SymbolPTCT
NamePTC THERAPEUTICS, INC.
SectorHEALTH CARE
RegionNorth America
IndustryBiotechnology: Pharmaceutical Preparations
Address500 WARREN CORPORATE CENTER DRIVE, WARREN, New Jersey, 07059, United States
Telephone+1 908 - 222-7000
Fax
Email
Websitehttps://www.ptcbio.com
IncorporationUNDEFINED
Incorporated On
Employees
Fiscal Year
Public Since
ExchangesNASDAQ
Auditor
Audit StatusNOT PROVIDED
Reporting Status
CIK0001070081
Description

PTC Therapeutics Inc is a global biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, small-molecule therapeutics. Its target area is RNA biology post-transcriptional control processes of the events occurring in a cell when a RNA messenger is copied from DNA during the transcription process. The discovered products address multiple therapeutic areas, including rare disorders, such as nonsense mutations in Duchenne muscular dystrophy and oncology.

Additional info from NASDAQ:
PTC Therapeutics Inc is a global biopharmaceutical company focused on the discovery, development, and commercialization of orally administered, small-molecule therapeutics. Its target area is RNA biology post-transcriptional control processes of the events occurring in a cell when a RNA messenger is copied from DNA during the transcription process. The discovered products address multiple therapeutic areas, including rare disorders, such as nonsense mutations in Duchenne muscular dystrophy and oncology.

2026-07-07 20:11

📋 Almstead Neil Gregory (Officer) plans to sell 25K shares of PTC THERAPEUTICS, INC. (at $88.88 each, total $2.2M) Filed: Jul 07, 2026 | ID: 005150

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2026-07-06 20:10

📋 Almstead Neil Gregory (Officer) plans to sell 22K shares of PTC THERAPEUTICS, INC. (at $86.00 each, total $1.9M) Filed: Jul 06, 2026 | ID: 005117

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2026-07-06 19:17

📋 Boulding Mark Elliott (Officer) plans to sell 5K shares of PTC THERAPEUTICS, INC. (at $84.14 each, total $427K) Filed: Jul 06, 2026 | ID: 005114

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2026-06-30 21:20

Almstead Neil Gregory 🟡 adjusted position in 8.1K shares (2 derivative) of PTC THERAPEUTICS, INC. (PTCT) at $85.26 ($1.3M) Transaction Date: Jun 26, 2026 | Filing ID: 000019

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2026-06-26 21:20

Almstead Neil Gregory 🟡 adjusted position in 26.3K shares (5 derivative) of PTC THERAPEUTICS, INC. (PTCT) at $87.00 ($4.3M) Transaction Date: Jun 24, 2026 | Filing ID: 000017

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2026-06-26 19:29

📋 Almstead Neil Gregory (Officer) plans to sell 8K shares of PTC THERAPEUTICS, INC. (at $85.26 each, total $693K) Filed: Jun 26, 2026 | ID: 004919

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2026-06-25 20:16

📋 Almstead Neil Gregory (Officer) plans to sell 21K shares of PTC THERAPEUTICS, INC. (at $85.97 each, total $1.8M) Filed: Jun 25, 2026 | ID: 004899

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2026-06-24 21:20

Almstead Neil Gregory 🟡 adjusted position in 23.7K shares (1 derivative) of PTC THERAPEUTICS, INC. (PTCT) at $81.21 ($3.1M) Transaction Date: Jun 22, 2026 | Filing ID: 000014

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2026-06-24 20:11

📋 Almstead Neil Gregory (Officer) plans to sell 5K shares of PTC THERAPEUTICS, INC. (at $85.03 each, total $453K) Filed: Jun 24, 2026 | ID: 004862

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2026-06-22 21:20

Utter Christine Marie 🟡 adjusted position in 19.7K shares (1 derivative) of PTC THERAPEUTICS, INC. (PTCT) at $75.56 ($2.5M) Transaction Date: Jun 17, 2026 | Filing ID: 000010

