RegulationFast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Priority Review (China), Orphan Drug (South Korea), Orphan Drug (Australia)

Structure/Sequence

Molecular FormulaC15H9FN2O3

InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N

CAS Registry775304-57-9

Clinical Trials associated with Ataluren

JPRN-jRCT2071220020

/ CompletedPhase 1

A Single-Dose Study to Assess the Food Effect on the Pharmacokinetics and Tolerability of Ataluren (PTC124) in Healthy Japanese Subjects

Start Date06 Jun 2022

Sponsor / Collaborator

PTC Therapeutics, Inc.

NCT04336826

/ CompletedPhase 2

An Open-Label Study Evaluating the Safety and Pharmacokinetics of Ataluren in Children From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy

This study is designed to evaluate safety, tolerability, and pharmacokinetics (PK) in male children with nmDMD aged ≥6 months to <2 years treated daily for 24 weeks with orally administered ataluren 10, 10, and 20 milligrams/kilogram (mg/kg) (morning, mid-day, and evening dose, respectively).

Start Date29 Dec 2021

Sponsor / Collaborator

PTC Therapeutics, Inc.

CTR20211795

/ CompletedPhase 3

一项 ATALUREN (PTC124) 在无义突变型杜兴氏肌营养不良患者中的长期安全性、疗效和耐受性的开放性研究

[Translation] An open-label study of the long-term safety, efficacy, and tolerability of ATALUREN (PTC124) in patients with nonsense mutations in Duchenne muscular dystrophy

本研究的目标在于评估长期 Ataluren 10 mg/kg 、10 mg/kg 和 20 mg/kg 给药在完成 III 期研究 (PTC124-GD-041-DMD )后希望继续接受治疗的 nmDMD 受试者中的安全性、疗效和耐受性。将根据所有不良事件 (AE)、实验室检查异常、生命体征、心电图 (ECG)、超声心动图和体格检查结果的类型、频率、严重程度、时间以及与 Ataluren 的关系来描述 Ataluren 的安全性特征。Ataluren 的疗效将根据上肢功能 (PUL)、DMD 上肢患者报告结局指标 (PROM)、肺量测定、行走能力丧失 (LoA)和 6 分钟步行试验 (6MWT)较基线的变化来评估。

[Translation]

The objective of this study was to evaluate the safety, efficacy, and tolerability of long-term dosing of Ataluren 10 mg/kg, 10 mg/kg, and 20 mg/kg in nmDMD subjects who wished to continue treatment after completing a Phase III study (PTC124-GD-041-DMD). The safety profile of ataluren will be described based on the type, frequency, severity, timing, and relationship to ataluren of all adverse events (AEs), laboratory abnormalities, vital signs, electrocardiogram (ECG), echocardiogram, and physical examination findings. The efficacy of ataluren will be evaluated based on change from baseline in upper limb function (PUL), DMD upper limb patient-reported outcome measures (PROMs), spirometry, loss of ambulation ability (LoA), and 6-minute walk test (6MWT).

Start Date14 Sep 2021

Sponsor / Collaborator

ROVI Pharma Industrial Services SA

[+2]

100 Clinical Results associated with Ataluren

100 Translational Medicine associated with Ataluren

100 Patents (Medical) associated with Ataluren

319

Literatures (Medical) associated with Ataluren

01 Jun 2026·JOURNAL OF DERMATOLOGICAL SCIENCE

Identification of novel small molecule compounds with readthrough activity in Nagashima-type palmoplantar keratosis

Article

Author: Miyauchi, Toshinari ; Ohguchi, Yuka ; Suzuki, Shotaro ; Sugiyama, Takato ; Takeda, Masae ; Nomura, Toshifumi ; Ujiie, Hideyuki ; Peh, Jin Teng ; Nobuta, Risa

BACKGROUND:

Nagashima-type palmoplantar keratosis (NPPK), the most common form of hereditary palmoplantar keratodermas in East Asian populations, is caused by biallelic loss-of-function mutations in SERPINB7 such as p.Arg266Ter (c.796 C>T). This mutation introduces a premature termination codon (PTC). Given the minimal efficacy of current drugs in improving NPPK skin phenotypes, we applied readthrough therapy to enable the production of functional proteins by allowing ribosomes to read through premature stop codons.

