Biotech Values

[ghmm]

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

If they show [only] 10-12% that would be a significant achievement and great progress for a Gene Therapy in Hemophilia as that would like eliminate the need for prophalactic transfusion and the therapy should be approvable. However, I certainly would not expect them to get the lion share of the market if Spark continues to show ~30% expression. There will be a market though for UniQure since:
1) There is less natural anti-AAV5 antibody in general population (UniQure's) then there is for AAV8 (Sparks).
2) Spark has only reported data on 4 patients. GT is still VERY early we don't know about long term response, why some patients don't respond to the same level as others, etc.
3) If retreatment becomes necessary Qure may also have opportunity to capture more patients.
A key point I think is Uniqure has 100% rights in US (partnered in Europe) and an EV value of ~40M, Spark (granted its not even their lead indication) is partnered with Pfizer (don't know % off hand) and has a market cap of over 1.5B.

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).

To their credit Sparks dose is considerably less than UniQure's probably in part explains the lack of ALT elevation. Perhaps this is true I really don't know. It should be pointed out that BMRN uses an insect viral line too and had arguably much higher expression (though it is Hemophilia A not B). BMRN also uses a much higher dose than Spark (and even higher than UniQure's high dose). If the ALT elevations are manageable with steroids and no other serious AE's occur while yes less would be better but how big a deal would it be for a (theoretically) once and done treatment as long as the factor expression is robust?