PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2024 Financial Results
PTC Therapeutics reported Q3 2024 total revenue of $196.8 million, leading to increased full-year guidance of $750-800 million. The DMD franchise generated $124 million, with Translarna contributing $72.3 million and Emflaza $51.9 million. The company has three FDA applications under review and plans a fourth submission for vatiquinone in December. Q3 net loss was $106.7 million, improved from $133.0 million in Q3 2023. Cash position strengthened to $1.01 billion as of September 30, 2024, up from $876.7 million at end of 2023.
PTC Therapeutics ha riportato un fatturato totale di $196,8 milioni per il terzo trimestre del 2024, portando a un'aggiornamento al rialzo delle previsioni annuali a $750-800 milioni. Il segmento DMD ha generato $124 milioni, con Translarna che ha contribuito con $72,3 milioni e Emflaza con $51,9 milioni. L'azienda ha tre domande di approvazione presso la FDA in fase di revisione e prevede una quarta presentazione per vatiquinone a dicembre. La perdita netta del terzo trimestre è stata di $106,7 milioni, in miglioramento rispetto ai $133,0 milioni del terzo trimestre del 2023. La posizione di cassa è migliorata a $1,01 miliardi al 30 settembre 2024, in aumento rispetto ai $876,7 milioni di fine 2023.
PTC Therapeutics reportó ingresos totales de $196,8 millones en el tercer trimestre de 2024, lo que llevó a una mejora en la guía anual a $750-800 millones. La franquicia DMD generó $124 millones, con Translarna contribuyendo con $72,3 millones y Emflaza con $51,9 millones. La compañía tiene tres solicitudes bajo revisión por parte de la FDA y planea una cuarta presentación para vatiquinona en diciembre. La pérdida neta del tercer trimestre fue de $106,7 millones, mejorando desde los $133,0 millones del tercer trimestre de 2023. La posición de efectivo se fortaleció a $1,01 mil millones hasta el 30 de septiembre de 2024, en comparación con los $876,7 millones a finales de 2023.
PTC Therapeutics는 2024년 3분기 총 수익이 1억 9680만 달러에 달했다고 보고하며 연간 가이드를 7억 5000만 ~ 8억 달러로 상향 조정했습니다. DMD 부문은 1억 2400만 달러를 창출하며, Translarna가 7230만 달러, Emflaza가 5190만 달러를 기여했습니다. 이 회사는 현재 FDA의 검토 중인 신청서가 3건 있으며 12월에 vatiquinone에 대한 네 번째 제출을 계획하고 있습니다. 3분기 순손실은 1억 670만 달러로, 2023년 3분기의 1억 3300만 달러에서 개선되었습니다. 2024년 9월 30일 기준으로 현금 보유액은 10억 1000만 달러로 증가했으며, 2023년 말의 8억 7670만 달러에서 상승했습니다.
PTC Therapeutics a reporté un chiffre d'affaires total de 196,8 millions de dollars pour le troisième trimestre 2024, ce qui a conduit à une révision à la hausse des prévisions annuelles à 750-800 millions de dollars. La franchise DMD a généré 124 millions de dollars, avec Translarna contribuant 72,3 millions de dollars et Emflaza 51,9 millions de dollars. L'entreprise a trois demandes d'approbation auprès de la FDA en cours d'examen et prévoit une quatrième soumission pour le vatiquinone en décembre. La perte nette au troisième trimestre était de 106,7 millions de dollars, en amélioration par rapport aux 133,0 millions de dollars du troisième trimestre 2023. La position de liquidités s'est renforcée à 1,01 milliard de dollars au 30 septembre 2024, contre 876,7 millions de dollars à la fin de 2023.
PTC Therapeutics meldete für das dritte Quartal 2024 einen Gesamtumsatz von 196,8 Millionen US-Dollar, was zu einer Anhebung der Jahresprognose auf 750-800 Millionen US-Dollar führte. Das DMD-Segment generierte 124 Millionen US-Dollar, wobei Translarna 72,3 Millionen US-Dollar und Emflaza 51,9 Millionen US-Dollar beitrugen. Das Unternehmen hat drei Anträge zur Genehmigung bei der FDA in Bearbeitung und plant im Dezember eine vierte Einreichung für Vatiquinon. Der Nettoverlust im dritten Quartal betrug 106,7 Millionen US-Dollar, eine Verbesserung gegenüber 133,0 Millionen US-Dollar im dritten Quartal 2023. Die Liquiditätsposition wurde zum 30. September 2024 auf 1,01 Milliarden US-Dollar gestärkt, im Vergleich zu 876,7 Millionen US-Dollar Ende 2023.