OBJECTIVE:

To discover small molecule compounds with readthrough potency.

METHODS:

Using high-throughput screening, we initially screened nearly 20,000 small molecule compounds. Using Western blot, we assessed the readthrough effectiveness of shortlisted compounds on the mutant SERPINB7 vector. We identified a promising compound and acquired structurally related compounds, comparing then their potency with that of gentamicin and ataluren. In addition, we evaluated the readthrough capability of the identified compounds on other 11 premature termination codons with different + 4 nucleotides.

RESULTS:

The identified hit compounds demonstrated superior readthrough efficacy than gentamicin and ataluren. Notably, our compounds were effective in enabling readthrough, particularly on UGAA and UAAG premature stop codons.

CONCLUSION:

Our research identified new compounds that effectively promote the readthrough of premature stop codons. In vitro, these compounds showed higher readthrough potency than drugs such as gentamicin or ataluren. These results suggest that these compounds could be promising therapeutic agents for genetic diseases caused by nonsense mutations.

01 Jun 2026·EPILEPSIA

Mutation type‐specific transcriptomic signatures and readthrough therapy rescue in SMC1A ‐related developmental and epileptic encephalopathy

Article

Author: Antonio Musio ; Iñigo Marcos-Alcalde ; Francesca Sardina ; Ian D. Krantz ; Maddalena Di Nardo ; Cinzia Rinaldo ; Paulino Gómez-Puertas ; Maria M. Pallotta

Abstract:

Objective:

This study was undertaken to investigate the molecular consequences of pathogenic variants in the SMC1A gene—particularly those associated with developmental and epileptic encephalopathy (DEE85)—and to evaluate the therapeutic potential of ataluren in restoring SMC1A function and mitigating disease‐related transcriptomic and genomic alterations.

Methods:

The study analyzed transcriptomic profiles from cell lines derived from individuals with DEE85 and Cornelia de Lange syndrome (CdLS), comparing the effects of different SMC1A variants. Particular focus was placed on nonsense variants and their impact on gene expression. Functional assays were conducted to assess the ability of ataluren to restore SMC1A protein expression, correct transcriptional defects, and reduce genomic instability.

Results:

Transcriptomic alterations were strongly dependent on variant type, with nonsense variants causing the most profound gene expression changes. DEE85 and CdLS cell lines exhibited distinct transcriptional signatures. Treatment with ataluren led to successful restoration of SMC1A protein levels, partial correction of gene expression abnormalities, and a reduction in genomic instability in cells harboring nonsense variants.

Significance:

These findings demonstrate that SMC1A ‐related epileptic encephalopathies are driven by variant‐specific molecular mechanisms and highlight the therapeutic promise of ataluren for DEE85. The study supports further development of precision medicine strategies targeting nonsense variants in SMC1A , with potential implications for improving diagnosis, treatment, and quality of life in affected individuals.

01 Apr 2026·Journal of Comparative Effectiveness Research

Ataluren for the treatment of people living with nonsense mutation Duchenne muscular dystrophy: a plain language summary of Study 041

Article

Author: de Queiroz Campos Araujo, Alexandra Prufer ; Wu, Shiwen ; Vlodavets, Dmitry ; Castle, Jill

What is this summary about?:

This article describes results from Study 041. Study 041 was a clinical study of ataluren, a treatment for people living with nonsense mutation Duchenne muscular dystrophy (nmDMD for short). Over time, people living with nmDMD experience muscle loss and reduced muscle strength and function (decline in muscle function). The researchers wanted to know whether 72 weeks of treatment with ataluren slowed the decline in muscle function in a large group of people living with nmDMD. To determine this, they compared the effects of ataluren with the effects of a placebo, which is a treatment that looks the same as the study treatment but has no active ingredients. The study also looked at the safety of ataluren over the 72 weeks of treatment. Ataluren has previously been compared with placebo in 48-week long clinical trials.

