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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"}]
Find Clinical Drug Pipeline Developments & Deals for Sepiapterin
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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
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
Details:
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