PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2024 Financial Results
PTC Therapeutics reported Q2 2024 financial results with total revenue of $186.7 million, down from $213.8 million in Q2 2023. Key highlights include:
- DMD franchise revenue of $118 million
- Translarna net product revenue of $70 million
- Emflaza net product revenue of $47 million
- Evrysdi royalty revenue of $53.2 million
The company submitted NDAs for sepiapterin and Translarna to the FDA. PTC reported positive interim data from the PTC518 PIVOT-HD study in Huntington's disease. Cash position strengthened to $1.09 billion. Full-year 2024 revenue guidance is $700-$750 million.
PTC Therapeutics ha riportato i risultati finanziari del Q2 2024 con un fatturato totale di 186,7 milioni di dollari, in calo rispetto ai 213,8 milioni di dollari del Q2 2023. Tra i principali punti salienti:
- Fatturato della franchigia DMD di 118 milioni di dollari
- Fatturato netto del prodotto Translarna di 70 milioni di dollari
- Fatturato netto del prodotto Emflaza di 47 milioni di dollari
- Fatturato delle royalty di Evrysdi di 53,2 milioni di dollari
La società ha presentato NDA per sepiapterin e Translarna alla FDA. PTC ha riportato dati preliminari positivi dallo studio PTC518 PIVOT-HD sulla malattia di Huntington. La posizione di cassa si è rafforzata a 1,09 miliardi di dollari. Le previsioni di fatturato per l'intero anno 2024 sono comprese tra 700 e 750 milioni di dollari.
PTC Therapeutics informó sobre los resultados financieros del Q2 2024, con ingresos totales de 186.7 millones de dólares, en comparación con 213.8 millones de dólares en el Q2 2023. Los puntos destacados incluyen:
- Ingresos de la franquicia DMD de 118 millones de dólares
- Ingresos netos del producto Translarna de 70 millones de dólares
- Ingresos netos del producto Emflaza de 47 millones de dólares
- Ingresos por regalías de Evrysdi de 53.2 millones de dólares
La compañía presentó NDA para sepiapterin y Translarna a la FDA. PTC reportó datos interinos positivos del estudio PTC518 PIVOT-HD para la enfermedad de Huntington. La posición de efectivo se fortaleció a 1.09 mil millones de dólares. La guía de ingresos para todo el año 2024 es de 700 a 750 millones de dólares.
PTC 제약은 2024년 2분기 재무 결과를 보고했으며, 총 수익은 1억 8,670만 달러로, 2023년 2분기의 2억 1,380만 달러에서 감소했습니다. 주요 내용은 다음과 같습니다:
- DMD 프랜차이즈 수익 1억 1,800만 달러
- Translarna 순 제품 수익 7,000만 달러
- Emflaza 순 제품 수익 4,700만 달러
- Evrysdi 로열티 수익 5,320만 달러
회사는 FDA에 sepiapterin과 Translarna에 대한 NDA를 제출했습니다. PTC는 헌팅턴병에 대한 PTC518 PIVOT-HD 연구의 긍정적인 중간 데이터를 보고했습니다. 현금 보유액은 10억 9,000만 달러로 강화되었습니다. 2024년 전체 연도 수익 가이드는 7억 ~ 7억 5천만 달러입니다.
PTC Therapeutics a rapporté ses résultats financiers du Q2 2024, avec un revenu total de 186,7 millions de dollars, en baisse par rapport à 213,8 millions de dollars au Q2 2023. Les principaux points forts incluent :
- Revenu de la franchise DMD de 118 millions de dollars
- Revenu net du produit Translarna de 70 millions de dollars
- Revenu net du produit Emflaza de 47 millions de dollars
- Revenu de royalties d'Evrysdi de 53,2 millions de dollars
La société a soumis des NDA pour le sepiapterin et le Translarna à la FDA. PTC a rapporté des données intermédiaires positives de l'étude PTC518 PIVOT-HD sur la maladie de Huntington. La position de trésorerie a été renforcée à 1,09 milliard de dollars. Les prévisions de revenus pour l'année complète 2024 sont de 700 à 750 millions de dollars.
