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 (PTCT) provides critical updates through this centralized news resource for investors and industry stakeholders. Track official announcements spanning clinical trial progress, regulatory milestones, and strategic partnerships in rare disease therapeutics.
Access timely updates on PTCT's small molecule developments, gene therapy advancements, and financial disclosures. Our curated feed ensures comprehensive coverage of material events including FDA submissions, research collaborations, and commercialization efforts.
This repository features verified press releases covering:
• Clinical trial results for neuromuscular and oncology programs
• Regulatory filings and orphan drug designations
• Strategic alliances with research institutions
• Financial performance and earnings communications
Bookmark this page for direct access to PTCT's evolving developments in post-transcriptional control therapeutics. Monitor breakthrough innovations addressing Duchenne muscular dystrophy, genetic disorders, and underserved patient populations.
PTC Therapeutics (NASDAQ: PTCT) released interim results from their Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. At Month 12, PTC518 showed dose-dependent reductions of mutant huntingtin (mHTT) protein in blood and cerebrospinal fluid (CSF), with 22% and 43% reductions at 5mg and 10mg doses, respectively. Additionally, clinical assessments indicated a slowing in motor symptom progression, with Total Motor Score (TMS) worsening by 2.0 points (5mg) and 1.3 points (10mg) compared to 4.9 points for placebo. Importantly, PTC518 was found to be safe and well-tolerated. Based on these findings, the FDA lifted the partial clinical hold on the program. The company will discuss these results in a conference call and webcast on June 20, 2024, at 8:00 am EDT.
PTC Therapeutics (NASDAQ: PTCT) announced its participation in William Blair's 44th Annual Growth Stock Conference on June 4, 2024, at 9:00 am ET / 8:00 am CT. The presentation will be webcast live and accessible through the Investor section of PTC Therapeutics' website. The webcast will be archived for 30 days. Attendees should join early to ensure a timely connection.
PTC Therapeutics (NASDAQ: PTCT) announced the validation and acceptance of its marketing application for sepiapterin aimed at treating PKU, by the European Medicines Agency (EMA). This marks an essential phase in making sepiapterin available in Europe. CEO Matthew B. Klein highlighted that this submission is the first of many planned for 2024, with the FDA submission expected by Q3 2024, and additional submissions in Brazil and Japan.
The sepiapterin MAA comprises data from the phase 3 APHENITY trial, which showed a 63% mean reduction in Phe levels, with 84% of participants achieving Phe control <360 µmol/L. Results also indicated dietary liberalization while maintaining Phe control. The open-label extension study confirmed the durability of sepiapterin's effects.
PTC Therapeutics (NASDAQ: PTCT) announced the European Commission's decision not to adopt the CHMP's negative opinion on the annual renewal of the marketing authorization for Translarna™ (ataluren). The authorization remains valid, allowing continued availability for patients in Europe. The EC has requested a re-evaluation, considering more comprehensive data. PTC's CEO emphasized the importance of Translarna for patients with nonsense mutation Duchenne muscular dystrophy. Due to the uncertainty of the continued authorization's impact on 2024 revenue, PTC has paused its revenue guidance, with updates to follow.
PTC Therapeutics announced the FDA's acceptance of the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy targeting AADC deficiency. The FDA has granted Priority Review status, setting a target action date of November 13, 2024. According to PTC's CEO, Matthew B. Klein, the collected data affirm Upstaza's potential transformative benefits. This acceptance marks a critical milestone in the effort to introduce an approved therapy for AADC deficiency in the U.S.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will be participating in the RBC Capital Markets 2024 Global Healthcare Conference on May 14 and the Bank of America Securities 2024 Health Care Conference on May 16. The presentations will be webcast live on the company's website and archived for 30 days.
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