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Trial ID Title Phase Indication Status Start Date Completion Date Source
NCT01370447 EPI-743 for Mitochondrial Respiratory Chain Diseases Mitochondrial Diseases No_Longer_Available ClinicalTrials.gov
NCT07159139 Vatiquinone Expanded Access Protocol Mitochondrial Disease No_Longer_Available ClinicalTrials.gov
NCT02592941 Deflazacort Expanded Access Program for Children, Adolescents and Adults With D… Duchenne Muscular Dystrophy Approved_For_Marketing ClinicalTrials.gov
NCT07681713 Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxi… Phase3 Friedreich's Ataxia Not_Yet_Recruiting 2026-08-15 2029-03-15 ClinicalTrials.gov
NCT07614126 Study of L-dopa Treatment in Patients With a Neurodevelopmental Disorder (CTNNB… Na CTNNB1 Recruiting 2026-04-08 2027-05-01 ClinicalTrials.gov
NCT06302348 A Study of Sepiapterin in Participants With Phenylketonuria (PKU) Phase3 Phenylketonuria Recruiting 2024-03-04 2031-02-28 ClinicalTrials.gov
NCT05515536 A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With F… Phase3 Friedreich Ataxia Active_Not_Recruiting 2022-12-08 2027-12-31 ClinicalTrials.gov
NCT05485987 A Study of Vatiquinone for the Treatment of Participants With Friedreich Ataxia Phase2 Friedreich Ataxia Completed 2022-10-12 2024-08-29 ClinicalTrials.gov
NCT05218655 A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With In… Phase3 Inherited Mitochondrial Disease Completed 2022-06-22 2025-04-15 ClinicalTrials.gov
NCT05269355 A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS) Phase2 Leiomyosarcoma Terminated 2022-05-23 2024-07-17 ClinicalTrials.gov
NCT05349721 Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophi… Phase2 Amyotrophic Lateral Sclerosis Terminated 2022-05-15 2025-01-30 ClinicalTrials.gov
NCT05166161 A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria Phase3 Phenylketonuria Active_Not_Recruiting 2022-02-14 2026-06-30 ClinicalTrials.gov
NCT04336826 A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants… Phase2 Nonsene Mutation Duchenne Muscular Dystrophy Completed 2021-12-29 2023-08-07 ClinicalTrials.gov
NCT05099640 A Study of PTC923 in Participants With Phenylketonuria Phase3 Phenylketonuria Completed 2021-09-30 2023-05-03 ClinicalTrials.gov
NCT04903288 A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for… Phase2 AADC Deficiency Active_Not_Recruiting 2021-05-12 2028-04-30 ClinicalTrials.gov
NCT04577352 A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of P… Phase2 Friedreich Ataxia Completed 2020-12-17 2023-10-02 ClinicalTrials.gov
NCT04378075 A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondri… Phase2 Mitochondrial Diseases Terminated 2020-09-28 2023-12-27 ClinicalTrials.gov
NCT04439071 A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized… Phase2 Pneumonia Terminated 2020-07-09 2022-07-20 ClinicalTrials.gov
NCT03783923 A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dys… Phase3 Limb-Girdle Muscular Dystrophy Terminated 2019-10-31 2021-01-01 ClinicalTrials.gov
NCT04014530 Pembrolizumab With Ataluren in Patients With Metastatic pMMR and dMMR Colorecta… Phase1 Colorectal Cancer Recruiting 2019-08-01 2023-08-01 ClinicalTrials.gov
NCT03796637 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duch… Phase2 Duchenne Muscular Dystrophy Completed 2019-04-11 2019-06-03 ClinicalTrials.gov
NCT03761095 A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants W… Phase1 Leiomyosarcoma Completed 2019-03-13 2024-02-05 ClinicalTrials.gov
NCT03712124 A Study of CNSA-001 in Women With Diabetic Gastroparesis Phase2 Gastroparesis Completed 2019-02-27 2019-10-08 ClinicalTrials.gov
NCT03519711 A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Par… Phase1 BH4 Deficiency Completed 2019-01-03 2020-10-02 ClinicalTrials.gov
NCT04117880 A Phase 2 Open Label Extension Study in Participants With Nonsense Mutation Ani… Phase2 Aniridia Withdrawn 2018-12-31 2021-01-31 ClinicalTrials.gov
NCT03648827 A Study to Assess Dystrophin Levels in Participants With Nonsense Mutation Duch… Phase2 Duchenne Muscular Dystrophy Completed 2018-12-21 2020-10-23 ClinicalTrials.gov
