PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results
– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue –
– Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million –
– Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion –
– Strong cash position of $2.2 billion as of June 30, 2026 –
WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026.
"Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule."
Key Corporate Updates
Second 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 June
30,
Six Months Ended June
30,
2026
2025
2026
2025
Revenues:
Net product revenue
$
238,819
$
118,329
$
464,392
$
271,755
Collaboration and license revenue
50,595
2,941
50,738
989,172
Royalty revenue
71,105
57,605
117,940
94,044
Total revenues
360,519
178,875
633,070
1,354,971
Operating expenses:
Cost of product, collaboration and license sales, excluding amortization of acquired
intangible assets
19,921
11,420
47,949
24,282
Amortization of acquired intangible assets
11,841
4,061
23,422
7,859
Research and development (1)
99,150
112,990
200,023
221,963
Selling, general and administrative (2)
80,630
85,262
166,813
166,223
Change in the fair value of contingent consideration
-
-
-
(800)
Tangible asset impairment and losses on transactions, net
-
99
927
176
Total operating expenses
211,542
213,832
439,134
419,703
Income (loss) from operations
148,977
(34,957)
193,936
935,268
Interest expense, net
(48,481)
(30,358)
(97,511)
(64,450)
Other expense, net
(2,951)
(5,737)
(1,342)
(12,042)
Income (loss) before income tax (expense) benefit
97,545
(71,052)
95,083
858,776
Income tax (expense) benefit
(14,049)
6,203
(14,396)
(57,063)
Net income (loss) attributable to common stockholders
$
83,496
$
(64,849)
$
80,687
$
801,713
Weighted-average shares outstanding:
Basic (in shares)
83,006,808
78,151,240
82,765,248
78,438,830
Diluted (in shares)
92,020,009
78,151,240
91,824,273
86,502,578
Net income (loss) per share—basic (in dollars per share)
$
1.01
$
(0.83)
$
0.97
$
10.22
Net income (loss) per share—diluted (in dollars per share)
$
0.92
$
(0.83)
$
0.90
$
9.29
(1) Research and development reconciliation
GAAP research and development
$
99,150
$
112,990
$
200,023
$
221,963
Less: share-based compensation expense
10,547
9,030
21,677
17,693
Non-GAAP research and development
$
88,603
$
103,960
$
178,346
$
204,270
(2) Selling, general and administrative reconciliation
GAAP selling, general and administrative
$
80,630
$
85,262
$
166,813
$
166,223
Less: share-based compensation expense
12,744
9,513
25,035
18,910
Non-GAAP selling, general and administrative
$
67,886
$
75,749
$
141,778
$
147,313
PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(in thousands, except share data)
June 30, 2026
December 31, 2025
Cash, cash equivalents and marketable securities
$
2,229,082
$
1,945,371
Total Assets
$
3,250,477
$
2,898,767
Total debt
$
590,970
$
286,631
Total deferred revenue
1,192
2,040
Total liability for sale of future royalties
2,321,434
2,308,366
Total liabilities
$
3,415,840
$
3,104,080
Total stockholders' deficit (83,327,286 and 81,474,366 common
shares issued and outstanding at June 30, 2026, and
December 31, 2025, respectively)
$
(165,363)
$
(205,313)
Total liabilities and stockholders' deficit
$
3,250,477
$
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
95
95
Projected non-GAAP R&D and SG&A expense
$
680
$
720
Acronyms
CHF: Confoederatio Helvetica Francs (Swiss francs)
cUHDRS: Composite Unified Huntington's Disease Rating Scale
DMD: Duchenne muscular dystrophy
FA: Friedreich's ataxia
FDA: US Food and Drug Administration
GAAP: Generally Accepted Accounting Principles
HD: Huntington's disease
NDA: New Drug Application
nmDMD: Nonsense mutation Duchenne muscular dystrophy
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) 618-8228
[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 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.