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ACER-001

Acer Therapeutics, Inc.

Drug Names(s): NaPBA, ACER001, ACER 001

Description: NaPBA is a phenylbutyrate therapy in development for maple syrup urine disease. BCKDC (branched-chain alpha-keto acid dehydrogenase complex) is a complex involved with catabolism of branched-chain amino acids; deficiency of enzymes making up the BCKDC complex leads to maple syrup urine disease. Phenylbutyrate prevents phosphorylation of the E1a subunit of BCKDC through inhibition of the BCKDC kinase. This activates BCKDC overall activity, providing a molecular explanation for the effect of phenylbutyrate in a subset of MSUD patients.

Phenylbutyrate treatment may be a valuable treatment for reducing the plasma levels of neurotoxic BCAA and their corresponding BCKA in a subset of MSUD patients.

Deal Structure: Acer Therapeutics entered into an exclusive license agreement with Baylor College of Medicine to commercialize phenylbutyrate (NaPBA).


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