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 |
|---|---|---|---|
| 3/11/2025 | $41.00 → $55.00 | Underperform → Neutral | BofA Securities |
| 3/7/2025 | $55.00 | Sector Perform | Scotiabank |
| 12/13/2024 | $45.00 → $67.00 | Equal-Weight → Overweight | Morgan Stanley |
| 12/3/2024 | $39.00 → $63.00 | Sector Perform → Outperform | RBC Capital Mkts |
| 10/10/2024 | Mkt Perform | Raymond James | |
| 9/4/2024 | $44.00 | Outperform | Robert W. Baird |
| 8/26/2024 | $47.00 | Buy | UBS |
| 5/20/2024 | Underperform → Mkt Perform | Raymond James | |
| 12/19/2023 | $28.00 | Equal-Weight → Underweight | Morgan Stanley |
| 12/8/2023 | $37.00 | Overweight | Wells Fargo |
— Results show 97% of phenylalanine (Phe) tolerance study participants were able to increase their dietary Phe intake with a mean increase of 126% — — Genetic variant analysis of Phase 3 APHENITY trial demonstrates meaningful effect in classical subjects with non-BH4 responsive genotypes — WARREN, N.J., March 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American College of Medical Genetics and Genomics (ACMG) Annual Clinical Genetics Meeting. These data provide further evidence of the potential meaningful benefits of sepiapterin treatment for the full s
– Full year 2024 revenue of $807 million, exceeding guidance – – All 2024 clinical and regulatory milestones were achieved on schedule, including four NDA submissions, all of which were accepted for filing – – License and collaboration agreement with Novartis for PTC518 Huntington's disease program closed in January 2025 – – Cash of over $2.0 billion as of January 2025 – WARREN, N.J., Feb. 27, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the fourth quarter ending December 31, 2024. "Our strong fourth quarter rounds out a year of significant accomplishment across every part of our company," said Matthew B. Klein, M.
WARREN, N.J., Feb. 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that its executives will participate in fireside chats at the following investor conferences: TD Cowen 45th Annual Health Care 2025 Conference Tuesday, March 4 at 11:10 a.m. ET Barclays 27th Annual Global Healthcare ConferenceTuesday, March 11 at 12:30 p.m. ET The 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 for 30 days following the
- PDUFA target action date of Aug. 19, 2025 - WARREN, N.J., Feb. 19, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the New Drug Application (NDA) for vatiquinone for the treatment of children and adults living with Friedreich's ataxia (FA). The application has been granted Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) target action date of Aug. 19, 2025. "We are excited to be one step closer to bringing an approved therapy to all patients with Friedreich's ataxia," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "If approved, vatiquinone woul
WARREN, N.J., Feb. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the company will host a webcast conference call to report its fourth quarter and full year 2024 financial results and provide an update on the company's business and outlook on Thursday, Feb. 27, at 4:30 p.m. EST. 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 participants dial in 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section
WARREN, N.J., Jan. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that on Jan. 3, 2025, the company approved 1,200 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to two 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 Jan. 3, 2025, and are being made as an inducement material to each employee's acceptance of employment with the company in accordance with Nasdaq Listing Rule 5635(c)(4). The RSUs each will vest over four years with
– Unaudited 2024 total revenue of approximately $814 million, exceeding guidance – – Four approval applications submitted to FDA in 2024 – – Global launch preparations underway for sepiapterin for PKU, a $1 billion market opportunity; CHMP opinion expected Q2 2025, U.S. approval decision in July 2025 – – License and collaboration agreement with Novartis for PTC518 program closed – – PIVOT-HD data readout for PTC518 expected Q2 2025 – WARREN, N.J., Jan. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today provided an update on the Company's progress and its outlook for 2025. Matthew B. Klein, M.D., Chief Executive Officer of PTC, will discuss these updates at the 43rd Annual
WARREN, N.J., Dec. 23, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that CEO Matthew B. Klein, M.D., will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 11:15 a.m. PST / 2:15 p.m. EST. The presentation will be webcast live on the Events and Presentations page under the Investors section of PTC Therapeutics' website at https://ir.ptcbio.com/events-presentations and will be archived for 30 days following the presentation. It is recommended that users connect to PTC's website several minutes prior to the start of the webcast to ensure a timely connection. About PTC Therapeutics, Inc.PTC is a global biopharmaceutical co
- If approved, vatiquinone would be the first and only authorized therapy for children with FA - - PTC's fourth approval application submitted to FDA in 2024 - WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA). "We are excited to have reached this important milestone in the development of vatiquinone," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The evidence of short- and long-term efficacy as well as the extensive safety data collec
WARREN, N.J., Dec. 17, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that on Dec. 13, 2024, the company approved non-statutory stock options to purchase an aggregate of 1,725 shares of its common stock and 5,060 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to eight 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 Dec
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
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
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BofA Securities upgraded PTC Therapeutics from Underperform to Neutral and set a new price target of $55.00 from $41.00 previously
