Biotech · Public company · Warren, NJ, US

Ptc Therapeutics, Inc.

Ticker PTCT · SEC CIK 0001070081

Track this companyFree. Adds Ptc Therapeutics, Inc. to your Dealase workspace and tells you when its record changes.

Clinical trials256 recruiting or active

Pipeline programs20From trials and the company's site

Disclosed deals0None found yet

Cash runwayNot availableNo recent cash burn filed

Pipeline20

AtalurenPhase 3 · Muscular Dystrophy, DuchenneClinicalTrials.gov · fetched Nov 12, 2026
AtalurenPhase 3 · Duchenne Muscular DystrophyClinicalTrials.gov · fetched Nov 12, 2026
DacarbazinePhase 3 · LeiomyosarcomaClinicalTrials.gov · fetched Nov 12, 2026
DeflazacortPhase 3 · Limb-Girdle Muscular DystrophyClinicalTrials.gov · fetched Nov 12, 2026
PTC299Phase 3 · PneumoniaClinicalTrials.gov · fetched Nov 12, 2026
PTC923Phase 3 · PhenylketonuriaClinicalTrials.gov · fetched Nov 12, 2026
SepiapterinPhase 3 · PhenylketonuriaClinicalTrials.gov · fetched Nov 12, 2026
SOCPhase 3 · PneumoniaClinicalTrials.gov · fetched Nov 12, 2026
UnesbulinPhase 3 · LeiomyosarcomaClinicalTrials.gov · fetched Nov 12, 2026
VatiquinonePhase 3 · Mitochondrial DiseasesClinicalTrials.gov · fetched Nov 12, 2026
VatiquinonePhase 3 · Friedreich AtaxiaClinicalTrials.gov · fetched Nov 12, 2026
VatiquinonePhase 3 · Friedreich's AtaxiaClinicalTrials.gov · fetched Nov 12, 2026
VatiquinonePhase 3 · Inherited Mitochondrial DiseaseClinicalTrials.gov · fetched Nov 12, 2026
AtalurenPhase 2 · Nonsene Mutation Duchenne Muscular DystrophyClinicalTrials.gov · fetched Nov 12, 2026
Eladocagene ExuparvovecPhase 2 · AADC DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
EPI-743Phase 2 · Leigh SyndromeClinicalTrials.gov · fetched Nov 12, 2026
PTC844Phase 2 · Rheumatoid ArthritisClinicalTrials.gov · fetched Nov 12, 2026
PTC857Phase 2 · Amyotrophic Lateral SclerosisClinicalTrials.gov · fetched Nov 12, 2026
PTC923Phase 2 · BH4 DeficiencyClinicalTrials.gov · fetched Nov 12, 2026
EPI-743Mitochondrial DiseasesClinicalTrials.gov · fetched Nov 12, 2026

