RegulationFast Track (US), Orphan Drug (EU), Priority Review (CN), Orphan Drug (KR), Orphan Drug (AU), Conditional marketing approval (EU), Orphan Drug (US)

Structure

Molecular FormulaC15H9FN2O3

InChIKeyOOUGLTULBSNHNF-UHFFFAOYSA-N

CAS Registry775304-57-9

Clinical Trials associated with Ataluren

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.

CTIS2024-518919-19-00 / RecruitingIIT

A single patient trial with Ataluren in one case of severe common variable immunodeficiency with autoimmunity due to homozygous stop codon mutations of LRBA - LRBA01

Start Date28 Jul 2021

Sponsor / Collaborator

Centre Hospitalier Universitaire de Liege

JPRN-jRCT2041200087 / RecruitingPhase 3IIT

An Open-Label, Long-Term Safety, Efficacy, and Tolerability Study for Ataluren (PTC124) in Patients with Nonsense Mutation Duchenne Muscular Dystrophy - PTC124-GD-049-DMD

Start Date07 Jan 2021

Sponsor / Collaborator-

100 Clinical Results associated with Ataluren

100 Translational Medicine associated with Ataluren

100 Patents (Medical) associated with Ataluren

611

Literatures (Medical) associated with Ataluren

09 Dec 2024·ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

N‐Halogenation by Vanadium‐Dependent Haloperoxidases Enables 1,2,4‐Oxadiazole Synthesis

Article

Author: Patton, Zoe E. ; Biegasiewicz, Kyle F. ; Sharma, Manik ; Bacsa, John ; Shoemaker, Carlie R.

Abstract:

Nitrogen‐containing compounds are valuable synthetic intermediates and targets in nearly every chemical industry. While methods for nitrogen‐carbon and nitrogen‐heteroatom bond formation have primarily relied on nucleophilic nitrogen atom reactivity, molecules containing nitrogen‐halogen bonds allow for electrophilic or radical reactivity modes at the nitrogen center. Despite the growing synthetic utility of nitrogen‐halogen bond‐containing compounds, selective catalytic strategies for their synthesis are largely underexplored. We recently discovered that the vanadium‐dependent haloperoxidase (VHPO) class of enzymes are a suitable biocatalyst platform for nitrogen‐halogen bond formation. Herein, we show that VHPOs perform selective halogenation of a range of substituted benzamidine hydrochlorides to produce the corresponding N’‐halobenzimidamides. This biocatalytic platform is applied to the synthesis of 1,2,4‐oxadiazoles from the corresponding N‐acylbenzamidines in high yield and with excellent chemoselectivity. Finally, the synthetic applicability of this biotechnology is demonstrated in an extension to nitrogen‐nitrogen bond formation and the chemoenzymatic synthesis of the Duchenne muscular dystrophy drug, ataluren.

01 Dec 2024·Molecular Genetics and Metabolism Reports

Ataluren-mediated nonsense variant readthrough in D-bifunctional protein deficiency: A case report

Article

Author: Lee, Ni-Chung ; Hwu, Wuh-Liang ; Chien, Yin-Hsiu ; Lee, Wang-Tso ; Hsu, Rai-Hseng ; Chen, Hui-An

D-bifunctional protein (DBP) deficiency, a fatal peroxisomal enzyme disorder, typically manifests with life-threatening symptoms in the first two years of childhood. We present the case of an infant with elevated lysophosphatidylcholine C26:0 (C26:0-LPC) levels identified during X-linked adrenoleukodystrophy (ALD) screening, leading to a diagnosis of DBP deficiency due to a homozygous HSD17B4 c.1041T>A, p.(Tyr347Ter) variant. Starting at two months of age, the infant experienced seizures, hypotonia, and developmental delays, prompting the initiation of experimental treatment with the readthrough agent PTC124 (ataluren) at six months. The treatment led to a decrease in C26:0-LPC levels from 0.65 μM to 0.53 μM; concomitant fish oil supplementation transiently increased C26:0-LPC to 0.74 μM before returning to 0.53 μM after cessation of supplementation. The patient demonstrated improved swallowing and progressive motor and speech development during a two-year treatment period, with no further seizures. This case report highlights the potential of nonsense readthrough therapy for peroxisomal disorders, a group of metabolic diseases that currently lack targeted treatments.

