PTCT

EX-99.1

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EX-99.1

Exhibit 99.1

​

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., October 29, 2026 – 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

·

Q3 2026 total revenue of $358 million, including $276 million of product revenue

·

Sephience launch momentum continues with steady growth in the US and ongoing global expansion

o

Q3 2026 revenue of $182 million, including $145 million revenue in the US and $37 million revenue ex-US, up over 20% from Q2 2026

o

Over 2,000 active patients worldwide as of September 30, 2026

·

ST-920 Fabry disease BLA submission expected to be completed in Q4 2026

o

ST-920 is a one-time administered AAV gene therapy with the potential to reduce the burden associated with chronic enzyme replacement therapy

o

Potential FDA accelerated approval and commercial launch in 2027

·

Votoplam Huntington’s disease program continues to advance

o

Due to FDA scheduling, Type C meeting to discuss votoplam Phase 2 data and potential accelerated approval path now scheduled for Q1 2027

o

Global Phase 3 INVEST-HD study actively enrolling at sites worldwide with target enrollment of approximately 770 early symptomatic patients

·

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

o

Global study will enroll approximately 120 patients ages 7 to 21

o

Study includes open-label treatment arm with matched natural history control arm

·

PTC pipeline advances across multiple programs

o

Initiated Phase 2a study of PTC844, a next-generation DHODH inhibitor

o

Ongoing Phase 1 study of PTC612, an oral NLRP3 inhibitor, continues to advance

o

PTC303, a splicing development candidate targeting MSH3, expected to enter clinic in 2027

​

Third Quarter 2026 Financial Highlights

·

Total net product revenue and royalty revenue was $357.6 million for the third quarter of 2026, compared to $201.7 million for the third quarter of 2025.

·

Total net product revenue across the commercial portfolio was $275.9 million for the third quarter of 2026, compared to $131.0 million for the third quarter of 2025, representing an over 100% increase.

·

Sephience net product revenues were $181.9 million for the third quarter of 2026, representing 20% growth compared to the second quarter of 2026.

·

Translarna™ (ataluren) net product revenues were $48.5 million for the third quarter of 2026, compared to $50.7 million for the third quarter of 2025. Translarna third quarter 2026 revenue includes a government purchase order from Russia.

·

Emflaza® (deflazacort) net product revenues were $25.1 million for the third quarter of 2026, compared to $35.2 million for the third quarter of 2025.

·

Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately CHF 1,470 million, resulting in royalty revenue of $81.7 million to PTC for the third quarter of 2026, compared to $70.8 million to PTC for the third quarter of 2025.

·

Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $105.4 million for the third quarter of 2026, compared to $100.2 million for the third quarter of 2025.

·

Non-GAAP R&D expenses were $94.9 million for the third quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $91.0 million for the third quarter of 2025, excluding $9.1 million in non-cash, stock-based compensation expense.

·

GAAP SG&A expenses were $80.1 million for the third quarter of 2026, compared to $84.0 million for the third quarter of 2025.

·

Non-GAAP SG&A expenses were $66.7 million for the third quarter of 2026, excluding $13.3 million in non-cash, stock-based compensation expense, compared to $73.8 million for the third quarter of 2025, excluding $10.3 million in non-cash, stock-base

EX-99.1

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EX-99.1

Exhibit 99.1

​

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

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