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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) announced its participation in the SVB Securities Global Biopharma Conference on February 14, 2023, at 9:20 a.m. EST. The company will present a comprehensive overview of its operations and strategic focus. A live webcast of the presentation will be available on PTC's Events and Presentations page and will be archived for 30 days. PTC is dedicated to developing innovative treatments for patients with rare disorders, aiming to maximize access to transformative medicines. For further details, visit www.ptcbio.com.
PTC Therapeutics (NASDAQ: PTCT) announced that the National Institute for Health and Care Excellence (NICE) has recommended Translarna™ (ataluren) for reimbursement within the National Health Service (NHS) in England and Wales. This decision makes Translarna the only approved treatment for patients aged 2 and older with nonsense mutation Duchenne muscular dystrophy who can walk. The NICE recommendation is based on clinical trials showing Translarna's effectiveness in slowing disease progression and improving outcomes. This marks a significant milestone for patients and reflects PTC's ongoing commitment to access for effective treatments.
PTC Therapeutics (PTCT) announced the launch of its ninth STRIVE™ awards program aimed at funding initiatives for the Duchenne Muscular Dystrophy (Duchenne) community. Submissions are due by March 17, 2023, with projects focusing on Innovation and Transition to Adulthood. Since its inception in 2015, STRIVE has supported 39 initiatives across 20 countries, addressing the needs of Duchenne patients who now live longer due to improved treatments. This year’s recipients will be announced in September, continuing PTC's 25-year commitment to support the Duchenne community.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on January 5, 2023, the approval of non-statutory stock options for 39,585 shares and 13,045 restricted stock units (RSUs) for 33 new employees as a part of their employment compensation. This follows the NASDAQ inducement grant exception guidelines. The stock options have an exercise price of $39.42 per share, vesting over four years, while the RSUs also vest over a four-year period. These grants are designed to attract talent and align new hires’ interests with the company's long-term goals.
PTC Therapeutics reported a 2022 total revenue of approximately $710 million, showing over 30% year-over-year growth. The company projects 2023 total revenue guidance between $940 million and $1 billion. Significant corporate milestones include the approval of Upstaza™ for AADC deficiency and the initiation of several clinical trials. The unaudited net product revenue in 2022 reached $535 million, with guidance for DMD franchise revenue in 2023 between $545 million and $565 million.
PTC Therapeutics, based in South Plainfield, N.J., will present a company overview at the 41st Annual J.P. Morgan Healthcare Conference on January 9, 2023, at 7:30 a.m. PST/10:30 a.m. EST.
The presentation can be accessed via a live webcast on the company’s Investor section and will be archived for 30 days. PTC focuses on developing medicines for rare disorders, aiming to provide access to transformative treatments for patients with unmet medical needs.
PTC Therapeutics (PTCT) announced that the Brazilian Health Regulatory Agency (ANVISA) has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This marks a significant milestone as it's the first global approval for Waylivra for this indication. The approval was based on the Phase 2/3 BROADEN study, which demonstrated an 88% mean reduction in triglyceride levels for patients treated with Waylivra at three months, compared to only 22% for the placebo group. Waylivra is also approved for Familial Chylomicronemia Syndrome (FCS) in Brazil.
PTC Therapeutics has received authorization from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK for Upstaza (eladocagene exuparvovec), the first gene therapy aimed at treating AADC deficiency. This rare disorder severely impacts motor development, posing life-threatening challenges. Upstaza has demonstrated clinical efficacy, allowing patients to achieve significant motor and cognitive milestones after treatment. This authorization follows a similar approval in the European Union, furthering PTC's commitment to innovative therapies for rare diseases.
PTC Therapeutics reported a 57% year-over-year revenue growth with total quarterly revenue of $217 million for Q3 2022. Key products: DMD revenue reached $131 million, with Translarna at $77 million and Emflaza at $55 million. The company secured strategic financing from Blackstone for up to $1 billion to support its pipeline. Despite growth, PTC recorded a net loss of $109.3 million and a significant decrease in cash equivalents to $288.4 million since 2021. Full-year revenue guidance increased to $710-$750 million.
PTC Therapeutics has entered a strategic financing collaboration with Blackstone, securing up to $500 million in funding to enhance its product pipeline and business development. This includes $350 million in low-cost capital at the close. The funds will help accelerate PTC's mission to deliver transformative medicines, with a goal of launching one therapy every 2-3 years. Blackstone's investment will be collateralized by PTC's existing products, including Translarna™ and Upstaza®. The collaboration aims to optimize capital structure and support new medicines.
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