Full Press Release Details
PTC Therapeutics Provides Corporate Update and
Reports Fourth Quarter and Full Year 2024 Financial Results
- Full year 2024 revenue of $807 million, exceeding guidance -
- All 2024 clinical and regulatory milestones were achieved on schedule, including four NDA submissions, all of which were accepted for filing -
- License and collaboration agreement with Novartis for PTC518 Huntington's disease program closed in January 2025 -
- Cash of over $2.0 billion as of January 2025 -
WARREN, N.J., February 27, 2025 - PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter ending December 31, 2024.
"Our strong fourth quarter rounds out a year of significant accomplishment across every part of our company," said Matthew B. Klein, M.D., Chief Executive Officer. "In 2024, our commercial team delivered another outstanding performance, we achieved all clinical and regulatory milestones on schedule and we solidified our balance sheet. We now have over $2 billion in cash to support our planned commercial and R&D activities in 2025 and beyond. With the many accomplishments of 2024 and our team's demonstrated ability to execute across every part of the business, we have built a strong foundation for continued success."
Key Corporate Updates:
Key Clinical and Regulatory Milestones:
Fourth Quarter and Full Year 2024 Financial Highlights:
PTC Full Year 2025 Financial Guidance:
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 December 31, | Twelve Months Ended December 31, | ||||||||||
| 2024 | 2023 | 2024 | 2023 | ||||||||
| Revenues: | |||||||||||
| Net product revenue | 154,706 | $ | 155,062 | $ | 600,951 | $ | 661,249 | ||||
| Collaboration revenue | 304 | 100,024 | 304 | 100,030 | |||||||
| Royalty revenue | 58,162 | 50,999 | 203,864 | 168,856 | |||||||
| Manufacturing revenue | - | 971 | 1,661 | 7,687 | |||||||
| Total revenues | 213,172 | 307,056 | 806,780 | 937,822 | |||||||
| Operating expenses: | |||||||||||
| Cost of product sales, excluding amortization of acquired intangible assets | 16,283 | 29,118 | 57,398 | 65,486 | |||||||
| Amortization of acquired intangible asset | 3,307 | 77,174 | 60,738 | 222,635 | |||||||
| Research and development (1) | 124,770 | 121,353 | 534,480 | 666,563 | |||||||
| Selling, general and administrative (2) | 84,683 | 76,291 | 300,911 | 332,540 | |||||||
| Change in the fair value of contingent consideration | (10,175) | (2,700) | (4,475) | (127,700) | |||||||
| Intangible asset impairment | 159,548 | - | 159,548 | 217,800 | |||||||
| Tangible asset impairment and losses (gains) on transactions, net | (2,855) | - | 750 | - | |||||||
| Total operating expenses | 375,561 | 301,236 | 1,109,350 | 1,377,324 | |||||||
| (Loss) income from operations | (162,389) | 5,820 | (302,570) | (439,502) | |||||||
| Interest expense, net | (41,060) | (44,274) | (166,993) | (129,180) | |||||||
| Other income, net | 8,850 | 18,961 | 6,544 | 10,130 | |||||||
| Gain on Sale of priority review voucher | 99,900 | - | 99,900 | - | |||||||
| Loss on extinguishment of debt | - | (137,558) | - | (137,558) | |||||||
| Loss before income tax benefit (expense) | (94,699) | (157,051) | (363,119) | (696,110) | |||||||
| Income tax benefit (expense) | 28,813 | 1,259 | (176) | 69,506 | |||||||
| Net loss attributable to common stockholders | $ | (65,886) | $ | (155,792) | $ | (363,295) | $ | (626,604) | |||
| Weighted-average shares outstanding: | |||||||||||
| Basic and diluted (in shares) | 77,201,783 | 75,490,569 | 76,845,055 | 74,838,392 | |||||||
| Net loss per share-basic and diluted (in dollars per share) | $ | (0.85) | $ | (2.06) | $ | (4.73) | $ | (8.37) | |||
| (1) Research and development reconciliation | |||||||||||
| GAAP research and development | $ | 124,770 | $ | 121,353 | $ | 534,480 | $ | 666,563 | |||
| Less: share-based compensation expense | 8,818 | 8,113 | 36,629 | 52,941 | |||||||
| Non-GAAP research and development | $ | 115,952 | $ | 113,240 | $ | 497,851 | $ | 613,622 | |||
| (2) Selling, general and administrative reconciliation | |||||||||||
| GAAP selling, general and administrative | $ | 84,683 | $ | 76,291 | $ | 300,911 | $ | 332,540 | |||
| Less: share-based compensation expense | 8,420 | 8,395 | 37,986 | 48,695 | |||||||
| Non-GAAP selling, general and administrative | $ | 76,263 | $ | 67,896 | $ | 262,925 | $ | 283,845 |
PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(in thousands, except share data)
| December 31, 2024 | December 31, 2023 | ||||
| Cash, cash equivalents and marketable securities | $ | 1,139,696 | $ | 876,739 | |
| Total Assets | $ | 1,705,024 | $ | 1,895,698 | |
| Total debt | $ | 285,412 | $ | 284,213 | |
| Total deferred revenue | 5,505 | 801 | |||
| Total liability for sale of future royalties | 2,081,776 | 1,814,097 | |||
| Total liabilities | $ | 2,803,095 | $ | 2,714,253 | |
| Total stockholders' deficit (77,704,188 and 75,708,889 common shares issued and outstanding at December 31, 2024 and December 31, 2023, respectively) | $ | (1,098,071) | $ | (818,555) | |
| Total liabilities and stockholders' deficit | $ | 1,705,024 | $ | 1,895,698 |
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2025 R&D and 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 |
AADC: Aromatic L-Amino Acid Decarboxylase
CHF: Confoederatio Helvetica Francs (Swiss francs)
CHMP: Committee for Medicinal Products for Human Use
DMD: Duchenne Muscular Dystrophy
FA: Friedreich's Ataxia
FDA: U.S. Food and Drug Administration
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
PRV: Priority Review Voucher
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.
For More Information:
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," 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 Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Commission adopts the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the conditional marketing authorization for Translarna in the EEA, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to sepiapterin, 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; expectations with respect to vatiquinone, 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; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's most recent Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.
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, sepiapterin 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.