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Details:
MC-1 (pyridoxal phosphate) is a naturally occuring purinergic receptor antagonist, which is being evaluated for the treatment of Pyridox(am)Ine 5'-Phosphate Oxidase (PNPO) Deficiency.
Lead Product(s): Pyridoxal Phosphate
Therapeutic Area: Genetic Disease Product Name: MC-1
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 April 23, 2024
Details:
FDA provides complete approval to enroll patients in its pivotal clinical trial to evaluate "MC-1" (a naturally occurring metabolite of pyridoxine) for treatment of a rare pediatric disease called pyridox(am)ine 5′-phosphate oxidase deficiency.
Lead Product(s): Pyridoxal Phosphate
Therapeutic Area: Rare Diseases and Disorders Product Name: MC-1
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 November 23, 2023
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