01 Oct 2024·EXPERIMENTAL NEUROLOGY

Investigating therapeutic nonsense suppression in a neurofibromatosis mouse model

Article

Author: Peker, Ahmet ; Wu, Chan ; Shazeeb, Mohammed Salman ; Han, George ; Gounis, Matthew J ; Jacobson, Allan ; Mangkalaphiban, Kotchaphorn ; Rentiya, Zubir S

Neurofibromatosis type 1 (NF1) is a human genetic disorder caused by variants in the NF1 gene. Plexiform neurofibromas, one of many NF1 manifestations, are benign peripheral nerve sheath tumors occurring in up to 50% of NF1 patients. A substantial fraction of NF1 pathogenetic variants are nonsense mutations, which result in the synthesis of truncated non-functional NF1 protein (neurofibromin). To date, no therapeutics have restored neurofibromin expression or addressed the consequences of this protein's absence in NF1 nonsense mutation patients, but nonsense suppression is a potential approach to the problem. Ataluren is a small molecule drug that has been shown to stimulate functional nonsense codon readthrough in several models of nonsense mutation diseases, as well as in Duchenne muscular dystrophy patients. To test ataluren's potential applicability in nonsense mutation NF1 patients, we evaluated its therapeutic effects using three treatment regimens in a previously established NF1 patient-derived (c.2041C > T; p.Arg681X) nonsense mutation mouse model. Collectively, our experiments indicate that: i) ataluren appeared to slow the growth of neurofibromas and alleviate some paralysis phenotypes, ii) female Nf1-nonsense mutation mice manifested more severe paralysis and neurofibroma phenotypes than male mice, iii) ataluren doses with apparent effectiveness were lower in female mice than in male mice, and iv) age factors also influenced ataluren's effectiveness.

News (Medical) associated with Ataluren

14 Nov 2024

·endpts.com

FDA approves PTC’s ultra-rare disease gene therapy for broader patient population than Europe

The FDA has cleared PTC Therapeutics’ gene therapy for AADC deficiency, an ultra-rare disease that impacts the nerve cells and brain. The gene therapy was granted an expansive label encompassing children and adults across “the full spectrum of disease severity,” according to PTC’s press release , and will be marketed as Kebilidi. Known generically as eladocagene exuparvovec, the gene therapy is marketed as Upstaza in the EU, UK and Israel, where it is indicated for children 18 months and older with severe AADC deficiency. It was first authorized in the EU and UK in 2022. In the UK, the therapy has a list price of about £3 million ($3.8 million). PTC has not yet disclosed the US list price for Kebilidi. In clinical trials with 26 patients, the therapy improved patients’ motor and cognitive function, according to results published in 2022 , with those improvements sustained for over five years in patients with long-term follow-up. In the US, it’s estimate d that fewer than 1,000 people have AADC deficiency. People with the genetic disease don’t make enough dopamine, which can result in developmental disabilities, sleep disorders, and motor and behavioral symptoms. Kebilidi is meant to increase dopamine production by introducing a functional copy of the DDC gene that is mutated in these patients. The gene therapy is administered directly to the brain and is the first such gene therapy to win approval in the US. Along with Kebilidi, PTC is waiting for FDA decisions for two other therapies it’s developing: Translarna, a Duchenne muscular dystrophy drug that was rejected in the US in 2016 and 2017 and is soon expected to lose its EU market authorization; and a PKU drug called sepiapterin.

