deuterated ambroxol

Zywie Bio is developing proprietary deuterated and halogenated analogues of ambroxol as small molecule pharmacological chaperones for the treatment of Gaucher disease and various neurodegenerative conditions, including Parkinson's disease and Alzheimer's disease. These compounds are designed to bind and stabilize glucocerebrosidase (GBA), an enzyme often deficient or dysfunctional in these diseases, thereby improving its lysosomal activity. Furthermore, the drug activates the Transcription Factor EB (TFEB) pathway, which stimulates lysosomal biogenesis and autophagy to facilitate the clearance of toxic protein aggregates like alpha-synuclein, tau, and amyloid-beta. While Zywie Bio leverages clinical data from Phase 2 trials of high-dose ambroxol in Parkinson's disease dementia and Alzheimer's disease, its proprietary deuterated analogues are intended to offer improved pharmacokinetic properties while maintaining the parent molecule's mechanism of action.

Other names

ambroxolhalogenated ambroxol