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, Inc. (NASDAQ: PTCT) will host a webcast conference call on April 27, 2023, at 4:30 p.m. ET to report its first quarter 2023 financial results and provide an update on the company's business outlook. Participants can register for the call here. A replay will be available on the company's website shortly after the call and archived for 30 days. PTC focuses on developing medicines for rare disorders and aims to maximize value for patients and stakeholders through its robust pipeline.
PTC Therapeutics (NASDAQ: PTCT) announced on March 22, 2023, the approval of stock options for 58 new employees, totaling 58,465 shares, along with 29,710 restricted stock units (RSUs). These inducement grants were made to incentivize new hires and adhere to NASDAQ Listing Rule 5635(c)(4). Each stock option has an exercise price of $46.35, in line with the company's closing stock price on the grant date. The options vest over four years, with 25% vesting on the first anniversary. The RSUs also vest over four years, with 25% vesting on each annual anniversary. This strategic move is aimed at attracting top talent to enhance PTC's capabilities.
PTC Therapeutics (NASDAQ: PTCT) announced the retirement of founding CEO Stuart Peltz, Ph.D., effective immediately. He will transition to a Senior Consultant role. Chief Operating Officer Matthew Klein, M.D., has been appointed the new CEO and will also join the Board of Directors. Klein brings extensive experience from BioElectron and has been integral to PTC's development pipeline, which includes five ongoing clinical trials and EMA approval for its gene therapy, Upstaza. A conference call will discuss the leadership transition today, March 24, 2023, at 8:30 am ET.
PTC Therapeutics reported total revenue of $699 million for 2022, a 30% year-over-year growth, or $740 million at constant exchange rates, a 37% increase. The company reaffirmed its 2023 guidance, expecting total revenue between $940 million and $1 billion. Key products include the Duchenne muscular dystrophy franchise, generating $507 million, and new therapies advancing in clinical trials. Notably, net loss for Q4 2022 was $170.9 million, compared to $143.3 million in Q4 2021. Cash and marketable securities fell to $410.7 million from $773.4 million in 2021.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced its participation in three upcoming investor conferences. The events include the Raymond James 44th Annual Institutional Investor Conference on March 6 at 8:05 a.m. EST, the Cowen 43rd Annual Health Care Conference on March 7 at 12:50 p.m. EST, and the Barclays Global Healthcare Conference on March 14 at 8:00 a.m. EST. Each presentation will be webcast live and archived on the company's Events and Presentations page for 30 days. PTC specializes in developing innovative medicines for rare disorders.
PTC Therapeutics (NASDAQ: PTCT) will host a webcast conference call on February 21, 2023, at 4:30 PM ET to report its fourth quarter and full year 2022 financial results. The call aims to provide an update on the company's business and future outlook. Interested participants can register here for dial-in information. The call will also be accessible via the PTC Investor website and archived for 30 days for those unable to attend live.
PTC Therapeutics, Inc. (NASDAQ: PTCT) has received the 2023 EURORDIS Black Pearl Company Award for Innovation, recognizing its 25-year commitment to rare disease research and treatment. The award celebrates organizations making significant advancements in the development of therapies for rare diseases.
CEO Stuart W. Peltz highlighted the importance of this recognition as PTC marks its quarter-century serving the rare disease community. The award ceremony is scheduled for February 21 in Brussels, coinciding with Rare Disease Day, which aims to raise awareness about rare diseases globally.
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