Shots:

• The company has stopped the P-II/III STAGED-PKD study for not meeting futility criteria that assess venglustat vs PBO in patients with ADPKD- following an independent analysis of the annualized rate of change in TKV
• The therapy did not bring a reduction in TKV growth rate and interim analysis showed the reduction of GSLs may not prevent kidney cyst growth and is not a primary pathway associated with the progression of ADPKD
• Biomarker data from the study confirmed that the therapy effectively inhibits the GSL pathway by a reduction in GL-1. The company is also evaluating the therapy for Gaucher disease type 3- Fabry disease- and GM2 Gangliosidosis

  Ref: Globe Newswire | Image: CNN