NCT03642145 A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystro… Phase3 Duchenne Muscular Dystrophy Withdrawn 2018-10-31 2021-07-31 ClinicalTrials.gov
NCT03761069 Study of PTC299 (Emvododstat) in Relapsed/Refractory Acute Leukemias Phase1 Leukemia, Myeloid, Acute Terminated 2018-10-29 2021-12-28 ClinicalTrials.gov
NCT03605550 A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic P… Phase1 High Grade Glioma Active_Not_Recruiting 2018-08-01 2029-03-01 ClinicalTrials.gov
NCT03206645 Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant Chemotherapy Phase1 Ovarian Cancer Completed 2017-08-28 2024-03-31 ClinicalTrials.gov
NCT03179631 Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy Phase3 Muscular Dystrophy, Duchenne Completed 2017-07-06 2023-07-25 ClinicalTrials.gov
NCT02926066 A Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylase (AADC) De… Phase2 Aromatic Amino Acid Decarboxylase Deficiency Completed 2016-11-09 2022-05-16 ClinicalTrials.gov
NCT02758626 Ataluren for Nonsense Mutation in CDKL5 and Dravet Syndrome Phase2 Epilepsy Completed 2016-11-01 2021-02-27 ClinicalTrials.gov
NCT02819557 Study of Ataluren in ≥2 to <5 Year-Old Male Participants With Duchenne Muscular… Phase2 Duchenne Muscular Dystrophy Completed 2016-06-09 2018-02-09 ClinicalTrials.gov
NCT02647359 Study of Ataluren in Participants With Nonsense Mutation Aniridia Phase2 Aniridia Completed 2016-01-31 2021-01-22 ClinicalTrials.gov
NCT02460679 Safety and Biomarker Study of EPI-589 in Participants With Amyotrophic Lateral … Phase2 Amyotrophic Lateral Sclerosis Completed 2016-01-14 2018-02-23 ClinicalTrials.gov
NCT02404480 PTC596 in Patients With Advanced Solid Tumors Phase1 Cancer Completed 2016-01-01 2017-02-06 ClinicalTrials.gov
NCT02456103 Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibro… Phase3 Cystic Fibrosis Terminated 2015-08-31 2017-06-02 ClinicalTrials.gov
NCT02485431 Food Effect and Bioavailability of Deflazacort Formulations in Healthy Voluntee… Phase1 Healthy Volunteers Completed 2015-06-01 2015-08-01 ClinicalTrials.gov
NCT02369731 Registry of Translarna (Ataluren) in Nonsense Mutation Duchenne Muscular Dystro… Muscular Dystrophy, Duchenne Completed 2015-04-30 2025-03-31 ClinicalTrials.gov
NCT02409004 Effects of Rifampin on the Pharmacokinetics of Ataluren Phase1 Healthy Completed 2015-02-01 2015-03-01 ClinicalTrials.gov
NCT02251600 A Pharmacokinetic Study of Oral Deflazacort in Children and Adolescent Subjects… Phase1 Duchenne Muscular Dystrophy Completed 2014-12-01 2015-10-01 ClinicalTrials.gov
NCT02295748 An Open-Label, Long-Term Extension Study to Evaluate the Safety and Tolerabilit… Phase1 Duchenne Muscular Dystrophy Completed 2014-12-01 2017-08-24 ClinicalTrials.gov
NCT02286622 A Single Dose Evaluation of the Effects of Renal Impairment on Deflazacort Phar… Phase1 Renal Impairment Completed 2014-12-01 2015-02-01 ClinicalTrials.gov
NCT02286609 A Single Dose Evaluation of the Effects of Moderate Hepatic Impairment on Defla… Phase1 Hepatic Impairment Completed 2014-12-01 2015-02-01 ClinicalTrials.gov
NCT02286635 Evaluate Effects of Multiple Doses of Rifampin and Clarithromycin on the Single… Phase1 Healthy Volunteers Completed 2014-11-01 2014-12-01 ClinicalTrials.gov
NCT01395641 A Phase I/II Clinical Trial for Treatment of Aromatic L-amino Acid Decarboxylas… Phase1 Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Completed 2014-10-22 2022-03-07 ClinicalTrials.gov
NCT02139306 Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF) Phase3 Cystic Fibrosis Completed 2014-08-01 2016-11-01 ClinicalTrials.gov
NCT02107859 Study of Ataluren (PTC124) in Cystic Fibrosis Phase3 Cystic Fibrosis Terminated 2014-05-23 2017-06-05 ClinicalTrials.gov
NCT02090959 An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation … Phase3 Muscular Dystrophy, Duchenne Terminated 2014-03-20 2018-06-12 ClinicalTrials.gov
NCT02352896 Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh Synd… Phase2 Leigh Syndrome Completed 2014-01-31 2023-10-30 ClinicalTrials.gov
