PTC Therapeutics posts Q1 2026 results, raises outlook

PTC Therapeutics, Inc. reported first quarter 2026 total revenue of $272.6 million, including $225.6 million in net product revenue and $46.8 million in royalty revenue.

Rhea-AI Filing Summary

PTC Therapeutics, Inc. reported first quarter 2026 total revenue of $272.6 million, including $225.6 million in net product revenue and $46.8 million in royalty revenue. Management said this strong start supports raising full-year 2026 product revenue guidance.

Global Sephience sepiapterin revenue reached $125 million in the quarter, reflecting 36% quarter-over-quarter growth. The company reported a small net loss of $2.8 million, or $(0.03) per basic and diluted share, compared with a large prior-year profit that included substantial collaboration revenue.

PTC highlighted positive topline results from a 24‑month interim analysis of the PIVOT‑HD votoplam extension study and continued work on the global Phase 3 INVEST‑HD study. An open-label vatiquinone registration study is planned to start in Q3 2026. Cash, cash equivalents and marketable securities were $1.89 billion as of March 31, 2026.

Positive

• Q1 2026 product and royalty revenue were strong, with net product revenue of $225.6 million and royalty revenue of $46.8 million, supporting management’s decision to raise full-year 2026 product revenue guidance.

Negative

• Capital structure remains stretched, with a $2.33 billion liability for sale of future royalties, $286.9 million of debt, and a stockholders’ deficit of $180.5 million as of March 31, 2026, despite a solid cash balance.

Insights

Biopharma equity analyst

Solid Q1 revenue and Sephience growth support higher 2026 guidance, but leverage and royalty obligations remain key constraints.

PTC Therapeutics generated Q1 2026 revenue of $272.6M, with net product revenue of $225.6M and royalty revenue of $46.8M. The company described this as an “outstanding” quarter that supports a full-year product revenue guidance raise, anchored by Sephience sepiapterin’s 36% quarter-over-quarter growth to $125M.

Despite solid operations, the bottom line showed a modest net loss of $2.8M versus a prior-year profit driven by $986.2M of collaboration and license revenue. Interest expense was material at $49.0M, and the balance sheet includes a liability for sale of future royalties of $2.33B, along with total debt of $286.9M, contributing to stockholders’ deficit.

On the pipeline side, positive topline data from the 24‑month PIVOT‑HD votoplam extension and plans to initiate an open-label vatiquinone registration study in Q3 2026 reinforce late-stage development activity in Huntington’s disease and Friedreich’s ataxia. Projected 2026 GAAP R&D and SG&A expense of $775M–$815M, and non-GAAP of $680M–$720M, signal continued heavy investment funded by a large $1.89B cash position.

8-K Event Classification

3 items: 2.02, 7.01, 9.01

3 items

Item 2.02 Results of Operations and Financial Condition Financial

Disclosure of earnings results, typically an earnings press release or preliminary financials.

Item 7.01 Regulation FD Disclosure Disclosure

Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.

Item 9.01 Financial Statements and Exhibits Exhibits

Financial statements, pro forma financial information, or exhibit attachments filed with this report.

Key Figures

Q1 2026 total revenues: $272.6M Q1 2026 net product revenue: $225.6M Q1 2026 Sephience revenue: $125M +5 more

8 metrics

Q1 2026 total revenues $272.6M Three months ended March 31, 2026 total revenues of $272,551,000

Q1 2026 net product revenue $225.6M Net product revenue for three months ended March 31, 2026 was $225,573,000

Q1 2026 Sephience revenue $125M Global Sephience sepiapterin launch revenue of $125 million with 36% QoQ growth

Q1 2026 net (loss) income -$2.8M Net loss attributable to common stockholders was $2,809,000 for Q1 2026

Q1 2026 EPS basic - $0.03/share Net loss per share—basic was $(0.03) for three months ended March 31, 2026

Cash, cash equivalents and marketable securities $1.89B Cash, cash equivalents and marketable securities were $1,892,463,000 at March 31, 2026

Liability for sale of future royalties $2.33B Total liability for sale of future royalties was $2,327,230,000 at March 31, 2026

Projected 2026 non-GAAP R&D and SG&A $680M–$720M Projected full-year 2026 non-GAAP R&D and SG&A expense range

Key Terms

non-GAAP financial measures, liability for sale of future royalties, Phase 3 INVEST-HD study, registration study, +2 more

6 terms

non-GAAP financial measures financial

"In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures."

