Replies to post #195199 on Biotech Values

[mcbio]

QURE -

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

Don't think so. Just the PR and also a CC today. I listened to the CC and there was some reference to there being an immune reaction to the body producing the enzyme (since it wasn't being produced before at all in the patients and so recognized as new to the immune system). There was a question from a caller about need for patients to be on immunosuppressive treatment for life. Didn't seem to be a definitive answer to this from the company if I recall but maybe that's not a big deal given unmet need and particularly if the therapy ultimately does prove to have meaningful benefit.

I'm still unclear on magnitude of treatment effect but I guess just too early to say. While the company did caution the small N they did note that the oldest patient (I think 4 or 5 years old) was still experiencing some improvement when natural progression would normally predict a sharp decline at that age.