidance to $650-800 million
The company received positive regulatory progress for multiple products, including Sephience for PKU patients and Vatiquinone for Friedreich's ataxia. PTC518's Phase 2 PIVOT-HD study met its primary endpoint with promising results.- Ricavi della franchise DMD pari a 134 milioni di dollari (Translarna: 86M, Emflaza: 48M)
- Solida posizione di cassa di 2,0 miliardi di dollari al 31 marzo 2025
- Parere positivo del CHMP per Sephience™ nell'aprile 2025, con decisione FDA prevista per il 29 luglio 2025
- Aggiornamento delle previsioni di fatturato 2025 a 650-800 milioni di dollari
L'azienda ha ottenuto progressi regolatori positivi per diversi prodotti, tra cui Sephience per pazienti con PKU e Vatiquinone per l'atassia di Friedreich. Lo studio di fase 2 PIVOT-HD su PTC518 ha raggiunto l'endpoint primario con risultati promettenti.- Ingresos de la franquicia DMD de 134 millones de dólares (Translarna: 86M, Emflaza: 48M)
- Sólida posición de efectivo de 2.0 mil millones de dólares al 31 de marzo de 2025
- Opinión positiva del CHMP para Sephience™ en abril de 2025, con decisión de la FDA prevista para el 29 de julio de 2025
- Actualización de la guía de ingresos para 2025 a 650-800 millones de dólares
La compañía recibió avances regulatorios positivos para varios productos, incluyendo Sephience para pacientes con PKU y Vatiquinone para ataxia de Friedreich. El estudio de fase 2 PIVOT-HD de PTC518 alcanzó su objetivo principal con resultados prometedores.- DMD 사업부 매출 1억 3,400만 달러 (Translarna: 8,600만 달러, Emflaza: 4,800만 달러)
- 2025년 3월 31일 기준 20억 달러의 강력한 현금 보유고
- 2025년 4월 Sephience™에 대한 CHMP 긍정 의견, FDA 결정은 2025년 7월 29일 예정
- 2025년 매출 가이던스를 6억 5천만~8억 달러로 상향 조정
회사는 PKU 환자를 위한 Sephience와 프리드라이히 운동실조증 치료제 Vatiquinone 등 여러 제품에서 긍정적인 규제 진전을 이루었습니다. PTC518의 2상 PIVOT-HD 연구는 유망한 결과로 주요 평가 지표를 충족했습니다.- Revenus de la franchise DMD de 134 millions de dollars (Translarna : 86 M$, Emflaza : 48 M$)
- Position de trésorerie solide de 2,0 milliards de dollars au 31 mars 2025
- Avis positif du CHMP pour Sephience™ en avril 2025, décision de la FDA attendue le 29 juillet 2025
- Mise à jour des prévisions de revenus 2025 à 650-800 millions de dollars
La société a obtenu des avancées réglementaires positives pour plusieurs produits, dont Sephience pour les patients atteints de PKU et Vatiquinone pour l'ataxie de Friedreich. L'étude de phase 2 PIVOT-HD de PTC518 a atteint son critère principal avec des résultats prometteurs.- Umsatz der DMD-Franchise von 134 Millionen US-Dollar (Translarna: 86 Mio., Emflaza: 48 Mio.)
- Starke Liquiditätsposition von 2,0 Milliarden US-Dollar zum 31. März 2025
- Positive CHMP-Empfehlung für Sephience™ im April 2025, FDA-Entscheidung erwartet am 29. Juli 2025
- Aktualisierte Umsatzprognose für 2025 von 650 bis 800 Millionen US-Dollar
Das Unternehmen erzielte positive regulatorische Fortschritte für mehrere Produkte, darunter Sephience für PKU-Patienten und Vatiquinone für Friedreich-Ataxie. Die Phase-2-Studie PIVOT-HD mit PTC518 erreichte den primären Endpunkt mit vielversprechenden Ergebnissen.- Net income of $866.6 million in Q1 2025, compared to net loss of $91.6 million in Q1 2024
- Strong cash position of $2.0 billion, up from $1.1 billion in December 2024
- Secured $986.2 million from PTC518 license and collaboration agreement with Novartis
- Positive CHMP opinion for Sephience with broad label approval
- PTC518 Phase 2 study met primary endpoint with favorable safety profile
- Total revenue declined to $190 million in Q1 2025 from $209 million in Q1 2024
- Translarna revenue decreased to $86.2 million from $103.6 million year-over-year
- Emflaza revenue dropped to $47.8 million from $57.5 million year-over-year
- Increased SG&A expenses to $81.0 million from $73.3 million in Q1 2024
Insights
PTC's $986M Novartis deal transforms financials to $867M profit with strong $2B cash position despite product revenue declines.
