Replies to post #115538 on Biotech Values

[HattieTheWitch]

Yes, that's a good point. I also liked during Q&A where one caller referred to Uplyso's hypersensitivity rate as 6% and management was quick to correctly note that it was actually 7%.

Am I correct to think that the lower the hypersensitivity level, the better?

[ghmm]

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.

[genisi]

One patient developed non-neutralizing IgG antibodies to taliglucerase at the end of the study

I believe data were from 25 patients, so the rate of antibodies to uplyso is still lower than that of Cerezyme. See also #msg-56216621. Last point, the non-neutralizing antibodies to Cerezyme have no effect on its safety or efficacy.

[jq1234]

"One patient developed non-neutralizing IgG antibodies to taliglucerase at the end of the study." I don't know exactly what this means and if this is something to potentially be concerned about. What are your thoughts on this?

That's to be expected when foreign enzyme is injected. The expected rate of developing IgG antibody from Cerezyme is 15%, roughly half of those patients had hypersensitivity symptom.

Here is Cerezyme safety information:

http://www.cerezyme.com/healthcare/safety/cz_hc_safety.asp