PTC Therapeutics, Inc.: Q2 FY2026 earnings call

What management said about Q2 FY2026, from the documents filed with the SEC on 30 July 2026: press release. Guidance figures: 2 raised, 3 kept. The quarter’s numbers.

Key takeaways

Product revenue more than doubled: Total net product revenue across the commercial portfolio was $238.8 million, against $118.3 million a year earlier. Total revenue was $360.5 million, against $178.9 million. Source
Net income against prior-year loss: Net income was $83.5 million, or $0.92 per diluted share, against a net loss of $64.8 million, or $(0.83) per share, a year earlier. Source
Full-year revenue guidance raised: Full-year total revenue and product revenue expectations were both raised for 2026. Figures are listed in the guidance section. Source
Sephience sales rose 21 percent: Sephience net product revenue was $151.3 million, up 21% from the first quarter's $124.6 million. Patients on commercial therapy reached 1,647 at June 30, 2026, against 1,244 at March 31. Source
Non-GAAP R&D expense fell: Non-GAAP R&D expense was $88.6 million, excluding $10.5 million of stock-based compensation, against $104.0 million a year earlier. Source
Votoplam slowed disease at 10 mg: PIVOT-HD 24-month results in Stage 2 participants showed dose-dependent slowing on the cUHDRS scale, reaching 52% at the 10 mg dose level. Source
Cash position rose to $2.2 billion: Cash, cash equivalents and marketable securities were $2,229.1 million at June 30, 2026, up from $1,945.4 million at December 31, 2025. Source
$550 million convertible notes issued: PTC issued $550.0 million of senior convertible notes due 2031 at a 0% coupon, with a conversion price 40% above the stock price at pricing. Total debt was $591.0 million at June 30, 2026. Source

“Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance,”
Matthew B. Klein, M.D., Chief Executive Officer

Guidance

Area	Period	Figure	Against last quarter
Total revenue Source	full year 2026	$1.18 to $1.28 billion	Raised from $1.08 to $1.18 billion
Total net product revenue Source	full year 2026	$850 to $950 million	Raised from $750 to $850 million
GAAP R&D and SG&A expense Source	full year 2026	$775 to $815 million	Kept
Non-GAAP R&D and SG&A expense Source	full year 2026	$680 to $720 million	Kept
Stock-based compensation expense (excluded from non-GAAP expense) Source	full year 2026	$95 million	Kept

What changed from last quarter

Guidance raised: Full-year total revenue and total product revenue guidance were both raised. Source
Guidance kept: GAAP and non-GAAP R&D and SG&A expense guidance were kept at prior-quarter levels. Source
New: PTC303 named as the MSH3 splicing program development candidate, with clinic entry planned for 2027. Source
New: Phase 1 study of PTC612, an oral NLRP3 inhibitor, was initiated in the second quarter. Source
New: Senior convertible notes due 2031 were issued in June 2026, with a 0% coupon. Source
New: Hege Sollie-Zetlmayer was appointed to the Board of Directors. Source

Results and growth drivers

Product revenue more than doubled: Total net product revenue across the commercial portfolio was $238.8 million, against $118.3 million a year earlier. Total revenue was $360.5 million, against $178.9 million. Source
Net income against prior-year loss: Net income was $83.5 million, or $0.92 per diluted share, against a net loss of $64.8 million, or $(0.83) per share, a year earlier. Source
Full-year revenue guidance raised: Full-year total revenue and product revenue expectations were both raised for 2026. Figures are listed in the guidance section. Source

Products and customers

Sephience sales rose 21 percent: Sephience net product revenue was $151.3 million, up 21% from the first quarter's $124.6 million. Patients on commercial therapy reached 1,647 at June 30, 2026, against 1,244 at March 31. Source
Sephience growth shifts beyond the US: Sephience revenue was mostly from the US, with contributions from outside the US increasing. The company said the global launch continues. Source
Translarna and Emflaza revenue declined: Translarna net product revenue was $42.2 million, against $59.5 million a year earlier. Emflaza was $24.6 million, against $36.4 million, due to continued generic erosion. Source
Evrysdi royalties grew: Roche reported Evrysdi sales of approximately 968 CHF million year to date. PTC's royalty was $71.1 million for the quarter, against $57.6 million a year earlier. Source

“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.”
Matthew B. Klein, M.D., Chief Executive Officer

Costs and margins

Non-GAAP R&D expense fell: Non-GAAP R&D expense was $88.6 million, excluding $10.5 million of stock-based compensation, against $104.0 million a year earlier. Source
GAAP R&D expense declined: GAAP R&D expense was $99.2 million, against $113.0 million in the second quarter of 2025. Source
SG&A expense lower year over year: GAAP SG&A expense was $80.6 million, against $85.3 million. Non-GAAP SG&A expense was $67.9 million, excluding $12.7 million of stock-based compensation, against $75.7 million. Source