PTC Therapeutics' Q1 2025 financial results reveal a dramatic transformation in their financial position. The $986.2 million collaboration revenue from Novartis for PTC518 propelled the company to $866.6 million in net income, compared to a $91.6 million loss in Q1 2024. This represents one of the most significant quarterly turnarounds in the company's history.
Core product revenues show some challenges with year-over-year declines - Translarna at $86.2 million (down 16.8% from Q1 2024) and Emflaza at $47.8 million (down 16.9%). However, royalty revenue from Evrysdi continues to grow, reaching $36.4 million for the quarter, up from $31.2 million year-over-year.
The most notable financial development is PTC's substantially improved cash position of $2.03 billion, providing extraordinary operational flexibility. Management specifically highlighted they can reach cashflow breakeven without additional capital raises - a critical inflection point for biotechnology companies.
The updated full-year 2025 revenue guidance of $650-800 million shows increasing confidence in their commercial outlook. The 10.5% increase in SG&A expenses signals investment in commercial infrastructure, likely preparing for Sephience's anticipated launch.
The balance sheet shows a $2.09 billion liability for sale of future royalties, indicating PTC has monetized certain future revenue streams to strengthen their current position. Despite product revenue headwinds, the combination of breakthrough pipeline progress and transformed financial standing positions PTC for potential long-term growth as they approach multiple regulatory milestones in 2025.
PTC's pipeline achieves critical regulatory milestones with positive CHMP opinion for Sephience and promising Huntington's disease data.
PTC Therapeutics' pipeline is advancing rapidly across multiple programs with significant near-term regulatory catalysts. The positive CHMP opinion for Sephience (sepiapterin) represents a major achievement, with the European Commission expected to formalize approval within approximately two months. Importantly, the label is broad, covering all age groups and disease severities in PKU, maximizing the addressable patient population.
The FDA review for Sephience remains on track with a July 29, 2025 PDUFA date, positioning the company for potential near-simultaneous transatlantic approvals. Japan's regulatory review is also progressing with a decision expected in Q4 2025, setting up a potential global commercial opportunity.
Vatiquinone has received Priority Review designation from the FDA for Friedreich's ataxia with an August 19, 2025 target date, reflecting the significant unmet need in this rare neurodegenerative condition.
The PTC518 program for Huntington's disease delivered promising Phase 2 PIVOT-HD results, demonstrating dose-dependent HTT protein lowering at Week 12 (the primary endpoint). The data showed trends toward clinical benefit in Stage 2 patients at Month 12 and dose-dependent clinical benefit versus matched natural history controls at Month 24. Critically, the compound maintained favorable safety without treatment-related NfL increases - a biomarker of neuronal damage that has challenged other programs in this space.
With multiple potential approvals in 2025, PTC is positioned to potentially evolve from primarily a DMD-focused company to a multi-therapeutic area rare disease leader with an expanding commercial portfolio.
– Strong revenue performance of
– Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date –
– Global Sephience launch activities progressing well –
– Strong cash position of over
"Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "Our strong cash balance supports all of our planned commercial and R&D activities and provides the ability to reach cashflow breakeven without raising additional capital. The positive CHMP opinion for Sephience kickstarts our anticipated global launch for what we see as a significant revenue opportunity."
Key Corporate Updates:
- First quarter 2025 total net product and royalty revenue of
$190 million - First quarter 2025 revenue for the DMD franchise of
$134 million $86 million $48 million
Key Clinical and Regulatory Milestones:
- Sephience
- Positive CHMP opinion on Sephience MAA for adult and pediatric PKU patients received on April 25, 2025; the opinion includes a broad label inclusive of all ages and disease severities. PTC expects the EC to adopt the opinion in approximately two months
- NDA currently under review by the FDA, with a target regulatory action date of July 29, 2025
Japan regulatory submission under review and a decision is expected in Q4 2025
- Vatiquinone
- NDA for pediatric and adult patients with Friedreich's ataxia accepted and granted Priority Review by the FDA, with a target regulatory action date of August 19, 2025
- Translarna
- NDA currently under review by the FDA
- PTC518
- Phase 2 PIVOT-HD study results announced on May 5, 2025:
- Met primary endpoint of dose-dependent blood HTT lowering at Week 12
- Dose-dependent trends of clinical benefit in Stage 2 patients at Month 12
- Signals of dose-dependent clinical benefit relative to matched natural history cohort and dose-dependent lowering of NfL in Stage 2 subjects at Month 24
- Continued favorable safety and tolerability with no treatment-related NfL spikes
- Plans to complete additional analyses and discuss next development and regulatory steps, including potential for accelerated approval
- Phase 2 PIVOT-HD study results announced on May 5, 2025:
First Quarter 2025 Financial Highlights:
- Total net product and royalty revenue was
$189.9 million $208.8 million - Total revenue includes net product revenue across the commercial portfolio of
$153.4 million $177.6 million $1,022.7 million $32.5 million $986.2 million - Translarna net product revenues were
$86.2 million $103.6 million - Emflaza net product revenues were
$47.8 million $57.5 million - Roche reported Evrysdi® full year 2025 sales of approximately
420 CHF million, resulting in royalty revenue of$36.4 million $31.2 million - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$109.0 million $116.1 million - Non-GAAP R&D expenses were
$100.3 million $8.7 million $107.2 million $9.0 million - GAAP SG&A expenses were
$81.0 million $73.3 million - Non-GAAP SG&A expenses were
$71.6 million $9.4 million $63.9 million $9.4 million - Net income was
$866.6 million $91.6 million - Cash, cash equivalents, and marketable securities were
$2,027.2 million $1,139.7 million - Shares issued and outstanding as of March 31, 2025 were 79,225,276.