Drug ApprovalGene TherapyClinical Result

08 Nov 2024

·medcitynews.com

FDA Takes Step Toward Removal of Ineffective Decongestants From the Market - MedCity News

A pharmaceutical ingredient that’s been a key component in nasal decongestants for decades is having its efficacy called into question, and the FDA is now weighing whether to change the compound’s regulatory status — potentially leading to many widely used over-the-counter cold and allergy products being pulled from pharmacy shelves. But before the FDA takes any action, it wants to hear what the public thinks. The ingredient, oral phenylephrine, by itself and in combination with other active ingredients, is in decongestants sold under numerous brand names, such as Sudafed and Mucinex. All products containing oral phenylephrine continue to be available to consumers for now. But on Thursday, the FDA proposed an order that would remove oral phenylephrine from its non-prescription drugs guidelines, called the OTC Monograph. The regulator emphasized that this proposed order is due to lack of efficacy, not because of safety. At one time, phenylephrine was rare in decongestants because pseudoephedrine was the preferred active ingredient. But that compound can be made into the methamphetamine. Efforts to curb meth production led to the Combat Methamphetamine Act of 2005, which moved pseudoephedrine-containing drugs behind pharmacy counters. Drugmakers responded by making more products with oral phenylephrine, which is not covered by this law. presented by Sponsored Post The Promise of Value-Based Care and MedTech Innovation Monica Vajani, Executive Director for mHUB’s MedTech Accelerator, discusses how mHUB is helping innovators transition healthcare towards value-based care. By Monica Vajani - mHUB Last year, an FDA advisory committee discussed phenylephrine and unanimously voted that current scientific data do not support the ingredient’s efficacy as a nasal decongestant. The committee raised no concerns about the safety of oral phenylephrine when used at the recommended dose. The FDA now says it has conducted a comprehensive review of all available safety and efficacy data for oral phenylephrine as a nasal decongestant, including historical data used to support the compound 30 years ago. Speaking during a conference call with journalists Thursday, Theresa Michele, director of the FDA’s office of nonprescription drugs, said new data had become available since the advisory committee meeting, which the agency also reviewed. “At this point, based on the available data, and taking into account the advice of the advisory committee, we are proposing that oral phenylephrine is not effective to relieve nasal congestion at the recommended dose set forth in the monograph,” Michele said. Phenylephrine is also an ingredient in some nasal spray decongestants. The FDA’s proposal does not extend to such products. The proposed FDA order can be found here. Public comments may be submitted here (use docket number FDA-2024-N-4734 ) through May 7, 2025. After considering the comments, the agency may issue a final order removing oral phenylephrine from its guidelines, which would mean nasal decongestants could no longer contain the compound. Michele said the FDA would then provide manufacturers with time to either reformulate their medicines or remove oral phenylephrine-containing products from the market. Don’t let the autumn sniffles get you down. Here’s a look back at other recent regulatory news, including U.S. and European actions on several novel medications (alas, no new decongestants): Exclusive Heard at HLTH 2024: Insights from Innovative Healthcare Executives Executives from Imagine360, Verily, BrightInsight, Lantern, and Rhapsody shared their approaches to reducing healthcare costs and facilitating digital transformation. By MedCity News Decisions for Drugs and Devices —The FDA needs more time to review Organon drug Vtama as a treatment for atopic dermatitis. The company said the regulator has pushed out the December target date for a decision by three months to March 12, 2025. Vtama is from Roivant Sciences subsidiary Dermavant, which Organon recently acquired for $175 million up front. Under Dermavant, the topical drug won FDA approval in 2022 as a treatment for plaque psoriasis. —Novartis