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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 (NASDAQ: PTCT) announced encouraging two-year data from the SUNFISH trial for Evrysdi™ (risdiplam) at the 2021 MDA Conference. Patients with Type 2 or Type 3 spinal muscular atrophy showed sustained or improved motor function after 24 months. Key measures like MFM32, RULM, and HFMSE indicated motor function stability and improvement. Adverse events decreased in the second year, with upper respiratory infections being the most common. Evrysdi aims to enhance SMN protein production, crucial for motor neuron health, and is marketed in the U.S. by Genentech.
On March 15, 2021, PTC Therapeutics announced results from a real-world study involving 92 patients with Duchenne and Becker muscular dystrophy who switched from prednisone to EMFLAZA (deflazacort) following FDA approval. The study indicated that most patients experienced improvement in disease progression after switching, with 95% of DMD and 90% of BMD patients reporting effective results. The study emphasized the need for better disease management and the potential of EMFLAZA in altering the natural history of DMD, reflecting PTC's commitment to offering differentiated treatments.
The SMA Foundation and PTC Therapeutics (NASDAQ: PTCT) have expanded their collaboration to advance research in regenerative medicine for Spinal Muscular Atrophy (SMA) and related neuromuscular disorders. The partnership aims to fund academic institutions to develop new treatments, filling a therapeutic gap in SMA care. This initiative builds on their successful history, including the recent approval of Evrysdi for SMA. SMA is a serious genetic disorder affecting 1 in 11,000 babies, leading to muscle atrophy and loss of function.
On February 26, 2021, PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) recommended approval of Evrysdi™ (risdiplam) for treating 5q spinal muscular atrophy (SMA) in patients aged 2 months and older. The decision follows accelerated assessment due to the drug's significance for public health. Evrysdi has already been approved in seven countries and is under review in 30 more. A final decision from the European Commission is anticipated in the next two months.
PTC Therapeutics (NASDAQ: PTCT) announced its financial results for Q4 and full year 2020, reporting total revenues of $380.8 million, up from $307 million in 2019. Key drivers included product revenues of $331 million from Translarna and Emflaza, with a 38% YoY growth in Emflaza. R&D expenses rose significantly to $477.6 million, reflecting investment in clinical trials and programs. The company holds $1.1 billion in cash. For 2021, PTC anticipates net product revenues between $355 million and $375 million and R&D and SG&A expenses of $825-$855 million.
PTC Therapeutics announced positive results from the pivotal FIREFISH study published in The New England Journal of Medicine, evaluating Evrysdi™ (risdiplam) for infants with symptomatic type 1 spinal muscular atrophy (SMA). The study showed significant improvements in survival and developmental milestones after 12 months, with 90% of infants alive without permanent ventilation. Despite some serious adverse events, the overall safety profile remains favorable. This study reinforces the therapeutic potential of Evrysdi in treating SMA, providing insight into its role in addressing unmet medical needs.
PTC Therapeutics (PTCT) announced the initiation of the second stage of the FITE19 clinical trial for PTC299, an oral investigational drug aimed at COVID-19 treatment. This phase follows a successful first stage, which involved 40 patients and an interim safety analysis. Approximately 340 participants will be enrolled in the second stage across multiple centers outside the U.S. PTC299 targets the enzyme dihydroorotate dehydrogenase (DHODH), potentially inhibiting viral replication and modifying inflammatory responses. The primary endpoint is to assess clinical efficacy compared to a placebo.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced its participation in several upcoming investor conferences, including the Raymond James 42nd Annual Institutional Investors Conference on March 2 at 7:30 a.m. ET, the Cowen 41st Annual Health Care Conference on March 4 at 10:30 a.m. ET, and the Barclays Global Healthcare Conference on March 10 at 9:10 a.m. ET. The presentations will be webcast live on PTC's website and archived for 30 days. PTC focuses on developing differentiated medicines for rare disorders, emphasizing its commitment to meeting unmet medical needs.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on February 25, 2021, at 4:30 p.m. (ET) to discuss its fourth quarter and year-end 2020 financial results. Investors can access the call by dialing (877) 303-9216 domestically or (973) 935-8152 internationally, using passcode 2174406. A live webcast will be available on PTC's investor relations website, with a replay accessible two hours post-call and archived for 30 days. PTC focuses on developing treatments for rare disorders, emphasizing a diversified pipeline.
PTC Therapeutics hosted a conference call on February 4, 2021, at 5:30 p.m. E.T. to discuss results from its clinical study 045 of Translarna™ (ataluren) for treating patients with nonsense mutation Duchenne muscular dystrophy. The call featured access via phone and a live webcast, both providing crucial insights into this rare genetic disorder. Duchenne muscular dystrophy leads to muscle weakness and fatal complications, primarily affecting males. Translarna focuses on restoring protein production in affected patients, currently licensed in the European Economic Area.
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