Earnings summaries and quarterly performance for PTC THERAPEUTICS.
Executive leadership at PTC THERAPEUTICS.
Matthew B. Klein
Detailed
Chief Executive Officer
CEO
CU
Christine Utter
Detailed
Senior Vice President, Chief Accounting Officer and Head of People Services
EP
Eric Pauwels
Detailed
Chief Business Officer
LG
Lee Golden
Detailed
Executive Vice President and Chief Medical Officer
ME
Mark E. Boulding
Detailed
Executive Vice President and Chief Legal Officer
NA
Neil Almstead
Detailed
Chief Technical Operations Officer
PG
Pierre Gravier
Detailed
Chief Financial Officer
Board of directors at PTC THERAPEUTICS.
AY
Alethia Young
Detailed
Director
AJ
Allan Jacobson
Detailed
Director
DP
David P. Southwell
Detailed
Director
ER
Emma Reeve
Detailed
Director
GD
Glenn D. Steele Jr.
Detailed
Director
JB
Jerome B. Zeldis
Detailed
Director
MS
Mary Smith
Detailed
Director
MS
Michael Schmertzler
Detailed
Chair of the Board
SS
Stephanie S. Okey
Detailed
Director
Research analysts who have asked questions during PTC THERAPEUTICS earnings calls.
Brian Abrahams
RBC Capital Markets
4 questions for PTCT
Also covers: ACAD, ATAI, BCRX +17 more
JS
Joseph Schwartz
Oppenheimer
4 questions for PTCT
Also covers: ASND, AUPH, BMRN +14 more
EM
Eliana Merle
UBS
3 questions for PTCT
Also covers: ALNY, APLS, ARVN +17 more
Eric Joseph
JPMorgan Chase & Co.
3 questions for PTCT
Also covers: BEAM, BLUE, ENTA +9 more
Gena Wang
Barclays
3 questions for PTCT
Also covers: ALNY, BCRX, BLUE +12 more
JB
Joel Beatty
Baird
3 questions for PTCT
Also covers: ACAD, ALKS, AMLX +11 more
JP
John Peyton Bohnsack
TD Cowen
3 questions for PTCT
Kelly Shi
Jefferies
3 questions for PTCT
Also covers: ADCT, AGEN, ALDX +17 more
Kristen Kluska
Cantor Fitzgerald
3 questions for PTCT
Also covers: ABEO, ADMA, AQST +24 more
PC
Paul Choi
Goldman Sachs
3 questions for PTCT
Also covers: AMRN, ARVN, ASND +12 more
TA
Tazeen Ahmad
Bank of America
3 questions for PTCT
Also covers: ACAD, ALNY, APLS +20 more
Judah Frommer
Morgan Stanley
2 questions for PTCT
Also covers: APLS, ARQT, ATAI +4 more
SC
Samantha Corwin
William Blair
2 questions for PTCT
Also covers: ALLO, BEAM, KRYS +3 more
BC
Brian Cheng
JPMorgan Chase & Co.
1 question for PTCT
Also covers: ALLO, CHRS, EDIT +6 more
DB
Danielle Brill Bongero
Raymond James
1 question for PTCT
DL
David Lebowitz
Citigroup Inc.
1 question for PTCT
Also covers: ALNY, ARWR, ASND +11 more
DS
Dingding Shi
Jefferies
1 question for PTCT
Also covers: ADCT, ASND, AUTL +6 more
EM
Ellie Merle
UBS Group AG
1 question for PTCT
Also covers: ALNY, ALT, ARVN +12 more
Geoff Meacham
Citigroup Inc.
1 question for PTCT
Also covers: ABBV, AMGN, BMY +8 more
Geoffrey Meacham
Citi
1 question for PTCT
Also covers: ABBV, BIIB, BMY +6 more
HW
Huidong Wang
Barclays
1 question for PTCT
Also covers: ALNY, BCRX, BEAM +15 more
JL
Joon Lee
Truist Securities
1 question for PTCT
Also covers: ADVM, AXSM, CNM +14 more
Joseph Thome
TD Cowen
1 question for PTCT
Also covers: ALKS, AMLX, ARDX +12 more
Khalil Fenina
Goldman Sachs
1 question for PTCT
Also covers: FGEN
Michael Riad
Morgan Stanley
1 question for PTCT
Also covers: ACAD, ALEC, ITCI +3 more
RM
Richard Miller
Cantor Fitzgerald
1 question for PTCT
Also covers: ADMA, QURE
SC
Sami Corwin
William Blair
1 question for PTCT
Also covers: ALLO, BEAM, KRYS +5 more
TF
Tiago Fauth
Wells Fargo
1 question for PTCT
Also covers: LQDA, VRNA, WVE
Recent press releases and 8-K filings for PTCT.
PTC Therapeutics Announces Preliminary 2025 Financial Results and 2026 Guidance
PTCT
Earnings
Guidance Update
Product Launch
- PTC Therapeutics reported unaudited 2025 total product and royalty revenue of approximately $823.4 million, with unaudited Sephience Q4 2025 net revenue at approximately $92.5 million and total 2025 Sephience net revenue of approximately $112.1 million since launch.
