PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of medicines to patients with rare disorders. Its portfolio pipeline includes commercial products and product candidates in various stages of development, including clinical, pre-clinical and research and discovery stages, focuses on the development of treatments for multiple therapeutic areas, including rare diseases and oncology. The company offers Translarna and Emflaza for the treatment of Duchenne muscular dystrophy in the European Economic Area and the United States; commercializes Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean; and markets Evrysdi for the treatment of SMA in adults and children two months and older in Brazil. The company's splicing platform includes PTC518, which is being developed for the treatment of Huntington's disease. PTC Therapeutics, Inc. has collaborations with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation to advance drug discovery and development research in regenerative medicine; research collaboration with Massachusetts General Hospital for the treatment of rare genetic disorders resulting from pre-mRNA; and Akcea Therapeutics, Inc. to commercialize Tegsedi and Waylivra for the treatment of rare diseases in Latin America and the Caribbean. PTC Therapeutics, Inc. was founded in 1998 and is headquartered in South Plainfield, New Jersey.
IPO Year: 2013
Exchange: NASDAQ
Website: ptcbio.com
| Date | Price Target | Rating | Analyst |
|---|---|---|---|
| 5/27/2026 | $85.00 | Hold → Buy | Jefferies |
| 5/8/2026 | $90.00 | Hold → Buy | TD Cowen |
| 4/10/2026 | $108.00 | Outperform | Raymond James |
| 1/28/2026 | $119.00 | Overweight | Barclays |
| 12/1/2025 | $91.00 | Outperform → Sector Perform | RBC Capital Mkts |
| 10/20/2025 | $73.00 | Overweight | Wells Fargo |
| 6/17/2025 | $80.00 | Buy | Truist |
| 5/9/2025 | $68.00 | Neutral → Buy | BofA Securities |
| 5/7/2025 | $40.00 | Sell → Neutral | Citigroup |
| 3/11/2025 | $41.00 → $55.00 | Underperform → Neutral | BofA Securities |
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– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – 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 – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback –WARREN, N.J., May 7, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and fi
WARREN, N.J., May 4, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that its executives will speak at the following conferences: Bank of America Securities Health Care Conference 2026Tuesday, May 12 at 8:40 a.m. PDT / 11:40 a.m. EDTRBC Capital Markets 2026 Global Healthcare ConferenceWednesday, May 20 at 10 a.m. EDTGoldman Sachs 47th Annual Global Healthcare ConferenceTuesday, June 9 at 9:20 a.m. EDTThe presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website at https://ir.ptcbio.co
- Dose-dependent benefit on cUHDRS in Stage 2 participants compared to matched natural history cohort, with 52% slowing at 10 mg dose -- Continued evidence of favorable safety profile - - Novartis initiated global Phase 3 INVEST-HD study of votoplam -- PTC will host a conference call to discuss results today, April 28, at 4:30 pm ET - WARREN, N.J., April 28, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 Huntington's disease (HD) patients following 24 months of votoplam treatment compared to a
WARREN, N.J., April 28, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam. PTC's partner Novartis stated on its quarterly earnings call today that long-term extension data are supportive of the now-initiated Novartis Phase 3 INVEST-HD study and Novartis and PTC will assess potential best next steps for the program including further actions with FDA. 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
WARREN, N.J., April 22, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the company will host a webcast conference call to report its first quarter 2026 financial results and provide an update on the company's business and outlook on Thursday, May 7, 2026, at 4:30 p.m. ET. 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 r
WARREN, N.J., April 17, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that on April 13, 2026, the company approved non-statutory stock options to purchase an aggregate of 1,155 shares of its common stock and 925 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to a new non-executive employee. The awards were made pursuant to the Nasdaq inducement grant exception as a component of the new hire's employment compensation. The inducement grant was approved by PTC's Compensation Committee o
WARREN, N.J., March 25, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced the appointment of Jessica Chutter to its Board of Directors. Ms. Chutter is a highly accomplished biotechnology investment banker with an extensive list of achievements over her long tenure at Morgan Stanley, including building and leading the bank's biotechnology franchise. "I have closely followed Jessica's illustrious career, and I am proud to welcome her to PTC's Board of Directors," said Michael Schmertzler, Chairman of the Board of Directors. "Jessica will play an integral role as the Board continues to work with the management team on charting the company's next stage of growth."Ms. Ch
WARREN, N.J., March 13, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that on March 10, 2026, the company approved non-statutory stock options to purchase an aggregate of 14,550 shares of its common stock and 17,515 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 18 new employees. The awards were made pursuant to the Nasdaq inducement grant exception as a component of the new hires' employment compensation. The inducement grants were approved by PTC's Compensation Committee on Marc