PTC Therapeutics berichtete über die Finanzergebnisse des Q2 2024 mit einem Gesamtumsatz von 186,7 Millionen Dollar, ein Rückgang von 213,8 Millionen Dollar im Q2 2023. Wichtige Highlights sind:
- Umsatz der DMD-Franchise von 118 Millionen Dollar
- Nettoumsatz des Produkts Translarna von 70 Millionen Dollar
- Nettoumsatz des Produkts Emflaza von 47 Millionen Dollar
- Lizenzgebühren von Evrysdi von 53,2 Millionen Dollar
Das Unternehmen reichte NDA für Sepiapterin und Translarna bei der FDA ein. PTC berichtete über positive Zwischenwerte aus der PTC518 PIVOT-HD-Studie zur Huntington-Krankheit. Die Liquidität stärkte sich auf 1,09 Milliarden Dollar. Die Umsatzprognose für das Gesamtjahr 2024 liegt bei 700 bis 750 Millionen Dollar.
- Submitted NDAs for sepiapterin and Translarna to FDA
- Positive interim data from PTC518 PIVOT-HD study in Huntington's disease
- Strong cash position of $1.09 billion, up from $876.7 million at end of 2023
- Evrysdi royalty revenue increased to $53.2 million from $36.9 million in Q2 2023
- On track to achieve remaining 2024 clinical and regulatory milestones
- Total revenue decreased to $186.7 million from $213.8 million in Q2 2023
- Translarna net product revenue declined to $70.4 million from $96.5 million in Q2 2023
- Emflaza net product revenue fell to $47.3 million from $65.7 million in Q2 2023
- Net loss of $99.2 million for Q2 2024
- Expiration of Emflaza's orphan drug exclusivity in February 2024
Insights
PTC Therapeutics' Q2 2024 results show a mixed financial picture. Total revenues of
The decline in product revenues, particularly for Translarna and Emflaza, is concerning. Translarna's revenue drop was attributed to timing of bulk orders, while Emflaza's decline was due to loss of exclusivity. On the positive side, royalty revenue from Evrysdi showed strong growth, increasing by
PTC's cost-cutting efforts are evident, with both R&D and SG&A expenses decreasing significantly. This has helped narrow the net loss to
PTC Therapeutics' pipeline progress is noteworthy. The company has submitted NDAs for sepiapterin (PKU treatment) and Translarna (nmDMD treatment) to the FDA, marking significant regulatory milestones. The positive interim data from the PTC518 PIVOT-HD study in Huntington's disease is also encouraging, potentially opening up a new market opportunity.
The BLA acceptance for PTC's gene therapy for AADC deficiency, with priority review and a target action date of November 13, 2024, is a major development. If approved, this could be PTC's first gene therapy product, diversifying its portfolio and potentially driving future growth.
However, the expiration of Emflaza's orphan drug exclusivity in February 2024 poses a challenge, as reflected in its declining revenues. The company will need to navigate this loss of exclusivity while leveraging its pipeline to maintain growth. The planned NDA submission for vatiquinone in Friedreich ataxia later this year could provide another growth avenue if successful.
PTC Therapeutics' market position appears to be in transition. While facing headwinds with its established products, the company is actively expanding its pipeline and geographic reach. The continued access and expansion for Translarna in existing and new geographies is positive, although the impact of bulk order timing makes it difficult to assess true demand trends.
The strong performance of Evrysdi, marketed by Roche, is a bright spot. With year-to-date sales of approximately
Investor focus is likely to shift towards the company's late-stage pipeline, particularly the AADC deficiency gene therapy and the potential launches of sepiapterin and Translarna in the US, if approved. The outcome of these regulatory decisions could significantly impact PTC's future market position and valuation. The company's ability to successfully commercialize these potential new products will be important for offsetting the challenges faced by its current commercial portfolio.
