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PTC THERAPEUTICS, INC. (PTCT)

PTC Therapeutics, Inc. (PTCT) is a biopharmaceutical company committed to the research, development, and commercialization of innovative therapies for patients afflicted with rare genetic disorders. Its robust pipeline encompasses both commercialized therapies and a variety of experimental drug candidates, spanning all stages of development—from early research and preclinical studies to clinical trials—with a primary focus on addressing various rare disease indications. Among its commercialized products, PTC Therapeutics provides Translarna and Emflaza, offering therapeutic options for Duchenne muscular dystrophy patients in the European Economic Area and the United States. Translarna also addresses nonsense mutation Duchenne muscular dystrophy in Brazil and Russia. The company additionally commercializes Tegsedi and Waylivra for various rare conditions throughout Latin America and the Caribbean. Furthermore, in Brazil, PTC Therapeutics distributes Evrysdi as a treatment for spinal muscular atrophy (SMA) in patients aged two months and older. Leveraging its proprietary splicing platform, the company is also actively developing PTC518, a promising therapeutic candidate aimed at treating Huntington's disease. To bolster its research and development capabilities, PTC Therapeutics has established strategic collaborations, including partnerships with F. Hoffman-La Roche Ltd and Hoffman-La Roche Inc., as well as the Spinal Muscular Atrophy Foundation, focusing on advancing drug discovery and development research, particularly in regenerative medicine. Additionally, it collaborates with Akcea Therapeutics, Inc. for the commercialization of Tegsedi and Waylivra in the Latin America and Caribbean region. Established in 1998, PTC Therapeutics, Inc. maintains its corporate headquarters in South Plainfield, New Jersey.

Sector: Pharmaceutical Preparations

Industry: Biotechnology

PTC Therapeutics, Inc. logo

PTC Therapeutics, Inc.

NASDAQ:PTCTHealthcare
-6.97%$59.97
AH$61.55+2.63%vs close
Mkt Cap$5.01B
Float79.27M
O/S83.54M
Vol
$ Vol
RVOL
Inst Own-
SI10.5%
Earnings
Dilution
PTC Therapeutics, Inc. logoNASDAQ:PTCT$59.97-6.97%
Upcoming Catalysts
Phase Distribution
65 lead-sponsored trials·3 active·39 completed
Phase 112
Phase 1/21
Phase 227
Phase 2/34
Phase 318
Not applicable3
(11 more as collaborator)

Trials need funding. See this company's dilution risk and cash position.

Recent Deals
  • Sangamo Therapeutics, Inc.LICENSE IN
    $111M upfront/ $211M totalJan 1, 1970
    ST-920
Clinical Trials
Sponsor role
Status
Phase
  • Phase 3
    Completed

    Study of Ataluren (PTC124™) in Cystic Fibrosis

    Ataluren, Placebo

    Cystic Fibrosis

    Enrollment
    238
    Start
    Sep 2009
    Primary Completion
    Nov 2011
    Completion
    Nov 2011
    NCT00803205
  • Phase 3
    Completed

    Extension Study of Ataluren (PTC124) in Cystic Fibrosis

    Ataluren

    Cystic Fibrosis

    Enrollment
    191
    Start
    Aug 2010
    Primary Completion
    Dec 2013
    Completion
    Dec 2013
    NCT01140451
  • Phase 3
    Terminated

    Study of Ataluren in Previously Treated Participants With Nonsense Mutation Dystrophinopathy (nmDBMD)

    Ataluren

    Duchenne Muscular Dystrophy

    Enrollment
    265
    Start
    Nov 2010
    Primary Completion
    Feb 2026
    Completion
    Feb 2026
    NCT01247207
  • Phase 3
    Completed

    Study of Ataluren for Previously Treated Participants With Nonsense Mutation Duchenne/Becker Muscular Dystrophy (nmDBMD) in Europe, Israel, Australia, and Canada

    Ataluren

    Duchenne Muscular Dystrophy, Becker Muscular Dystrophy +1

    Enrollment
    94
    Start
    May 2012
    Primary Completion
    Jan 2018
    Completion
    Jan 2018
    NCT01557400
  • Phase 3
    Completed

    Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD)

    Ataluren, Placebo

    Muscular Dystrophy, Duchenne, Muscular Dystrophies +7

    Enrollment
    230
    Start
    Mar 2013
    Primary Completion
    Aug 2015
    Completion
    Aug 2015
    NCT01826487
  • Phase 3
    Terminated

    An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy

    Ataluren

    Muscular Dystrophy, Duchenne, Muscular Dystrophies +7

    Enrollment
    219
    Start
    Mar 2014
    Primary Completion
    Jun 2018
    Completion
    Jun 2018
    NCT02090959
  • Phase 3
    Terminated

    Study of Ataluren (PTC124) in Cystic Fibrosis

    Ataluren

    Cystic Fibrosis

    Enrollment
    61
    Start
    May 2014
    Primary Completion
    Jun 2017
    Completion
    Jun 2017
    NCT02107859
  • Phase 3
    Completed

    Study of Ataluren in Nonsense Mutation Cystic Fibrosis (ACT CF)

    Ataluren (PTC124®), Placebo

    Cystic Fibrosis

    Enrollment
    279
    Start
    Aug 2014
    Primary Completion
    Nov 2016
    Completion
    Nov 2016
    NCT02139306
  • Phase 3
    Terminated

    Extension Study of Ataluren in Participants With Nonsense Mutation Cystic Fibrosis

