The phase III slide is not important because since then they got a SPA.
The drug will be given to untreated, symptomatic Gaucher patients (no healthy control required) and the relief in symptoms such as spleen size etc will be measured. This will prove efficacy in sick humans.
There was no efficacy in phase I, just safety and Pharmacology in healthy subjects.
Apart from that, they have shown via 3D crystallography that their molecule is similar to Cerezyme .
They have shown in vitro enzymatic activity and superiority in 2 models.
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