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 launched the seventh annual STRIVE Awards program aimed at supporting Duchenne muscular dystrophy patient advocacy organizations. This year, a new category has been added to facilitate the transition of Duchenne patients from adolescence to adulthood. The awards emphasize the increasing life expectancy of Duchenne patients, now reaching their 30s and 40s, which highlights the need for enhanced quality of life support. The submission deadline is March 15, with winners announced in September 2021.
On January 6, 2021, PTC Therapeutics (NASDAQ: PTCT) approved inducement grants for 10 new employees, comprising 13,465 stock options and 4,665 restricted stock units (RSUs). These grants are part of the employee's compensation and comply with NASDAQ Listing Rule 5635(c)(4). The options have an exercise price of $66.49 and a 10-year term, vesting over four years. RSUs will also vest over four years with annual anniversaries. This initiative reflects PTC's commitment to attracting talent in the biopharmaceutical sector.
PTC Therapeutics (NASDAQ: PTCT) presented its R&D pipeline updates and commercial progress at the J.P. Morgan Healthcare Conference on January 11, 2021. Highlights from 2020 include a DMD franchise revenue of approximately $331 million, with significant contributions from Translarna and Emflaza. The company anticipates 2021 revenues between $355 and $375 million for the DMD franchise and plans to conduct several clinical trials. Preliminary financial results indicate approximately $1.1 billion in cash and equivalents by year-end 2020.
PTC Therapeutics announced its participation in the 39th Annual J.P. Morgan Healthcare Conference on January 11, 2021, at 7:30 a.m. EST. The event will be webcast live, with a replay available for 30 days. The company focuses on developing and commercializing innovative medicines for rare disorders, aiming to improve patient access to necessary treatments. Interested parties are encouraged to join the webcast via the investor relations section on PTC's website.
PTC Therapeutics (NASDAQ: PTCT) announced the approval of non-statutory stock options for 12,500 shares and 4,360 restricted stock units (RSUs) to 10 new employees as part of their employment compensation. These awards were granted under NASDAQ's inducement grant exception on December 15, 2020. The stock options have an exercise price of $66.37 and will vest over four years. The RSUs will follow a similar vesting schedule. PTC, focused on rare disorders, continues to strengthen its workforce to support its mission in biopharmaceutical development.
PTC Therapeutics will host a webinar on December 15, 2020, at 12:00 noon ET, focusing on Translarna, a treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD). The session will discuss clinical and real-world data, commercial success, and potential regulatory milestones in the United States. Results from Study PTC124-GD-045-DMD are expected in Q1 2021. The webinar can be accessed via phone or through the company's website, with a replay available for 30 days.
PTC Therapeutics (NASDAQ: PTCT) announced on December 2, 2020, the approval of stock options and restricted stock units (RSUs) for 47 new employees as part of their compensation package. The grant includes 41,955 stock options at an exercise price of $63.86 and 20,735 RSUs, both vesting over four years. These inducement grants comply with NASDAQ Listing Rule 5635(c)(4) and were approved by the Compensation Committee to attract new talent.
PTC Therapeutics announced that Translarna (ataluren) has received marketing approval in the Russian Federation for treating nonsense mutation Duchenne muscular dystrophy (nmDMD), impacting an estimated 13% of DMD patients. This approval is significant as Translarna is the only medicine targeted at the underlying cause of nmDMD in approved regions. CEO Stuart W. Peltz highlighted the demand from Russian physicians and the identification of nmDMD patients who may benefit. This marks an important milestone in PTC's goal to globally provide access to this treatment.
PTC Therapeutics (NASDAQ: PTCT) has initiated the registration-directed Phase 3 MOVE-FA study for vatiquinone (PTC743) in children and young adults with Friedreich ataxia (FA), a rare neurodegenerative disorder. This pivotal trial comes after vatiquinone showed positive results in a Phase 2 trial, impacting disease severity over 24 months. The trial will enroll approximately 110 participants across multiple regions, with primary endpoints focused on the modified Friedreich ataxia rating scale. The FDA has granted vatiquinone both Orphan Drug and Fast Track Designations.
PTC Therapeutics (NASDAQ: PTCT) will present at the Evercore ISI 3rd Annual HealthCONx Conference on December 1, 2020, at 11:45 a.m. ET. This presentation, featuring a company overview, will be available via live webcast on PTC's website, with an archived version accessible for 30 days. PTC Therapeutics is a global biopharmaceutical company specializing in innovative treatments for rare disorders, emphasizing its commitment to meeting unmet medical needs through a diversified pipeline of transformative medicines.
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