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PTC Therapeutics Announces Vatiquinone NDA Submission to FDA for the Treatment of Children and Adults Living with Friedreich Ataxia
Prnewswire· 2024-12-19 13:00
- If approved, vatiquinone would be the first and only authorized therapy for children with FA -- PTC's fourth approval application submitted to FDA in 2024 -WARREN, N.J., Dec. 19, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today the submission of the vatiquinone New Drug Application (NDA) for the treatment of children and adults living with Friedreich ataxia (FA) to the U.S. Food and Drug Administration (FDA)."We are excited to have reached this important milestone in the developm ...
PTC Partners with Microsoft and Volkswagen Group to Develop Codebeamer Generative AI Copilot
Prnewswire· 2024-12-03 13:30
Collaboration brings together industry leaders to advance generative AI use cases for manufacturing Codebeamer Copilot expected to simplify requirements management as part of the product development process Codebeamer Copilot beta expected to release in early 2025BOSTON, Dec. 3, 2024 /PRNewswire/ -- PTC (NASDAQ: PTC) today announced that it has partnered with Microsoft and Volkswagen Group to develop a generative artificial intelligence (AI) copilot based on the PTC Codebeamer® application lifecycle manage ...
PTC Therapeutics Stock Soars on Novartis Licensing Agreement
Investopedia· 2024-12-02 18:50
Key TakeawaysShares of PTC Therapeutics soared Monday when the pharmaceutical firm announced it struck a nearly $2 billion license and collaboration deal with Novartis.The deal gives Novartis global development, manufacturing, and commercial responsibility for PTC's experimental Huntington's Disease drug.PTC will receive $1.0 billion upfront, and said it would use the money to expand its splicing platform and increase its commercial and development portfolio moves. Shares of PTC Therapeutics (PTCT) soared 1 ...
PTC Therapeutics Inks Licensing Pact With Novartis Worth Up To ~$3 Billion For Huntington's Program
Benzinga· 2024-12-02 17:58
On Monday, PTC Therapeutics, Inc. PTCT signed an exclusive global license and collaboration agreement with Novartis AG NVS for its PTC518 Huntington’s disease program, which includes related molecules.Under the agreement, PTC will receive an upfront payment of $1.0 billion, up to $1.9 billion in development, regulatory and sales milestones, a profit share in the U.S., and double-digit tiered royalties on ex-U.S. sales.Also Read: FDA Grants Conditional Approval For PTC Therapeutics’ Gene Therapy As First Eve ...
PTC Therapeutics Enters into a Global License and Collaboration Agreement with Novartis for PTC518 Huntington's Disease Program
Prnewswire· 2024-12-02 12:00
- PTC to receive $1.0B in cash at closing -- PTC is eligible to receive up to $1.9B in development, regulatory and sales milestones -- PTC to share profits in the U.S. and tiered double-digit royalties on ex-U.S. net sales -- Novartis will assume global development, manufacturing and commercial responsibilities following completion of placebo-controlled portion of ongoing PIVOT-HD study -- PTC will host a conference call on Dec. 2, 2024, at 8:30 am EST- WARREN, N.J., Dec. 2, 2024 /PRNewswire/ -- PTC Therape ...
PTC Therapeutics Enters into Agreement to Sell Priority Review Voucher for $150 Million
Prnewswire· 2024-11-27 12:00
Core Viewpoint - PTC Therapeutics, Inc. has announced an agreement to sell its Rare Pediatric Disease Priority Review Voucher for $150 million, contingent upon the closing of the transaction [1][2]. Group 1: Transaction Details - The Rare Pediatric Disease Priority Review Voucher was granted to PTC on November 13, 2024, alongside FDA approval for KEBILIDI™ for treating AADC deficiency [2]. - The transaction is subject to customary closing conditions, including the expiration of the applicable waiting period under the Hart-Scott Rodino Antitrust Improvements Act [2]. Group 2: About the PRV Program - The FDA awards priority review vouchers to sponsors of rare pediatric disease product applications that meet specific criteria, aimed at encouraging the development of new drugs for rare pediatric diseases [3]. Group 3: Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing clinically differentiated medicines for rare disorders [4]. - The company aims to provide access to best-in-class treatments for patients with limited treatment options, leveraging its scientific expertise and global commercial infrastructure [4].
PTC Therapeutics Announces Topline Results of CardinALS Trial of Utreloxastat in ALS Patients
Prnewswire· 2024-11-26 21:30
Core Insights - PTC Therapeutics announced that the Phase 2 CardinALS study did not meet its primary endpoint of slowing disease progression in ALS patients, with a p-value of 0.52 indicating lack of statistical significance [1][2] - Despite showing a modest numerical benefit and correlation with a biomarker (NfL), the study's secondary efficacy endpoints also did not achieve significance [1][2] - Utreloxastat was found to be safe and well tolerated, but due to the lack of efficacy, further development is not planned [2] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, aiming to provide access to treatments for patients with limited options [3] - The company's strategy leverages scientific expertise and global infrastructure to maximize value for stakeholders [3]
PTC Therapeutics Announces FDA Approval of AADC Deficiency Gene Therapy
Prnewswire· 2024-11-13 23:02
Core Points - The FDA has granted accelerated approval for PTC Therapeutics' gene therapy KEBILIDI™ for AADC deficiency, marking the first gene therapy approved for direct brain administration in the U.S. [1][4] - KEBILIDI is indicated for both children and adults, addressing a rare genetic disorder that severely impacts motor function due to dopamine synthesis inability [3][8]. - The therapy is delivered via a minimally invasive stereotactic neurosurgical procedure, with trained surgeons and specialized centers prepared for its administration [7][8]. Company Overview - PTC Therapeutics is focused on developing and commercializing innovative treatments for rare disorders, leveraging scientific expertise and a global commercial infrastructure [14]. - The company aims to maximize value for stakeholders by providing access to transformative medicines for patients with limited treatment options [14]. Product Details - KEBILIDI is a recombinant adeno-associated virus-based gene therapy designed to correct the genetic defect causing AADC deficiency by delivering a functioning DDC gene directly into the brain [6]. - Clinical trials have shown that KEBILIDI leads to the synthesis of dopamine and improvement in motor development milestones [3][4]. Regulatory Milestones - The approval was based on safety and efficacy findings from an ongoing global clinical trial (PTC-AADC-GT-002), with further confirmatory evidence expected from long-term patient follow-up [4]. - A Rare Disease Priority Review Voucher was also granted, which the company plans to monetize [4].
PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2024 Financial Results
Prnewswire· 2024-11-07 21:01
– Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA –– On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone –WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024. "I am proud of ...
PTC's Q4 Earnings Surpass Estimates, Revenues Rise Y/Y, Shares Jump
ZACKS· 2024-11-07 14:35
PTC Inc (PTC) reported fourth-quarter fiscal 2024 non-GAAP earnings per share (EPS) of $1.54, up 28% year over year. The figure beat the Zacks Consensus Estimate by 7.7%.Stay up-to-date with all quarterly releases: See Zacks Earnings Calendar.Quarterly revenues soared 15% year over year to $627 million. The Zacks Consensus Estimate for revenues was pegged at $620.5 million. The top line neared the high end of PTC’s guidance, driven by continued demand for its product lifecycle management (PLM) and computer- ...