Scotiabank initiated coverage of PTC Therapeutics with a rating of Sector Perform and set a new price target of $55.00
Morgan Stanley upgraded PTC Therapeutics from Equal-Weight to Overweight and set a new price target of $67.00 from $45.00 previously
RBC Capital Mkts upgraded PTC Therapeutics from Sector Perform to Outperform and set a new price target of $63.00 from $39.00 previously
Raymond James resumed coverage of PTC Therapeutics with a rating of Mkt Perform
Robert W. Baird initiated coverage of PTC Therapeutics with a rating of Outperform and set a new price target of $44.00
UBS resumed coverage of PTC Therapeutics with a rating of Buy and set a new price target of $47.00
Raymond James upgraded PTC Therapeutics from Underperform to Mkt Perform
Morgan Stanley downgraded PTC Therapeutics from Equal-Weight to Underweight and set a new price target of $28.00
Wells Fargo initiated coverage of PTC Therapeutics with a rating of Overweight and set a new price target of $37.00
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– Full year 2024 revenue of $807 million, exceeding guidance – – All 2024 clinical and regulatory milestones were achieved on schedule, including four NDA submissions, all of which were accepted for filing – – License and collaboration agreement with Novartis for PTC518 Huntington's disease program closed in January 2025 – – Cash of over $2.0 billion as of January 2025 – WARREN, N.J., Feb. 27, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the fourth quarter ending December 31, 2024. "Our strong fourth quarter rounds out a year of significant accomplishment across every part of our company," said Matthew B. Klein, M.
WARREN, N.J., Feb. 13, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the company will host a webcast conference call to report its fourth quarter and full year 2024 financial results and provide an update on the company's business and outlook on Thursday, Feb. 27, at 4:30 p.m. EST. 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 participants dial in 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section
- PTC to receive $1.0B in cash at closing -- PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones -- PTC to share profits in the U.S. and tiered double-digit royalties on ex-U.S. net sales -- Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of ongoing PIVOT-HD study -- PTC will host a conference call on Dec. 2, 2024, at 8:30 am EST- WARREN, N.J., Dec. 2, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today the signing of an exclusive global license and collaboration agreement with Novartis Pharmaceuticals Corporation, a subsidiary of Novartis AG (NY
– Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA – – On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone – WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024. "I am proud of our team's strong execution," said Matthew Klein, M.D., Chief Executive Officer. "We continue to achieve excellent revenue performance allowing us to raise full-year revenue guidance.
WARREN, N.J., Oct. 24, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the company will host a webcast conference call to report its third quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, Nov. 7, at 4:30 p.m. EST. 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 participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call will be available app
– Continued strong revenue performance – – NDAs for sepiapterin and Translarna™ submitted to FDA – – Positive interim data readout from PTC518 PIVOT-HD study – – On track to achieve remaining 2024 clinical and regulatory milestones – WARREN, N.J., Aug. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the second quarter ending June 30, 2024. "I am proud of our team's continued outstanding execution as we have accomplished all our objectives so far this year," said Matthew Klein, M.D., Chief Executive Officer. "We are in a strong cash position, submitted three drug approval applications to the FDA and remain on schedul
WARREN, N.J., July 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the company will host a webcast conference call to report its second quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, Aug. 8, at 4:30 p.m. EDT. 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 participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call will be available
- FDA lifts PTC518 partial clinical hold based on PIVOT-HD data - - Conference call and webcast to be held June 20th at 8:00 am EDT - WARREN, N.J., June 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today shared interim results from the Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. At Month 12, PTC518 treatment resulted in dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF) in the interim cohort of patients. In addition, favorable trends were demonstrated on several relevant HD clinical assessments including Total Motor Score (TMS) and Composite Unified Huntington's Disease Rating Scale (cUHDRS). Fu
– Strong revenue performance across product portfolio – – Submitted MAA for sepiapterin and BLA for Upstaza – – On target to achieve remaining 2024 clinical and regulatory milestones, including global submissions for sepiapterin – WARREN, N.J., April 25, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2024. "We are off to a strong start in 2024, with outstanding commercial performance and achievement of all planned clinical and regulatory milestones for the first quarter," said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. "We remain on track to achieve the ma
SOUTH PLAINFIELD, N.J., April 11, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the company will host a webcast conference call to report its first quarter 2024 financial results and provide an update on the company's business and outlook on Thursday, April 25, 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 participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at https://ir.ptcbio.com/events-presentations. A replay of the call will be
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