What were the results?:

In this study, 359 people took at least one dose of ataluren or placebo, even if they later switched or didn't stick to the treatment plan. Over 72 weeks, ability to walk and physical function declined less in people receiving ataluren than in people receiving placebo. This means that their physical abilities were maintained for longer if they took ataluren. The number of side effects were generally similar between people who took ataluren and those who took placebo.

What do the results of Study 041 mean?:

These results help to confirm that 72 weeks of treatment with ataluren (compared with 72 weeks of placebo treatment) slows down the decline in muscle function experienced by people living with nmDMD. Results also confirmed that side effects reported were similar with ataluren and placebo. These results likely show that people who receive ataluren can maintain their independence for a longer time. This study is the largest phase 3 clinical study of people living with nmDMD that has been done so far.ClinicalTrials.gov, NCT number:NCT03179631

News (Medical) associated with Ataluren

03 Aug 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Second Quarter 2026 Financial Results

– Robust Q2 performance with total revenue of $361 million, including $239 million of product revenue – – Full-year 2026 expected total revenue increased to $1.18 to $1.28 billion, with full-year product revenue guidance raised to $850 to $950 million – – Sephience™ (sepiapterin) Q2 2026 revenue of $151 million, with continued broad uptake and geographical expansion – – Strong cash position of $2.2 billion as of June 30, 2026 – WARREN, N.J., July 30, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the second quarter ended June 30, 2026. "Our teams delivered another strong quarter enabling us to again raise 2026 product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "We also made a number of advances in our R&D pipeline, including identification of our next small molecule splicing development candidate, PTC303, targeting somatic expansion disorders including Huntington's disease and myotonic dystrophy. In addition, we initiated the Phase 1 study of PTC612, our differentiated NLRP3 small molecule." Key Corporate Updates Q2 2026 total revenue of $361 million, including $239 million of product revenue Sephience global launch continues with strong momentum Q2 2026 revenue of $151 million, with the majority from the US and increasing contributions internationally 1,647 patients on commercial therapy worldwide as of June 30, 2026 Positive results from votoplam PIVOT-HD long-term extension study reported and enrollment in global Phase 3 INVEST-HD study ongoing PIVOT-HD 24-month results demonstrated dose-dependent disease slowing on cUHDRS scale in Stage 2 participants, reaching 52% at 10 mg dose level Plan to engage with FDA in 2H 2026 to discuss PIVOT-HD 24-month results being finalized INVEST-HD study being conducted and funded by partner Novartis with target enrollment of approximately 770 individuals with early symptomatic disease INVEST-HD initiation triggered a $50 million milestone payment from Novartis to PTC in Q2 2026 Vatiquinone PROVE-FA study to support NDA resubmission for treatment of Friedreich's ataxia expected to initiate in Q3 2026 Global study will enroll approximately 120 individuals with Friedreich's ataxia age 7 to 21 Study includes open-label treatment arm with matched natural history control group PTC pipeline continues to advance PTC303 named as MSH3 splicing program development candidate targeting Huntington's disease and myotonic dystrophy with plan to enter clinic in 2027 Phase 1 study of PTC612, an oral NLRP3 inhibitor, initiated in Q2 2026 Phase 2a study of PTC844, a next-generation DHODH inhibitor, expected to initiate in Q3 2026 Hege