Saturday, February 26, 2011 11:04:55 AM
genisi could give you a better answer and all the statistics (I am lazy 
) but I think most (perhaps all) ERT's in general have some degree of antibody formation. If they are neutralizing it will limit the potency of the ERT sometimes dosing can help get around it in some patients.
A couple things for long term PLX investors to keep in mind. Assuming the manufacturing issues are minor they can't just compete with every existing ERT. In addition to the Orphan drug period they need to develop enzymes that are at least as good (just ask John Crowley or Henri Termir about the difficulties of doing so in Pompe). And now companies are trying to improve on the ERT's. Genzyme has an oral Gaucher program, Amicus is co-administering their chaperone with ERT, Biomarin is attempting approach to improve the binding, I also think PTC has a technology that may be applicable to some subsets or LSD (but not too familiar with them).
I am most intrigued by PLX's oral program but its early. Their cheap/high capacity technology could be a plus too though to me that isn't enough by itself unless their goal is to be a high end "generics" play.
) but I think most (perhaps all) ERT's in general have some degree of antibody formation. If they are neutralizing it will limit the potency of the ERT sometimes dosing can help get around it in some patients. A couple things for long term PLX investors to keep in mind. Assuming the manufacturing issues are minor they can't just compete with every existing ERT. In addition to the Orphan drug period they need to develop enzymes that are at least as good (just ask John Crowley or Henri Termir about the difficulties of doing so in Pompe). And now companies are trying to improve on the ERT's. Genzyme has an oral Gaucher program, Amicus is co-administering their chaperone with ERT, Biomarin is attempting approach to improve the binding, I also think PTC has a technology that may be applicable to some subsets or LSD (but not too familiar with them).
I am most intrigued by PLX's oral program but its early. Their cheap/high capacity technology could be a plus too though to me that isn't enough by itself unless their goal is to be a high end "generics" play.
Join the InvestorsHub Community
Register for free to join our community of investors and share your ideas. You will also get access to streaming quotes, interactive charts, trades, portfolio, live options flow and more tools.
