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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) reported full year 2024 revenue of $807 million, with Q4 revenue at $213.2 million. The company achieved all clinical and regulatory milestones, including four FDA-accepted NDA submissions.
Key financial highlights include DMD franchise Q4 revenue of $144 million, with Translarna generating $93.7 million and Emflaza $50.5 million. The company secured a significant deal with Novartis for its PTC518 Huntington's disease program in January 2025, strengthening its cash position to over $2 billion.
For 2025, PTC projects total revenues between $600-800 million, including product sales and Evrysdi royalties. The company expects GAAP R&D and SG&A expenses of $805-835 million, and non-GAAP expenses of $730-760 million for 2025.
PTC Therapeutics (NASDAQ: PTCT) has announced its executive team's participation in two major upcoming investor conferences in March 2025. The company will be featured in fireside chat sessions at the TD Cowen 45th Annual Health Care Conference on March 4 at 11:10 a.m. ET and the Barclays 27th Annual Global Healthcare Conference on March 11 at 12:30 p.m. ET.
Both presentations will be accessible through live webcasts on PTC Therapeutics' investor relations website under the Events and Presentations section. The webcasts will remain archived for 30 days after the presentations. Interested parties are advised to connect to the website several minutes before the scheduled start times to ensure proper connection.
PTC Therapeutics (NASDAQ: PTCT) announced FDA acceptance and Priority Review for its New Drug Application (NDA) for vatiquinone, targeting the treatment of children and adults with Friedreich's ataxia (FA). The FDA has set a PDUFA target action date of August 19, 2025.
The application is supported by data from the placebo-controlled MOVE-FA study and two long-term studies involving pediatric and adult FA patients. These studies demonstrated significant evidence of slowing disease progression and confirmed vatiquinone's safety and tolerability across all age groups.
If approved, vatiquinone would become the first therapy available for pediatric FA patients while offering an alternative treatment option for adults. This marks PTC's fourth FDA application acceptance in 2024.
PTC Therapeutics (NASDAQ: PTCT) has announced it will host a webcast conference call to discuss its fourth quarter and full year 2024 financial results on Thursday, February 27, at 4:30 p.m. EST. The call will also include updates on the company's business and outlook.
Participants can access the call via phone by registering through a provided link and are advised to dial in 15 minutes before the start time to avoid delays. The webcast will be accessible through PTC's Investor Relations website, with a replay available for 30 days after the call.
PTC Therapeutics (NASDAQ: PTCT) has announced the approval of 1,200 restricted stock units (RSUs) to two new employees as part of their employment compensation. The RSUs were granted on January 3, 2025, under the Nasdaq inducement grant exception and approved by PTC's Compensation Committee.
Each RSU represents the right to receive one share of common stock upon vesting. The vesting schedule spans four years, with 25% of the original shares vesting annually on each employee's hire date anniversary, contingent upon continued employment with the company.
PTC Therapeutics reported unaudited 2024 total revenue of approximately $814 million, exceeding guidance. The company's DMD franchise generated strong performance with Translarna™ revenue of ~$340 million and Emflaza® revenue of ~$207 million. The company ended 2024 with a cash balance of ~$1.1 billion.
Key developments include four FDA approval applications submitted in 2024, including Kebilidi™ (approved November 2024) and sepiapterin for PKU (decision expected July 2025). PTC closed a significant collaboration with Novartis for PTC518, receiving $1 billion upfront with potential for $1.9 billion in additional milestones.
For 2025, PTC anticipates total revenues between $600-800 million and projects GAAP R&D and SG&A expenses of $805-835 million. Important upcoming milestones include PIVOT-HD data readout and sepiapterin regulatory decisions in Q2 2025.
PTC Therapeutics (NASDAQ: PTCT) has announced that its CEO, Matthew B. Klein, M.D., will deliver a presentation at the 43rd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled for Monday, January 13, 2025, at 11:15 a.m. PST / 2:15 p.m. EST.
The event will be accessible via webcast on PTC Therapeutics' website under the Investors section's Events and Presentations page. Viewers can access the webcast at https://ir.ptcbio.com/events-presentations, and the recording will remain available for 30 days after the presentation. Attendees are advised to connect several minutes before the start time to ensure proper connection.
PTC Therapeutics (NASDAQ: PTCT) has submitted a New Drug Application (NDA) to the FDA for vatiquinone, aimed at treating children and adults with Friedreich ataxia (FA). If approved, vatiquinone would become the first authorized therapy for children with FA. The application is supported by data from the placebo-controlled MOVE-FA study and two long-term studies, demonstrating significant disease progression slowdown and safety across all age groups.
This marks PTC's fourth FDA approval application in 2024, following their successful AADC gene therapy approval in November 2024, and pending NDAs for sepiapterin (PKU treatment) and Translarna™ (Duchenne muscular dystrophy treatment).
PTC Therapeutics (NASDAQ: PTCT) has announced the approval of inducement grants for eight new employees on December 13, 2024. The grants include non-statutory stock options to purchase 1,725 shares of common stock and 5,060 restricted stock units (RSUs). The stock options were priced at $46.37 per share, matching the closing price on the grant date, and have a 10-year term. Both options and RSUs vest over four years, with options vesting 25% after one year and 6.25% quarterly thereafter, while RSUs vest 25% annually. These awards were approved by PTC's Compensation Committee as part of new hire compensation packages under Nasdaq Listing Rule 5635(c)(4).
PTC Therapeutics (NASDAQ: PTCT) has entered into a global license and collaboration agreement with Novartis for its PTC518 Huntington's disease program. The deal includes a $1.0 billion upfront payment and potential earnings of up to $1.9 billion in development, regulatory, and sales milestones. PTC will share profits in the U.S. on a 40/60 basis and receive tiered double-digit royalties on ex-U.S. sales.
Novartis will take over global development, manufacturing, and commercialization responsibilities after completing the ongoing PIVOT-HD study's placebo-controlled portion, expected in H1 2025. Interim results from June 2024 showed PTC518 achieved durable, dose-dependent reduction in HTT protein levels and demonstrated favorable safety profiles.
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