Non-GAAP financial measures are numbers companies use to show their financial performance that exclude certain expenses or income. They help investors see how the company might perform without one-time costs or other unusual items, giving a different perspective from official reports. However, since they can be adjusted, they don’t always tell the full story and should be looked at alongside standard financial figures.

liability for sale of future royalties financial

"Total liability for sale of future royalties | ​ | 2,327,230 | ​ | ​ | 2,308,366"

Phase 3 INVEST-HD study medical

"supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions"

registration study medical

"Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback"

A registration study is a late-stage clinical trial designed to prove a drug or medical device is safe and effective enough for regulatory approval. Think of it as the final exam before a product can be sold: its results directly determine whether authorities will grant market authorization, which in turn can make or break future sales, revenue forecasts and investment decisions. Investors watch these studies for clear go/no-go signals.

Composite Unified Huntington's Disease Rating Scale medical

"cUHDRS: Composite Unified Huntington's Disease Rating Scale"

A composite Unified Huntington’s Disease Rating Scale is a single numerical score that combines tests of movement, thinking, behavior and daily function to summarize how advanced Huntington’s disease is for a patient. Investors watch it because drug developers use changes in this score as a clear, measurable sign that a therapy is helping or failing—think of it as a car’s dashboard light that bundles several warning indicators into one reading for decision-making.

Non-GAAP research and development financial

"Non-GAAP research and development | $ | 89,744 | ​ | $ | 100,310"

Earnings Snapshot

Total revenue: $272.6M · Guidance included

Q1 2026

Total revenue $272.6M -77% YoY

Net income (loss) -$2.8M vs. $866.6M prior year

EPS basic -$0.03 vs. $11.09 prior year

Net product revenue $225.6M up from $153.4M prior year

Royalty revenue $46.8M up from $36.4M prior year

Guidance

Company stated that strong Q1 2026 revenue performance supports raising full-year 2026 product revenue guidance and projected GAAP and non-GAAP R&D and SG&A expense ranges for 2026.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

How did PTC Therapeutics (PTCT) perform financially in Q1 2026?

PTC Therapeutics reported Q1 2026 total revenue of $272.6 million, including $225.6 million in net product revenue and $46.8 million in royalty revenue. The quarter resulted in a small net loss of $2.8 million, or $(0.03) per basic and diluted share.

What drove PTC Therapeutics’ revenue in the first quarter of 2026?

Q1 2026 revenue of $272.6 million was driven mainly by net product revenue of $225.6 million and royalty revenue of $46.8 million. The Sephience sepiapterin launch contributed $125 million, reflecting 36% quarter-over-quarter growth and forming a key pillar of the company’s top line.

How is the Sephience (sepiapterin) launch progressing for PTC Therapeutics?

The Sephience sepiapterin launch is progressing strongly, generating $125 million in Q1 2026 revenue and delivering 36% quarter-over-quarter growth. Management highlighted sustained momentum in the US and increasing international contributions as more countries participate in the global launch.

What were PTC Therapeutics’ earnings per share in Q1 2026?

For Q1 2026, PTC Therapeutics reported net loss per share of $(0.03) on both a basic and diluted basis. This compares with prior-year earnings per basic share of $11.09, which benefited from substantial one-time collaboration and license revenue of $986.2 million.

What is PTC Therapeutics’ cash and debt position as of March 31, 2026?