cancer drug Scemblix expanded its FDA-approved uses to include the treatment of newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ PML). The tablet, part of a class of drugs called STAMP inhibitors, initially landed accelerated FDA approval in 2021 as a treatment for advanced cases of Ph+ PML. —The FDA approved Iterum Therapeutics antibiotic Orlynvah. The regulatory decision covers the treatment of uncomplicated urinary tract infections in women who have limited or no oral antibacterial treatment options. The drug, whose main ingredient is sulopenem, is taken as a tablet twice daily for five days. —Astellas Pharma landed FDA approval for zolbetuximab, brand name Vyloy, as a treatment for gastric or gastroesophageal junction adenocarcinoma. It’s the first FDA approval for a drug targeting the cancer protein claudin 18.2 (CLDN18.2). The regulatory decision specifically covers patients whose cancer is negative for a different cancer protein called HER2. Drugs are already available for HER2-positive cancers. —In other Astellas news, the Japanese pharma company withdrew its European Medicines Agency application for geographic atrophy drug avacincaptad pegol. The company said it made the decision after discussions with the agency’s Committee for Medicinal Products for Human Use. The drug, administered as a once-monthly eye injection, won FDA approval in geographic atrophy last year and is marketed in the U.S. under the brand name Izervay. Failure to secure approval in Europe will make it harder for Astellas to recoup the $5.9 billion it spent to acquire Iveric Bio, the company that developed the drug. —Pfizer respiratory syncytial virus vaccine Abrysvo expanded its FDA approval to include adults age 18 through 59, broadening the market for the product. The initial FDA approval of Abrysvo last year covered patients age 60 and older. It’s also approved for use by pregnant individuals as a maternal vaccine; antibodies produced by the mother confer protection against RSV intended to last from the baby’s birth up to six months of age. —Gilead Sciences’ Trodelvy will no longer be available in the U.S. for treating urothelial carcinoma, an aggressive bladder cancer. After discussions with the FDA, the company decided to voluntarily withdraw the product, which had won accelerated approval in this indication in 2021. In May, the company reported Trodelvy failed its confirmatory Phase 3 test in advanced urothelial carcinoma. The withdrawal in urothelial carcinoma does not affect drug’s approved uses in breast cancer. —After previous regulatory setbacks, AbbVie finally landed FDA approval for Vyalev, a Parkinson’s disease drug/device combination product that provides 24 hour subcutaneous infusion of the medications foscarbidopa and foslevodopa. Last year, the regulator turned down the product’s application, asking for more information about the pump in the device. The FDA did not raise any questions about safety or efficacy, nor did it ask for another clinical trial. This past June, the agency again turned down AbbVie’s application for issues at the product’s third-party manufacturer. —There’s a new Pfizer drug for both hemophilia A and B. The FDA approved the antibody drug marstacimab for preventing or reducing frequency of bleeding episodes in patients 12 and older. Pfizer will market the drug under the brand name Hympavzi. As a once-weekly injectable medication, Hympavzi will provide an alternative to IV-infused hemophilia therapies. —The FDA needs more time to review Intercept Pharmaceuticals’ application seeking full regulatory approval of Ocaliva, which had received accelerated approval in 2016 for the rare liver disease primary biliary cholangitis. Intercept, now a subsidiary of Alfasigma, said the agency did not provide a new date for a regulatory decision. —Alzheimer’s disease drug Leqembi hit a regulatory obstacle in Australia, where the Therapeutic Goods Administration decided against registering the medication. Eisai said it will request a reconsideration as permitted under Australian law. It’s the second setback for the drug in recent months. Over the summer, the drug received a negative opinion from a European Medicines Agency committee concerned about the risk of severe side effects and limited benefit. Leqembi has