PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2026 Financial Results

– Strong Q3 2026 performance with total revenue of $358 million, including $276 million of product revenue –

– Full-year 2026 expected total revenue increased to $1.29 to $1.32 billion, with full-year product revenue guidance raised to $960 to $990 million –

– Sephience™ (sepiapterin) Q3 2026 revenue of $182 million, with continued growth in the US and ongoing global expansion –

– Acquired ST-920 Fabry disease gene therapy; BLA submission expected to be completed in Q4 2026 –

WARREN, N.J., Oct. 29, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ended September 30, 2026.

"We delivered another strong quarter of record product revenue led by the continued momentum of the Sephience launch, putting us firmly on track to achieve our cash flow breakeven milestone this year," said Matthew B. Klein, M.D., Chief Executive Officer. "Now at the one-year mark, the launch has been a success by all measures. With a strong patient base, the large global market, and the reports of meaningful treatment benefits, we look forward to continued growth both in the US and internationally."

Key Corporate Updates

Third Quarter 2026 Financial Highlights

PTC Updates 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 September

30,

Nine Months Ended September

30,

2026

2025

2026

2025

Revenues:

Net product revenue

275,894

130,956

740,286

402,711

Collaboration and license revenue

229

9,258

50,967

998,430

Royalty revenue

81,684

70,793

199,624

164,837

Total revenues

357,807

211,007

990,877

1,565,978

Operating expenses:

Cost of product, collaboration and license sales, excluding

amortization of acquired intangible assets

23,664

15,781

71,613

40,063

Amortization of acquired intangible assets

12,595

7,534

36,017

15,393

Research and development (1)

105,353

100,158

305,376

322,121

Selling, general and administrative (2)

80,081

84,046

246,894

250,269

Change in the fair value of contingent consideration

(800)

Tangible asset impairment and losses on transactions, net

472

927

648

Acquired in-process research and development *

111,000

Total operating expenses

332,693

207,991

771,827

627,694

Income from operations

25,114

3,016

219,050

938,284

Interest expense, net

(45,866)

(32,592)

(143,377)

(97,042)

Other (expense) income, net

(1,073)

6,502

(2,415)

(5,540)

Loss on extinguishment of debt

(19,263)

(Loss) income before income tax (expense) benefit

(41,088)

(23,074)

53,995

835,702

Income tax (expense) benefit

(10,605)

38,970

(25,001)

(18,093)

Net (loss) income attributable to common stockholders

(51,693)

15,896

28,994

817,609

Weighted-average shares outstanding:

Basic (in shares)

83,476,100

78,471,119

83,004,625

78,642,478

Diluted (in shares)

83,476,100

87,115,369

92,253,675

86,899,727

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

(0.62)

0.20

0.35

10.40

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

(0.62)

0.20

0.34

9.45

(1) Research and development reconciliation

GAAP research and development

105,353

100,158

305,376

322,121

Less: share-based compensation expense

10,479

9,133

32,155

26,827

Non-GAAP research and development

94,874

91,025

273,221

295,294

(2) Selling, general and administrative reconciliation

GAAP selling, general and administrative

80,081

84,046

246,894

250,269

Less: share-based compensation expense

13,348

10,293

38,384

29,202

Non-GAAP selling, general and administrative

66,733

73,753

208,510

221,067

* In Q3 2026, the Company purchased the ST-920 asset from Sangamo Therapeutics, Inc. and paid an upfront cash consideration of $111.0 million which was recorded as Acquired in-process research and development expense.

PTC Therapeutics, Inc.

Summary Consolidated Balance Sheets

(in thousands, except share data)

September 30,

2026

December 31,

2025

Cash, cash equivalents and marketable securities

2,127,918

1,945,371

Total Assets

3,207,715

2,898,767

Total debt

536,268

286,631

Total deferred revenue

1,975

2,040

Total liability for sale of future royalties

2,305,603

2,308,366

Total liabilities

3,398,924

3,104,080

Total stockholders' deficit (83,535,767 and 81,474,366 common shares

issued and outstanding at September 30, 2026 and December 31, 2025,

respectively)

(191,209)

(205,313)

Total liabilities and stockholders' deficit

3,207,715

2,898,767

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

AAV: adeno-associated virus

BLA: Biologics License Application

CHF: Confoederatio Helvetica Francs (Swiss francs)

DHODH: dihydroorotate dehydrogenase

FA: Friedreich's ataxia

FDA: US Food and Drug Administration

GAAP: Generally Accepted Accounting Principles

HD: Huntington's disease

MSH3: MutS Homolog 3 (human gene)

NDA: New Drug Application

NLRP3: NOD-, LRR- and pyrin domain-containing 3 (multi-protein complex)

R&D: Research and Development

SG&A: Selling, General, and Administrative

Today's Conference Call and Webcast Reminder

To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details.

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, Facebook and Instagram.

For more information please contact:

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 "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total 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 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 US 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 ST-920, including the expected timing of regulatory submissions and potential approvals, and the potential achievement of regulatory milestones and contingent payments PTC may be obligated to make; 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; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; 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.