I noted a couple things on twitter. It seems like the super responders (1 in particular) had the Factor level drop to normal (briefly) and is now at near normal. Considering his level was over 300 at one point that is rather substantial. Its above my paygrade to speculate but I would be concerned if its a sign of loss of expression or two could it be the body is somehow self regulating and adjusting? Also if so the lower dose being tested in the second P3 would seem less necessary.
The n's are small and some fluctuation is normal... with that caveat though the median level is also dropping (though slightly) without individual data hard to speculate but durability has been one of my concerns.
Those things being said the results are quite remarkable! It seems ONCE (Hemophilia B) gets all the spotlight in the Hemophilia GT space but BMRN is actually putting the vast majority of patients at normal Factor levels with minimal AE's (1 SAE was because of a patient needing knee replacement and the other resolved with Tylenol).
I also wonder about pricing if Roche is charging ~500k one would think 4-5x that if durability remains would be cheap for BMRN's GT.
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