Sollie-Zetlmayer has been appointed to PTC's Board of Directors. Ms. Sollie-Zetlmayer has over 30 years of experience in the biopharmaceutical and medical device industry as a global business operations and human resources leader. She most recently served as PTC's Chief Human Resources Officer prior to her retirement in June 2026. Second Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $309.9 million for the second quarter of 2026, compared to $175.9 million for the second quarter of 2025. Total net product revenue across the commercial portfolio was $238.8 million for the second quarter of 2026, compared to $118.3 million for the second quarter of 2025, representing over 100% increase. Sephience net product revenues were $151.3 million for the second quarter of 2026, representing 21% growth compared to the first quarter of 2026. Translarna™ (ataluren) net product revenues were $42.2 million for the second quarter of 2026, compared to $59.5 million for the second quarter of 2025. Emflaza® (deflazacort) net product revenues were $24.6 million for the second quarter of 2026, compared to $36.4 million for the second quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) year-to-date sales of approximately 968 CHF million, resulting in royalty revenue of $71.1 million to PTC for the second quarter of 2026, compared to $57.6 million to PTC for the second quarter of 2025. In the second quarter of 2026, PTC recorded a development milestone of $50.0 million from Novartis for the first patient dosed in the ongoing INVEST-HD Phase 3 study. This sales milestone was recorded as collaboration revenue. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $99.2 million for the second quarter of 2026, compared to $113.0 million for the second quarter of 2025. Non-GAAP R&D expenses were $88.6 million for the second quarter of 2026, excluding $10.5 million in non-cash, stock-based compensation expense, compared to $104.0 million for the second quarter of 2025, excluding $9.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $80.6 million for the second quarter of 2026, compared to $85.3 million for the second quarter of 2025. Non-GAAP SG&A expenses were $67.9 million for the second quarter of 2026, excluding $12.7 million in non-cash, stock-based compensation expense, compared to $75.7 million for the second quarter of 2025, excluding $9.5 million in non-cash, stock-based compensation expense. Net income was $83.5 million for the second quarter of 2026, compared to net loss of $64.8 million for the second quarter of 2025. In June 2026, PTC issued $550.0 million of senior convertible notes due in 2031 at 0% coupon and a conversion price representing a 40% premium over the stock's closing price at the time of the deal. Concurrent with the transaction, PTC repurchased the majority of its 1.5% senior convertible notes due in September 2026. Cash, cash equivalents, and marketable securities were $2,229.1 million on June 30, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of June 30, 2026, were 83,327,286. PTC Updates Full-Year 2026 Financial Guidance Expected total revenue increased to $1.18 to $1.28 billion, with total product revenue guidance raised to $850 to $950 million from $750 to $850 million GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease NDA: New Drug Application nmDMD: Nonsense mutation Duchenne muscular dystrophy R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including commercialization and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; expectations regarding PTC's product candidates, including the timing of clinical trials and studies; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Phase 1Financial StatementExecutive ChangePhase 2Phase 3

07 May 2026

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports First Quarter 2026 Financial Results

