WARREN, N.J., Dec. 23, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that CEO Matthew B. Klein, M.D., will present at the 43rd Annual J.P. Morgan Healthcare Conference on Monday, January 13, 2025, at 11:15 a.m. PST / 2:15 p.m. EST.The presentation will be webcast live on the Events and Presentations page under the Investors section of PTC Therapeutics' website at https://ir.ptcbio.com/events-presentations and will be archived for 30 days following the presentation. It is recomme ...

Industry Overview - The fourth industrial revolution is characterized by the integration of smart digital technologies that work together iteratively in real time, greatly benefiting the manufacturing industry [16] - The first industrial revolution was driven by steam power and mechanical production, the second by electricity and mass production, and the third by the information age (semiconductors, computing, and the internet) [2] - Spending on digital transformation is expected to grow at a 16.2% compound annual growth rate from 2022 to 2027, reaching $3.9 trillion compared to $1.9 trillion in 2022 [5] PTC's Role in the Industry - PTC's software plays a pivotal role in the fourth industrial revolution, particularly through its closed-loop digital thread, which gathers data across a product's entire life cycle to improve processes iteratively [3][16] - The company's software suite includes computer-aided design (CAD), product life-cycle management (PLM), Internet of Things (IoT), augmented reality (AR), and software life cycle management (SLM) solutions [10][17] - PTC's software enables manufacturers to shorten development timelines while maintaining high quality, integrating hardware, mechanical, electronic, and embedded software processes [18] PTC's Growth Strategy - PTC is refocusing on its five key industry verticals: industrial products, aerospace and defense, electronics and technology, automaking, and medical technology and life sciences to drive ARR growth [19] - The company recently appointed Robert Dahdah, former Microsoft vice president, as chief revenue officer to lead sales and customer success for its Digital Thread group and new market strategy [13] - PTC's annual run rate (ARR), which includes subscription software, SaaS, and support contracts, continues to show double-digit growth despite economic challenges [18] Market Potential - The increasing adoption of digital technologies is expected to amplify the value PTC provides to its manufacturing customers, leading to significant growth for the company in the medium and long term [15] - Industry forecasters expect PTC to grow at a mid-teens rate over the next decade, driven by the ongoing investment in digital technology and the fourth industrial revolution [20] - Lower interest rates and a potential pick-up in industrial spending are seen as additional benefits for PTC's growth prospects [14]

- 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 ...

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 ...

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 ...

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 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 ...

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].

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]

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].