Biotech Values

[genisi]

FOLD/amigal

They have 17 (out of 26) phase II patients remained on amigal monotherapy (slide 8) and my guess is these are the patients having 'amenable' mutations. If so, I am less bearish on the monotherapy treatment than before because certain mutations were one of the phase III study 011 inclusion criteria. Chaperone monotherapy failed in Gaucher and Pompe but perhaps it has a better chance in Fabry due to better selection of patients (mutations, urine GL-3 levels etc). When used in combo with ERT, chaperone treatment should work for all mutations as it binds to the exogenous enzyme not just the endogenous one and safety should also be better due to less frequent dosing.