approvals in the U.S., Japan, China, and several other markets. —The FDA approved a Thermo Fisher Scientific companion diagnostic to Voranigo, a Servier Pharmaceuticals’ drug approved over the summer for treating patients whose glioma is driven by mutations to IDH1 or IDH2 enzymes. The diagnostic, called Ion Torrent Oncomine Dx Target Test, identifies patients whose cancer carries the genetic signature making them eligible for treatment with the Servier drug. —Advanced cases of non-small cell lung cancer can now be treated with a wearable device. The FDA approved Novocure’s Optune Lua, a wearable technology that kills cancer cells with electrical signals administered via electrodes placed on the skin. Novocure’s “tumor-treating field” technology was first approved in 2011 as a treatment for a recurrent type of brain cancer. —The FDA rejected Zealand Pharma’s dasiglucagon, a drug developed to prevent and treat hypoglycemia in children who have the rare disease congenital hyperinsulinism. According to the Copenhagen-based biotech, the regulator cited issues at a third-party manufacturer. The FDA did not raise any concerns about the drug’s clinical data or safety. Advisory Committee Outcomes —An FDA advisory committee voted 11 to 3 that the benefits of Lexicon Pharmaceuticals’ sotagliflozin do not outweigh its risks when used as an adjunct to insulin to treat adults with type 1 diabetes and chronic kidney disease. But some committee members expressed support for use of the drug in subpopulations of patients. Sotagliflozin, given the brand name Zynquista, is expected to receive an FDA decision by Dec. 20. Sotagliflozin was approved last year for the treatment of heart failure and is marketed as Inpefa in this indication. —The European Medicine Agency’s Committee for Medicinal Products for Human Use yet again recommended against renewing the marketing authorization for Translarna, a PTC Therapeutics drug for Duchenne muscular dystrophy. Translarna received conditional marketing authorization in 2014. After the drug failed its Phase 3 study, the committee twice recommended against renewing the authorization. The latest opinion comes after the EMA directed the committee to reconsider, taking into account “the totality of evidence.” The committee said its reassessment “concluded that the effectiveness of Translarna has not been confirmed.” —Stealth BioTherapeutics moved a step closer to potential approval of the first drug for Barth syndrome. An FDA advisory committee voted 10 to 6 that Stealth’s drug, elamipretide, was effective at treating the ultra-rare mitochondrial disorder. The committee vote follows several regulatory setbacks, including a 2021 refuse-to-file letter in which the FDA pointed out that elamipretide failed its clinical trial. Stealth maintains there was improvement in a subgroup of patients and the short duration of treatment is the reason for the trial failure. An FDA decision is expected by Jan. 29. What’s New in Covid-19 —The European Commission granted marketing authorization to Novavax’s updated Covid-19 vaccine, Nuvaxovid. Authorization for the protein-based vaccine covers its use in those age 12 and older. The FDA authorized the updated version of Novavax’s Covid-19 vaccine in September. —In other Novavax Covid-19 news, the FDA pressed pause on the company’s plans for late-stage testing of a combination flu/Covid-19 vaccine as well as a standalone influenza vaccine. According to Novavax, the clinical hold stems from a report of motor neuropathy in a single patient outside the U.S. who received the combination vaccine in a Phase 2 study. The trial was completed in July 2023 and the serious adverse event was reported in September. Novavax said previous Covid-19 and influenza trials showed no signs of motor neuropathy. The company’s standalone Covid-19 vaccine is unaffected by the clinical hold. —In Covid-19 and flu testing news, the FDA granted marketing authorization to a Healgen Scientific over-the-counter, at-home test for both pathogens. The OTC Healgen Rapid Check Covid-19/Flu A&B Antigen Test uses a sample from a nasal swab. The authorization covers use of the product by people age 14 and older within five days of the start of symptoms. For children as young as 2, the sample must be collected by an adult.