- Increased full-year revenue guidance to $750-800 million
- Strong cash position of $1.01 billion, up from $876.7 million in December 2023
- Royalty revenue increased to $61.4 million from $50.2 million year-over-year
- Reduced net loss to $106.7 million from $133.0 million year-over-year
- Decreased SG&A expenses to $73.5 million from $80.9 million year-over-year
- Total product revenue declined to $135.4 million from $144.0 million year-over-year
- Emflaza revenue decreased to $51.9 million from $67.4 million year-over-year
- Operating loss of $54.5 million in Q3 2024
- Increased interest expense to $41.6 million from $28.2 million year-over-year
Insights
The Q3 results show mixed performance with flat total revenues of
The company reduced operating expenses, with R&D and SG&A costs decreasing year-over-year. However, net loss remains substantial at
The regulatory pipeline shows strong momentum with three FDA applications under review: sepiapterin for PKU (PDUFA date: July 2025), Translarna for nmDMD and AADC gene therapy (decision expected November 2024). The upcoming NDA submission for vatiquinone in Friedreich ataxia and Fast Track designation for PTC518 in Huntington's disease demonstrate pipeline diversification.
The portfolio includes both rare disease treatments and novel therapeutic approaches. The anticipated Q4 data readout for utreloxastat in ALS could provide additional growth catalyst. Strategic portfolio prioritization has helped optimize R&D spending while maintaining focus on high-potential programs.
– Strong revenue performance, supporting increase in full-year revenue guidance to
– Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA –
– On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone –
WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024.
"I am proud of our team's strong execution," said Matthew Klein, M.D., Chief Executive Officer. "We continue to achieve excellent revenue performance allowing us to raise full-year revenue guidance. In addition, we have submitted three approval applications to FDA so far this year, all of which have been accepted for review, and plan a fourth submission for vatiquinone for Friedreich ataxia in December. We are on our way to accomplishing all of our 2024 objectives and are in a strong financial position to execute on our long-term strategic plans."
Key Corporate Updates:
- Third quarter 2024 total revenue of
$197 million - Third quarter 2024 revenue for the DMD franchise was
$124 million - Translarna™ (ataluren) net product revenue was
$72 million - Emflaza® (deflazacort) net product revenue was
$52 million
- Translarna™ (ataluren) net product revenue was
Key Clinical and Regulatory Milestones:
- PTC submitted an NDA to the FDA for sepiapterin for the treatment of PKU. The file was accepted for review, and the regulatory action date is set for July 29, 2025.
- PTC resubmitted the NDA for Translarna for the treatment of nmDMD; the file was accepted, and a regulatory action date was not provided.
- The BLA for PTC's gene therapy for AADC deficiency remains on schedule for a regulatory action date of November 13, 2024.
- PTC received Fast Track Designation for the PTC518 Huntington's disease program.
- PTC plans to submit an NDA for vatiquinone for the treatment of Friedreich ataxia in December 2024.
- PTC expects to share topline data for the CardinALS trial of utreloxastat for the treatment of ALS in the fourth quarter of 2024.
Third Quarter 2024 Financial Highlights:
- Total revenues were
$196.8 million $196.6 million - Total revenue includes net product revenue across the commercial portfolio of
$135.4 million $144.0 million $61.4 million $52.5 million - Translarna net product revenues were
$72.3 million $69.0 million - Emflaza net product revenues were
$51.9 million $67.4 million - Roche reported Evrysdi® 2024 year-to-date sales of approximately
CHF 1.2 billion , resulting in royalty revenue of$61.4 million $50.2 million - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$161.4 million $164.2 million $50.0 million - Non-GAAP R&D expenses were
$152.0 million $9.4 million $150.2 million $14.0 million - GAAP SG&A expenses were
$73.5 million $80.9 million - Non-GAAP SG&A expenses were
$63.1 million $10.3 million $67.9 million $13.0 million - Change in the fair value of deferred and contingent consideration was a loss of
$0.7 million $1.5 million - Tangible asset impairment and losses (gains) on transactions, net, was a loss of
$1.8 million $0.0 million - Net loss was
$106.7 million $133.0 million - Cash, cash equivalents, and marketable securities was
$1,013.4 million $876.7 million - Shares issued and outstanding as of September 30, 2024, were 76,952,124.
