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PTC Therapeutics, Inc. - PTCT STOCK NEWS

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.

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PTC Therapeutics (NASDAQ: PTCT) will host a webcast conference call on February 22, 2022, at 4:30 p.m. ET to discuss fourth quarter and year-end 2021 financial results.

Interested parties can participate by calling (877) 303-9216 in the U.S. or (973) 935-8152 internationally. A live webcast will also be available on the company's investor website, with a replay accessible for 30 days post-call.

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PTC Therapeutics announced that the FDA has granted priority review for its Evrysdi (risdiplam) to treat pre-symptomatic infants under 2 months with spinal muscular atrophy (SMA). If approved, Evrysdi will be the first at-home treatment for this vulnerable group. Interim data from the RAINBOWFISH study showed that 80% of treated infants achieved key motor milestones within 12 months. The company emphasizes the need for early treatment in SMA cases, reinforcing the significance of Evrysdi in improving patients' lives.

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PTC Therapeutics (NASDAQ: PTCT) has announced the launch of the eighth annual STRIVE™ awards program to support initiatives for the Duchenne muscular dystrophy community. Grants will be awarded to patient advocacy organizations focusing on Innovation or Transition to Adulthood. The application process is open until March 18, 2022, with recipients announced in September. The STRIVE program has funded over 35 initiatives across 20 countries since its inception, enhancing awareness and support for Duchenne patients.

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PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on Jan. 7, 2022, the approval of non-statutory stock options for 26 new employees, totaling 18,255 shares, along with 10,675 restricted stock units (RSUs). These awards aim to incentivize new hires as part of their employment compensation, following NASDAQ's inducement grant exception. All stock options have an exercise price of $38.10 per share and vest over four years, contingent on continued service. This initiative reflects PTC's commitment to attracting talent for its rare disorder therapies.

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PTC Therapeutics, Inc. (NASDAQ: PTCT) has launched its 2022 Talent Pipeline Program, a global initiative aimed at providing recent college graduates with real-world job experiences. The program, which begins accepting applications on Feb. 11, 2022, will include around 30 Fellows who will receive mentorship, job coaching, and leadership training throughout the year. Successful participants will earn a Certificate of Completion and will be considered for future positions. The initiative aims to attract and retain top talent while enhancing PTC's organizational capabilities.

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PTC Therapeutics presented an update on its commercial progress and R&D pipeline at the 40th J.P. Morgan Healthcare Conference on January 10, 2022. The company reported a 29% growth in net product revenue to $429 million in 2021, driven by its Duchenne muscular dystrophy (DMD) franchise. Key milestones for 2022 include expected results from clinical studies for Translarna and vatiquinone, alongside a financial guidance projecting revenues between $700 million and $750 million. The company holds approximately $773 million in cash as of year-end 2021.

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PTC Therapeutics announced that Waylivra™ (volanesorsen) has received Category 1 classification from Brazil's CMED, making it the only treatment for familial chylomicronemia syndrome (FCS) available in the country. This classification recognizes its greater efficacy over current treatments and allows for international pricing. Furthermore, PTC submitted an application to ANVISA for Waylivra’s approval for familial partial lipodystrophy (FPL), potentially marking the first approved treatment for FPL globally. A decision from ANVISA is expected in the latter half of 2022.

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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.

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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.

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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.

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FAQ

What is the current stock price of PTC Therapeutics (PTCT)?

The current stock price of PTC Therapeutics (PTCT) is $45.04 as of January 9, 2025.

What is the market cap of PTC Therapeutics (PTCT)?

The market cap of PTC Therapeutics (PTCT) is approximately 3.5B.

What is PTC Therapeutics focused on?

PTC Therapeutics focuses on the discovery, development, and commercialization of orally-administered small-molecule therapeutics targeting post-transcriptional control processes in RNA biology.

What is Translarna™ (ataluren)?

Translarna™ (ataluren) is a treatment developed by PTC Therapeutics for nonsense mutation Duchenne muscular dystrophy (nmDMD), designed to enable the formation of functioning proteins in patients with genetic disorders.

What is Upstaza™ (eladocagene exuparvovec)?

Upstaza™ (eladocagene exuparvovec) is a one-time gene replacement therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, aimed at correcting the underlying genetic defect and restoring dopamine production.

What is sepiapterin used for?

Sepiapterin is being developed for the treatment of phenylketonuria (PKU), a rare metabolic disease. It has demonstrated significant reductions in blood phenylalanine levels in clinical trials.

Where is PTC Therapeutics headquartered?

PTC Therapeutics is headquartered in Warren, New Jersey, USA.

How does PTC Therapeutics generate revenue?

PTC generates revenue through the commercialization of its clinically differentiated medicines, such as Translarna and Upstaza, and through strategic partnerships.

What is the company's strategy for growth?

PTC's strategy includes leveraging its scientific and clinical expertise, global commercial infrastructure, and robust product pipeline to bring transformative therapies to patients with rare disorders.

What are some key products in PTC Therapeutics' pipeline?

Key products include Translarna for nmDMD, Upstaza for AADC deficiency, and sepiapterin for PKU.

What recent regulatory updates has PTC Therapeutics announced?

PTC recently announced the re-evaluation of Translarna by the EMA, the FDA's acceptance of the Upstaza BLA with Priority Review, and the EMA's validation of the sepiapterin MAA for PKU.

How can investors and media contact PTC Therapeutics?

Investors can contact Kylie O'Keefe at kokeefe@ptcbio.com, and media can reach Jeanine Clemente at jclemente@ptcbio.com.
PTC Therapeutics, Inc.

Nasdaq:PTCT

PTCT Rankings

PTCT Stock Data

3.47B
75.06M
2.34%
103.75%
5.95%
Biotechnology
Pharmaceutical Preparations
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United States of America
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