– Continued strong revenue performance –
– NDAs for sepiapterin and Translarna™ submitted to FDA –
– Positive interim data readout from PTC518 PIVOT-HD study –
– On track to achieve remaining 2024 clinical and regulatory milestones –
"I am proud of our team's continued outstanding execution as we have accomplished all our objectives so far this year," said Matthew Klein, M.D., Chief Executive Officer. "We are in a strong cash position, submitted three drug approval applications to the FDA and remain on schedule to achieve the many milestones we have set for the remainder of 2024."
Key Corporate Updates:
- Second quarter 2024 total revenue of
$187 million - Second quarter 2024 revenue for the DMD franchise was
$118 million
- Translarna™ (ataluren) net product revenue was
$70 million - Emflaza® (deflazacort) net product revenue was
$47 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 in July 2024. Further regulatory submissions are planned for
Japan andBrazil later in 2024. - PTC resubmitted the NDA for Translarna for the treatment of nmDMD in July 2024.
- PTC achieved all objectives in the interim readout of the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease patients, which were disclosed in the second quarter.
- PTC plans to submit an NDA for vatiquinone for the treatment of Friedreich ataxia in late 2024.
- PTC expects to share topline data for the CardinALS trial of utreloxastat for the treatment of ALS in the fourth quarter of 2024.
- The BLA for PTC's gene therapy for AADC deficiency was accepted by FDA with priority review; the target regulatory action date is November 13, 2024.
Second-Quarter 2024 Financial Highlights:
- Total revenues were
$186.7 million $213.8 million - Total revenues include net product revenue across the commercial portfolio of
$133.2 million $174.6 million $53.5 million $39.2 million - Translarna net product revenues were
$70.4 million $96.5 million - Emflaza net product revenues were
$47.3 million $65.7 million - Roche reported Evrysdi 2024 year-to-date sales of approximately
CHF 838 million , resulting in royalty revenue of$53.2 million $36.9 million - Based on
U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were$132.2 million $185.9 million $15.0 million - Non-GAAP R&D expenses were
$122.7 million $9.4 million $170.3 million $15.5 million - GAAP SG&A expenses were
$69.5 million $88.4 million - Non-GAAP SG&A expenses were
$59.7 million $9.8 million $74.6 million $13.8 million - Change in the fair value of deferred and contingent consideration was a loss of
$5.1 million $128.9 million $0 $129.8 million - Tangible asset impairment and losses (gains) on transactions, net, was a loss of
$1.8 million $0 million - Net loss was
$99.2 million $198.9 million - Cash, cash equivalents, and marketable securities was
$1,093.3 million $876.7 million $250 million - Shares issued and outstanding as of June 30, 2024, were 76,900,123.
PTC Updates Full Year 2024 Financial Guidance:
- PTC anticipates total revenues for full year 2024 to be between
$700 million $750 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 $35 million $15 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. | |||||||||||
Consolidated Statements of Operations | |||||||||||
(In thousands, except share and per share data) | |||||||||||
Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||
2024 | 2023 | 2024 | 2023 | ||||||||
Revenues: | |||||||||||
Net product revenue | $ | 133,220 | $ | 174,592 | $ | 310,824 | $ | 362,149 | |||
Collaboration revenue | - | - | - | 6 | |||||||
Royalty revenue | 53,183 | 36,853 | 84,337 | 67,684 | |||||||
Manufacturing revenue | 301 | 2,363 | 1,661 | 4,351 | |||||||
Total revenues | 186,704 | 213,808 | 396,822 | 434,190 | |||||||
Operating expenses: | |||||||||||
