Replies to post #204084 on Biotech Values

[mcbio]

QURE -

1) The data to date in their Hemophilia B program has shown about 5% expression and some ALT elevations. Sparks has shown ~30% (21-42) and no ALT elevations which is perhaps why people are writing off QURE's Hemophilia B program. I find it interesting BMRN (which also uses AAV5) had more than a log increase in expression with a 3 fold increase in dose. Also BMRN's ALT elevations have been managed with prophylactic steroids (with apparently no loss of expression). The High dose in QURE's Hemophilia B program (4x the low dose) should have up to 6 months data by year end. The analysts notes I've seen have expectations in the 10-12% expression range. I would not be shocked for it to be substantially above that.

If QURE only shows expression in 10-12% range, is it safe to assume that will not be competitive with ONCE showing ~30% expression? How close to 30% does QURE need to be to be competitive?

5) Though they use a much higher dose than Spark they do their own manufacturing with 2 facilities (insect viral line). They've said they have enough capacity to treat all the CV patients from one year of production (one reason sited for BMS choosing them).

ONCE really touts their mammalian cell line approach and I think they believe that is what may be leading them to show initial stronger data than QURE (vs. QURE insect line approach).

[DewDiligence]

QURE receives Nasdaq deficiency letter wrt BoD composition:

http://www.uniqure.com/news/328/182/uniQure-Receives-NASDAQ-Deficiency-Letter-Related-to-Board-Composition.html

[DewDiligence]

QURE CEO resigns immediately for personal/family reasons:

http://www.uniqure.com/news/329/182/uniQure-Announces-Management-and-Board-Changes.html

[ghmm]

UniQure (QURE)

Its been a while since I posted about why I was buying and the price is lower than when I first started buying. Still the stock has been doing quite well of late. Even before DMTX reported (questionable) data in their program. I think it is because the street is starting to realize there is a place for their Hemophilia GT.

While the High dose Hemophilia B data was not what I had hoped (in terms of FIX) the data appears to certainly be more than adequate in terms of providing efficacy and durability with no major safety concern to date. To me the stock could go much higher on just the Hemophilia program the short term key will be how their meeting with FDA goes and what type of guidance is given. If FDA signs off on a reasonable duration and size the company should do quite well even if they only get 10-20% (and it could be much higher considering what I posted earlier) of the GT hemophilia B market this could be generating a few hundred million annually in the not-to-distant future.