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) reported a 66% increase in total revenue to $118.4 million for Q3 2020, driven by the launch of Evrysdi for spinal muscular atrophy. The company achieved significant milestones, including $35 million in collaboration revenue from Roche. However, net loss widened to $69.7 million, compared to $60 million last year, with R&D expenses rising to $93 million. Cash and equivalents increased to $1.14 billion. PTC aims to enhance long-term value through innovative therapies.
On October 28, 2020, PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the approval of non-statutory stock options and restricted stock units (RSUs) for 18 new employees. A total of 18,610 stock options and 6,860 RSUs were granted as part of the employees' compensation. The stock options have an exercise price of $51.96 per share and a vesting schedule over four years. The inducement grants align with NASDAQ Listing Rule 5635(c)(4), established to attract new talent within the company.
PTC Therapeutics, trading under NASDAQ: PTCT, will present an overview at two upcoming virtual conferences. The Credit Suisse 29th Annual Virtual Healthcare Conference is scheduled for November 11, 2020, at 9:30 a.m. ET, and the Barclays Virtual Gene Editing & Gene Therapy Summit will occur on November 16, 2020, at 3:30 p.m. ET. These presentations will be streamed live and archived for two weeks on PTC's investor relations webpage. PTC focuses on developing innovative therapies for patients with rare disorders.
PTC Therapeutics (NASDAQ: PTCT) announced the launch of a Phase 2/3 clinical trial for vatiquinone (PTC743) aimed at treating mitochondrial epilepsy, a severe condition affecting children with inherited mitochondrial diseases. With approximately 11,000-13,000 potential patients across the US, EU, Japan, and Latin America, the trial is critical as it addresses a significant unmet medical need. Vatiquinone has shown promise in earlier studies, impacting seizure frequency and related morbidity in over 500 patients. The trial's primary endpoint focuses on reducing seizure frequency in a controlled setting.
PTC Therapeutics, along with the AADC Family Network, has announced October 23 as the first AADC Deficiency Awareness Day in Massachusetts. This designation aims to raise awareness about Aromatic L-amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder that significantly impacts affected children. To commemorate the day, PTC will host a Facebook Live event featuring industry experts, including Kelly Heger and Matthew Klein. The initiative highlights the ongoing efforts towards finding treatment options for this condition, which currently has no approved therapies.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that its drug, Evrysdi™ (risdiplam), received approval from Brazil's ANVISA for spinal muscular atrophy (SMA), marking its second country approval. Additionally, PTC filed a New Drug Application in Japan, which triggers a $7.5 million milestone payment from Roche. Evrysdi™ has been submitted for approval in 15 other markets and is undergoing reviews in four health authorities. The drug offers a new treatment option for SMA, which affects 1 in 10,000 babies. Its oral formulation is significant for patient accessibility during the COVID-19 pandemic.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on October 29, 2020, at 4:30 p.m. (ET), to report third quarter 2020 financial results and update its business outlook. Investors can access the call by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) with the passcode 7096445. A live webcast will also be available on the company’s website and a replay will be archived for 30 days. PTC focuses on developing medicines for rare disorders and aims to provide access to innovative treatments.
On September 28, 2020, PTC Therapeutics announced positive two-year data from the FIREFISH study on Evrysdi™ (risdiplam) for treating spinal muscular atrophy (SMA). The study indicated that 88% of infants were alive without permanent ventilation after two years. Significant motor milestones were achieved, including increased independent sitting and head control. The treatment's safety profile remained consistent, with no new safety signals. Evrysdi™ was recently approved and shows a strong commercial launch, highlighting its importance amid the ongoing pandemic.
PTC Therapeutics announced on September 21, 2020, the approval of stock options for 75,670 shares and 12,845 restricted stock units (RSUs) to 22 new employees, as part of their employment compensation. These inducement grants comply with NASDAQ's listing rules and aim to attract new talent. The options have an exercise price of $45.46 per share, with a 10-year term and a four-year vesting schedule. The RSUs will also vest over four years. PTC focuses on developing innovative treatments for rare disorders, driving their investment in a strong pipeline of medicines.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will present a company overview on October 1, 2020, at the Jefferies Virtual Gene Therapy/Editing Summit at 9:00 a.m. ET. The presentation will be live-streamed on their website and available for 30 days afterward. PTC Therapeutics focuses on developing medicines for rare disorders, aiming to provide access to innovative treatments for patients with unmet needs. For optimal viewing, it's recommended to connect a few minutes before the start of the webcast.
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