- The company provided 2026 product revenue guidance of $700 million to $800 million, which represents 19-36% year-over-year growth.
- Cash, cash equivalents, and marketable securities totaled approximately $1.94 billion as of December 31, 2025.
- PTC Therapeutics plans to initiate a Phase 3 trial for its votoplam Huntington's disease program in the first half of 2026, with the FDA indicating openness to a potential Accelerated Approval pathway.
Jan 12, 2026, 5:00 PM
PTCT Reports Strong 2025 Performance and Provides 2026 Guidance
PTCT
Guidance Update
Product Launch
New Projects/Investments
- PTCT exceeded its 2025 revenue guidance, achieving $823 million in total revenue and ending the year with over $1.94 billion in cash.
- The Safiyance launch demonstrated strong momentum, generating $112 million in total revenue since launch, with $92.5 million in Q4 2025 net revenue, and 946 patients on commercial therapy worldwide as of December 31st.
- For 2026, PTCT provided revenue guidance of $700-$800 million and OpEx guidance of $680-$720 million, positioning the company for potential cash flow breakeven.
- The company advanced its pipeline, including the Vodaplam Huntington's Disease Program with a planned Phase 3 study, and sold the remaining Evrysdi royalty for $240 million in milestones in December 2025.
Jan 12, 2026, 5:00 PM
PTC Therapeutics and BillionToOne Presentations at J.P. Morgan Healthcare Conference
PTCT
Guidance Update
Product Launch
New Projects/Investments
- PTC Therapeutics (PTCT) reported strong 2025 revenue performance and provided 2026 revenue and OpEx guidance, with expectations to reach cash flow breakeven in 2026. Key financial figures are detailed in the table below.
- PTCT's Safiyance launch had a strong start, and the Vodaplam Huntington's Disease program received FDA support for a potential accelerated approval pathway, with a Phase 3 study fully funded by Novartis. Safiyance performance metrics are provided in the table below.
- BillionToOne provided 2025 revenue guidance and 2026 revenue projections, while achieving GAAP profitability in 2025 and expecting to maintain it in 2026. Key financial figures are detailed in the table below.
- BillionToOne is expanding its growth strategy into the Minimal Residual Disease (MRD) market, which represents a significant potential opportunity. Financial details are provided in the table below.
| Metric | PTC Therapeutics | BillionToOne |
|---|---|---|
| Revenue (Millions USD) | ||
| FY 2025 | $823 | $293-$299 (guidance) |
| FY 2026 (Guidance/Projection) | $700-$800 | $415-$430 |
| Operating Expenses (Millions USD) | ||
| FY 2026 (Guidance) | $680-$720 | N/A |
| Safiyance Total Revenue Since Launch (Millions USD) | $112 | N/A |
| Safiyance Patients on Therapy (as of Dec 31st) | 946 | N/A |
| Blended Gross Margin (%) | N/A | 70% |
| MRD Market Opportunity (Billions USD) | N/A | $30 |
Jan 12, 2026, 5:00 PM
PTC Therapeutics and BillionToOne Present 2025 Performance and 2026 Outlook
PTCT
Guidance Update
Product Launch
Revenue Acceleration/Inflection
- PTC Therapeutics (PTCT) reported $823 million in total revenue for 2025, exceeding its guidance, with $588 million in product revenue and closing the year with over $1.94 billion in cash.
- The launch of PTC Therapeutics' Sephience product generated $112 million in net revenue since launch as of December 31, 2025, with 946 patients on commercial therapy worldwide.
- For 2026, PTC Therapeutics provided revenue guidance of $700-$800 million and OpEx guidance of $680-$720 million, with an expectation to reach cash flow breakeven. The 2026 product revenue guidance does not include Evrysdi royalty revenue, which was sold for $240 million in milestones in December 2025.
- BillionToOne achieved a $334 million annualized revenue run rate as of Q3 2025, with 70% gross margins and GAAP profitability.
- BillionToOne provided updated revenue guidance, projecting $293-$299 million for full-year 2025 and $415-$430 million for 2026.
Jan 12, 2026, 5:00 PM
PTC Therapeutics Provides 2025 Unaudited Results and 2026 Guidance
PTCT
Earnings
Guidance Update
Product Launch
- PTC Therapeutics reported unaudited 2025 total product and royalty revenue of approximately $823.4 million, exceeding guidance, with unaudited 2025 product revenue of approximately $587.8 million.
- The company's Sephience launch showed strong performance, with unaudited Q4 2025 net revenue of approximately $92.5 million and total net revenue of approximately $112.1 million for 2025 since launch.
- For 2026, PTC anticipates total product revenue of $700 to $800 million, representing 19% to 36% year-over-year growth.
- PTC ended 2025 with a strong cash position of approximately $1.94 billion as of December 31, 2025.