WARREN, N.J., Feb. 23, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that its executives will speak at the following conferences: TD Cowen 46th Annual Health Care Conference 2026Monday, March 2 at 9:50 a.m. ETLeerink 2026 Global Healthcare ConferenceTuesday, March 10 at 4:20 p.m. ETBarclays Global Healthcare Conference 2026Wednesday, March 11 at 8 a.m. ETThe presentations will be webcast live on the Events and Presentations page under the Investor section of PTC Therapeutics' website at https://ir.ptcbio.com/events-presentations and will be archived f
– Full-year 2025 product and royalty revenue of $831M, exceeding guidance –– Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively –– Cash of $1.95B as of December 31, 2025 –WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025. "We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs,
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Jefferies upgraded PTC Therapeutics from Hold to Buy and set a new price target of $85.00
TD Cowen upgraded PTC Therapeutics from Hold to Buy and set a new price target of $90.00
Raymond James initiated coverage of PTC Therapeutics with a rating of Outperform and set a new price target of $108.00
Barclays resumed coverage of PTC Therapeutics with a rating of Overweight and set a new price target of $119.00
RBC Capital Mkts downgraded PTC Therapeutics from Outperform to Sector Perform and set a new price target of $91.00
Wells Fargo initiated coverage of PTC Therapeutics with a rating of Overweight and set a new price target of $73.00
Truist initiated coverage of PTC Therapeutics with a rating of Buy and set a new price target of $80.00
BofA Securities upgraded PTC Therapeutics from Neutral to Buy and set a new price target of $68.00
Citigroup upgraded PTC Therapeutics from Sell to Neutral and set a new price target of $40.00
BofA Securities upgraded PTC Therapeutics from Underperform to Neutral and set a new price target of $55.00 from $41.00 previously
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4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
3 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
SCHEDULE 13G/A - PTC THERAPEUTICS, INC. (0001070081) (Subject)
10-Q - PTC THERAPEUTICS, INC. (0001070081) (Filer)
8-K - PTC THERAPEUTICS, INC. (0001070081) (Filer)
8-K - PTC THERAPEUTICS, INC. (0001070081) (Filer)
DEFA14A - PTC THERAPEUTICS, INC. (0001070081) (Filer)
DEF 14A - PTC THERAPEUTICS, INC. (0001070081) (Filer)
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SCHEDULE 13G/A - PTC THERAPEUTICS, INC. (0001070081) (Subject)
8-K - PTC THERAPEUTICS, INC. (0001070081) (Filer)
10-K - PTC THERAPEUTICS, INC. (0001070081) (Filer)
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4 - PTC THERAPEUTICS, INC. (0001070081) (Issuer)
Live FDA approvals issued by the Food and Drug Administration and FDA breaking news
For Immediate Release: November 14, 2024 The U.S. Food and Drug Administration approved Kebilidi (eladocagene exuparvovec-tneq), an adeno-associated virus vector-based gene therapy indicated for the treatment of adult and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency. Kebilidi is the first FDA-approved gene therapy for treatment of AADC deficiency. “Clinical advancements in the f
Live Leadership Updates
WARREN, N.J., March 25, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced the appointment of Jessica Chutter to its Board of Directors. Ms. Chutter is a highly accomplished biotechnology investment banker with an extensive list of achievements over her long tenure at Morgan Stanley, including building and leading the bank's biotechnology franchise. "I have closely followed Jessica's illustrious career, and I am proud to welcome her to PTC's Board of Directors," said Michael Schmertzler, Chairman of the Board of Directors. "Jessica will play an integral role as the Board continues to work with the management team on charting the company's next stage of growth."Ms. Ch
PONTE VEDRA, Fla., Dec. 01, 2025 (GLOBE NEWSWIRE) -- Cadrenal Therapeutics, Inc. (NASDAQ:CVKD), a biopharmaceutical company developing transformative therapeutics to overcome current gaps in anticoagulation therapy, today announced the appointment of Lee Scott Golden, M.D., to its Board of Directors, effective immediately. Dr. Golden will serve as an independent director. Dr. Golden currently serves as Executive Vice President and Chief Medical Officer at PTC Therapeutics, Inc. (NASDAQ:PTCT), where he leads global clinical development across a broad rare disease pipeline. Before joining PTC, Dr. Golden served as the Chief Medical Officer at Espero BioPharma, Inc., a development-stage card
NEW YORK, Nov. 24, 2025 /PRNewswire/ -- S&P Dow Jones Indices will make the following changes to the S&P 500, S&P SmallCap 600: S&P SmallCap 600 constituent Sandisk Corp. (NASD: SNDK) will replace The Interpublic Group of Companies Inc. (NYSE:IPG) in the S&P 500, and PTC Therapeutics Inc. (NASD: PTCT) will replace Sandisk in the S&P SmallCap 600 effective prior to the opening of trading on Friday, November 28. S&P 500 constituent Omnicom Group Inc. (NYSE:OMC) is acquiring The Interpublic Group of Companies in a deal expected to close soon, pending final conditions.Upwork Inc. (NASD: UPWK) will replace Premier Inc. (NASD: PINC) in the S&P SmallCap 600 effective prior to the open of trading
BOSTON, Sept. 12, 2023 (GLOBE NEWSWIRE) -- Sensorium Therapeutics (Sensorium), a biotechnology company translating insights from nature and human experience to deliver transformational therapeutics, today announced the appointment of veteran life sciences executive David Southwell to its Board of Directors. "We are excited to welcome David to our Board," said Dick Simon, Chief Executive Officer of Sensorium. "He is a seasoned life sciences executive with a track record of success that spans multiple therapeutic areas and development stages. David's depth of experience will be a key asset as we advance our lead program, SENS-01, towards the clinic and expand our drug pipeline into addition