As of March 31, 2026, PTC Therapeutics held $1.89 billion in cash, cash equivalents and marketable securities, against $286.9 million of total debt. The company also reported a $2.33 billion liability for sale of future royalties and total stockholders’ deficit of $180.5 million.

What guidance and expense outlook did PTC Therapeutics provide for 2026?

PTC indicated that Q1 2026 performance supports raising full-year 2026 product revenue guidance. It projected full-year 2026 GAAP R&D and SG&A expense of $775–$815 million, and non-GAAP R&D and SG&A expense of $680–$720 million, excluding $95 million of stock-based compensation.

What are the key clinical updates mentioned by PTC Therapeutics in this filing?

PTC reported positive topline results from a 24‑month interim analysis of the PIVOT‑HD votoplam extension study, supporting the ongoing global Phase 3 INVEST‑HD trial. The company also plans to start an open-label vatiquinone registration study in Q3 2026, following FDA feedback on trial design and expectations.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Accepted by SEC EDGAR 05/07/2026 - 04:09 PM Learn about SEC filing dates

0001070081false00010700812026-05-072026-05-07

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UNITED STATES

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SECURITIES AND EXCHANGE COMMISSION

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WASHINGTON, D.C. 20549

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FORM 8-K

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CURRENT REPORT

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Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934

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Date of Report (Date of earliest event reported): May 7, 2026

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PTC THERAPEUTICS, INC.

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(Exact Name of Company as Specified in Charter)

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Delaware	​ ​ ​	001-35969	​ ​ ​	04-3416587
(State or Other Jurisdiction	​	(Commission	​	(IRS Employer
of Incorporation)	​	File Number)	​	Identification No.)

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500 Warren Corporate Center Drive	​	​ ​ ​	​
Warren, NJ	​	​	07059
(Address of Principal Executive Offices)	​	​	(Zip Code)

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Registrant’s telephone number, including area code: (908) 222-7000

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Not applicable

(Former Name or Former Address, if Changed Since Last Report)

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Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

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☐Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

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☐Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

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☐Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

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☐Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

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Securities registered pursuant to Section 12(b) of the Act:

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Title of each class	​ ​ ​	Trading Symbol(s)	​ ​ ​	Name of each exchange on which registered
Common Stock, $0.001 par value per share	​	PTCT	​	Nasdaq Global Select Market

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Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

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Emerging growth company ☐

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If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

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Item 2.02. Results of Operations and Financial Condition.

On May 7, 2026, PTC Therapeutics, Inc. (the “Company”) announced its financial results for the quarter ended March 31, 2026. The full text of the press release issued in connection with the announcement is furnished as Exhibit 99.1 to this Current Report on Form 8-K (this "Report") and is incorporated by reference into this Item 2.02.

Item 7.01. Regulation FD Disclosure.

The Company will host a conference call on May 7, 2026 at 4:30 p.m. eastern time, as previously announced. During this call the Company expects to review financial results for the quarter ended March 31, 2026, as well as other corporate highlights and updates. Instructions on how to access the conference call are included in the press release furnished as Exhibit 99.1 hereto.

The information in this Report (including Items 2.02 and 7.01 and Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing. All website addresses given in this Report or incorporated herein by reference are for information only and are not intended to be an active link or to incorporate any website information into this Report.

Item 9.01. Financial Statements and Exhibits.

(d) Exhibits

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Exhibit No.	​ ​ ​	Description
99.1	​	Press Release, dated May 7, 2026 issued by PTC Therapeutics, Inc.
104	​	The cover page from this Current Report on Form 8-K, formatted in Inline XBRL

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Signature

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this Report to be signed on its behalf by the undersigned hereunto duly authorized.

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​	PTC Therapeutics, Inc.
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Date: May 7, 2026	By:	/s/ Pierre Gravier
​	Name:	Pierre Gravier
​	Title:	Chief Financial Officer

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Exhibit 99.1

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

– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise –

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– Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth –

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– Positive topline results from 24-month interim analysis of PIVOT-HD extension study of votoplam, supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions –

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– Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback –

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WARREN, N.J., May 7, 2026 – PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2026.  

