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]

Core Points - PTC Therapeutics has announced that the FDA accepted the New Drug Application (NDA) for vatiquinone, targeting a PDUFA action date of August 19, 2025, for the treatment of Friedreich's ataxia [1][2] - Vatiquinone, if approved, would be the first therapy for pediatric patients with Friedreich's ataxia and aims to provide a safe and effective treatment alternative for adults [2] - The NDA is based on data from the placebo-controlled MOVE-FA study and two long-term studies, demonstrating significant evidence of slowing disease progression and safety across all age groups [2][5] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing medicines for rare disorders [7] - The company has submitted four approval applications to the FDA in 2024, all of which have been accepted for review [3] Product Information - Vatiquinone is a first-in-class selective inhibitor of 15-Lipoxygenase, targeting pathways disrupted in Friedreich's ataxia, and has shown potential in alleviating mitochondrial dysfunction and oxidative stress [4] - The MOVE-FA trial enrolled 146 patients, primarily under 18 years, and while the primary endpoint did not reach statistical significance, significant effects were observed on specific subscales [5] Disease Context - Friedreich's ataxia is a rare, debilitating neuromuscular disorder affecting approximately 25,000 people globally, characterized by progressive loss of coordination and muscle strength [6]

Group 1: Company Overview - PTC Therapeutics (PTCT) shares increased by 9% to close at $50.01, following a notable trading volume compared to typical sessions, contrasting with a 1.4% loss over the past four weeks [1] - Cantor Fitzgerald raised its price target for PTC Therapeutics from $76 to $113, maintaining an Overweight rating, driven by confidence in the FDA approval potential for sepiapterin to treat phenylketonuria, with a decision expected by July 29, 2025 [2] - If approved, sepiapterin could achieve peak annual sales of $1.5 billion in the U.S. and EU [2] Group 2: Financial Performance Expectations - PTC Therapeutics is projected to report a quarterly loss of $1 per share, reflecting a year-over-year change of -316.7%, with expected revenues of $247.89 million, down 19.3% from the previous year [3] - The consensus EPS estimate for the upcoming quarter has been revised 3.8% lower over the last 30 days, indicating a negative trend in earnings estimate revisions, which typically does not lead to price appreciation [4] Group 3: Industry Context - PTC Therapeutics operates within the Zacks Medical - Biomedical and Genetics industry, where Aligos Therapeutics, another company in the same sector, experienced a 12.5% decline in its stock price [4] - Aligos Therapeutics has a consensus EPS estimate of -$2.50 for its upcoming report, representing a 54.6% change from the previous year, and also holds a Zacks Rank of 3 (Hold) [5]

Core Viewpoint - PTC Therapeutics has entered into an exclusive global licensing and collaboration agreement with Novartis for its investigational oral therapy PTC518 for Huntington's disease, resulting in a nearly 19% increase in PTC's shares [1][4]. Group 1: Licensing Agreement Details - Novartis will develop and market PTC518 after the ongoing phase II PIVOT-HD study, expected to complete in the first half of next year [2]. - PTC Therapeutics will receive an upfront cash payment of $1 billion and may earn up to $1.9 billion in milestone payments from Novartis [2]. - Profit and loss sharing in the U.S. will be in a 40:60 ratio (PTC:Novartis), while Novartis will handle ex-U.S. sales, with PTC eligible for tiered double-digit royalties on those sales [3]. Group 2: Stock Performance and Market Reaction - The licensing deal has positively impacted PTC Therapeutics' stock, which has surged 88.9% year-to-date, contrasting with a 5.2% decline in the industry [6]. - Management plans to utilize proceeds from the deal to enhance its drug development platform and commercial activities [4]. Group 3: Recent Developments and Challenges - The FDA granted accelerated approval to PTC's gene therapy Kebilidi for AADC deficiency, marking a significant milestone as the first gene therapy directly administered to the brain in the U.S. [8]. - PTC has resubmitted a regulatory filing for Translarna in nmDMD, following a previous complete response letter in 2016 [9][10]. - A mid-stage study for PTC's investigational ALS drug utreloxastat did not meet its primary and secondary endpoints, indicating some setbacks for the company [11].

Financial Data and Key Metrics Changes - Total revenue for Q3 2024 was $197 million, with $124 million coming from the DMD franchise, including $72 million from Translarna and $52 million from Emflaza [6][39] - The company raised its total revenue guidance for 2024 to a range of $750 million to $800 million, reflecting strong performance [7][38] - Cash, cash equivalents, and marketable securities totaled $1.0 billion as of September 30, 2024, up from $877 million at the end of 2023 [42] Business Line Data and Key Metrics Changes - The DMD franchise generated $124 million in revenue, with Emflaza contributing $52 million [20][39] - The company submitted two NDAs to the FDA for sepiapterin and Translarna, with sepiapterin's action date set for July 29, 2025 [8][10] - Vatiquinone NDA submission is planned for December 2024, targeting treatment for Friedreich ataxia [12][35] Market Data and Key Metrics Changes - The company reported strong support for Translarna in Europe despite a recent negative opinion from CHMP, with ongoing efforts to maintain availability [21] - In Latin America, the company is expanding its presence with new orders for Translarna and Tegsedi [37] Company Strategy and Development Direction - The company is focused on launching sepiapterin globally in 2025, targeting a significant unmet need in PKU patients [25][27] - The strategy includes leveraging existing rare disease infrastructure to maximize access and maintain a premium pricing strategy for sepiapterin [32][34] - The company is preparing for multiple product launches in 2025, including AADC Gene Therapy, Translarna, sepiapterin, and vatiquinone [38] Management's Comments on Operating Environment and Future Outlook - Management expressed optimism about the upcoming FDA meetings regarding Huntingtin lowering as a surrogate endpoint for accelerated approval [62] - The company is confident in the potential of sepiapterin to address high unmet needs in PKU patients, with a projected $1 billion opportunity in the US alone [25][74] - Management highlighted the importance of the recent Lancet publication in raising awareness and excitement among healthcare providers regarding sepiapterin [58] Other Important Information - The company received Fast Track Designation from the FDA for PTC518, aimed at Huntington's disease [14] - The AADC Gene Therapy BLA has a scheduled FDA action date of November 13, 2024, which could lead to a new treatment approach for brain diseases [19] Q&A Session Summary Question: Update on Type C meeting regarding Huntingtin's - Management indicated that discussions will focus on endpoints for an efficacy trial and the potential for Huntingtin lowering as a surrogate endpoint, with clarity expected post-meeting [44] Question: Regulatory flexibility for Translarna - Management discussed the unique efficacy data from Study 041 and the STRIDE registry, emphasizing the compelling package of evidence for Translarna [45] Question: CardinALS trial endpoint changes - Management clarified that the change in primary endpoint was to include deaths in the analysis, as recommended by the FDA, and that enrollment adjustments were made based on screening yields [48] Question: Q4 revenue expectations - Management expects continued strong contributions from the DMD franchise, with stable revenue from Translarna and ongoing performance from Emflaza [51] Question: Sepiapterin provider and payer reactions - Management reported positive feedback from providers and payers regarding the APHENITY data, indicating a willingness to pay a premium price due to the high unmet need [57][59] Question: Translarna sales and European Commission decision - Management noted that approximately 46% of Translarna revenue comes from Europe, which could be at risk depending on the European Commission's decision [67] Question: Vatiquinone payer discussions - Management stated that discussions are still early, but there is a clear unmet need for pediatric and adolescent patients, with a strong data package supporting its efficacy [83]