1. PTC reports $190 million in strong revenue for Q1 2025. 2. Positive CHMP opinion for Sephience supports global launch strategy. 3. Cash position of over $2 billion ensures R&D and commercial activities. 4. Revised revenue guidance shows increased confidence in financial outlook. 5. NDA for Sephience targets July 29, 2025, potential market impact.
Strong revenue performance and positive CHMP opinion enhance growth potential. Examples include significant price rises after past approvals.
Strong quarterly results and positive feedback for a new drug indicate potential price increase.
Upcoming PDUFA date for Sephience may lead to immediate market movements.
WARREN, N.J., May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2025.
"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."
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.
(In thousands, except share and per share data)
| Revenues: | 2025 | 2024 |
|---|---|---|
| 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 |
| Description | 2025 | 2024 |
|---|---|---|
| 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) |
| Net income (loss) attributable to common stockholders | $866,562 | $ (91,576) |
| 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 |
| 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 |
(in thousands, except share data)
| 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 | $(185,760) | $(1,098,071) |
| Total liabilities and stockholders' deficit | $2,655,387 | $1,705,024 |
| Low End of Range | Projected GAAP R&D and SG&A Expense | $805 |
| High End of Range | $835 | |
| Less: projected non-cash, stock-based compensation expense | 75 | |
| Projected non-GAAP R&D and SG&A expense | $730 | $760 |
CHF: Confoederatio Helvetica Francs (Swiss francs), CHMP: Committee for Medicinal Products for Human Use, DMD: Duchenne Muscular Dystrophy, EC: European Commission, FDA: U.S. Food and Drug Administration, 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
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 here. 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.
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.
Investors: Ellen Cavaleri
+1 (615) 618-8228
[email protected]
Media: Jeanine Clemente
+1 (908) 912-9406
[email protected]
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 Brazil, Russia and other regions; the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna on other regulatory bodies; 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 Sephience, 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, 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.
SOURCE PTC Therapeutics, Inc.