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“We are off to a strong start to 2026 with outstanding revenue performance this quarter that supports raising our full-year product revenue guidance,” said Matthew B. Klein, M.D., Chief Executive Officer. “The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch.”

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Key Corporate Updates

● Q1 2026 total revenue of $273 million, with $226 million of product revenue

● Sephience global launch continues strong momentum

o Q1 2026 revenue of $125 million, including $112 million revenue in the US and $13 million revenue ex-US, up 36% from Q4 2025

o 1,244 total patients on commercial therapy worldwide as of March 31, 2026

o Continued strong cadence of patient start forms in the US, with ~140 per month over past several months, surpassing 1,500 mark in the quarter

o First commercial sales in Japan in Q1 2026, with revenue expected from up to 30 countries by year-end 2026

● Reported positive topline results in April 2026 from 24-month interim analysis of PIVOT-HD long-term extension study of votoplam, an oral small molecule splicing agent

o Dose-dependent benefit on cUHDRS in Stage 2 participants relative to natural history cohort, with 52% slowing of disease progression at 10 mg dose

o Continued favorable safety and tolerability with no treatment-related NfL spikes

o PTC and Novartis will complete data review and align on potential regulatory interactions

o Findings support ongoing global Phase 3 INVEST-HD study of votoplam led by Novartis

o First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026

● Type C meeting with FDA held in April 2026 to discuss vatiquinone study to support NDA resubmission for Friedreich’s ataxia program

o Based on meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026

o Primary endpoint will be change in mFARS from baseline to 24 months

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First Quarter 2026 Financial Highlights

● Total net product revenue and royalty revenue was $272.4 million for the first quarter of 2026, compared to $189.9 million for the first quarter of 2025.

● Total net product revenue across the commercial portfolio was $225.6 million for the first quarter of 2026, compared to $153.4 million for the first quarter of 2025, representing a 47% increase. 

● Sephience net product revenues were $124.6 million for the first quarter of 2026, representing 36% growth compared to fourth quarter of 2025.

● Translarna™ (ataluren) net product revenues were $59.0 million for the first quarter of 2026, compared to $86.2 million for the first quarter of 2025. Translarna first quarter of 2026 revenue includes a large government purchase order from Brazil.

● Emflaza^® (deflazacort) net product revenues were $21.5 million for the first quarter of 2026, compared to $47.8 million for the first quarter of 2025, due to continued generic erosion.

● Roche reported Evrysdi^® (risdiplam) sales of approximately 464 CHF million, resulting in royalty revenue of $46.8 million to PTC for the first quarter of 2026, compared to $36.4 million to PTC for the first quarter of 2025.

● Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $100.9 million for the first quarter of 2026, compared to $109.0 million for the first quarter of 2025.

● Non-GAAP R&D expenses were $89.7 million for the first quarter of 2026, excluding $11.1 million in non-cash, stock-based compensation expense, compared to $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense.

● GAAP SG&A expenses were $86.2 million for the first quarter of 2026, compared to $81.0 million for the first quarter of 2025.

● Non-GAAP SG&A expenses were $73.9 million for the first quarter of 2026, excluding $12.3 million in non-cash, stock-based compensation expense, compared to $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense.

● Net loss was $2.8 million for the first quarter of 2026, compared to net income of $866.6 million for the first quarter of 2025.

● Cash, cash equivalents, and marketable securities were $1,892.5 million on March 31, 2026, compared to $1,945.4 million on December 31, 2025.

● Shares issued and outstanding as of March 31, 2026, were 82,882,024.

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PTC Updates Full-Year 2026 Financial Guidance

● Total product revenue guidance raised to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion

● 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

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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.