– First quarter 2026 total revenue of $273 million, including $226 million of product revenue, supporting full-year 2026 guidance raise – – Global Sephience™ (sepiapterin) launch momentum continues with first quarter 2026 revenue of $125 million, representing 36% quarter-over-quarter growth – – Positive topline results from 24-month interim analysis of PIVOT-HD extension study of votoplam, supporting ongoing global Phase 3 INVEST-HD study and potential regulatory interactions – – Open-label vatiquinone registration study to be initiated in Q3 2026 based on FDA feedback – WARREN, N.J., May 7, 2026 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ended March 31, 2026. "We are off to a strong start to 2026 with outstanding revenue performance this quarter that supports raising our full-year product revenue guidance," said Matthew B. Klein, M.D., Chief Executive Officer. "The Sephience launch continues to be strong, with sustained momentum in the US and growing momentum internationally as more countries contribute to the global launch." Key Corporate Updates Q1 2026 total revenue of $273 million, with $226 million of product revenue Sephience global launch continues strong momentum Q1 2026 revenue of $125 million, including $112 million revenue in the US and $13 million revenue ex-US, up 36% from Q4 2025 1,244 total patients on commercial therapy worldwide as of March 31, 2026 Continued strong cadence of patient start forms in the US, with ~140 per month over past several months, surpassing 1,500 mark in the quarter First commercial sales in Japan in Q1 2026, with revenue expected from up to 30 countries by year-end 2026 Reported positive topline results in April 2026 from 24-month interim analysis of PIVOT-HD long-term extension study of votoplam, an oral small molecule splicing agent Dose-dependent benefit on cUHDRS in Stage 2 participants relative to natural history cohort, with 52% slowing of disease progression at 10 mg dose Continued favorable safety and tolerability with no treatment-related NfL spikes PTC and Novartis will complete data review and align on potential regulatory interactions Findings support ongoing global Phase 3 INVEST‑HD study of votoplam led by Novartis First patient dosed in Phase 3 INVEST-HD study, triggering $50 million milestone payment from Novartis to PTC in Q2 2026 Type C meeting with FDA held in April 2026 to discuss vatiquinone study to support NDA resubmission for Friedreich's ataxia program Based on meeting discussion and written feedback, PTC expects to initiate an open-label study using matched natural history control in Q3 2026 Primary endpoint will be change in mFARS from baseline to 24 months First Quarter 2026 Financial Highlights Total net product revenue and royalty revenue was $272.4 million for the first quarter of 2026, compared to $189.9 million for the first quarter of 2025. Total net product revenue across the commercial portfolio was $225.6 million for the first quarter of 2026, compared to $153.4 million for the first quarter of 2025, representing a 47% increase. Sephience net product revenues were $124.6 million for the first quarter of 2026, representing 36% growth compared to fourth quarter of 2025. Translarna™ (ataluren) net product revenues were $59.0 million for the first quarter of 2026, compared to $86.2 million for the first quarter of 2025. Translarna first quarter of 2026 revenue includes a large government purchase order from Brazil. Emflaza® (deflazacort) net product revenues were $21.5 million for the first quarter of 2026, compared to $47.8 million for the first quarter of 2025, due to continued generic erosion. Roche reported Evrysdi® (risdiplam) sales of approximately 464 CHF million, resulting in royalty revenue of $46.8 million to PTC for the first quarter of 2026, compared to $36.4 million to PTC for the first quarter of 2025. Based on US GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $100.9 million for the first quarter of 2026, compared to $109.0 million for the first quarter of 2025. Non-GAAP R&D expenses were $89.7 million for the first quarter of 2026, excluding $11.1 million in non-cash, stock-based compensation expense, compared to $100.3 million for the first quarter of 2025, excluding $8.7 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $86.2 million for the first quarter of 2026, compared to $81.0 million for the first quarter of 2025. Non-GAAP SG&A expenses were $73.9 million for the first quarter of 2026, excluding $12.3 million in non-cash, stock-based compensation expense, compared to $71.6 million for the first quarter of 2025, excluding $9.4 million in non-cash, stock-based compensation expense. Net loss was $2.8 million for the first quarter of 2026, compared to net income of $866.6 million for the first quarter of 2025. Cash, cash equivalents, and marketable securities were $1,892.5 million on March 31, 2026, compared to $1,945.4 million on December 31, 2025. Shares issued and outstanding as of March 31, 2026, were 82,882,024. PTC Updates Full-Year 2026 Financial Guidance Total product revenue guidance raised to $750 to $850 million, with expected total revenue