NEW YORK, June 04, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it will participate in a fireside chat at the Goldman Sachs 46th Annual Global Healthcare Conference on Tuesday, June 10, 2025 at 2:00 p.m. ET. The webcast will be accessible from Royalty Pharma’s “Events” page at https://www.royaltypharma.com/investors/events/. The webcast will also be archived for a minimum of thirty days. About Royalty Pharma Founded in 1996, Royalty Pharma is the largest buyer of biopharm ...

Core Viewpoint - Roche has received approval from the European Commission for a new room-temperature stable tablet formulation of Evrysdi® (risdiplam) for the treatment of spinal muscular atrophy (SMA), enhancing treatment flexibility and convenience for patients [1][2]. Company Overview - Roche is a leading biotechnology company focused on developing innovative medicines and diagnostics, with a strong emphasis on neuroscience and chronic disease management [10][12]. Product Details - The new 5mg Evrysdi tablet can be taken with or without food, does not require refrigeration, and is suitable for individuals aged two years and older who weigh at least 20kg (44 lbs) [1][4]. - Evrysdi is designed to increase and sustain the production of SMN protein, critical for maintaining healthy motor neurons, thereby improving the course of SMA [2][6]. Clinical Development - The approval of the tablet formulation is based on a bioequivalence study demonstrating that it provides the same efficacy and safety as the original oral solution [3][8]. - Over 18,000 individuals with SMA have been treated with Evrysdi globally, highlighting its established efficacy and safety profile [2][7]. Industry Context - SMA is a severe neuromuscular disease affecting approximately one in 10,000 babies, leading to significant muscle weakness and potential fatality [9]. - Roche's commitment to neuroscience includes investigating multiple treatments for various neurological disorders, including SMA [11].

Core Viewpoint - Roche has received approval from the European Commission for a new room-temperature stable tablet formulation of Evrysdi® (risdiplam) for the treatment of spinal muscular atrophy (SMA), enhancing treatment options for patients [1][2][3]. Company Overview - Roche is a leading biotechnology company focused on developing innovative medicines and diagnostics, with a strong emphasis on neuroscience and chronic disease management [10][12]. - The company has been involved in the clinical development of Evrysdi in collaboration with the SMA Foundation and PTC Therapeutics [4][7]. Product Details - The new 5mg Evrysdi tablet can be taken with or without food, does not require refrigeration, and is suitable for individuals aged two years and older who weigh at least 20kg (44 lbs) [1][4]. - Evrysdi is designed to increase and sustain the production of SMN protein, which is critical for maintaining healthy motor neurons [5][6]. Clinical Efficacy - Over 18,000 patients have been treated with Evrysdi globally, demonstrating its proven efficacy and safety [2][7]. - The approval of the tablet formulation is based on a bioequivalence study that confirmed it provides the same efficacy and safety as the original oral solution [3][8]. Market Impact - The introduction of the tablet formulation is expected to simplify disease management for SMA patients, offering greater portability and convenience [2][3]. - Evrysdi is currently approved in more than 100 countries, indicating its widespread acceptance and potential market reach [7].

NEW YORK, May 16, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced that it has successfully closed the acquisition of its external manager, RP Management, LLC (“RP Management”). The acquisition received overwhelming support from Royalty Pharma’s shareholders, with 99.9% of votes cast in favor of the transaction. “The completion of the internalization marks an exciting new chapter for Royalty Pharma,” said Pablo Legorreta, founder and Chief Executive Officer. “It reinforces our comm ...

Core Viewpoint - Royalty Pharma plc has received overwhelming shareholder approval (99.9%) for its transition from an external management model to an integrated corporate structure, marking a significant milestone for the company [1][4]. Financial Impact - The internalization transaction is expected to generate cash savings exceeding $100 million in 2026 and over $175 million in 2030, with cumulative savings projected to exceed $1.6 billion over the next ten years [3][8]. - The total transaction value is approximately $1.1 billion, which includes 24.5 million shares of Royalty Pharma equity, $100 million in cash, and the assumption of $380 million in existing Manager debt [5][6]. Corporate Governance and Structure - The internalization is anticipated to enhance corporate governance, increase transparency, and align the leadership team more closely with shareholder interests [2][3]. - Following the transaction, all employees of the external Manager will become direct employees of Royalty Pharma, simplifying the corporate structure [7][8]. Shareholder Benefits - The acquisition is expected to strengthen shareholder alignment and ensure management continuity, with shares received by management vesting over 5 to 9 years [3][9]. - The internalization may also expand Royalty Pharma's shareholder base and enhance the company's valuation over time [3][8]. Background Information - Royalty Pharma has operated under an external management model since its founding in 1996, paying quarterly fees to the Manager based on portfolio receipts and security investments [7].

Core Insights - PTC Therapeutics reported strong revenue performance of $190 million for the first quarter of 2025, with a significant cash position exceeding $2 billion as of March 31, 2025, supporting ongoing commercial and R&D activities [1][8]. Financial Performance - Total net product and royalty revenue for Q1 2025 was $190 million, a decrease from $208.8 million in Q1 2024 [5]. - Revenue from the DMD franchise was $134 million, with Translarna™ contributing $86 million and Emflaza® contributing $48 million [5]. - Collaboration and license revenue included $986.2 million from the PTC518 agreement with Novartis, which closed in January 2025 [5][7]. - Net income for Q1 2025 was $866.6 million, compared to a net loss of $91.6 million in Q1 2024 [8]. Regulatory and Clinical Updates - Positive CHMP opinion for Sephience™ (sepiapterin) received on April 25, 2025, with an expected European Commission adoption in approximately two months [5]. - NDA for Sephience is under review by the FDA, with a target action date of July 29, 2025 [5]. - NDA for vatiquinone accepted and granted Priority Review by the FDA, with a target action date of August 19, 2025 [5]. - Phase 2 PIVOT-HD study for PTC518 met primary endpoint of dose-dependent blood HTT lowering at Week 12, with favorable safety and tolerability [5]. Guidance and Future Outlook - PTC anticipates full-year 2025 revenue between $650 million and $800 million, including in-line products and potential new launches [8]. - Full-year 2025 GAAP R&D and SG&A expenses are projected to be between $805 million and $835 million [8]. - Non-GAAP R&D and SG&A expenses are expected to be between $730 million and $760 million, excluding estimated non-cash, stock-based compensation of $75 million [8].

NEW YORK, April 08, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (NASDAQ:RPRX) today announced the appointment of Vlad Coric, M.D. to the company's Board of Directors, effective immediately. Vlad Coric is the Chairman and Chief Executive Officer of Biohaven, a biopharmaceutical company focused on the discovery, development and commercialization of life-changing treatments in key therapeutic areas including neuroscience, immunology and oncology. "I am excited to announce that we are strengthening our Board wi ...

NEW YORK, April 08, 2025 (GLOBE NEWSWIRE) -- Royalty Pharma plc (Nasdaq: RPRX) today announced the appointment of Vlad Coric, M.D. to the company’s Board of Directors, effective immediately. Vlad Coric is the Chairman and Chief Executive Officer of Biohaven, a biopharmaceutical company focused on the discovery, development and commercialization of life-changing treatments in key therapeutic areas including neuroscience, immunology and oncology. “I am excited to announce that we are strengthening our Board w ...