Earnings summaries and quarterly performance for PTC THERAPEUTICS.
Executive leadership at PTC THERAPEUTICS.
Matthew B. Klein
Detailed
Chief Executive Officer
CEO
Christine Utter
Detailed
Senior Vice President, Chief Accounting Officer and Head of People Services
Eric Pauwels
Detailed
Chief Business Officer
Lee Golden
Detailed
Executive Vice President and Chief Medical Officer
Mark E. Boulding
Detailed
Executive Vice President and Chief Legal Officer
Neil Almstead
Detailed
Chief Technical Operations Officer
Pierre Gravier
Detailed
Chief Financial Officer
Board of directors at PTC THERAPEUTICS.
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
Joseph Schwartz
Oppenheimer
4 questions for PTCT
Also covers: ASND, AUPH, BMRN +14 more
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
Joel Beatty
Baird
3 questions for PTCT
Also covers: ACAD, ALKS, AMLX +11 more
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
Paul Choi
Goldman Sachs
3 questions for PTCT
Also covers: AMRN, ARVN, ASND +12 more
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
Samantha Corwin
William Blair
2 questions for PTCT
Also covers: ALLO, BEAM, KRYS +3 more
Brian Cheng
JPMorgan Chase & Co.
1 question for PTCT
Also covers: ALLO, CHRS, EDIT +6 more
Danielle Brill Bongero
Raymond James
1 question for PTCT
David Lebowitz
Citigroup Inc.
1 question for PTCT
Also covers: ALNY, ARWR, ASND +11 more
Dingding Shi
Jefferies
1 question for PTCT
Also covers: ADCT, ASND, AUTL +6 more
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
Huidong Wang
Barclays
1 question for PTCT
Also covers: ALNY, BCRX, BEAM +15 more
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
Richard Miller
Cantor Fitzgerald
1 question for PTCT
Also covers: ADMA, QURE
Sami Corwin
William Blair
1 question for PTCT
Also covers: ALLO, BEAM, KRYS +5 more
Tiago Fauth
Wells Fargo
1 question for PTCT
Also covers: LQDA, VRNA, WVE
Recent press releases and 8-K filings for PTCT.
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.
4 days ago
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.
4 days ago
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.
6 days ago
PTC Therapeutics Provides R&D Day Update on Splicing and Ferroptosis Platforms
PTCT
New Projects/Investments
Guidance Update
- PTC Therapeutics has significantly advanced its small molecule splicing platform, including the development of the PTCEK discovery engine, which has expanded the universe of druggable splicing targets.
- The company plans to select clinical candidates in early 2026 for its MSH3 program (targeting Huntington's disease and DM1), SCAT3 program, and an oncology program, with the MSH3 and SCAT3 programs aiming for Phase 1 readiness by the end of 2026.
- PTC is also progressing its ferroptosis platform, focusing on a Parkinson's disease program targeting 15-LO, and an NLRP3 program for inflammatory diseases, demonstrating superior potency with new compounds.
- Following a significant restructuring, PTC is committed to achieving cash flow break-even and reducing OpEx, while also seeking strategic partnerships for non-core therapeutic areas to leverage its science.
6 days ago
PTC Therapeutics Outlines R&D Strategy and Program Updates at 2025 R&D Day
PTCT
New Projects/Investments
- PTC Therapeutics is strategically focusing on small molecule therapies, leveraging its splicing and ferroptosis platforms to develop treatments for diseases with high unmet medical needs.
- The company provided updates on its splicing platform, which has already yielded commercial successes like Evrysdi and Branaplam, and is now advancing new programs targeting MSH3 (for Huntington's and DM1, with a clinical candidate selection planned for Q1 2026), SCAT3, fetal hemoglobin induction, and protein aggregation in neurodegenerative diseases.
- Updates on the ferroptosis and inflammation platforms include progress in Parkinson's disease (development candidate selection in Q1 2026, Phase 1 in H2 2026), NRF2 activation, and two clinical-stage programs: PTC612 (NLRP3 inhibitor, Phase 1 in H1 2026) and PTC844 (DHODH inhibitor, Phase 1 completed, phase two-ready).
- PTC remains committed to achieving cash flow break-even and reducing operating expenses, with an expectation of further OpEx reduction guidance in January, and is open to strategic partnerships for larger indications.
