Replies to post #195167 on Biotech Values

[lgonber]

The stock whent for +20% in pre market to -7% now. Another weird action

[mcbio]

QURE -

In all four patients, researchers verified the restoration of catalytical activity of the NaGlu protein in the cerebrospinal fluid (CSF) from 0% at baseline up to 14-17% of normal at 3 months with persistent effect at 12 months. [The vagueness of the “up to” phrase will presumably be addressed on the CC.]

Haven't listened to the CC yet, but I'm even more curious more generally about the significance of even 14-17% at best. How does one translate that in to ultimate actual patient benefit? Did QURE give prior guidance on a figure they wanted to see? And again, any hard numbers or support for what this means in terms of ultimate patient benefit? E.g., is 25% or 50% restoration of catalytic activity of the protein needed for meaningful benefit? Also, what does the "persistent effect at 12 months" entail? Did the percentage of restoration of catalytic activity of the protein at 3 months generally stay the same at 12 months or did it drop off some?

The fact that all treated Sanfilippo B subjects continued to gain skills throughout the study is extremely encouraging.

This again seems pretty vague to me. What exactly does "gain skills" entail?

[poorgradstudent]

QURE:

I've been away. Is there a data presentation for this?

TIA.