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 will present a company overview at the 40th Annual J.P. Morgan Virtual Healthcare Conference on January 10, 2022, at 7:30 a.m. EST. The live webcast can be accessed on PTC's Investor Events and Presentations page and will be archived for 30 days. PTC Therapeutics focuses on developing and commercializing medicines for rare disorders, with a commitment to providing access to innovative treatments for patients in need.
PTC Therapeutics (PTCT) announced the publication in Nature Communications detailing a novel splicing mechanism from its Huntington Disease (HD) program. This mechanism selectively lowers huntingtin protein levels via innovative splicing modifiers. CEO Stuart W. Peltz highlighted the breakthrough's potential to regulate gene expression, benefiting various diseases. HD affects around 45,000 individuals in the U.S., with no existing treatments targeting its underlying cause. PTC aims to leverage its splicing platform for new therapies, enhancing its pipeline of medicines for rare disorders.
PTC Therapeutics announced that its oral therapy Evrysdi® (risdiplam) has received prestigious awards for its innovation and life-saving effects. Recognized as the 2022 Drug Discovery of the Year by the British Pharmacological Society and the 2021 Drug Discovery Prize by the Society for Medicines Research, Evrysdi aims to treat spinal muscular atrophy (SMA) by enhancing SMN protein levels. The drug is commercialized by Roche and Genentech in the U.S., showcasing PTC's commitment to addressing unmet medical needs through innovative therapies.
PTC Therapeutics, Inc. (PTCT) has announced the approval of stock options for 49 new employees, totaling 83,750 shares, and 21,550 restricted stock units (RSUs). The awards, effective as of November 29, 2021, serve as an employment inducement under NASDAQ Listing Rule 5635(c)(4). Each stock option has an exercise price of $36.89 and a 10-year term, with vesting occurring over four years. The RSUs will also vest over four years. These grants reflect PTC's commitment to attract talent in the biopharmaceutical sector.
PTC Therapeutics (NASDAQ: PTCT) will present a company overview at the 4th Annual Evercore ISI HealthCONx Conference on Tuesday, Nov. 30 at 8:25 a.m. ET. The presentation will be available via a live webcast on PTC's Investor website and archived for 30 days. PTC focuses on developing clinically differentiated medicines for rare disorders, leveraging its scientific expertise and global infrastructure to address unmet medical needs. For more details, visit www.ptcbio.com.
PTC Therapeutics (NASDAQ: PTCT) reported a 39% growth in its Duchenne muscular dystrophy (DMD) franchise, with third-quarter net product revenue reaching $114 million. Total revenues increased to $138.7 million, compared to $118.4 million in Q3 2020. The company anticipates full-year DMD franchise revenues between $400 and $420 million, adjusting previous guidance. However, net losses rose to $133.6 million from $69.7 million year-on-year, and cash reserves decreased to $867.9 million. PTC continues advancements across its clinical pipeline with several ongoing trials.
PTC Therapeutics will present a company overview at the Credit Suisse 30th Annual Healthcare Conference on Nov. 9, 2021, at 8:00 a.m. ET. The presentation will be accessible via a live webcast on the company's investor site and archived for 30 days post-event. PTC focuses on developing innovative treatments for rare disorders, emphasizing a strong scientific foundation and global commercial capabilities to meet unmet patient needs.
PTC Therapeutics, Inc. (NASDAQ: PTCT) has announced that Brazil's ANVISA has approved the expansion of Translarna™ (ataluren) to include children aged 2 to 5 with nonsense mutation Duchenne muscular dystrophy (Duchenne). This broadens the prior indication of 5 years and older, allowing earlier treatment that may preserve muscle function and slow disease progression. Translarna is the sole treatment targeting the underlying cause of Duchenne due to a nonsense mutation and is licensed in the EEA for ambulatory patients aged 2 and older.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on Oct. 28, 2021, at 4:30 p.m. ET to report its third quarter financial results and provide a business update. Investors can join the call by dialing (877) 303-9216 in the U.S. or (973) 935-8152 internationally, using passcode 8574103. A live webcast will be available on the company's investor website, with a replay accessible two hours post-call and archived for 30 days. PTC focuses on developing innovative treatments for rare disorders, backed by a robust product pipeline.
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