6 days ago
PTC Therapeutics Discusses SEPHIENCE Launch and Pipeline Updates
PTCT
Product Launch
Guidance Update
New Projects/Investments
- PTC Therapeutics highlighted the successful global launch of SEPHIENCE, an oral therapy for PKU, which is expected to be a multi-billion dollar opportunity and become the standard of care.
- SEPHIENCE generated $19.6 million in revenue in its first six weeks of Q3, with $14.2 million from the U.S. and $5.4 million internationally, and had over 300 patients on therapy by the Q3 earnings call.
- The company is nearing profitability, driven by SEPHIENCE, with a full-year revenue guidance of $750 million-$800 million and OpEx of $730 million.
- PTC has upcoming FDA meetings in Q4 for its pipeline programs: vatiquinone for Friedreich's ataxia and PTC518 for Huntington's disease, with positive Phase 2 results for PTC518 showing dose-dependent Huntington lowering and clinical benefit.
Nov 18, 2025, 8:00 AM
PTC Therapeutics Discusses Strong Safiance Launch and Pipeline Updates
PTCT
Product Launch
Guidance Update
New Projects/Investments
- Safiance, PTC Therapeutics' oral therapy for PKU, had a strong initial launch, generating $19.6 million in revenue in the first six weeks of Q3, with $14.2 million from the U.S. and $5.4 million internationally. The company reported over 300 patients on therapy and over 520 patient start forms by the end of Q3.
- The company anticipates Safiance to be a multi-billion dollar opportunity and the key driver for achieving profitability, with current year revenue guidance of $750 million-$800 million and unchanged OpEx of $730 million.
- Despite losing exclusivity in February 2024, the Amplify program in the U.S. for DMD has maintained over 70% of its peak revenue due to brand loyalty and patient services.
- PTC Therapeutics has upcoming FDA meetings in Q4 for Vatiquinone in Friedreich's ataxia to discuss resubmission pathways after a CRL, and for PTC518 in Huntington's disease to align on the next efficacy trial design and explore accelerated approval following positive Phase 2 results.
Nov 18, 2025, 8:00 AM
PTC Therapeutics Reports Strong Sephience Launch and Provides Pipeline Updates
PTCT
Product Launch
Guidance Update
Revenue Acceleration/Inflection
- PTC Therapeutics reported $211 million in revenue for the third quarter, with a strong financial position of approximately $1.7 billion on its balance sheet.
- The company's new PKU drug, Sephience, generated $19.6 million in global revenue in Q3 2025, including $14.2 million from the US, and recorded over 500 patient start forms in the first six weeks of its launch.
- PTC Therapeutics received a Complete Response Letter (CRL) from the FDA for vatiquinone for Friedreich's Ataxia and plans a meeting with the FDA in Q4 2025 to discuss potential paths to resubmission or a new clinical trial.
- The company aims to achieve cash flow breakeven and profitability in the near future with Sephience, with a subsequent goal of reaching a $2 billion top line.
Nov 11, 2025, 8:30 PM
PTCT Announces Q3 2025 Results and Narrows Full-Year Guidance
PTCT
Earnings
Guidance Update
Product Launch
- PTCT reported third-quarter 2025 revenue of $211 million and narrowed its 2025 full-year revenue guidance to $750-$800 million, reflecting confidence in the Sephience launch and consistent performance of the DMD franchise.
- The global launch of Sephience generated $19.6 million in revenue in Q3 2025, with 521 patient start forms received from US centers and 341 patients on commercial therapy as of September 30.
- The company ended Q3 2025 with a strong financial position, holding approximately $1.68 billion in cash, cash equivalents, and marketable securities.
- Updates were provided on other programs, including a planned Q4 meeting with the FDA for the Huntington's Disease program to discuss study design and potential accelerated approval, and ongoing FDA review for Translarna's NDA.
Nov 4, 2025, 9:30 PM
PTC Therapeutics Reports Third Quarter 2025 Financial Results and Updates Full-Year Guidance
PTCT
Earnings
Guidance Update
Product Launch
- PTC Therapeutics reported total revenue of $211.0 million and net income of $15.9 million for the third quarter of 2025.
- The company initiated the US and EU launch of Sephience, generating $19.6 million in global revenue and receiving 521 patient start forms in the US as of September 30, 2025.
- PTC Therapeutics narrowed its full-year 2025 revenue guidance to $750 million to $800 million.
- Cash, cash equivalents, and marketable securities stood at $1,687.8 million as of September 30, 2025.
Nov 4, 2025, 9:10 PM
Quarterly earnings call transcripts for PTC THERAPEUTICS.
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