PTC Updates Full Year 2024 Financial Guidance:
- PTC anticipates total revenues for full year 2024 to be between
$750 million $800 million - PTC anticipates GAAP R&D and SG&A expenses for full year 2024 to be between
$740 million $835 million $65 million - PTC anticipates non-GAAP R&D and SG&A expenses for full year 2024 to be between
$660 million $755 million $65 million $80 million - PTC anticipates up to
$90 million $65 million $85 million $65 million $20 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 | Nine Months Ended September | ||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||
Revenues: | |||||||||||
Net product revenue | $ | 135,421 | $ | 144,038 | $ | 446,245 | $ | 506,187 | |||
Collaboration revenue | - | - | - | 6 | |||||||
Royalty revenue | 61,365 | 50,173 | 145,702 | 117,857 | |||||||
Manufacturing revenue | - | 2,365 | 1,661 | 6,716 | |||||||
Total revenues | 196,786 | 196,576 | 593,608 | 630,766 | |||||||
Operating expenses: | |||||||||||
Cost of product sales, excluding amortization of acquired | 10,848 | 9,493 | 41,115 | 36,368 | |||||||
Amortization of acquired intangible asset | 3,036 | 58,649 | 57,431 | 145,461 | |||||||
Research and development (1) | 161,412 | 164,212 | 409,710 | 545,210 | |||||||
Selling, general and administrative (2) | 73,456 | 80,886 | 216,228 | 256,249 | |||||||
Change in the fair value of deferred and contingent | 700 | 1,500 | 5,700 | (125,000) | |||||||
Intangible asset impairment | - | - | - | 217,800 | |||||||
Tangible asset impairment and losses (gains) on transactions, | 1,844 | - | 3,605 | - | |||||||
Total operating expenses | 251,296 | 314,740 | 733,789 | 1,076,088 | |||||||
Loss from operations | (54,510) | (118,164) | (140,181) | (445,322) | |||||||
Interest expense, net | (41,609) | (28,160) | (125,933) | (84,905) | |||||||
Other expense, net | (1,872) | (20,266) | (2,306) | (8,832) | |||||||
Loss before income tax (expense) benefit | (97,991) | (166,590) | (268,420) | (539,059) | |||||||
Income tax (expense) benefit | (8,663) | 33,620 | (28,989) | 68,247 | |||||||
Net loss attributable to common stockholders | $ | (106,654) | $ | (132,970) | $ | (297,409) | $ | (470,812) | |||
Weighted-average shares outstanding: | |||||||||||
Basic and diluted (in shares) | 76,925,523 | 75,377,997 | 76,716,340 | 74,618,611 | |||||||
Net loss per share—basic and diluted (in dollars per share) | $ | (1.39) | $ | (1.76) | $ | (3.88) | $ | (6.31) | |||
(1) Research and development reconciliation | |||||||||||
GAAP research and development | $ | 161,412 | $ | 164,212 | $ | 409,710 | $ | 545,210 | |||
Less: share-based compensation expense | 9,416 | 13,986 | 27,810 | 44,828 | |||||||
Non-GAAP research and development | $ | 151,996 | $ | 150,226 | $ | 381,900 | $ | 500,382 | |||
(2) Selling, general and administrative reconciliation | |||||||||||
GAAP selling, general and administrative | $ | 73,456 | $ | 80,886 | $ | 216,228 | $ | 256,249 | |||
Less: share-based compensation expense | 10,339 | 12,956 | 29,566 | 40,300 | |||||||
Non-GAAP selling, general and administrative | $ | 63,117 | $ | 67,930 | $ | 186,662 | $ | 215,949 | |||
PTC Therapeutics, Inc. | |||||
September 30, 2024 | December 31, 2023 | ||||
Cash, cash equivalents and marketable securities | $ | 1,013,357 | $ | 876,739 | |
Total assets | $ | 1,842,236 | $ | 1,895,698 | |
Total debt | $ | 285,106 | $ | 284,213 | |
Total deferred revenue | - | 801 | |||
Total liability for sale of future royalties | 2,082,051 | 1,814,097 | |||
Total liabilities | $ | 2,896,588 | $ | 2,714,253 | |
Total stockholders' deficit (76,952,124 and 75,708,889 | $ | (1,054,352) | $ | (818,555) | |
Total liabilities and stockholders' deficit | $ | 1,842,236 | $ | 1,895,698 | |
PTC Therapeutics, Inc. | ||
Full Year 2024 | ||
(in millions) | ||
Projected GAAP R&D Expense Related Milestone Payments | $ | 65 |
Projected GAAP Contingent Consideration Payable Related Milestone Payments | 25 | |
Total Projected GAAP Milestone Payments | $ | 90 |
PTC Therapeutics, Inc. | ||||||
Low End | High End | |||||
Projected GAAP R&D and SG&A Expense | $ | 740 | $ | 835 | ||
Less: projected non-cash, stock-based compensation expense | 80 | 80 | ||||
Projected non-GAAP R&D and SG&A expense | $ | 660 | $ | 755 | ||
Acronyms:
AADC: Aromatic l-Amino Acid Decarboxylase
ALS: Amyotrophic Lateral Sclerosis
BLA: Biologics License Application
CHF: Confoederatio Helvetica Francs (Swiss francs)
DMD: Duchenne Muscular Dystrophy
FA: Friedreich Ataxia
FDA:
GAAP: Generally Accepted Accounting Principles
HD: Huntington's Disease
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 to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor 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 following the call.
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 us at www.ptcbio.com and follow us on Facebook, X, and LinkedIn.
For More Information:
Investors:
Ellen Cavaleri
+1 (615) 618-6228
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 2024 Financial Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment 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; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; 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, Evrysdi, Tegsedi, Waylivra or sepiapterin.
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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