- 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 ...

Core Viewpoint - The European Commission has decided not to renew the authorization of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy, although individual EU member states may still allow its use under specific provisions [1][2]. Company Overview - PTC Therapeutics, Inc. 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 [5]. Product Information - Translarna (ataluren) is a protein restoration therapy aimed at enabling the formation of functional proteins in patients with genetic disorders caused by nonsense mutations, specifically targeting Duchenne muscular dystrophy [3]. - Translarna is licensed in multiple countries for treating nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged 2 years and older, while it remains an investigational new drug in the United States [3]. Disease Context - Duchenne muscular dystrophy is a rare and fatal genetic disorder primarily affecting males, leading to progressive muscle weakness and premature death due to heart and respiratory failure, with the absence of functional dystrophin protein being a critical factor [4].

— Results show 97% of phenylalanine (Phe) tolerance study participants were able to increase their dietary Phe intake with a mean increase of 126% — — Genetic variant analysis of Phase 3 APHENITY trial demonstrates meaningful effect in classical subjects with non-BH4 responsive genotypes — WARREN, N.J., March 20, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today shared new data being presented from the Phase 3 APHENITY trial and subsequent open-label extension study at the 2025 American Colle ...

PTC Therapeutics (PTCT) Q4 2024 Earnings Call February 28, 2025 03:30 AM ET Company Participants Ellen Cavaleri - Head of Investor RealtionsMatthew Klein - President & CEOEric Pauwels - Chief Business OfficerPierre Gravier - Chief Financial OfficerKelly Shi - Senior Vice PresidentTiago Fauth - Director - Equity Research Small/Mid Cap BiotechnologyEllie Merle - Executive DirectorGena Wang - MD - Biotech Equity ResearchPeyton Bohnsack - Biotechnology Equity Research - Vice PresidentTazeen Ahmad - MD - US Equi ...

For the quarter ended December 2024, PTC Therapeutics (PTCT) reported revenue of $213.17 million, down 30.6% over the same period last year. EPS came in at -$0.24, compared to -$0.24 in the year-ago quarter.The reported revenue represents a surprise of -3.96% over the Zacks Consensus Estimate of $221.96 million. With the consensus EPS estimate being -$0.96, the EPS surprise was +75.00%.While investors closely watch year-over-year changes in headline numbers -- revenue and earnings -- and how they compare to ...

Core Insights - PTC Therapeutics reported a quarterly loss of $0.24 per share, significantly better than the Zacks Consensus Estimate of a loss of $0.96, representing a 75% earnings surprise [1] - The company generated revenues of $213.17 million for the quarter ended December 2024, missing the Zacks Consensus Estimate by 3.96% and down from $307.06 million year-over-year [2] - PTC Therapeutics has outperformed the S&P 500 with a 12.9% increase in share price since the beginning of the year [3] Earnings Outlook - The current consensus EPS estimate for the upcoming quarter is -$1.22 on revenues of $168.72 million, and for the current fiscal year, it is -$4.49 on revenues of $738.12 million [7] - The estimate revisions trend for PTC Therapeutics is mixed, resulting in a Zacks Rank 3 (Hold), indicating expected performance in line with the market [6] Industry Context - The Medical - Biomedical and Genetics industry, to which PTC Therapeutics belongs, is currently in the top 28% of over 250 Zacks industries, suggesting a favorable outlook compared to lower-ranked industries [8] - Another company in the same industry, Zymeworks Inc., is expected to report a quarterly loss of $0.32 per share, reflecting a year-over-year change of -60%, with revenues projected at $36.74 million, up 117% from the previous year [9][10]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-K (Mark One) ☑ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended: December 31, 2024 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 Commission file number: 001-35969 PTC THERAPEUTICS, INC. (Exact name of registrant as specified in its charter) Delaware 04-3416587 (State or other jurisdiction of incorporati ...

Financial Performance - Unaudited total revenue for 2024 was approximately $814 million, exceeding guidance[1] - Unaudited net product revenue for 2024 was approximately $601 million, driven by the DMD franchise with $340 million from Translarna and $207 million from Emflaza[3] - Cash balance as of December 31, 2024, was approximately $1.1 billion, with an additional $1.0 billion from the PTC518 collaboration agreement with Novartis[3] - PTC anticipates approximately $211 million in royalty revenue from Evrysdi for the full year 2024[8] Future Projections - PTC expects total revenues for 2025 to be between $600 million and $800 million, including in-line products and potential new product launches[8] - Anticipated GAAP R&D and SG&A expenses for 2025 are projected to be between $805 million and $835 million[8] Regulatory Approvals - Four regulatory approval applications were submitted to the FDA in 2024, including sepiapterin for PKU with an FDA action date of July 29, 2025[3] - CHMP opinion on sepiapterin MAA is expected in Q2 2025[8] Collaborations and Milestones - PTC signed a global license and collaboration agreement with Novartis for PTC518, with potential milestone payments up to $1.9 billion[3] - Results from the PIVOT-HD Phase 2 study of PTC518 are expected in Q2 2025[8]

Core Insights - Analysts project that PTC Therapeutics (PTCT) will report a quarterly loss of $0.96 per share, reflecting a 300% decline year over year [1] - Revenue is expected to reach $252.51 million, a decrease of 17.8% from the same quarter last year [1] - The consensus EPS estimate has remained unchanged over the last 30 days, indicating a reevaluation of initial estimates by analysts [1][2] Revenue Projections - Analysts estimate that 'Revenues- Net product revenue' will be $141.85 million, showing an 8.5% decline from the previous year [4] - 'Revenues- Royalty revenue' is projected to reach $73.16 million, indicating a significant increase of 43.4% year over year [4] - 'Revenues- Net product revenue- Emflaza' is expected to be $50.95 million, reflecting a 24.4% decrease [5] - 'Revenues- Net product revenue- Translarna' is projected at $78.77 million, showing a 4.8% increase from the same quarter last year [5] Market Performance - PTC Therapeutics shares have increased by 9.1% over the past month, contrasting with a -0.5% change in the Zacks S&P 500 composite [6] - The company holds a Zacks Rank 3 (Hold), suggesting it is expected to closely follow overall market performance in the near term [6]

Company Overview - PTC Therapeutics is expected to report a quarterly loss of $0.96 per share, reflecting a year-over-year change of -300% [3] - Revenues are anticipated to be $252.51 million, down 17.8% from the same quarter last year [3] Earnings Expectations - The earnings report is scheduled for release on February 27, 2025, and could influence stock movement based on actual results compared to estimates [2] - The consensus EPS estimate has remained unchanged over the last 30 days, indicating stability in analyst expectations [4] Earnings Surprise Prediction - The Most Accurate Estimate for PTC Therapeutics is higher than the Zacks Consensus Estimate, resulting in an Earnings ESP of +12.84%, suggesting a bullish outlook from analysts [10] - The stock currently holds a Zacks Rank of 3, indicating a likelihood of beating the consensus EPS estimate [11] Historical Performance - In the last reported quarter, PTC Therapeutics was expected to post a loss of $1.54 per share but delivered a loss of $1.39, resulting in a surprise of +9.74% [12] - Over the past four quarters, the company has beaten consensus EPS estimates two times [13] Industry Context - Editas Medicine, another player in the Zacks Medical - Biomedical and Genetics industry, is expected to post a loss of $0.39 per share, indicating a year-over-year change of -69.6% [17] - Editas Medicine's revenues are projected to be $37.87 million, down 36.9% from the previous year [17]