Minakem Generic APIs Minakem Generic APIs

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[{"orgOrder":0,"company":"Censa Pharmaceuticals","sponsor":"PTC Therapeutics","pharmaFlowCategory":"D","amount":"$366.0 million","upfrontCash":"$10.0 million","newsHeadline":"PTC Therapeutics to Acquire Censa Pharmaceuticals","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"PTC Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"PTC Therapeutics Announces APHENITY Trial Achieved Primary Endpoint with Sepiapterin in PKU Patients","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 Announces Submission of Sepiapterin MAA for Treatment of PKU to EMA","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]

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            Details:

            PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin that acts as nitric oxide synthase modulator. It is being evaluated for the treatment of Phenylketonuria.

            Lead Product(s): Sepiapterin

            Therapeutic Area: Genetic Disease Product Name: PTC923

            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 March 28, 2024

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            Details:

            PTC923 (sepiapterin) is a precursor to intracellular tetrahydrobiopterin, which is a critical enzymatic cofactor involved in the metabolism and synthesis of numerous metabolic products and has the potential to treat the broad range of PKU patients.

            Lead Product(s): Sepiapterin

            Therapeutic Area: Genetic Disease Product Name: PTC923

            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 17, 2023

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            Censa Pharmaceuticals acquisition diversifies and strengthens PTC 's portfolio of rare disorders by adding the lead orphan metabolic disease drug, CNSA-001.

            Lead Product(s): Sepiapterin

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: PTC Therapeutics

            Deal Size: $366.0 million Upfront Cash: $10.0 million

            Deal Type: Acquisition May 06, 2020

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