Dublin, July 22, 2025 (GLOBE NEWSWIRE) -- The "Catalyst Monitor: Q3 2025 Outlook" report has been added to ResearchAndMarkets.com's offering.The report delivers forward-looking intelligence derived from interviews with key opinion leaders (KOLs), covering trial initiations, completions, top-line results, regulatory filings, PDUFA dates, and expected approvals. The report covers 18 significant catalyst events that are expected to occur in Q3 2025. Some of the catalysts covered in the report, include FDA app ...

Core Viewpoint - PTC Therapeutics has received marketing authorization from the European Commission for Sephience™ (sepiapterin) to treat phenylketonuria (PKU) in patients of all ages and disease severities, marking a significant advancement in treatment options for this rare metabolic disorder [1][2][3] Group 1: Product Information - Sephience™ (sepiapterin) is indicated for the treatment of hyperphenylalaninaemia (HPA) in both adult and pediatric patients with PKU, acting as a natural precursor of the enzymatic co-factor BH4, which is essential for the enzyme phenylalanine hydroxylase (PAH) [4] - The product functions as a dual pharmacological chaperone, improving the activity of defective PAH enzymes and effectively reducing blood phenylalanine (Phe) levels [4] - The European launch of Sephience will begin in Germany in the first half of July 2025, with ongoing review for approval in other countries including Japan and Brazil [3] Group 2: Clinical Evidence - The European approval is based on statistically significant results from the Phase 3 APHENITY trial, demonstrating a durable treatment effect and allowing participants to liberalize their diet in the long-term extension study [2] Group 3: Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on developing and commercializing medicines for rare disorders, leveraging scientific expertise and a global commercial infrastructure to maximize value for patients [6] - The company aims to provide access to best-in-class treatments for patients with unmet medical needs, supported by a robust and diversified pipeline of transformative medicines [6]

PTC Therapeutics June 2025 Matthew B. Klein, MD CEO Pam and Kelsey living with PKU 1 Corporate Presentation – June 2025 Forward Looking Statements This presentation contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this presentation, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the ...

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].

PTC Therapeutics, Inc. PTCT on Monday announced results from the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Stage 2 and Stage 3 Huntington’s disease (HD) patients.Huntington’s disease is a progressive neurodegenerative disorder caused by mutation of the huntingtin (HTT) gene, leading to the breakdown of brain cells. It is characterized by uncontrolled movements (chorea), cognitive decline, and psychiatric disturbances.The study met its primary endpoint of reduction in blood Huntingtin (HTT) protein leve ...

- Label includes full spectrum of PKU patients - - European launch preparations underway - WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusi ...

- Label includes full spectrum of PKU patients -- European launch preparations underway -WARREN, N.J., April 25, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has issued a positive opinion on the marketing authorization application for Sephience™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The opinion includes a broad label inclusive ...

Financial Data and Key Metrics Changes - Fourth quarter revenue totaled $213 million, and full year 2024 revenue was $807 million, exceeding guidance [8][31] - Cash, cash equivalents, and marketable securities totaled approximately $1.1 billion as of December 31, 2024, compared to $877 million as of December 31, 2023 [35] Business Line Data and Key Metrics Changes - DMD franchise revenue for the full year 2024 was $547 million, with Translarna net product revenue of $94 million and Emflaza net product revenue of $50 million in the fourth quarter [32][33] - The company achieved all clinical and regulatory milestones on schedule, including four FDA approval applications submitted [12][11] Market Data and Key Metrics Changes - The US represents the largest opportunity for Sepiapterin, with approximately 17,000 PKU patients, most of whom are not on medical treatments [23] - The estimated prevalence of Friedreich's ataxia in the US is about 6,000 patients, with one-third being pediatric [27] Company Strategy and Development Direction - The company plans to support 2025 commercial launches and continue investing in R&D platforms while exploring business development opportunities [10][11] - Anticipated milestones for 2025 include the global launch of Sepiapterin and potential launch of Vatiquinone in the US [15][16] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the company's strong financial position and ability to reach cash flow breakeven without raising additional capital [10][11] - The management highlighted the excitement surrounding Sepiapterin due to its potential to address significant unmet needs in the PKU patient population [15] Other Important Information - The company received a $1 billion upfront payment from Novartis as part of the PTC518 collaboration, with potential for up to $1.9 billion in development and sales milestones [13][35] - The company is preparing for multiple new product launches throughout the year, leveraging its experienced global commercial infrastructure [29] Q&A Session Summary Question: What is the expected impact of diet liberalization for PKU patients? - Management highlighted that over 97% of patients in the feed protocol can liberalize their diet, with many achieving or exceeding the recommended daily allowance of protein [40][41] Question: What is the market opportunity for Friedreich's ataxia? - Management expressed excitement about the opportunity to provide therapy for all FA patients, including pediatric patients, and emphasized the strong safety profile of the treatment [45][46] Question: What is the visibility on the Translarna review process? - Management indicated that the review is ongoing, and while they do not expect a formal PDUFA date, they anticipate more information in the first half of the year [57] Question: How should investors frame expectations for the PIVOT-HD update? - Management stated that the update will include both stage 2 and stage 3 patients, with a focus on biomarker data and clinical outcome scales [68][69] Question: How is the company preparing for the Sepiapterin launch? - Management noted that they are actively engaging with key stakeholders and preparing for a strategic launch sequence targeting access for PKU patients [22][24]