Methylsuccinic acid is a normal metabolite found in human fluids. Increased urinary levels of Methylsuccinic acid (together with ethylmalonic acid) are the main biochemical measurable features in ethylmalonic encephalopathy a rare metabolic disorder with an autosomal recessive mode of inheritance that is clinically characterized by neuromotor delay hyperlactic acidemia recurrent petechiae orthostatic acrocyanosis and chronic diarrhea.

KHK-IN-1 is a reversible and ATP-competitive inhibitor of ketohexokinase (KHK) with IC50 of 12 nM. In the ATP-binding region of KHK, KHK-IN-1 interacts with Asp-27B.

TFEB activator 1 is an activator?of transcription factor EB (TFEB) ?with promise for the prevention or treatment of Alzheimer's disease.