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 (PTCT) provides critical updates through this centralized news resource for investors and industry stakeholders. Track official announcements spanning clinical trial progress, regulatory milestones, and strategic partnerships in rare disease therapeutics.
Access timely updates on PTCT's small molecule developments, gene therapy advancements, and financial disclosures. Our curated feed ensures comprehensive coverage of material events including FDA submissions, research collaborations, and commercialization efforts.
This repository features verified press releases covering:
• Clinical trial results for neuromuscular and oncology programs
• Regulatory filings and orphan drug designations
• Strategic alliances with research institutions
• Financial performance and earnings communications
Bookmark this page for direct access to PTCT's evolving developments in post-transcriptional control therapeutics. Monitor breakthrough innovations addressing Duchenne muscular dystrophy, genetic disorders, and underserved patient populations.
PTC Therapeutics (NASDAQ: PTCT) has received a positive opinion from the European Medicines Agency's CHMP for Sephience™ (sepiapterin), a treatment for phenylketonuria (PKU) in children and adults. The approval recommendation includes a comprehensive label covering all age groups and disease severities.
The company plans to launch immediately after European Commission ratification, expected in approximately two months. The authorization will apply to all 27 EU member states plus Iceland, Norway, and Liechtenstein, with initial focus on Germany and key European markets offering immediate named patient access.
Additionally, Sephience is under review by the FDA with a PDUFA date of July 29, 2025, and approval applications are being processed in Japan and Brazil.
PTC Therapeutics (NASDAQ: PTCT) has announced its participation in four major healthcare investor conferences in May and June 2025. The schedule includes:
- Bank of America Securities Healthcare Conference on May 13 at 1:00 p.m. ET
- RBC Capital Markets Global Healthcare Conference on May 20 at 2:05 p.m. ET
- William Blair 45th Annual Growth Stock Conference on June 3 at 10:20 a.m. ET
- Goldman Sachs 46th Annual Global Healthcare Conference on June 10 at 9:20 a.m. ET
All presentations will be available via live webcast on PTC's investor relations website and will remain accessible for 30 days after each event. Viewers are advised to connect several minutes before the start time.
PTC Therapeutics (NASDAQ: PTCT) has announced a webcast conference call scheduled for Tuesday, May 6, at 4:30 p.m. ET. The call will present the company's first quarter 2025 financial results along with updates on business operations and future outlook.
Participants are advised to register in advance for dial-in details and join 15 minutes before the start time to avoid delays. The webcast will be accessible through PTC's investor relations website, with a replay available for 30 days after the call.
PTC Therapeutics (NASDAQ: PTCT) has announced the approval of equity compensation packages for 35 new employees on March 24, 2025. The compensation includes 12,000 non-statutory stock options and 28,220 restricted stock units (RSUs).
The stock options were granted at an exercise price of $57.03 per share, matching PTC's closing price on the grant date. These options have a 10-year term and vest over four years, with 25% vesting after the first year and 6.25% quarterly thereafter. The RSUs also follow a four-year vesting schedule, with 25% vesting annually.
These inducement grants, approved by PTC's Compensation Committee, comply with Nasdaq Listing Rule 5635(c)(4) and are part of the new employees' compensation packages.
PTC Therapeutics (NASDAQ: PTCT) announced that the European Commission (EC) has adopted the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) opinion to not renew the authorization of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy.
This decision effectively removes the drug's conditional marketing authorization in the European Economic Area. However, the EC indicated that individual EU countries can use Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued Translarna use. PTC Therapeutics plans to work on a country-by-country basis to provide commercial drug where possible.
PTC Therapeutics (NASDAQ: PTCT) has presented new data from their Phase 3 APHENITY trial and open-label extension study for sepiapterin treatment in phenylketonuria (PKU) patients at the 2025 ACMG Annual Meeting.
Key findings demonstrate that 97% of participants in the Phe tolerance protocol showed improved dietary flexibility, with a mean increase of 126% in protein intake. Notably, 66% of subjects reached or exceeded the age-adjusted recommended daily protein intake while maintaining blood Phe control.
The genetic variant analysis revealed that over 70% of study participants had a Genotype-Phenotype Value consistent with classical PKU. The results suggest significant treatment benefits, including for patients with mutations not responsive to BH4 and those with classical disease phenotype.
PTC Therapeutics (NASDAQ: PTCT) reported full year 2024 revenue of $807 million, with Q4 revenue at $213.2 million. The company achieved all clinical and regulatory milestones, including four FDA-accepted NDA submissions.
Key financial highlights include DMD franchise Q4 revenue of $144 million, with Translarna generating $93.7 million and Emflaza $50.5 million. The company secured a significant deal with Novartis for its PTC518 Huntington's disease program in January 2025, strengthening its cash position to over $2 billion.
For 2025, PTC projects total revenues between $600-800 million, including product sales and Evrysdi royalties. The company expects GAAP R&D and SG&A expenses of $805-835 million, and non-GAAP expenses of $730-760 million for 2025.
PTC Therapeutics (NASDAQ: PTCT) has announced its executive team's participation in two major upcoming investor conferences in March 2025. The company will be featured in fireside chat sessions at the TD Cowen 45th Annual Health Care Conference on March 4 at 11:10 a.m. ET and the Barclays 27th Annual Global Healthcare Conference on March 11 at 12:30 p.m. ET.
Both presentations will be accessible through live webcasts on PTC Therapeutics' investor relations website under the Events and Presentations section. The webcasts will remain archived for 30 days after the presentations. Interested parties are advised to connect to the website several minutes before the scheduled start times to ensure proper connection.
PTC Therapeutics (NASDAQ: PTCT) announced FDA acceptance and Priority Review for its New Drug Application (NDA) for vatiquinone, targeting the treatment of children and adults with Friedreich's ataxia (FA). The FDA has set a PDUFA target action date of August 19, 2025.
The application is supported by data from the placebo-controlled MOVE-FA study and two long-term studies involving pediatric and adult FA patients. These studies demonstrated significant evidence of slowing disease progression and confirmed vatiquinone's safety and tolerability across all age groups.
If approved, vatiquinone would become the first therapy available for pediatric FA patients while offering an alternative treatment option for adults. This marks PTC's fourth FDA application acceptance in 2024.
PTC Therapeutics (NASDAQ: PTCT) has announced it will host a webcast conference call to discuss its fourth quarter and full year 2024 financial results on Thursday, February 27, at 4:30 p.m. EST. The call will also include updates on the company's business and outlook.
Participants can access the call via phone by registering through a provided link and are advised to dial in 15 minutes before the start time to avoid delays. The webcast will be accessible through PTC's Investor Relations website, with a replay available for 30 days after the call.
