PTC Therapeutics Provides Corporate Update and Reports Fourth Quarter and Full Year 2025 Financial Results

– Full-year 2025 product and royalty revenue of $831M, exceeding guidance –

– Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively –

– Cash of $1.95B as of December 31, 2025 –

WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025.

"We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs, and strong financial position, we look forward to continued success as we approach cash flow breakeven."

Key Corporate Highlights

Fourth Quarter and Full Year 2025 Financial Highlights

Full Year 2026 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 accordance with 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,

2025

2024

2025

2024

Revenues:

Net product revenue

183,992

150,088

586,703

582,145

Collaboration and license revenue

(73)

304

998,357

304

Royalty revenue

79,387

58,162

244,224

203,864

Translarna France*

(98,629)

4,618

(98,629)

18,806

Manufacturing revenue

1,661

Total revenues

164,677

213,172

1,730,655

806,780

Operating expenses:

Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets

6,950

16,283

47,013

57,398

Amortization of acquired intangible assets

9,349

3,307

24,742

60,738

Research and development (1)

133,128

124,770

455,249

534,480

Selling, general and administrative (2)

96,874

84,683

347,143

300,911

Change in the fair value of contingent consideration

(10,175)

(800)

(4,475)

Intangible asset impairment

159,548

Tangible asset impairment and (gains) losses on transactions, net

(10,275)

(2,855)

(9,627)

750

Total operating expenses

236,026

375,561

863,720

1,109,350

(Loss) income from operations

(71,349)

(162,389)

866,935

(302,570)

Interest expense, net

(55,188)

(41,060)

(152,230)

(166,993)

Other (expense) income, net

(12,546)

8,850

(18,086)

6,544

Gain on sale of priority review voucher

99,900

(Loss) income before income tax benefit (expense)

(139,083)

(94,699)

696,619

(363,119)

Income tax benefit (expense)

4,118

28,813

(13,975)

(176)

Net (loss) income attributable to common stockholders

(134,965)

(65,886)

682,644

(363,295)

Weighted-average shares outstanding:

Basic (in shares)

80,689,810

77,201,783

79,534,290

76,845,055

Diluted (in shares)

80,689,810

77,201,783

88,311,494

76,845,055

Net (loss) income per share—basic (in dollars per share)

(1.67)

(0.85)

8.58

(4.73)

Net (loss) income per share—diluted (in dollars per share)

(1.67)

(0.85)

7.78

(4.73)

(1) Research and development reconciliation

GAAP research and development

133,128

124,770

455,249

534,480

Less: share-based compensation expense

8,843

8,818

35,669

36,629

Non-GAAP research and development

124,285

115,952

419,580

497,851

(2) Selling, general and administrative reconciliation

GAAP selling, general and administrative

96,874

84,683

347,143

300,911

Less: share-based compensation expense

9,675

8,420

38,878

37,986

Non-GAAP selling, general and administrative

87,199

76,263

308,265

262,925

*In the fourth quarter of 2025, PTC changed its estimates for its sales allowance related to Translarna revenues in France. The $98.6 million change in sales allowance estimate represents a life-to-date adjustment for the historical sales of Translarna in France. The 2024 amounts relate to historical Translarna sales recorded in France.

PTC Therapeutics, Inc.

Summary Consolidated Balance Sheets

(in thousands, except share data)

December 31, 2025

December 31, 2024

Cash, cash equivalents and marketable securities

1,945,371

1,139,696

Total assets

2,898,767

1,705,024

Total debt

286,631

285,412

Total deferred revenue

2,040

5,505

Total liability for sale of future royalties

2,308,366

2,081,776

Total liabilities

3,104,080

2,803,095

Total stockholders' deficit (81,474,366 and 77,704,188 common shares issued

and outstanding at December 31, 2025 and December 31, 2024, respectively)

(205,313)

(1,098,071)

Total liabilities and stockholders' deficit

2,898,767

1,705,024

PTC Therapeutics, Inc.

Reconciliation of GAAP to Non-GAAP Projected Full Year 2026 R&D and SG&A Expense

(in millions)

Low End of Range

High End of Range

Projected GAAP R&D and SG&A Expense

775

815

Less: projected non-cash, stock-based compensation expense

Projected non-GAAP R&D and SG&A expense

680

720

Acronyms

CHF: Confoederatio Helvetica Francs (Swiss francs)

DMD: Duchenne muscular dystrophy

FA: Friedreich's ataxia

FDA: U.S. Food and Drug Administration

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. 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 after completion of the call and will be archived on the company's website.

About PTC Therapeutics, Inc.

PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit www.ptcbio.com and follow on LinkedIn, X, Instagram and Facebook.

For more Information

Investors

Ellen Cavaleri

+1 (615) 618-6228

[email protected]

Media

Jeanine Clemente

+1 (908) 912-9406

[email protected]

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 "Full Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and (ii) 2026 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, meetings with regulatory agencies, 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," "aim," 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; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the U.S. on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; 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 vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; 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 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 Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra.

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.