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 announced that the FDA and European Commission have granted Orphan Drug Designation (ODD) for PTC923, aimed at treating hyperphenylalaninemia, primarily caused by Phenylketonuria (PKU). PKU can lead to severe cognitive disabilities and seizures. PTC923, an oral synthetic sepiapterin, is expected to enter a Phase 3 trial, APHENITY, in mid-2021. ODD provides various benefits, including grant funding, tax advantages, and potential market exclusivity in both the U.S. and EU.
PTC Therapeutics, based in South Plainfield, N.J., will present a company overview at the William Blair 41st Annual Growth Stock Conference on June 2, 2021, at 3:40 p.m. ET. The presentation will be accessible via a live webcast on their website and available for archiving for 30 days post-event. PTC is dedicated to developing innovative treatments for rare disorders and emphasizes maximizing patient access to its medicines.
PTC Therapeutics, Inc. (NASDAQ: PTCT) revealed findings on the impact of caring for children with aromatic L-Amino acid decarboxylase deficiency (AADC-d) at the Virtual ISPOR North America 2021 Conference. The study highlights that caregivers dedicate an average of 105 hours weekly to support, often leading 75% to leave jobs or reduce hours. The findings underscore the critical need for effective treatments, as AADC-d causes severe disabilities and requires constant care. Data also show a positive correlation between treatment efficacy and improved health outcomes for affected children.
PTC Therapeutics (PTCT) reported a strong Q1 2021 with total revenues of $117.9 million, a 32% increase from Q1 2020. The Duchenne muscular dystrophy (DMD) franchise revenue reached $90 million, driven by new patient uptake and geographic expansion. Preliminary results from the PTC518 trial showed a positive dose-dependent reduction in Huntington mRNA. However, the net loss for Q1 2021 was $128.6 million, compared to $112.7 million a year earlier. PTC reaffirms its 2021 guidance, projecting DMD net product revenues between $355 and $375 million.
PTC Therapeutics (NASDAQ: PTCT) has announced its participation in several upcoming virtual investor conferences. The presentations include:
- BofA Securities 2021 Virtual Health Care Conference on May 11 at 8:45 a.m. ET
- 2021 RBC Capital Markets Global Healthcare Virtual Conference on May 18 at 10:20 a.m. ET
- 2021 UBS Global Healthcare Virtual Conference on May 26 at 12:00 p.m. ET
These presentations will be available live and archived on PTC's investor relations website for 30 days.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on May 4, 2021, at 4:30 p.m. (ET) to discuss its first quarter 2021 financial results. Investors can access the call by dialing (877) 303-9216 domestically or (973) 935-8152 internationally. The call will be available for replay on the company’s website for 30 days after the event. PTC emphasizes its focus on developing differentiated medicines for rare disorders, reinforcing its commitment to providing best-in-class treatments for patients with unmet medical needs.
PTC Therapeutics (NASDAQ: PTCT) has granted stock options for 75,085 shares and 33,740 restricted stock units (RSUs) to 79 new employees as a part of their compensation package. Approved on April 13, 2021, these inducement grants comply with NASDAQ Listing Rule 5635(c)(4). The options have an exercise price of $47.31, based on the stock's closing price on the grant date. The vesting period spans four years, with a portion vesting annually and quarterly, contingent on continued employment.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced positive results from Part 2 of the pivotal FIREFISH trial for Evrysdi™ (risdiplam) in infants with type 1 spinal muscular atrophy (SMA). After 24 months, 93% of infants were alive, with 83% surviving without permanent ventilation. Key motor milestones improved significantly, such as sitting without support (from 29% at 1 year to 61% at 2 years). The treatment was associated with fewer hospitalizations, and safety profiles remained consistent. Evrysdi promotes SMN protein production, crucial for healthy motor neuron function.
PTC Therapeutics (NASDAQ: PTCT) has launched the PTC Pinpoint Direct™-CP Spectrum, a no-charge genetic testing program for individuals in the U.S. with symptoms of cerebral palsy (CP) but without evidence of brain injury. This initiative aims to reduce misdiagnosis and connect patients with potential genetic disorders, which may have existing treatments. The program collaborates with Genome Medical for genetic counseling and Invitae for genetic testing, enhancing patient access to care and eliminating unnecessary treatments.
PTC Therapeutics, Inc. (NASDAQ: PTCT) has been awarded the 2021 Don Clifton Strengths-Based Culture Award by Gallup. This recognition honors organizations that excel in developing engaging workplace cultures focused on employee strengths. CEO Stuart Peltz emphasized the company's commitment to a collaborative environment, especially amid challenges posed by the COVID-19 pandemic. PTC is dedicated to discovering and commercializing innovative medicines for rare disorders, leveraging its scientific expertise and robust global commercial infrastructure.
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