SOUTH PLAINFIELD, N.J., July 17, 2023 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced the appointment of Pierre Gravier to the role of Chief Financial Officer (CFO). Mr. Gravier brings more than 17 years of experience as an investment banker, venture capitalist and scientist to PTC. Most recently, Pierre was a managing director in the healthcare group of Perella Weinberg Partners, a leading independent global advisory firm. At Perella Weinberg, he focused on advising companies in the biopharmaceutical and pharmaceutical sectors on finance strategy and corporate development. "We are incredibly excited to have Pierre as part of PTC's leadership team," said Matthew B. Klein
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– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – 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 – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback –WARREN, N.J., May 7, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and fi
- Dose-dependent benefit on cUHDRS in Stage 2 participants compared to matched natural history cohort, with 52% slowing at 10 mg dose -- Continued evidence of favorable safety profile - - Novartis initiated global Phase 3 INVEST-HD study of votoplam -- PTC will host a conference call to discuss results today, April 28, at 4:30 pm ET - WARREN, N.J., April 28, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today reported positive topline results from the 24-month interim analysis of the PIVOT-HD long-term extension study, with favorable dose-dependent effects on disease progression for Stage 2 Huntington's disease (HD) patients following 24 months of votoplam treatment compared to a
WARREN, N.J., April 28, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) will host a webcast conference call today, April 28 at 4:30 p.m. ET to share results from the 24-month interim analysis of the PIVOT-HD long-term extension study of votoplam. PTC's partner Novartis stated on its quarterly earnings call today that long-term extension data are supportive of the now-initiated Novartis Phase 3 INVEST-HD study and Novartis and PTC will assess potential best next steps for the program including further actions with FDA. 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
WARREN, N.J., April 22, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the company will host a webcast conference call to report its first quarter 2026 financial results and provide an update on the company's business and outlook on Thursday, May 7, 2026, at 4:30 p.m. ET. 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 r
– Full-year 2025 product and royalty revenue of $831M, exceeding guidance –– Strong Sephience™ (sepiapterin) uptake since 2H 2025 launch with fourth quarter and 2025 revenue of $92M and $111M, respectively –– Cash of $1.95B as of December 31, 2025 –WARREN, N.J., Feb. 19, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2025. "We delivered another strong quarter and finish to 2025, building on the successful global launch of Sephience," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "With our robust commercial engine, innovative R&D programs,
WARREN, N.J., Feb. 5, 2026 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the company will host a webcast conference call to report its fourth quarter and full year 2025 financial results and provide an update on the company's business and outlook on Thursday, Feb. 19, 2026, at 4:30 p.m. ET. To access the call by phone, please click here to register and you will be provided with dial-in details. The webcast conference call can be accessed on the Investors section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call w
– Initiated US and EU launch of Sephience™ (sepiapterin) – – Strong initial Sephience uptake with global revenue of $19.6M and 521 start forms in the US as of September 30 – – Robust Q3 performance with total revenue of $211M – – Full-year 2025 revenue guidance narrowed to $750 - $800M – WARREN, N.J., Nov. 4, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the third quarter ended September 30, 2025. "We achieved an outstanding quarter, highlighted by the strong start to the global Sephience launch," said Matthew B. Klein, M.D., Chief Executive Officer. "The broad initial uptake supports the potential of Sephience to b
WARREN, N.J., Oct. 21, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the company will host a webcast conference call to report its third quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, November 4, at 4:30 p.m. ET. To access the call by phone, please click here to register, and you will be provided with dial-in details. To avoid delays, we recommend that participants dial in for the conference call 15 minutes before the start of the call. The webcast conference call can be accessed on the In
– European and FDA approval of Sephience™ (sepiapterin) with broad labeling for PKU – – Global launch underway in Europe and U.S. – – Total Q2 Revenue of $179M – WARREN, N.J., Aug. 7, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2025. "We had another strong quarter highlighted by the first approvals of Sephience for the treatment of children and adults with PKU," said Matthew B. Klein, M.D., Chief Executive Officer. "We have initiated the global launch and expect Sephience to be the foundational product for PTC's future growth and path to profitability." Key Corporate Updates: Sec
- Broad labeling inclusive of all disease subtypes for individuals 1 month of age and older - - PTC will host a conference call on Monday, July 28th at 5:00 pm ET - WARREN, N.J., July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in adult and pediatric patients 1 month of age and older with sepiapterin-responsive PKU. "We are excited to have reached this important milestone for those affected by PKU," said Matthew
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