Clinical trials25

Study of PTC844 in Participants With Active Rheumatoid ArthritisPHASE2 · RECRUITING · started Sep 24, 2026ClinicalTrials.gov NCT07779616 · fetched Nov 12, 2026
Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)PHASE3 · NOT_YET_RECRUITING · started Aug 15, 2026ClinicalTrials.gov NCT07681713 · fetched Nov 12, 2026
A Study of Sepiapterin in Participants With Phenylketonuria (PKU)PHASE3 · RECRUITING · started Mar 4, 2024ClinicalTrials.gov NCT06302348 · fetched Nov 12, 2026
A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich AtaxiaPHASE3 · ACTIVE_NOT_RECRUITING · started Dec 8, 2022ClinicalTrials.gov NCT05515536 · fetched Nov 12, 2026
A Study of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaPHASE2 · COMPLETED · started Oct 12, 2022ClinicalTrials.gov NCT05485987 · fetched Nov 12, 2026
A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial DiseasePHASE3 · COMPLETED · started Jun 22, 2022ClinicalTrials.gov NCT05218655 · fetched Nov 12, 2026
A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)PHASE2_PHASE3 · TERMINATED · started May 23, 2022ClinicalTrials.gov NCT05269355 · fetched Nov 12, 2026
Study to Assess the Effects of PTC857 Treatment in Participants With Amyotrophic Lateral Sclerosis ALSPHASE2 · TERMINATED · started May 15, 2022ClinicalTrials.gov NCT05349721 · fetched Nov 12, 2026
A Long-Term Safety Study of PTC923 in Participants With PhenylketonuriaPHASE3 · COMPLETED · started Feb 14, 2022ClinicalTrials.gov NCT05166161 · fetched Nov 12, 2026
A Study to Evaluate the Safety and Pharmacokinetics of Ataluren in Participants From ≥6 Months to <2 Years of Age With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)PHASE2 · COMPLETED · started Dec 29, 2021ClinicalTrials.gov NCT04336826 · fetched Nov 12, 2026
A Study of PTC923 in Participants With PhenylketonuriaPHASE3 · COMPLETED · started Sep 30, 2021ClinicalTrials.gov NCT05099640 · fetched Nov 12, 2026
A Study of SmartFlow Magnetic Resonance (MR) Compatible Ventricular Cannula for Administering Eladocagene Exuparvovec to Pediatric ParticipantsPHASE2 · ACTIVE_NOT_RECRUITING · started May 12, 2021ClinicalTrials.gov NCT04903288 · fetched Nov 12, 2026
A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich AtaxiaPHASE2_PHASE3 · COMPLETED · started Dec 17, 2020ClinicalTrials.gov NCT04577352 · fetched Nov 12, 2026
A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory EpilepsyPHASE2_PHASE3 · TERMINATED · started Sep 28, 2020ClinicalTrials.gov NCT04378075 · fetched Nov 12, 2026
A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)PHASE2_PHASE3 · TERMINATED · started Jul 9, 2020ClinicalTrials.gov NCT04439071 · fetched Nov 12, 2026
A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)PHASE3 · TERMINATED · started Oct 31, 2019ClinicalTrials.gov NCT03783923 · fetched Nov 12, 2026
A Study of Unesbulin (PTC596) in Combination With Dacarbazine in Participants With Advanced Leiomyosarcoma (LMS)PHASE1 · COMPLETED · started Mar 13, 2019ClinicalTrials.gov NCT03761095 · fetched Nov 12, 2026
A Study of PTC923 (CNSA-001) in Primary Tetrahydrobiopterin (BH4) Deficient Participants With HyperphenylalaninemiaPHASE1_PHASE2 · COMPLETED · started Jan 3, 2019ClinicalTrials.gov NCT03519711 · fetched Nov 12, 2026
A Phase 1b Study of PTC596 in Children With Newly Diagnosed Diffuse Intrinsic Pontine Glioma and High Grade GliomaPHASE1 · ACTIVE_NOT_RECRUITING · started Aug 1, 2018ClinicalTrials.gov NCT03605550 · fetched Nov 12, 2026
Unesbulin in Women With Ovarian Cancer Receiving Neoadjuvant ChemotherapyPHASE1 · COMPLETED · started Aug 28, 2017ClinicalTrials.gov NCT03206645 · fetched Nov 12, 2026
Long-Term Outcomes of Ataluren in Duchenne Muscular DystrophyPHASE3 · COMPLETED · started Jul 6, 2017ClinicalTrials.gov NCT03179631 · fetched Nov 12, 2026
Ataluren for Nonsense Mutation in CDKL5 and Dravet SyndromePHASE2 · COMPLETED · started Nov 1, 2016ClinicalTrials.gov NCT02758626 · fetched Nov 12, 2026
Long-Term Safety and Efficacy Evaluation of EPI-743 in Children With Leigh SyndromePHASE2 · COMPLETED · started Jan 31, 2014ClinicalTrials.gov NCT02352896 · fetched Nov 12, 2026
Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)PHASE3 · TERMINATED · started Nov 22, 2010ClinicalTrials.gov NCT01247207 · fetched Nov 12, 2026
EPI-743 for Mitochondrial Respiratory Chain DiseasesNO_LONGER_AVAILABLEClinicalTrials.gov NCT01370447 · fetched Nov 12, 2026

SEC filings12

8-K · 8-KSEC EDGAR · filed Nov 3, 2026 · fetched Nov 12, 2026
10-Q · 10-QSEC EDGAR · filed Oct 29, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Oct 29, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Sep 21, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Aug 28, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Aug 13, 2026 · fetched Nov 12, 2026
10-Q · 10-QSEC EDGAR · filed Jul 30, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Jul 30, 2026 · fetched Nov 12, 2026
8-K · FORM 8-KSEC EDGAR · filed Jun 18, 2026 · fetched Nov 12, 2026
8-K · FORM 8-KSEC EDGAR · filed Jun 16, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Jun 9, 2026 · fetched Nov 12, 2026
8-K · 8-KSEC EDGAR · filed Jun 5, 2026 · fetched Nov 12, 2026