of $1.08 to $1.18 billion GAAP R&D and SG&A expense guidance remains $775 to $815 million Non-GAAP R&D and SG&A expense guidance remains $680 to $720 million, excluding estimated non-cash, stock-based compensation expense of $95 million Non-GAAP Financial Measures In this press release, the financial results of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP R&D and SG&A expense financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in accordance with GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management's opinion, these non-GAAP financial measures are useful to investors and other users of PTC's financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company's future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below. Acronyms CHF: Confoederatio Helvetica Francs (Swiss francs) cUHDRS: Composite Unified Huntington's Disease Rating Scale DMD: Duchenne muscular dystrophy FA: Friedreich's ataxia FDA: US Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's disease mFARS: Modified Friedreich's Ataxia Rating Scale NDA: New Drug Application NfL: Neurofilament light chain nmDMD: Nonsense mutation Duchenne muscular dystrophy PKU: Phenylketonuria R&D: Research and Development SG&A: Selling, General, and Administrative Today's Conference Call and Webcast Reminder To access the live webcast, please visit the "Events & Presentations" page within the Investors section of the PTC website. A replay of the webcast will be available on the PTC website for 30 days following the event. To participate via phone, please register in advance here to receive dial-in details. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company dedicated to the discovery, development and commercialization of clinically differentiated medicines for children and adults living with rare disorders. PTC is advancing a robust and diversified pipeline of transformative medicines as part of its mission to provide access to best-in-class treatments for patients with unmet medical needs. The company's strategy is to leverage its scientific expertise and global commercial infrastructure to optimize value for patients and other stakeholders. To learn more about PTC, please visit and follow on LinkedIn, X, Facebook and Instagram. For more information please contact: Investors: Ellen Cavaleri +1 (615) 618-8228 [email protected] Media: Jeanine Clemente +1 (908) 912-9406 [email protected] Forward-Looking Statements: This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including the information provided under the heading "PTC Updates Full-Year 2026 Financial Guidance", including with respect to (i) 2026 total product revenue guidance and total revenue guidance and (ii) 2026 GAAP and non-GAAP R&D and SG&A expense guidance, and statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses, meetings with regulatory agencies, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words, "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," "aim," and similar expressions. PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; expectations with respect to Sephience, including any regulatory submissions and potential approvals, commercialization, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in geographies in which it has been approved and the effect of the European Commission's adoption of the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) on Translarna and the withdrawal of the Translarna NDA in the US on other regulatory bodies; expectations with respect to PTC's license and collaboration agreement with Novartis Pharmaceuticals Corporation for votoplam for the treatment of Huntington's disease including its right to receive development, regulatory and sales milestones, profit sharing and royalty payments from Novartis, the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses, including potential accelerated approval; expectations with respect to Upstaza/Kebilidi, including commercialization, manufacturing capabilities, and the potential achievement of sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to vatiquinone, including with respect to the design and expected timing of clinical trials and studies, the availability of data, and regulatory submissions and responses and potential approvals and other matters; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; the eligible patient base and commercial potential of PTC's products and product candidates; PTC's scientific approach and general development progress; PTC's ability to satisfy its obligations under the terms of its lease agreements; the sufficiency of PTC's cash resources and its ability to obtain adequate financing in the future for its foreseeable and unforeseeable operating expenses and capital expenditures; and the factors discussed in the "Risk Factors" section of PTC's Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors. As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Sephience, Translarna, Emflaza, Upstaza, Kebilidi, Evrysdi, Tegsedi or Waylivra. The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law. SOURCE PTC Therapeutics, Inc. 21% more press release views with Request a Demo