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PTC Therapeutics, Inc. Consolidated Statements of Operations
(in thousands, except share and per share data)

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​	Three Months Ended March 31,					​
​	2026		​	2025		​
Revenues:	​		​	​		​
Net product revenue	$	225,573	​	$	153,426	​
Collaboration and license revenue	​	143	​	986,231		​
Royalty revenue	​	46,835	​	36,439		​
Total revenues	272,551		​	1,176,096		​
Operating expenses:	​		​	​		​
Cost of product, collaboration and license sales, excluding amortization of acquired intangible assets	28,028		​	12,862		​
Amortization of acquired intangible assets	11,581		​	3,798		​
Research and development (1)	100,873		​	108,973		​
Selling, general and administrative (2)	86,183		​	80,961		​
Change in the fair value of contingent consideration	-		​	(800)		​
Tangible asset impairment and losses on transactions, net	​	927	​	​	77	​
Total operating expenses	227,592		​	205,871		​
Income from operations	44,959		​	970,225		​
Interest expense, net	(49,030)		​	(34,092)		​
Other income (expense), net	1,609		​	(6,305)		​
(Loss) income before income tax expense	(2,462)		​	929,828		​
Income tax expense	(347)		​	(63,266)		​
Net (loss) income attributable to common stockholders	$	(2,809)	​	$	866,562	​
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Weighted-average shares outstanding:	​		​	​		​
Basic (in shares)	82,521,023		​	78,115,836		​
Diluted (in shares)	82,521,023		​	​	86,385,922	​
Net (loss) income per share—basic (in dollars per share)	$	(0.03)	​	$	11.09	​
Net (loss) income per share—diluted (in dollars per share)	$	(0.03)	​	$	10.04	​
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(1) Research and development reconciliation	​		​	​		​
GAAP research and development	$	100,873	​	$	108,973	​
Less: share-based compensation expense	11,129		​	8,663		​
Non-GAAP research and development	$	89,744	​	$	100,310	​
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(2) Selling, general and administrative reconciliation	​		​	​		​
GAAP selling, general and administrative	$	86,183	​	$	80,961	​
Less: share-based compensation expense	12,292		​	9,397		​
Non-GAAP selling, general and administrative	$	73,891	​	$	71,564	​
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PTC Therapeutics, Inc.
Summary Consolidated Balance Sheets
(in thousands, except share data)

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​	March 31, 2026		​	December 31, 2025
Cash, cash equivalents and marketable securities	$	1,892,463	​	$	1,945,371
Total assets	$	2,869,104	​	$	2,898,767
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Total debt	$	286,937	​	$	286,631
Total deferred revenue	​	498	​	​	2,040
Total liability for sale of future royalties	​	2,327,230	​	​	2,308,366
Total liabilities	$	3,049,569	​	$	3,104,080
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Total stockholders' deficit (82,882,024 and 81,474,366 common shares issued and outstanding at March 31, 2026, and December 31, 2025, respectively)	$	(180,465)	​	$	(205,313)
Total liabilities and stockholders' deficit	$	2,869,104	​	$	2,898,767
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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
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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

mFARS: Modified Friedreich’s Ataxia Rating Scale

NDA: New Drug Application

NfL: Neurofilament light chain

nmDMD: Nonsense mutation Duchenne muscular dystrophy

PKU: Phenylketonuria

R&D: Research and Development

SG&A: Selling, General, and Administrative

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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.

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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.

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For more information please contact:

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Investors:
Ellen Cavaleri +1 (615) 618-8228 ecavaleri@ptcbio.com

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

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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.

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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 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; 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.

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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.

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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.

Filing Exhibits & Attachments

5 documents

Press Releases

• EX-99.1 EX-99.1 171.4 KB

Other Documents

• EX-101 EX-101.SCH 3.3 KB
• EX-101 EX-101.DEF 2.7 KB
• EX-101 EX-101.LAB 16.2 KB
• EX-101 EX-101.PRE 9.4 KB