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PTC Therapeutics (PTCT) came out with a quarterly loss of $0.83 per share versus the Zacks Consensus Estimate of a loss of $1.07. This compares to a loss of $1.16 per share a year ago. These figures are adjusted for non-recurring items.This quarterly report represents an earnings surprise of +22.43%. A quarter ago, it was expected that this biopharmaceutical company would post a loss of $1.08 per share when it actually produced earnings of $10.04, delivering a surprise of +1029.63%.Over the last four quarte ...

Financial Data and Key Metrics Changes - Total revenue for the second quarter was $179 million, with contributions from the DMD franchise amounting to $96 million [4][19] - Cash, cash equivalents, and marketable securities totaled approximately $1.989 billion as of June 30, 2025, compared to $1.14 billion as of December 31, 2024 [20] Business Line Data and Key Metrics Changes - DMD franchise revenue included $59 million from Translarna and $36 million from Emflaza [19] - The company expects to maintain approximately 25% of European revenue from prior to the nonrenewal of Translarna's conditional marketing authorization [5][28] Market Data and Key Metrics Changes - The EU approval of SUFIANCE in late June included a broad label for all disease subtypes and age groups, with FDA approval following shortly after [5][6] - The company anticipates SUFIANCE revenue opportunity in the U.S. to exceed $1 billion [6] Company Strategy and Development Direction - SUFIANCE is positioned as the foundational product for sustainable growth and profitability [4] - The company plans to leverage early access mechanisms in Europe while formal pricing and reimbursement discussions proceed [6][17] - The company is exploring business development opportunities to enhance its commercial portfolio and pipeline for long-term growth [20][98] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the market opportunity for SUFIANCE, highlighting strong initial feedback from healthcare providers [17][18] - The company remains in a strong financial position, allowing it to support planned commercial and R&D initiatives without the need for additional capital [8][20] Other Important Information - The company has reached an agreement to purchase the annual global net sales payment obligation related to the acquisition of Scenta Pharmaceuticals for approximately $225 million [7] - The company is preparing for the potential launch of vutiquinone in the U.S. and anticipates regulatory approvals in Japan and Brazil [18][71] Q&A Session Summary Question: What is on your wish list related to the trial design for Huntington's? - Management plans to discuss the design of the efficacy trial and potential pathways to accelerated approval with the FDA, focusing on key elements and data needed [24] Question: Do you have to renew Article 117 every year for Translarna in Europe? - Article 117 allows individual countries to decide on the availability of Translarna, and the company expects to maintain about 25% revenue through the rest of 2025 [25][28] Question: Can you clarify the metrics expected during the early launch of SUFIANCE? - The company will provide metrics on prescriptions, patient start forms, and healthcare provider dynamics as the launch progresses [31] Question: What feedback have you received from payers regarding SUFIANCE? - Payers have shown a high willingness to cover SUFIANCE with minimal restrictions, and the clinical profile is being well received [42] Question: What is the expected uptake of SUFIANCE in other EU countries? - The company anticipates significant opportunities in Europe, targeting 5 to 10 markets with early access programs [71] Question: What is the status of the Translarna review in the U.S.? - The NDA for Translarna remains under active review, with clinical site inspections completed and additional information being addressed [96]

Table of Contents UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 10-Q (Mark One) ☑ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended June 30, 2025 or ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission file number: 001-35969 PTC Therapeutics, Inc. (Exact name of registrant as specified in its charter) Securities registered purs ...

Exhibit 99.1 PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2025 Financial Results – European and FDA approval of Sephience™ (sepiapterin) with broad labeling for PKU – – Global launch underway in Europe and U.S. – – Total Q2 Revenue of $179M – WARREN, N.J., August 7, 2025 – PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2025. "We had another strong quarter highlighted by the first approvals of Seph ...

Core Viewpoint - The U.S. FDA has approved PTC Therapeutics' PTCT SEPHIENCE (sepiapterin) for treating phenylketonuria (PKU), a rare genetic disorder, which is expected to significantly impact the company's future revenue and position in the market [1][4][7]. Group 1: Product Approval and Market Potential - The approval includes broad labeling for hyperphenylalaninemia (HPA) in patients aged one month and older with sepiapterin-responsive PKU [2]. - The FDA's decision is based on significant efficacy and safety data from the Phase 3 APHENITY trial and its long-term extension study [4]. - PTC Therapeutics anticipates a potential revenue opportunity exceeding $1 billion for Sephience [6]. Group 2: Commercial Strategy - The initial commercial launch strategy will target 1,200 prescribers across 104 centers, covering over 80% of PKU claims data in the U.S. [4]. - The wholesale acquisition cost (WAC) for Sephience is approximately $40,000 per month or $480,000 per year [5]. - The company will prioritize patients who have not responded well to existing oral therapies, estimated to include 40% of the PKU population [5][6]. Group 3: Analyst Insights and Stock Performance - Analysts view the approval as a significant achievement for PTC, with expectations that Sephience could become the new standard of care for PKU patients [7]. - William Blair projects peak global sales for Sephience to reach $2.2 billion by 2031 and total revenue of $1.71 billion in 2025 [7]. - PTC stock has seen a price increase of 12.44%, reaching $49.86 at the time of publication [9].