PTC Updates Full-Year 2025 Financial Guidance:
- PTC now 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 March 31, | ||||||
2025 | 2024 | |||||
Revenues: | ||||||
Net product revenue | $
| 153,426 | $ | 177,604 | ||
Collaboration and license revenue | 986,231 | - | ||||
Royalty revenue | 36,439 | 31,154 | ||||
Manufacturing revenue | - | 1,360 | ||||
Total revenues | 1,176,096 | 210,118 | ||||
Operating expenses: | ||||||
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets | 12,862 | 14,740 | ||||
Amortization of acquired intangible assets | 3,798 | 51,530 | ||||
Research and development (1) | 108,973 | 116,129 | ||||
Selling, general and administrative (2) | 80,961 | 73,272 | ||||
Change in the fair value of contingent consideration | (800) | (100) | ||||
Tangible asset impairment and losses on transactions, net | 77 | - | ||||
Total operating expenses | 205,871 | 255,571 | ||||
Income (loss) from operations | 970,225 | (45,453) | ||||
Interest expense, net | (34,092) | (40,834) | ||||
Other (expense) income, net | (6,305) | 1,591 | ||||
Income (loss) before income tax expense | 929,828 | (84,696) | ||||
Income tax expense | (63,266) | (6,880) | ||||
Net income (loss) attributable to common stockholders | $ | 866,562 | $ | (91,576) | ||
Weighted-average shares outstanding: | ||||||
Basic (in shares) | 78,115,836 | 76,496,127 | ||||
Diluted (in shares) | 86,385,922 | 76,496,127 | ||||
Net income (loss) per share—basic (in dollars per share) | $ | 11.09 | $ | (1.20) | ||
Net income (loss) per share—diluted (in dollars per share) | $ | 10.04 | $ | (1.20) | ||
(1) Research and development reconciliation | ||||||
GAAP research and development | $ | 108,973 | $ | 116,129 | ||
Less: share-based compensation expense | 8,663 | 8,967 | ||||
Non-GAAP research and development | $ | 100,310 | $ | 107,162 | ||
(2) Selling, general and administrative reconciliation | ||||||
GAAP selling, general and administrative | $ | 80,961 | $ | 73,272 | ||
Less: share-based compensation expense | 9,397 | 9,411 | ||||
Non-GAAP selling, general and administrative | $ | 71,564 | $ | 63,861 | ||
PTC Therapeutics, Inc. | |||||
March 31, 2025 | December 31, 2024 | ||||
Cash, cash equivalents and marketable securities | $ | 2,027,181 | $ | 1,139,696 | |
Total Assets | $ | 2,655,387 | $ | 1,705,024 | |
Total debt | $ | 285,712 | $ | 285,412 | |
Total deferred revenue | 12,833 | 5,505 | |||
Total liability for sale of future royalties | 2,098,463 | 2,081,776 | |||
Total liabilities | $ | 2,841,147 | $ | 2,803,095 | |
Total stockholders' deficit (79,225,276 and 77,704,188 common shares | $ | (185,760) | $ | (1,098,071) | |
Total liabilities and stockholders' deficit | $ | 2,655,387 | $ | 1,705,024 | |
PTC Therapeutics, Inc. | |||||
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 | |
Acronyms:
CHF: Confoederatio Helvetica Francs (Swiss francs)
CHMP: Committee for Medicinal Products for Human Use
DMD: Duchenne Muscular Dystrophy
EC: European Commission
FDA:
GAAP: Generally Accepted Accounting Principles
HD: Huntington's Disease
HTT: Huntingtin protein
MAA: Marketing Authorization Application
NDA: New Drug Application
NfL: Neurofilament light chain
nmDMD: Nonsense mutation Duchenne muscular dystrophy
PDUFA: Prescription Drug User Fee Act
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 of the call and will be archived on the company's website for 30 days.
About PTC Therapeutics, Inc.
PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit www.ptcbio.com and follow on Facebook, X, and LinkedIn.
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 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," 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 Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi, Waylivra, Sephience 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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SOURCE PTC Therapeutics, Inc.
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