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 |
|---|---|---|---|
| 5/9/2025 | $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 |
| 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 |
– Strong revenue performance of $190 million – – Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date – – Global Sephience launch activities progressing well – – Strong cash position of over $2.0 billion as of March 31, 2025 – WARREN, N.J., May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2025. "Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year r
- Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 - - Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 - - Signals of dose-dependent clinical benefit relative to matched natural history cohort as well as dose-dependent lowering of NfL in Stage 2 patients at Month 24 - - Continued favorable safety and tolerability profile with no treatment-related NfL spikes - - PTC will host a conference call on May 5, 2025, at 8:00 am ET- WARREN, N.J., May 5, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disea
WARREN, N.J., May 1, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that on April 25, 2025, the company approved non-statutory stock options to purchase an aggregate of 7,000 shares of its common stock and 10,780 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 12 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 April
- Label includes full spectrum of PKU patients - - European launch preparations underway - WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive of all ages and disease severities. "This positive opinion is great news for the PKU patient community in Europe for whom there remains a significant unmet medical need," said Matthew B
WARREN, N.J., April 24, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that its executives will speak at the following conferences: Bank of America Securities 2025 Health Care ConferenceTuesday, May 13 at 10:00 a.m. PT / 1:00 p.m. ET RBC Capital Markets Global Healthcare Conference 2025Tuesday, May 20 at 2:05 p.m. ET William Blair 45th Annual Growth Stock Conference 2025Tuesday, June 3 at 9:20 a.m. CT / 10:20 a.m. ET Goldman Sachs 46th Annual Global Healthcare Conference 2025Tuesday, June 10 at 9:20 a.m. ET The presentations will be webcast live on t
WARREN, N.J., April 17, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the company will host a webcast conference call to report its first quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, May 6, 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 for the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investors
WARREN, N.J., March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that on March 24, 2025, the company approved non-statutory stock options to purchase an aggregate of 12,000 shares of its common stock and 28,220 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 35 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 Ma
WARREN, N.J., March 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) to not renew the authorization of Translarna™ (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy. While this action effectively removes the drug's conditional marketing authorization in the European Economic Area, the EC indicated that individual countries within the European Union can leverage Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued use of Translarna. "We are of course disappointe
— 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.
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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
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
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
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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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– Strong revenue performance of $190 million – – Positive CHMP opinion for Sephience™ (sepiapterin) received in April 2025, NDA review remains on track for July 29, 2025 PDUFA date – – Global Sephience launch activities progressing well – – Strong cash position of over $2.0 billion as of March 31, 2025 – WARREN, N.J., May 6, 2025 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ:PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2025. "Following a year of outstanding execution across every part of the Company, we have built on this positive momentum with solid revenue performance in the first quarter, allowing us to narrow our full-year r
- Study met primary endpoint with dose-dependent blood HTT protein lowering at Week 12 - - Favorable dose-dependent trends across clinical scales in Stage 2 patients at Month 12 - - Signals of dose-dependent clinical benefit relative to matched natural history cohort as well as dose-dependent lowering of NfL in Stage 2 patients at Month 24 - - Continued favorable safety and tolerability profile with no treatment-related NfL spikes - - PTC will host a conference call on May 5, 2025, at 8:00 am ET- WARREN, N.J., May 5, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington's disea
WARREN, N.J., April 17, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) announced today that the company will host a webcast conference call to report its first quarter 2025 financial results and provide an update on the company's business and outlook on Tuesday, May 6, 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 for the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investors
– 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
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