Financial StatementPhase 3License out/inNDA

27 Apr 2026

·allsci.com

Alltrna puts engineered tRNA therapeutic into first-in-human study

Alltrna, a Cambridge, Massachusetts-based Flagship Pioneering company, has received approval from Australia’s Therapeutic Goods Administration to initiate a first-in-human trial of AP003, a chemically modified transfer RNA therapeutic designed to restore full-length protein production in patients carrying an arginine-to-TGA premature termination codon. The AP003 clinical trial, approved under the TGA Clinical Trial Notification scheme following Human Research Ethics Committee review, will enroll healthy volunteers in a single ascending dose design, with safety and pharmacokinetics as primary endpoints. No enrolment number or trial end date has been disclosed publicly. AP003 is a tRNA oligonucleotide encapsulated in a liver-directed lipid nanoparticle. Its mechanism operates at the level of ribosomal translation: the engineered molecule decodes the Arg-TGA stop codon and reinserts arginine into the growing polypeptide chain, preventing premature termination and restoring functional protein. Alltrna describes Arg-TGA as the most frequent nonsense mutation in human genetic disease, accounting for approximately 21–22% of cases. The company frames AP003 not as a therapy for a single condition but as a mutation-centric platform applicable across any liver-expressed gene disrupted by this specific codon change — a strategy it terms treatment of “Stop Codon Disease.” Research context: the science behind engineered suppressor tRNA The conceptual basis for tRNA-mediated readthrough has existed for decades, but clinical translation has lagged. A 2022 Nature study demonstrated that AAV-delivered suppressor tRNA could overcome a nonsense mutation in a mouse model, providing direct in vivo proof of concept that engineered tRNAs can rescue PTC-driven phenotypes. A parallel analysis of anticodon-engineered suppressor tRNA therapy outlined why this approach may offer codon-level precision that small-molecule readthrough agents cannot match. That distinction matters because the small-molecule precedent is mixed. Ataluren, the most clinically advanced PTC readthrough compound, has faced inconsistent efficacy data across trials, with context-dependence around stop codon identity and flanking sequence complicating generalisation. A 2023 review of readthrough pharmacology reinforced that broad-spectrum small molecules have not reliably translated preclinical activity into clinical benefit. Alltrna’s approach bypasses this by directly supplying the missing tRNA species rather than modulating release factor competition — a mechanistically cleaner intervention, though one that introduces its own delivery and fidelity questions. The choice of liver as the initial tissue target reflects the maturity of LNP delivery platforms. Systemic LNP delivery of nucleic acid cargo to hepatocytes is well-characterised, with biodistribution data and formulation precedents supporting predictable hepatic uptake. Alltrna states that AP003’s preclinical safety profile is consistent with established oligonucleotide and LNP experience, though no primary AP003 publication has been made available to independently assess insertion fidelity, off-target readthrough at endogenous stop codons, or immunogenicity. Stop Codon Disease, as Alltrna defines it, encompasses roughly 10% of all genetic disease patients — approximately 30 million people globally. The diseases most directly in scope for a liver-directed Arg-TGA therapeutic include conditions such as certain urea cycle disorders, organic acidemias including methylmalonic acidemia, and phenylketonuria subtypes driven by this specific mutation class. Across these indications, existing approved therapies — nitrogen scavengers, dietary restriction, cofactor supplementation — address metabolic consequences rather than the causative genetic lesion. No approved pharmacological therapy corrects the underlying protein truncation in nonsense mutation patients across these disease categories. Liver transplantation remains the only functional cure for several of them, constrained by donor availability and procedural risk. The provided data does not include a comprehensive review of recent regulatory approvals in these specific indications, so a detailed current standard-of-care comparison cannot be fully constructed from available sources. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

OligonucleotideClinical Study

100 Deals associated with Ataluren

External Link

KEGG	Wiki	ATC	Drug Bank
D09323	Ataluren	M09AX03	DB05016

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Muscular Dystrophy, Duchenne	European Union	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	Iceland	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	Liechtenstein	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	Norway	PTC Therapeutics International Ltd.	31 Jul 2014

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Cystic Fibrosis	NDA/BLA	European Union	PTC Therapeutics, Inc.	-
Becker Muscular Dystrophy	Phase 3	Australia	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Belgium	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Canada	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	France	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Germany	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Israel	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Italy	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Spain	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	Sweden	PTC Therapeutics, Inc.	20 May 2012