Summary of PTC Therapeutics (PTCT) Update / Briefing July 28, 2025 Company Overview - **Company**: PTC Therapeutics - **Product**: SUFIANCE (cepiaterin), a once-daily oral therapy for phenylketonuria (PKU) patients Key Points FDA Approval and Product Details - SUFIANCE received FDA approval for all disease subtypes and ages from one month and older, marking a pivotal moment for PTC and the PKU community [3][4] - The therapy demonstrated robust phenylalanine level reductions, allowing PKU patients to liberalize their restrictive diets [4][5] - There are approximately 17,000 PKU patients in the U.S., with over 300 new diagnoses annually through newborn screening [5][6] Market Opportunity - Despite two existing therapies, a significant unmet need remains, as most patients are not on any therapy [6] - The revenue opportunity for SUFIANCE in the U.S. is projected to exceed $1 billion due to its strong clinical data and ability to address all key patient segments [6][19] - PTC has established customer-facing teams experienced in commercializing rare disease therapies [7][9] Launch Strategy - The commercial launch is set for August, with a focus on engaging over 1,200 potential prescribers at 104 PKU Centers of Excellence [10][11] - Initial targeting will focus on patients who have failed existing therapies, accounting for approximately 40% of the PKU population [12][56] - PTC plans to utilize digital tools and social media to promote SUFIANCE and engage with the PKU community [12][13] Pricing and Access - The wholesale acquisition cost of SUFIANCE is $41,000 per month, based on an average patient weight of 45 kg [18] - PTC aims to ensure low barriers for access, with expectations of minimal prior authorization requirements from payers [16][19] - All commercially insured PKU patients can expect a $0 copay per month upon enrolling in the PTC care program [15][81] Patient Engagement and Support - The PTC Cares team will provide comprehensive support, including insurance verification and copay assistance [14][81] - PTC is committed to ensuring that all eligible patients receive access to SUFIANCE [14][19] Clinical Data and Efficacy - Clinical trials showed that 97% of patients could liberalize their diet, with significant reductions in phenylalanine levels [61][87] - The therapy is expected to be the new standard of care for PKU patients, with a focus on both pediatric and adult populations [20][56] Future Outlook - PTC anticipates contributions from international markets, including Europe and Japan, as part of its global launch strategy [36][94] - The company is prepared to track and share launch metrics, including patient uptake and payer dynamics, starting from the third quarter earnings call [19][70] Additional Insights - There is a strong emphasis on the importance of diet management alongside therapy, with healthcare providers expected to guide patients in diet liberalization [61][89] - The company has engaged with payers to ensure a favorable pricing strategy that reflects the therapy's value and efficacy [67][69] - PTC is focused on addressing the needs of both treatment-naive patients and those who have previously failed therapies, ensuring a comprehensive approach to patient care [38][56]

- Broad labeling inclusive of all disease subtypes for individuals 1 month of age and older -- PTC will host a conference call on Monday, July 28th at 5:00 pm ET -WARREN, N.J., July 28, 2025 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the U.S. Food and Drug Administration (FDA) has approved SEPHIENCE™ (sepiapterin) for the treatment of children and adults living with phenylketonuria (PKU). The approval includes broad labeling for the treatment of hyperphenylalaninemia (HPA) in ...

Company Announcement - PTC Therapeutics, Inc. will host a webcast conference call to report its second quarter 2025 financial results and provide a business update on August 7, 2025, at 4:30 p.m. ET [1] - Participants are encouraged to register for the call in advance and dial in 15 minutes prior to the start to avoid delays [2] Company Overview - PTC Therapeutics is a global biopharmaceutical company focused on discovering, developing, and commercializing clinically differentiated medicines for rare disorders [3] - The company aims to provide access to best-in-class treatments for patients with unmet medical needs through a robust and diversified pipeline of transformative medicines [3] - PTC leverages its scientific expertise and global commercial infrastructure to optimize value for patients and stakeholders [3]

Core Viewpoint - The Schall Law Firm is investigating PTC Therapeutics, Inc. for potential violations of securities laws related to misleading statements and failure to disclose important information to investors [1][2]. Group 1: Investigation Details - The investigation centers on whether PTC issued false or misleading statements regarding the results of the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Huntington's disease patients [2]. - PTC announced that the study met its primary endpoint of reducing blood Huntingtin (HTT) protein levels with a significant p-value of less than 0.0001 at Week 12, along with favorable safety and tolerability [2]. - Analysts suggest that a Phase 3 study is necessary to confirm if the treatment effectively slows the progression of Huntington's disease, leading to a significant drop in PTC's share price by over 18.6% on the announcement day [2].