Drug ApprovalPhase 3VaccineAcquisitionClinical Result

07 Nov 2024

·www.prnewswire.com

PTC Therapeutics Provides Corporate Update and Reports Third Quarter 2024 Financial Results

– Strong revenue performance, supporting increase in full-year revenue guidance to $750-800 million – – Regulatory filings for sepiapterin, Translarna™ and AADC gene therapy under review by FDA – – On track to achieve remaining 2024 clinical and regulatory milestones, including NDA submission for vatiquinone – WARREN, N.J., Nov. 7, 2024 /PRNewswire/ -- PTC Therapeutics, Inc., (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2024. "I am proud of our team's strong execution," said Matthew Klein, M.D., Chief Executive Officer. "We continue to achieve excellent revenue performance allowing us to raise full-year revenue guidance. In addition, we have submitted three approval applications to FDA so far this year, all of which have been accepted for review, and plan a fourth submission for vatiquinone for Friedreich ataxia in December. We are on our way to accomplishing all of our 2024 objectives and are in a strong financial position to execute on our long-term strategic plans." Key Corporate Updates: Third quarter 2024 total revenue of $197 million Third quarter 2024 revenue for the DMD franchise was $124 million Translarna™ (ataluren) net product revenue was $72 million, driven by continued access for patients in existing geographies, continued geographic expansion, high compliance and adherence, and dose adjustments. Emflaza® (deflazacort) net product revenue was $52 million, driven by new patient starts, continued brand loyalty, high compliance and adherence, and dose adjustments. Key Clinical and Regulatory Milestones: PTC submitted an NDA to the FDA for sepiapterin for the treatment of PKU. The file was accepted for review, and the regulatory action date is set for July 29, 2025. PTC resubmitted the NDA for Translarna for the treatment of nmDMD; the file was accepted, and a regulatory action date was not provided. The BLA for PTC's gene therapy for AADC deficiency remains on schedule for a regulatory action date of November 13, 2024. PTC received Fast Track Designation for the PTC518 Huntington's disease program. PTC plans to submit an NDA for vatiquinone for the treatment of Friedreich ataxia in December 2024. PTC expects to share topline data for the CardinALS trial of utreloxastat for the treatment of ALS in the fourth quarter of 2024. Third Quarter 2024 Financial Highlights: Total revenues were $196.8 million for the third quarter of 2024, compared to $196.6 million for the third quarter of 2023. Total revenue includes net product revenue across the commercial portfolio of $135.4 million for the third quarter of 2024, compared to $144.0 million for the third quarter of 2023. Total revenue also includes royalty and manufacturing revenue of $61.4 million in the third quarter of 2024, compared to $52.5 million for the third quarter of 2023. Translarna net product revenues were $72.3 million for the third quarter of 2024, compared to $69.0 million for the third quarter of 2023. Emflaza net product revenues were $51.9 million for the third quarter of 2024, compared to $67.4 million for the third quarter of 2023. Roche reported Evrysdi® 2024 year-to-date sales of approximately CHF 1.2 billion, resulting in royalty revenue of $61.4 million to PTC for the third quarter of 2024, compared to $50.2 million for the third quarter of 2023. Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $161.4 million for the third quarter of 2024, compared to $164.2 million for the third quarter of 2023. The decrease in research and development expenses reflects strategic portfolio prioritization, as PTC continues to focus its resources on its differentiated, high-potential R&D programs. Included in third quarter 2024 R&D expense is $50.0 million in regulatory success-based milestones related to the Censa acquisition. Non-GAAP R&D expenses were $152.0 million for the third quarter of 2024, excluding $9.4 million in non-cash, stock-based compensation expense, compared to $150.2 million for the third quarter of 2023, excluding $14.0 million in non-cash, stock-based compensation expense. GAAP SG&A expenses were $73.5 million for the third quarter of 2024, compared to $80.9 million for the third quarter of 2023. The decrease in selling, general and administrative expenses reflects lower employee costs as a result of the reduction in workforce in 2023. Non-GAAP SG&A expenses were $63.1 million for the third quarter of 2024, excluding $10.3 million in non-cash, stock-based compensation expense, compared to $67.9 million for the third quarter of 2023, excluding $13.0 million in non-cash, stock-based compensation expense. Change in the fair value of deferred and contingent consideration was a loss of $0.7 million for the third quarter of 2024, compared to a loss of $1.5 million for the third quarter of 2023. Tangible asset impairment and losses (gains) on transactions, net, was a loss of $1.8 million for the third quarter of 2024, compared to $0.0 million for the third quarter of 2023. These results were primarily related to fixed asset impairments. Net loss was $106.7 million for the third quarter of 2024, compared to net loss of $133.0 million for the third quarter of 2023. Cash, cash equivalents, and marketable securities was $1,013.4 million on September 30, 2024, compared to $876.7 million on December 31, 2023. Shares issued and outstanding as of September 30, 2024, were 76,952,124. PTC Updates Full Year 2024 Financial Guidance: PTC anticipates total revenues for full year 2024 to be between $750 million and $800 million. PTC anticipates GAAP R&D and SG&A expenses for full year 2024 to be between $740 million and $835 million, including expected R&D expense milestone payments of up to $65 million. PTC anticipates non-GAAP R&D and SG&A expenses for full year 2024 to be between $660 million and $755 million, including expected R&D expense milestone payments of up to $65 million and excluding estimated non-cash, stock-based compensation expense of $80 million. PTC anticipates up to $90 million of payments for full year 2024 upon achievement of potential regulatory success-based milestones from previous acquisitions, of which up to $65 million will be recorded as R&D operating expenses. As of September 30, 2024, $85 million in milestones have been achieved, of which $65 million has been recorded as R&D operating expenses and $20 million as contingent consideration payable. 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 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: AADC: Aromatic l-Amino Acid Decarboxylase ALS: Amyotrophic Lateral Sclerosis BLA: Biologics License Application CHF: Confoederatio Helvetica Francs (Swiss francs) DMD: Duchenne Muscular Dystrophy FA: Friedreich Ataxia FDA: U.S. Food and Drug Administration GAAP: Generally Accepted Accounting Principles HD: Huntington's Disease NDA: New Drug Application 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 call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at . A replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call. About PTC Therapeutics, Inc. PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to children and adults living with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company's strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at and follow us on Facebook, X, and LinkedIn. For More Information: Investors: Ellen Cavaleri +1 (615) 618-6228 [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 2024 Financial Guidance", including with respect to (i) 2024 total revenue guidance, (ii) 2024 GAAP and non-GAAP R&D and SG&A expense guidance and (iii) 2024 acquisition related milestone payment 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, commercialization and other matters with respect to its products and product candidates; PTC's strategy, future operations, future financial position, future revenues, projected costs; the extent, timing and financial aspects of our strategic pipeline prioritization and reductions in workforce; 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," 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; PTC's ability to maintain its marketing authorization of Translarna for the treatment of nmDMD in Brazil, Russia, the European Economic Area (EEA) and other regions, including whether the European Commission adopts the negative opinion from the Committee for Medicinal Products for Human Use (CHMP) for the conditional marketing authorization for Translarna in the EEA, or PTC's ability to identify other potential mechanisms by which it may provide Translarna to nmDMD patients in the EEA; PTC's ability to use the clinical data from its international drug registry study and real-world evidence concerning Translarna's benefits to support a continued marketing authorization for Translarna for the treatment of nmDMD in the EEA; PTC's ability to use the results of Study 041, a randomized, 18-month, placebo-controlled clinical trial of Translarna for the treatment of nmDMD followed by an 18-month open-label extension, and from its international drug registry study to support a marketing approval for Translarna for the treatment of nmDMD in the United States; whether investigators agree with PTC's interpretation of the results of clinical trials and the totality of clinical data from its trials in Translarna; expectations with respect to Upstaza, including any regulatory submissions and potential approvals, commercialization, manufacturing capabilities, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to sepiapterin, including any regulatory submissions and potential approvals, commercialization, the potential achievement of development, regulatory and sales milestones and contingent payments that PTC may be obligated to make; expectations with respect to the commercialization of Evrysdi under PTC's SMA collaboration; expectations with respect to the commercialization of Tegsedi and Waylivra; the timing of and actual expenses incurred in connection with the discontinuation of PTC's preclinical and early research programs in gene therapy and reductions in workforce, which may be in different periods and may be materially higher than estimated; the savings that may result from the discontinuation of PTC's strategic pipeline prioritization and reductions in workforce, which may be materially less than expected; 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 most recent 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 Translarna, Emflaza, Upstaza, Evrysdi, Tegsedi, Waylivra or sepiapterin. 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Financial StatementNDA

