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 pioneering global biopharmaceutical company dedicated to discovering, developing, and commercializing orally-administered small-molecule therapeutics. The company is focused on post-transcriptional control processes in RNA biology, which are crucial for regulating protein synthesis and ensuring proper cellular function. PTC's innovative approach targets a range of therapeutic areas, including neuromuscular disorders, oncology, and infectious diseases, with a particular emphasis on treatments for orphan and ultra-orphan disorders.
One of PTC's notable achievements is the development of Translarna™ (ataluren), a treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD). Despite a recent setback with the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) issuing a negative opinion, the European Commission has asked for a re-evaluation, keeping the medication available in Europe. This reflects the strong backing from the patient community and expert physicians who believe in its efficacy and safety.
The company’s robust product pipeline also includes Upstaza™ (eladocagene exuparvovec), a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. This therapy has recently been submitted for review by the U.S. FDA and granted Priority Review status. Upstaza has shown transformative clinical benefits in clinical trials and compassionate use programs, demonstrating significant neurological improvements in patients.
Additionally, PTC is advancing sepiapterin for the treatment of phenylketonuria (PKU). The company's Marketing Authorization Application (MAA) for sepiapterin has been accepted by the EMA, with submissions planned in the U.S., Brazil, and Japan. Sepiapterin has demonstrated significant reductions in blood phenylalanine levels, offering a potential new treatment option for PKU patients who currently have limited therapeutic choices.
Financially, PTC has shown strong revenue performance and is well-positioned for future success. Recent financial results indicate a thriving operational status, supported by a diversified portfolio and strategic partnerships. The company's commitment to providing best-in-class treatments for rare disorders, combined with its global commercial infrastructure, underscores its strategy to maximize value for all stakeholders.
PTC's mission is to leverage its scientific and clinical expertise to bring innovative therapies to patients in need, ensuring access to treatments that can significantly improve quality of life. For more information, please visit their official website.
PTC Therapeutics (NASDAQ: PTCT) has confirmed ongoing enrollment for its Phase 2 PIVOT-HD trial of PTC518, aimed at treating Huntington's disease. The 12-month study has received approval in several European countries and Australia, focusing on two dose levels: 5mg and 10mg. Enrollment began in the US but has paused due to FDA requests for additional data. There have been no treatment-related adverse events reported to date. PTC plans to share data from the initial 12-week study phase within the first half of 2023.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on October 27, 2022, at 4:30 p.m. ET to discuss its third quarter 2022 financial results and business updates. Participants can register for the call online to receive dial-in details and are advised to join 15 minutes early. A replay will be available approximately two hours after the call and can be accessed on the company's website for 30 days. PTC is focused on developing medicines for rare disorders, leveraging its scientific expertise and global commercial capabilities.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced it will present new data on Translarna™ (ataluren) at the 27th International Annual Congress of the World Muscle Society in Halifax, Nova Scotia, on October 12-14, 2022. Key presentations will highlight updated demographics and safety data, age at loss of ambulation, pulmonary function, and the efficacy of ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna is currently licensed in the European Economic Area for nmDMD treatment in ambulatory patients aged 2 and older, while being investigational in the U.S.
PTC Therapeutics (NASDAQ: PTCT) announced the recipients of its 2022 STRIVE Awards, supporting Duchenne Muscular Dystrophy initiatives. The program grants funds to four organizations: ACDM (Brazil) for educational rehabilitation, KASDER (Turkey) for expanding psychological services, MDA Hellas (Greece) for a film on sexuality, and Action Duchenne (U.K.) for skills training. This year, PTC received 40 applications, highlighting diverse needs in the Duchenne community. The STRIVE Awards enhance awareness and support for patients, aiming to provide a broader impact across four countries.
PTC Therapeutics (NASDAQ: PTCT) will present an overview at upcoming investor conferences: Wells Fargo's 17th Annual Healthcare Conference on Sept. 8 at 1:55 p.m. EDT and Morgan Stanley's 20th Annual Global Healthcare Conference on Sept. 12 at 4:15 p.m. EDT. These presentations will be available via live webcast on PTC's Investor page and archived for 30 days. PTC focuses on developing treatments for rare disorders, enhancing access to innovative medicines for patients with unmet medical needs.
PTC Therapeutics reported $166 million in total revenue for Q2 2022, marking a 42% year-over-year growth. The DMD franchise showed strong revenue performance with $134 million net product revenue, a 32% increase from the previous year. Notably, Upstaza™ gene therapy received marketing authorization from the European Commission, beginning its commercial rollout. However, the company faced a net loss of $152 million compared to $118 million in Q2 2021. PTC reaffirms its full-year guidance of $700-$750 million in total revenues.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on August 4, 2022, at 4:30 p.m. ET, to discuss its second quarter 2022 financial results and provide a business outlook. Participants can register for the call in advance to receive dial-in details and are encouraged to join 15 minutes early to avoid delays. A replay of the conference call will be available on PTC's website shortly after the event and archived for 30 days. The company is committed to developing treatments for rare disorders.
PTC Therapeutics announced the European Commission's approval of Upstaza™ (eladocagene exuparvovec), the first gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, applicable in all EU member states and select countries. This therapy is significant for patients aged 18 months and older, offering a disease-modifying treatment option. Clinical trials showed substantial improvements in motor and cognitive skills in treated patients. CEO Stuart W. Peltz emphasized the therapy's potential impact on the quality of life for patients suffering from this severe genetic disorder.
PTC Therapeutics (NASDAQ: PTCT) will hold a conference call on June 21, 2022, at 8:00 a.m. E.T. to discuss topline results from Study 041 of Translarna™ (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy. The call will include a slide presentation accessible via the company’s website. Translarna is a protein restoration therapy aimed at addressing genetic disorders caused by nonsense mutations. More details about Duchenne muscular dystrophy and Translarna can be found on PTC's website.
PTC Therapeutics announces the election of Alethia Young to its Board of Directors. Young brings over 20 years of experience in healthcare and biotech equity research, currently serving as CFO of Graphite Bio. Previously, she held senior positions at Cantor Fitzgerald, Credit Suisse, and Deutsche Bank. Young expressed enthusiasm about PTC's diverse pipeline aimed at rare diseases, emphasizing her commitment to leveraging her background for the company's growth. CEO Stuart W. Peltz highlighted her industry knowledge as a valuable asset for PTC's future success.
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