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[{"orgOrder":0,"company":"Flatley Discovery Lab","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces Initiation of a Registration-Directed Clinical Trial to Evaluate Vatiquinone in Mitochondrial Epilepsy","therapeuticArea":"Neurology","highestDevelopmentStatus":"Phase II\/ Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Neurology","graph2":"Phase II\/ Phase III"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces Topline Results from Vatiquinone MOVE-FA Registration-Directed Trial","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces Results from MIT-E Clinical Trial of Vatiquinone for the Treatment of Mitochondrial Disease Associated Seizures","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            Details:

            PTC743 (vatiquinone) is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Mitochondrial Disease Associated Seizures.

            Lead Product(s): Vatiquinone

            Therapeutic Area: Genetic Disease Product Name: PTC743

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 29, 2023

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            Vatiquinone is a small molecule, first-in-class selective inhibitor of 15-Lipoxygenase (15-LO), an enzyme that is a key regulator of the energetic and oxidative stress pathways that are disrupted in Friedreich ataxia.

            Lead Product(s): Vatiquinone

            Therapeutic Area: Genetic Disease Product Name: PTC743

            Highest Development Status: Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable May 23, 2023

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            Vatiquinone, developed from PTC's Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways underpinning many neurological disease pathologies, including epilepsy.

            Lead Product(s): Vatiquinone

            Therapeutic Area: Neurology Product Name: PTC743

            Highest Development Status: Phase II/ Phase III Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable October 26, 2020

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