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).