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	EU	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	IS	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	LI	PTC Therapeutics International Ltd.	31 Jul 2014
Muscular Dystrophy, Duchenne	NO	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	EU	PTC Therapeutics, Inc.	-
Becker Muscular Dystrophy	Phase 3	AU	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	BE	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	CA	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	FR	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	DE	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	IL	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	IT	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	ES	PTC Therapeutics, Inc.	20 May 2012
Becker Muscular Dystrophy	Phase 3	SE	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 \| NCT01826487 \| NCT00592553 (Study 041 \| Study 007 \| ACT DMD, AAN2024)	Phase 2/3	Muscular Dystrophy, Duchenne	-	Ataluren	hyunaxdfpy(crdpdjwbsl) = nhadgeiwxn lrnfznwckp (xgexfjmnwn ) View more	Positive	09 Apr 2024
NCT04336826 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	qaxcyaznqf(spmhqzwegp) = dnsvndeqhw tbhknpiehz (lmzrhujsbf, jibmezgeot - iwllhhwsdo) View more	-	01 Apr 2024
NCT03179631 (Study 041, AAN2023)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation (nm) DMD	-	Ataluren	tdznolnraz(mgqtdwwpnm) = cfluuzyxfg hqcvuyuvww (mwpzbfbmkp ) View more	Positive	25 Apr 2023
NCT03179631 (Study 041, MDA2023)	Phase 3	Muscular Dystrophy, Duchenne	-	Ataluren	mmorgfkcfh(sgbyzlvrwh) = yoauhzyfvj nhdpklcnlp (wwvfsixkhz ) View more	Positive	19 Mar 2023
Pubmed \| Cochrane Database Syst Rev	Not Applicable	Cystic Fibrosis	517	Ataluren and similar compounds	nidhgdywul(xuzbaafoah) = 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) omkwjaljsv (jezkjgkife ) View more	-	03 Mar 2023
Pubmed \| Cochrane Database Syst Rev	Not Applicable	Cystic Fibrosis	517	Placebo		-	03 Mar 2023
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Ataluren (Ataluren)	rmidwbfgon(gahqfcmsbo) = qfothkrrzk etlygyohsy (iaachqrlgh, swqdzooizr - nvuwfzqpzx) View more	-	27 May 2022
NCT02647359 (STAR, CTgov)	Phase 2	Aniridia	39	Placebo+Ataluren (Placebo)	rmidwbfgon(gahqfcmsbo) = rsdummllvw etlygyohsy (iaachqrlgh, buzgnglnxh - ticwxvnsaw) View more	-	27 May 2022
NCT03648827 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	20	Ataluren	tjizdgzwtd(opoamiswkx) = mvqyglhaos raapyuedcf (mdkkxoeutp, icixfbxsio - zxozutkfre) View more	-	05 Apr 2022
NCT03796637 (CTgov)	Phase 2	Muscular Dystrophy, Duchenne	6	Ataluren	twupigkytv(xhyzltxhml) = veolqekync cpfssdcybj (bdzzuhxlfv, cidixdktbg - jnhaeakqdf) View more	-	05 Apr 2022
NCT01557400 (Study 019, MDA2021)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation	94	Ataluren 10, 10, 20 mg/kg	rilhyhcnir(eusudkbjmc) = a trend in delay in decline to predicted FVC <50% by ~1 year pwnsgqowuu (dfmpfbkdtu )	Positive	01 Mar 2021
NCT01557400 (Study 019, MDA2021)	Phase 3	Muscular Dystrophy, Duchenne nonsense mutation	94	Ataluren		Positive	01 Mar 2021
NCT01557400 (Study 019, CTgov)	Phase 3	Becker Muscular Dystrophy \| Duchenne and Becker Muscular Dystrophy \| Muscular Dystrophy, Duchenne	94	Ataluren	sjnfhlxvos(szkhfvtddg) = geenaleurf jgncgqcggv (kfunnwigaw, ivikathrzh - sfhtkbctch) View more	-	25 Nov 2020

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

Chat with Hiro

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.

Start your data trial now!

Synapse data is also accessible to external entities via APIs or data packages. Empower better decisions with the latest in pharmaceutical intelligence.

Bio

Bio Sequences Search & Analysis

Chemical

Chemical Structures Search & Analysis