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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 IIIProduct 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 IIIProduct 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 IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: PTC Therapeutics
Deal Size: $366.0 million Upfront Cash: $10.0 million
Deal Type: Acquisition May 06, 2020