Cost of product sales, excluding amortization of acquired intangible assets | 15,527 | 12,731 | 30,267 | 26,875 | |||||||
Amortization of acquired intangible asset | 2,865 | 47,397 | 54,395 | 86,812 | |||||||
Research and development (1) | 132,169 | 185,874 | 248,298 | 380,998 | |||||||
Selling, general and administrative (2) | 69,500 | 88,449 | 142,772 | 175,363 | |||||||
Change in the fair value of deferred and contingent consideration | 5,100 | (128,900) | 5,000 | (126,500) | |||||||
Intangible asset impairment | - | 217,800 | - | 217,800 | |||||||
Tangible asset impairment and losses (gains) on transactions, net | 1,761 | - | 1,761 | - | |||||||
Total operating expenses | 226,922 | 423,351 | 482,493 | 761,348 | |||||||
Loss from operations | (40,218) | (209,543) | (85,671) | (327,158) | |||||||
Interest expense, net | (43,490) | (29,415) | (84,324) | (56,745) | |||||||
Other (expense) income, net | (2,025) | 1,479 | (434) | 11,434 | |||||||
Loss before income tax (expense) benefit | (85,733) | (237,479) | (170,429) | (372,469) | |||||||
Income tax (expense) benefit | (13,446) | 38,596 | (20,326) | 34,627 | |||||||
Net loss attributable to common stockholders | $ | (99,179) | $ | (198,883) | $ | (190,755) | $ | (337,842) | |||
Weighted-average shares outstanding: | |||||||||||
Basic and diluted (in shares) | 76,725,070 | 74,730,433 | 76,610,598 | 74,232,624 | |||||||
Net loss per share—basic and diluted (in dollars per share) | $ | (1.29) | $ | (2.66) | $ | (2.49) | $ | (4.55) | |||
(1) Research and development reconciliation | |||||||||||
GAAP research and development | $ | 132,169 | $ | 185,874 | $ | 248,298 | $ | 380,998 | |||
Less: share-based compensation expense | 9,428 | 15,529 | 18,395 | 30,842 | |||||||
Non-GAAP research and development | $ | 122,741 | $ | 170,345 | $ | 229,903 | $ | 350,156 | |||
(2) Selling, general and administrative reconciliation | |||||||||||
GAAP selling, general and administrative | $ | 69,500 | $ | 88,449 | $ | 142,772 | $ | 175,363 | |||
Less: share-based compensation expense | 9,815 | 13,842 | 19,226 | 27,344 | |||||||
Non-GAAP selling, general and administrative | $ | 59,685 | $ | 74,607 | $ | 123,546 | $ | 148,019 | |||
PTC Therapeutics, Inc. | |||||
Summary Consolidated Balance Sheets | |||||
(in thousands, except share data) | |||||
June 30, 2024 | December 31, 2023 | ||||
Cash, cash equivalents and marketable securities | $ | 1,093,293 | $ | 876,739 | |
Total Assets | $ | 1,916,355 | $ | 1,895,698 | |
Total debt | $ | 284,806 | $ | 284,213 | |
Total deferred revenue | - | 801 | |||
Total liability for sale of future royalties | 2,084,880 | 1,814,097 | |||
Total liabilities | $ | 2,896,536 | $ | 2,714,253 | |
Total stockholders' deficit (76,900,123 and 75,708,889 common | $ | (980,181) | $ | (818,555) | |
Total liabilities and stockholders' deficit | $ | 1,916,355 | $ | 1,895,698 | |
PTC Therapeutics, Inc. | ||
Reconciliation of GAAP Milestone Payments Full Year 2024 | ||
(in millions) | ||
Milestone Payments | ||
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. | |||||
Reconciliation of GAAP to Non-GAAP Projected Full Year 2024 R&D and SG&A Expense | |||||
(In millions) | |||||
Low End of | High End of | ||||
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
EMA: European Medicines Agency
FA: Friedreich Ataxia
FDA:
GAAP: Generally Accepted Accounting Principles
HD: Huntington's Disease
MAA: Marketing Authorization Application
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 patients 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, on Twitter at @PTCBio, and on LinkedIn.
For More Information:
Investors:
Kylie O'Keefe
+1 (908) 300-0691
kokeefe@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 Revenue 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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