Welcome to our dedicated page for Ptc Therapeutics news (Ticker: PTCT), a resource for investors and traders seeking the latest updates and insights on Ptc Therapeutics stock.
PTC Therapeutics, Inc. (NASDAQ: PTCT) is a global biopharmaceutical company focused on clinically differentiated medicines for children and adults living with rare disorders. The PTCT news feed on Stock Titan aggregates company announcements, financial updates and regulatory milestones that PTC discloses through press releases and SEC filings.
Investors following PTCT news can see updates on the commercial performance and global rollout of products such as Sephience (sepiapterin) for phenylketonuria (PKU), as well as revenue contributions from the company’s Duchenne muscular dystrophy franchise, which includes Translarna (ataluren) and Emflaza (deflazacort). News items also cover royalty revenue from Evrysdi (risdiplam) and changes in royalty arrangements, such as the sale of certain Evrysdi royalty rights to Royalty Pharma.
PTC Therapeutics regularly reports quarterly and annual financial results, including total revenue, product revenue, royalty and collaboration revenue, and details on GAAP and non-GAAP R&D and SG&A expenses. Corporate updates often highlight pipeline and regulatory developments, including FDA meetings for programs like votoplam in Huntington’s disease and vatiquinone in Friedreich’s ataxia, as well as ongoing review of the Translarna NDA.
The company also issues news about regulatory approvals and launches in different regions, such as Sephience approvals in the EU, US and Japan, and about R&D-focused events and investor conference presentations. In addition, PTC discloses equity inducement grants under Nasdaq Listing Rule 5635(c)(4), illustrating its use of stock options and restricted stock units as part of employee compensation.
By monitoring the PTCT news page, readers can track how product launches, regulatory decisions, collaborations, royalty transactions and capital markets activities shape the company’s progress in the rare disease biopharmaceutical sector.
PTC Therapeutics (NASDAQ: PTCT) said executives will present at three investor conferences in early March 2026: TD Cowen on March 2, Leerink on March 10, and Barclays on March 11.
All presentations will be webcast live via the company’s Events and Presentations page and archived for 30 days. Viewers are advised to connect several minutes early.
PTC Therapeutics (NASDAQ: PTCT) reported full-year 2025 product and royalty revenue of $831M, total 2025 revenues of $1.731B, and cash of $1.945B at year-end. Sephience launched in 2H 2025, generating $111M in 2025 and $92M in Q4. PTC closed a royalty sale for Evrysdi for $240M upfront and up to $60M in milestones. Full-year 2025 net income was $682.6M driven by a Novartis collaboration payment; GAAP R&D and SG&A guidance given for 2026.
Phenylketonuria market (includes BioMarin - BMRN) is forecast to expand through 2034 driven by rising diagnosis rates, expanded newborn screening, and multiple emerging therapies.
Key datapoints: US had 18,800 diagnosed PKU cases in 2024; sepiapterin received regulatory approvals in 2025 and pegvaliase sBLA entered FDA review.
PTC Therapeutics (NASDAQ: PTCT) announced on February 12, 2026 that it has withdrawn the New Drug Application resubmission for Translarna (ataluren) for nonsense mutation Duchenne muscular dystrophy (DMD) following feedback from the U.S. Food and Drug Administration.
According to the company, FDA reviewers indicated the NDA data are unlikely to meet the Agency's threshold of substantial evidence of effectiveness to support approval, and PTC therefore withdrew the submission; leadership expressed disappointment after more than two decades of development.
PTC Therapeutics (NASDAQ: PTCT) will report fourth quarter and full year 2025 financial results on Thursday, Feb. 19, 2026 at 4:30 p.m. ET. The company will host a webcast conference call to discuss results, business updates, and outlook.
Investors can register for phone access or join the webcast on the company's Investors site. A replay will be archived on the company website for 30 days.
PTC Therapeutics (NASDAQ: PTCT) reported unaudited 2025 product and royalty revenue of approximately $823.4M, with unaudited product revenue of about $587.8M. Sephience generated unaudited Q4 2025 net revenue of approximately $92.5M and total 2025 net revenue since launch of $112.1M. PTC ended 2025 with approximately $1.94B in cash, cash equivalents and marketable securities. In December 2025 PTC sold remaining Evrysdi royalty rights for $240M upfront plus up to $60M in milestones while retaining a separate $150M milestone right. For 2026 PTC guides total product revenue of $700–$800M (ex-Evrysdi royalties), GAAP R&D and SG&A of $775–$815M, and non-GAAP R&D and SG&A of $680–$720M. Clinical updates include Phase 3 alignment and Accelerated Approval openness for votoplam HD and FDA follow-up requests for vatiquinone MOVE-FA.
PTC Therapeutics (NASDAQ: PTCT) approved an inducement equity award on Jan. 2, 2026 granting 300 restricted stock units (RSUs) to a new non-executive employee. Each RSU represents the right to one share of common stock upon vesting. The award was approved by the company's Compensation Committee and made under the Nasdaq inducement grant exception (Listing Rule 5635(c)(4)) as part of the employee's hire compensation. The RSUs vest over four years, with 25% vesting on each annual anniversary of the hire date, subject to continued service through each vesting date.
PTC Therapeutics (NASDAQ: PTCT) announced MHLW approval of Sephience (sepiapterin) in Japan for the treatment of children and adults living with phenylketonuria (PKU).
The Japanese label covers all ages and the full spectrum of disease severity. This is PTC's first product approval in Japan and follows approvals in the EU and US within less than six months, enabling a coordinated global launch. PTC said pricing discussions in Japan are expected to conclude in Q1 2026, with commercial launch occurring shortly thereafter.
PTC Therapeutics (NASDAQ: PTCT) announced inducement equity grants approved Dec. 16, 2025 for five new employees under Nasdaq Listing Rule 5635(c)(4).
The company granted non-statutory stock options to purchase an aggregate of 3,825 shares and 4,385 restricted stock units (RSUs). Options have an exercise price of $75.42 (closing price on Dec. 16, 2025), a 10-year term, and vest over four years (25% after one year, then 6.25% quarterly). RSUs vest over four years with 25% vesting on each annual anniversary, subject to continued service.
PTC Therapeutics (NASDAQ: PTCT) announced that CEO Matthew B. Klein, M.D., will present at the 44th Annual J.P. Morgan Healthcare Conference on Monday, Jan. 12, 2026 at 9:00 a.m. PST / 12:00 p.m. EST.
The presentation will be webcast live on the company’s Events and Presentations investor page at https://ir.ptcbio.com/events-presentations and will be archived for 30 days following the presentation.