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03179631 (Study 041, CTgov)	Phase 3	Muscular Dystrophy, Duchenne	360	Ataluren (Ataluren)	oyiojwnqzq(cugcbobumu) = kpoiolacca wizunqrcmx (nrrdxnzitl, 6.461) View more	-	10 Mar 2026
NCT03179631 (Study 041, CTgov)	Phase 3	Muscular Dystrophy, Duchenne	360	PLACEBO (Placebo)	oyiojwnqzq(cugcbobumu) = ksfnuylarh wizunqrcmx (nrrdxnzitl, 6.377) View more	-	10 Mar 2026
NCT03179631 \| NCT01826487 \| NCT00592553 (Study 041 \| Study 007 \| ACT DMD, AAN2024)	Phase 2/3	Muscular Dystrophy, Duchenne	-	Ataluren	ilkffuevot(houlndfvpt) = axxqjkgyij ymlodmdkes (ejwfqeqrpv ) View more	Positive	09 Apr 2024
NCT04336826 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	avighcrnmr = erccjcwdfe lvqcfdwtcy (ficzgifsjh, cucqcukjet - jojybyywjg) View more	-	01 Apr 2024
NCT03179631 (Study 041, AAN2023)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation (nm) DMD	-	Ataluren	uqrnssxynk(tukpslugiu) = jgwfthuzhu hsxffyiczv (nlatcnbnrh ) View more	Positive	25 Apr 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	-	Ataluren	wzavyywioa(ebbsvzklcv) = qquslsrefj idiydjzkzz (icftssapzc ) View more	Positive	19 Mar 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	701	Ataluren	tcfggbjzff(uxxfmerptl) = zuuybxtxef yojbgvcnqk (rwyumnjxzf ) View more	Positive	19 Mar 2023
NCT02139306 \| NCT00803205 \| NCT02107859 (Pubmed \| Cochrane Database Syst Rev)	Not Applicable	Cystic Fibrosis	517	Ataluren and similar compounds	lxiagqczrp(ljptlddmnf) = Ataluren was associated with a higher rate of episodes of renal impairment (risk ratio 12.81, 95% confidence interval 2.46 to 66.65; P = 0.002; I2 = 0%; 2 trials, 517 participants) aguyiekwvb (gqinpoqhrc ) View more	-	03 Mar 2023
	Not Applicable	Cystic Fibrosis	517	Placebo		-	03 Mar 2023
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Ataluren (Ataluren)	zzcrukmmxl(vekhkuhrcr) = dlepkrzred dqtmsyrvoc (euosmaibbu, 7.425) View more	-	27 May 2022
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Placebo+Ataluren (Placebo)	zzcrukmmxl(vekhkuhrcr) = cpbcvkrviq dqtmsyrvoc (euosmaibbu, 11.809) View more	-	27 May 2022
NCT03648827 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	20	Ataluren	qvmupojwjs(dbnwcqevjw) = rpdzuueurp nlzvlgsfmq (yynlnisrom, xznvmnypoz - pctwepsqle) View more	-	05 Apr 2022
NCT03796637 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	fivqsiemak(llifewnhhv) = mblmjczehc mmulhbecwu (wnmjjeinpg, 0.05874) View more	-	05 Apr 2022

Translational Medicine

Boost your research with our translational medicine data.

view full example data

Deal

Boost your decision using our deal data.

view full example data

Core Patent

Boost your research with our Core Patent data.

view full example data

Clinical Trial

Identify the latest clinical trials across global registries.

view full example data

Approval

Accelerate your research with the latest regulatory approval information.

view full example data

Regulation

Understand key drug designations in just a few clicks with Synapse.

view full example data

Hiro LS Researcher

The AI Search Engine Built to Accelerate Biopharma Decisions

Search across billion-scale life sciences data to uncover signals, validate evidence, and act with confidence.

Ask any biopharma research question→

Get started for free today!

Accelerate Strategic R&D decision making with Synapse, Patsnap’s AI-powered Connected Innovation Intelligence Platform Built for Life Sciences Professionals.

Discover Synapse Data Servers

Synapse data is now integrated into the PatSnap LS Model Context Protocol (MCP) service. Customize your LLM agent now using our MCP server!

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis