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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, 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.
PTC Therapeutics (NASDAQ: PTCT) announced the winners of its sixth annual STRIVE Awards, recognizing initiatives aimed at supporting the Duchenne muscular dystrophy community. Awarded projects include an online platform in Argentina, events in Hungary connecting families with healthcare professionals, a support program in Russia, and a student ambassador program in the U.S. These initiatives aim to enhance community connections and awareness during challenging times. Mary Frances Harmon emphasized the importance of patient advocacy amid the ongoing challenges faced by rare disease patients.
PTC Therapeutics (NASDAQ: PTCT) will present a company overview at the Cantor Virtual Global Healthcare Conference on September 17, 2020, at 9:20 a.m. ET. The presentation will be available via live webcast on the PTC Therapeutics website, with an archive accessible for 30 days post-event. PTC focuses on developing innovative treatments for rare disorders, driving investment through a robust pipeline aimed at addressing unmet medical needs.
PTC Therapeutics announced a significant $20 million milestone payment from Roche triggered by the first commercial sale of Evrysdi™ (risdiplam) in the U.S. The FDA approved Evrysdi™ on August 7, 2020, for treating spinal muscular atrophy (SMA) in individuals 2 months and older. This oral treatment can be administered at home, enhancing patient accessibility during the COVID-19 pandemic. Evrysdi™ aims to improve survival motor neuron protein levels, addressing a critical unmet need in SMA care.
On August 18, 2020, PTC Therapeutics (NASDAQ: PTCT) approved non-statutory stock options for 49,165 shares and 14,175 restricted stock units (RSUs) to five new employees as part of their compensation. The stock options have an exercise price of $49.13 and vest over four years, rewarding 25% after the first year, with additional vesting every three months. The RSUs will vest similarly over four years. These inducement grants comply with NASDAQ Listing Rule 5635(c)(4) and are aimed at enhancing the company's talent acquisition strategy.
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