    Ataluren

    Cystic Fibrosis

    Enrollment
    246
    Start
    Aug 2015
    Primary Completion
    Jun 2017
    Completion
    Jun 2017
    NCT02456103
  • Phase 3
    Completed

    Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy

    Ataluren, PLACEBO

    Muscular Dystrophy, Duchenne, Muscular Dystrophies +7

    Enrollment
    360
    Start
    Jul 2017
    Primary Completion
    Mar 2022
    Completion
    Jul 2023
    NCT03179631
  • Phase 3
    Withdrawn

    A Study of Deflazacort (Emflaza®) in Participants With Duchenne Muscular Dystrophy (DMD)

    Deflazacort

    Duchenne Muscular Dystrophy

    Enrollment
    0
    Start
    Oct 2018
    Primary Completion
    Jul 2021est.
    Completion
    Jul 2021est.
    NCT03642145
  • Phase 3
    Terminated

    A Study of Deflazacort (Emflaza®) in Participants With Limb-Girdle Muscular Dystrophy 2I (LGMD2I)

    Deflazacort

    Limb-Girdle Muscular Dystrophy

    Enrollment
    11
    Start
    Oct 2019
    Primary Completion
    Jan 2021
    Completion
    Jan 2021
    NCT03783923
  • Phase 3
    Completed

    A Study of PTC923 in Participants With Phenylketonuria

    PTC923, Placebo

    Phenylketonuria

    Enrollment
    157
    Start
    Sep 2021
    Primary Completion
    Apr 2023
    Completion
    May 2023
    NCT05099640
  • Phase 3
    Completed

    A Long-Term Safety Study of PTC923 in Participants With Phenylketonuria

    PTC923

    Phenylketonuria

    Enrollment
    247
    Start
    Feb 2022
    Primary Completion
    Jul 2026
    Completion
    Jul 2026
    NCT05166161
  • Phase 3
    Completed

    A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease

    Vatiquinone

    Inherited Mitochondrial Disease

    Enrollment
    101
    Start
    Jun 2022
    Primary Completion
    Apr 2025
    Completion
    Apr 2025
    NCT05218655
  • Phase 3
    Active, not recruiting

    A Study to Assess the Safety and Efficacy of Vatiquinone in Participants With Friedreich Ataxia

    Vatiquinone

    Friedreich Ataxia

    Enrollment
    130
    Start
    Dec 2022
    Primary Completion
    Dec 2027est.
    Completion
    Dec 2027est.
    NCT05515536
  • Phase 3
    Recruiting

    A Study of Sepiapterin in Participants With Phenylketonuria (PKU)

    Sepiapterin

    Phenylketonuria

    Enrollment
    56
    Start
    Mar 2024
    Primary Completion
    Feb 2031est.
    Completion
    Feb 2031est.
    NCT06302348
  • Phase 3
    Not yet recruiting

    Long-Term Efficacy Study of Vatiquinone for the Treatment of Friedreich's Ataxia (FA)

    Vatiquinone

    Friedreich's Ataxia

    Enrollment
    120
    Start
    Aug 2026est.
    Primary Completion
    Mar 2029est.
    Completion
    Mar 2029est.
    NCT07681713
  • Phase 2/3
    Terminated

    A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy

    Vatiquinone

    Mitochondrial Diseases, Drug Resistant Epilepsy +6

    Enrollment
    68
    Start
    Sep 2020
    Primary Completion
    Mar 2023
    Completion
    Dec 2023
    NCT04378075
  • Phase 2/3
    Terminated

    A Study to Evaluate Efficacy and Safety of PTC299 (Emvododstat) in Hospitalized Participants With Coronavirus (COVID-19)

    PTC299, Placebo

    Pneumonia, COVID-19 +1

    Enrollment
    189
    Start
    Jul 2020
    Primary Completion
    Jul 2022
    Completion
    Jul 2022
    NCT04439071
  • Phase 2/3
    Completed

    A Study to Assess the Efficacy and Safety of Vatiquinone for the Treatment of Participants With Friedreich Ataxia

    Vatiquinone, Placebo

    Friedreich Ataxia

    Enrollment
    146
    Start
    Dec 2020
    Primary Completion
    Apr 2023
    Completion
    Oct 2023
    NCT04577352
  • Phase 2/3
    Terminated

    A Study of Unesbulin in Participants With Advanced Leiomyosarcoma (LMS)

    Unesbulin, Dacarbazine

    Leiomyosarcoma

    Enrollment
    359
    Start
    May 2022
    Primary Completion
    Jun 2024
    Completion
    Jul 2024
    NCT05269355
  • Phase 2
    Completed

    PTC124 for Cystic Fibrosis

    PTC124

    Cystic Fibrosis

    Enrollment
    24
    Start
    Sep 2005
    Primary Completion
    Aug 2006
    Completion
    Aug 2006
    NCT00234663
  • Phase 2
    Completed

    Safety and Efficacy of Ataluren (PTC124) for Cystic Fibrosis

    Ataluren

    Cystic Fibrosis

    Enrollment
    24
    Start
    Nov 2005
    Primary Completion
    May 2006
    Completion
    May 2006
    NCT00237380
  • Phase 2
    Completed

    Safety and Efficacy Study of PTC124 in Duchenne Muscular Dystrophy

    PTC124

    Duchenne Muscular Dystrophy

    Enrollment
    38
    Start
    Dec 2005
    Primary Completion
    May 2007
    Completion
    May 2007
    NCT00264888

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