Development Strategy

• We shifted our strategy about two years ago from Gaucher disease to Alzheimer’s. Based on information Steve Pasternak found while he was doing his clinical study of Parkinson’s. We discovered that Ambroxol stimulates the TFEB system and does this by the lysosomal pathway clearing misfolded proteins, such as tau and Abeta.
• These three papers describe the unique characteristics of Ambroxol
  Ambroxol AD placement memo.pdf, Ambroxol as mAb adjuvant.pdf, AmbroxolMechanismsOfAction.pdf
• Ambroxol exerts strong effects on the autophagy lysosomal pathway. In addition, suppresses inflammation.
• Opportunities still exist in Gaucher disease, Type 3 as orphan and Type 1 as a larger market.
• In addition, the mechanism of clearing misfolded proteins is applicable for a wide range of neurological diseases; such as Parkinson’s, Lewy bodies, etc.
• Ambroxol operates primarily as a chaperone in Gaucher disease and Parkinson’s.