Drug intelligence / Profile preview

thiamine + biotin

Development stage
Phase 2
Lead developer
Lydian
Modality
Orthosteric Ligands → Classical Binding Small Molecules → Small Molecules, Metabolically Activated Prodrugs → Prodrugs/Conditional Activator Small Molecules → Small Molecules
Administration
Oral, Intravenous, Intramuscular
01

Overview

Thiamine + biotin is a combination of two water-soluble B vitamins, thiamine (vitamin B1) and biotin (vitamin B7). Thiamine acts as an essential cofactor in carbohydrate metabolism and energy production, playing a critical role in the function of the nervous system. Biotin functions as a coenzyme for carboxylase enzymes involved in fatty acid synthesis, amino acid catabolism, and gluconeogenesis. The combination is used therapeutically to treat or prevent deficiencies of these vitamins due to poor diet, certain illnesses, alcoholism, or increased requirements such as during pregnancy. Notably, high-dose thiamine plus biotin supplementation is the standard treatment for biotin-thiamine-responsive basal ganglia disease (BTRBGD), a rare inherited neurological disorder caused by mutations affecting thiamine transport into cells[2][3][7]. In this context, early administration can be life-saving and may prevent irreversible neurological damage.

Other names
combined thiamine and biotin therapyhigh-dose thiamine and biotin
02

Targets

ACC (Acetyl-CoA Carboxylase 1)α-KGDH (Alpha-ketoglutarate dehydrogenase complex)PCCA (Propionyl-CoA carboxylase alpha subunit)PROC (Protein C)PDHA1 (Pyruvate dehydrogenase (lipoamide) alpha 1)TKT (Transketolase)PDH (Pyruvate dehydrogenase complex)

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