NCT01910168 A Study to Collect Blood Samples From Patients With Spinal Muscular Atrophy for… Muscular Atrophy, Spinal Completed 2013-08-01 2014-02-01 ClinicalTrials.gov
NCT01826487 Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscu… Phase3 Muscular Dystrophy, Duchenne Completed 2013-03-26 2015-08-20 ClinicalTrials.gov
NCT01728064 Safety and Efficacy of EPI-743 in Patients With Friedreich's Ataxia Phase2 Friedreich's Ataxia Completed 2012-12-31 2016-02-29 ClinicalTrials.gov
NCT01721733 Safety and Efficacy Study of EPI-743 in Children With Leigh Syndrome Phase2 Leigh Syndrome Completed 2012-10-31 2015-05-31 ClinicalTrials.gov
NCT01557400 Study of Ataluren for Previously Treated Participants With Nonsense Mutation Du… Phase3 Duchenne Muscular Dystrophy Completed 2012-05-20 2018-01-19 ClinicalTrials.gov
NCT01247207 Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dys… Phase3 Duchenne Muscular Dystrophy Completed 2010-11-30 2026-02-10 ClinicalTrials.gov
NCT01158300 PTC299 in Treating Young Patients With Refractory or Recurrent Primary Central … Phase1 Brain and Central Nervous System Tumors Completed 2010-11-01 2015-01-01 ClinicalTrials.gov
NCT01140451 Extension Study of Ataluren (PTC124) in Cystic Fibrosis Phase3 Cystic Fibrosis Completed 2010-08-12 2013-12-02 ClinicalTrials.gov
NCT01141075 Ataluren for Nonsense Mutation Methylmalonic Acidemia Phase2 Amino Acid Metabolism, Inborn Errors Terminated 2010-07-19 2011-11-03 ClinicalTrials.gov
NCT01009294 Study of Ataluren (PTC124) in Nonambulatory Participants With Nonsense-Mutation… Phase2 Duchenne Muscular Dystrophy Terminated 2010-01-13 2010-03-23 ClinicalTrials.gov
NCT00947193 Study of Ataluren (PTC124) in Hemophilia A and B Phase2 Hemophilia A Terminated 2009-10-14 2011-08-30 ClinicalTrials.gov
NCT00803205 Study of Ataluren (PTC124™) in Cystic Fibrosis Phase3 Cystic Fibrosis Completed 2009-09-08 2011-11-12 ClinicalTrials.gov
NCT00911248 PTC299 for Treatment of Neurofibromatosis Type 2 Phase2 Neurofibromatosis 2 Terminated 2009-07-31 2012-03-31 ClinicalTrials.gov
NCT00847379 Phase 2B Extension Study of Ataluren (PTC124) in Duchenne/Becker Muscular Dystr… Phase2 Duchenne Muscular Dystrophy Terminated 2009-01-31 2010-05-24 ClinicalTrials.gov
NCT00686842 PTC299 in Treating Patients With HIV-Related Kaposi Sarcoma Phase1 Kaposi's Sarcoma Terminated 2008-09-01 2010-12-01 ClinicalTrials.gov
NCT00759876 Phase 2a Extension Study of Ataluren (PTC124) in Duchenne Muscular Dystrophy (D… Phase2 Duchenne Muscular Dystrophy Terminated 2008-08-13 2010-05-17 ClinicalTrials.gov
NCT00704821 PTC299 for Treatment of Advanced Cancer Phase1 Advanced Cancer Terminated 2008-07-03 2012-02-24 ClinicalTrials.gov
NCT00592553 Phase 2B Study of PTC124 (Ataluren) in Duchenne/Becker Muscular Dystrophy (DMD/… Phase2 Duchenne Muscular Dystrophy Completed 2008-02-29 2009-12-31 ClinicalTrials.gov
NCT00508586 PTC299 and Hormonal Agent for Treatment of Metastatic Breast Cancer Phase1 Metastatic Breast Cancer Completed 2007-11-01 2012-03-01 ClinicalTrials.gov
NCT00458341 A Study of Ataluren in Pediatric Participants With Cystic Fibrosis Phase2 Cystic Fibrosis Completed 2007-03-23 2008-02-29 ClinicalTrials.gov
NCT00351078 PTC124 for the Treatment of Cystic Fibrosis Phase2 Cystic Fibrosis Completed 2006-12-01 2007-07-01 ClinicalTrials.gov
NCT00264888 Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy Phase2 Duchenne Muscular Dystrophy Completed 2005-12-01 2007-05-01 ClinicalTrials.gov
NCT00237380 Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis Phase2 Cystic Fibrosis Completed 2005-11-30 2006-05-31 ClinicalTrials.gov
NCT00234663 PTC124 for Cystic Fibrosis Phase2 Cystic Fibrosis Completed 2005-09-01 2006-08-01 ClinicalTrials.gov
Total clinical trials: 75
Product Name Type Development Stage Therapeutic Area Study Status Trial ID
Vatiquinone Other Phase PHASE3 Friedreich's Ataxia NOT_YET_RECRUITING NCT07681713
PTC923 Other Phase PHASE3 Phenylketonuria ACTIVE_NOT_RECRUITING NCT05166161
Sepiapterin Other Phase PHASE3 Phenylketonuria RECRUITING NCT06302348
L-Dopa Other Approved CTNNB1 RECRUITING NCT07614126
Total products: 4