- Key pipeline updates include alignment with the FDA on the design of a global Phase 3 trial for the votoplam Huntington's disease program, planned to initiate in H1 2026, with the FDA open to a potential Accelerated Approval pathway.
Jan 12, 2026, 1:30 PM
PTC Therapeutics sells remaining Evrysdi royalty interest for $240 million upfront
PTCT
- On December 29, 2025, PTC Therapeutics, Inc. sold its retained interest in the Evrysdi® (risdiplam) royalty to Royalty Pharma Investments 2019 ICAV.
- The company received $240.0 million in upfront cash consideration for this sale.
- PTC Therapeutics is eligible for three potential additional cash payments of $20.0 million each, contingent on specific royalty payment thresholds being met by RPI in 2027, 2028, and 2029.
- Following this transaction, PTC Therapeutics now owns 0% of the Evrysdi® royalty, with Royalty Pharma owning 100%.
Dec 29, 2025, 9:05 PM
PTC Therapeutics Announces Approval of Sephience in Japan
PTCT
Product Launch
- PTC Therapeutics received approval from the Japanese Ministry of Health, Labor and Welfare (MHLW) for Sephience™ (sepiapterin) for the treatment of phenylketonuria (PKU) in children and adults.
- This approval is the first product approval for PTC in Japan and includes individuals of all ages and the full spectrum of disease severity.
- The company anticipates concluding pricing discussions in Q1 2026, with the product launch expected shortly thereafter.
- Sephience has now been approved in the EU, US, and Japan within less than six months, facilitating a global launch.
Dec 22, 2025, 12:00 PM
PTC Therapeutics Discusses Strong Sapphiens Launch and Future Outlook at Citi Conference
PTCT
Product Launch
New Projects/Investments
Guidance Update
- PTC Therapeutics reported a strong launch for Sapphiens, its oral therapy for PKU, in the US and EU during 2025, with continued momentum into Q4. This product is anticipated to drive the company towards cash flow break-even in the near future.
- The company is expanding the global reach of Sapphiens, with expected approvals in Japan and Brazil before the end of 2025, complementing its simultaneous US and European launches. Early uptake has been observed across all patient segments, including switches from existing therapies and therapy-naive patients.
- PTC Therapeutics is committed to achieving cash flow break-even soon and plans to reduce operating expenses in 2026 relative to 2025. The company also highlighted significant advancements in its small molecule splicing platform, identifying new therapeutic targets and potential for strategic partnerships.
Dec 4, 2025, 3:30 PM
PTC Therapeutics Reports Strong Sapphiens Launch and Pipeline Progress
PTCT
Product Launch
Guidance Update
New Projects/Investments
- PTC Therapeutics (PTCT) has experienced an incredibly successful year in 2025, driven by the US and EU approval of Sapphiens, an oral therapy for PKU, which is demonstrating an incredibly strong launch and continued strong momentum into the fourth quarter. This product is anticipated to lead the company to cash flow break-even and beyond in the near future.
- The company is actively preparing for the global launch of Sapphiens, with approvals expected in Japan and Brazil before the end of 2025. To support this, PTC has expanded its API manufacturers from one to two, with plans to add a third, ensuring it can meet anticipated global demand.
- PTC's R&D day highlighted the significant evolution of its small molecule splicing platform, which has identified 256 additional sequences that could be targeted, offering substantial potential for new therapies and strategic partnerships.
- In its pipeline, Votaplam for Huntington's disease is noted as a differentiated oral small molecule with placebo-controlled data, and its partner Novartis is moving forward with the next trial. The company also received a Complete Response Letter (CRL) for vatiquinone, and discussions with the FDA are scheduled for Q4 2025 to explore potential pathways.
- PTC has committed to reducing operating expenses (OpEx) in 2026 relative to 2025 as part of its strategy to achieve cash flow break-even.
Dec 4, 2025, 3:30 PM
PTC Therapeutics Outlines R&D Strategy and Pipeline Progress at R&D Day
PTCT
New Projects/Investments
- PTC Therapeutics has strategically refocused its R&D efforts on small molecule splicing and ferroptosis platforms to develop transformative therapies for diseases with high unmet medical needs.
- The company's PTSeek splicing platform has enabled the discovery of novel modulators, with plans to select clinical candidates in early 2026 for programs targeting Huntington's disease/DM1 (MSH3 lowering), SCA3 (ataxin-3 lowering), and an oncology target, aiming for Phase I readiness by end of 2026 for the NRD and SCA3 programs.
- In its inflammation and ferroptosis platform, PTC is advancing a preclinical program for Parkinson's disease and an NRF2 activation program, with a development candidate expected in H2 2026. The clinical pipeline includes a highly potent NLRP3 inhibitor (PTC612) for autoimmune and inflammatory disorders.
- These R&D initiatives are being conducted with a significantly reduced research budget, aligning with the company's commitment to OpEx reduction and achieving cash flow break-even, while also exploring strategic partnerships for certain programs.
Dec 2, 2025, 2:30 PM
Quarterly earnings call transcripts for PTC THERAPEUTICS.
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