Pipeline and partnered programs

Votoplam slowed disease at 10 mg: PIVOT-HD 24-month results in Stage 2 participants showed dose-dependent slowing on the cUHDRS scale, reaching 52% at the 10 mg dose level. Source
Novartis-funded Phase 3 enrolling: Novartis conducts and funds the INVEST-HD Phase 3 study, targeting about 770 people with early symptomatic disease. Its initiation triggered a $50 million milestone payment to PTC in the second quarter. Source
PTC303 named as development candidate: PTC303 was named as the MSH3 splicing program development candidate, targeting Huntington's disease and myotonic dystrophy. Source
PTC612 Phase 1 started: The oral NLRP3 inhibitor PTC612 entered a Phase 1 study in the second quarter of 2026. Source
Vatiquinone registration study planned: An open-label PROVE-FA study is expected to start in Q3 2026 to support the NDA resubmission for Friedreich's ataxia, enrolling about 120 people aged 7 to 21 with a matched natural history control group. Source
PTC844 Phase 2a expected: A Phase 2a study of PTC844, a next-generation DHODH inhibitor, is expected to start in Q3 2026. Source

“In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule.”
Matthew B. Klein, M.D., Chief Executive Officer

Capital, debt and cash

Cash position rose to $2.2 billion: Cash, cash equivalents and marketable securities were $2,229.1 million at June 30, 2026, up from $1,945.4 million at December 31, 2025. Source
$550 million convertible notes issued: PTC issued $550.0 million of senior convertible notes due 2031 at a 0% coupon, with a conversion price 40% above the stock price at pricing. Total debt was $591.0 million at June 30, 2026. Source
Most 2026 convertible notes repurchased: Alongside the new issue, PTC repurchased the majority of its 1.5% senior convertible notes due September 2026. Source
Shares outstanding reached 83.3 million: Shares issued and outstanding were 83,327,286 at June 30, 2026, against 81,474,366 at December 31, 2025. Source

Corporate and board changes

Former HR chief joins board: Hege Sollie-Zetlmayer was appointed to the Board of Directors. Source
Director has operations and HR background: The new director has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. Source
Retired as chief HR officer: She most recently served as PTC's Chief Human Resources Officer and retired in June 2026. Source

Coming up, as management said on 30 July 2026

Vatiquinone study start: The PROVE-FA study is expected to start in Q3 2026. Source
PTC844 Phase 2a start: The Phase 2a study of PTC844 is expected to start in Q3 2026. Source
FDA engagement on PIVOT-HD: PTC plans to engage with the FDA in the second half of 2026 on the PIVOT-HD 24-month results, which are being finalized. Source
PTC303 clinic entry: PTC303 is planned to enter the clinic in 2027. Source

Figures management reported

Figure	Reported
Sephience net product revenue, Q2 2026 Source	$151.3 million
Translarna net product revenue, Q2 2026 Source	$42.2 million (Q2 2025: $59.5 million)
Emflaza net product revenue, Q2 2026 Source	$24.6 million (Q2 2025: $36.4 million)
Evrysdi royalty revenue to PTC, Q2 2026 Source	$71.1 million
Evrysdi sales reported by Roche, year to date Source	approximately 968 CHF million
GAAP R&D expense, Q2 2026 Source	$99.2 million (Q2 2025: $113.0 million)
Non-GAAP R&D expense, Q2 2026 Source	$88.6 million (Q2 2025: $104.0 million)
Votoplam cUHDRS slowing at 10 mg, PIVOT-HD 24-month Source	52%
INVEST-HD target enrollment Source	approximately 770 individuals
Vatiquinone PROVE-FA planned enrollment Source	approximately 120 individuals

Risks management discussed

External: Emflaza net product revenue fell year over year, with management citing continued generic erosion. Source

Earlier plans management reported on

Not stated: The second-quarter release restates the Q3 2026 start for the open-label PROVE-FA study. Source
Not stated: The first-quarter release had said the INVEST-HD start would trigger a milestone in Q2 2026; the second-quarter release says the $50.0 million was recorded in Q2. Source
Not stated: The first-quarter release said PTC and Novartis would complete data review and align on regulatory interactions; the second-quarter release gives a plan to engage FDA in the second half of 2026. Source

Other quarters

Summarised with AI from the documents the company filed with the SEC. Every point is checked against the filing before it is shown: management’s own words word for word, figures against the line or table row they come from. A point that fails the check is left out. Source opens the filing at that line.

As filed with the SEC

Open the filing on EDGAR

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 - 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

Q2 2026 total revenue of $361 million, including $239 million of product revenue
Sephience global launch continues with strong momentum

oQ2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally o1,647 patients on commercial therapy worldwide as of June 30, 2026

Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing

oPIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level oPlan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized oINVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease oINVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026

Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich’s ataxia expected to initiate in Q3 2026

oGlobal study will enroll approximately 120 individuals with Friedreich’s ataxia age 7 to 21 oStudy includes open-label treatment arm with matched natural history control group

PTC pipeline continues to advance

oPTC303 named as MSH3 splicing program development candidate targeting Huntington’s disease and myotonic dystrophy with plan to enter clinic in 2027 oPhase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 oPhase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026

Hege Sollie-Zetlmayer has been appointed to PTC’s Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC’s Chief Human Resources Officer prior to her retirement in June 2026.

Second Quarter 2026 Financial Highlights

Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025.
Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase.
Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026.
Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025.
Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion.
Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025.
In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue.
Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025.
Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025.
Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense.
Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025.
In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock’s closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026.
Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025.
Shares issued and outstanding as of June 30, 2026, were 83,327,286.

PTC Updates Full-Year 2026 Financial Guidance

Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million
GAAP R&D and SG&A expense guidance remains $775 to $815 million
Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million

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 ecavaleri@ptcbio.com

Media: Jeanine Clemente +1 (908) 912-9406 jclemente@ptcbio.com

Forward-Looking Statements:

Forward-looking statements notice

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.

Forward-looking statements notice

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.